RARE Daily - Global Genes

Articles

RARECast Podcasts

September 24, 2021

Creating a Playbook for Bespoke Gene Therapies

Drug Development

September 24, 2021

Long-Term Treatment with Crysvita Reduces the Burden of Disease in Adults With XLH

Drug Development

September 24, 2021

AGTC Reports Serious Adverse Events in Three Patients

Drug Development

September 24, 2021

Capricor Reports Positive Final Data From its Phase 2 DMD Trial of Experimental Cell Therapy

Drug Development

September 24, 2021

Roche Presents New Data Supporting Safety and Efficacy of its Neuromuscular Therapies

Research

September 24, 2021

CIIC and Trio Health Introduce the Creation of a Dynamic Rare Disease Patient Registry Platform

Rare Advocate

September 23, 2021

Rare Leader: Shari Hume, Co-Founder, Cure JM Foundation

Finance

September 23, 2021

Garuda Therapeutics Launches with $72 Million Series A Financing

Finance

September 23, 2021

Synlogic Raises $45 Million in Public Offering to Advance Treatments for Metabolic Disorders

Finance

September 23, 2021

GenEdit Raises $26 Million to Support Nanoparticle Delivery Platform for Genetic Medicines

Drug Development

September 23, 2021

FDA Expands Use of Incyte’s Jakafi to Include GVHD

Finance

September 22, 2021

Opus Genetics Launches with $19 Million to Advance Gene Therapies for Retinal Diseases

Drug Development

September 22, 2021

Rhythm Presents New Data for Setmelanotide in Multiple Rare Genetic Diseases of Obesity

Regulatory

September 21, 2021

FDA Should Look to Past Precedents When Applying Flexibility to Approvals to Maintain Public Trust, Study...

Deals

September 21, 2021

Novartis Acquires Arctos Medical, Expanding Portfolio of Gene Therapies for Rare Eye Disorders

Deals

September 21, 2021

Mirum and Takeda Partner to Develop and Commercialize Rare Pediatric Liver Disease Drug in Japan

Drug Development

September 21, 2021

Sunitinib Prolongs Progression Free Survival in Rare Neuroendocrine Tumors

Diagnosis

September 21, 2021

Horizon Awards First Horizon Prize to ThinkGenetic, Congenica, for Work to Accelerate Diagnosis of Rare Diseases

Finance

September 20, 2021

Rare Disease Dealmaking Heats Up in August

Diagnosis

September 20, 2021

Genomenon and Alexion Collaboration to Accelerate Genetic Diagnosis for Rare Disease Patients

RARECast Podcasts

September 17, 2021

Finding Answers for Undiagnosed Patients with Rare Genetic Diseases

Drug Development

September 17, 2021

FDA Places Hold on Protagonist’s Blood Disorders Program after Skin Tumors Observed in Mouse Study

Uncategorized

September 16, 2021

Rare Leader: Geoff Rhyne, Co-Founder and CEO, IDefine

Deals

September 16, 2021

Travere Licenses Sparsentan to Vifor Pharma for Commercialization in Europe, Australia, and New Zealand

Finance

September 16, 2021

Amolyt Raises $80 Million to Advance Rare Endocrine Pipeline

Finance

September 16, 2021

Anjarium Raises $61 Million to Develop Next-Generation Non-viral Gene Therapies

Drug Development

September 16, 2021

FDA Approves Takeda’s Exkivity for Subset of NSCLC

Finance

September 15, 2021

Avalo Raises $27.5 Million to Advance Pipeline of Cancer and Rare Genetic Disease Therapies

Drug Development

September 15, 2021

Rarebase Launches a Neuroscience Drug Discovery Platform Collaborating with 15 Rare Disease Patient Groups

Drug Development

September 15, 2021

FDA Grants Fast Track Designation to BridgeBio’s Experimental Therapy for LGMD2i

Drug Development

September 15, 2021

FDA Grants Fast Track Designation to Sage for Huntington’s Disease Therapy

Drug Development

September 15, 2021

FDA Extends Action Date for Calliditas Nefecon

Uncategorized

September 15, 2021

FDA Lifts Clinical Hold on KalVista HAE Therapy

Finance

September 14, 2021

Skyhawk Raises $133 Million to Advance its RNA-Modifying Candidates into the Clinic

Finance

September 14, 2021

Vanqua Bio Launches with $85 Million to Target Neurodegenerative Diseases

Finance

September 14, 2021

Walking Fish Launches with $50 Million to Advance B Cell Therapies for Multiple Diseases

Drug Development

September 14, 2021

Astellas Reports Fourth Patient Dies in XLMTM Gene Therapy Trial

Legislation

September 13, 2021

NORD Warns Provision in Biden Administration’s Ambitious Legislation Would Cut Benefits of Orphan Drug Tax Credit

Deals

September 13, 2021

RARE-X and C-Path Establish New Collaboration for Rare Disease Data Sharing

Drug Development

September 13, 2021

Chiesi Global Rare Diseases Wins Approval in Brazil for Treatment of Iron Overload in SCD

RARECast Podcasts

September 10, 2021

How a Familiar Face Can Lead to a Rare Disease Diagnosis

Research

September 10, 2021

Research Collaboration Seeks to Accelerate Rare Disease Diagnostics

Drug Development

September 10, 2021

FDA Grants Decibel Rare Pediatric Disease Designations for Congenital Hearing Loss Therapy

Drug Development

September 10, 2021

Sanofi Experimental Therapy for Rare Skin Condition Fails in Late-Stage Study

Rare Advocate

September 9, 2021

Rare Leader: Cyndi Roemer, Board Chair, The MSA Coalition

Finance

September 9, 2021

Mammoth Raises $195 Million to Build Next-Generation CRISPR Therapeutics and Diagnostics

Finance

September 9, 2021

iECURE Launches With $50 Million to Develop In Vivo Gene Insertion

Finance

September 9, 2021

LEXEO Therapeutics Closes $100 Million to Advance Gene Therapy Pipeline

Deals

September 8, 2021

Albireo Sells Priority Review Voucher (PRV) for $105 Million

Finance

September 8, 2021

ADARx Raises $75 Million to Advance Pipeline of RNA Targeting Therapeutics

Finance

September 8, 2021

Attralus Raises $116 Million to Advance Pan-Amyloid Removal Therapeutics

Finance

September 8, 2021

Bluebird Bio Secures $75 Million Ahead of Separation into Two Companies

Deals

September 8, 2021

Sanofi to Acquire Kadmon for $1.9 Billion, Gaining Approved Therapy for cGVHD

Strategy

September 8, 2021

Catabasis Takes New Name to Highlight New Focus

Deals

September 7, 2021

Invitae to Acquire Ciitizen for $325 Million

Drug Development

September 7, 2021

FDA Places Clinical Hold on BioMarin’s Gene Therapy Study in Adults with PKU

Deals

September 7, 2021

Moderna and ILCM to Collaborate to Develop mRNA Therapeutic for Crigler-Najjar Syndrome Type 1

Deals

September 7, 2021

NeuExcell Therapeutics Enters Research Collaboration with Spark for Huntington’s Disease Gene Therapy

Drug Development

September 7, 2021

EU Expands Approval of Ultomiris to Include Children and Adolescents with PNH

RARECast Podcasts

September 3, 2021

Bringing the Trial to the Patient

Deals

September 3, 2021

SOBI Board of Directors Recommends $8.1 Billion Takeover by Private Equity Consortium

Rare Advocate

September 2, 2021

Rare Leader: Earl Cole, Founder and Chairman, Perthes Kids Foundation

Finance

September 2, 2021

Disc Medicine Raises $90 Million to Advance Therapies for Hematologic Diseases

Research

September 2, 2021

Researchers Develop Test to Determine Patients with Rare Blood Disorder Likely to Benefit from Existing Therapy

Deals

September 2, 2021

Polyphor and EnBiotix Sign Merger Agreement and sale of Inhaled Murepavadin to EnBiotix

Drug Development

September 2, 2021

Takeda’s Breakthrough Therapy for Rare Cancer Fails in Phase 3

Drug Development

September 2, 2021

FDA Approves Beigene’s Brukinsa to Treat Waldenström’s Macroglobulinemia

Research

September 1, 2021

CZI Award $3 Million to Researchers to Build Pediatric Cell Atlas of Skeletal Muscle

Drug Development

September 1, 2021

Astellas Voluntarily Halts X-linked Myotubular Myopathy Gene Therapy Trial

Deals

August 31, 2021

VectivBio Acquires Comet, Expanding Rare Metabolic Disease Pipeline

Finance

August 30, 2021

Laronde Raises $440 Million to Further Advance New Class of Programmable Medicines

RARECast Podcasts

August 27, 2021

Targeting Rare and Chronic Kidney Diseases

Drug Development

August 27, 2021

BioMarin’s Voxzogo Approved in Europe to Treat Children with Achondroplasia

Uncategorized

August 27, 2021

Earlier Diagnosis Can Reduce Cost of Raising a Child with a Rare Genetic Disorder

Drug Development

August 27, 2021

EMA Grants Marinus Accelerated Assessment of Ganaxolone for Treatment of CDKL5 Deficiency Disorder

Drug Development

August 27, 2021

FDA Approves Expanded Use of Servier’s Tibsovo to Include Rare Bile Duct Cancer

Rare Advocate

August 26, 2021

Rare Leader: Casey Gorman, Executive Director, PRISMS (Patients and Researchers Interested in Smith-Magenis Syndrome)

Uncategorized

August 26, 2021

Alexion Reports Positive Late-Stage Results for Experimental Wilson Disease Therapeutic

Drug Development

August 26, 2021

FDA Approves Ascendis Pharma’s Skytrofa for Pediatric Growth Hormone Deficiency

Drug Development

August 25, 2021

Stealth Seeks FDA Approval for Barth Syndrome Treatment, Despite Call for More Data

Research

August 24, 2021

As FDA Readies for PDUFA VII, Rare Disease, Cell and Gene Therapies in the Mix

Finance

August 24, 2021

AllStripes Raises $50 Million to Advance Global Rare Disease Research

Deals

August 24, 2021

Vertex and Arbor Partner to Develop Novel Ex Vivo Engineered Cell Therapies

Deals

August 24, 2021

Shape and Roche Collaborate to advance AAV-based RNA Editing for Neuroscience and Rare Diseases

Deals

August 23, 2021

Pfizer to Acquire Trillium For $2.3 Billion to Beef Up Rare Blood Cancer Pipeline

Deals

August 23, 2021

Genevant and Takeda Partner to Develop Nonviral Rare Liver Disease Gene Therapies

Research

August 23, 2021

Researchers Uncover Cause of Rare Bone Disorder

RARECast Podcasts

August 20, 2021

Addressing the Delivery Challenges of Genetic Medicines

Drug Development

August 20, 2021

Alexion Discontinues Late-Stage ALS Trial of Ultomiris Due to Lack of Efficacy

Uncategorized

August 20, 2021

Discovery of the Mechanism for A Rare Autoinflammatory Disease Brings Repurposed Treatment

Rare Advocate

August 19, 2021

Rare Leader: Amber Olsen, Executive Director, United MSD Foundation

Uncategorized

August 19, 2021

FDA Grants Fast Track Designation to Senhwa’s Silmitasertib to Treat Recurrent SHH Medulloblastoma

Drug Development

August 19, 2021

NICE Recommends First Treatment for Rare Blood Disorder

Finance

August 18, 2021

Jnana Therapeutics Raises $50 Million to Advance Lead PKU Program and Pipeline

Finance

August 18, 2021

Vigil Raises $90 Million to Advance Pipeline to Treat Neurodegenerative Diseases

In Rare Form

August 18, 2021

Why Becoming a Rare Disease Expert Isn’t Enough for Parents

Drug Development

August 18, 2021

Marinus Reports Positive Topline Ganaxolone Phase 2 Results in Tuberous Sclerosis Complex

Deals

August 17, 2021

Aurinia Acquires Novel Pipeline Assets Targeting Autoimmune and Kidney-Related Diseases

Drug Development

August 16, 2021

Merck Wins Approval for Treatment for Von Hippel-Lindau Disease-Associated Tumors

Drug Development

August 16, 2021

FDA Lifts Clinical Hold on Rocket Pharma’s Gene Therapy Trial for Danon Disease

Finance

August 16, 2021

Beckley Psytech Raises $80 Million to Develop Portfolio of Psychedelic Medicines

Drug Development

August 16, 2021

Travere Reports Positive Topline Interim Results from Phase 3 Study of Sparsentan in IgAN

Drug Development

August 16, 2021

FDA Grants Edgewise Fast Track Designation for Becker Muscular Dystrophy Therapy

RARECast Podcasts

August 13, 2021

Targeting Tissues Throughout the Body with RNA Therapies

Drug Development

August 13, 2021

FDA Grants First of its Kind Indication for Chronic Sleep Disorder Treatment

Uncategorized

August 13, 2021

Ipsen Withdraws Application Seeking Approval for Rare Bone Disease Therapy

Drug Development

August 13, 2021

FDA Grants Fast Track Designation to Vivet’s Therapy for the Treatment of Wilson Disease

Uncategorized

August 13, 2021

FDA Grants Breakthrough Therapy Designation to Junshi’ and Coherus’ Treatment for Nasopharyngeal Carcinoma

Rare Advocate

August 12, 2021

Rare Leader, Jennifer Martin, President, Helping Hands for GAND

Finance

August 12, 2021

Fulcrum Raises $125 Million in Public Offering to Advance Rare Disease Pipeline

Drug Development

August 12, 2021

CRO MMS Launches Pro-Bono Regulatory Assistance Program for Ultra-Rare Disease Patient Advocacy Groups

Finance

August 11, 2021

Harmony Biosciences Enters into Strategic Financing Collaboration with Blackstone

Drug Development

August 11, 2021

First Intravenous Infusion Gene Therapy for Rare Disease in China Cleared for Clinical Testing

Finance

August 10, 2021

Money Continues to Flow into Rare Disease Drug Development

Deals

August 10, 2021

Healx Enters Partnership with Ono Pharmaceutical to Repurpose Drugs for Rare Diseases

Deals

August 10, 2021

VGXI Partners with Rare Trait Hope Fund in Support of Gene Therapy for Rare Disease AGU

Drug Development

August 9, 2021

FDA Places Clinical Hold on Bluebird Bio CALD Candidate for Safety Concerns

Drug Development

August 9, 2021

Liminal BioSciences to Sell Priority Review Voucher for $105 Million

Deals

August 9, 2021

Epizyme and Hutchmed Form Strategic Collaboration to Develop and Commercialize Tazverik in Greater China

Drug Development

August 9, 2021

FDA Approves Sanofi Genzyme’s New Treatment for Pompe Disease

Finance

August 9, 2021

Sierra Oncology Updates Timeline for Momelotinib in Myelofibrosis and Raises $34 Million

Drug Development

August 9, 2021

Harmony Biosciences Adds Novel Asset to Rare Neurological Disease Pipeline

RARECast Podcasts

August 6, 2021

Delivering Rare Disease Therapies to Patients in Need

Deals

August 6, 2021

Sierra Oncology Licenses AstraZeneca BET Inhibitor to Expand Myelofibrosis Pipeline

Drug Development

August 6, 2021

Dicerna Reports Positive Results from Pivotal Trial of Experimental RNAi Therapy for Primary Hyperoxaluria

Rare Advocate

August 5, 2021

Rare Leader: Stacy Pike-Langenfeld, Co-Founder and President, KrabbeConnect

Finance

August 5, 2021

PepGen Raises $112.5 Million to Advance Transformative Therapies for Neuromuscular Diseases

Finance

August 5, 2021

Aardvark Raises $29 Million to Fund Trials of Therapeutic for Prader-Willi Syndrome

Deals

August 5, 2021

Sarepta Enters Licensing Agreement for Nationwide Children’s Gene Therapy Program for Limb-Girdle Muscular Dystrophy

Drug Development

August 5, 2021

FDA Grants Breakthrough Therapy Designation to Eiger’s Avexitide for Congenital Hyperinsulinism

Policy

August 4, 2021

Industry Group Respond to Proposals that Could Undermine Accelerated Approval

Finance

August 4, 2021

Avidity Biosciences Raises $144 Million to Support RNA Therapeutic Rare Disease Pipeline

Research

August 4, 2021

Rare Disorder Offers Roadmap for Understanding Roots of Inflammatory Disease

Deals

August 3, 2021

Sanofi Takes Major Step into mRNA with Acquisition of Translate Bio for $3.2 Billion

Deals

August 3, 2021

Marinus Enters Collaboration with Orion for European Commercialization of Rare Disease Drug

Drug Development

August 3, 2021

FDA Clears Novartis to Start Phase 3 Study of Intrathecal Zolgensma in Older Patients with SMA

Drug Development

August 3, 2021

FDA Issues Complete Response Letter Received for Medac’s Allo-HSCT Preparative Regimen Treosulfan

Deals

August 2, 2021

Ipsen and Exicure Enter Exclusive Collaboration Targeting Rare Neurodegenerative Disorders

Finance

August 2, 2021

Immunovant Receives $200 Million Strategic Investment from Roivant to Advance Pipeline

Drug Development

August 2, 2021

EU Expands Approval for Amicus’s Fabry Disease Therapy to Adolescents

Drug Development

August 2, 2021

FDA Approves J&J’s Uptravi for Intravenous Use in Adult Patients with PAH

RARECast Podcasts

July 30, 2021

An Advocate’s Journey through Diagnosis, Loss, and Hope

Finance

July 30, 2021

Tenaya Raises $180 Million in Upsized IPO to Address Underlying Causes of Heart Disease

Rare Advocate

July 29, 2021

Rare Leader: Kimberly Ventarola, Co-Founder and President, Malan Syndrome Foundation

Finance

July 29, 2021

Rallybio Raises $81 Million in IPO to Advance Therapies for Rare Diseases

Finance

July 29, 2021

Crinetics Raises $15 Million from Frazier Healthcare Partners

Deals

July 29, 2021

Rhythm Signs Research Agreement with Clinical Registry Investigating Bardet-Biedl Syndrome

Drug Development

July 29, 2021

Alnylam Reports Positive Results from Phase 3 Study of Lumasiran in Advanced Primary Hyperoxaluria Type 1

Drug Development

July 29, 2021

FDA Grants Breakthrough Therapy Designation to Takeda’s Experimental Narcolepsy Drug

Drug Development

July 29, 2021

Alector Presents Positive Phase 2 Results of Experimental Therapy in FTD

Uncategorized

July 29, 2021

FDA Grants Breakthrough Therapy Designation to Arrowhead’s Experimental Treatment for AATD

Finance

July 28, 2021

Deep Genomics Raises $180 Million to Advance Programmable RNA Therapeutics

Deals

July 28, 2021

Aristea Enters into Collaboration with Arena in Neutrophil-Mediated Diseases and Raises $63 Million

Deals

July 28, 2021

Bluebird Bio Forms Strategic Alliance with Resilience to Develop Next Generation Cell Therapies

Finance

July 28, 2021

Ring Raises $117 Million to Advance Vector Platform to Unlock Full Potential of Gene Therapy

Drug Development

July 28, 2021

FDA Grants Fast Track Designation to Apic Bio’s APB-102 for the Treatment of Patients with SOD1...

Drug Development

July 28, 2021

FDA Grants Rare Pediatric Disease Designation to Lexeo Therapeutics CLN2 Batten Disease Therapy

Research

July 27, 2021

Study Links Autism to New Set of Rare Gene Variants

People & Organizations

July 27, 2021

RARE-X Appoints Charlene Son Rigby as Chief Executive Officer

Drug Development

July 27, 2021

FDA Grants Rare Pediatric Disease Designation to Day One for Treatment of Pediatric Low-Grade Glioma

Drug Development

July 27, 2021

Abeona Reports New Positive Data from Study of Gene Therapy for Sanfilippo Syndrome

Awareness

July 27, 2021

RareStone and Tencent Collaborate on Service Ecosystem Focused on Rare Disease Patients in China

Policy

July 27, 2021

Australian Government Provides $3.3 Million to Support Rare Disease Patients

Drug Development

July 26, 2021

European Commission Approves Rhythm’s Drug for Rare Obesity Disorders

Drug Development

July 26, 2021

Denali Reports Positive Interim Data from Phase 1/2 Hunter Syndrome Trial

RARECast Podcasts

July 23, 2021

Using Model Systems to Find Drugs to Repurpose for Rare Diseases

Rare Advocate

July 22, 2021

Rare Leader: Cyndi Frank, Co-President and Co-Founder, Gaucher Community Alliance

Finance

July 22, 2021

Hemab Raises $55 Million to Advance Therapeutics for Rare Bleeding Disorders

Healthcare

July 22, 2021

NHS England Unveils New Innovative Medicines Fund to Fast-Track Promising New Drugs

Drug Development

July 22, 2021

Spark Says Ongoing Phase 1/2 Hemophilia A Gene Therapy Data Suggests Durability

Research

July 22, 2021

Researchers Find Immune Component to Rare Neurodegenerative Disease

Drug Development

July 22, 2021

FDA Approves Octapharma’s Therapy for Adult Dermatomyositis

Deals

July 22, 2021

Parexel Partners with Rare Disease Foundation in China

Finance

July 21, 2021

Amylyx Raises $135 Million to Support Development and Potential Launch of ALS Treatment

Drug Development

July 21, 2021

European Commission Approves Bluebird Bio’s Gene Therapy for CALD

Drug Development

July 21, 2021

FDA Approves Albireo’s Bylvay as First Drug for PFIC

Deals

July 21, 2021

Lexeo Acquires Stelios to Expand Pipeline into Rare Cardiac Gene Therapies

Finance

July 21, 2021

Horama Raises $39 Million to Develop its Gene Therapy Pipeline and Changes Name to Coave Therapeutics

Drug Development

July 21, 2021

FDA Grants Breakthrough Therapy Designation to Genentech for Therapy to Treat Myelodysplastic Syndromes

Diagnosis

July 20, 2021

UW Opens Clinic for Patients with Undiagnosed Genetic Diseases

Policy

July 20, 2021

Netherland Fines Rare Disease Drugmaker for Excessive Price

Drug Development

July 19, 2021

Cytokinetics Reports Positive Results from Mid-State Trial of Hypertrophic Cardiomyopathy

Deals

July 19, 2021

GentiBio and Forge Biologics Form GMP Manufacturing Partnership

RARECast Podcasts

July 16, 2021

How a Small and Young Foundation Catalyzed Research into a Rare Disease

Research

July 16, 2021

NIH Funds New Effort to Discover Genetic Causes of Single-Gene Disorders T

Rare Advocate

July 15, 2021

Rare Leader: Chelsey McCarthy, Executive Director, DDX3X Foundation

Finance

July 15, 2021

Shape Therapeutics Raises $112 Million to Advance RNA Technology Platforms

Deals

July 15, 2021

Rady Children’s Institute for Genomic Medicine Enters Research Collaboration with Takeda

Deals

July 14, 2021

AstraZeneca Clears Final Hurdle for Acquisition of Alexion

Drug Development

July 14, 2021

FDA Grants Breakthrough Therapy Designation to Alkeus Stargardt Disease Therapy

Finance

July 13, 2021

Prime Medicine Launches with $315 Million to Deliver on the Promise of Prime Editing

Data Center

July 13, 2021

Rare Disease Drug Developers Bring in $13.7 Billion in New Capital in First Half of 2021

Research

July 13, 2021

Researchers Use Prenatal Editing in Animal Model to Correct Lysosomal Storage Disease

Deals

July 13, 2021

Novo Nordisk Acquires Prothena’s Antibody Targeting ATTR Amyloidosis

Strategy

July 13, 2021

Poxel Shifts Focus to Rare Metabolic Diseases

Drug Development

July 13, 2021

Gene Therapy Reverses Neurological Deficiencies in AADC

RARECast Podcasts

July 9, 2021

Overcoming the Limitations of Conventional Cell Engineering

Research

July 9, 2021

Browsable Resource Linking Rare Protein-Coding Genetic Variants to Human Health and Disease

Drug Development

July 9, 2021

FDA Places Clinical Hold on Sigilon’s Phase 1/2 Study in Hemophilia A

Drug Development

July 9, 2021

BlueBird Bio Resumes Marketing TDT Gene Therapy in Europe

Rare Advocate

July 8, 2021

Rare Leader: Deanna Fournier, Executive Director, Histiocytosis Association

Deals

July 8, 2021

BridgeBio Enters Collaborations with Three Academic Research Institutions

Deals

July 8, 2021

Lilly Enters Collaboration with Verge to Use Its AI to Discover and Develop New Treatments for...

Drug Development

July 8, 2021

FDA Grants Lysogene Fast Track Designation for GM1 Gangliosidosis Gene Therapy

Research

July 8, 2021

CMT Research Foundation Enters Partnership with University of Illinois Chicago

Research

July 7, 2021

Patient Groups Begin Using RARE-X’s Data Collection Platform

Drug Development

July 7, 2021

ChemoCentryx Addresses FDA Advisory Panel Questions in Amendment to Filing on Vasculitis Drug

Deals

July 6, 2021

EC Clears AstraZeneca’s Acquisition of Alexion

Deals

July 6, 2021

Orchard and Pharming Collaborate on Gene Therapy for HAE

Deals

July 6, 2021

Eureka and MSK License Novel Multiple Myeloma Target Domain to Sanofi

Drug Development

July 6, 2021

FDA Grants Priority Review to Levo for Prader-Willi Syndrome Therapy

Deals

July 6, 2021

Abbisko and Sperogenix Partner to Develop ABSK021 for ALS, Other Rare CNS Diseases in Greater China

RARECast Podcasts

July 2, 2021

Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care

Drug Development

July 2, 2021

Arrowhead Pauses RNAi Study in Cystic Fibrosis After Tox Study Signals

Deals

July 2, 2021

GSK Enters into Global Collaboration with Alector to Develop Antibodies for Neurodegenerative Diseases

Rare Advocate

July 1, 2021

Rare Leader: Diane Powell, CEO, The Hypersomnia Foundation

Deals

July 1, 2021

Apellis and Beam Collaborate to Apply Base Editing for Complement-Driven Diseases Drug Discovery

Drug Development

July 1, 2021

FDA Approves Jazz’s Rylaze as Component of Treatment Regimen for Most Common Childhood Cancer

Finance

June 30, 2021

Intellia Raises $600 Million to Advance Gene Editing Therapeutic Pipeline

Finance

June 30, 2021

Sofinnova Invests in Three New Rare Disease Gene Therapy Startups

Healthcare

June 30, 2021

NHS Creates First-of-Its-Kind Rare Disease Network in UK

Drug Development

June 30, 2021

FDA Grants Allogene Fast Track Designation for Multiple Myeloma Therapy

Drug Development

June 30, 2021

GenSight Reports Phase 3 Study Misses Endpoint, but Improves Vision in Patients with Degenerative Eye Disease

Finance

June 30, 2021

Aerovate Raises $121.5 Million in Upsized IPO to Advance PAH Therapy

Drug Development

June 30, 2021

FDA Grants Lexeo Therapeutics Rare Pediatric Disease Designation

Strategy

June 29, 2021

Orphazyme Lays Off Two-Thirds of Staff After FDA Setback

Research

June 29, 2021

Natural History Study Informs Potent Lifesaving Update to Standard of Care for PMM2-CDG Patients

Research

June 29, 2021

Pediatricians Find Cause of Muscle Breakdown in Rare Disease

Drug Development

June 28, 2021

Intellia and Regeneron Report First-Ever Data Supporting In Vivo CRISPR Editing in ATTR Patients

Drug Development

June 28, 2021

EU Approves Roche’s Enspryng as First At-home Subcutaneous Treatment for NMOSD

Drug Development

June 28, 2021

FDA Grants Fast Track Designation to Three Experimental Rare Disease Therapies

RARECast Podcasts

June 25, 2021

Forging Gene Therapy Capacity and a Pipeline at the Same Time

Finance

June 25, 2021

Graphite Bio Raises $238 Million in IPO to Advance Precision Gene Editing Pipeline

Legislation

June 25, 2021

Senators Introduce Legislation to Help Diagnose Children with Rare Diseases

Research

June 25, 2021

International Study of Rare Childhood Cancer Finds Genetic Clues, Potential for Tailored Therapy

Rare Advocate

June 24, 2021

Rare Leader: Kevin Mead, Executive Director of the International Pemphigus & Pemphigoid Foundation

Deals

June 24, 2021

AbbVie Exercises Right to Acquire TeneoOne and Lead Asset Targeting Multiple Myeloma

Deals

June 24, 2021

4D Molecular Reports Trial Data for Rare Eye Disorders and Termination of Roche Agreement

Research

June 24, 2021

Pacific Biosciences and Rady Children’s Collaborate on WGS Study of Rare Diseases

Drug Development

June 24, 2021

Corbus Reports Lenabasum Fails to Meet Primary Endpoint in Late-Stage Trial for Dermatomyositis

Drug Development

June 24, 2021

FDA Approves Santen’s Verkazia to Treat Vernal Keratoconjunctivitis in Children and Adults

Finance

June 23, 2021

Glycomine Raises $35 Million to Advance Novel Treatment for Rare Glycosylation Disease

Drug Development

June 23, 2021

UniQure Reports Positive 52-Week Data from Pivotal Trial of Hemophilia B Gene Therapy

Research

June 22, 2021

MIND Institute Gets $1.25 Million Grant for SYNGAP1 Syndrome

Finance

June 22, 2021

Protagonist Raises $132 Million to Advance Rare Blood Cancer Therapy

Finance

June 22, 2021

Neurona Raises $41.5 Million to Advance Cell Therapies for Epilepsy and Neurological Disorders

Deals

June 22, 2021

Neurelis Acquires Rights to Portfolio of Compounds Targeting Rare CNS Disorder

Deals

June 22, 2021

PerkinElmer to Acquire Viral Vector Gene Delivery Specialist Sirion Biotech

Deals

June 22, 2021

Jasper and Aruvant Collaborate on Treatment for Sickle Cell Disease

Research

June 22, 2021

New UAE Center will Diagnose and Treat Patients with Genetic Diseases

Drug Development

June 21, 2021

EC Expands Approval of Sanofi’s Oral MS Therapy to Include Children and Adolescents

Drug Development

June 21, 2021

Agios Seek Approval for Mitapivat for Rare Inherited Blood Disorder

RARECast Podcasts

June 18, 2021

Using A Natural DNA Repair Process to Improve Genetic Medicines

Drug Development

June 18, 2021

FDA Says It Won’t Approve Orphazyme’s NPC Therapy without Additional Data

Drug Development

June 18, 2021

FDA Grant Applied Therapeutics Fast Track Designation for Experimental Galactosemia Therapy

Drug Development

June 18, 2021

FDA Grants United Therapeutics Priority Review for PAH Drug

Deals

June 18, 2021

Perlara Enters Collaboration with Vivan for Multiple Rare Diseases

Rare Advocate

June 17, 2021

Rare Leader: Fallon Schultz, President and Founder, International FPIES Association

Finance

June 17, 2021

Gene Editing Therapeutics Developer Verve Completes $266.7 Million IPO

Deals

June 17, 2021

Horizon Therapeutics Buy Manufacturing Facility to Support Its Growth

Drug Development

June 16, 2021

FDA Expands Approval of Blueprint Medicines’ Ayvakit to Include Rare Blood Disorder

Uncategorized

June 16, 2021

CAMP4 Raises $45 Million for Programmable Therapeutics to Upregulate Genes

Deals

June 16, 2021

n-Lorem Foundation Forms Partnership with Manufacturer Oligonucleotide Nitto Avecia

Research

June 15, 2021

Researchers Use Base Editing to Turn Pathogenic Gene Benign in Mice with SCD

Drug Development

June 15, 2021

Biogen Choroideremia Gene Therapy Fails in Phase 3 Study

Deals

June 15, 2021

CRISPR Therapeutics and Capsida Enter Strategic Collaboration to Develop Gene-Edited Therapies for ALS, FA

Drug Development

June 15, 2021

Novartis Reports Positive Results for PNH Therapy

Research

June 15, 2021

Nanoparticle Therapy Shows Early Promise at Preventing a Rare, Fatal Newborn Lung Disease

Deals

June 14, 2021

Cure Rare Disease Enters Partnership with Curi Bio for DMD Gene Therapy

Drug Development

June 14, 2021

Study Shows Evrysdi Improved Motor Function in Pre-Symptomatic Babies

Strategy

June 14, 2021

CDMO Yposkesi Launches Construction of Advanced Therapy Facility Near Paris

Drug Development

June 14, 2021

FDA Grants Jasper Therapeutics Rare Pediatric Disease Designation for Conditioning Treatment Prior to Stem Cell Transplant

RARECast Podcasts

June 11, 2021

Empowering Rare Disease Patients with their Own Health Records

In Rare Form

June 11, 2021

A Mother’s Prosecution Alarms Rare Patient Advocates

Drug Development

June 11, 2021

Sanofi Presents Positive New Data for Sutimlimab for the Treatment of CAD

Drug Development

June 11, 2021

Vertex Achieves Primary Endpoint in AATD Study but Drops Program Due to Insufficient Clinical Benefit

Rare Advocate

June 10, 2021

Rare Leader: Kim Hollander, Executive Director, Oxalosis & Hyperoxaluria Foundation

Deals

June 10, 2021

Codexis and Takeda Expand Collaboration to Additional Gene Therapy for a Rare Genetic Disorder

Finance

June 10, 2021

Kurome Closes $15 Million Financing to Develop Targeted Kinase Inhibitors to Treat Rare Blood Cancers

Drug Development

June 10, 2021

New Data Point to Potential Long-Term Efficacy of Biogen’s Spinraza for SMA

Data Center

June 9, 2021

Rare Disease Therapeutics Developers Continued to Rake in Cash in May

Finance

June 9, 2021

Alcyone Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders

Drug Development

June 9, 2021

FDA Approves Trikafta in Children with Cystic Fibrosis Ages 6 through 11 with Certain Mutations

Finance

June 8, 2021

CyGenica Secures Seed Funding to Accelerate Cancer and Rare Genetic Disease Drug Delivery

Drug Development

June 8, 2021

FDA Approves Alexion’s Ultomiris for Children and Adolescents with PNH

Policy

June 8, 2021

Rare Disease Company Coalition Names Executive Committee

Drug Development

June 7, 2021

FDA Lifts Clinical Hold on Bluebird Bio’s Sickle Cell Disease and β-Thalassemia Studies

Drug Development

June 7, 2021

FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder

Drug Development

June 7, 2021

Novartis Reports Positive Interim Analysis from Phase 2 Study in Rare Kidney Disease

RARECast Podcasts

June 4, 2021

Targeting CNS Diseases with Gene Therapies

Drug Development

June 4, 2021

Nanoscope Gene Therapy Restores Vision in 11 Patients Blinded by Retinitis Pigmentosa

Rare Advocate

June 3, 2021

Rare Leader: Krista Vasi, Executive Director, Usher Syndrome Coalition

Strategy

June 3, 2021

Auris Medical Reboots to RNA Therapeutics with Acquisition of Trasir Therapeutics

Finance

June 3, 2021

Amyl Raises $22 million to Develop Novel Therapies for Amyloidosis

Finance

June 3, 2021

Stablix Therapeutics Launches with $63 Million to Target Protein Stabilization

Drug Development

June 3, 2021

AavantiBio and Resilience Form Strategic Collaboration for Gene Therapy Development and Manufacturing

Drug Development

June 3, 2021

FDA Grants Protagonist Breakthrough Therapy Designation for Rusfertide in PV

Drug Development

June 3, 2021

FDA Grants Priority Review to Amryt’s EB Drug

Deals

June 2, 2021

MorphoSys to Acquire Rare Cancer Drug Developer Constellation Pharma for $1.7 Billion

Finance

June 2, 2021

Transine $12.8 Million Seed Funding to Advance Novel Class of Therapeutic RNAs

Drug Development

June 2, 2021

Santhera and ReveraGen Report Positive Topline Results in Pivotal DMD Study

Research

June 2, 2021

Barth Syndrome Foundation and American Heart Association Collaborate to Advance ResearchRare Daily Staff

Research

June 2, 2021

Treatabolome Project Seeks to Shorten Diagnosis-to-Treatment Time for Rare Disease Patients

Drug Development

June 2, 2021

FDA Grants Breakthrough Therapy Designation to Janssen Off-the-Shelf Cell Therapy for Multiple Myeloma

Drug Development

June 1, 2021

FDA Approves QED and Helsinn’s Truseltiq to Treat Patients with Cholangiocarcinoma

Research

June 1, 2021

Scientists Discover a New Genetic Form of ALS in Children

Diagnosis

June 1, 2021

Solve-RD Collaboration Makes Case for Data Sharing and Periodic Reanalysis

Drug Development

June 1, 2021

FDA Grants Fast Track Designation to BridgeBio’s Encaleret for the Treatment of ADH1

Deals

June 1, 2021

Genomenon and Nostos Genomics Partner to Accelerate Rare Genetic Disease Diagnoses

Drug Development

May 27, 2021

A New Therapy Offers A Different Approach to Inhibiting the Complement System

Rare Advocate

May 27, 2021

Rare Leader: Kyle Dempsey, President, Superficial Siderosis Research Alliance

Drug Development

May 27, 2021

Eloxx and Metagenomi Receive Funding Awards from Cystic Fibrosis Foundation

Finance

May 27, 2021

Day One Raises $160 Million in IPO to Advance Genetically Defined Cancer Therapies

Drug Development

May 26, 2021

Larimar Says $95 Million Placement Cancelled After FDA Places Clinical Hold on Lead Program

Business

May 26, 2021

Penn’s Gene Therapy Program Becomes Anchor Tenant of Discovery Labs

Finance

May 26, 2021

Rgenta Therapeutics Completes Seed Financing, Raising $38 Million for RNA-Targeted Pipeline

Drug Development

May 26, 2021

FDA Asks Travere for Additional Data Ahead of Marketing Application for FSGS Therapy

Deals

May 26, 2021

BlueRock and Senti Bio Collaborate to Develop Engineered Cell Therapies for Regenerative Medicine

Drug Development

May 25, 2021

Apellis and Sobi Report Positive Results from Empaveli Study in Treatment Naïve Patients with PNH

Drug Development

May 25, 2021

FDA Grants Scholar Rock Fast Track Designation for SMA Therapy

Regulatory

May 25, 2021

FDA Approves First Therapy for Rare Form of NSCLC with Companion Diagnostic

Regulatory

May 24, 2021

Xeris to Acquire Strongbridge

Deals

May 24, 2021

Xeris to Acquire Strongbridge

Regulatory

May 24, 2021

Bipartisan Legislation Introduced to Advance Biomedical Research Stalled by Pandemic

Regulatory

May 24, 2021

CHMP Recommends Approval of Therapies to Treat the Rare Disorders PFIC and POMC

Drug Development

May 21, 2021

Bringing Aberrant Proteins Back into the Fold

Finance

May 21, 2021

Haya Therapeutics Raises $20 Million Seed Financing for RNA-based Fibrosis Therapies

Deals

May 21, 2021

Mallinckrodt Reaches Agreement to Sell Experimental NPC-1 Drug Adrabetadex to Mandos

Drug Development

May 21, 2021

European Regulators Give Bluebird Bio Positive Opinion for CALD Gene Therapy

Drug Development

May 21, 2021

Biogen and Ginkgo Bioworks Enter Collaboration to Create Novel Gene Therapy Manufacturing Platform

Drug Development

May 21, 2021

European Medicines Agency Grants PRIME Designation to Larimar’s Treatment for Friedreich’s Ataxia

Rare Advocate

May 20, 2021

Rare Leader: Trish Flanagan, co-founder and president, YBRP

Drug Development

May 20, 2021

Adaptimmune Reports Positive Initial Data from Mid-Stage Trial in Rare Cancer

Data Center

May 19, 2021

Investors Pile $2.2 Billion into Rare Disease Therapeutics Developers in April

Finance

May 19, 2021

Eloxx Raises $52 Million in Public Offering to Advance Rare Disease Pipeline

Deals

May 19, 2021

Ciitizen and RARE-X Form Collaboration

Finance

May 18, 2021

G2 Bio Companies Launch with $200 Million to Develop Transformative Genetic-Based Therapies

Finance

May 18, 2021

Gamma Biosciences Acquires Controlling Stake in Mirus Bio

Finance

May 18, 2021

Vedere Bio II Launches with $77 Million to Develop Next Generation Ocular Gene Therapies

Finance

May 18, 2021

Stealth Bio Raises $30 Million to Advance Elamipretide Clinical Trials

Finance

May 18, 2021

Interius Raises $76 Million for Cell and Gene Therapy Platform

Deals

May 17, 2021

Charles River to Acquire Gene Therapy CDMO Vigene Biosciences

Drug Development

May 17, 2021

FDA Approves Apellis’ Empaveli for Adults with Paroxysmal Nocturnal Hemoglobinuria

RARECast Podcasts

May 14, 2021

A Cat who Curates Rare Mice

Finance

May 14, 2021

Praxis Precision Medicines Raises $91.3 Million in Public Offering

Deals

May 14, 2021

PerkinElmer to Expand Cell and Gene Therapy and Biologics Manufacturing with $260 Million Nexcelom Acquisition

Finance

May 14, 2021

Vera Therapeutics Raises $48 Million in IPO

Drug Development

May 14, 2021

Biogen XLRP Gene Therapy Fails Late-Stage Study

Drug Development

May 14, 2021

FDA Grants BridgeBio Fast Track Designation for CAH Gene Therapy

Rare Advocate

May 13, 2021

Rare Leader: Julie Breneiser, Executive Director, Gorlin Syndrome Alliance

Finance

May 13, 2021

SpliSense Secures $28.5 Million in Series B Financing Including Backing from CFF

Finance

May 13, 2021

Flare Launches with $82 Million to Advance Novel Drug Discovery Approach for Transcription Factors

Finance

May 13, 2021

Magenta Raises $86.4 Million to Advance Novel Antibody-Drug Conjugate Conditioning Platform

Policy

May 13, 2021

Rare Disease Drug Developers Form Coalition to Address Challenges

Deals

May 13, 2021

CRO Emmes Acquires UK-Based Orphan Reach

Deals

May 12, 2021

MD Anderson and Broad Institute Launch Translational Research Platform Focused on Rare Cancers

Finance

May 12, 2021

Huma Raises $200 Million to Scale Digital Health Platform for Better Care and Research

Finance

May 12, 2021

Gennao Bio Closes $40 Million in Financing to Develop Pipeline of Targeted Nucleic Acid Therapeutics

Drug Development

May 12, 2021

FDA Grants Fulcrum Fast Track for FSHD Drug

Finance

May 12, 2021

Xontogeny Seeds NephroDI to Develop a Treatment for Nephrogenic Diabetes Insipidus

Research

May 11, 2021

Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency

Deals

May 11, 2021

Biogen Enters Collaboration with Capsigen for AAV Capsids for CNS and Neuromuscular Disorders

Research

May 11, 2021

Mutations Cause Rare Genetic Disease in Children

Uncategorized

May 11, 2021

FDA Halts Rocket Pharma Trial of Experimental Gene Therapy for Danon Disease

Drug Development

May 10, 2021

Laronde Unveiled to Advance New Class of Programmable Medicines

Drug Development

May 10, 2021

Ionis Stops Development of Inhaled Antisense Therapy for CF

Drug Development

May 10, 2021

FDA Grants Fast-Track Designation to Smart Immune’s T Cell Immunity Enhancement Product

RARECast Podcasts

May 7, 2021

Putting the Patient at the Center of Rare Disease Clinical Trials

Finance

May 7, 2021

Talaris Raises $150 Million in IPO to Advance Cell Therapy Pipeline

Finance

May 7, 2021

Science 37 to Become Publicly Listed via Merger with SPAC

Drug Development

May 7, 2021

ChemoCentryx Continues Slide on Divided FDA Advisory Committee Votes

Drug Development

May 7, 2021

Orphazyme Drug Fails in Pivotal Study

Rare Advocate

May 6, 2021

Rare Leader: Tiffany House, President Acid Maltase Deficiency Association

Finance

May 6, 2021

Dyno Therapeutics Raises $100 Million to Accelerate AI‑powered Gene Therapy Platform

Finance

May 6, 2021

TwinStrand Raises $50 Million to Grow Market for Duplex Sequencing Technology

Deals

May 5, 2021

Amryt Acquires Chiasma, Strengthening Its Rare and Orphan Disease Portfolio

Research

May 5, 2021

Centogene Discovers Six New Rare Diseases

Drug Development

May 5, 2021

ChemoCentryx Shares Crash After FDA Questions Data Supporting ANCA Vasculitis Therapy

Research

May 5, 2021

Study Finds People with Rare Diseases Often Feel Stigmatized

Drug Development

May 4, 2021

Sarepta Reports High-Dose SRP-5051 Increases Dystrophin in Certain DMD Patients

Finance

May 4, 2021

Mogrify Raises $17 Million to Advance Reprogrammed Cell Therapies

Deals

May 4, 2021

Passage Bio Partners with InformedDNA to Offer Genetic Counseling and Testing for FTD

Regulatory

May 4, 2021

Acella Issues Voluntary Nationwide Recall of Certain Lots of Thyroid Tablets Due to Sub Potency

Finance

May 3, 2021

Affinia Raises $110 Million to Advance Engineered AAV Gene Therapy Vector Platform

Finance

May 3, 2021

Aceragen Launches with $35 Million and Experimental Therapy for Farber Disease

Drug Development

May 3, 2021

With Accelerated Approval Off the Table, Avrobio Seeks Registration Trial for Fabry Disease Gene Therapy

Drug Development

May 3, 2021

Alnylam Reports Positive Early Results of Phase 3 PH1 Study

Drug Development

May 3, 2021

FDA Expands Approval of Chiesi’s Ferriprox for Transfusional Iron Overload due to Sickle Cell Disease

RARECast Podcasts

April 30, 2021

Creating a Toolkit to Accelerate the Development of Gene Editing Therapies

Rare Advocate

April 29, 2021

Rare Leader: Nancy Lazarus, Founder, Achalasia Awareness Organization

Finance

April 29, 2021

Capsida Debuts with $140 Million to Advance Next Generation Gene Therapies

Deals

April 29, 2021

Mirum Grants CANbridge Exclusive License to Maralixibat in Greater China for Rare Liver Diseases

Drug Development

April 29, 2021

FDA Grants Fast Track to Akari Bullous Pemphigoid Therapy

Drug Development

April 29, 2021

Astellas Takes $532 Million Charge Over Delay of Gene Therapy for XLMTM

Deals

April 28, 2021

Allen Institute and BioMarin Collaborate to Develop Gene Therapies for Rare Brain Diseases

Drug Development

April 28, 2021

FDA Tell Protalix and Chiesi It won’t Grant Accelerated Approval for Fabry Therapy

Deals

April 27, 2021

LogicBio Enters Gene-Editing Collaborations with CANBridge and Daiichi Sankyo

Deals

April 27, 2021

AllStripes Collaborates with NIH’s NCATS to Launch Effort in Rare Disease Diagnosis

Drug Development

April 27, 2021

FDA Removes Clinical Hold on Voyager, Clears IND for Huntington’s Gene Therapy

Drug Development

April 27, 2021

Biogen Will Provide Early Access to Experimental ALS Drug

Drug Development

April 27, 2021

EMA Grants PRIME Designation to Vertex and CRISPR’s Therapy for Transfusion-Dependent Beta Thalassemia

Drug Development

April 27, 2021

AavantiBio Partners with Catalent to Advance Gene Therapies for Rare Genetic Diseases

Regulatory

April 27, 2021

FDA Issues Guidance on Individualized ASOs for Ultra-Rare Conditions

Drug Development

April 26, 2021

FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program

Drug Development

April 26, 2021

FDA Approves ADC Therapeutics’ Zynlonta to Treat Rare Cancer

RARECast Podcasts

April 23, 2021

PTC Looks to Advanced Therapies

Deals

April 23, 2021

CDISC and NORD to Develop Data Standards for Rare Disease

Rare Advocate

April 22, 2021

Rare Leader: Heidi Edwards, President and Founder, Sister’s Hope Foundation

Deals

April 22, 2021

Vertex and Obsidian Enter into Collaboration to Discover Novel Therapies that Regulate Gene Editing

Finance

April 22, 2021

ITM raises $109 Million to Advance Radiopharmaceutical Precision Oncology Pipeline

Finance

April 21, 2021

Code Biotherapeutics Launches with $10 Million to Develop Therapies for Debilitating Genetic Diseases

Finance

April 21, 2021

EdiGene Raises $62 Million to Advance and Scale Up Clinical Translation of Gene Editing Technologies

Drug Development

April 21, 2021

Beam Publishes Data Demonstrating Ability to Rationally Design Base Editors for Precise Editing

Pricing

April 21, 2021

Bluebird Withdraws Zynteglo from German Market, Cuts Workforce

Drug Development

April 21, 2021

EC Grants Approval to GW Pharmaceuticals’ Epidyolex for TSC-Associated Seizures

Uncategorized

April 21, 2021

FDA Grants RMAT Designation to Allogene’s Cell Therapy for Multiple Myeloma

Research

April 20, 2021

The Making of a Gene Therapy for an Ultra-Rare Disease

Drug Development

April 20, 2021

FDA Puts Clinical Hold on KalVista’s HAE Therapy

Deals

April 20, 2021

Citrine and Sinopharm Enter Strategic Partnership to Broaden Access to Rare Disease Drugs in China

Deals

April 20, 2021

Vertex and CRISPR Amend Sickle Cell Disease and Beta Thalassemia Collaboration

Research

April 20, 2021

IndoUSrare and Rare-X Conduct Feasibility Study for a Patient-Owned Health Data Collection Program for India

Business

April 20, 2021

Castle Creek Biosciences Appoints Matthew Gantz as President and CEO

Finance

April 19, 2021

Rezolute Enters $30 Million Debt Agreement to Advance Rare and Metabolic Disease Pipeline

Drug Development

April 19, 2021

Study Finds Positive Impact of Fintepla on Dravet Patients and Caregivers

Drug Development

April 19, 2021

Biogen Reports New Spinraza Data in SMA

RARECast Podcasts

April 16, 2021

Going to Extreme Lengths

Research

April 16, 2021

Study Identifies Key Target in Treatment-Resistant Hemophilia A

Finance

April 16, 2021

Recursion Raises $436 Million in Upsized IPO to Advance Rare Disease Pipeline

Rare Advocate

April 15, 2021

Lauren Dunlap, Co-Founder and Executive Director, AAIDA

Finance

April 15, 2021

Medable Raises $78 Million to Decentralize Clinical Trials

Finance

April 15, 2021

Alchemab Raises $82 Million to Advance Antibody Therapeutics in Neurodegeneration and Cancer

Deals

April 15, 2021

Ciitizen Partners with STXBP1 Foundation to Accelerate Patient Outcomes

Business

April 15, 2021

DOJ Subpoenas Documents from Alnylam Over Marketing of Onpattro

Drug Development

April 15, 2021

FDA Grants Rare Pediatric Disease Designation to FibroGen Treatment for DMD

Drug Development

April 15, 2021

New Evrysdi Data Shows Improvement in Motor Function Over Time for SMA Patients

Finance

April 14, 2021

Investment in Rare Disease Drug Development Continues to Surge

Research

April 14, 2021

Gene Therapy Shows Promise in Treating Rare Eye Disease in Mice

Deals

April 14, 2021

Huma Enters Partnership with RARE-X to Accelerate Rare Disease Research

Drug Development

April 14, 2021

FDA Grants Rare Pediatric Disease Designation to Therapies from Moleculin and Akuous

Deals

April 13, 2021

Senti Bio and Spark Collaborate to Develop Next-Generation Precision Gene Therapies

Deals

April 13, 2021

BridgeBio Pharma Enters Seven Collaborations with University Partners

Finance

April 13, 2021