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

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.

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What’s up with Vertex and CRISPR Therapeutics?

Here’s the news on these biotechs. Vertex cuts ties to CRISPR Therapeutics’ type 1 diabetes stem cell therapy, Fierce Biotech.  From the piece, referring to Vertex:
“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 disagreements related to stem cell clinics marketing MSCs. He was a great 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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5 thoughts on “Weekly reads: Vertex & CRISPR Therapeutics, Arnold Caplan death, MS genetics”

  1. Arnold Caplan (born Jan. 5, 1942 to Jan. 10, 2024) who discovered the MSC had an important affect on my life. Since 2018 I have been studying the secretome, which for my father had saved his life. My father had blood sepsis for nearly 2 years. All the standard medication did nothing to help my father. After one single treatment, my father had a blood test 9 days later at Watford General, his blood sepsis had gone. That was 2019 and the sepsis has not returned. RIP Arnold Caplan

  2. Bill Jones, thanks for correcting my error (“medicinal stem cells”). Yes, “medicinal signaling cells,” and more to the point of the effect of this promotion in terminology to obscure the biologically significant cell heterogeneity of the preparations.

    Kind regards,


  3. Dear Admin:

    We are advised to speak well of the dead, especially the recently dead; and I am sure that many praising eulogies for Caplan by his trainees and colleagues will follow soon in elitist research journals. But we should not speak well of the misdeeds, or if we are kinder, the missteps of the dead. This consideration is particularly applicable in the case of scientists like Caplan who are responsible for impeding progress in scientific knowledge and its medical development.

    Now that Caplan is no longer with us, perhaps the field of mesenchymal stem cell research can achieve a more physiologically valid representation of important mesenchymal tissue cell preparations. My interactions with Caplan were through reading his scientific writings, attending his more recent virtual research conferences which he sponsored, and occasional exchanges between him and me, as a member of audiences, when he pontificated in his conference presentations. He was oppressive of other ideas from his position of prominence in the field, which to his is credit, he was a major founder and contributor. In my experiences with him, he gave little consideration to ideas that differed from his own and actively suppressed other perspectives.

    Caplan publicly pronounced that his claim of having named mesenchymal tissue cell preparations to be “mesenchymal stem cells” was a mistaken appellation in light of subsequent studies showing the evident cell heterogeneity of these cell preparations. However, in the meantime, he destined this field of investigation, including professional science organizations like the International Society for Cell Therapy (ISCT), to treat these cell-heterogeneous preparations as if they were homogeneous for stem cells.

    Once admitting his error, Caplan proceeded to drive the field into turmoil with a next extreme opinion of his own. He promoted either calling the cells “medicinal stem cells,” which would ignore the important cellular biology of the cell preparations altogether; or “mesenchymal stromal cells,” with which he made a worse error for stem cell science and medicine by throwing the critical stem cells in the preparations out with his self-cleansing bath water.

    I hope that in the coming lionizations of the death of Caplan that the actual cellular biology of mesenchymal tissue cells is not further obscured by the same type of politics in science that investigators like Caplan deployed to promote their own ideas above the broader and richer work and ideas of the greater scientific research community.


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