Neurogene reported a patient experienced systemic hyperinflammatory syndrome during their Rett syndrome gene therapy trial. CEO Rachel McMinn confirmed the female patient is in critical condition. The ...
Syndax Pharmaceuticals won the first FDA approval last week in a new class of medicines called menin inhibitors.
The FDA on Monday offered new draft guidance to help drugmakers understand the nuts and bolts of FDA processes for developing and submitting an application for a new cell or gene therapy, from pre-IND ...
In 2010, neuroscientist and former Eli Lilly research leader Steve Paul started a company that he hoped would spark a revolution in drug discovery built around a class of compounds dubbed ...
Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...
Alnylam's nucresiran showed promising results in Phase 1 trial for rare cardiomyopathy, achieving up to 96% TTR reduction at ...
Aclaris partners with Biosion for anti-TSLP programs and seeks $80M funding, Keymed Biosciences signs $626M potential deal with Ouro Medicines, ReCode receives CF Foundation funding, Quell gets $10M ...
Atlas Venture, one of biotech’s go-to VC firms, is eyeing a $450 million 14th fund, partner Bruce Booth said in its ...
Novartis and Ratio Therapeutics announced a collaboration deal worth up to $745 million, focusing on SSTR2 radiotherapeutic ...
Trogenix has emerged from stealth to advance its pipeline of viral immunotherapies for cancer, starting with a lead candidate ...
San Diego biotech Avenzo Therapeutics has in-licensed another asset, this time securing the global ex-China ...
Cybin said its psilocin analog has the potential to wipe out symptoms in most patients with a serious form of ...
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