Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle ...

For nearly one-third of the 3 million adults in the United States living with epilepsy, medications fail to control seizures.

To help improve gene therapy, a multidisciplinary team has generated a comprehensive atlas that researchers can use to select ...

Variant, a company dedicated to developing innovative treatments for orphan inherited eye diseases, and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) in Naples ...

Gene therapies are a revolution in medicine but over the past few years, companies developing them for many serious ...

Base editing corrected a mutation that causes macular degeneration, highlighting the potential of gene therapy to treat ...

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the ...

A recent study demonstrated the potential of gene therapy to restore motor capacity in an ultra-rare disease, megalencephalic ...

The therapy is administered as a single subretinal ... LUXTURNA uses an adeno-associated viral type 2 (AAV2) vector to ...

Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing ...

The European Medicines Agency (EMA) provided scientific advice supporting the non-clinical development plan of VAR002, an adeno-associated viral vector gene therapy targeting inherited retinal ...