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 Endocrinology specialty topic page houses video interviews with key opinion leaders in the field of endocrinology about the latest relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for endocrine disorders. It also contains up-to-date clinical news coverage in the field of endocrinology gene and cell therapy.

Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.

Considerations for Gene Therapy Logistics and Implementation

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. 

Around the Helix: Cell and Gene Therapy Company Updates – May 15, 2024

Around the Helix: Cell and Gene Therapy Company Updates – May 8, 2024

Around the Helix: Cell and Gene Therapy Company Updates – May 1, 2024

Another recent program in the investigational landscape was Helixmith’s deprioritized Engensis diabetic neuropathy gene therapy. 

First Patient With Diabetic Foot Ulcers Screened for SkinTE Pivotal Trial 

The cofounder, chairman, and CEO of Kriya Therapeutics discussed the company’s goal of bringing gene therapy to a much broader population of patients.

Shankar Ramaswamy, MD, on Bringing Gene Therapy to Common Diseases

“The technology has really matured to a point where we can take that next leap as a field to bring gene therapies into a broader universe of diseases. I think we as a field need to figure out how we can scale the technology, how we can bring down the cost, and how we can improve throughput and quality of the products that we engineer and manufacture. But those are solvable challenges.”

Gene therapy, particularly adeno-associated virus vector-based gene therapy, first gained its foothold in the field of medicine by targeting relatively rare inherited disorders like Duchenne muscular dystrophy and hemophilia A, both of which are now among the indications for the 6 FDA-approved AAV vector-based gene therapies. Indeed, this trend has continued, with many companies and academic institutions continuing to pursue the development of AAV vector-based gene therapies for rare and even ultrarare genetic disorders. Because of the high cost and lengthy and complex manufacturing process for gene therapy products, targeting smaller patient populations for both clinical trials and commercial products is in some ways a good fit for the modality. That said, gene therapy may have the potential to treat much more common diseases, as well. One company currently seeking to bring AAV vector-based gene therapy to common diseases is Kriya Therapeutics. Kriya gave 6 presentations regarding their work on this endeavor at 

the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting

, held May 7 to 10, 2024, in Baltimore, MD.

® sat down with Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics, to learn more about the company’s efforts. Ramaswamy explained that gene therapy has significant potential to address unmet needs for relatively common diseases like type 1 diabetes and geographic atrophy, but that bringing gene therapy to such indications will require overcoming challenges such as scaling manufacturing, reducing costs, and characterizing products. He noted that Kriya is currently working on technologies and methods to address these concerns, and gave his view that gene therapy is on the precipice of reaching much broader use in clinical practice.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.

Videos

Head of Seattle Children's Research Institute's Center for Immunity and Immunotherapies discussed overcoming limitations of Treg cells via genetic engineering

David Rawlings, MD, on the Potential of Engineered Treg Cells to Treat Autoimmune Diseases

“Putting this all together: The concept of therapy with regulatory T-cells is that if you can target them to the autoimmune tissue of interest—for example, the islet in type 1 diabetes, where the autoimmune process is taking place—then you can have a tissue-specific therapeutic that that turns off an autoimmune process.”

T-regulatory (Treg) cells have the natural ability to dampen immune responses, and as such are of great interest for potential use in the treatment of autoimmune diseases. Although, a number of factors makes the utilization of unmodified Treg cells unlikely to be effective as the basis of a cell therapy. For example, Treg cells require the presence of interleukin-2 (IL-2) in order to remain active and polyclonal Treg cells have limited efficacy. 

In an attempt to overcome challenges like this, investigators in the lab of David Rawlings, MD, the director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute, have been conducting preclinical research on the development of antigen-specific engineered regulatory T-cells (EngTregs). These EngTregs are derived from autologous CD4 T-cells and are genetically modified to maintain the Treg cell phenotype continuously and to target a specific antigen. A third modification allows the EngTregs to be activated by the presence of rapamycin rather than IL2. This is important because administration of sufficient levels of rapamycin for the purpose of activating the EngTregs avoids common issues with using exogenous IL2, such as activation of other, unintended cell types. These results from their work were presented at 

the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting

, held May 16-20, in Los Angeles, California.

™ shortly after the conference, Rawlings discussed the engineering techniques his lab has been researching and how they might help to overcome the issues holding back the therapeutic potential of Treg cells. He also went over the results of further research that involved administering EngTregs in a mouse model of diabetes. He noted that a mouse surrogate of the EngTregs targeted at mouse pancreatic islet cells showed the ability to prevent the development of diabetes in vivo.

REFERENCE
Seattle Children’s Researchers Share Progress at American Society of Gene and Cell Therapy Annual Meeting. Website. Seattle Children’s Research Institute. Accessed October 12, 2023. https://www.seattlechildrens.org/research/featured-research/asgct-2023/

The director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute discussed preclinical research on overcoming limitations of Treg cells through genetic engineering.  

The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed possible applications of the procedure.

Massimo Trucco, MD, on the Potential of Islet Cell Transplant

“Chronic pancreatitis can lead to cancer. So, that is a consideration for doing the auto transplant in children to avoid a problem that can develop in the future. In adults, the organs are already working sub-optimally and the cell-to-liver transplant is not the best because the liver is not the best. The general body of the recipient is not working very well.”

The pancreatic islet cell replacement therapy VX-880 reduced insulin dependence in the first 2 patients with type 1 diabetes (T1D) treated as part of a phase 1/2 study (NCT04786262).

Islet cells are emerging as a possible approach to cell therapies in such indications including T1D and chronic pancreatitis.

Recently, Alleghany Health Network performed the first islet cell transplant for the treatment of chronic pancreatitis.

The project leader is Massimo Trucco, MD, director, Alleghany Health Network Institute of Cellular Therapeutics. As part of the treatment, Trucco and colleagues remove the pancreas to eliminate the chronic pancreatitis and transplant islet cells to the liver to continue insulin production.

spoke with Trucco to learn more about the islet cell transplant procedure. He discussed eligibility and potential other applications for the transplants.

1. Markmann JF, Naji A, Rickels MR, et al. Stem cell–derived, fully differentiated islet cells for type 1 diabetes. Presented at: ADA 82nd Scientific Sessions Conference, June 6, 2022, New Orleans, Louisiana. Abstract #259-OR.
2. AHN performs its first islet cell transplants to treat chronic pancreatitis. News release. AHN. October 5, 2021. https://www.prweb.com/releases/ahn_performs_its_first_islet_cell_transplants_to_treat_chronic_pancreatitis/prweb18241294.htm

The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed possible applications of the procedure. 

Massimo Trucco, MD, on the Potential of Islet Cell Transplant 

The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed islet cell transplant for the treatment of chronic pancreatitis.

Massimo Trucco, MD, on Improving Access to Islet Cell Transplant

“We are working very hard to make the auto transplant also available, which uses the same procedure for the allo-transplant in which you can take the islet cell from any diseased donor. Transplant in a diabetic patient is technically the same thing and is very feasible.”

Islet cell transplant is emerging as a cell therapy approach to treat conditions including type 1 diabetes (T1D) and chronic pancreatitis. Recent data from Vertex Pharmaceuticals showcased the efficacy of this approach, with VX-880 reducing insulin dependence in the first 2 patients with T1D treated in a phase 1/2 study (NCT04786262).

Alleghany Health Network recently performed its first islet cell transplant to treat chronic pancreatitis under the lead of Massimo Trucco, MD, director, Alleghany Health Network Institute of Cellular Therapeutics.

 As part of this procedure, the pancreas is removed to avoid the effects of chronic pancreatitis and islet cells are transplanted into the liver to maintain insulin production.

 spoke with Trucco to learn more about islet cell transplant as a treatment for chronic pancreatitis and T1D. He discussed improving access to the procedure.

The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed islet cell transplant for the treatment of chronic pancreatitis. 

Massimo Trucco, MD, on Improving Access to Islet Cell Transplant  

The Director of the Islet Transplant Program at the University of Alberta discussed the implantable VC-02 device's success in type 1 diabetes.

Stem Cell Therapy Device Restores Insulin Production in T1 Diabetes: James Shapiro, MD

This content originally appeared on our sister site, 

Novel cell replacement therapies have recently produced promising data in several severe diseases, including type 1 diabetes (T1D).

One such study is evaluating a stem cell therapy to restore insulin productinon in people with severe T1D. The ongoing, first-in-human phase 1/2 study has enrolled implanted a total of 17 patients with the ViaCyte PEC-Direct device at 6 different centers. The device is comprised of pancreatic cells (PEC-01) contained within pouches for subcutaneous placement.

In an interview with HCPLive, James Shapiro MD PhD, Canada Research Chair and Director of the Islet Transplant Program at the University of Alberta and lead author of the Cell Reports Medicine report, discussed the findings of the study and what they ultimately represent.

“It was a very successful trial in terms of demonstrating the safety, it was absolutely safe for patients, while they were, you know, many different potential side effects on the anti rejection drugs and the minor surgeries that the patients went through, they tolerated the placement and the removal of the devices exceedingly well,” Shapiro said.

The trial results indicated 34% of patients had evidence of C-peptide production, while 63% of patients had evidence of surviving insulin producing cells at different time points when the devices were taken out and examined under a microscope.

Shapiro went on to describe the next wave of trials using gene-edited products that will not require anti-rejection drugs, called PEC-QT. He noted the difference between a treatment and a cure is the limitless source of cells and lack of need for rejection drugs.

“I think if that happened, then we really would have a therapy that could be given to children just diagnosed with diabetes, they could be given to patients with all forms of diabetes, not just patients with T1D,” he said. “So, I think this does herald a big step forward for for stem cell based therapists in the cure potential curative treatment for all forms of diabetes.”

The Director of the Islet Transplant Program at the University of Alberta discussed the implantable VC-02 device's success in type 1 diabetes.



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