Medical Innovation Exchange

JPM 24: CRISPR Tx on active deal hunt but director cautions it's 'still early days’

Some four 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 green lit 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. 

https://www.fiercebiotech.com/biotech/jpm-24-crispr-tx-investor-its-still-early-days-sealing-new-deals

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