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 ...

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

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 ...

New hepatitis D treatment data from Vir Biotechnology and Bluejay Therapeutics shows promising results in reducing viral levels. Both companies plan further trials, with Vir targeting Phase 3 in 2025.

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

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 ...

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The FDA on Monday offered new draft guidance to help drugmakers understand the nuts and bolts of FDA processes for developing and submitting an application for a new cell or gene therapy, from pre-IND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