Vertex’s VX-880, an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy for T1D, has already achieved proof-of-concept with ‘highly promising’ safety and efficacy results from an ongoing Phase 1/2 study which continues to enroll and dose patients.
The acquisition of ViaCyte provides Boston-headquartered Vertex - whose approved medicines to date are for cystic fibrosis - with the tools to accelerate its work in T1D. Through the acquisition, it will gain complementary assets, capabilities and technologies including additional human stem cell lines, intellectual property around stem cell differentiation, and Good Manufacturing Practice (GMP) manufacturing facilities for cell-based therapies that could accelerate Vertex’s ongoing T1D programs.
The acquisition also provides access to novel hypoimmune stem cell assets via the ViaCyte collaboration with CRISPR Therapeutics.
“VX-880 has successfully demonstrated clinical proof of concept in T1D, and the acquisition of ViaCyte will accelerate our goal of transforming, if not curing T1D by expanding our capabilities and bringing additional tools, technologies and assets to our current stem cell-based programs,” said Reshma Kewalramani, M.D., CEO and President of Vertex.
San Diageo headquartered ViaCyte is a private cellular therapy company with a clinical-stage stem cell platform that delivers therapeutic proteins. The company has significant clinical experience in patients with T1D; this includes a first-in-class gene-edited, immune-evasive investigational islet cell replacement therapy for diabetes that could potentially eliminate the need for exogenous insulin without requiring immunosuppression.
ViaCyte has received support for its research from JDRF and the California Institute of Regenerative Medicine and has established collaborative partnerships with leading companies, including CRISPR Therapeutics, to advance its therapies for T1D.
“ViaCyte’s commitment to finding a functional cure for T1D is shared by Vertex, and this acquisition will allow Vertex to deploy ViaCyte’s tools, technologies and assets toward the development of Vertex’s multiple cell replacement therapy approaches designed to reduce the burden of millions of people living with T1D worldwide,” said Michael Yang, President and CEO of ViaCyte.
The acquisition – for $320m in cash – will close later this year subject to certain conditions.
Vertex has multiple approved medicines that treat the underlying cause of cystic fibrosis and several ongoing clinical and research programs in this area. Type 1 diabetes is one of the focuses in its pipeline of investigational small molecule, cell and genetic therapies: which also cover sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.