Drug Pipeline

3 drugs associated with Aromatic L-amino acid decarboxylase deficiency

DrugPhaseSponsor
AAV2-hAADC
Phase 1Krzysztof Bankiewicz↗
VGN-R09b
Phase 1Shanghai Jiao Tong University School of Medicine↗
Eladocagene Exuparvovec

KEBILIDI is a recombinant adeno-associated virus serotype 2 (rAAV2) based gene therapy designed to deliver a copy of the DDC gene which encodes the AADC enzyme. Intraputaminal infusion of KEBILIDI results in AADC enzyme expression and subsequent production of dopamine in the putamen.

Phase 2PTC Therapeutics↗