July 28, 2021
Deep Genomics Raises $180 Million to Advance Programmable RNA Therapeutics
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July 28, 2021
Aristea Enters into Collaboration with Arena in Neutrophil-Mediated Diseases and Raises $63 Million
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July 28, 2021
Ring Raises $117 Million to Advance Vector Platform to Unlock Full Potential of Gene Therapy
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July 28, 2021
Bluebird Bio Forms Strategic Alliance with Resilience to Develop Next Generation Cell Therapies
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July 28, 2021
FDA Grants Fast Track Designation to Apic Bio’s APB-102 for the Treatment of Patients with SOD1 ALS
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July 28, 2021
FDA Grants Rare Pediatric Disease Designation to Lexeo Therapeutics CLN2 Batten Disease Therapy
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July 27, 2021
Study Links Autism to New Set of Rare Gene Variants
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July 27, 2021
RARE-X Appoints Charlene Son Rigby as Chief Executive Officer
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July 27, 2021
FDA Grants Rare Pediatric Disease Designation to Day One for Treatment of Pediatric Low-Grade Glioma
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July 27, 2021
Abeona Reports New Positive Data from Study of Gene Therapy for Sanfilippo Syndrome
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July 27, 2021
RareStone and Tencent Collaborate on Service Ecosystem Focused on Rare Disease Patients in China
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July 27, 2021
Australian Government Provides $3.3 Million to Support Rare Disease Patients
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July 26, 2021
European Commission Approves Rhythm’s Drug for Rare Obesity Disorders
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July 26, 2021
Denali Reports Positive Interim Data from Phase 1/2 Hunter Syndrome Trial
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July 23, 2021
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
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July 22, 2021
Rare Leader: Cyndi Frank, Co-President and Co-Founder, Gaucher Community Alliance
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July 22, 2021
Hemab Raises $55 Million to Advance Therapeutics for Rare Bleeding Disorders
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July 22, 2021
NHS England Unveils New Innovative Medicines Fund to Fast-Track Promising New Drugs
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July 22, 2021
Spark Says Ongoing Phase 1/2 Hemophilia A Gene Therapy Data Suggests Durability
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July 22, 2021
Researchers Find Immune Component to Rare Neurodegenerative Disease
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July 22, 2021
FDA Approves Octapharma’s Therapy for Adult Dermatomyositis
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July 22, 2021
Parexel Partners with Rare Disease Foundation in China
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July 21, 2021
Amylyx Raises $135 Million to Support Development and Potential Launch of ALS Treatment
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July 21, 2021
European Commission Approves Bluebird Bio’s Gene Therapy for CALD
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July 21, 2021
FDA Approves Albireo’s Bylvay as First Drug for PFIC
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July 21, 2021
Lexeo Acquires Stelios to Expand Pipeline into Rare Cardiac Gene Therapies
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July 21, 2021
Horama Raises $39 Million to Develop its Gene Therapy Pipeline and Changes Name to Coave Therapeutics
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July 21, 2021
FDA Grants Breakthrough Therapy Designation to Genentech for Therapy to Treat Myelodysplastic Syndromes
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July 20, 2021
UW Opens Clinic for Patients with Undiagnosed Genetic Diseases
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July 20, 2021
Netherland Fines Rare Disease Drugmaker for Excessive Price
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July 19, 2021
Cytokinetics Reports Positive Results from Mid-State Trial of Hypertrophic Cardiomyopathy
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July 19, 2021
GentiBio and Forge Biologics Form GMP Manufacturing Partnership
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July 16, 2021
How a Small and Young Foundation Catalyzed Research into a Rare Disease
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July 16, 2021
NIH Funds New Effort to Discover Genetic Causes of Single-Gene Disorders T
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July 15, 2021
Rare Leader: Chelsey McCarthy, Executive Director, DDX3X Foundation
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July 15, 2021
Shape Therapeutics Raises $112 Million to Advance RNA Technology Platforms
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July 15, 2021
Rady Children’s Institute for Genomic Medicine Enters Research Collaboration with Takeda
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July 14, 2021
AstraZeneca Clears Final Hurdle for Acquisition of Alexion
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July 14, 2021
FDA Grants Breakthrough Therapy Designation to Alkeus Stargardt Disease Therapy
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July 13, 2021
Prime Medicine Launches with $315 Million to Deliver on the Promise of Prime Editing
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July 13, 2021
Rare Disease Drug Developers Bring in $13.7 Billion in New Capital in First Half of 2021
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July 13, 2021
Researchers Use Prenatal Editing in Animal Model to Correct Lysosomal Storage Disease
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July 13, 2021
Novo Nordisk Acquires Prothena’s Antibody Targeting ATTR Amyloidosis  
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July 13, 2021
Poxel Shifts Focus to Rare Metabolic Diseases
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July 13, 2021
Gene Therapy Reverses Neurological Deficiencies in AADC
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July 9, 2021
Overcoming the Limitations of Conventional Cell Engineering
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July 9, 2021
Browsable Resource Linking Rare Protein-Coding Genetic Variants to Human Health and Disease
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July 9, 2021
FDA Places Clinical Hold on Sigilon’s Phase 1/2 Study in Hemophilia A
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July 9, 2021
BlueBird Bio Resumes Marketing TDT Gene Therapy in Europe
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July 8, 2021
Rare Leader: Deanna Fournier, Executive Director, Histiocytosis Association
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July 8, 2021
BridgeBio Enters Collaborations with Three Academic Research Institutions
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July 8, 2021
Lilly Enters Collaboration with Verge to Use Its AI to Discover and Develop New Treatments for ALS
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July 8, 2021
FDA Grants Lysogene Fast Track Designation for GM1 Gangliosidosis Gene Therapy
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July 8, 2021
CMT Research Foundation Enters Partnership with University of Illinois Chicago
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July 7, 2021
Patient Groups Begin Using RARE-X’s Data Collection Platform
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July 7, 2021
ChemoCentryx Addresses FDA Advisory Panel Questions in Amendment to Filing on Vasculitis Drug
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July 6, 2021
EC Clears AstraZeneca’s Acquisition of Alexion
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July 6, 2021
Orchard and Pharming Collaborate on Gene Therapy for HAE
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July 6, 2021
Eureka and MSK License Novel Multiple Myeloma Target Domain to Sanofi
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July 6, 2021
FDA Grants Priority Review to Levo for Prader-Willi Syndrome Therapy
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July 6, 2021
Abbisko and Sperogenix Partner to Develop ABSK021 for ALS, Other Rare CNS Diseases in Greater China
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July 2, 2021
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
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July 2, 2021
Arrowhead Pauses RNAi Study in Cystic Fibrosis After Tox Study Signals
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July 2, 2021
GSK Enters into Global Collaboration with Alector to Develop Antibodies for Neurodegenerative Diseases
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July 1, 2021
Rare Leader: Diane Powell, CEO, The Hypersomnia Foundation
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July 1, 2021
Apellis and Beam Collaborate to Apply Base Editing for Complement-Driven Diseases Drug Discovery
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July 1, 2021
FDA Approves Jazz’s Rylaze as Component of Treatment Regimen for Most Common Childhood Cancer
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June 30, 2021
Intellia Raises $600 Million to Advance Gene Editing Therapeutic Pipeline
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June 30, 2021
Sofinnova Invests in Three New Rare Disease Gene Therapy Startups
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June 30, 2021
NHS Creates First-of-Its-Kind Rare Disease Network in UK
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June 30, 2021
FDA Grants Allogene Fast Track Designation for Multiple Myeloma Therapy
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June 30, 2021
GenSight Reports Phase 3 Study Misses Endpoint, but Improves Vision in Patients with Degenerative Eye Disease
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June 30, 2021
Aerovate Raises $121.5 Million in Upsized IPO to Advance PAH Therapy
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June 30, 2021
FDA Grants Lexeo Therapeutics Rare Pediatric Disease Designation
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June 29, 2021
Orphazyme Lays Off Two-Thirds of Staff After FDA Setback
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June 29, 2021
Natural History Study Informs Potent Lifesaving Update to Standard of Care for PMM2-CDG Patients
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June 29, 2021
Pediatricians Find Cause of Muscle Breakdown in Rare Disease
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June 28, 2021
Intellia and Regeneron Report First-Ever Data Supporting In Vivo CRISPR Editing in ATTR Patients
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June 28, 2021
EU Approves Roche’s Enspryng as First At-home Subcutaneous Treatment for NMOSD
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June 28, 2021
FDA Grants Fast Track Designation to Three Experimental Rare Disease Therapies
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June 25, 2021
Forging Gene Therapy Capacity and a Pipeline at the Same Time
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June 25, 2021
Graphite Bio Raises $238 Million in IPO to Advance Precision Gene Editing Pipeline
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June 25, 2021
Senators Introduce Legislation to Help Diagnose Children with Rare Diseases
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June 25, 2021
International Study of Rare Childhood Cancer Finds Genetic Clues, Potential for Tailored Therapy
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June 24, 2021
Rare Leader: Kevin Mead, Executive Director of the International Pemphigus & Pemphigoid Foundation
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June 24, 2021
AbbVie Exercises Right to Acquire TeneoOne and Lead Asset Targeting Multiple Myeloma
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June 24, 2021
4D Molecular Reports Trial Data for Rare Eye Disorders and Termination of Roche Agreement
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June 24, 2021
Pacific Biosciences and Rady Children’s Collaborate on WGS Study of Rare Diseases
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June 24, 2021
Corbus Reports Lenabasum Fails to Meet Primary Endpoint in Late-Stage Trial for Dermatomyositis
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June 24, 2021
FDA Approves Santen’s Verkazia to Treat Vernal Keratoconjunctivitis in Children and Adults
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June 23, 2021
Glycomine Raises $35 Million to Advance Novel Treatment for Rare Glycosylation Disease
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June 23, 2021
UniQure Reports Positive 52-Week Data from Pivotal Trial of Hemophilia B Gene Therapy
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June 22, 2021
MIND Institute Gets $1.25 Million Grant for SYNGAP1 Syndrome
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June 22, 2021
Protagonist Raises $132 Million to Advance Rare Blood Cancer Therapy
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June 22, 2021
Neurona Raises $41.5 Million to Advance Cell Therapies for Epilepsy and Neurological Disorders
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June 22, 2021
Neurelis Acquires Rights to Portfolio of Compounds Targeting Rare CNS Disorder
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June 22, 2021
PerkinElmer to Acquire Viral Vector Gene Delivery Specialist Sirion Biotech
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June 22, 2021
Jasper and Aruvant Collaborate on Treatment for Sickle Cell Disease
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June 22, 2021
New UAE Center will Diagnose and Treat Patients with Genetic Diseases
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June 21, 2021
EC Expands Approval of Sanofi’s Oral MS Therapy to Include Children and Adolescents
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June 21, 2021
Agios Seek Approval for Mitapivat for Rare Inherited Blood Disorder
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June 18, 2021
Using A Natural DNA Repair Process to Improve Genetic Medicines
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June 18, 2021
FDA Says It Won’t Approve Orphazyme’s NPC Therapy without Additional Data
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June 18, 2021
FDA Grant Applied Therapeutics Fast Track Designation for Experimental Galactosemia Therapy
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June 18, 2021
FDA Grants United Therapeutics Priority Review for PAH Drug
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June 18, 2021
Perlara Enters Collaboration with Vivan for Multiple Rare Diseases
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June 17, 2021
Rare Leader: Fallon Schultz, President and Founder, International FPIES Association
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June 17, 2021
Gene Editing Therapeutics Developer Verve Completes $266.7 Million IPO
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June 17, 2021
Horizon Therapeutics Buy Manufacturing Facility to Support Its Growth
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June 16, 2021
FDA Expands Approval of Blueprint Medicines’ Ayvakit to Include Rare Blood Disorder
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June 16, 2021
CAMP4 Raises $45 Million for Programmable Therapeutics to Upregulate Genes
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June 16, 2021
n-Lorem Foundation Forms Partnership with Manufacturer Oligonucleotide Nitto Avecia
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June 15, 2021
Researchers Use Base Editing to Turn Pathogenic Gene Benign in Mice with SCD
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June 15, 2021
Biogen Choroideremia Gene Therapy Fails in Phase 3 Study
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June 15, 2021
CRISPR Therapeutics and Capsida Enter Strategic Collaboration to Develop Gene-Edited Therapies for ALS, FA
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June 15, 2021
Novartis Reports Positive Results for PNH Therapy
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June 15, 2021
Nanoparticle Therapy Shows Early Promise at Preventing a Rare, Fatal Newborn Lung Disease
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June 14, 2021
Cure Rare Disease Enters Partnership with Curi Bio for DMD Gene Therapy
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June 14, 2021
Study Shows Evrysdi Improved Motor Function in Pre-Symptomatic Babies
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June 14, 2021
CDMO Yposkesi Launches Construction of Advanced Therapy Facility Near Paris
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June 14, 2021
FDA Grants Jasper Therapeutics Rare Pediatric Disease Designation for Conditioning Treatment Prior to Stem Cell Transplant
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June 11, 2021
Empowering Rare Disease Patients with their Own Health Records
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June 11, 2021
A Mother’s Prosecution Alarms Rare Patient Advocates
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June 11, 2021
Sanofi Presents Positive New Data for Sutimlimab for the Treatment of CAD
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June 11, 2021
Vertex Achieves Primary Endpoint in AATD Study but Drops Program Due to Insufficient Clinical Benefit
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June 10, 2021
Rare Leader: Kim Hollander, Executive Director, Oxalosis & Hyperoxaluria Foundation
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June 10, 2021
Codexis and Takeda Expand Collaboration to Additional Gene Therapy for a Rare Genetic Disorder
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June 10, 2021
Kurome Closes $15 Million Financing to Develop Targeted Kinase Inhibitors to Treat Rare Blood Cancers
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June 10, 2021
New Data Point to Potential Long-Term Efficacy of Biogen’s Spinraza for SMA 
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June 9, 2021
Rare Disease Therapeutics Developers Continued to Rake in Cash in May
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June 9, 2021
Alcyone Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders
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June 9, 2021
FDA Approves Trikafta in Children with Cystic Fibrosis Ages 6 through 11 with Certain Mutations
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June 8, 2021
CyGenica Secures Seed Funding to Accelerate Cancer and Rare Genetic Disease Drug Delivery
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June 8, 2021
FDA Approves Alexion’s Ultomiris for Children and Adolescents with PNH
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June 8, 2021
Rare Disease Company Coalition Names Executive Committee
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June 7, 2021
FDA Lifts Clinical Hold on Bluebird Bio’s Sickle Cell Disease and β-Thalassemia Studies
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June 7, 2021
FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder
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June 7, 2021
Novartis Reports Positive Interim Analysis from Phase 2 Study in Rare Kidney Disease
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June 4, 2021
Targeting CNS Diseases with Gene Therapies
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June 4, 2021
Nanoscope Gene Therapy Restores Vision in 11 Patients Blinded by Retinitis Pigmentosa
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June 3, 2021
Rare Leader: Krista Vasi, Executive Director, Usher Syndrome Coalition
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June 3, 2021
Auris Medical Reboots to RNA Therapeutics with Acquisition of Trasir Therapeutics
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June 3, 2021
Amyl Raises $22 million to Develop Novel Therapies for Amyloidosis
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June 3, 2021
Stablix Therapeutics Launches with $63 Million to Target Protein Stabilization
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June 3, 2021
AavantiBio and Resilience Form Strategic Collaboration for Gene Therapy Development and Manufacturing
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June 3, 2021
FDA Grants Protagonist Breakthrough Therapy Designation for Rusfertide in PV
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June 3, 2021
FDA Grants Priority Review to Amryt’s EB Drug
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June 2, 2021
MorphoSys to Acquire Rare Cancer Drug Developer Constellation Pharma for $1.7 Billion
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June 2, 2021
Transine $12.8 Million Seed Funding to Advance Novel Class of Therapeutic RNAs
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June 2, 2021
Santhera and ReveraGen Report Positive Topline Results in Pivotal DMD Study
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June 2, 2021
Barth Syndrome Foundation and American Heart Association Collaborate to Advance Research<br><br><em>Rare Daily Staff</em>
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June 2, 2021
Treatabolome Project Seeks to Shorten Diagnosis-to-Treatment Time for Rare Disease Patients
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June 2, 2021
FDA Grants Breakthrough Therapy Designation to Janssen Off-the-Shelf Cell Therapy for Multiple Myeloma
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June 1, 2021
FDA Approves QED and Helsinn’s Truseltiq to Treat Patients with Cholangiocarcinoma
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June 1, 2021
Scientists Discover a New Genetic Form of ALS in Children
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June 1, 2021
Solve-RD Collaboration Makes Case for Data Sharing and Periodic Reanalysis
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June 1, 2021
FDA Grants Fast Track Designation to BridgeBio’s Encaleret for the Treatment of ADH1
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June 1, 2021
Genomenon and Nostos Genomics Partner to Accelerate Rare Genetic Disease Diagnoses
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May 27, 2021
A New Therapy Offers A Different Approach to Inhibiting the Complement System
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May 27, 2021
Rare Leader: Kyle Dempsey, President, Superficial Siderosis Research Alliance
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May 27, 2021
Eloxx and Metagenomi Receive Funding Awards from Cystic Fibrosis Foundation
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May 27, 2021
Day One Raises $160 Million in IPO to Advance Genetically Defined Cancer Therapies
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May 26, 2021
Larimar Says $95 Million Placement Cancelled After FDA Places Clinical Hold on Lead Program
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May 26, 2021
Penn’s Gene Therapy Program Becomes Anchor Tenant of Discovery Labs
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May 26, 2021
Rgenta Therapeutics Completes Seed Financing, Raising $38 Million for RNA-Targeted Pipeline
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May 26, 2021
FDA Asks Travere for Additional Data Ahead of Marketing Application for FSGS Therapy
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May 26, 2021
BlueRock and Senti Bio Collaborate to Develop Engineered Cell Therapies for Regenerative Medicine
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May 25, 2021
Apellis and Sobi Report Positive Results from Empaveli Study in Treatment Naïve Patients with PNH
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May 25, 2021
FDA Grants Scholar Rock Fast Track Designation for SMA Therapy
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May 25, 2021
FDA Approves First Therapy for Rare Form of NSCLC with Companion Diagnostic
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May 24, 2021
Bipartisan Legislation Introduced to Advance Biomedical Research Stalled by Pandemic
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May 24, 2021
CHMP Recommends Approval of Therapies to Treat the Rare Disorders PFIC and POMC
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May 21, 2021
Bringing Aberrant Proteins Back into the Fold
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May 21, 2021
Haya Therapeutics Raises $20 Million Seed Financing for RNA-based Fibrosis Therapies
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May 21, 2021
Mallinckrodt Reaches Agreement to Sell Experimental NPC-1 Drug Adrabetadex to Mandos
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May 21, 2021
European Regulators Give Bluebird Bio Positive Opinion for CALD Gene Therapy
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May 21, 2021
Biogen and Ginkgo Bioworks Enter Collaboration to Create Novel Gene Therapy Manufacturing Platform
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May 21, 2021
European Medicines Agency Grants PRIME Designation to Larimar’s Treatment for Friedreich’s Ataxia
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May 20, 2021
Rare Leader: Trish Flanagan, co-founder and president, YBRP
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May 20, 2021
Adaptimmune Reports Positive Initial Data from Mid-Stage Trial in Rare Cancer
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May 19, 2021
Investors Pile $2.2 Billion into Rare Disease Therapeutics Developers in April
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May 19, 2021
Eloxx Raises $52 Million in Public Offering to Advance Rare Disease Pipeline
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May 19, 2021
Ciitizen and RARE-X Form Collaboration
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May 18, 2021
G2 Bio Companies Launch with $200 Million to Develop Transformative Genetic-Based Therapies
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May 18, 2021
Gamma Biosciences Acquires Controlling Stake in Mirus Bio
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May 18, 2021
Vedere Bio II Launches with $77 Million to Develop Next Generation Ocular Gene Therapies
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May 18, 2021
Stealth Bio Raises $30 Million to Advance Elamipretide Clinical Trials
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May 18, 2021
Interius Raises $76 Million for Cell and Gene Therapy Platform
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May 17, 2021
Charles River to Acquire Gene Therapy CDMO Vigene Biosciences
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May 17, 2021
FDA Approves Apellis’ Empaveli for Adults with Paroxysmal Nocturnal Hemoglobinuria
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May 14, 2021
Praxis Precision Medicines Raises $91.3 Million in Public Offering
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May 14, 2021
PerkinElmer to Expand Cell and Gene Therapy and Biologics Manufacturing with $260 Million Nexcelom Acquisition
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May 14, 2021
Vera Therapeutics Raises $48 Million in IPO
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May 14, 2021
Biogen XLRP Gene Therapy Fails Late-Stage Study
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May 14, 2021
FDA Grants BridgeBio Fast Track Designation for CAH Gene Therapy
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May 13, 2021
Rare Leader: Julie Breneiser, Executive Director, Gorlin Syndrome Alliance
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May 13, 2021
SpliSense Secures $28.5 Million in Series B Financing Including Backing from CFF
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May 13, 2021
Flare Launches with $82 Million to Advance Novel Drug Discovery Approach for Transcription Factors
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May 13, 2021
Magenta Raises $86.4 Million to Advance Novel Antibody-Drug Conjugate Conditioning Platform
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May 13, 2021
Rare Disease Drug Developers Form Coalition to Address Challenges
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May 13, 2021
CRO Emmes Acquires UK-Based Orphan Reach
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May 12, 2021
MD Anderson and Broad Institute Launch Translational Research Platform Focused on Rare Cancers
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May 12, 2021
Huma Raises $200 Million to Scale Digital Health Platform for Better Care and Research
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May 12, 2021
Gennao Bio Closes $40 Million in Financing to Develop Pipeline of Targeted Nucleic Acid Therapeutics
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May 12, 2021
FDA Grants Fulcrum Fast Track for FSHD Drug
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May 12, 2021
Xontogeny Seeds NephroDI to Develop a Treatment for Nephrogenic Diabetes Insipidus
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May 11, 2021
Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency
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May 11, 2021
Biogen Enters Collaboration with Capsigen for AAV Capsids for CNS and Neuromuscular Disorders
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May 11, 2021
Mutations Cause Rare Genetic Disease in Children
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May 11, 2021
FDA Halts Rocket Pharma Trial of Experimental Gene Therapy for Danon Disease
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May 10, 2021
Laronde Unveiled to Advance New Class of Programmable Medicines
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May 10, 2021
Ionis Stops Development of Inhaled Antisense Therapy for CF
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May 10, 2021
FDA Grants Fast-Track Designation to Smart Immune’s T Cell Immunity Enhancement Product
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May 7, 2021
Putting the Patient at the Center of Rare Disease Clinical Trials
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May 7, 2021
Talaris Raises $150 Million in IPO to Advance Cell Therapy Pipeline
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May 7, 2021
Science 37 to Become Publicly Listed via Merger with SPAC
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May 7, 2021
ChemoCentryx Continues Slide on Divided FDA Advisory Committee Votes
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May 7, 2021
Orphazyme Drug Fails in Pivotal Study
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May 6, 2021
Rare Leader: Tiffany House, President Acid Maltase Deficiency Association
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May 6, 2021
Dyno Therapeutics Raises $100 Million to Accelerate AI‑powered Gene Therapy Platform
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May 6, 2021
TwinStrand Raises $50 Million to Grow Market for Duplex Sequencing Technology
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May 5, 2021
Amryt Acquires Chiasma, Strengthening Its Rare and Orphan Disease Portfolio
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May 5, 2021
Centogene Discovers Six New Rare Diseases
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May 5, 2021
ChemoCentryx Shares Crash After FDA Questions Data Supporting ANCA Vasculitis Therapy
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May 5, 2021
Study Finds People with Rare Diseases Often Feel Stigmatized
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May 4, 2021
Sarepta Reports High-Dose SRP-5051 Increases Dystrophin in Certain DMD Patients
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May 4, 2021
Mogrify Raises $17 Million to Advance Reprogrammed Cell Therapies  
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May 4, 2021
Passage Bio Partners with InformedDNA to Offer Genetic Counseling and Testing for FTD
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May 4, 2021
Acella Issues Voluntary Nationwide Recall of Certain Lots of Thyroid Tablets Due to Sub Potency
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May 3, 2021
Affinia Raises $110 Million to Advance Engineered AAV Gene Therapy Vector Platform
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May 3, 2021
Aceragen Launches with $35 Million and Experimental Therapy for Farber Disease
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May 3, 2021
With Accelerated Approval Off the Table, Avrobio Seeks Registration Trial for Fabry Disease Gene Therapy
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May 3, 2021
Alnylam Reports Positive Early Results of Phase 3 PH1 Study
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May 3, 2021
FDA Expands Approval of Chiesi’s Ferriprox for Transfusional Iron Overload due to Sickle Cell Disease
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April 30, 2021
Creating a Toolkit to Accelerate the Development of Gene Editing Therapies
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April 29, 2021
Rare Leader: Nancy Lazarus, Founder, Achalasia Awareness Organization
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April 29, 2021
Capsida Debuts with $140 Million to Advance Next Generation Gene Therapies
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April 29, 2021
Mirum Grants CANbridge Exclusive License to Maralixibat in Greater China for Rare Liver Diseases
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April 29, 2021
FDA Grants Fast Track to Akari Bullous Pemphigoid Therapy
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April 29, 2021
Astellas Takes $532 Million Charge Over Delay of Gene Therapy for XLMTM
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April 28, 2021
Allen Institute and BioMarin Collaborate to Develop Gene Therapies for Rare Brain Diseases
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April 28, 2021
FDA Tell Protalix and Chiesi It won’t Grant Accelerated Approval for Fabry Therapy
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April 27, 2021
LogicBio Enters Gene-Editing Collaborations with CANBridge and Daiichi Sankyo
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April 27, 2021
AllStripes Collaborates with NIH’s NCATS to Launch Effort in Rare Disease Diagnosis
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April 27, 2021
FDA Removes Clinical Hold on Voyager, Clears IND for Huntington’s Gene Therapy
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April 27, 2021
Biogen Will Provide Early Access to Experimental ALS Drug
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April 27, 2021
EMA Grants PRIME Designation to Vertex and CRISPR’s Therapy for Transfusion-Dependent Beta Thalassemia
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April 27, 2021
AavantiBio Partners with Catalent to Advance Gene Therapies for Rare Genetic Diseases
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April 27, 2021
FDA Issues Guidance on Individualized ASOs for Ultra-Rare Conditions
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April 26, 2021
FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
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April 26, 2021
FDA Approves ADC Therapeutics’ Zynlonta to Treat Rare Cancer
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April 23, 2021
PTC Looks to Advanced Therapies
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April 23, 2021
CDISC and NORD to Develop Data Standards for Rare Disease
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April 22, 2021
Rare Leader: Heidi Edwards, President and Founder, Sister’s Hope Foundation
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April 22, 2021
Vertex and Obsidian Enter into Collaboration to Discover Novel Therapies that Regulate Gene Editing
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April 22, 2021
ITM raises $109 Million to Advance Radiopharmaceutical Precision Oncology Pipeline
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April 21, 2021
Code Biotherapeutics Launches with $10 Million to Develop Therapies for Debilitating Genetic Diseases
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April 21, 2021
EdiGene Raises $62 Million to Advance and Scale Up Clinical Translation of Gene Editing Technologies
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April 21, 2021
Beam Publishes Data Demonstrating Ability to Rationally Design Base Editors for Precise Editing
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April 21, 2021
Bluebird Withdraws Zynteglo from German Market, Cuts Workforce
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April 21, 2021
EC Grants Approval to GW Pharmaceuticals’ Epidyolex for TSC-Associated Seizures
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April 21, 2021
FDA Grants RMAT Designation to Allogene’s Cell Therapy for Multiple Myeloma
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April 20, 2021
The Making of a Gene Therapy for an Ultra-Rare Disease
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April 20, 2021
FDA Puts Clinical Hold on KalVista’s HAE Therapy
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April 20, 2021
Citrine and Sinopharm Enter Strategic Partnership to Broaden Access to Rare Disease Drugs in China
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April 20, 2021
Vertex and CRISPR Amend Sickle Cell Disease and Beta Thalassemia Collaboration
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April 20, 2021
IndoUSrare and Rare-X Conduct Feasibility Study for a Patient-Owned Health Data Collection Program for India
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April 20, 2021
Castle Creek Biosciences Appoints Matthew Gantz as President and CEO
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April 19, 2021
Rezolute Enters $30 Million Debt Agreement to Advance Rare and Metabolic Disease Pipeline
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April 19, 2021
Study Finds Positive Impact of Fintepla on Dravet Patients and Caregivers
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April 19, 2021
Biogen Reports New Spinraza Data in SMA
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April 16, 2021
Study Identifies Key Target in Treatment-Resistant Hemophilia A
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April 16, 2021
Recursion Raises $436 Million in Upsized IPO to Advance Rare Disease Pipeline
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April 15, 2021
Lauren Dunlap, Co-Founder and Executive Director, AAIDA
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April 15, 2021
Medable Raises $78 Million to Decentralize Clinical Trials
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April 15, 2021
Alchemab Raises $82 Million to Advance Antibody Therapeutics in Neurodegeneration and Cancer
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April 15, 2021
Ciitizen Partners with STXBP1 Foundation to Accelerate Patient Outcomes  
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April 15, 2021
DOJ Subpoenas Documents from Alnylam Over Marketing of Onpattro
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April 15, 2021
FDA Grants Rare Pediatric Disease Designation to FibroGen Treatment for DMD
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April 15, 2021
New Evrysdi Data Shows Improvement in Motor Function Over Time for SMA Patients
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April 14, 2021
Investment in Rare Disease Drug Development Continues to Surge
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April 14, 2021
Gene Therapy Shows Promise in Treating Rare Eye Disease in Mice
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April 14, 2021
Huma Enters Partnership with RARE-X to Accelerate Rare Disease Research
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April 14, 2021
FDA Grants Rare Pediatric Disease Designation to Therapies from Moleculin and Akuous
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April 13, 2021
Senti Bio and Spark Collaborate to Develop Next-Generation Precision Gene Therapies
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April 13, 2021
BridgeBio Pharma Enters Seven Collaborations with University Partners
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April 13, 2021
Theseus Debuts with $100 Million Series B to for Targeted Treatment-Resistant Cancer Mutations
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April 13, 2021
Arcellx Raises $115 Million to Advance Pipeline of Adaptive and Controllable Cell Therapies
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April 13, 2021
Jaguar Gene Therapy Closes $139 Million Funding to Advance Preclinical Rare Disease Pipeline
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April 13, 2021
ChemGenes to Provide n-Lorem Support for ASO Manufacturing
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April 12, 2021
Taysha Acquires Global Rights to Experimental Gene Therapy for Rare Neurodegenerative Disease
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April 12, 2021
Aytu Licenses Late-Stage Pediatric Onset Rare Disease Asset from Rumpus Therapeutics
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April 12, 2021
Mirum Signs Option to License Vivet’s Experimental Gene Therapy Programs for PFIC
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April 12, 2021
Sanofi Acquires Tidal Therapeutics with Its Innovative mRNA-based Research Platform
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April 12, 2021
FDA Grants FibroGen Fast Track Designation for DMD Therapy
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April 9, 2021
Everything’s up to Date in Kansas City, at Least When It Comes to Genomics
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April 9, 2021
Two Rare Disease Biotechs Go Public, Raising Combined $222 Million in IPOs
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April 8, 2021
ValenzaBio Raises $70 million in Venture Round
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April 8, 2021
Three Rare Disease Biotechs Raise Combined $675 Million in Public and Private Offerings
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April 8, 2021
Dicerna Sells Oxlumo Royalty Interest to Royalty Pharma for up to $240 Million
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April 8, 2021
Rare Leader: Melody Burchett, President, A Cure In Sight
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April 7, 2021
Ionis Signs Distribution Deal with Sobi and Cuts 70 Percent of Akcea Workforce
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April 7, 2021
U.K.’s University of Sheffield Establishes New Gene Therapy Innovation Center
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April 7, 2021
Metagenomi Extends Series A Financing to Advance Next-Gen Gene Editing Technology
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April 7, 2021
NIH Scientists Develop Breath Test for Rare Metabolic Condition
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April 7, 2021
Harvard Awards First Round of Rare Cancer Research Grants
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April 7, 2021
FDA Grants Breakthrough Therapy to Taiho’s Treatment for Advanced Cholangiocarcinoma
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April 6, 2021
Invitae Acquires Personalized Oncology Testing Company Generosity, Scores $1.2 B Financing
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April 6, 2021
Progenity and Ionis Pharmaceuticals Enter into Agreement for Oral Delivery of Antisense Therapies
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April 6, 2021
Scholar Rock Reports Positive Topline Results from Therapy for SMA Types 2 and 3
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April 5, 2021
Foundation for USP7 Related Diseases Makes Grant to Neurolentech
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April 5, 2021
n-Lorem Enters Partnership with Ultragenyx for Personalized Treatments for Patients with Ultra-Rare Conditions
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April 5, 2021
FDA Approves Praluent as Add-on Therapy for Patients with HoFH
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April 5, 2021
Takeda’s Rare Disease Drug Natpara Won’t Be Available for Another Year
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April 2, 2021
Choosing the Right Viral Vector for a Gene Therapy
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April 2, 2021
Penn Researchers Restore Vision in Blind Patient with Rare Genetic Condition with RNA Therapy
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April 1, 2021
Rare Leader: Marissa Penrod, Founder and CEO, Team Joseph
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April 1, 2021
Nitto to Invest $226 Million to Expand Oligonucleotide Manufacturing in Massachusetts
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April 1, 2021
Eloxx Acquires Zikani to Focus on Ribosomal RNA-Targeted Gene Therapy
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April 1, 2021
FDA Approves New Orphan Indications for Three Marketed Drugs
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April 1, 2021
Uplifting Athletes Issues $140,000 in Rare Disease Research Grants
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March 31, 2021
BridgeBio’s QED Therapeutics Enters Blockbuster Partnership with Helsinn for Infigratinib
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March 31, 2021
Scribe Raises $100 Million to Further Develop Its “CRISPR by Design” Platform
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March 31, 2021
Entrada Raises $116 Million to Advance Intracellular Biologics Pipeline into the Clinic
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March 31, 2021
Forma Reports Positive Data in SCD Trial
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March 30, 2021
Europe Approves Incyte’s Pemazyre for Rare Cancer
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March 30, 2021
Omega Raises $126 Million to Advance Epigenomic Programming Pipeline and Platform
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March 30, 2021
Wave Dumps Two Huntington’s Treatments after Failure to Show Benefit in Studies
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March 30, 2021
CZI Begins New Round of Funding for Rare Disease Organizations
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March 30, 2021
Imara Opens Applications for Real Impact Grants
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March 29, 2021
Report Finds Most Rare Disease Drugs Protected by Patents Rather than Exclusivity
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March 29, 2021
Ionis Reports Positive Topline Phase 2 Data of Antisense Treatment for HAE
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March 29, 2021
Cyclo NPC1 Phase 1/2 Trial Hits Primary Endpoint
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March 29, 2021
FDA Approves First Cell-Based Gene Therapy for Adult Patients with Multiple Myeloma
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March 29, 2021
Orphazyme’s Treatment for Rare Muscle Wasting Disease Fails in Late Stage Trial
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March 26, 2021
A Software Platform to Give Patients the Tools to Build Treatments for Rare Genetic Diseases
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March 26, 2021
Two Rare Disease Drug Developers Raise Combined $416 Million in IPOs
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March 26, 2021
Gyroscope Raises $148 Million to Advance AMD Gene Therapy
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March 26, 2021
FDA Expands Use of Myrbetriq to Include Rare Pediatric Indication
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March 26, 2021
FDA Approves Kiniksa’s Pericarditis Therapy Arcalyst
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March 25, 2021
Rare Leader: Lisa Bonebrake, Executive Director, Alport Syndrome Foundation
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March 25, 2021
Sanford Health Enters Partnership with Congenica to Provide Answers for Undiagnosed Rare Disease Patients
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March 24, 2021
Rare Disease Drug Developers’ Deals and IPOs Boost February Numbers
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March 24, 2021
Step Pharma Raises $41.3 Million to Advance First-in-Class Therapy to Clinic in T Cell Malignancies
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March 24, 2021
Horizon Therapeutics Launches Prize with MIT to Advance Solutions for the Rare Disease Community
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March 24, 2021
FDA Grants Fast Track Designation to Reneo’s PMM Therapy
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March 24, 2021
ProQR Reports Positive Results of QR-421a in Usher Syndrome
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March 24, 2021
Researchers Find New Genetic Links to Rare Eye Disease
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March 23, 2021
PhoreMost Raises $46 Million to Advance Brain Cancer Treatment into Clinic
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March 23, 2021
Maze Therapeutics Reveals First Three Programs Targeting Genetic Drivers of Disease
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March 23, 2021
Genentech Halting Late-Stage Huntington’s Disease Study
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March 23, 2021
Recordati Reports Positive Results from Late-Stage Cushing’s Disease Study
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March 22, 2021
Study Shows Proof-of-Concept of Gene Editing in DMD
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March 22, 2021
Whole Genome Sequencing Enables Rare Disease Diagnoses
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March 22, 2021
China Grants Citrine Clinical Trial Waiver for Narcolepsy Therapy Wakix
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March 22, 2021
Aura Raises $80 Million to Advance Pipeline Targeting Rare Ocular Cancers
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March 22, 2021
VeriSIM Launches PulmoSIM to Develop Treatments for Rare Respiratory Diseases
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March 22, 2021
BridgeBio’s Calcilytix Reports Proof-Of-Concept for ADH1 Therapy
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March 19, 2021
Side Effects: The Toll a Rare Disease Can Take on a Family in Pursuit of a Cure
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March 18, 2021
Two Rare Disease Biotechs Rake in $170 Million in Public Equity Offerings
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March 18, 2021
Sarepta Reports Positive 2-Year Results from LGMD2E Gene Therapy Trial
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March 18, 2021
Chris Austin Will Leave NCATS to Join VC Firm Flagship Pioneering
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March 18, 2021
Gain Therapeutics Raises $40M in IPO to Advance Rare Disease Pipeline
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March 18, 2021
Rare Leader: Melanie McKay, Founder, Ryker’s Foundation for Pompe Disease
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March 18, 2021
Anima and Takeda to Collaborate on Neurological Disease Therapeutics
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March 18, 2021
Translate Bio Reports Results from Trial of mRNA Therapy for Cystic Fibrosis
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March 17, 2021
Amathus and Merck Enter into Neurodegenerative Disease Pact
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March 16, 2021
GBT In-Licenses Two Sickle Cell Small Molecule Programs from Sanofi
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March 16, 2021
StrideBio Raises $81.5M to Advance AAV-Based Gene Therapies into Clinic
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March 16, 2021
Scientist Develop Novel Gene Editor to Correct CF and Other Mutations
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March 16, 2021
Genentech Reports Positive New Data for Evrysdi in SMA Type 2 or Type 3
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March 16, 2021
Nurix Enters Pediatric Cancer Collaboration Sponsored by ALSF
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March 16, 2021
Solid Reports New Data from DMD Clinical Trial, Resumes Patient Dosing
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March 15, 2021
Bluebird bio Reports Positive Long-Term Data for CALD Gene Therapy
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March 15, 2021
Graphite Raises $150 Million to Advance Gene Editing Therapies for Rare Diseases
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March 15, 2021
Genevant and Takeda Partner to Develop RNA Therapies for Liver Fibrosis
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March 15, 2021
ElevateBio Raises $525M to Advance Cell/ Gene Therapy Business Model
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March 15, 2021
Savara Raises $130 Million to Advance Development of aPAP Therapy
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March 12, 2021
Realizing the Potential of CRISPR Gene Editing
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March 12, 2021
Congress Introduces Legislation Intended to Speed Development of Rare Disease Therapies
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March 12, 2021
Longboard Raises $80 Million in IPO to Advance Rare Neurological Pipeline
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March 11, 2021
Rare Leader: Blynda Killian, President, Cyclic Vomiting Syndrome Association
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March 11, 2021
Intellia Presents Preclinical Proof of Concept for CRISPR-Based in Vivo Editing
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March 11, 2021
Science 37 and Xperiome Partner to Speed Rare Disease Studies for Patients and Providers
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March 11, 2021
FDA Approves Compassionate Use of Longeveron Therapy in Child with Rare Heart Defect
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March 11, 2021
Inability to Enroll Patients Terminates More Than a Quarter of Rare Disease Drug Trials
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March 10, 2021
FDA Approves Second Sight Medical Products’ Retinal Prosthesis System
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March 10, 2021
Bluebird Says Case of AML Unlikely Related to Gene Therapy Vector
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March 10, 2021
FDA Tells Soleno It Will Need to Conduct Additional Study for Prader-Willi Therapy
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March 9, 2021
Spinal Muscular Atrophy Foundation and PTC Therapeutics Expand Partnership
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March 9, 2021
FDA Grants RMAT Designation to Rocket’s Gene Therapy for Rare Pediatric Disease LAD-I
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March 8, 2021
FDA Approves Kite’s Yescarta for Relapsed or Refractory Follicular Lymphoma as Third Line Therapy
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March 8, 2021
NICE Recommends Novartis’ Gene Therapy for SMA
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March 8, 2021
AnaptysBio’s Experimental Therapy for Palmoplantar Pustulosis Fails in Phase 2 Trial
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March 8, 2021
FDA Grants Fast Track Designation to Three Passage Bio Gene Therapy Candidates for Rare CNS Disorders
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March 8, 2021
FDA Grants BioMarin RMAT Designation for Experimental Hemophilia A Gene Therapy
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March 5, 2021
Understanding the Economic Toll of Rare Disease in the United States
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March 5, 2021
FDA Approves Genentech’s Actemra to Slow Decline in Pulmonary Function in Adults with SSc
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March 5, 2021
Rare Diseases Account for 11 percent of U.S. Drug Spending, Report Finds
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March 4, 2021
When Blockbusters Garner the Benefits of Orphan Drugs
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March 4, 2021
Rare Leader: Danae’ Bartke, executive director and co-founder, HCU Network America
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March 4, 2021
Advocacy Groups Call on Governors to Prioritize Vaccination of Rare Disease Patients and Caregivers
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March 4, 2021
Biogen Will Construct $200 Million Gene Therapy Manufacturing Facility in North Carolina
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March 3, 2021
Takeda Secures Global Rights to Rare Epilepsy Treatment from Ovid for $860 Million
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March 3, 2021
Caribou Raises $115 Million to Advance Next-Generation CRISPR Technologies
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March 3, 2021
Study Shows Potential for Lipid Nanonparticles to Deliver Gene Editing Therapy to Liver
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March 2, 2021
FDA approves Oncopeptides’ Multiple Myeloma Therapy
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March 2, 2021
eGenesis Completes $125 Million Financing
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March 2, 2021
Mesoblast Raises $110 Million
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March 2, 2021
Children’s National Hospital Rare Disease Institute Partners with Takeda to Standardize Care for Patients with Rare Diseases
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March 2, 2021
GBT Grant Program to Support to the Sickle Cell Disease Community
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March 1, 2021
DTx Pharma Raises $100 Million to Advance its Platform and Pipeline of RNA Therapeutics
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March 1, 2021
FDA Grants BridgeBio and Its Affiliate Origin Biosciences Approval for MoCD Therapy
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March 1, 2021
SalioGen Therapeutics Emerges from Stealth, Closes $20 Million Series A
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March 1, 2021
Ionis Establishes New Grant Program to Advance Research and Understanding of ATTR
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February 26, 2021
The Promise of Gene-Based Therapies for Neurodegenerative Conditions
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February 25, 2021
When a Dollar Sign Becomes an Exclamation Point
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February 25, 2021
Former AveXis Executives Launch Jaguar Gene Therapy to Target Rare Diseases
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February 25, 2021
Rare Leader: Mark Stone, President and CEO, FSHD Society
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February 25, 2021
FDA Grants Rare Pediatric Disease Designation to Retrotope’s Therapy for Two Neurodegenerative Indications
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February 24, 2021
Orna Raises $80 Million to Develop a New Class of Engineered RNA Therapies
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February 24, 2021
Scailyte and Volv Global Enter Strategic Partnership
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February 24, 2021
Children’s Mercy Kansas City Expands Sequencing Capabilities
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February 24, 2021
Cyprium and Sentynl Sign Development and Purchase Agreement for Menkes Disease Therapy
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February 23, 2021
Shepherd Enters Collaboration with NCATS to Improve Treatments for Rare Head and Neck Cancer
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February 23, 2021
Soleno Collaborates with Vanderbilt on KATP Channel Activators for the Treatment of Rare Diseases
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February 23, 2021
Day One Licenses Merck KGaA’s MEK Inhibitor Pimasertib, Expands Pipeline
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February 23, 2021
BC Platforms Partners with Genomenon to Boost Rare Disease Diagnostics and Research
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February 23, 2021
Beam Completes Acquisition of GuideTx
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February 23, 2021
Protalix and Chiesi Report Positive Results from Open-Label, Switch Over Trial of Therapy for Fabry Disease
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February 22, 2021
FDA Tells BrainStorm Evidence Appears Lacking to Support Seeking Approval for ALS Therapy
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February 19, 2021
Moving Beyond Viral Vectors for Gene Therapies
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February 18, 2021
Rare Leader: Paula Evans, founder and chairperson, Foundation for Angelman Syndrome Therapeutics (FAST)
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February 18, 2021
NCI Awards $16 Million Grant to Advance Research of Two Rare Cancers
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February 18, 2021
Charles River to Acquire Cell and Gene Therapy Manufacturer Cognate BioServices for $875 million
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February 18, 2021
FDA Approves First-of-Its-Kind Implant to Treat Rare Bone Disease as a Humanitarian Use Device
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February 18, 2021
FDNA Launches Telehealth AI Telegenetics Platform
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February 18, 2021
Evox Therapeutics Raises $95.4 Million To Advance Exosome-Based Pipeline
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February 18, 2021
Otsuka and Eikonizo to Collaborate on Treatments for ALS and Other Rare Diseases
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February 17, 2021
Novartis Teams with Gates Foundation to Develop an Accessible in Vivo SCD Gene Therapy
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February 17, 2021
Rare Disease Drug Developers Busy Raising Money in January
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February 17, 2021
Health Canada Approves Ultragenyx’ Drug for Group of Rare Metabolic Disorders
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February 17, 2021
Eurordis Survey Finds People with Rare Diseases Have Concerns with Quality of Healthcare Experience
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February 16, 2021
Study Finds Commercial Genetic Testing Technology Falsely Detects Very Rare Variants
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February 16, 2021
Bluebird Bio Temporarily Suspends SCD Gene Therapy Trials after Case of AML
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February 16, 2021
FDA Approves Pfizer’s Panzyga to Treatment of Adults with CIDP
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February 16, 2021
Shepherd and Oncoheroes Partner to on Medicines for Rare Cancers
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February 16, 2021
FDA Grants Rare Pediatric Disease Designation to Forge’s Gene Therapy for Krabbe Disease
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February 12, 2021
Mila Makovec, Recipient of Bespoke ASO for Ultra-Rare Condition, Dies at 10
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February 12, 2021
Acquisitions Help Jazz Build Toward Commercial Crescendo
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February 12, 2021
Patients’ Cells Offer Insight into New Gene Therapy Approach to Rare Eye Disease
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February 12, 2021
Travere Raises $175 Million in Public Offering to Advance Rare Kidney Disease Pipeline
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February 12, 2021
Decibel Raises $127 Million in IPO to Advance Gene Therapies for Hearing and Balance Disorders
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February 12, 2021
Denali Reports Positive Early Data from Phase 1/2 Study in Hunter Syndrome
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February 12, 2021
FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Rescindo’s Treatment for Kabuki Syndrome
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February 11, 2021
FDA Approves Regeneron’s Evkeeza for Patients with Ultra-Rare Form of High Cholesterol
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February 11, 2021
Rare Leader: Monica Coenraads, CEO, Rett Syndrome Research Trust
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February 11, 2021
Cyteir Secures $80 Million for Synthetic Lethalities
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February 11, 2021
Chinook Tackles Rare Chronic Kidney Diseases
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February 11, 2021
KalVista Raises $193.5 Million in Public Offering to Advance HAE Treatment
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February 11, 2021
Ensoma Launches with $70 Million for In Vivo Gene Therapy, Partners with Takeda
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February 10, 2021
Day One Raises $130 Million to Accelerate New Targeted Cancer Treatments for Children
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February 10, 2021
AbbVie and Caribou Biosciences Enter Collaboration for Off-the-Shelf CAR-T Therapies
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February 10, 2021
Ipsen Enters Agreement with Mendelian to Use Company’s Software to Diagnose Rare Diseases
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February 10, 2021
Galapagos and Gilead Discontinue Late-Stage Study in IPF, Stop Development of Drug
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February 10, 2021
HemoShear Raises $40 Million to Advance Rare Disease Portfolio
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February 10, 2021
Mereo BioPharma Raises $100 Million in Public Offering
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February 10, 2021
FDA Expands Use of Botox to include Rare Pediatric Neurologic Condition
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February 10, 2021
Study Suggests Way to Arrest Two Rare Disease Before Birth
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February 9, 2021
Not the Way it Normally Happens
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February 9, 2021
KalVista Reports Positive Results in Trial of Oral Treatment for HAE Attacks
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February 8, 2021
Avrobio Reports Positive Results of Its Gene Therapy for Fabry Disease
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February 8, 2021
Editas’ Collins Steps Down, Chairman Mullen Takes Reins
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February 8, 2021
Ocugen Raises $23 Million to Advance Ocular Gene Therapies
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February 8, 2021
FDA Approves Two Rare Cancer Therapies
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February 5, 2021
Powerful Gene Editing Approach Offers the Promise of Correcting a Range of Rare Diseases
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February 5, 2021
Two Rare Disease Biotechs Hit the Market in Upsized IPOs
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February 5, 2021
Rhythm and Orchard Each Raise $150 Million to Advance Their Rare Disease Programs
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February 4, 2021
Rare Leader: Karen Chen, CEO, Spinal Muscular Atrophy Foundation
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February 4, 2021
Kaleido Raises $60 Million in Public Offering to Advance Pipeline of Microbiome Targeted Therapies
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February 4, 2021
Sana Raises $588 Million in IPO to Advance Engineered Cell Therapies
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February 4, 2021
Coya Completes Merger and Raises $10 Million to Advance Pipeline of Tregs for Neurodegenerative Diseases
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February 4, 2021
Prothena Revives Birtamimab Program in AL Amyloidosis
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February 3, 2021
Jazz Pharmaceuticals to Acquire GW Pharmaceuticals for $7.2 Billion
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February 3, 2021
The Making of A Rare Parent
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February 3, 2021
Neurocrine Terminates Parkinson’s Gene Therapy Collaboration with Voyager Therapeutics
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February 2, 2021
Travere Achieves Interim Proteinuria Endpoint in Ongoing Study of Sparsentan in FSGS
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February 2, 2021
Immunovant Shares Tumble as It Pauses Clinical Dosing of TED Therapy
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February 2, 2021
FDA Removes Clinical Hold on Mustang Gene Therapies for SCID
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February 2, 2021
FDA Grants Rare Pediatric Disease Designation to Chinook’s Treatment of Primary Hyperoxaluria
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February 1, 2021
Horizon to Acquire Viela Bio to Expand Pipeline and Grow Rare Disease Portfolio
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February 1, 2021
CHOP Researchers Develop Improved Vector for Blood Disorder Gene Therapies
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January 29, 2021
A Child’s-Eye-View of Clinical Trials
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January 29, 2021
AGTC Raises $74.5 Million to Advance Pipeline of Gene Therapies for Rare Diseases
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January 29, 2021
Catabasis Pharmaceuticals Acquires Quellis Biosciences and Raises $110 Million in Private Offering
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January 28, 2021
Gina Glass, Executive Director, Dreamsickle Kids Foundation
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January 28, 2021
Health Canada Seeks Input from Canadians on a National Strategy for High-Cost Drugs for Rare Diseases
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January 28, 2021
FDA Grants M6P Therapeutics Six Rare Pediatric Disease designations for LSDs
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January 28, 2021
NORD Issues State Report Cards on Policy Issues Critical to Rare Disease Patients
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January 27, 2021
Study in Flies Reveals Interaction Between Genes Drives Severity of Ultra Rare Disease
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January 27, 2021
Agios’ Mitapivat Meets Primary Endpoint in Late-Stage Study in Pyruvate Kinase Deficiency
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January 27, 2021
Design Secures $125 Million to Advance Treatments for Repeat Expansion Disorders
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January 27, 2021
FDA Approves Recordati’s Carbaglu to Treat Acute Hyperammonemia
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January 27, 2021
Catalyst Biosciences Raises $50 Million to Advance Rare Disease Pipeline
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January 26, 2021
Rhythm Reports Positive Data with Setmelanotide in Additional Rare Obesities
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January 26, 2021
BridgeBio Prices Upsized Offering of $650 Million Convertible Senior Notes
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January 26, 2021
Relief and Acer Sign Option Agreement for Exclusivity on Treatment for UCDs
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January 25, 2021
Patients with Chronic Rare Conditions Feel Unsupported
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January 25, 2021
Eiger Launches Zokinvy for Treatment of Progeria
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January 22, 2021
Using Nanoliposomes to Make Cancer Therapies Safer and More Effective
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January 22, 2021
Spate of Public Offerings Continues as Two More Companies Raise Capital in Public Markets
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January 21, 2021
Rare Leader: Keith Harper, Board President, Narcolepsy Network
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January 21, 2021
Editas Medicine and Dyne Therapeutics Raise Combined $399 Million in Public Offerings
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January 20, 2021
EC Approves Extension of SOBI’s Doptelet for ITP
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January 20, 2021
UCSD and Ionis Use ASO to Target Multiple Myeloma
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January 20, 2021
Three Rare Disease Companies Raise Combined $380 Million in Public Offerings
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January 19, 2021
FDA Grants Taysha Rare Pediatric Disease Designation for SLC13A5 Deficiency Candidate
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January 18, 2021
FDA Approves Janssen’s Darzalex Faspro to Treat Newly Diagnosed AL Amyloidosis
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January 15, 2021
Accelerating Treatments for Rare Disease through Data Sharing
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January 14, 2021
Rare Leader: Heidi Wallis, President, Association for Creatine Deficiencies
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January 14, 2021
Sarepta and Genevant Enter Research Collaboration for LNP-Based Gene Editing Therapeutics
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January 14, 2021
Aldeyra Therapeutics Raises $65 Million to Advance Therapies Targeting Immune-Mediated Diseases
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January 14, 2021
FDA Grants RMAT Designation to Orchard Therapeutics Treatment for Metachromatic Leukodystrophy
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January 13, 2021
Study Suggests Genetic Editing Could Correct Rare Disease that Causes Rapid Aging
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January 13, 2021
Taysha and UT Southwestern Launch Innovation Fund to Advance CNS Gene Therapies into Clinic
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January 13, 2021
Inzen Therapeutics Unveiled to Explore Novel Medicines Based on Input from Dying Cells
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January 12, 2021
Tessera Raises More Than $230 Million to Advance ‘Gene Writing’ Platform
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January 11, 2021
The United Kingdom Releases a Rare Disease Framework
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January 11, 2021
Atalanta Therapeutics Launches $110 Million to Pioneer RNAi Therapeutics for Neurodegenerative Diseases
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January 11, 2021
Bluebird Bio Retains Gene Therapy and Spins Out Oncology into Separate Company
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January 8, 2021
Sarepta DMD Gene Therapy Meets Primary Endpoint but Fails to Show Functional Benefit, Shares Fall
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January 8, 2021
Treating the Root Cause of Sickle Cell Disease
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January 7, 2021
FDA Issues Guidance on Individualized Medicines
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January 7, 2021
A Stellar Year for Rare Disease Financings
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January 7, 2021
Rare Leader: Nasha Fitter, CEO, FOXG1 Research Foundation
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January 7, 2021
Lexeo Therapeutics Launches with $85 Million to Develop Gene Therapies for Monogenic Diseases
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January 7, 2021
Abcuro Raises $42 Million to Develop Immune Modulating Therapies
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January 7, 2021
Argenx and Zai Lab Enter Strategic Collaboration to Expand Reach of Autoimmune Therapy
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January 7, 2021
Endeavor BioMedicines Launches with $62 Million to Attack Idiopathic Pulmonary Fibrosis
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January 7, 2021
Alnylam Reports Positive Topline Results from Late-Stage hATTR Study
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January 7, 2021
Gene Therapy Developer Generation Bio Raises $196 Million in Public Offering
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January 7, 2021
FDA Grants Rare Pediatric Disease Designations to Inversago and to Praxis Precision Medicines
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January 6, 2021
Rhythm Pharmaceuticals Sells Priority Review Voucher to Alexion for $100M
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January 6, 2021
Mount Sinai and Rumi Scientific Partner to Advance Drug Discovery for Rare Genetic Disorders Tied to Autism
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January 5, 2021
Biogen and ViGeneron Enter into Global Development Collaboration for Ophthalmic Gene Therapy
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January 5, 2021
Aro Biotherapeutics Raises $88 Million to Advance Development of Genetic Medicines
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January 5, 2021
A Go-Go Market for Rare Disease IPOs
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January 5, 2021
A Banner Year for Venture Funding
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January 5, 2021
Rare Disease Secondary and Debt Offerings Rise in 2020
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January 5, 2021
Rare Partnering Slows in 2020, but Deal Values Grow
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January 5, 2021
AstraZeneca Acquisition of Alexion Tops M&A in 2020
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January 4, 2021
Taysha Gene Therapies Collaborates with AllStripes on Leigh Syndrome
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December 31, 2020
Targeting A Common Pathway in Genetic Forms of Obesity
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December 30, 2020
Incyte and Cellenkos Enter into Global Development Collaboration of Combo Treatment for Myelofibrosis
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December 30, 2020
Protalix and Chiesi Report Final Results of Phase 3 Study of Therapy for Fabry Disease
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December 29, 2020
HKUMed Researchers Discover Novel Gene Implicated in Rare Heterotaxy Syndrome
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December 28, 2020
Aprea’s Experimental Therapy Misses Primary Endpoint in Late Stage MDS Trial
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December 28, 2020
United Therapeutics Acquires Rare Disease Priority Review Voucher
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December 28, 2020
FDA Lifts Hold on Audentes’ Gene Therapy Trial for the Treatment of X-Linked Myotubular Myopathy
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December 24, 2020
After Pruning Orchard, Gaspar Focuses on High Value Opportunities
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December 24, 2020
Rare Leader: Allison Kaczenski, Founder and President, SATB2 Gene Foundation
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December 23, 2020
RegenxBio Sells Portion of Zolgensma Royalties for $200 Million
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December 23, 2020
FDA Grants Rare Pediatric Disease Designation to Novadip’s Treatment for Rare Bone Disease
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December 23, 2020
BridgeBio Enters Collaboration with UCSF to Accelerate the Development of Therapies for Genetic Diseases
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December 22, 2020
Vertex Partners with Skyhawk to Modulate RNA Splicing for Serious Diseases
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December 22, 2020
Rhythm Obesity Drug Hits Endpoint in Bardet-Biedl Patients, but Misses in Alström Syndrome Patients
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December 21, 2020
Agios Sells Oncology Business for $2 Billion to Focus on Genetic Diseases
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December 21, 2020
FDA Places Clinical Hold on UniQure Hemophilia B Gene Therapy Program
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December 18, 2020
Writing a New Chapter of Genetic Medicine
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December 18, 2020
Mereo BioPharma and Ultragenyx Partner to Develop Setrusumab for Rare Bone Disease
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December 18, 2020
GSK and Adrestia and Enter into Precision Medicine Discovery Collaboration
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December 18, 2020
FDA Grants Rare Pediatric Disease Designation to Polaryx Therapeutics’ Treatment for Niemann Pick Disease
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December 17, 2020
Rare Leader: Mike Graglia, Managing Director, Syngap Research Fund
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December 17, 2020
Reengineering Gene Therapy Development
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December 17, 2020
Novartis to Acquire Cadent Therapeutics for Up to $770 Million
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December 16, 2020
Neurogene Raises $115 Million to Advance Gene Therapies for Rare Neurological Diseases