Phase I
ALS is the 5,000-piece jigsaw puzzle of the therapeutic world. As the regulatory edges come together, new research pieces are also connecting.
The hits kept coming for the sickle cell disease space last week as the FDA placed a full clinical hold on Fulcrum Therapeutics’ Investigational New Drug application FTX-6058.
Novartis recently announced it is returning a Phase II-ready nonalcoholic steatohepatitis asset to Pliant. The program stemmed from a 2019 collaboration.
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.
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