“rare-diseases” Archives

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Entry Author Date Location
Vertex Stops Test of Rare Disease Drug, Dimming Pipeline Prospects 10/15/20 Boston
With Phase 2 Data in Hand, Ovid Eyes Multiple Pivotal Trials for Epilepsy Drug 09/30/20 New York
Dyne’s Upsized IPO Brings In $233M for Muscle Disorders Drug R&D 09/17/20 Boston
With FDA Nod, NS Pharma Duchenne Drug Becomes Sarepta Competitor 08/12/20 New York
AbbVie Exits Editas Alliance, Pruning Another Genetic Medicines Pact 08/07/20 Boston
Forge Bio’s Combo Model for Gene Therapies Attracts $40M in Funding 07/21/20 National
CSL Behring Bolsters Hemophilia Lineup With Deal for uniQure’s Gene Therapy 06/25/20 Philadelphia
Orca Bio Surfaces With $192M and Recipes for “Custom” Cell Therapies 06/17/20 San Francisco
Chinook, Aduro Merger Plan Sets Up New Kidney Disease-Focused Biotech 06/02/20 Seattle
BridgeBio’s Pipeline Expansion Continues as Portfolio’s Milestones Approach 04/30/20 San Francisco
Taysha Launches With $30M to Bring Gene Therapy to CNS Disorders 04/29/20 Texas
AstraZeneca Wins First FDA Drug Nod for NF1, a Rare Genetic Disorder 04/10/20 National
Bio Roundup: Pandemic Declared, Rubius Restarts, Kymera’s Cash & More 03/13/20 National
Acceleron Drops Drug for Rare Muscle Disease After Mixed Phase 2 Data 03/10/20 Boston
Spruce Bio Raises $88M for Clinical Tests of Rare Endocrine Disease Drug 02/20/20 San Francisco
Bio Roundup: Goodbye Belviq, LogicBio on Hold, Revolution’s IPO & More 02/14/20 National
FDA Clinical Hold Stops LogicBio’s Gene Editing Study Before It Starts 02/10/20 Boston
Emendo Biotherapeutics Nabs $61M for “Next-Generation CRISPR” R&D 01/15/20 New York
Generation Bio Pockets $110M for Next Wave of Gene Therapy, IPO Plans 01/13/20 Boston
Zafgen’s Path Forward Is Reverse Merger With Chondrial Therapeutics 12/18/19 Boston
FDA Puts Partial Hold on Tests of Ipsen Drug Acquired in $1B Deal 12/06/19 National
FDA Rejects Enzyvant’s Regenerative Therapy for Rare Immune Disorder 12/05/19 Boston
AI Startup Healx Looks to Find Repurposable Drugs for Rare Diseases 11/26/19 National
Alnylam Gets Quick FDA OK for Second RNAi Rare Disease Drug 11/20/19 Boston
Anemia Drug From Acceleron Pharma and Celgene Wins FDA Approval 11/08/19 New York
FDA Halts Novartis Gene Therapy Study Amid Animal Safety Concerns 10/30/19 National
Bio Roundup: Ichnos Emerges, Beyond CAR-T, BridgeBio Bails & More 10/18/19 National
Healx Bags $56M to Advance Affordable, AI-Based Orphan Drugs 10/17/19 National
Alexion to Buy Achillion in $930M Marriage of Rare Disease Drug Firms 10/16/19 Boston
Dova Pharmaceuticals Agrees to Sobi Acquisition, Shares Jump 38% 09/30/19 Raleigh Durham
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