Articles

RARECast Podcasts
September 24, 2021
Creating a Playbook for Bespoke Gene Therapies
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Drug Development
September 24, 2021
Long-Term Treatment with Crysvita Reduces the Burden of Disease in Adults With XLH
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Drug Development
September 24, 2021
AGTC Reports Serious Adverse Events in Three Patients
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Drug Development
September 24, 2021
Capricor Reports Positive Final Data From its Phase 2 DMD Trial of Experimental Cell Therapy
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Drug Development
September 24, 2021
Roche Presents New Data Supporting Safety and Efficacy of its Neuromuscular Therapies
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Research
September 24, 2021
CIIC and Trio Health Introduce the Creation of a Dynamic Rare Disease Patient Registry Platform
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Rare Advocate
September 23, 2021
Rare Leader: Shari Hume, Co-Founder, Cure JM Foundation
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Finance
September 23, 2021
Garuda Therapeutics Launches with $72 Million Series A Financing
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Finance
September 23, 2021
Synlogic Raises $45 Million in Public Offering to Advance Treatments for Metabolic Disorders
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Finance
September 23, 2021
GenEdit Raises $26 Million to Support Nanoparticle Delivery Platform for Genetic Medicines
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Drug Development
September 23, 2021
FDA Expands Use of Incyte’s Jakafi to Include GVHD
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Finance
September 22, 2021
Opus Genetics Launches with $19 Million to Advance Gene Therapies for Retinal Diseases
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Drug Development
September 22, 2021
Rhythm Presents New Data for Setmelanotide in Multiple Rare Genetic Diseases of Obesity
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Regulatory
September 21, 2021
FDA Should Look to Past Precedents When Applying Flexibility to Approvals to Maintain Public Trust, Study...
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Deals
September 21, 2021
Novartis Acquires Arctos Medical, Expanding Portfolio of Gene Therapies for Rare Eye Disorders
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Deals
September 21, 2021
Mirum and Takeda Partner to Develop and Commercialize Rare Pediatric Liver Disease Drug in Japan
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Drug Development
September 21, 2021
Sunitinib Prolongs Progression Free Survival in Rare Neuroendocrine Tumors
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Diagnosis
September 21, 2021
Horizon Awards First Horizon Prize to ThinkGenetic, Congenica, for Work to Accelerate Diagnosis of Rare Diseases
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Finance
September 20, 2021
Rare Disease Dealmaking Heats Up in August
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Diagnosis
September 20, 2021
Genomenon and Alexion Collaboration to Accelerate Genetic Diagnosis for Rare Disease Patients
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RARECast Podcasts
September 17, 2021
Finding Answers for Undiagnosed Patients with Rare Genetic Diseases
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Drug Development
September 17, 2021
FDA Places Hold on Protagonist’s Blood Disorders Program after Skin Tumors Observed in Mouse Study
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Uncategorized
September 16, 2021
Rare Leader: Geoff Rhyne, Co-Founder and CEO, IDefine
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Deals
September 16, 2021
Travere Licenses Sparsentan to Vifor Pharma for Commercialization in Europe, Australia, and New Zealand
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Finance
September 16, 2021
Amolyt Raises $80 Million to Advance Rare Endocrine Pipeline
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Finance
September 16, 2021
Anjarium Raises $61 Million to Develop Next-Generation Non-viral Gene Therapies
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Drug Development
September 16, 2021
FDA Approves Takeda’s Exkivity for Subset of NSCLC
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Finance
September 15, 2021
Avalo Raises $27.5 Million to Advance Pipeline of Cancer and Rare Genetic Disease Therapies
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Drug Development
September 15, 2021
Rarebase Launches a Neuroscience Drug Discovery Platform Collaborating with 15 Rare Disease Patient Groups
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Drug Development
September 15, 2021
FDA Grants Fast Track Designation to BridgeBio’s Experimental Therapy for LGMD2i
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Drug Development
September 15, 2021
FDA Grants Fast Track Designation to Sage for Huntington’s Disease Therapy
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Drug Development
September 15, 2021
FDA Extends Action Date for Calliditas Nefecon
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Uncategorized
September 15, 2021
FDA Lifts Clinical Hold on KalVista HAE Therapy
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Finance
September 14, 2021
Skyhawk Raises $133 Million to Advance its RNA-Modifying Candidates into the Clinic
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Finance
September 14, 2021
Vanqua Bio Launches with $85 Million to Target Neurodegenerative Diseases
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Finance
September 14, 2021
Walking Fish Launches with $50 Million to Advance B Cell Therapies for Multiple Diseases
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Drug Development
September 14, 2021
Astellas Reports Fourth Patient Dies in XLMTM Gene Therapy Trial
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Legislation
September 13, 2021
NORD Warns Provision in Biden Administration’s Ambitious Legislation Would Cut Benefits of Orphan Drug Tax Credit
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Deals
September 13, 2021
RARE-X and C-Path Establish New Collaboration for Rare Disease Data Sharing
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Drug Development
September 13, 2021
Chiesi Global Rare Diseases Wins Approval in Brazil for Treatment of Iron Overload in SCD
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RARECast Podcasts
September 10, 2021
How a Familiar Face Can Lead to a Rare Disease Diagnosis
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Research
September 10, 2021
Research Collaboration Seeks to Accelerate Rare Disease Diagnostics
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Drug Development
September 10, 2021
FDA Grants Decibel Rare Pediatric Disease Designations for Congenital Hearing Loss Therapy
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Drug Development
September 10, 2021
Sanofi Experimental Therapy for Rare Skin Condition Fails in Late-Stage Study
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Rare Advocate
September 9, 2021
Rare Leader: Cyndi Roemer, Board Chair, The MSA Coalition
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Finance
September 9, 2021
Mammoth Raises $195 Million to Build Next-Generation CRISPR Therapeutics and Diagnostics
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Finance
September 9, 2021
iECURE Launches With $50 Million to Develop In Vivo Gene Insertion
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Finance
September 9, 2021
LEXEO Therapeutics Closes $100 Million to Advance Gene Therapy Pipeline
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Deals
September 8, 2021
Albireo Sells Priority Review Voucher (PRV) for $105 Million
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Finance
September 8, 2021
ADARx Raises $75 Million to Advance Pipeline of RNA Targeting Therapeutics
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Finance
September 8, 2021
Attralus Raises $116 Million to Advance Pan-Amyloid Removal Therapeutics
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Finance
September 8, 2021
Bluebird Bio Secures $75 Million Ahead of Separation into Two Companies
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Deals
September 8, 2021
Sanofi to Acquire Kadmon for $1.9 Billion, Gaining Approved Therapy for cGVHD
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Strategy
September 8, 2021
Catabasis Takes New Name to Highlight New Focus
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Deals
September 7, 2021
Invitae to Acquire Ciitizen for $325 Million
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Drug Development
September 7, 2021
FDA Places Clinical Hold on BioMarin’s Gene Therapy Study in Adults with PKU
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Deals
September 7, 2021
Moderna and ILCM to Collaborate to Develop mRNA Therapeutic for Crigler-Najjar Syndrome Type 1
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Deals
September 7, 2021
NeuExcell Therapeutics Enters Research Collaboration with Spark for Huntington’s Disease Gene Therapy
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Drug Development
September 7, 2021
EU Expands Approval of Ultomiris to Include Children and Adolescents with PNH
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RARECast Podcasts
September 3, 2021
Bringing the Trial to the Patient
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Deals
September 3, 2021
SOBI Board of Directors Recommends $8.1 Billion Takeover by Private Equity Consortium
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Rare Advocate
September 2, 2021
Rare Leader: Earl Cole, Founder and Chairman, Perthes Kids Foundation
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Finance
September 2, 2021
Disc Medicine Raises $90 Million to Advance Therapies for Hematologic Diseases
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Research
September 2, 2021
Researchers Develop Test to Determine Patients with Rare Blood Disorder Likely to Benefit from Existing Therapy
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Deals
September 2, 2021
Polyphor and EnBiotix Sign Merger Agreement and sale of Inhaled Murepavadin to EnBiotix
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Drug Development
September 2, 2021
Takeda’s Breakthrough Therapy for Rare Cancer Fails in Phase 3
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Drug Development
September 2, 2021
FDA Approves Beigene’s Brukinsa to Treat Waldenström’s Macroglobulinemia
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Research
September 1, 2021
CZI Award $3 Million to Researchers to Build Pediatric Cell Atlas of Skeletal Muscle
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Drug Development
September 1, 2021
Astellas Voluntarily Halts X-linked Myotubular Myopathy Gene Therapy Trial
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Deals
August 31, 2021
VectivBio Acquires Comet, Expanding Rare Metabolic Disease Pipeline
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Finance
August 30, 2021
Laronde Raises $440 Million to Further Advance New Class of Programmable Medicines
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RARECast Podcasts
August 27, 2021
Targeting Rare and Chronic Kidney Diseases
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Drug Development
August 27, 2021
BioMarin’s Voxzogo Approved in Europe to Treat Children with Achondroplasia
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Uncategorized
August 27, 2021
Earlier Diagnosis Can Reduce Cost of Raising a Child with a Rare Genetic Disorder
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Drug Development
August 27, 2021
EMA Grants Marinus Accelerated Assessment of Ganaxolone for Treatment of CDKL5 Deficiency Disorder
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Drug Development
August 27, 2021
FDA Approves Expanded Use of Servier’s Tibsovo to Include Rare Bile Duct Cancer
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Rare Advocate
August 26, 2021
Rare Leader: Casey Gorman, Executive Director, PRISMS (Patients and Researchers Interested in Smith-Magenis Syndrome)
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Uncategorized
August 26, 2021
Alexion Reports Positive Late-Stage Results for Experimental Wilson Disease Therapeutic
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Drug Development
August 26, 2021
FDA Approves Ascendis Pharma’s Skytrofa for Pediatric Growth Hormone Deficiency
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Drug Development
August 25, 2021
Stealth Seeks FDA Approval for Barth Syndrome Treatment, Despite Call for More Data
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Research
August 24, 2021
As FDA Readies for PDUFA VII, Rare Disease, Cell and Gene Therapies in the Mix
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Finance
August 24, 2021
AllStripes Raises $50 Million to Advance Global Rare Disease Research
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Deals
August 24, 2021
Vertex and Arbor Partner to Develop Novel Ex Vivo Engineered Cell Therapies
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Deals
August 24, 2021
Shape and Roche Collaborate to advance AAV-based RNA Editing for Neuroscience and Rare Diseases
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Deals
August 23, 2021
Pfizer to Acquire Trillium For $2.3 Billion to Beef Up Rare Blood Cancer Pipeline
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Deals
August 23, 2021
Genevant and Takeda Partner to Develop Nonviral Rare Liver Disease Gene Therapies
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Research
August 23, 2021
Researchers Uncover Cause of Rare Bone Disorder
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RARECast Podcasts
August 20, 2021
Addressing the Delivery Challenges of Genetic Medicines
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Drug Development
August 20, 2021
Alexion Discontinues Late-Stage ALS Trial of Ultomiris Due to Lack of Efficacy
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Uncategorized
August 20, 2021
Discovery of the Mechanism for A Rare Autoinflammatory Disease Brings Repurposed Treatment
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Rare Advocate
August 19, 2021
Rare Leader: Amber Olsen, Executive Director, United MSD Foundation
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Uncategorized
August 19, 2021
FDA Grants Fast Track Designation to Senhwa’s Silmitasertib to Treat Recurrent SHH Medulloblastoma
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Drug Development
August 19, 2021
NICE Recommends First Treatment for Rare Blood Disorder
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Finance
August 18, 2021
Jnana Therapeutics Raises $50 Million to Advance Lead PKU Program and Pipeline
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Finance
August 18, 2021
Vigil Raises $90 Million to Advance Pipeline to Treat Neurodegenerative Diseases
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In Rare Form
August 18, 2021
Why Becoming a Rare Disease Expert Isn’t Enough for Parents
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Drug Development
August 18, 2021
Marinus Reports Positive Topline Ganaxolone Phase 2 Results in Tuberous Sclerosis Complex
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Deals
August 17, 2021
Aurinia Acquires Novel Pipeline Assets Targeting Autoimmune and Kidney-Related Diseases
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Drug Development
August 16, 2021
Merck Wins Approval for Treatment for Von Hippel-Lindau Disease-Associated Tumors
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Drug Development
August 16, 2021
FDA Lifts Clinical Hold on Rocket Pharma’s Gene Therapy Trial for Danon Disease
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Finance
August 16, 2021
Beckley Psytech Raises $80 Million to Develop Portfolio of Psychedelic Medicines
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Drug Development
August 16, 2021
Travere Reports Positive Topline Interim Results from Phase 3 Study of Sparsentan in IgAN
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Drug Development
August 16, 2021
FDA Grants Edgewise Fast Track Designation for Becker Muscular Dystrophy Therapy
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RARECast Podcasts
August 13, 2021
Targeting Tissues Throughout the Body with RNA Therapies
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Drug Development
August 13, 2021
FDA Grants First of its Kind Indication for Chronic Sleep Disorder Treatment
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Uncategorized
August 13, 2021
Ipsen Withdraws Application Seeking Approval for Rare Bone Disease Therapy
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Drug Development
August 13, 2021
FDA Grants Fast Track Designation to Vivet’s Therapy for the Treatment of Wilson Disease
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Uncategorized
August 13, 2021
FDA Grants Breakthrough Therapy Designation to Junshi’ and Coherus’ Treatment for Nasopharyngeal Carcinoma
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Rare Advocate
August 12, 2021
Rare Leader, Jennifer Martin, President, Helping Hands for GAND
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Finance
August 12, 2021
Fulcrum Raises $125 Million in Public Offering to Advance Rare Disease Pipeline
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Drug Development
August 12, 2021
CRO MMS Launches Pro-Bono Regulatory Assistance Program for Ultra-Rare Disease Patient Advocacy Groups
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Finance
August 11, 2021
Harmony Biosciences Enters into Strategic Financing Collaboration with Blackstone
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Drug Development
August 11, 2021
First Intravenous Infusion Gene Therapy for Rare Disease in China Cleared for Clinical Testing
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Finance
August 10, 2021
Money Continues to Flow into Rare Disease Drug Development
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Deals
August 10, 2021
Healx Enters Partnership with Ono Pharmaceutical to Repurpose Drugs for Rare Diseases
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Deals
August 10, 2021
VGXI Partners with Rare Trait Hope Fund in Support of Gene Therapy for Rare Disease AGU
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Drug Development
August 9, 2021
FDA Places Clinical Hold on Bluebird Bio CALD Candidate for Safety Concerns
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Drug Development
August 9, 2021
Liminal BioSciences to Sell Priority Review Voucher for $105 Million
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Deals
August 9, 2021
Epizyme and Hutchmed Form Strategic Collaboration to Develop and Commercialize Tazverik in Greater China
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Drug Development
August 9, 2021
FDA Approves Sanofi Genzyme’s New Treatment for Pompe Disease
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Finance
August 9, 2021
Sierra Oncology Updates Timeline for Momelotinib in Myelofibrosis and Raises $34 Million
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Drug Development
August 9, 2021
Harmony Biosciences Adds Novel Asset to Rare Neurological Disease Pipeline
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RARECast Podcasts
August 6, 2021
Delivering Rare Disease Therapies to Patients in Need
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Deals
August 6, 2021
Sierra Oncology Licenses AstraZeneca BET Inhibitor to Expand Myelofibrosis Pipeline
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Drug Development
August 6, 2021
Dicerna Reports Positive Results from Pivotal Trial of Experimental RNAi Therapy for Primary Hyperoxaluria
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Rare Advocate
August 5, 2021
Rare Leader: Stacy Pike-Langenfeld, Co-Founder and President, KrabbeConnect
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Finance
August 5, 2021
PepGen Raises $112.5 Million to Advance Transformative Therapies for Neuromuscular Diseases
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Finance
August 5, 2021
Aardvark Raises $29 Million to Fund Trials of Therapeutic for Prader-Willi Syndrome
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Deals
August 5, 2021
Sarepta Enters Licensing Agreement for Nationwide Children’s Gene Therapy Program for Limb-Girdle Muscular Dystrophy
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Drug Development
August 5, 2021
FDA Grants Breakthrough Therapy Designation to Eiger’s Avexitide for Congenital Hyperinsulinism
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Policy
August 4, 2021
Industry Group Respond to Proposals that Could Undermine Accelerated Approval
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Finance
August 4, 2021
Avidity Biosciences Raises $144 Million to Support RNA Therapeutic Rare Disease Pipeline
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Research
August 4, 2021
Rare Disorder Offers Roadmap for Understanding Roots of Inflammatory Disease
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Deals
August 3, 2021
Sanofi Takes Major Step into mRNA with Acquisition of Translate Bio for $3.2 Billion
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Deals
August 3, 2021
Marinus Enters Collaboration with Orion for European Commercialization of Rare Disease Drug
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Drug Development
August 3, 2021
FDA Clears Novartis to Start Phase 3 Study of Intrathecal Zolgensma in Older Patients with SMA
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Drug Development
August 3, 2021
FDA Issues Complete Response Letter Received for Medac’s Allo-HSCT Preparative Regimen Treosulfan
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Deals
August 2, 2021
Ipsen and Exicure Enter Exclusive Collaboration Targeting Rare Neurodegenerative Disorders
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Finance
August 2, 2021
Immunovant Receives $200 Million Strategic Investment from Roivant to Advance Pipeline
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Drug Development
August 2, 2021
EU Expands Approval for Amicus’s Fabry Disease Therapy to Adolescents
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Drug Development
August 2, 2021
FDA Approves J&J’s Uptravi for Intravenous Use in Adult Patients with PAH
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RARECast Podcasts
July 30, 2021
An Advocate’s Journey through Diagnosis, Loss, and Hope
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Finance
July 30, 2021
Tenaya Raises $180 Million in Upsized IPO to Address Underlying Causes of Heart Disease
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Rare Advocate
July 29, 2021
Rare Leader: Kimberly Ventarola, Co-Founder and President, Malan Syndrome Foundation
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Finance
July 29, 2021
Rallybio Raises $81 Million in IPO to Advance Therapies for Rare Diseases
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Finance
July 29, 2021
Crinetics Raises $15 Million from Frazier Healthcare Partners
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Deals
July 29, 2021
Rhythm Signs Research Agreement with Clinical Registry Investigating Bardet-Biedl Syndrome
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Drug Development
July 29, 2021
Alnylam Reports Positive Results from Phase 3 Study of Lumasiran in Advanced Primary Hyperoxaluria Type 1
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Drug Development
July 29, 2021
FDA Grants Breakthrough Therapy Designation to Takeda’s Experimental Narcolepsy Drug
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Drug Development
July 29, 2021
Alector Presents Positive Phase 2 Results of Experimental Therapy in FTD
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Uncategorized
July 29, 2021
FDA Grants Breakthrough Therapy Designation to Arrowhead’s Experimental Treatment for AATD
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Finance
July 28, 2021
Deep Genomics Raises $180 Million to Advance Programmable RNA Therapeutics
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Deals
July 28, 2021
Aristea Enters into Collaboration with Arena in Neutrophil-Mediated Diseases and Raises $63 Million
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Deals
July 28, 2021
Bluebird Bio Forms Strategic Alliance with Resilience to Develop Next Generation Cell Therapies
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Finance
July 28, 2021
Ring Raises $117 Million to Advance Vector Platform to Unlock Full Potential of Gene Therapy
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Drug Development
July 28, 2021
FDA Grants Fast Track Designation to Apic Bio’s APB-102 for the Treatment of Patients with SOD1...
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Drug Development
July 28, 2021
FDA Grants Rare Pediatric Disease Designation to Lexeo Therapeutics CLN2 Batten Disease Therapy
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Research
July 27, 2021
Study Links Autism to New Set of Rare Gene Variants
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People & Organizations
July 27, 2021
RARE-X Appoints Charlene Son Rigby as Chief Executive Officer
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Drug Development
July 27, 2021
FDA Grants Rare Pediatric Disease Designation to Day One for Treatment of Pediatric Low-Grade Glioma
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Drug Development
July 27, 2021
Abeona Reports New Positive Data from Study of Gene Therapy for Sanfilippo Syndrome
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Awareness
July 27, 2021
RareStone and Tencent Collaborate on Service Ecosystem Focused on Rare Disease Patients in China
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Policy
July 27, 2021
Australian Government Provides $3.3 Million to Support Rare Disease Patients
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Drug Development
July 26, 2021
European Commission Approves Rhythm’s Drug for Rare Obesity Disorders
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Drug Development
July 26, 2021
Denali Reports Positive Interim Data from Phase 1/2 Hunter Syndrome Trial
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RARECast Podcasts
July 23, 2021
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
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Rare Advocate
July 22, 2021
Rare Leader: Cyndi Frank, Co-President and Co-Founder, Gaucher Community Alliance
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Finance
July 22, 2021
Hemab Raises $55 Million to Advance Therapeutics for Rare Bleeding Disorders
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Healthcare
July 22, 2021
NHS England Unveils New Innovative Medicines Fund to Fast-Track Promising New Drugs
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Drug Development
July 22, 2021
Spark Says Ongoing Phase 1/2 Hemophilia A Gene Therapy Data Suggests Durability
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Research
July 22, 2021
Researchers Find Immune Component to Rare Neurodegenerative Disease
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Drug Development
July 22, 2021
FDA Approves Octapharma’s Therapy for Adult Dermatomyositis
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Deals
July 22, 2021
Parexel Partners with Rare Disease Foundation in China
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Finance
July 21, 2021
Amylyx Raises $135 Million to Support Development and Potential Launch of ALS Treatment
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Drug Development
July 21, 2021
European Commission Approves Bluebird Bio’s Gene Therapy for CALD
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Drug Development
July 21, 2021
FDA Approves Albireo’s Bylvay as First Drug for PFIC
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Deals
July 21, 2021
Lexeo Acquires Stelios to Expand Pipeline into Rare Cardiac Gene Therapies
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Finance
July 21, 2021
Horama Raises $39 Million to Develop its Gene Therapy Pipeline and Changes Name to Coave Therapeutics
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Drug Development
July 21, 2021
FDA Grants Breakthrough Therapy Designation to Genentech for Therapy to Treat Myelodysplastic Syndromes
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Diagnosis
July 20, 2021
UW Opens Clinic for Patients with Undiagnosed Genetic Diseases
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Policy
July 20, 2021
Netherland Fines Rare Disease Drugmaker for Excessive Price
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Drug Development
July 19, 2021
Cytokinetics Reports Positive Results from Mid-State Trial of Hypertrophic Cardiomyopathy
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Deals
July 19, 2021
GentiBio and Forge Biologics Form GMP Manufacturing Partnership
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RARECast Podcasts
July 16, 2021
How a Small and Young Foundation Catalyzed Research into a Rare Disease
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Research
July 16, 2021
NIH Funds New Effort to Discover Genetic Causes of Single-Gene Disorders T
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Rare Advocate
July 15, 2021
Rare Leader: Chelsey McCarthy, Executive Director, DDX3X Foundation
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Finance
July 15, 2021
Shape Therapeutics Raises $112 Million to Advance RNA Technology Platforms
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Deals
July 15, 2021
Rady Children’s Institute for Genomic Medicine Enters Research Collaboration with Takeda
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Deals
July 14, 2021
AstraZeneca Clears Final Hurdle for Acquisition of Alexion
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Drug Development
July 14, 2021
FDA Grants Breakthrough Therapy Designation to Alkeus Stargardt Disease Therapy
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Finance
July 13, 2021
Prime Medicine Launches with $315 Million to Deliver on the Promise of Prime Editing
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Data Center
July 13, 2021
Rare Disease Drug Developers Bring in $13.7 Billion in New Capital in First Half of 2021
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Research
July 13, 2021
Researchers Use Prenatal Editing in Animal Model to Correct Lysosomal Storage Disease
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Deals
July 13, 2021
Novo Nordisk Acquires Prothena’s Antibody Targeting ATTR Amyloidosis  
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Strategy
July 13, 2021
Poxel Shifts Focus to Rare Metabolic Diseases
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Drug Development
July 13, 2021
Gene Therapy Reverses Neurological Deficiencies in AADC
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RARECast Podcasts
July 9, 2021
Overcoming the Limitations of Conventional Cell Engineering
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Research
July 9, 2021
Browsable Resource Linking Rare Protein-Coding Genetic Variants to Human Health and Disease
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Drug Development
July 9, 2021
FDA Places Clinical Hold on Sigilon’s Phase 1/2 Study in Hemophilia A
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Drug Development
July 9, 2021
BlueBird Bio Resumes Marketing TDT Gene Therapy in Europe
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Rare Advocate
July 8, 2021
Rare Leader: Deanna Fournier, Executive Director, Histiocytosis Association
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Deals
July 8, 2021
BridgeBio Enters Collaborations with Three Academic Research Institutions
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Deals
July 8, 2021
Lilly Enters Collaboration with Verge to Use Its AI to Discover and Develop New Treatments for...
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Drug Development
July 8, 2021
FDA Grants Lysogene Fast Track Designation for GM1 Gangliosidosis Gene Therapy
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Research
July 8, 2021
CMT Research Foundation Enters Partnership with University of Illinois Chicago
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Research
July 7, 2021
Patient Groups Begin Using RARE-X’s Data Collection Platform
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Drug Development
July 7, 2021
ChemoCentryx Addresses FDA Advisory Panel Questions in Amendment to Filing on Vasculitis Drug
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Deals
July 6, 2021
EC Clears AstraZeneca’s Acquisition of Alexion
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Deals
July 6, 2021
Orchard and Pharming Collaborate on Gene Therapy for HAE
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Deals
July 6, 2021
Eureka and MSK License Novel Multiple Myeloma Target Domain to Sanofi
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Drug Development
July 6, 2021
FDA Grants Priority Review to Levo for Prader-Willi Syndrome Therapy
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Deals
July 6, 2021
Abbisko and Sperogenix Partner to Develop ABSK021 for ALS, Other Rare CNS Diseases in Greater China
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RARECast Podcasts
July 2, 2021
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
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Drug Development
July 2, 2021
Arrowhead Pauses RNAi Study in Cystic Fibrosis After Tox Study Signals
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Deals
July 2, 2021
GSK Enters into Global Collaboration with Alector to Develop Antibodies for Neurodegenerative Diseases
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Rare Advocate
July 1, 2021
Rare Leader: Diane Powell, CEO, The Hypersomnia Foundation
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Deals
July 1, 2021
Apellis and Beam Collaborate to Apply Base Editing for Complement-Driven Diseases Drug Discovery
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Drug Development
July 1, 2021
FDA Approves Jazz’s Rylaze as Component of Treatment Regimen for Most Common Childhood Cancer
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Finance
June 30, 2021
Intellia Raises $600 Million to Advance Gene Editing Therapeutic Pipeline
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Finance
June 30, 2021
Sofinnova Invests in Three New Rare Disease Gene Therapy Startups
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Healthcare
June 30, 2021
NHS Creates First-of-Its-Kind Rare Disease Network in UK
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Drug Development
June 30, 2021
FDA Grants Allogene Fast Track Designation for Multiple Myeloma Therapy
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Drug Development
June 30, 2021
GenSight Reports Phase 3 Study Misses Endpoint, but Improves Vision in Patients with Degenerative Eye Disease
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Finance
June 30, 2021
Aerovate Raises $121.5 Million in Upsized IPO to Advance PAH Therapy
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Drug Development
June 30, 2021
FDA Grants Lexeo Therapeutics Rare Pediatric Disease Designation
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Strategy
June 29, 2021
Orphazyme Lays Off Two-Thirds of Staff After FDA Setback
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Research
June 29, 2021
Natural History Study Informs Potent Lifesaving Update to Standard of Care for PMM2-CDG Patients
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Research
June 29, 2021
Pediatricians Find Cause of Muscle Breakdown in Rare Disease
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Drug Development
June 28, 2021
Intellia and Regeneron Report First-Ever Data Supporting In Vivo CRISPR Editing in ATTR Patients
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Drug Development
June 28, 2021
EU Approves Roche’s Enspryng as First At-home Subcutaneous Treatment for NMOSD
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Drug Development
June 28, 2021
FDA Grants Fast Track Designation to Three Experimental Rare Disease Therapies
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RARECast Podcasts
June 25, 2021
Forging Gene Therapy Capacity and a Pipeline at the Same Time
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Finance
June 25, 2021
Graphite Bio Raises $238 Million in IPO to Advance Precision Gene Editing Pipeline
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Legislation
June 25, 2021
Senators Introduce Legislation to Help Diagnose Children with Rare Diseases
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Research
June 25, 2021
International Study of Rare Childhood Cancer Finds Genetic Clues, Potential for Tailored Therapy
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Rare Advocate
June 24, 2021
Rare Leader: Kevin Mead, Executive Director of the International Pemphigus & Pemphigoid Foundation
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Deals
June 24, 2021
AbbVie Exercises Right to Acquire TeneoOne and Lead Asset Targeting Multiple Myeloma
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Deals
June 24, 2021
4D Molecular Reports Trial Data for Rare Eye Disorders and Termination of Roche Agreement
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Research
June 24, 2021
Pacific Biosciences and Rady Children’s Collaborate on WGS Study of Rare Diseases
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Drug Development
June 24, 2021
Corbus Reports Lenabasum Fails to Meet Primary Endpoint in Late-Stage Trial for Dermatomyositis
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Drug Development
June 24, 2021
FDA Approves Santen’s Verkazia to Treat Vernal Keratoconjunctivitis in Children and Adults
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Finance
June 23, 2021
Glycomine Raises $35 Million to Advance Novel Treatment for Rare Glycosylation Disease
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Drug Development
June 23, 2021
UniQure Reports Positive 52-Week Data from Pivotal Trial of Hemophilia B Gene Therapy
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Research
June 22, 2021
MIND Institute Gets $1.25 Million Grant for SYNGAP1 Syndrome
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Finance
June 22, 2021
Protagonist Raises $132 Million to Advance Rare Blood Cancer Therapy
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Finance
June 22, 2021
Neurona Raises $41.5 Million to Advance Cell Therapies for Epilepsy and Neurological Disorders
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Deals
June 22, 2021
Neurelis Acquires Rights to Portfolio of Compounds Targeting Rare CNS Disorder
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Deals
June 22, 2021
PerkinElmer to Acquire Viral Vector Gene Delivery Specialist Sirion Biotech
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Deals
June 22, 2021
Jasper and Aruvant Collaborate on Treatment for Sickle Cell Disease
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Research
June 22, 2021
New UAE Center will Diagnose and Treat Patients with Genetic Diseases
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Drug Development
June 21, 2021
EC Expands Approval of Sanofi’s Oral MS Therapy to Include Children and Adolescents
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Drug Development
June 21, 2021
Agios Seek Approval for Mitapivat for Rare Inherited Blood Disorder
Read More
RARECast Podcasts
June 18, 2021
Using A Natural DNA Repair Process to Improve Genetic Medicines
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Drug Development
June 18, 2021
FDA Says It Won’t Approve Orphazyme’s NPC Therapy without Additional Data
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Drug Development
June 18, 2021
FDA Grant Applied Therapeutics Fast Track Designation for Experimental Galactosemia Therapy
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Drug Development
June 18, 2021
FDA Grants United Therapeutics Priority Review for PAH Drug
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Deals
June 18, 2021
Perlara Enters Collaboration with Vivan for Multiple Rare Diseases
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Rare Advocate
June 17, 2021
Rare Leader: Fallon Schultz, President and Founder, International FPIES Association
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Finance
June 17, 2021
Gene Editing Therapeutics Developer Verve Completes $266.7 Million IPO
Read More
Deals
June 17, 2021
Horizon Therapeutics Buy Manufacturing Facility to Support Its Growth
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Drug Development
June 16, 2021
FDA Expands Approval of Blueprint Medicines’ Ayvakit to Include Rare Blood Disorder
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Uncategorized
June 16, 2021
CAMP4 Raises $45 Million for Programmable Therapeutics to Upregulate Genes
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Deals
June 16, 2021
n-Lorem Foundation Forms Partnership with Manufacturer Oligonucleotide Nitto Avecia
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Research
June 15, 2021
Researchers Use Base Editing to Turn Pathogenic Gene Benign in Mice with SCD
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Drug Development
June 15, 2021
Biogen Choroideremia Gene Therapy Fails in Phase 3 Study
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Deals
June 15, 2021
CRISPR Therapeutics and Capsida Enter Strategic Collaboration to Develop Gene-Edited Therapies for ALS, FA
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Drug Development
June 15, 2021
Novartis Reports Positive Results for PNH Therapy
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Research
June 15, 2021
Nanoparticle Therapy Shows Early Promise at Preventing a Rare, Fatal Newborn Lung Disease
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Deals
June 14, 2021
Cure Rare Disease Enters Partnership with Curi Bio for DMD Gene Therapy
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Drug Development
June 14, 2021
Study Shows Evrysdi Improved Motor Function in Pre-Symptomatic Babies
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Strategy
June 14, 2021
CDMO Yposkesi Launches Construction of Advanced Therapy Facility Near Paris
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Drug Development
June 14, 2021
FDA Grants Jasper Therapeutics Rare Pediatric Disease Designation for Conditioning Treatment Prior to Stem Cell Transplant
Read More
RARECast Podcasts
June 11, 2021
Empowering Rare Disease Patients with their Own Health Records
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In Rare Form
June 11, 2021
A Mother’s Prosecution Alarms Rare Patient Advocates
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Drug Development
June 11, 2021
Sanofi Presents Positive New Data for Sutimlimab for the Treatment of CAD
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Drug Development
June 11, 2021
Vertex Achieves Primary Endpoint in AATD Study but Drops Program Due to Insufficient Clinical Benefit
Read More
Rare Advocate
June 10, 2021
Rare Leader: Kim Hollander, Executive Director, Oxalosis & Hyperoxaluria Foundation
Read More
Deals
June 10, 2021
Codexis and Takeda Expand Collaboration to Additional Gene Therapy for a Rare Genetic Disorder
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Finance
June 10, 2021
Kurome Closes $15 Million Financing to Develop Targeted Kinase Inhibitors to Treat Rare Blood Cancers
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Drug Development
June 10, 2021
New Data Point to Potential Long-Term Efficacy of Biogen’s Spinraza for SMA 
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Data Center
June 9, 2021
Rare Disease Therapeutics Developers Continued to Rake in Cash in May
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Finance
June 9, 2021
Alcyone Therapeutics Launches to Advance Next-Generation Gene Therapies for CNS Disorders
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Drug Development
June 9, 2021
FDA Approves Trikafta in Children with Cystic Fibrosis Ages 6 through 11 with Certain Mutations
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Finance
June 8, 2021
CyGenica Secures Seed Funding to Accelerate Cancer and Rare Genetic Disease Drug Delivery
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Drug Development
June 8, 2021
FDA Approves Alexion’s Ultomiris for Children and Adolescents with PNH
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Policy
June 8, 2021
Rare Disease Company Coalition Names Executive Committee
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Drug Development
June 7, 2021
FDA Lifts Clinical Hold on Bluebird Bio’s Sickle Cell Disease and β-Thalassemia Studies
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Drug Development
June 7, 2021
FDA Approves First Treatment for Patients with Plasminogen Deficiency, a Rare Genetic Disorder
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Drug Development
June 7, 2021
Novartis Reports Positive Interim Analysis from Phase 2 Study in Rare Kidney Disease
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RARECast Podcasts
June 4, 2021
Targeting CNS Diseases with Gene Therapies
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Drug Development
June 4, 2021
Nanoscope Gene Therapy Restores Vision in 11 Patients Blinded by Retinitis Pigmentosa
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Rare Advocate
June 3, 2021
Rare Leader: Krista Vasi, Executive Director, Usher Syndrome Coalition
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Strategy
June 3, 2021
Auris Medical Reboots to RNA Therapeutics with Acquisition of Trasir Therapeutics
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Finance
June 3, 2021
Amyl Raises $22 million to Develop Novel Therapies for Amyloidosis
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Finance
June 3, 2021
Stablix Therapeutics Launches with $63 Million to Target Protein Stabilization
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Drug Development
June 3, 2021
AavantiBio and Resilience Form Strategic Collaboration for Gene Therapy Development and Manufacturing
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Drug Development
June 3, 2021
FDA Grants Protagonist Breakthrough Therapy Designation for Rusfertide in PV
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Drug Development
June 3, 2021
FDA Grants Priority Review to Amryt’s EB Drug
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Deals
June 2, 2021
MorphoSys to Acquire Rare Cancer Drug Developer Constellation Pharma for $1.7 Billion
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Finance
June 2, 2021
Transine $12.8 Million Seed Funding to Advance Novel Class of Therapeutic RNAs
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Drug Development
June 2, 2021
Santhera and ReveraGen Report Positive Topline Results in Pivotal DMD Study
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Research
June 2, 2021
Barth Syndrome Foundation and American Heart Association Collaborate to Advance ResearchRare Daily Staff
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Research
June 2, 2021
Treatabolome Project Seeks to Shorten Diagnosis-to-Treatment Time for Rare Disease Patients
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Drug Development
June 2, 2021
FDA Grants Breakthrough Therapy Designation to Janssen Off-the-Shelf Cell Therapy for Multiple Myeloma
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Drug Development
June 1, 2021
FDA Approves QED and Helsinn’s Truseltiq to Treat Patients with Cholangiocarcinoma
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Research
June 1, 2021
Scientists Discover a New Genetic Form of ALS in Children
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Diagnosis
June 1, 2021
Solve-RD Collaboration Makes Case for Data Sharing and Periodic Reanalysis
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Drug Development
June 1, 2021
FDA Grants Fast Track Designation to BridgeBio’s Encaleret for the Treatment of ADH1
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Deals
June 1, 2021
Genomenon and Nostos Genomics Partner to Accelerate Rare Genetic Disease Diagnoses
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Drug Development
May 27, 2021
A New Therapy Offers A Different Approach to Inhibiting the Complement System
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Rare Advocate
May 27, 2021
Rare Leader: Kyle Dempsey, President, Superficial Siderosis Research Alliance
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Drug Development
May 27, 2021
Eloxx and Metagenomi Receive Funding Awards from Cystic Fibrosis Foundation
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Finance
May 27, 2021
Day One Raises $160 Million in IPO to Advance Genetically Defined Cancer Therapies
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Drug Development
May 26, 2021
Larimar Says $95 Million Placement Cancelled After FDA Places Clinical Hold on Lead Program
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Business
May 26, 2021
Penn’s Gene Therapy Program Becomes Anchor Tenant of Discovery Labs
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Finance
May 26, 2021
Rgenta Therapeutics Completes Seed Financing, Raising $38 Million for RNA-Targeted Pipeline
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Drug Development
May 26, 2021
FDA Asks Travere for Additional Data Ahead of Marketing Application for FSGS Therapy
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Deals
May 26, 2021
BlueRock and Senti Bio Collaborate to Develop Engineered Cell Therapies for Regenerative Medicine
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Drug Development
May 25, 2021
Apellis and Sobi Report Positive Results from Empaveli Study in Treatment Naïve Patients with PNH
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Drug Development
May 25, 2021
FDA Grants Scholar Rock Fast Track Designation for SMA Therapy
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Regulatory
May 25, 2021
FDA Approves First Therapy for Rare Form of NSCLC with Companion Diagnostic
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Regulatory
Deals
Regulatory
May 24, 2021
Bipartisan Legislation Introduced to Advance Biomedical Research Stalled by Pandemic
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Regulatory
May 24, 2021
CHMP Recommends Approval of Therapies to Treat the Rare Disorders PFIC and POMC
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Drug Development
May 21, 2021
Bringing Aberrant Proteins Back into the Fold
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Finance
May 21, 2021
Haya Therapeutics Raises $20 Million Seed Financing for RNA-based Fibrosis Therapies
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Deals
May 21, 2021
Mallinckrodt Reaches Agreement to Sell Experimental NPC-1 Drug Adrabetadex to Mandos
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Drug Development
May 21, 2021
European Regulators Give Bluebird Bio Positive Opinion for CALD Gene Therapy
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Drug Development
May 21, 2021
Biogen and Ginkgo Bioworks Enter Collaboration to Create Novel Gene Therapy Manufacturing Platform
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Drug Development
May 21, 2021
European Medicines Agency Grants PRIME Designation to Larimar’s Treatment for Friedreich’s Ataxia
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Rare Advocate
May 20, 2021
Rare Leader: Trish Flanagan, co-founder and president, YBRP
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Drug Development
May 20, 2021
Adaptimmune Reports Positive Initial Data from Mid-Stage Trial in Rare Cancer
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Data Center
May 19, 2021
Investors Pile $2.2 Billion into Rare Disease Therapeutics Developers in April
Read More
Finance
May 19, 2021
Eloxx Raises $52 Million in Public Offering to Advance Rare Disease Pipeline
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Deals
May 19, 2021
Ciitizen and RARE-X Form Collaboration
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Finance
May 18, 2021
G2 Bio Companies Launch with $200 Million to Develop Transformative Genetic-Based Therapies
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Finance
May 18, 2021
Gamma Biosciences Acquires Controlling Stake in Mirus Bio
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Finance
May 18, 2021
Vedere Bio II Launches with $77 Million to Develop Next Generation Ocular Gene Therapies
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Finance
May 18, 2021
Stealth Bio Raises $30 Million to Advance Elamipretide Clinical Trials
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Finance
May 18, 2021
Interius Raises $76 Million for Cell and Gene Therapy Platform
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Deals
May 17, 2021
Charles River to Acquire Gene Therapy CDMO Vigene Biosciences
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Drug Development
May 17, 2021
FDA Approves Apellis’ Empaveli for Adults with Paroxysmal Nocturnal Hemoglobinuria
Read More
RARECast Podcasts
Finance
May 14, 2021
Praxis Precision Medicines Raises $91.3 Million in Public Offering
Read More
Deals
May 14, 2021
PerkinElmer to Expand Cell and Gene Therapy and Biologics Manufacturing with $260 Million Nexcelom Acquisition
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Finance
May 14, 2021
Vera Therapeutics Raises $48 Million in IPO
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Drug Development
May 14, 2021
Biogen XLRP Gene Therapy Fails Late-Stage Study
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Drug Development
May 14, 2021
FDA Grants BridgeBio Fast Track Designation for CAH Gene Therapy
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Rare Advocate
May 13, 2021
Rare Leader: Julie Breneiser, Executive Director, Gorlin Syndrome Alliance
Read More
Finance
May 13, 2021
SpliSense Secures $28.5 Million in Series B Financing Including Backing from CFF
Read More
Finance
May 13, 2021
Flare Launches with $82 Million to Advance Novel Drug Discovery Approach for Transcription Factors
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Finance
May 13, 2021
Magenta Raises $86.4 Million to Advance Novel Antibody-Drug Conjugate Conditioning Platform
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Policy
May 13, 2021
Rare Disease Drug Developers Form Coalition to Address Challenges
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Deals
May 13, 2021
CRO Emmes Acquires UK-Based Orphan Reach
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Deals
May 12, 2021
MD Anderson and Broad Institute Launch Translational Research Platform Focused on Rare Cancers
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Finance
May 12, 2021
Huma Raises $200 Million to Scale Digital Health Platform for Better Care and Research
Read More
Finance
May 12, 2021
Gennao Bio Closes $40 Million in Financing to Develop Pipeline of Targeted Nucleic Acid Therapeutics
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Drug Development
May 12, 2021
FDA Grants Fulcrum Fast Track for FSHD Drug
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Finance
May 12, 2021
Xontogeny Seeds NephroDI to Develop a Treatment for Nephrogenic Diabetes Insipidus
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Research
May 11, 2021
Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency
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Deals
May 11, 2021
Biogen Enters Collaboration with Capsigen for AAV Capsids for CNS and Neuromuscular Disorders
Read More
Research
May 11, 2021
Mutations Cause Rare Genetic Disease in Children
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Uncategorized
May 11, 2021
FDA Halts Rocket Pharma Trial of Experimental Gene Therapy for Danon Disease
Read More
Drug Development
May 10, 2021
Laronde Unveiled to Advance New Class of Programmable Medicines
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Drug Development
May 10, 2021
Ionis Stops Development of Inhaled Antisense Therapy for CF
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Drug Development
May 10, 2021
FDA Grants Fast-Track Designation to Smart Immune’s T Cell Immunity Enhancement Product
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RARECast Podcasts
May 7, 2021
Putting the Patient at the Center of Rare Disease Clinical Trials
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Finance
May 7, 2021
Talaris Raises $150 Million in IPO to Advance Cell Therapy Pipeline
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Finance
May 7, 2021
Science 37 to Become Publicly Listed via Merger with SPAC
Read More
Drug Development
May 7, 2021
ChemoCentryx Continues Slide on Divided FDA Advisory Committee Votes
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Drug Development
May 7, 2021
Orphazyme Drug Fails in Pivotal Study
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Rare Advocate
May 6, 2021
Rare Leader: Tiffany House, President Acid Maltase Deficiency Association
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Finance
May 6, 2021
Dyno Therapeutics Raises $100 Million to Accelerate AI‑powered Gene Therapy Platform
Read More
Finance
May 6, 2021
TwinStrand Raises $50 Million to Grow Market for Duplex Sequencing Technology
Read More
Deals
May 5, 2021
Amryt Acquires Chiasma, Strengthening Its Rare and Orphan Disease Portfolio
Read More
Research
May 5, 2021
Centogene Discovers Six New Rare Diseases
Read More
Drug Development
May 5, 2021
ChemoCentryx Shares Crash After FDA Questions Data Supporting ANCA Vasculitis Therapy
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Research
May 5, 2021
Study Finds People with Rare Diseases Often Feel Stigmatized
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Drug Development
May 4, 2021
Sarepta Reports High-Dose SRP-5051 Increases Dystrophin in Certain DMD Patients
Read More
Finance
May 4, 2021
Mogrify Raises $17 Million to Advance Reprogrammed Cell Therapies  
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Deals
May 4, 2021
Passage Bio Partners with InformedDNA to Offer Genetic Counseling and Testing for FTD
Read More
Regulatory
May 4, 2021
Acella Issues Voluntary Nationwide Recall of Certain Lots of Thyroid Tablets Due to Sub Potency
Read More
Finance
May 3, 2021
Affinia Raises $110 Million to Advance Engineered AAV Gene Therapy Vector Platform
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Finance
May 3, 2021
Aceragen Launches with $35 Million and Experimental Therapy for Farber Disease
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Drug Development
May 3, 2021
With Accelerated Approval Off the Table, Avrobio Seeks Registration Trial for Fabry Disease Gene Therapy
Read More
Drug Development
May 3, 2021
Alnylam Reports Positive Early Results of Phase 3 PH1 Study
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Drug Development
May 3, 2021
FDA Expands Approval of Chiesi’s Ferriprox for Transfusional Iron Overload due to Sickle Cell Disease
Read More
RARECast Podcasts
April 30, 2021
Creating a Toolkit to Accelerate the Development of Gene Editing Therapies
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Rare Advocate
April 29, 2021
Rare Leader: Nancy Lazarus, Founder, Achalasia Awareness Organization
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Finance
April 29, 2021
Capsida Debuts with $140 Million to Advance Next Generation Gene Therapies
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Deals
April 29, 2021
Mirum Grants CANbridge Exclusive License to Maralixibat in Greater China for Rare Liver Diseases
Read More
Drug Development
April 29, 2021
FDA Grants Fast Track to Akari Bullous Pemphigoid Therapy
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Drug Development
April 29, 2021
Astellas Takes $532 Million Charge Over Delay of Gene Therapy for XLMTM
Read More
Deals
April 28, 2021
Allen Institute and BioMarin Collaborate to Develop Gene Therapies for Rare Brain Diseases
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Drug Development
April 28, 2021
FDA Tell Protalix and Chiesi It won’t Grant Accelerated Approval for Fabry Therapy
Read More
Deals
April 27, 2021
LogicBio Enters Gene-Editing Collaborations with CANBridge and Daiichi Sankyo
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Deals
April 27, 2021
AllStripes Collaborates with NIH’s NCATS to Launch Effort in Rare Disease Diagnosis
Read More
Drug Development
April 27, 2021
FDA Removes Clinical Hold on Voyager, Clears IND for Huntington’s Gene Therapy
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Drug Development
April 27, 2021
Biogen Will Provide Early Access to Experimental ALS Drug
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Drug Development
April 27, 2021
EMA Grants PRIME Designation to Vertex and CRISPR’s Therapy for Transfusion-Dependent Beta Thalassemia
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Drug Development
April 27, 2021
AavantiBio Partners with Catalent to Advance Gene Therapies for Rare Genetic Diseases
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Regulatory
April 27, 2021
FDA Issues Guidance on Individualized ASOs for Ultra-Rare Conditions
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Drug Development
April 26, 2021
FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
Read More
Drug Development
April 26, 2021
FDA Approves ADC Therapeutics’ Zynlonta to Treat Rare Cancer
Read More
RARECast Podcasts
April 23, 2021
PTC Looks to Advanced Therapies
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Deals
April 23, 2021
CDISC and NORD to Develop Data Standards for Rare Disease
Read More
Rare Advocate
April 22, 2021
Rare Leader: Heidi Edwards, President and Founder, Sister’s Hope Foundation
Read More
Deals
April 22, 2021
Vertex and Obsidian Enter into Collaboration to Discover Novel Therapies that Regulate Gene Editing
Read More
Finance
April 22, 2021
ITM raises $109 Million to Advance Radiopharmaceutical Precision Oncology Pipeline
Read More
Finance
April 21, 2021
Code Biotherapeutics Launches with $10 Million to Develop Therapies for Debilitating Genetic Diseases
Read More
Finance
April 21, 2021
EdiGene Raises $62 Million to Advance and Scale Up Clinical Translation of Gene Editing Technologies
Read More
Drug Development
April 21, 2021
Beam Publishes Data Demonstrating Ability to Rationally Design Base Editors for Precise Editing
Read More
Pricing
April 21, 2021
Bluebird Withdraws Zynteglo from German Market, Cuts Workforce
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Drug Development
April 21, 2021
EC Grants Approval to GW Pharmaceuticals’ Epidyolex for TSC-Associated Seizures
Read More
Uncategorized
April 21, 2021
FDA Grants RMAT Designation to Allogene’s Cell Therapy for Multiple Myeloma
Read More
Research
April 20, 2021
The Making of a Gene Therapy for an Ultra-Rare Disease
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Drug Development
April 20, 2021
FDA Puts Clinical Hold on KalVista’s HAE Therapy
Read More
Deals
April 20, 2021
Citrine and Sinopharm Enter Strategic Partnership to Broaden Access to Rare Disease Drugs in China
Read More
Deals
April 20, 2021
Vertex and CRISPR Amend Sickle Cell Disease and Beta Thalassemia Collaboration
Read More
Research
April 20, 2021
IndoUSrare and Rare-X Conduct Feasibility Study for a Patient-Owned Health Data Collection Program for India
Read More
Business
April 20, 2021
Castle Creek Biosciences Appoints Matthew Gantz as President and CEO
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Finance
April 19, 2021
Rezolute Enters $30 Million Debt Agreement to Advance Rare and Metabolic Disease Pipeline
Read More
Drug Development
April 19, 2021
Study Finds Positive Impact of Fintepla on Dravet Patients and Caregivers
Read More
Drug Development
April 19, 2021
Biogen Reports New Spinraza Data in SMA
Read More
RARECast Podcasts
Research
April 16, 2021
Study Identifies Key Target in Treatment-Resistant Hemophilia A
Read More
Finance
April 16, 2021
Recursion Raises $436 Million in Upsized IPO to Advance Rare Disease Pipeline
Read More
Rare Advocate
April 15, 2021
Lauren Dunlap, Co-Founder and Executive Director, AAIDA
Read More
Finance
April 15, 2021
Medable Raises $78 Million to Decentralize Clinical Trials
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Finance
April 15, 2021
Alchemab Raises $82 Million to Advance Antibody Therapeutics in Neurodegeneration and Cancer
Read More
Deals
April 15, 2021
Ciitizen Partners with STXBP1 Foundation to Accelerate Patient Outcomes  
Read More
Business
April 15, 2021
DOJ Subpoenas Documents from Alnylam Over Marketing of Onpattro
Read More
Drug Development
April 15, 2021
FDA Grants Rare Pediatric Disease Designation to FibroGen Treatment for DMD
Read More
Drug Development
April 15, 2021
New Evrysdi Data Shows Improvement in Motor Function Over Time for SMA Patients
Read More
Finance
April 14, 2021
Investment in Rare Disease Drug Development Continues to Surge
Read More
Research
April 14, 2021
Gene Therapy Shows Promise in Treating Rare Eye Disease in Mice
Read More
Deals
April 14, 2021
Huma Enters Partnership with RARE-X to Accelerate Rare Disease Research
Read More
Drug Development
April 14, 2021
FDA Grants Rare Pediatric Disease Designation to Therapies from Moleculin and Akuous
Read More
Deals
April 13, 2021
Senti Bio and Spark Collaborate to Develop Next-Generation Precision Gene Therapies
Read More
Deals
April 13, 2021
BridgeBio Pharma Enters Seven Collaborations with University Partners
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Finance
April 13, 2021
Theseus Debuts with $100 Million Series B to for Targeted Treatment-Resistant Cancer Mutations
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