First Type 1 Diabetes Patient Receiving Novel Gene-Edited Cell Replacement Therapy

Vertex Pharmaceuticals, a biotech based in Boston, is acquiring ViaCyte’s stem-cell derived cell replacement therapies to take the next step of providing a cure for Type 1 diabetes (T1D) to the public. In fact, they’re buying the whole company for 320 million dollars.
Back in February, ViaCyte and partner CRISPR Therapeutics dosed their first patient in a Phase I trial of VCTX210 for T1D. The therapy is an investigational, patient-derived, gene-edited, immune-evasive, stem cell therapy. The therapy combines CRISPR’s gene-editing expertise with ViaCyte’s proprietary pluripotent stem cell line. VCTX210 produces pancreatic cells that won’t be rejected by the patient’s immune system but should differentiate into pancreatic endoderm cells, which will then produce insulin.