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 ...

Novartis and Ratio Therapeutics announced a collaboration deal worth up to $745 million, focusing on SSTR2 radiotherapeutic ...

Patients who would benefit from a GLP-1 face significant challenges when it comes to access. Shortages have made it difficult ...

CSL Behring is closing its Pasadena R&D site, relocating operations to Waltham, Massachusetts, affecting 30 employees. The company is deprioritizing ex vivo lentiviral gene therapy work and focusing ...

Trogenix has emerged from stealth to advance its pipeline of viral immunotherapies for cancer, starting with a lead candidate ...

Rocket Pharmaceuticals reported Monday that benefits from its Danon disease gene therapy were seen up to around five years after treatment.

Syndax Pharmaceuticals won the first FDA approval last week in a new class of medicines called menin inhibitors. The treatment, Rejuforj, will be available as soon as this month for patients with an ...

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 ...

The FDA on Monday offered new draft guidance to help drugmakers understand the nuts and bolts of FDA processes for developing and submitting an application for a new cell or gene therapy, from pre-IND ...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 ...

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 ...