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

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

The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company. It is the first gene therapy directly administered to the brain and ...

The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...

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 ...

Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...

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 ...

Why one trial participant's healthy birth after her CAR-T treatment for lupus is catching resesarchers' attention: ...

Immunotherapy, which enhances the immune system's T cell response to eliminate cancer cells, has emerged as a key approach in ...

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.