WALTHAM, Mass - TScan Therapeutics, Inc. (NASDAQ:TCRX), a biopharmaceutical company specializing in T cell receptor (TCR) therapies, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for its TSC-100 and TSC-101 therapies. These therapies are designed for the treatment of hematological malignancies in patients undergoing allogeneic hematopoietic cell transplantation (HCT) with reduced intensity conditioning.
The RMAT designation, part of the 21st Century Cures Act, aims to expedite the development and review of promising gene and cell therapies for serious or life-threatening diseases. TSC-100 and TSC-101, targeting minor histocompatibility antigens HA-1 and HA-2 respectively, are intended to eliminate malignant and pre-malignant recipient hematopoietic cells post-transplant.
Chrystal U. Louis, M.D., Chief Medical Officer of TScan, expressed the company's delight in receiving the RMAT designation and emphasized the transformative potential of TSC-100 and TSC-101 in treating cancers such as acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS).
The company's Phase 1 trial data, updated on May 13, 2024, showed that all eight patients treated with TSC-100 or TSC-101 remained relapse-free with no detectable disease after more than 10 months. This contrasts with the control-arm patients, where relapses and post-transplant mortality were observed.
TScan's therapeutic approach involves selecting HLA A02:01-positive transplant patients who are either HA-1- or HA-2-positive, with antigen-negative donors. This selective process aims to increase the cure rate for HCT recipients, as approximately 40% of patients with AML, ALL, and MDS relapse within two years of undergoing allogeneic haploidentical HCT with reduced intensity conditioning.
The RMAT designation provides TScan with benefits like intensive FDA guidance on drug development, discussions on surrogate endpoints for early interactions, and potential for accelerated approval.
This news is based on a press release statement from TScan Therapeutics, Inc.
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