Release Date: 10-Sep-2021
Bioheng Biotech Co., Ltd. received Orphan Drug Designation from the U.S FDA for the treatment of T-cell acute lymphoblastic leukemia (T-ALL). CTD401 is a universal CAR-T (UCAR-T) cell therapy product developed for the treatment of patients with T-ALL. The product is prepared by collecting peripheral blood mononuclear cells (PBMC) from healthy donors, using a virus encoding a CD7 CAR gene for transduction, and using CRISPR/cas9 technology to knock out key genes.
The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.