Specialised in the development of gene therapies for liver-related diseases, Vivet Therapeutics’ solutions are currently in preclinical and clinical phases. 

Many metabolic diseases are of genetic origin. In Wilson’s disease, the ATP7B gene has a mutation that leads to a non-functional protein. The gene therapy developed by Vivet aims to deliver the correct sequence of the gene into the liver, using a viral vector, in order to produce the functional protein. “It’s about restoring the homeostasis of copper metabolism to avoid both hepatic and/or neurological accumulation,” explains Jean-Philippe Combal, CEO and co-founder of Vivet Therapeutics. 

A single injection into the liver to restore copper metabolism 

The aim is to use this viral vector with high hepatic tropism for many orphan metabolic diseases: Wilson’s disease, progressive familial intrahepatic cholestasis or metabolic diseases of the urea cycle. Today, patients with these orphan or ultra-orphan diseases (fewer than 2,000 patients worldwide) have conventional drug treatments that treat the symptoms but do not provide a cure.  Many are eligible for liver transplantation if treatment fails. Vivet could mimic, thanks to the new gene, a transplant with a single injection. This injection would allow the liver to restore the targeted metabolic pathways, in particular to correctly restore copper metabolism. 

“We miniaturised the gene sequence to make it as functional as the native protein, to ensure, in the case of Wilson’s disease, the homeostasis of copper metabolism and other metabolic pathways in our other indications,” Jean-Philippe Combal, CEO and co-founder of Vivet Therapeutics.

Currently in clinical trials, Vivet hopes to provide evidence of efficacy in humans in Europe and the United States within the next two years. The company signed a partnership agreement with Pfizer announced in March 2019, with an option to purchase the company, as well as a production partnership for the product targeting Wilson’s disease, VTX-801. It is within the framework of this partnership with the American pharmaceutical company and with Mirum Pharmaceuticals – which is also targeting orphan diseases – that Vivet Therapeutics was nominated for the Prix Galien Medstartup, in the category “Best cooperation dedicated to vulnerable populations or in developing countries”.