Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...

PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

PTC Therapeutics (PTCT) stock on watch as company receives FDA approval for gene therapy Kebilidi for AADC deficiency. Read ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

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 ...

The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.

These preclinical findings could lead ... Single-Dose Gene Therapy Is Potentially Life-Changing for Adults With Hemophilia B Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding ...

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.

This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) ...