ViaCyte, Inc.

CRISPR Tx starts 2024 with a shifting set of priorities following the approval of exa-cel. Some three months after the company first disclosed partnership interest, CRISPR Therapeutics board member and SR One CEO Simeon George, M.D., says it’s “still early days” for sealing new deals.  George’s level-setting brings back down to earth the speed at which CRISPR Tx is likely to land suitors for backburner pipeline assets even as CEO Sam Kulkarni, Ph.D., maintains an aggressive slate of business development meetings at this year's J.P. Morgan Healthcare Conference. “Part of the challenge, if I'm honest, is we have shown how good we are at taking this technology and moving it forward,” George said in an interview with Fierce Biotech Tuesday. But peers in the space have not, he added.  CRISPR Tx recently made the jump to a commercial-stage company following the historic FDA approval for exa-cel in sickle cell, now marketed as Casgevy. But the gene editing biotech has already run into some serious growing pains. The company laid off 10% of its staff a week after FDA advisors all but greenlit exa-cel ahead of the FDA approval. Then, Chief Medical Officer Phuong Khanh Morrow, M.D., resigned six days after the FDA's nod. And earlier this week, Vertex-run ViaCyte announced it was opting out of a collaboration on CRISPR Tx’s gene-edited stem cell therapy for diabetes.  Kulkarni said during the company's J.P. Morgan presentation on Tuesday that ViaCyte's decision provides "operational flexibility and ease" to focus on other Vertex-partnered diabetes work.  With respect to new deals, George says that there needs to be a “meeting of the minds” on potential terms and CRISPR Tx’s confidence that a partner could take forward a program the gene editor doesn’t have the time or money to advance itself. Kulkarni said during his presentation that he had to rejig his schedule to make time for more partnership talks.  "Almost every pharma company now wants to have a cell and gene therapy strategy," he said.  Leading up to the exa-cel approval, CRISPR Tx had teased an openness to partnering the off-the-shelf cell therapy and in vivo programs. Alex Harding, head of the biotech's business development effort, told Fierce Biotech in October that pharmas expressed interest in the cell therapy programs should they show efficacy in either autoimmune diseases or solid tumors. CRISPR is testing its off-the-shelf programs to show just that.  Kulkarni told the conference crowd that auto-immune disease could be a huge opportunity for pharma partnerships.  For George, this new chapter will be similar to the last, underscoring CRISPR’s natural move to a new set of goals post-approval. “The way we think about the goals and deliverables now, it's shifting from the focus on exa-cel now to these next sets of programs and the resourcing to support those,” he said. “And then ultimately the company needs to be held accountable for those deliverables.”  Annalee Armstrong contributed reporting. 

Pictured: Entrance to Vertex's office in Boston, Massachusetts/iStock, hapabapa Vertex Pharmaceuticals’ subsidiary ViaCyte Inc. is not pushing through with its diabetes deal with CRISPR Therapeutics, the Swiss biotech announced Monday. The partners—which had recently won the FDA’s approval for Casgevy (exagamglogene autotemcel), the first CRISPR-based gene therapy for sickle cell disease—were supposed to advance a gene-edited stem cell therapy for diabetes. Vertex’s opt-out of the deal will take effect in early February 2024. As per the original agreement, which was signed in September 2018 between ViaCyte and CRISPR, the biotechs would combine ViaCyte’s stem cell expertise with CRISPR’s gene editing technologies to develop a potential beta-cell replacement product that could help patients produce their own insulin without inducing an immune reaction. In February 2022, the partners dosed the first patient in their Phase I trial of this allogeneic gene-edited cell replacement therapy, dubbed VCTX210. Vertex bought ViaCyte a few months later, in July 2022, for $320 million in cash. At the time, CEO and president Reshma Kewalramani said that the acquisition would “accelerate our goal of transforming, if not curing T1D.” VCTX210, along with ViaCyte’s platform, was meant to complement Vertex’s own diabetes portfolio, headlined by VX-880, likewise a stem-cell derived insulin-producing islet cell therapy. Now, because Vertex is backing out of the partnership, all ongoing collaboration assets will be completely owned by CRISPR, though ViaCyte will still be eligible for royalties on net future sales. Outside of the ViaCyte collaboration, Vertex is still partnered with CRISPR for a non-exclusive license to the latter’s CRISPR/Cas9 platform to develop a potentially curative cell treatment for type 1 diabetes. This agreement was signed in March 2023 and saw Vertex pay $100 million upfront, as well as pledge up to $230 million in research and development milestones, plus royalties. ViaCyte opting out of the CRISPR partnership comes one day after Vertex Pharmaceuticals announced that it had paused the Phase I/II study of VX-880 following two patient deaths. The company had found that the mortalities were unrelated to the study drug and said that the suspension was “protocol-specified.” Global regulatory authorities and an independent data monitoring committee will review the data from that study. Aside from VX-880, Vertex is also advancing VX-264 for type 1 diabetes, for which it is currently in Phase I/II assessments. The candidate uses stem cell–derived islet cells encapsulated in a protective device, which in turn will be implanted into a patient and help produce insulin. According to Vertex’s website, the device protects VX-264 from the immune system, thereby eliminating the need for immunosuppressive therapy. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Vertex Pharmaceuticals’ subsidiary ViaCyte is backing out of one of its collaborations with CRISPR Therapeutics on gene-edited stem cell therapies for diabetes. CRISPR said Monday that ViaCyte has decided to opt-out of a co-development and co-commercialization agreement for CTX211, its beta cell replacement therapy “that is transplanted into patients in a device and intended to produce insulin in a glucose-dependent manner.” While CRISPR in February will wholly own the candidate again, ViaCyte will be paid a royalty on future sales. You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.