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CAMBRIDGE, Mass. – Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced that the U.S. Food and Drug Administration (FDA) has accepted the investigational new drug (IND) application for NTLA-5001, the company’s first wholly-owned ex vivoCRISPR genome... -
WASHINGTON – Following Amylyx’s announcement that it intends to submit a New Drug Application (NDA) for AMX0035, The ALS Association today urges the Food and Drug Administration to approve the treatment for all people with ALS as soon as possible. The Association was an original funder of Amylyx and has...
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PARIS – GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the independent Data Safety Monitoring Board (DSMB) completed its third safety review of the ongoing PIONEER Phase I/II...
Videos
Rare disease spotlight
This Fact Friday we are highlighting studies on DMD affected females! Check out these interesting quotes from 2 research articles on DMD carriers. #Duchenne #MuscularDystrophy #RareDisease #NotJustCarriers #DMD
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