Sinobio's Innovative CAR-T Therapy for Malignant Glioma Receives FDA Orphan Drug Designation

Orphan drugs refer to pharmaceuticals used for the prevention, treatment, and diagnosis of rare diseases. In the United States, rare diseases are defined as conditions that affect fewer than 200,000 people. According to the U.S. Orphan Drug Act, drugs granted orphan drug designation in the U.S. can enjoy a range of accelerated review and special support policies, including seven years of market exclusivity after new drug approval, exemption from BLA application fees, potential waiver of certain clinical data submission requirements, and tax deductions for clinical research expenses.

  

The CAR-T cell carries two CAR molecules on its surface, targeting IL13Rα2 and HER2, which are expressed in glioblastoma. At the same time, this CAR-T therapy can secrete multispecific antibodies (BiTE) targeting EGFR and EGFRvIII. The BiTE molecule consists of two nanobodies (VHH) and a single-chain variable fragment (scFv), connected in tandem by a flexible fusion linker, capable of activating autologous T cells to exert cytotoxic effects. Glioblastoma is the most malignant type of astrocytic tumor, with rapid growth; 70%-80% of patients have a disease course of 3-6 months, and only 10% survive longer than one year. Current treatment methods primarily include surgery, radiotherapy, chemotherapy, tumor-treating fields, and other comprehensive treatments, indicating significant unmet clinical needs.

Simnova is a cell therapy biotechnology company at the clinical development stage. Established in 2019, it was originally a subsidiary of Simcere Pharmaceutical Group and became an independently operated company after being spun off in 2021. The company is currently focusing on developing globally competitive universal cell therapy products while actively advancing the clinical research and application of multiple highly differentiated solid tumor products. Additionally, through collaboration with ORNA, a prominent foreign biotechnology company, Simnova is also developing next-generation universal in vivo cell therapy products based on circular mRNA and targeted delivery platforms. This technology represents another potentially disruptive form of universal cell therapy beyond CAR-NK, with significant potential for broad applications in treating cancer, autoimmune diseases, and more.