FDA grants RMAT designation to CRISPR-edited CAR-T for cutaneous T-cell lymphomas
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The FDA granted regenerative medicine advanced therapy designation to CTX130, a chimeric antigen receptor T-cell therapy under development for the treatment of mycosis fungoides and Sézary syndrome.
CTX130 (CRISPR Therapeutics) is an allogeneic, CRISPR/Cas9 gene-edited CAR T-cell therapy derived from healthy donor cells. The investigational agent targets CD70, an antigen known to be expressed by certain solid tumors and hematologic malignancies, on the surface of cancer cells.
The novel cell therapy is being evaluated in the phase 1 COBALT-LYM trial, a single-arm, multicenter dose-escalation study designed to assess the safety and efficacy of CTX130 in adults with relapsed or refractory T-cell or B-cell malignancies.
“The RMAT designation is an important milestone for the CTX130 program that recognizes the transformative potential of our cell therapy in patients with T-cell lymphomas based upon encouraging clinical data to date,” Phuong Khanh (P.K.) Morrow, MD, FACP, chief medical officer at CRISPR Therapeutics, said in a company-issued press release. “We continue to work with a sense of urgency to bring our broad portfolio of allogeneic cell therapies to patients in need.”
The FDA’s RMAT designation program, part of the 21st Century Cures Act, is designed to expedite review of regenerative medicine therapies intended to treat, modify, reverse or cure serious or life-threatening diseases or conditions. Preliminary clinical evidence must indicate the therapy has the potential to address unmet medical needs.
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