SHANGHAI, Dec. 4, 2023 /PRNewswire/ -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announces that the U.S. Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance for CT071, an autologous CAR T-cell therapy candidate targeting G protein-coupled receptor class C group 5 member D (GPRC5D), for the treatment of patients with relapsed/refractory multiple myeloma (MM) or relapsed/refractory primary plasma cell leukemia (PCL).

GPRC5D is emerging as an important target in the landscape of MM, a common but incurable hematologic malignancy characterized by the uncontrolled proliferation of plasma cells. The notable overexpression of GPRC5D on the surface of malignant plasma cells, coupled with its limited presence on normal tissues, makes GPRC5D an ideal candidate for the treatment of MM and PCL. CT071 incorporates a fully-human single-chain variable fragment (scFV) developed by CARsgen, specifically designed to target GPRC5D.

CT071 is manufactured with CARsgen's proprietary CARcelerate^TM platform, which shortens the manufacturing time to less than 2 days and therefore yields younger, healthier, and possibly more potent CAR T cells compared to conventional manufacturing. The improved manufacturing efficiency also enhances the supply capacity, reduces the manufacturing costs, and expedites the availability of the product to the patients.

An investigator-initiated trial (IIT) is already under way in China to assess the safety and efficacy of CT071 in treating relapsed/refractory MM or PCL (NCT05838131). Preliminary clinical data from the IIT shows an acceptable safety profile with preliminary efficacy.

About CT071

CT071 is a CAR T-cell therapy candidate developed utilizing proprietary CARcelerate^TM platform of CARsgen targeting GPRC5D for the treatment of relapsed/refractory MM or relapsed/refractory PCL. An IIT (NCT05838131) is ongoing in China to evaluate the safety and efficacy of CT071 for the treatment of relapsed/refractory multiple myeloma or plasma cell leukemia.

About CARcelerate^TM

CARcelerate^TM is a proprietary platform CARsgen developed that can shorten the manufacturing time for the CAR T cells to less than 2 days, as compared to the conventional CAR T manufacturing process. The CARcelerate^TM platform produces CAR T cells that are younger, healthier, are more likely to remain in a 'naïve' state and less likely to be exhausted; as such, these CAR T cells from the CARcelerate^TM platform are expected to exhibit more potent tumor killing activity.

About CARsgen Therapeutics Holdings Limited

CARsgen is a biopharmaceutical company with operations in China and the U.S. and is focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. CARsgen has established a comprehensive CAR T-cell research and development platform, encompassing target discovery, innovative CAR T-cell development, clinical trials, and commercial-scale production. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs. CARsgen's vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

Forward-looking Statements

All statements in this press release that are not historical fact or that do not relate to present facts or current conditions are forward-looking statements. Such forward-looking statements express the Group's current views, projections, beliefs and expectations with respect to future events as of the date of this press release. Such forward-looking statements are based on a number of assumptions and factors beyond the Group's control. As a result, they are subject to significant risks and uncertainties, and actual events or results may differ materially from these forward-looking statements and the forward-looking events discussed in this press release might not occur. Such risks and uncertainties include, but are not limited to, those detailed under the heading "Principal Risks and Uncertainties" in our most recent annual report and interim report and other announcements and reports made available on our corporate website, https://www.carsgen.com. No representation or warranty is given as to the achievement or reasonableness of, and no reliance should be placed on, any projections, targets, estimates or forecasts contained in this press release.

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