A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

Unlike existing treatments for Alzheimer’s that target unhealthy protein deposits, the new approach developed at UC San Diego ...

The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, ...

Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on ...

VG801 is a dual AAV gene therapy that leverages mRNA trans-splicing via the vgRNA REVeRT and vgAAV platforms to deliver the ...

FDA alignment on outcomes for the key Part B trial was supported by caregiver-reported milestone gains. Taysha Gene Therapies ...

The former workers cited various reasons why they think Novo was cautious: its conservative culture and history of investing ...

MarkHerz inks MoU with Technical University of Munich Hospital to develop cardiovascular and diabetes-targeted AAV gene therapies: Munich Thursday, June 5, 2025, 15:00 Hrs [IST] M ...

AnaptysBio announces positive results from phase 2b trial of rosnilimab to treat rheumatoid arthritis: San Diego Thursday, June 5, 2025, 18:00 Hrs [IST] AnaptysBio, Inc., a clinic ...