13 Best Biotech Stocks To Invest In Now

2. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)

Number of Hedge Fund Holders: 60

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.

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.