Vertex Subsidiary ViaCyte Has Opted Out of Collaboration with CRISPR Therapeutics for Diabetes Treatment
10 January 2024
CRISPR Therapeutics recently released a press release highlighting its strategic priorities and 2024 outlook.
Samarth Kulkarni, PhD, Chairman and CEO of CRISPR Therapeutics reflected on the ‘landmark year in 2023’ which marked the ‘first-ever approval of a CRISPR-based gene-editing therapy in addition to entering the clinic with our in vivo therapies.’ Kulkarni claims that the company is ‘well positioned to execute on [our] clinical trials across various therapeutic areas including oncology, autoimmune, cardiovascular and diabetes, setting up a catalyst-rich 12-18 months for the company.’
However, the company’s outlook for 2024 featured an announcement that ViaCyte, Inc., a subsidiary of Vertex Therapeutics, Inc. has decided to opt out of the co-development and co-commercialization collaboration with CRISPR Therapeutics, effective in early February. According to the opt-out terms, CRISPR Therapeutics will now own all collaboration assets and pay royalties to ViaCyte for future sales. The ViaCyte collaboration assets include VC-01™ (formerly VCT01™). This allogeneic, gene-edited, immune-evasive, stem cell-derived product candidate is transplanted into patients in a device and is intended to produce insulin in a glucose-dependent manner.
CRISPR Therapeutics is advancing a Phase I clinical trial of CTX211 to treat Type I Diabetes (T1D). The company is committed to developing a beta-cell replacement product that doesn’t require chronic immunosuppression.
CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of T1D, which incorporates gene edits that aim to enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose.
Excluding the collaboration with ViaCyte, Vertex retains non-exclusive rights to specific CRISPR Therapeutics’ CRISPR/Cas9 technology aimed at accelerating the development of potentially curative cell therapies for Type I Diabetes (T1D). In 2023, Vertex made upfront and milestone payments totaling $170 million to CRISPR Therapeutics under the licensing agreement. Additionally, CRISPR Therapeutics stands to earn an extra $160 million in research and development milestones, along with royalties on any future products resulting from the collaboration.
In the fourth quarter of 2023, regulatory approvals were secured for CASGEVY in the United States for the treatment of sickle cell disease (SCD), as well as in Great Britain and Bahrain for the treatment of both SCD and transfusion-dependent beta thalassemia (TDT). Furthermore, a positive opinion was received from the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) for CASGEVY in the treatment of both SCD and TDT. Notably, Exa-cel, the inaugural therapy resulting from the strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals, is under the global development, manufacturing, regulatory, and commercialization leadership of Vertex, with support from CRISPR Therapeutics.
Xavier De Mollerat Du Jeu, Senior Director R&D at Thermo Fisher Scientific is accompanied by Gianluca Pettiti, Executive Vice President and President, Life Sciences Group at Thermo Fisher Scientific and Fred Parietti, Co-Founder and CEO at Multiply Labs for this Workshop at Advanced Therapies Week 2024.
Manufacturing capacity, complexity and cost are core constraints that limit commercialization of cell and gene therapies, and ultimately patient access. In this Fireside Chat series, join Georgi Makin, VP of Digital & Editorial at Phacilitate and the Germfree team as we discuss the challenges and opportunities associated with delivering cell therapies in a decentralized manufacturing model.