The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to iCell Gene Therapeutics‘ Chimeric Antigen Receptor (CAR) engineered T-cells targeting CD4 (CD4CAR) for the treatment of peripheral T-cell lymphoma (PTCL).
“We are very excited to have this opportunity to partner with iCell Gene Therapeutics to lead the efforts of preparing this cutting-edge immunotherapy into first-in-human clinical trial for patients suffering this extremely difficult-to-treat T-cell lymphoma,” Dr. William Tse, chief of the Blood and Marrow Transplantation Division at the University of Louisville’s Department of Medicine, said in a press release.
CAR engineered T-cells are patient T-cells that have been genetically modified to express a protein on its surface with the ability to bind to a target protein or another cell. After binding the target protein, a CAR T-cell will send signals that will ultimately trigger mechanisms to selectively kill the targeted cell.
CD4CAR is now in development for CD4+ T-cell malignancies.
“CD4CAR could significantly enhance currently available treatment options for these patients. The orphan drug designation is an important achievement as we advance our development plans for this promising treatment in T-cell hematologic cancers,” said Dr. Yupo Ma, PhD, a pathology researcher at Stony Brook University and iCell Gene Therapeutics’ chairman and chief scientific officer.
Though clinical development programs are ongoing with CAR T-cells for CD19+ cell hematological malignancies, CD4+ PTCLs — which account for 10 percent to 15 percent of all non-Hodgkin’s lymphomas (NHLs) and are harder to treat than B-cell NHLs — have not been targeted by a CAR therapy in a human trial.
An initial Phase 1 clinical study is being prepared in collaboration with the National Institutes of Health, Indiana Clinical and Translational Sciences Institute, Stony Brook University Hospital, and the Blood and Marrow Transplantation Division and the Clinical Trial Research Unit at James Graham Brown Cancer Center at University of Louisville.
The FDA’s orphan drug designation program provides orphan drug status and related development incentives to drugs and biologics meant to safely and effectively treat, diagnose, or prevent rare diseases or disorders that impact fewer than 200,000 people in the United States.