Work

FDA approval: Kebilidi (from Catapult's regulatory round-up for November 2024)

Year: 2025

Type: other

Abstract: FDA has approvedeladocagene exuparvovec-tneq (Kebilidi) a recombinant adeno-associated virus serotype 2 (rAAV2)-based gene therapy, containing the human DDC gene (dopa decarboxylase) indicated for the treatment of adult and paediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. It is designed to correct the underlying genetic defect by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production. AADC defi...

Source: Industrias Culturais (Universidade de Coimbra)

DOI: 10.58079/13axk

Author: Adelage

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

OpenAlex ID: W6898950784

Cites:

Cited by:

Related to: