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  • Mass General Brigham Ventures

In Quest for Liver Disease Gene Therapy Vertex Invests $340M in Verve

Massachusetts-based biopharmaceutical companies Vertex and Verve Therapeutics announced Wednesday that they had entered into an exclusive four-year research partnership to discover and nurture a gene editing therapy for a yet-undisclosed liver disease.

The agreement will see Vertex make an upfront payment of $60 million to Verve, including a $35-million equity investment. In return, Verve will be in charge of all research work, including drug discovery and preclinical development of the in vivo gene editing program for their target of interest, all of which will be funded by Vertex.

Vertex will then be in charge of subsequent clinical studies, further development, manufacturing and eventual commercialization of the resulting candidate. Verve will continue to reap the benefits of its research efforts, as it will remain eligible to receive up to $66 million in success payments, up to $340 million in development and commercial milestones and tiered royalties on future sales.

“Vertex is committed to discovering and developing transformative medicines for people with serious diseases,” David Altshuler, M.D., Ph.D., executive vice president, global research and chief scientific officer of Vertex said in a statement. “We are impressed by the progress Verve has made and look forward to combining the expertise in gene editing and drug development of our two companies to serve more patients in need.”

Vertex's Quest for Liver Disease Therapy

Yesterday’s deal represents the latest in Vertex’s quest to establish its presence in the liver disease space. The company was banking its hopes on small molecule candidate VX-864 for alpha-1 antitrypsin deficiency (AATD), a rare genetic disease connected to the lungs and the liver. In June of last year, the candidate met its Phase II primary endpoints and proved well-tolerated but at magnitudes unlikely to translate to clinical benefit. Vertex decided to forego late-stage development.

After its setback with VX-864, Vertex is back in its early research phase for AATD. Currently, the company lists no dedicated candidates for liver diseases.

Gene therapies treat or prevent diseases by addressing their genetic roots. Often, this approach sees the introduction of a healthy and functional copy of a gene (or a new gene altogether) into the patients’ cells to fight their illnesses. More recently, doctors have been afforded more flexibility with the advent of the game-changing CRISPR technology. This has allowed them to fix disease-causing mutations, turn off a faulty gene, turn on a dormant gene or even remove a piece of DNA that somehow interferes with a gene’s normal function.

While gene therapies have been widely used in other therapeutic areas, their uptake in liver diseases has been relatively slow. Most recently, Germany’s CEVEC Pharma and its South Korean partner RZNOMICS announced that their candidate RZ001, which uses a proprietary trans-splicing ribozyme technology for primary liver cancer, was given Investigational New Drug approval by South Korea’s chief regulatory body. Senti Bio also recently revealed data suggesting its proprietary Gene Circuit technology could be promising against hepatocellular carcinoma.

The field is also wide open for Vertex outside of liver cancers. Last May, Precision BioScience announced that its in vivo gene editing program strongly suppressed hepatitis B infection. A month before, China-based Shenzhen HighTide Biopharmaceutical successfully delivered the first Phase II dose of HTD1801, its candidate for patients with type 2 diabetes and nonalcoholic fatty liver disease.

Source: BioSpace

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