Pfizer Invests $51 Million to Access Gene Therapy Platform for Liver Disorders

Mar 31, 2019 | Gene Therapy, Leading Pharma, Liver Disease, Metabolic Disease, Pharma Watch, Wilson Disease

FINSMES reports Pfizer has invested $51 million for a 15% stake in Vivet Therapeutics, a France-based gene therapy venture dedicated to developing inherited liver disorder treatments.  Pfizer secured an exclusive option to buy all outstanding shares. Pfizer’s Monika Vnuk, MD, will joint Vivet’s Board of Directors. The two companies will jointly develop VTX-801, Vivet’s proprietary asset for Wilson disease, a rare chronic and potentially life-threatening liver disorder of impaired copper transport that causes serious copper poisoning. According to the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Wilson’s disease occurs in about 1 in 30,000 people.

Who is Vivet?

Founded in 2016, Vivet was launched to develop innovative gene therapy treatments for orphan diseases. It is focused on optimizing gene therapy through collaboration with the Fundacion para la Investigacion Medica Aplicada (CIMA, Universidad de Navarra) to develop new adeno-associated virus AAV vectors specifically targeting the live and generating new technologies to optimize gene delivery and long term expression. Its lead investigational product, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). In addition to its Wilson disease program, Vivet is also advancing liver-directed gene therapy programs for progressive familial intrahepatic cholestasis (PFIC) for bile excretion defects and citrullinemia for defects in the urea cycle, which leads to the buildup of ammonia and other toxic substances in the blood.

IP Building Blocks

Vivet’s novel technology has been developed through partnerships with, and exclusive license from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain; and Massachusetts Eye and Ear (MEE), Boston, USA. These licenses cover exclusive use of novel proprietary AAV vector gene therapy to treat metabolic diseases and certain MEE patent-protected Anc80 AAV gene therapy vectors. Anc80 is a next generation gene therapy technology designed to increase gene expression levels in the liver, while reducing risk of undesired immunogenicity.


Prior to this recent Pfizer investment, the company is backed by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners.


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