FDA News

FDA grants orphan drug designation to CD4CAR therapy for peripheral T-cell lymphoma

The FDA granted orphan drug designation to iCell Gene Therapeutics for its chimeric antigen receptor-engineered T cells directed against the target protein CD4 to treat peripheral T-cell lymphoma.

Chimeric antigen receptor (CAR)-engineered T cells are patients’ own T cells that have been genetically modified to express a protein on its surface. CAR T cells can bind to a target protein on another cell, and the CAR protein sends a signal to the interior of the T cell to unleash mechanisms that selectively kill the targeted cell.

The CD4CAR T cells from iCell Gene Therapeutics is in development for CD4-positive T-cell malignancies.

“CD4CAR could significantly enhance currently available treatment options for these patients,” Yupo Ma, MD, PhD, professor of pathology at Stony Brook University and chairman and chief scientific officer at iCell Gene Therapeutics, said in a press release. “The orphan drug designation is an important achievement as we advance our development plans for this promising treatment in T-cell hematologic cancers.”

A phase 1 study is planned through a collaboration between iCell Gene Therapeutics, NIH, Indiana Clinical and Translational Sciences Institute, Stony Brook Hospital, and the blood and marrow transplantation division and clinical trial research unit at James Graham Brown Cancer Center at University of Louisville.

“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,” William Tse, MD, chief of the blood and marrow transplantation division in the department of medicine at University of Louisville School of Medicine, said in the press release.

The FDA granted orphan drug designation to iCell Gene Therapeutics for its chimeric antigen receptor-engineered T cells directed against the target protein CD4 to treat peripheral T-cell lymphoma.

Chimeric antigen receptor (CAR)-engineered T cells are patients’ own T cells that have been genetically modified to express a protein on its surface. CAR T cells can bind to a target protein on another cell, and the CAR protein sends a signal to the interior of the T cell to unleash mechanisms that selectively kill the targeted cell.

The CD4CAR T cells from iCell Gene Therapeutics is in development for CD4-positive T-cell malignancies.

“CD4CAR could significantly enhance currently available treatment options for these patients,” Yupo Ma, MD, PhD, professor of pathology at Stony Brook University and chairman and chief scientific officer at iCell Gene Therapeutics, said in a press release. “The orphan drug designation is an important achievement as we advance our development plans for this promising treatment in T-cell hematologic cancers.”

A phase 1 study is planned through a collaboration between iCell Gene Therapeutics, NIH, Indiana Clinical and Translational Sciences Institute, Stony Brook Hospital, and the blood and marrow transplantation division and clinical trial research unit at James Graham Brown Cancer Center at University of Louisville.

“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,” William Tse, MD, chief of the blood and marrow transplantation division in the department of medicine at University of Louisville School of Medicine, said in the press release.