NEW YORK – Vivet Therapeutics has received €4.9 million (about $5.3 million) from the French government to advance development of a gene therapy program for cerebrotendinous xanthomatosis (CTX), the Paris-based biotech firm said Thursday.
The gene therapy, VTX-806, uses an adeno-associated virus vector to deliver a functional copy of the CYP27A1 gene, mutations in which cause CTX, with the aim of halting or reversing progression of the rare neurodegenerative disease. CTX is an autosomal recessive genetic disorder in which the body isn't able to metabolize cholesterols.
The investment from France's Health Innovation 2030 program, operated by French national investment bank Bpifrance, will support preclinical R&D activities, manufacturing process development, and a clinical study to identify biomarkers of effectiveness.