Vivet wins $5.3m grant to develop gene therapy for rare metabolic disorder

2024-02-01
·
交易
Pharmaceutical Technology
基因疗法临床研究
Vivet’s VTX-806 is an adeno-associated virus (AAV) vector gene therapy that reinstates CYP27A1 gene activity. Image Credit: Anusorn Nakdee / Shutterstock.
VivetrenVTX-806rnment has awarded a €4.9m ($5.3m) grant to Paris-based Vivet TherapeutiCYP27A1evelop VTX-806, a gene therapy to treat a rare condition that leads to the accumulation of cholesterol in nerve cells called cerebrotendinous xanthomatosis (CTX).
The funding is part of the France Health Innovation Plan 2023 under the acceleration strategy of Biotherapies – Bioproduction in innovative therapies. The funding is operated by French investment bank Bpifrance and will be disbursed over three years, as per a 1 February press release.
ViveFrench Governmentene therapies for different rare disorders and hasVivet Therapeuticsh Pfizer, whVTX-806he latter owns 15% equity in the company.cerebrotendinous xanthomatosis (CTX)
VTX-806 is an adeno-associated virus (AAV) vector gene therapy that reinstates CYP27A1 gene activity. The therapy is being developed to stop or reverse disease progression over the long term, or to potBpifrancecure CTX.
Vivets a rare autosomal recessive genetic disorder caused by an abnormality in the CYP27A1 PfizerThe gene is responsible for producing mitochondrial enzyme sterol 27-hydroxylase enzyme, responsible for converting cholesterol into a bile acid.
VTX-806o:Almirall and Microsoft partner for dermatological drug developmentCYP27A1CTX
If untreaAlmirall can Microsoftgressive neurologic problems such as seizures, cognitive impairment, and ataxia. Current disease treatment includes oral bile acid replacement therapy and treatment with chenodeoxycholic acid to normalise the production of cholestanol.
Vivet’s lead candidate, VTX-801, is a gene therapy for Wilson’s disease being developed with Pfizer. The AAV vector gene therapy targets the ATP7B gene, responsible for causing the condition. It is currently being investigated in an open-label Phase I/II GATEWAY trial (NCT04537377), that has a clinical readout expected by the end of the year.
The study’s endpoint is to arusfertidesafety and tolerability of VTX-801 at 52 weeks after a single infusion. Additional endpoints include changes in disease-related biomarkers, including free serum copper and serum ceruloplasmin activity, as well as radiocopper-related parameters and VTX-801 responder status to allow standard-of-care withdrawal.
Other therapies in Vivet’s preclinical pipeline include VTX-804 for the treatment of a rare neurological disorder, citrullinemia type 1, along with VTX-802 and VTX-803 for the treatment of progressive familial intrahepatic cholestasis (PFIC) – a set of rare genetic disorders.
In 2021, US-baCTX Mirum Pharmaceuticals acquired development and comseizuressacognitive impairment802 anataxia803.chenodeoxycholic acid
Cell & Gene Therapy coveVTX-801 Pharmaceutical TechnoloWilson’s diseaseby Cytiva.PfizerATP7B
Editorial content is independently produced and follows the higheVTX-801dards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.VTX-801
The cell and gene Mirum Pharmaceuticalsomentum, with a new wave of therapies promising to tranVTX-802he waVTX-803rs treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead.
By Cytiva Thematic
-->
By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy
更多内容，请访问原始网站
文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。
机构
适应症
靶点
药物
生物医药百科问答
全新生物医药AI Agent 覆盖科研全链路，让突破性发现快人一步
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台，助您全方位提升您的研发与决策效率。
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台，以API或者数据包形式对外开放，助您更加充分利用智慧芽新药情报信息。