Wugen Receives RMAT and PRIME Designations for CAR-T Cell Therapy for Treatment of Rare Cancers

Rare Daily Staff

Wugen received Regenerative Medicine Advanced Therapy designation in the United States and PRIME designation in the European Union for WU-CART-007 for the treatment of the rare cancers relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma.

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant, preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease. WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global phase 1/2 clinical trial for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma.

Regulators granted Wugen these designations following their reviews of data including results from the global phase 1/2 clinical trial for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma. The newest data from the phase 1/2 study will be presented by Ibrahim Aldoss, City of Hope, on June 14 during the European Hematology Association 2024 Hybrid Congress in Madrid, Spain.

“Our goal is to bring this investigational off-the-shelf allogeneic CAR-T treatment to patients as soon as possible,” said Kumar Srinivasan, Wugen president and CEO. “These designations represent a significant opportunity to accelerate regulatory reviews for WU-CART-007 as we conclude phase 1/2 trials and begin pivotal studies later this year.”

The U.S. Food and Drug Administration’s Center for Biologic Research and Evaluation grants RMAT designation for regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition as well as having preliminary clinical evidence indicating their potential to address unmet medical needs for a serious condition. EMA’s PRIME scheme is designed for medicines that demonstrate the potential to address an unmet medical need.

WU-CART-007 previously received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma.

Photo: Kumar Srinivasan, Wugen president and CEO