Phase I
There are currently no disease-modifying treatments for Friedreich’s ataxia. That could change when the FDA makes a decision about Reata Pharmaceuticals’ omaveloxolone on Feb. 28.
Novartis has abandoned its ex vivo sickle cell disease program developed using Intellia Therapeutics’ CRISPR gene editing platform, Intellia announced Thursday.
Graphite Bio is discontinuing the development of nulabeglogene autogedtemcel (nula-cel), its lead asset, and shaving off about 50% of its workforce.
After overcoming a clinical hold, Astellas presented preliminary safety and efficacy data from the Phase I/II FORTIS trial of AT845 in late-onset Pompe disease at the 19th Annual WORLDSymposium 2023.
Hemab Therapeutics announced the closing of an oversubscribed Series B round to bring new treatment options for lesser-known bleeding disorders.
Bristol Myers Squibb has returned the rights for interleukin-12 immunotherapy program DF6002 back to its original owner, Dragonfly Therapeutics, the companies announced Monday.
Merck and Nectin Therapeutics announced a research collaboration agreement to study the safety and efficacy of Nectin’s investigational antibody in locally advanced and metastatic solid tumors.
The FDA placed 4D Molecular Therapeutics’ gene therapy program, 4D-310, on clinical hold, the company announced Thursday.
uniQure inked a deal with Apic Bio to gain development and commercialization rights to APB-102, a gene therapy for a rare, genetic form of ALS.
Phase I/II data showed AC Immune’s anti-amyloid-beta vaccine candidate could safely elicit an antibody response in patients with Alzheimer’s disease. Now, it will expand to Down Syndrome patients.
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