Genome editing has been a much-debated topic in recent years, with the emergence of CRISPR technology in the spotlight. Scientists and investors alike have been captivated by the potential of this revolutionary technology, and its implications for the future of medicine, genetics, and more.
In a groundbreaking move, CRISPR Therapeutics and ViaCyte have performed the world’s first-ever human transplant of gene-edited, stem cell-derived pancreatic cells to treat Type 1 diabetes (T1D), a milestone that will go down in the history books. The transplant took place in February 2022, marking a major breakthrough in the fight against T1D.
In February 2023, Genprex made a breakthrough in the search for a functional cure to Type 1 Diabetes, by publishing the results of an animal study that adds credibility to their genome editing approach. This milestone could be a major step forward in finding a cure for this chronic condition.
Located in Austin, Texas, Genprex is dedicated to revolutionizing the way we treat cancer and diabetes. By utilizing cutting-edge technology, Genprex is developing life-changing treatments and therapies that could have a major impact on the lives of those suffering from these conditions. With a focus on improving the quality of life for patients, Genprex is pushing the boundaries of medical science and pioneering new treatments that could make a real difference.
GlycoMimetics is making strides in the healthcare industry with its innovative pipeline of three promising products: Reqorsa, GPX-002, and GPX-003. Reqorsa is currently in Phase I trials for cancer, while GPX-002 and GPX-003 are being preclinically tested for diabetes, with GPX-002 potentially offering a solution for T1D and GPX-003 for T2D. This cutting-edge research could revolutionize the lives of countless individuals suffering from these diseases.
Genprex is pioneering a novel approach to treat diabetes: using Adeno-Associated Virus (AAV) pancreatic intraductal infusions to deliver Pdx1 and MafA genes to the pancreas. This innovative gene therapy stands apart from traditional treatments like replacement gene therapy, gene editing and CAR-T, as it seeks to improve the efficacy, safety, and affordability of diabetes treatments.
Non-Human Primate Study
One month after eight non-human primates were injected with streptozocin to induce diabetes, the subjects underwent surgical procedures to receive infusions, marking a significant milestone in the study of diabetes treatment.
The results of the study showed remarkable improvements in the health of the non-human primates. Not only did they require less insulin, their C-peptide levels increased and their glucose tolerance improved. What’s more, immunohistochemistry revealed that the number of insulin-positive cells in the NHPs was higher than the non-treated diabetic controls.
The researchers monitored blood glucose and insulin levels during the diabetes induction period. On average, participants required 8 to 10 units of exogenous insulin daily and had daily blood glucose levels between 50 and 100 mg/dL.
After Genprex’s investigational AAV-CMV-Pdx1-mafA construct was injected during the surgical procedure, a remarkable trend was observed: average daily blood glucose levels initially spiked to 200 mg/dL, yet total daily exogenous insulin levels dropped dramatically to below 4 units.
After undergoing a surgical procedure, the patient’s average daily blood glucose levels dropped dramatically to 100 mg/dL within 56 days. Not only did this remarkable achievement provide substantial relief to the patient, but it also resulted in improved glucose suppression capabilities eight weeks later. Notably, the patient’s average glucose-stimulated insulin secretion remained higher post-operation.
At the 3-month mark, an astonishing result was seen in the recent glucose tolerance test results of NHP2. Both the treated diabetic NHPs and healthy NHPs showed virtually no difference between them, proving the effectiveness of the treatment!
This remarkable accomplishment is a major step forward, but there are still unknowns that need to be considered before we can fully appreciate its impact. Through further exploration, we can begin to understand the true scope of this breakthrough.
Genprex announced that their gene therapy could be administered without an operation in humans through a routine procedure known as endoscopic retrograde cholangiopancreatography, which is distinct from the method used in non-human primates.
Living with diabetes can be a challenge, but the promise of a quick cure may offer a glimmer of hope. Patients may need to adjust their expectations, however, as they become accustomed to novel treatments that differ from the traditional course of oral and injectable medications. Nevertheless, the prospect of a swift resolution to this chronic condition can be an appealing one.
With the promise of potentially groundbreaking treatments for cancer, Genprex’s human trials have yet to commence as the road to FDA approval looms ahead. Although the timeline for such an approval is uncertain, the hope is that this treatment will be available on the market sometime in the latter half of the 2020s.
CRISPR and ViaCyte in Phase I
Vertex made a major move in July 2022, acquiring ViaCyte and its groundbreaking diabetes genome editing treatment, VCTX210. This treatment, developed by ViaCyte and CRISPR, has put them at the forefront of diabetes treatments, offering an exciting new way to tackle the disease. With the acquisition, Vertex is now well-positioned to bring this innovative therapy to more people, and make a real difference in the fight against diabetes.
ViaCyte’s new product, VCTX210, is a groundbreaking solution offering a permanent solution to diabetes without the need for long-term immunosuppression with drugs. This product is a revolutionary step towards a future free from the need for medications to control the condition.
As of February 2023, the companies had made impressive progress, having completed dosing in Phase I subjects. This marks a significant milestone, as the Phase I trial is the first stage in evaluating the safety and efficacy of the treatment. Genprex is one step ahead in this process, as the results of the trial will provide a better understanding of the potential benefits and risks.
VCTX210 and Genprex offer two distinct approaches to the modern treatment of diabetes: VCTX210 is an allogeneic, gene-edited, stem cell-derived therapy designed to provide a best-in-class treatment for both Type 1 Diabetes and insulin-dependent Type 2 Diabetes, while Genprex utilizes a viral delivery system. Both aim to offer comprehensive treatment options for diabetes, but with different approaches.
Genprex and CRISPR/Viacyte are revolutionizing the search for a cure to diabetes, with the potential to provide answers to millions of patients worldwide. As these gene therapy agents charge forward, many are keeping a close eye on how they will shape the future of this debilitating disease. With so much at stake, the next few years are sure to be an exciting journey in seeing how these pioneering treatments will fare.