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Weekly reads: Vertex & CRISPR Therapeutics, Arnold Caplan death, MS genetics – The Niche

The biotechs Vertex and CRISPR Therapeutics have an interesting relationship as biotechs. They are partners are multiple levels but also are very different as companies including in size.

CRISPR Therapeutics
CRISPR Therapeutics scientists including CEO Samarth Kulkarni (second from left).

There’s been a key development in one of their partnerships.

Before we jump into that, please check out the video version of my 20 stem cell and regenerative medicine predictions for 2024 below. Subscribe to our YouTube channel too as we head for 1,000+ subscribers!

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What’s up with Vertex and CRISPR Therapeutics?

“The big biotech has chosen to opt out of the diabetes gene-edited stem cell therapy it gained through the acquisition of ViaCyte, leaving CRISPR to take the clinical-phase program forward itself.”
It’s not clear why Vertex isn’t pursuing this area in addition to its other diabetes efforts. Vertex acquired ViaCyte soon after also buying the other key cell therapy diabetes biotech Semma. At that point one possible concern was that some of the promising, unique diabetes approaches at ViaCyte might be put on the back burner. It’s hard to say what’s up with the former ViaCyte stuff now at Vertex. At least it seems that CRISPR Therapeutics will continue this line of gene-editing-based diabetes research. Also, CRISPR and Vertex are still partners in other ways:
“Vertex also remains active in diabetes cell therapy, including in partnership with CRISPR. In March, Vertex paid $100 million for non-exclusive rights to its partner’s CRISPR-Cas9 technology in hypoimmune cell therapies for type 1 diabetes. CRISPR received a further $70 million from Vertex later in the year and is still in line to receive up to $160 million in R&D milestones.”

Arnold Caplan death

The stem cell field lost one of the early pioneers recently when Professor Arnold Caplan passed away on Jan. 10. Caplan was considered the father of the mesenchymal stem cell or MSC field. He and others later felt that the MSC acronym should not be defined simply as stem cells since the cultures are somewhat heterogeneous. Other meanings for the MSC acronym began to be used.

I got to know him a little bit over the years and some good conversations. I had a long interview with him for The Niche about a decade ago. We even had some mild disagreements related to stem cell clinics marketing MSCs. He was a wonderful person to talk with about anything related to stem cells. Arnold was the kind of person you felt would just be around forever and him dying at 82 is a bit of a shock. It seems way too young.

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