Pharma Focus Europe

Hemogenyx Pharmaceuticals PLC Seeks Full FDA Review for HEMO-CAR-T IND

Monday, July 10, 2023

Hemogenyx Pharmaceuticals plc (LSE:HEMO) has recently received a comprehensive review letter from the U.S. Food and Drug Administration (FDA) concerning the Investigational New Drug (IND) application for their product candidate, HEMO-CAR-T. HEMO-CAR-T is a Chimeric Antigen Receptor T-cell therapy intended for the treatment of acute myeloid leukemia (AML). The FDA has placed HEMO-CAR-T on clinical hold (CH) due to an issue related to splicing during the lentivirus manufacturing process used to produce CAR-T cells. The company has identified the source of the splicing problem and has developed a solution to address it. They are currently remanufacturing the lentivirus. The FDA's letter also provides suggestions for improving the safety of HEMO-CAR-T, which are unrelated to the clinical hold and can be easily addressed.

Dr. Vladislav Sandler, CEO and Co-Founder of Hemogenyx Pharmaceuticals, expressed confidence in their ability to address the FDA's concerns and questions regarding the IND. AML has poor survival rates, and the company is committed to resolving the clinical hold in order to continue the development of this potentially life-saving treatment.

AML is the most common form of acute leukemia in adults and is currently treated primarily with chemotherapy. Hemogenyx Pharmaceuticals aims to develop a more effective and less harmful therapy for AML. Successful development of a new therapy for AML would have a significant impact on treatment and survival rates for the disease.

CAR-T therapy involves modifying a patient's own T-cells, a type of immune cell, to recognize and destroy cancer cells. The process includes isolating the patient's T-cells, modifying them in a laboratory using a CAR gene construct to target the patient's specific cancer, amplifying the modified cells, and then reintroducing them into the patient.

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