“rare-diseases” Archives

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Entry Author Date Location
X4 Pharma Pulls In $27M for Immune Drug in Cancer, Rare Diseases 11/16/17 Boston
Xconomy Q&A: An Update with Arcturus Therapeutics CEO Joseph Payne 10/23/17 San Diego
Alnylam Data Puts a Nobel Discovery One Step Closer to Approved Drug 09/20/17 Boston
BridgeBio Bags $135M For “Hub and Spoke” Rare Disease Drug R&D 09/13/17 San Francisco
Startup Builders, IPO Closers & More: Meet Xconomy’s CEO Award Finalists 08/10/17 Boston
FDA Approves Houston Biotech Lexicon’s Carcinoid Syndrome Diarrhea Drug 02/28/17 Texas
Insurer UHC Will Cover Biogen Spine Drug, But With Limits And Proof 02/02/17 Boston
Duchenne Lessons: Advocates For Rare Spinal Disease Have Eyes on FDA 08/26/16 National
Cloud Therapy Uses Watson to Help Doctors, Pharma With Diagnoses 06/08/16 Texas
Moderna’s Third mRNA Startup, Elpidera, Sets Sights on Rare Diseases 05/12/15 Boston
After raising $76M in Series E Round, aTyr Pharma Files for IPO 04/07/15 San Diego
West Coast Biotech Roundup: Tracon, Mirati, Third Rock, & Dendreon 02/06/15 San Diego
Breaking Bottlenecks, Opening the Floodgates to Genomic Data 11/18/14 National
With $22M, True North Points Toward Rare-Disease Clinical Trials 06/17/14 San Francisco
LTI Corrals $5M to Exploit Link Between Gaucher, Parkinson’s 05/12/14 Boston
Repligen, Continuing Overhaul, Flips Rare Disease Drugs to BioMarin 01/21/14 Boston
Moderna, Living a Charmed Life, Pulls in Another $125M From Alexion 01/13/14 Boston
Gates Foundation Health Chief on Coaxing Pharma to Do Good 11/25/13 National
NEA, Genzyme Join $18M Round For Rare Disease Startup Edimer 07/30/13 Boston
Aegerion Shares Soar on New Numbers For Rare Disease Drug 07/30/13 Boston
Sarepta to Seek FDA Approval For Duchenne Muscular Dystrophy Drug 07/24/13 Boston
Ensemble Adds to Cast of Partners With Alexion Deal 07/18/13 Boston
Alnylam Shares Boom on Early Data For Subcutaneous RNA Drug 07/11/13 Boston
Alnylam’s Amyloidosis Drug Holds up in Second Clinical Trial 07/01/13 Boston
Amicus Shelves New-Drug Filing For Fabry Disease Drug 06/17/13 New York
Profiles in Long-Termism: Sarepta Therapeutics CEO Chris Garabedian 06/10/13 National
Nimbus Discovery, Shire to Co-Develop Oral Rare Disease Drugs 05/08/13 Boston
Repligen in Drug Development Licensing Deal with Pfizer 01/04/13 Boston
Synageva BioPharma Announces $80 Million Stock Offering 01/03/13 Boston
Amicus-GlaxoSmithkline Drug for Fabry Disease Fails Clinical Trial 12/20/12 New York
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