Xiangxue Pharmaceutical Co., Ltd (SHE: 300147) and Innostellar Biotherapeutics Co., Ltd have both been noted by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) as potential candidates for breakthrough therapy designations (BTDs). Xiangxue’s TAEST16001, a T-cell receptor TCR affinity-enhanced specific T-cell (TAEST) therapy, and Innostellar’s LX101, an RPE65-targeted gene therapy, are set to be recognized for their innovative approaches in treating specific conditions.
TAEST16001 marks a significant advancement as the first T-cell receptor-engineered T-cell (TCR-T) therapy in China. It targets the cancer-testis antigen NY-ESO-1, which is prevalent in various solid tumors such as epithelial ovarian cancer but limited in normal tissues. In July 2018, Xiangxue partnered with US-based Athenex Inc. (NASDAQ: ATNX) to establish Axis Therapeutics, a joint venture aimed at global development of TAEST technology. The BTD status for TAEST16001 is in relation to its clinical trial for patients with solid tumors that are both HLA-A*02:01 positive and NY-ESO-1 positive.
On the other hand, LX101 employs adeno-associated virus 2 (AAV2) to deliver the normal RPE65 gene, targeting and transfecting diseased retinal pigment epithelial cells. This therapy enables the continuous expression of the RPE65 protein, which is deficient in patients, thereby restoring normal visual circulation and improving visual function. The BTD indication for LX101 is for the treatment of patients with hereditary retinal malnutrition (IRD) associated with RPE65 double allele mutations.- Flcube.com
