diabetes Archives - The Niche

After getting Vertex’s Zimislecel most appear cured of type 1 diabetes in small trial

June 21, 2025 / Professor Paul Knoepfler, Ph.D. / 4 Comments

In groundbreaking results, Vertex Pharmaceuticals presented and published results showing that ten out of twelve patients in a small trial exhibited signs of a cure for their type 1 diabetes after receiving the stem cell therapy Zimislecel. This is extremely exciting. While still early days for this therapy and there are some caveats (discussed below), […]

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Weekly reads: More good news on stem cells for diabetes, Panda stem cells, pricey longevity club

September 29, 2024 / Professor Paul Knoepfler, Ph.D. / 1 Comment

People often ask me what is the most promising new cell therapy and while it is difficult to choose just one, I often highlight the promise of stem cells for diabetes. Here in the U.S., early data from diabetes clinical trials by Vertex look encouraging. Other teams worldwide are also working in this space and

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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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16 predictions for stem cells & regenerative medicine in 2022

January 4, 2022 / Professor Paul Knoepfler, Ph.D. / 4 Comments

The last few years in the stem cell and regenerative medicine arena have been complicated in many ways. Variables like surging stem cell clinics and COVID have made predictions harder. Even so, trying to make predictions is useful and it’s fun to go over past year’s predictions. You can see my scorecard on my 2021

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Vertex upbeat on 1st stem cell therapy for diabetes trial participant

October 19, 2021 / Professor Paul Knoepfler, Ph.D. / 14 Comments

It’s hard in the cell therapy field not to get excited even with bits of seemingly encouraging news but with N=1 reports we have to be very cautious and that’s the case with news from Vertex on their 1st stem cell therapy for diabetes trial participant. Vertex stem cell therapy for diabetes VX-880: N=1 This

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Quick reaction to milestone ViaCyte data on a diabetes patient

June 25, 2021 / Professor Paul Knoepfler, Ph.D. / 3 Comments

VC-01-post-implant-final1-e1503703572933, viacyte

ViaCyte released encouraging data just now on a patient with implanted stem cell-derived pancreas-like device whose diabetes improved. You can read more about this in the Endpoints piece that I linked to in the previous sentence. Stem cells for diabetes; the ViaCyte update The idea of stem cells for diabetes has been more on my

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Weekly recommended science reads: stem cells, CRISPR, cancer & more

March 1, 2020 / Professor Paul Knoepfler, Ph.D. / 1 Comment

CRISPR-for-muscular-dystrophy.-Zhang-et-al.-Science-2020.-Screenshot-of-Fig.-2-featured-image

There’s never enough time to get all of one’s science reading done, but we can try! It helps to have a list of “to-read” articles, whether actual research articles or media pieces. In the old days, I remember my mentors saying they literally had “piles” of journal articles on their coffee tables, bathrooms, bedrooms, etc.

ViaCyte Q&A: CRISPR Tx quick progress, ongoing diabetes trial, & more

September 25, 2019 / Professor Paul Knoepfler, Ph.D. / 9 Comments

ViaCyte is one of the most exciting stem cell and regenerative medicine biotech companies so I like to try to check in with them regularly. Today’s post is my new interview with ViaCyte leader Paul Laikind on recent developments. We had a great chat about the science and how things are looking upbeat for the

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Quick Q&A with ViaCyte CEO on CRISPR-stem cell work

September 19, 2018 / Professor Paul Knoepfler, Ph.D. / 4 Comments

We heard this week about the encouraging news of a new CRISPR-stem cell collaboration between stem cell biotech ViaCyte and CRISPR Therapeutics. The goal of the team is to make genetically engineered pancreatic derivatives from stem cells that would have a unique immune stealthiness. If such an approach was successful, patients would not need to

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ViaCyte & CRISPR Therapeutics team up to battle diabetes

September 17, 2018 / Professor Paul Knoepfler, Ph.D. / 2 Comments

What happens when one of the most exciting stem cell biotechs ViaCyte teams up with a firm like CRISPR Therapeutics to use a combination of gene editing and cell therapy? Hopefully a synergistic partnership emerges that in this case can develop an effective stem cell-based therapy for diabetes. You can see a press release on

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