Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

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

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

Baby KJ Muldoon, the first patient to successfully receive personalized CRISPR gene editing therapy has returned home after over 300 days at the hospital.

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

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

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

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

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

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

Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting, which will review recent TSHA-102 Clinical Program ...

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...