First CRISPR Therapy for Type 1 Diabetes Set to Enter Clinical Trial
A press release shared yesterday by CRISPR Therapeutics and ViaCyte marks a turning point in the gene-editing field and for diabetes patients. The companies jointly-developed CRISPR-edited VCTX210 stem cell therapy candidate for type 1 diabetes (and insulin-dependant type 2 diabetes) has been approved for a clinical trial in Canada.
Type 1 diabetes (T1D) is an autoimmune disease that arises when an individual’s own immune cells attack and destroy the insulin-producing beta cells in the pancreas. The resulting deficiency in beta cells causes a halt in insulin production, which in turn leads to uncontrolled blood glucose levels (see Fact Box).
The current standard treatment for T1D is insulin replacement therapy with regular injections of insulin and no cure exists. According to the World Health Organisation, there were 9 million people living with T1D worldwide in 2017, and multiple studies have shown that this number is rising steadily. In 2017, the number of people living with type 2 diabetes was estimated at 462 million worldwide, and a proportion of these also require insulin replacement therapy.
VCTX210 – the first-ever gene-edited therapy for type 1 diabetes
VCTX210 is an allogeneic (donor-derived), gene-edited, stem cell-derived therapy designed as a best-in-class treatment for type 1 diabetes (T1D) and insulin-dependant T2D. The candidate is designed to replace the bta cells that are lost in diabetes, and is developed using a CRISPR-Cas9 gene-editing approach that involves disruption and insertion.
VCTX210 is also engineered to evade the immune response for increased persistance, which also eliminates the requirement for immune suppression therapy. No further details about how VCTX210 is developed are available at this time.
Type 1 diabetes (T1D) is an autoimmune disease that results in destruction of the insulin-producing beta cells of the pancreas. The resuling insulin deficiency leads to rising blood glucose levels that over time can lead to a range of complications that include the heart, eyes, kidneys, nerves, and gums and teeth.
T1D may debut at any age but it usually begins in childhood or early adulthood, and is fatal if untreated. The standard current treatment is regular blood glucose monitoring and subcutaneous administration of modified human insulin expressed in heterologous hosts, e.g., E. coli.
The related disease type 2 diabetes (T2D) is caused by insulin resistance, whereby cells don’t respond normally to insulin. The pancreas makes more insulin to try to get cells to respond, and over time the pancreas fails to keep up with the demand for insulin, leading to rising blood sugar levels. Although the general treatment approach to T2D is dietary and other lifestyle changes, a proportion of these patients will need insulin replacement therapy.
CRISPR Therapeutics is a leading biopharmaceutical company in the CRISPR space that focuses on developing transformative gene-based medicines for serious diseases with a pipeline that encompasses haemoglobinopathies, cancer and rare genetic diseases, including but not limited to, duchenne muscular dystropy and cystic fibrosis.
ViaCyte is a regenerative medicine company whose pipeline includes two novel clinical-stage stem cell-derived cell replacement therapy candidates designed as a functional cure for all T1D patients. and a next-generation treatment for insulin-requiring type 2 diabetes. The co-development of VCTX210 combines ViaCyte’s stem cell capabilities and CRISPR Therapeutics’ gene-editing platform to advance a functional cure for all T1D patients as well as insulin-dependant T2D patients.
The VCTX210 trial
The Phase 1 clinical trial of VCTX210 is designed to evaluate its safety, tolerability, and immune evasion in T1D patients with T1D, and patient enrollment is expected to be initated by the end of this year.
We will continue to provide updates about VCTX210 and the upcoming clinical trial as further details emerge.
For a complete overview of CRISPR IND approvals and ongoing gene-editing clinical trials, check out CRISPR Medicine News' Clinical Trials Database.
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Arsenal Biosciences, Inc.
M.D. Anderson Cancer Center