Sarepta says early filing for DMD gene therapy is back on
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy […]
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy […]
The FDA rejected the new drug application for a Levo Therapeutics treatment for the rare metabolic disorder Prader-Willi syndrome. According to Levo, the agency said […]
Acadia Pharma’s trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has […]
Shots: Entos will use its Fusogenix nucleic acid delivery system to develop BioMarin products for multiple genetic diseases. BioMarin will be responsible for the preclinical […]
The team behind the World Without Disease Accelerator, part of Janssen Research & Development, one of the Janssen Pharmaceutical companies of Johnson & Johnson, is […]
Shots: Metagenomi to receive an up front, equity investment & is eligible to receive target option exercise fees in addition to development, regulatory, and commercial […]
Shots: Mammoth to receive $41M up front, $650M as future payments based upon the achievement of prespecified research, development & commercial milestones across 2 programs […]
Catabasis has conceded defeat with its Duchenne muscular dystrophy drug edasalonexent, pulling the plug on the drug after a phase 3 trial echoed the results […]
Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after […]
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