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Weekly reads: Vertex, stem cells for MS, Athersys, 900-day paper review at Nature

March 3, 2024 / Professor Paul Knoepfler, Ph.D. / 1 Comment

“While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression,” said Bastiano Sanna, Ph.D., executive vice president and chief of cell and genetic therapies at Vertex.

Vertex Pharmaceuticals has a lot going for it right now on the cell therapy front. For instance, they have the strongest type 1 diabetes cell therapy pipeline after some recent acquisitions. Still it’s not a simple matter to succeed in the cell therapy space even with one therapy for one targeted disease. Trials are tough […]

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9th Circuit hearing on FDA appeal of Cell Surgical Network case: maybe encouraging?

February 7, 2024 / Professor Paul Knoepfler, Ph.D. / 5 Comments

I was able to watch some of the 9th Circuit Court hearing today on the FDA appeal of its loss in the Cell Surgical Network lawsuit. This post is my brief impressions as a scientist. What went down? Do the three judges seem to be leaning one way or another? The ruling in this case

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Perspectives 10 years after STAP cells: the culture of science, misconduct, & hopes for progress

January 29, 2024 / Professor Paul Knoepfler, Ph.D. / 6 Comments

Exactly ten years ago today, on January 29, 2014, I wrote about two new Nature papers on so-called STAP cells. The papers claimed that stress alone could convert regular non-stem cells into some of the most powerful stem cells. More specifically, the authors claimed to make pluripotent stem cells similar to iPS cells this way.

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

January 14, 2024 / Professor Paul Knoepfler, Ph.D. / 5 Comments

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. 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

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Weekly reads: Verve Therapeutics, Casgevy UK OK, GDNF, MYC

November 19, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Some folks can view data from early, small clinical trials too skeptically or overly enthusiastically, and maybe that’s going on with some preliminary results from Verve Therapeutics. Good news? Bad news? I’m going to start with two articles about the same news that have very different vibes. What do I think? On the whole, I

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3 cell therapy companies doing well: Vertex, Vericel, & Regeneron

November 15, 2023 / Professor Paul Knoepfler, Ph.D. / 5 Comments

Cell therapy companies face many hurdles. I wrote recently about four cell therapy biotechs that have been really struggling. Blogging here at The Niche for more than a dozen years, I’ve seen many companies even go under. That’s bad news for the field, for patients, and for investors. Today I wanted to highlight some cell therapy

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Weekly reads: Conception Bio & IVG, MYC, Stanford Prez resigns

July 23, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

The Bay Area biotech Conception Bio has an intriguing name. What does “conception” mean here? Scientists can make many things from stem cells. For example, lately I’ve written a lot about stem cell-based human embryo models. Another intriguing area is called in vitro gametogenesis or IVG, where Conception Bio is a leader. IVG means making

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Weekly reads: cell therapy trials, melanoma, β-thalassemia

June 4, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

I’m kind of in grant writing hell at the moment, as many of you may have been at various times, but I’m still finding time to read a little on cell therapy and stem cell research. Here are some things that caught my eye as I work on multiple grants with impending deadlines. Clinical trials

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Weekly reads: big CRISPR mtg, Mesoblast good news, Neuralink spiked

March 12, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Where do things stand with potential applications of CRISPR and other gene editing technologies in patients? Overall, things are looking very positive. CRISPR human trials This week the third big international human genome editing meeting took place in London. The summit addressed numerous potential clinical applications. It was good to see the agenda included quite

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Stem cell clinic ‘doc’ sentenced to 202 years in prison

January 17, 2023 / Professor Paul Knoepfler, Ph.D. / 4 Comments

Jimmy Lee Taylor going, stem cell clinic

I had heard that we might see criminal charges or convictions in 2022 for unproven stem cell clinic activities. This led to my prediction for last year of a criminal conviction in the stem cell clinic sphere. The prediction was correct as a Florida man selling unproven stem cell injections was been sentenced in late December

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