Funds advance gene therapy for cerebrotendinous xanthomatosis 

Neuroscience

Vivet Therapeutics has received €4.9 million from the French government to advance the development of a gene therapy for the treatment of cerebrotendinous xanthomatosis (CTX), a rare neurodegenerative disease.  

This funding is from the “Innovations in biotherapies” framework of the France Health Innovation Plan 2030 and specifically the acceleration strategy Biotherapies – Bioproduction in innovative therapies. Coordinated by the French Health Innovation agency, the funding, will be distributed over a three-year period and operated by Bpifrance. Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. 

CTX is a rare autosomal recessive genetic disorder that affects the body’s ability to metabolise 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 characterised 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 characterised 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. 

Gloria Gonzalez, Chief Scientific Officer at Vivet Therapeutics, said: “Our commitment to developing treatment options for metabolic disorders is demonstrated by our lead clinical programme, VTX-801 which targets Wilson Disease, alongside a second programme, VTX-806, which is in development as a potential treatment option for CTX patients. Our advanced pipeline highlights our ability to provide strong translational support for rare genetic disorders and patients’ needs, notably with the help of the CTX Alliance and the Spanish CTX patient association.” 

The funding programme 

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 programme 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 programme. 

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