Beyond Insulin: DNA Technology Offers Potential Treatment For Type 1 Diabetes
As DNA technology continues to evolve, the dream of an insulin-free future for individuals living with Type 1 diabetes is becoming more tangible
Genetic predisposition, environmental triggers, and immune dysregulation contribute to the development of Type 1 diabetes (T1D)—a complex and chronic autoimmune disease. Out of an estimated 8.75 million people with T1D globally, approximately 1.5 million are under the age of twenty. Maintenance therapy, consisting of insulin injections paired with glucose monitoring, remains the predominant therapeutic option accessible to most patients. In recent years, advancements in DNA technology, cell and gene therapy, and antibody treatments have been leveraged to improve outcomes and quality of life for individuals with T1D.
Unlike Type 2 diabetes, which is linked to lifestyle factors, Type 1 diabetes is an autoimmune condition where the body destroys insulin-producing β-cells. Curing it requires both restoring insulin production and suppressing the autoimmune response—making therapeutic development far more complex than standard lifelong glucose monitoring and insulin therapy. Akshay Ray, Associate Vice President, Technology Research & Advisory, Aranca shares insights:
DNA technology has existed for over four decades, and advances in recent years have been instrumental in developing biologic therapies for conventionally difficult or untreatable diseases. For a complex disease like T1D, DNA technology has been used to develop vaccines, create antibodies that target T-cells attacking pancreatic β-cells, and re-engineer cells to produce insulin.
Kick-starting Insulin Production
Given the destruction of pancreatic β-cells and the resulting insulin deficiency, transplanting insulin-producing cells has emerged as a relatively successful strategy for 'curing" T1D, as shown by promising clinical trials, albeit with small sample sizes. Vertex Pharmaceuticals has developed Zimislecel (VX-880), an allogenic stem cell-based therapy comprising insulin-producing pancreatic β-cells generated from stem cells. The therapy has shown remarkable results, with 11 out of 12 participants demonstrating improved glycemic control since administration. These findings have paved the way for a larger ongoing Phase 3 trial.
Mitigating Immunosuppression Needs
While transplanting β-cells—lab-made or donor-derived—seems like a logical solution, the body's immune system often rejects these foreign cells. Immunosuppressants can prevent graft rejection but leave patients vulnerable due to compromised immunity.
Several companies are exploring innovative ways to bypass this problem.
Sana Biotechnology's UP421 uses donor islet cells engineered to be hypoimmune, eliminating the need for immunosuppressants. They are also working on lab-grown islet cells to reduce donor dependence.
Sernova Biotherapeutics has developed a Cell Pouch™, which enables vascularization—providing oxygen and nutrients to the cells. They also plan to use this system with manufactured islet cells.
Seraxis has introduced SR-02, a manufactured islet cell therapy implanted into the omentum (a protective fat layer around abdominal organs). Their upcoming candidate, SR-03, includes gene-edited islet cells to minimize immune rejection.
CRISPR Therapeutics has developed CTX211, which uses gene-edited manufactured islet cells to help them evade immune attack.
Tackling Autoimmunity
Vaccine strategies for T1D aim to reduce the autoimmune destruction of β-cells. These approaches may activate antigen-specific T-reg cells, eliminate autoreactive T-cells, or halt immune cell interactions. One vaccine candidate combines Cholera Toxin Subunit B with the insulin B chain to improve immune tolerance. Another strategy targets Antigen Presenting Cells (APCs) to activate them; once activated, APCs downregulate the Th2 pathway responsible for producing autoantibodies against β-cells. Advances in DNA technology have allowed researchers to explore finer immune mechanisms of T1D, helping to identify other vaccine targets such as GAD65 and IA-2. Several vaccine candidates have now reached human trial stages, offering hope for broader therapeutic options.
As DNA technology continues to evolve, the dream of an insulin-free future for individuals living with Type 1 diabetes is becoming more tangible. From restoring insulin production to eliminating the need for immunosuppressants and developing targeted vaccines, these scientific advancements are addressing the disease at its core. Though challenges remain, ongoing research offers renewed hope for more effective and lasting treatments that go beyond lifelong disease management.
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Beyond Insulin: DNA Technology Offers Potential Treatment For Type 1 Diabetes
Last Updated: As DNA technology continues to evolve, the dream of an insulin-free future for individuals living with Type 1 diabetes is becoming more tangible Genetic predisposition, environmental triggers, and immune dysregulation contribute to the development of Type 1 diabetes (T1D)—a complex and chronic autoimmune disease. Out of an estimated 8.75 million people with T1D globally, approximately 1.5 million are under the age of twenty. Maintenance therapy, consisting of insulin injections paired with glucose monitoring, remains the predominant therapeutic option accessible to most patients. In recent years, advancements in DNA technology, cell and gene therapy, and antibody treatments have been leveraged to improve outcomes and quality of life for individuals with T1D. Unlike Type 2 diabetes, which is linked to lifestyle factors, Type 1 diabetes is an autoimmune condition where the body destroys insulin-producing β-cells. Curing it requires both restoring insulin production and suppressing the autoimmune response—making therapeutic development far more complex than standard lifelong glucose monitoring and insulin therapy. Akshay Ray, Associate Vice President, Technology Research & Advisory, Aranca shares insights: DNA technology has existed for over four decades, and advances in recent years have been instrumental in developing biologic therapies for conventionally difficult or untreatable diseases. For a complex disease like T1D, DNA technology has been used to develop vaccines, create antibodies that target T-cells attacking pancreatic β-cells, and re-engineer cells to produce insulin. Kick-starting Insulin Production Given the destruction of pancreatic β-cells and the resulting insulin deficiency, transplanting insulin-producing cells has emerged as a relatively successful strategy for 'curing" T1D, as shown by promising clinical trials, albeit with small sample sizes. Vertex Pharmaceuticals has developed Zimislecel (VX-880), an allogenic stem cell-based therapy comprising insulin-producing pancreatic β-cells generated from stem cells. The therapy has shown remarkable results, with 11 out of 12 participants demonstrating improved glycemic control since administration. These findings have paved the way for a larger ongoing Phase 3 trial. Mitigating Immunosuppression Needs While transplanting β-cells—lab-made or donor-derived—seems like a logical solution, the body's immune system often rejects these foreign cells. Immunosuppressants can prevent graft rejection but leave patients vulnerable due to compromised immunity. Several companies are exploring innovative ways to bypass this problem. Sana Biotechnology's UP421 uses donor islet cells engineered to be hypoimmune, eliminating the need for immunosuppressants. They are also working on lab-grown islet cells to reduce donor dependence. Sernova Biotherapeutics has developed a Cell Pouch™, which enables vascularization—providing oxygen and nutrients to the cells. They also plan to use this system with manufactured islet cells. Seraxis has introduced SR-02, a manufactured islet cell therapy implanted into the omentum (a protective fat layer around abdominal organs). Their upcoming candidate, SR-03, includes gene-edited islet cells to minimize immune rejection. CRISPR Therapeutics has developed CTX211, which uses gene-edited manufactured islet cells to help them evade immune attack. Tackling Autoimmunity Vaccine strategies for T1D aim to reduce the autoimmune destruction of β-cells. These approaches may activate antigen-specific T-reg cells, eliminate autoreactive T-cells, or halt immune cell interactions. One vaccine candidate combines Cholera Toxin Subunit B with the insulin B chain to improve immune tolerance. Another strategy targets Antigen Presenting Cells (APCs) to activate them; once activated, APCs downregulate the Th2 pathway responsible for producing autoantibodies against β-cells. Advances in DNA technology have allowed researchers to explore finer immune mechanisms of T1D, helping to identify other vaccine targets such as GAD65 and IA-2. Several vaccine candidates have now reached human trial stages, offering hope for broader therapeutic options. As DNA technology continues to evolve, the dream of an insulin-free future for individuals living with Type 1 diabetes is becoming more tangible. From restoring insulin production to eliminating the need for immunosuppressants and developing targeted vaccines, these scientific advancements are addressing the disease at its core. Though challenges remain, ongoing research offers renewed hope for more effective and lasting treatments that go beyond lifelong disease management. view comments Disclaimer: Comments reflect users' views, not News18's. Please keep discussions respectful and constructive. Abusive, defamatory, or illegal comments will be removed. News18 may disable any comment at its discretion. By posting, you agree to our Terms of Use and Privacy Policy.
