Vertex Terminates Gene Therapy Partnership with Verve, Highlighting Industry Challenges

Vertex Pharmaceuticals has ended its collaboration with Verve Therapeutics on liver gene therapies, marking another setback in the gene therapy sector. The termination comes two years into a four-year agreement that was initially valued at up to $400 million.

Partnership Dissolution and Industry Trends

Verve Therapeutics announced the split in a recent statement, citing Vertex's decision to terminate the research collaboration "due to changing priorities within its development portfolio." The partnership, established in 2022, aimed to develop in vivo gene editing treatments for an undisclosed liver disease.

This development follows a series of challenges in the gene therapy field, including recent layoffs at companies such as Editas, Intellia, and Encoded. Additionally, Pfizer recently discontinued its hemophilia gene editing treatment, Beqvez, citing market disinterest and the high costs associated with gene therapies.

Verve's Ongoing Programs and Partnerships

Despite the setback with Vertex, Verve Therapeutics continues to advance its gene editing programs. The company's lead candidate, VERVE-102, targets the PCSK9 gene in liver cells to reduce LDL cholesterol levels in patients with heterozygous familial hypercholesterolemia (HeFH). VERVE-102 is currently in a Phase Ib trial, with data expected in the second quarter of 2025.

Verve maintains other strategic partnerships, including a $60 million deal with Eli Lilly in 2023 for rights to its PCSK9-targeting program. CEO Sekar Kathiresan stated that the company is preparing to deliver an opt-in package to Lilly, with a decision anticipated in the second half of 2025.

Mixed Signals in Gene Therapy Progress

While the gene therapy sector faces challenges, there have been some positive developments. Intellia Therapeutics reported significant reductions in angioedema events with its NTLA-2002 treatment, and Regeneron recently presented promising data on a treatment for otoferlin-related deafness.

However, the high cost of gene therapies remains a significant hurdle. Pfizer's discontinued Beqvez treatment was priced at $3.5 million per dose, highlighting the economic challenges facing the industry and potential barriers to market adoption.

As the gene therapy landscape continues to evolve, companies in the sector face the dual challenges of advancing complex scientific programs and navigating a challenging market environment. The termination of the Vertex-Verve partnership underscores the ongoing volatility and realignment within this cutting-edge field of medicine.