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Can CRISPR Finally Cure Type 1 Diabetes for Good?

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Understanding Diabetes and Its Challenges

Diabetes exists in two forms. Type 2 diabetes is the most common form and is mostly a metabolic disease, stemming from unhealthy lifestyles and obesity, where the body’s cells stop reacting to insulin properly.

A more dangerous form of diabetes is Type 1, where the immune system attacks its own cells, especially the islets of Langerhans in the pancreas, which are responsible for the production of insulin. As a result, this disease was 100% deadly until the invention of insulin injections.

Source: Nature

Insulin is, however, not a magical solution, as it requires constant monitoring of blood sugar, and still does not remove all the potential complications of type 1 diabetes. It also mostly starts in childhood, drastically affecting the quality of life of patients at an early age.

This is why a more durable cure would be ideal. Some biotech companies are making progress, with implantation of insulin-producing cells extracted either from organ donors, or from lab-grown stem cells.

However, these methods do not remove the tendency of the patient’s immune system to destroy these pancreatic cells. So to make the treatment “stick”, they also need to take immunosuppressant drugs.

While it protects the implanted cells, suppressing immune activity carries risks of infection, cancer, and other serious side effects.

For now, there are no credible therapeutic paths to reprogram the immune system to stop the autoimmune syndrome. But a new method is emerging, where the implanted pancreas cells are modified genetically to not trigger an immune response, removing the need for immunosuppressants.

The prestigious scientific journal Nature reports new progress for this concept, in an article titled “Hope for diabetes: CRISPR-edited cells pump out insulin in a person — and evade immune detection”.

New Approaches to Treating Type 1 Diabetes

Restoring Functions, But With Caveats

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Company Approach Stage Key Limitation
Vertex Pharmaceuticals Stem-cell-derived islets Clinical (12 patients) Requires immunosuppression
Reprogenix Bioscience Islets from reprogrammed fat cells Preclinical/Early reports Still immune destruction risk
Sana Therapeutics CRISPR-edited hypoimmune cells Early (1 patient) Insulin output too low; needs scaling

As mentioned, some progress has been made in the implantation of insulin-producing cells.

Vertex Pharmaceuticals (VRTX -0.29%) is leading the charge, after a series of acquisitions:

  • First, in 2019, acquiring Semma Therapeutics, a startup founded on patents developed by Doug Melton at Harvard University.
  • Secondly, with the acquisition of ViaCyte in 2021, injecting pancreas progenitor stem cells into the pancreas.

In June 2025, Vertex reported that it had transplanted embryonic stem-cell-derived islets into 12 people with type 1 diabetes, one year ago. Out of 12 people, 10 participants no longer required insulin injections. The company plans to seek regulatory approval for this cell therapy next year.

Similarly, Reprogenix Bioscience in Hangzhou, China, is creating islets from reprogrammed stem cells derived from a recipient’s own fat tissue, with early reports of success.

These approaches still require immunosuppression, even in the case of using the patient’s own cells, as the immune system destroying the insulin-producing cells is what caused the disease in the first place.

With 9.4 million children and adults having type-1 diabetes in 2025, and 16.4 million expected by 2040, causing 201,600 deaths per year, a more durable treatment is required.

Can CRISPR Enable Full Recovery?

A different approach is being taken by Sana Biotechnology (SANA -5.04%), a startup based in Seattle, Washington.

In a breakthrough announcement, Sana declared that they have implanted CRISPR-edited pancreas cells into a person with type 1 diabetes.

The genetically modified cells pumped out sugar-regulating insulin for months without the need for the recipient to take immune-dampening drugs.

“The preliminary data has definitely lifted the spirits of our community — and it’s a really elegant approach,”

Aaron Kowalski – Chief executive of Breakthrough T1D, a non-profit organization

They detailed their results in the New England Journal of Medicine1, under the title “Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression”.

Is It Truly Enough?

Other scientists are a little less enthusiastic about the announcement. They point out a few key limitations to these preliminary results.

First, the study covered only one person, making it hard to understand if the protocol is reliable enough for mass deployment.

Another issue is that the patient received a low dose of the cells, leading to too low insulin production, so not removing (yet?) the need for taking insulin.

Still, a very important step seems to have been achieved, which is to create a genetic modification that hides the implanted cells from the immune system.

Investing in CRISPR Solutions

Sana Therapeutics

Sana Biotechnology, Inc. (SANA -5.04%)

Sana Therapeutics was founded in 2019 by former Juno Therapeutics executives Hans Bishop and Steve Harr.

Juno Therapeutics was an immunotherapy company acquired by Celgene for $9 billion, itself acquired by Bristol-Myers Squibb (BMS) a year later for $74B.

Sana’s modified hypoimmune” cells are designed to avoid both parts of the immune system: the adaptive and innate systems.

To avoid the lymphocytes of the adaptive system, they modified the HLA-1 & 2 markers. But alone, this would just make the cells more sensitive to attack by the innate system.

So they also make the cell overexpress a protein called CD37. This protein serves as a protective “do not kill me” signal that prevents immune watchdogs, known as natural killer cells, from attacking the edited cells.

The next stage for the company is to not use organ donor cells, but to produce the same type of hypoimmune cells from stem cells, allowing for mass production.

This would bring the company closer to a commercially viable product, but clinical trials will likely not start before 2027 and will take several years after that.

This concept might have a lot of potential beyond diabetes as well. No less than >75 different types of autoimmune disorders are driven by immune B-cell pathology.

If the strategy to create hypoimmune cells can be applied to other types of cells than pancreatic cells, this could be used to save the lives of more than 5 million other patients.

In the same way, allogenic CAR-T cells used in cancer treatment could benefit from that technology as well. The company’s SC291 research program, now in phase I of clinical trials, is looking at the potential of this concept.

Overall, the preliminary results of Sana Therapeutics are very promising and might outmatch even the impressive results of the early clinical trials of Vertex, thanks to this method not needing immunosuppressants.

However, investors need to be aware that the company is still at a very early stage, many years before reaching an FDA-approved therapy.

Considering the average failure rate of drug development and clinical trials, this means the company’s stock carries an inherent risk tied to potential medical or technical hiccups down the road.

Latest Sana Therapeutics (SANA) Stock News and Developments

References:

1. Per-Ola Carlsson, Xiaomeng Hu, Ph.D., et al. Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression. New England Journal of Medicine. VOL. 393 NO. 9. (2025) DOI: 10.1056/NEJMoa2503822

Jonathan is a former biochemist researcher who worked in genetic analysis and clinical trials. He is now a stock analyst and finance writer with a focus on innovation, market cycles and geopolitics in his publication 'The Eurasian Century".

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