Vertex Collaborates with Verve to Discover and Develop an In Vivo Gene Editing Program for Liver Disease

Vertex Pharmaceuticals and Verve Therapeutics entered an exclusive, four-year global research collaboration focused on discovering and developing an in vivo gene editing program for a single undisclosed liver disease.

Photo: Sekar Kathiresan, co-founder and CEO of Verve

Under the terms of the collaboration, Verve will advance the discovery, research, and certain preclinical development of a novel in vivo gene editing program for the target of interest, with all program costs funded by Vertex. Vertex will be responsible for subsequent development, manufacturing and commercialization of any program stemming from Verve’s research efforts.

“This partnership with Vertex enables an important step forward for Verve as we build out our leading gene editing capabilities and pipeline of in vivo gene editing medicines to address serious diseases,” said Sekar Kathiresan, co-founder and CEO of Verve. “This agreement validates the pioneering work at Verve to develop liver-directed gene editing medicines and expands the reach of our capabilities and breadth of our pipeline.”

Under the terms of the agreement, Verve will receive an upfront payment of $60 million, including a $35 million equity investment in Verve. Verve is also eligible to receive up to $66 million in success payments, up to $340 million in development and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement.

Verve’s lead product candidate, VERVE-101, is designed to permanently turn off the PCSK9 gene in the liver in order to disrupt blood PCSK9 protein production and thereby durably reduce blood LDL-C levels, with the goal of reducing a patient’s risk for cardiovascular disease. VERVE-101 is being developed initially for the treatment of patients with heterozygous familial hypercholesterolemia, a potentially fatal genetic heart disease.

Author: Rare Daily Staff