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 .
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Disclosure: None. This article is originally published at Insider Monkey.
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