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Vertex Pharmaceuticals Continues to Innovate in Type 1 Diabetes Treatment with Gene Editing Technology

Vertex Pharmaceuticals Continues to Innovate in Type 1 Diabetes Treatment with Gene Editing Technology

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Gene editing is an immensely potent technique that can be wielded to manipulate the hereditary code of an individual. This cutting-edge technology has revolutionized the field of genetics, enabling scientists to make precise and specific alterations to an organism's DNA, thereby modulating their physical characteristics and biological behavior.

One way it can be utilized in diabetes 1 treatment is by correcting genetic mutations that lead to the development of the disease. Type 1 diabetes is caused by an autoimmune response that destroys insulin-producing cells in the pancreas. While it is not a genetic disorder, certain genes are associated with an increased risk of developing the disease. For example, the HLA genes, which are involved in regulating the immune system, have been linked to an increased risk of developing type 1 diabetes.

  • One potential application of gene editing in diabetes 1 treatment is to use it to modify the HLA genes in individuals who are at high risk of developing the disease. This could potentially prevent the autoimmune response from occurring, thereby protecting the insulin-producing cells in the pancreas.
  • Another potential application of gene editing in diabetes 1 treatment is to use it to modify the genetic code of insulin-producing cells themselves. This could involve introducing genes that promote the survival of these cells or altering genes that are responsible for the production of insulin to improve its efficiency.
  • Vertex Pharmaceuticals is spearheading the development of two cutting-edge clinical-stage programs to tackle type 1 diabetes, with each initiative employing a distinct methodology to stave off rejection of the cell treatments by the immune system. In a bold move to create a third program in preclinical development, the pharmaceutical company is keen to leverage the power of gene editing to circumvent rejection. To this end, Vertex Pharmaceuticals has forged a lucrative partnership with CRISPR Therapeutics, shelling out a hefty $100 million to leverage their gene editing technology for the development of novel edited cell therapies.
  • The disclosed initial payment has conferred upon Boston-based Vertex the esteemed privilege of non-exclusive access to CRISPR's revolutionary gene editing technology for the development of innovative modified cell therapies aimed at treating type 1 diabetes. This strategic transaction marks a significant milestone in cementing the enduring partnership between the two enterprises. Vertex has already engaged in a collaborative effort with CRISPR in Zug, Switzerland to produce a cure for a rare blood condition through the ex vivo editing of a patient's stem cells.
  • Type 1 diabetes is a chronic autoimmune disorder that destroys pancreatic islet cells responsible for insulin production by the immune system. Vertex Pharmaceuticals entered the pursuit of cell therapy for the disease by acquiring Semma Therapeutics in 2019, which was developing a therapy using stem cell-derived human islets. Vertex's therapy, VX-880, necessitates standard immunosuppressive measures to protect the implanted cells. The company is currently conducting a Phase 1/2 clinical trial to evaluate the program. In June, Vertex demonstrated the clinical proof of concept for this approach using data from two participants in the study.
  • Vertex's second program, VX-264, involves housing and implanting insulin-producing islet cells in an immunoprotective device. Although the company has received approval to conduct clinical testing in Canada, its application for U.S. trials is still subject to an FDA clinical hold.
  • Vertex Pharmaceuticals is pursuing a third strategy for the treatment of type 1 diabetes, which entails creating hypoimmune cells that have a decreased likelihood of provoking an immune response. Although this research is still in the preclinical phase, the organization announced on Monday that CRISPR's gene-editing technology could hasten the development of these cell therapies. If successful, this approach has the potential to circumvent the need for immunosuppressive treatments, which can pose additional hazards.
  • Sana Biotechnology's potential competition with Vertex in the development of hypoimmune cell therapy for type 1 diabetes. Sana has been working on pancreatic islet cells derived from stem cells that are modified to be hypoimmune and can survive without immunosuppression. These cells also can evade immune responses that could lead to rejection. The results of their preclinical research were presented in June 2022 during the annual meeting of the International Society for Stem Cell Research.
  • Vertex also has a foothold in type 1 diabetes through ViaCyte, which it purchased last year for $320 million. While ViaCyte has two type 1 diabetes programmes that require immunosuppressive medications, it also has a third programme that may not require them due to gene editing. This programme is the result of a collaboration between ViaCyte and CRISPR Therapeutics before the company's acquisition by Vertex. According to the terms of their agreement, the ViaCyte/CRISPR collaboration will continue. Phase 1/2 testing has been completed for that programme.
  • Under the terms of the new agreement, Vertex and CRISPR may receive up to $230 million in research and development milestone payments, as well as royalty from sales of any commercialised hypoimmune cell treatments for type 1 diabetes. CRISPR will have no share in any of Vertex's type 1 diabetes medication pipeline programmes, including VX-880 and VX-264, under the terms of the agreement. The prior collaboration between Vertex and CRISPR to develop a gene-editing medicine for rare blood illnesses is progressing. Vertex initiated a rolling FDA filing in November to seek regulatory approval in sickle cell disease and transfusion-dependent beta-thalassemia. Vertex stated in its 2022 financial results report last month that it anticipates the application to be completed by the end of the first quarter of this year.

 

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