CRISPR Therapeutics and Vertex Pharmaceuticals have signed a non-exclusive licensing agreement for the use of CRISPR Therapeutics’ gene editing technology, known as CRISPR /Cas9. Under the terms of their agreement, the Zug based biotech will receive a $100M upfront payment plus milestone and royalty payments on potential future gene-edited hypoimmune T1D products.
Based on its revolutionary gene editing technology, CRISPR/Cas9, that allows for precise, directed changes to genomic DNA, CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, the company has established strategic collaborations with leading companies, including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc.
In a partnership deal, global biotech company Vertex Pharmaceuticals has acquired exclusive rights to CRISPR /Cas9 to accelerate the development of its hypoimmune cell therapies for type-1 diabetes (T1D). Vertex will pay CRISPR Therapeutics $100 million up-front. The Swis startup will also be eligible for up to an additional $230 million in research and development milestones and receive royalties on any future products resulting from this agreement.
CRISPR and ViaCyte, Inc., which was acquired by Vertex in 2022, will continue to collaborate on their existing gene-edited allogeneic stem cell therapies, using ViaCyte cells, for the treatment of diabetes under the terms of their collaboration. A Phase 1/2 study of VCTX211, an allogeneic, gene-edited, stem cell-derived product candidate for T1D, which originated under the CRISPR Therapeutics and ViaCyte collaboration, has been initiated and is on-going. CRISPR Therapeutics will not obtain any interest in Vertex’s pre-existing pipeline of T1D products, including VX-880 and VX-264.
Alongside its headquarters in Zug, CRISPR Therapeutics has a wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom.
"We are pleased to expand our long and successful relationship with Vertex with this collaboration which fully leverages our gene editing platform to develop hypoimmune cell therapies for T1D,” said Samarth Kulkarni, Chief Executive Officer of CRISPR Therapeutics. “In parallel, we continue to expand our capabilities in regenerative medicine and advance our existing allogeneic gene-edited cell therapy programs.”
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