Latest news with #Zimislecel
News18
10-07-2025
- Health
- News18
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.
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Business Standard
26-06-2025
- Health
- Business Standard
Stem cell breakthrough: New therapy raises hopes of curing type 1 diabetes
Living with type 1 diabetes is a constant battle where patients need to track blood sugar throughout the day and need insulin injections or pump-based therapies for life. But what if one day you could stop taking insulin for good? Today, it might sound impossible, but US-based scientists are working towards making it a reality. They have developed a new stem cell-derived treatment that has allowed the majority of patients with severe type 1 diabetes, who participated in the trial, to discontinue insulin use. The study titled Stem cell–derived, fully differentiated islets for Type 1 diabetes, published in The New England Journal of Medicine, tested an experimental treatment called zimislecel, developed by US biopharmaceutical company Vertex Pharmaceuticals. According to the research paper, it helped 83 per cent of participants achieve insulin independence and maintain stable blood sugar levels for at least one year, marking a potentially transformative step in diabetes care. What is zimislecel and what did the trial find? Researchers conducted a trial on 14 people with severe type 1 diabetes who suffered from hypoglycemic unawareness, a dangerous condition where blood sugar drops suddenly without warning signs. These patients are at risk of passing out, having seizures, or even dying from low blood sugar. According to the study, the participants received a stem cell-based infusion called zimislecel, which introduced lab-grown islet cells into their bodies. These new cells travelled to the liver and started working like natural insulin-producing cells. The findings show: 10 out of 12 surviving participants were able to completely stop using insulin within a year The remaining two patients needed significantly less insulin Patients spent over 70 per cent of their time within the healthy blood sugar range (70–180 mg/dL) Episodes of low blood sugar stopped within the first 90 days of treatment How zimislecel could change type 1 diabetes treatment According to the researchers, type 1 diabetes happens when the immune system mistakenly destroys insulin-producing islet cells in the pancreas. Without insulin, the body cannot regulate blood sugar levels, requiring lifelong insulin injections or pumps. Zimislecel changes this as it uses stem cells engineered to become islet cells, replacing what the immune system destroyed. Once inside the body, these cells settle in the liver and start regulating blood sugar like a healthy pancreas would. However, the participants in the trial also received glucocorticoid-free immunosuppressive therapy to prevent the body from attacking the newly introduced islet cells. This means that the patient might need to take immunosuppressants for life, which reduces immunity and increases the risk of infections. The study was presented at the American Diabetes Association's 85th Scientific Sessions held in Chicago from June 21–23. According to media reports, experts have cautioned that while this immunosuppression may be less risky than what's used for organ transplants, the long-term safety of zimislecel will need more years of follow-up. What's next for zimislecel and when could it be approved? Vertex Pharmaceuticals is now moving forward with phase 3 clinical trials of zimislecel. If larger studies confirm these results, the company may apply for approval from the US Food and Drug Administration (FDA) as early as next year. The cost of the treatment is still unknown, but experts agree that even the possibility of freedom from insulin is a groundbreaking leap forward. Can this stem cell therapy help with type 2 diabetes too? Zimislecel is currently being tested only in people with type 1 diabetes. Type 2 diabetes, which typically develops later in life and is related to insulin resistance rather than insulin absence, is a different disease that may not respond the same way to this therapy. However, other researchers are experimenting with procedures to cure type 2 diabetes through cell therapy. For example, scientists in China developed an artificial version of the insulin-producing cells found in the pancreas, which were then transplanted into the patient during trials that produced positive results. More research is going on in this regard to confirm the efficacy and safety of this approach before it can be widely used as a treatment. For more health updates, follow #HealthWithBS
Yahoo
25-06-2025
- Business
- Yahoo
Vertex Pharmaceuticals Presents 'Unprecedented' Data for T1D Cell Therapy Zimislecel, Shows Durable Benefit
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of the best biotech stocks to invest in now. On June 20, Vertex Pharmaceuticals announced the simultaneous presentation and publication of updated data from the Phase 1/2 portion of the Phase 1/2/3 FORWARD-101 clinical trial of zimislecel (VX-880). Zimislecel is an investigational stem cell-derived, fully differentiated islet cell therapy for individuals with type 1 diabetes (T1D) who experience impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). The data were presented at the American Diabetes Association/ADA annual conference in Chicago, specifically during the symposium 'Innovation and Progress in Stem Cell-Derived Islet-Cell Replacement Therapy' and were simultaneously published online by the New England Journal of Medicine. The presented data included 12 patients who received a full dose of zimislecel as a single infusion and were followed for at least one year, as of October 2024. A pharmacist delivering a specific medication to a patient in a specialty pharmacy. The results consistently demonstrated the transformative potential of zimislecel, showing durable patient benefit with longer follow-up. All 12 participants exhibited engraftment with glucose-responsive endogenous C-peptide production, which remained durable throughout one year of follow-up. Zimislecel was generally well-tolerated, with most adverse events being mild or moderate. Type 1 diabetes (T1D) is an autoimmune disease where the body's insulin-producing beta cells in pancreatic islets are destroyed, leading to insulin deficiency and hyperglycemia. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that develops and commercializes therapies for treating cystic fibrosis/CF. While we acknowledge the potential of VRTX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Daily Mail
24-06-2025
- Health
- Daily Mail
Major breakthrough as 10 patients have their diabetes CURED with new drug
Ten people have been effectively cured of their type 1 diabetes after a breakthrough infusion of stem cells on the path toward FDA approval. One year after being treated, 10 of the 12 patients who took the drug, called Zimislecel, no longer needed insulin, while the other two needed much smaller doses. The groundbreaking therapy's foundation uses stem cells that researchers manipulated to become pancreatic islet cells, tiny clusters of specialized cells scattered throughout the pancreas that produce hormones to regulate blood sugar. The cells were injected into the patients, traveling through the liver, implanted there, and began producing insulin where their bodies had previously produced none. Their blood sugar spikes were less severe after meals. Their insulin production also kept improving, and their time spent in a healthy glucose range went from about 50 percent at baseline to over 93 percent at one year. The study's participants are among the 30 percent of type 1 diabetes patients with a complication that makes it impossible for them to tell when their blood sugar is low or high, lacking the normal signs like shakiness or sweating. All of the patients in the study had this subtype of type 1, known as hypoglycemic unawareness. The condition can also cause patients to pass out, have seizures, or even die. Researchers behind the study believe their drug paves the way toward a cure for type 1 diabetes overall. The condition, which is due to a combination of genetics and environmental factors like childhood viral infections, affects roughly 1.6 million Americans. Trevor Reichman, a study co-author and surgeon at University Health Network in Toronto, told STAT: 'This study represents for the first time that biologic replacement can be administered to patients with type 1 diabetes in a single safe and effective procedure with minimal risk to the recipient.' 'This study has the potential to get us one step closer to a 'functional cure' for patients with type 1 diabetes,' Reichman added. Researchers expect to apply for approval of this drug with the FDA within the next five years. Patients had to have a history of hypoglycemic unawareness to participate in the study, causing seizures, coma, loss of consciousness, or hospital stays. Once they started taking Zimislecel, made by Vertex Pharmaceuticals of Boston, the patients also had to take immune-suppressing drugs to prevent the body from attacking the foreign islet cells. One study enrollee, Amanda Smith, 36, from London, told the New York Times that she jumped at the chance to join the groundbreaking trial. Six months after receiving the infusion, she no longer needed insulin. 'It's like a whole new life,' she said. Traditionally, stem cells are isolated from the pancreas of a deceased organ donor. However, the cells in the latest research were grown in the lab rather than taken from cadavers, offering a scalable, renewable source of islet cells without having to rely on a limited supply of donors. Type 1 is less common than type 2 diabetes, which affects 32 million Americans and typically comes on later in life due to a confluence of lifestyle factors and genes. Without insulin, type 1 diabetics' bodies have no way to regulate blood sugar, which can build up in the bloodstream and skyrocket. The body starts breaking down fat for fuel, creating ketones, or acidic byproducts. A buildup of ketones in the blood can cause diabetic ketoacidosis, a condition that causes nausea, vomiting, rapid breathing, dehydration, and confusion. Without proper treatment with insulin and fluids, diabetic ketoacidosis can cause a laundry list of potentially fatal effects, including brain swelling, kidney failure, cardiac arrest, and death. This therapy is 25 years in the making, pioneered by the father of a baby who was diagnosed with type 1 diabetes, followed by his teen daughter. He pledged to find a cure for the disease. Their findings were published in the New England Journal of Medicine. The first patient to receive this therapy was Brian Sheton, who got it in 2021. He had been living with low blood sugar that often plunged him into a state of unconsciousness, even crashing his motorcycle into a wall at one point. The infusion cured him, but Vertex said he died afterward due to dementia symptoms that were present before he was treated. Stem cell therapy is the newest frontier in disease research, starting with niche conditions, such as hypoglycemic unawareness. Still, it has the potential to be scaled up to cover a broader umbrella of diseases. After 25 years of taking insulin shots, Illinois mom Marlaina Goedel finally said what she had longed to say for years: 'I am cured.' The 30-year-old was diagnosed with type 1 diabetes at five, and became one of a handful of people to have received an islet cell transplant. Her blood sugar normalized within a month after the infusion, and she no longer needed insulin injections. Now, she's chasing long-postponed dreams: riding her horse, going back to school, and soaking in the sweetness of a life no longer dictated by blood sugar numbers. 'We hope in the next five to 10 years that this therapy will have the potential to be given with minimal or zero immunosuppression, further minimizing the risk for patients long-term,' Dr Reichman said, adding that more research is still needed on a larger population.
Yahoo
28-03-2025
- Business
- Yahoo
Vertex Announces Program Updates for Type 1 Diabetes Portfolio
- VX-264 Phase 1/2 enrollment and dosing complete in Parts A and B: VX-264 was generally safe and well tolerated; efficacy data are not supportive of further clinical advancement - - Zimislecel (VX-880) pivotal trial on track to complete enrollment and dosing in H1 2025; Vertex expects to submit marketing applications to global regulators in 2026 - - Continue to progress multiple novel, research-stage immunoprotective approaches - BOSTON, March 28, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced several updates on the Company's type 1 diabetes (T1D) portfolio. VX-264 UpdateVertex has completed enrollment and dosing in Parts A and B of the Phase 1/2 VX-264 (cells + device) study and the planned analysis at Day 90 for Part B. In Part B of the study, participants received the full dose of the investigational fully differentiated pancreatic islet cell therapy encapsulated in a proprietary immunoprotective device. There were two primary endpoints in Part B, safety and change in peak C-peptide during a mixed-meal tolerance test (MMTT) from baseline at Day 90. VX-264 was generally safe and well tolerated; however, the study did not meet the efficacy endpoint. Increases in C-peptide, a marker of insulin production, were not observed at levels necessary to deliver benefit. Therefore, VX-264 will not be advancing further in clinical trials. Vertex plans to conduct further analyses, including of explanted devices, to better understand these findings. Zimislecel UpdateZimislecel (formerly VX-880), Vertex's investigational fully differentiated islet cell therapy with standard immunosuppression, is in the Phase 3 portion of the Phase 1/2/3 study in patients with T1D with severe hypoglycemic events (SHEs) and impaired awareness of hypoglycemia. This pivotal trial is well underway and on track to complete enrollment and dosing in the first half of 2025, setting up global regulatory submissions in 2026. Zimislecel has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration, Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), and has secured an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the UK Medicines and Healthcare products Regulatory Agency (MHRA). Consistent with this progress, Vertex is investing in expanding its manufacturing and commercial capabilities to ensure launch readiness. If approved, eligible patients across the U.S. and Europe with recurrent SHEs despite best available care could benefit from zimislecel, and Vertex anticipates that initial approval could serve approximately 60,000 people with severe T1D. T1D Research UpdateVertex is also pursuing research-stage T1D programs to evaluate additional approaches that could provide transformative benefit to people with T1D and reduce or eliminate the need for standard immunosuppressive regimens. These approaches include alternative immunosuppressive regimens, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel devices to encapsulate islet cells. "We'd like to thank the patients, physicians and T1D community who participated in the VX-264 study. Today's data show that more work needs to be done to advance the 'cells plus device' program, and we are committed to doing so," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex Pharmaceuticals. "Equally, we are very pleased with the rapid progress of our zimislecel program, which is on track to complete enrollment and dosing in the Phase 3 study this summer, positioning us for global regulatory submissions in 2026. We're excited for the opportunity to bring the promise of zimislecel to patients as quickly as possible." About Type 1 Diabetes T1D results from the autoimmune destruction of insulin-producing beta cells in pancreatic islets. Insulin deficiency results in hyperglycemia and can lead to acute life-threatening complications such as diabetic ketoacidosis. People with T1D are reliant on lifelong treatment with exogenous insulin that requires careful monitoring of blood glucose levels. Even with the availability of advanced exogenous insulin delivery and glucose monitoring systems, people with T1D can have periods of very low and very high blood sugar levels. Exogenous insulin has a narrow therapeutic range and carries an inherent risk of causing low blood sugar levels or hypoglycemic events, which can potentially result in arrhythmias, seizures, coma and even death. Due to the limitations and complexities of exogenous insulin treatment, it can be difficult for people with T1D to achieve and maintain good glucose control. Exposure to prolonged periods of high blood glucose levels, or hyperglycemia, can lead to long-term complications such as nerve damage, kidney disease/failure, eye disease (including vision loss), cardiovascular disease, stroke and even death. HbA1c is a measure of average blood glucose over the most recent ~2-3 months, and the consensus guidance is to maintain an HbA1c of <7% to reduce the risk of long-term complications; only ~1 in 4 people with T1D globally meet this clinical target. Current standards of care do not address the underlying cause of the disease and leave people with T1D susceptible to both hypo- and hyperglycemia and their associated morbidity and mortality. There is no cure for T1D. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Carmen Bozic, M.D., and statements regarding Vertex's plans and expectations for the clinical trial evaluating VX-264, including plans to conduct further analyses, expectations for the clinical trial evaluating zimislecel, including expectations for the trial to complete enrollment and dosing in the first half of 2025 and expectations for global regulatory submissions in 2026, plans to invest in manufacturing and commercial capabilities to ensure zimislecel launch readiness, expectations for the patient population that could benefit from zimislecel, and plans to progress multiple novel, research-stage immunoprotective approaches. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's research and development programs may not support development or registration of its compounds due to safety, efficacy or other reasons, that clinical trial data might not be available on the expected timeline, and other risks listed under Risk Factors in Vertex's most recent annual report filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on Contacts Vertex Pharmaceuticals IncorporatedInvestors:InvestorInfo@ Media:mediainfo@ Sign in to access your portfolio
