Vivet Receives $5.3 Million Grant to Advance Gene Therapy for Rare Genetic Metabolic Disease

Rare Daily Staff   

Vivet Therapeutics, a clinical stage biotech developing gene therapies for rare inherited metabolic disorders, said it received $5.3 million (€4.9 million) from the French government to advance the development of a gene therapy for the treatment of cerebrotendinous xanthomatosis, a rare neurodegenerative disease.

The funding is from the Innovations in biotherapies framework of the France Health Innovation Plan 2030. Coordinated by the French Health Innovation agency, the funding, will be distributed over a three-year period and operated by Bpifrance.

Cerebrotendinous xanthomatosis (CTX) is a rare autosomal recessive genetic disorder that affects the body’s ability to metabolize fats known as cholesterols. Patients with CTX are unable to break down different forms of cholesterol, which build up in certain areas of the body and are characterized by fatty yellow nodules (tendon xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body. Disease symptomatology is characterized by chronic diarrhea during infancy and cataracts in late childhood. When patients enter adulthood, they begin to demonstrate progressive neurological symptoms, which may include dementia, seizures, hallucinations, depression and difficulty with coordination and speech. At present there is no cure for CTX, only treatment to slow disease progression.

Vivet will use the funds to further develop its gene therapy product VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure CTX in patients. By reinstating the CYP27A1 gene activity, which is impaired in CTX patients, using an adeno-associated viruses (AAV) vector, Vivet aims to demonstrate a safe and feasible treatment option for CTX disease.

The funding is for the CrisTauX program which will cover preclinical research and development activities of VTX-806 and includes the manufacturing process development within medicine good manufacturing practices rules and a clinical study aiming at identifying biomarkers for the effectiveness of treatment in patients under standard of care. CrisTauX will enable accelerated clinical development of VTX-806 upon completion of the program.

“CTX is a progressive, neurodegenerative disorder, that if left untreated can lead to debilitating consequences and a poor prognosis affecting a patient’s lifespan,” said Anne Douar, chief operating officer at Vivet Therapeutics. “The funding granted as part of France Health Innovation Plan 2030 will enable Vivet to accelerate the development of a transformative and potentially curative treatment with our gene therapy product VTX-806 for patients with CTX.”

Photo: Anne Douar, chief operating officer at Vivet Therapeutics