CARsgen Therapeutics Receives Orphan Medicinal Product Designation from the European Medicines Agency for CT041 CLDN18.2 CAR T Cells for the Treatment of Gastric Cancers

SHANGHAI,March 1,2021 -- CARsgen Therapeutics Holdings Limited,a clinical-stage biopharmaceutical company,today announced that the European Commission (EC) has grantedorphan designation for CT041,CARsgen's first-in-class Claudin 18.2 (CLDN18.2) targeted CAR-T product candidate for the treatment of gastric cancer. CT041 consists of the patient's own T cells,genetically modified to express a humanized anti-CLDN18.2 chimeric antigen receptor (CAR) to treat patients with CLDN18.2-positive tumors.

"The orphan medicinal product designation of CT041 by the EC is another important recognition of CARsgen's commitment to the development of CAR T-cell treatment for patients with advanced gastric cancer," said Dr. Zonghai Li,founder,CEO and CSO of CARsgen. "According to the World Health Organization,over one million new cases of gastric adenocarcinoma are expected each year,and it is the seventh most prevalent cancer type worldwide[1]. Despite the development of novel therapies,gastric cancer is still a disease with one of the highest unmet medical needs. We reaffirm our long-standing commitment to cancer patients worldwide by expanding upon our CT041 clinical trial data to advance novel,safe and effective immunotherapies."

The EC grants orphan drug designation to investigational treatments for rare conditions,those affecting fewer than five in 10,000 people in the European Union. Treatments that meet the European Medicines Agency's orphan designation criteria qualify for incentives to encourage advancement of drug development.

CT041 is the first CLDN18.2-targeted CAR T-cell treatment that has received Investigational New Drug (IND) clearance by the US Food and Drug Administration (FDA) and the National Medical Products Administration (NMPA) in China. Three open-label,multicenter,Phase 1b clinical trials (NCT04404595,NCT04581473,and NCT03874897) to evaluate the safety and efficacy of autologous CT041 cell treatment in patients with advanced gastric,gastroesophageal,or pancreatic adenocarcinoma are currently underway.

References:

[1] Bray F,Ferlay J,Soerjomataram I,Siegel RL,Torre LA,Jemal A. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018;68(6):394-424.

About CARsgen Therapeutics

CARsgen Therapeutics is a China and U.S.-based clinical-stage biopharmacetucial company focused on innovative cell therapies for the treatment of solid tumors and hematological malignancies.

Founded in 2014,CARsgen is based in Shanghai,with operations in both China and the United States. CARsgen has established a broad pipeline of CAR T-cell product candidates covering several solid and blood tumors in areas of significant unmet medical need. The company has launched first-in-class CAR T-cell clinical trials for the treatment of relapsed/refractory tumors,includingCLDN18.2-targeted CAR-T product candidate for gastric/gastroesophageal junction cancer and pancreatic cancer and GPC3-targeted CAR-T product candidate for hepatocellular carcinoma (HCC). CARsgen also has ongoing clinical CAR T-cell programs with a fully human CAR-BCMA for multiple myeloma for the treatment of refractory/relapsed multiple myeloma and a humanized CAR-CD19 for the treatment of refractory/relapsed B-cell non-Hodgkin lymphoma.

For more information,please visit: www.carsgen.com

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