Pfizer will make a candidate gene therapy for the liver condition Wilson disease under an agreement with Vivet Therapeutics.

The US pharmaceutical firm will make supplies of the candidate – known as VTX-801 – for a Phase I/II clinical trial due to start early next year.

Wilson disease is characterized by the accumulation of copper in tissue. It is an inherited disorder caused by mutations in the gene encoding the ATP7B copper transporter.

Image: Stock/Shidlovski

VTX-801 is an AAV liver tropic capsid containing a single-stranded DNA genome carrying a shortened version of the ATP7B gene, dubbed the ATP7B-minigene. Vivet release promising preclinical data in 2019.

The candidate has been granted orphan designation by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Pfizer will make the candidate therapy at its facility in Chapel Hill, North Carolina.

Pfizer gained the plant when it paid $150 million (€136 million) for gene therapy developer Bamboo Therapeutics in 2016.

Gene relationships

Pfizer has been working with Vivet since March 2019, when it paid €45 million for a minority interest in the French Biotech.

The agreement also gave Pfizer the option to buy all outstanding shares in the French biotech after the delivery of certain data from the Phase I/II clinical trial for VTX-801.

In a press release Vivet CEO Jean-Philippe Combal said, “The manufacture of potentially transformative gene therapy technologies is an incredibly complex undertaking.

He added, “We are hopeful that this key milestone further strengthens our relationship with Pfizer while ensuring the long-term development of VTX-801.”

Vivet is one of a number of gene therapy firms Pfizer has partnered with in recent years.

It has a deal with Spark Therapeutics – for a project focus on hemophilia B – and is also working with Sangamo Therapeutics on a gene therapy for hemophilia A.