The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...

Ascend Advanced Therapies, which has been on an expansion tear since debuting three years ago, inked a deal with EW ...

PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...

Developed by PTC Therapeutics, Kebilidi is an adeno-associated virus vector-based gene therapy indicated for the treatment of ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.

Biotech stock Neurogene collapsed Tuesday after a patient experienced a serious side effect in the company's Rett syndrome ...

WARREN, NJ, USA I November 13, 2024 I PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of ...

at 08:57 FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an ...

The addition of the ABL Rockville team and facility aligns more than three decades of development and manufacturing experience with additional virus modalities and GMP capacity for adeno-associated ...