Vivet Therapeutics Secures EUR 4.9 Million to Progress Development of Gene Therapy Targeting Cerebrotendinous Xanthomatosis

Vivet Therapeutics Secures EUR 4.9 Million Government Funding for Gene Therapy Advancement in Cerebrotendinous Xanthomatosis Treatment”

Vivet Therapeutics, a biotech company specializing in gene therapies for rare inherited metabolic disorders, has received EUR 4.9 million in funding from the French government. The financing, part of the “Innovations in biotherapies” framework within the France Health Innovation Plan 2030, aims to support the development of a gene therapy for cerebrotendinous xanthomatosis (CTX), a rare neurodegenerative disease. Administered over three years, the funding will be managed by Bpifrance under the coordination of the French Health Innovation agency.

CTX is a rare autosomal recessive genetic disorder impacting the metabolism of fats, particularly cholesterols. Patients with CTX face challenges in breaking down various forms of cholesterol, leading to their accumulation in specific body areas, marked by fatty yellow nodules called tendon xanthomas within the brain’s connective tissues. These deposits can result in progressive damage to the brain and other bodily regions, accompanied by symptoms such as chronic diarrhea during infancy, cataracts in late childhood, and later, neurological issues like dementia, seizures, hallucinations, depression, coordination difficulties, and speech problems. Currently, there is no cure for CTX, with available treatments focused on slowing disease progression.

Vivet intends to utilize the funding to advance the development of its gene therapy candidate, VTX-806, as a potential long-term treatment option to halt or reverse CTX progression, potentially leading to a cure. VTX-806 seeks to restore the activity of the CYP27A1 gene, which is impaired in CTX patients, utilizing an adeno-associated viruses (AAV) vector for delivery. The goal is to demonstrate a safe and viable treatment option for CTX.

Gloria Gonzalez, Chief Scientific Officer at Vivet Therapeutics, expressed the company’s dedication to developing treatment options for metabolic disorders, highlighting their lead clinical program, VTX-801, targeting Wilson Disease, and the VTX-806 program for CTX patients. Anne Douar, Chief Operating Officer at Vivet Therapeutics, emphasized the urgency of developing a transformative and potentially curative treatment for CTX patients, made possible by the funding from the France Health Innovation Plan 2030, to improve patient outcomes and prognosis.

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