FDA approves Vertex clinical test stem cell-derived fully differentiated islet cell therapy

Vertex Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has approved a new drug clinical trial application (IND) to enable the company to launch a VX-880 clinical trial.
VX-880 is a stem cell-derived, fully differentiated islet cell therapy used to treat type 1 diabetes (T1D).
Vertex plans to launch a phase 1/2 single-arm, open-label clinical trial in the first half of 2021, using VX-880 to treat patients with type 1 diabetes (T1D) with impaired and severe hypoglycemia.
the trial will assess the safety and effectiveity of different doses of VX-880, with approximately 17 patients participating in the clinical trial. Dr Bastiano Sanna, executive vice president and head of cell and gene therapy at
Vertex, said: "As we celebrate the 100th anniversary of the discovery of insulin this year, we are pleased to have introduced the first of its kind (first-in-class) cell therapy into the clinic, with the potential to have a meaningful impact on patients with T1D.
We look forward to launching our clinical program and testing our unique approach to producing islet cells from stem cell-derived, fully differentiated insulin to replace destroyed islet cells in people with type 1 diabetes.
type 1 diabetes (T1D) is the autoimmune destruction of insulin-producing islet cells in the pancreas, resulting in reduced insulin secretion and impaired blood sugar control.
insulin can lead to abnormal treatment of nutrients in the body, leading to high blood sugar levels.
high blood sugar can lead to diabetic ketoacidosis and, over time, complications such as kidney disease/failure, eye disease (including loss of vision), heart disease, stroke, nerve damage and even death.
the limitations and complexity of the insulin delivery system, blood sugar control in T1D patients is difficult to achieve and maintain.
blood sugar remains a key limiting factor in blood sugar management, which can lead to loss of consciousness, coma, seizures, injuries and even death.
there are currently limited options for treating T1D in addition to insulin therapy.
VX-880 (formerly STx-02) is a research isogenetic human stem cell-derived islet cell therapy that is currently being planned to evaluate patients with impaired hypoglycemia awareness and severe hypoglycemia.
VX-880 has the potential to restore the body's ability to regulate blood sugar levels by restoring islet cell function, including insulin production.
VX-880 clinical trials will include infusions of fully differentiated functional islet cells, as well as long-term combined immunosuppression therapy to protect islet cells from immune rejection.
original source: Vertex Announces FDA Clearance of Investigational New Drug (IND) application for VX-880, a Novel Cell Therapy for The Treatment of Type 1 Diabetes (T1D) This article was originally published from Bio Valley, for more information please download Bio Valley APP (T1D)