CRISPR Therapeutics

Type 1 diabetes therapy gets go-ahead for clinical trial

/ Kevin McCormack / 1 Comment
ViaCyte’s implantable cell-based therapy for type 1 diabetes

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Taking even the most promising therapy and moving it out of the lab and into people is an incredibly complex process and usually requires a great team. Now, two great teams have paired up to do just that with a therapy for type 1 diabetes (T1D). ViaCyte and CRISPR Therapeutics have put their heads together and developed an approach that has just been given clearance by Health Canada to start a clinical trial.

Regular readers of this blog know that CIRM has been a big supporter of ViaCyte for many years, investing more than $72 million in nine different awards. They have developed an implantable device containing embryonic stem cells that develop into pancreatic progenitor cells, which are precursors to the islet cells destroyed by T1D. The hope is that when this device is transplanted under a patient’s skin, the progenitor cells will develop into mature insulin-secreting cells that can properly regulate the glucose levels in a patient’s blood.

One of the challenges in earlier testing was developing a cell-based therapy that could evade the immune system, so that people didn’t need to have their immune system suppressed to prevent it attacking and destroying the cells. This particular implantable version sprang out of an early stage award we made to ViaCyte (DISC2-10591). ViaCyte and CRISPR Therapeutics helped with the design of the therapeutic called VCTX210.

In a news release, Michael Yang, the President and CEO of ViaCyte, said getting approval for the trial was a major milestone: “Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies. This approach builds on previous accomplishments by both companies and represents a major step forward for the field as we strive to provide a functional cure for this devastating disease.”

The clinical trial, which will be carried out in Canada, is to test the safety of the therapy, whether it creates any kind of reaction after being implanted in the body, and how well it does in evading the patient’s immune system. In October our podcast – Talking ‘Bout (re)Generation – highlighted work in T1D and included an interview with Dr. Manasi Jaiman, ViaCyte’s Vice President for Clinical Development. Here’s an excerpt from that podcast.

Dr. Manasi Jaimin, ViaCyte VP Clinical development

New partnership to make CIRM supported treatment for type 1 diabetes even better

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ViaCyte images

ViaCyte’s PEC-Direct device. Image courtesy of ViaCyte

ViaCyte, a regenerative medicine company long backed by CIRM, announced a partnership with CRISPR Therapeutics to increase the number of people with Type 1 Diabetes (T1D) who could benefit from their PEC-Direct therapeutic implant.

Last year, CIRM granted ViaCyte $20 million to facilitate development of PEC-Direct, a device that both transplants pancreatic progenitor stem cells (the immature version of  islet cells, the insulin-producing cells that are destroyed in TID), and allows those cells to connect to the patient’s bloodstream to help them function more like normal islet cells. This treatment, currently in clinical trials, was initially targeted towards high risk patients because of the need to treat them with immunosuppressive therapy, to ensure that the patient’s immune system does not attack the implanted cells.

ViaCyte’s partnership with CRISPR Therapeutics aims to eliminate the need for immunosuppressive therapy by engineering the transplanted stem cells to evade the immune system prior to implanting in the patient. CRISPR Therapeutics is already using this gene editing approach in CAR-T based cancer therapies and has developed an important knowledge base in “immune-evasive gene editing.” Paul Laikind Ph.D., CEO and President of ViaCyte explains the importance of this partnership in a news release:

“Creating an immune-evasive gene-edited version of our technology would enable us to address a larger patient population than we could with a product requiring immunosuppression. CRISPR Therapeutics is the ideal partner for this program given their leading gene editing technology and expertise and focus on immune-evasive editing.”

Samarth Kulkarni, Ph.D., and CEO of CRISPR Therapeutics adds:

“We believe the combination of regenerative medicine and gene editing has the potential to offer durable, curative therapies to patients in many different diseases, including common chronic disorders like insulin-requiring diabetes.”

The hope is that this new approach could make this treatment available to everyone with T1D. The benefits of such a treatment option would be considerable as TID affects around 1.25 million Americans, and can lead to severe health complications such as kidney damage and heart disease. The initial goals of this collaboration are to develop a stem cell line that successfully evades the immune system, followed by developing a product that can be used in patients.