NGGT001

NGGT001 is an investigational gene therapy designed to treat Bietti's Crystalline Dystrophy (BCD), a rare inherited retinal dystrophy characterized by yellow-white crystals and lipid deposits in the retina that can progress to blindness. The therapy uses an adeno-associated virus serotype 2 (AAV2) vector to deliver a functional copy of the *CYP4V2* gene directly into the subretinal space, aiming to restore enzymatic fatty acid metabolism in patients with confirmed *CYP4V2* mutations. Clinical trials have shown promising safety and efficacy results, including improvements in best corrected visual acuity (BCVA) and quality of life for treated patients. No severe adverse events related to the drug have been reported, and mild procedure-related effects resolved spontaneously. There are currently no approved treatments for BCD; NGGT001 is being evaluated as a potential first-in-class therapy[1][2][3][4][5][6][7].

Other names

rAAV-hCYP4V2rAAV-hCYP-4V2rAAV-hCYP 4V2