LX101 and TAEST16001 Among Innovative Therapies Granted Breakthrough Therapy Designation in China

　　【Pharmaceutical Network Industry Dynamics】With the continuous advancement of medical technology, drug research and development has made breakthroughs in the search for cures for diseases. However, for those life-threatening diseases that lack effective treatment options, new breakthrough therapies are particularly crucial. To accelerate the development and market entry of breakthrough treatments, China's "Drug Registration Administration Measures" has introduced a breakthrough therapy designation process, providing a more flexible pathway for the discovery and application of innovative drugs.
 

It is reported that recently, several innovative drugs have been included in the breakthrough therapy drug program, including "LX101 Injection" jointly applied by Shanghai Langsheng Biotechnology Co., Ltd., and "TAEST16001 Injection" jointly applied by GuangDong XIANGXUE LIFE SCIENCES LTD, etc.
 

Data shows that LX101 injection is suitable for treating patients with inherited retinal dystrophy (IRD) associated with biallelic RPE65 mutations. This injection is a gene therapy drug using rAAV as a vector, and its treatment for RPE65 mutation-related IRD has entered the phase 3 clinical trial stage.
 

Public data shows that IRD caused by RPE65 gene mutations is a typical blinding rare disease, with onset during infancy or early childhood. Nearly all patients eventually progress to complete blindness. Currently, there are no effective clinical treatments for this condition. LX101, developed by Shanghai Langsheng Biotechnology Co., Ltd., is a gene therapy drug targeting inherited retinal degeneration. The drug uses adeno-associated virus type 2 (AAV2) to carry the normal RPE65 gene, specifically recognizing and transfecting diseased retinal pigment epithelial cells, enabling continuous and efficient expression of the RPE65 protein that patients lack. This restores normal visual cycling and achieves the therapeutic goal of improving visual function. In previously conducted investigator-initiated clinical studies, LX101 demonstrated good safety and has shown efficacy in improving vision among several patients.
 

It is reported that Shanghai Langsheng Biotechnology Co., Ltd. is dedicated to the research, development, and manufacturing of innovative gene therapy drugs, focusing on gene therapy for hereditary and chronic eye diseases. The company has advanced 5 candidate molecules into clinical trials.
 

TAEST16001 Injection from GuangDong XIANGXUE LIFE SCIENCES LTD is a TCR-T cell immunotherapy product intended for the treatment of soft tissue sarcoma with HLA-A*02:01 genotype and positive expression of tumor antigen NY-ESO-1.
 

Data shows that AEST16001 is a TCR-T cell immunotherapy product independently developed by GuangDong XIANGXUE LIFE SCIENCES LTD. It targets NY-ESO-1 cancer-testis antigen TCR-T cells. The patient's T cells are transduced ex vivo with a lentivirus carrying genetically engineered tumor antigen-specific TCR genes. These T cells expressing the anti-tumor antigen TCR are then massively expanded to approximately 1×10⁹~2×10¹⁰ cells. After the patient undergoes lymphodepletion with an appropriate dose of medication (lymphocyte depletion), the TCR-T cells are reinfused into the patient.
 

Currently, the Phase I clinical trial of TAEST16001 has been completed, and a Phase II clinical trial for the treatment of patients with advanced soft tissue sarcoma is underway. It is reported that at the ASCO meeting held in June 2024, researchers presented a summary of interim data from this Phase II clinical trial.
 

Statistics show that in the first half of 2024 (up to June 21), 39 new drugs were officially included in the list of breakthrough therapies after public announcement. Most of these drugs are in the field of cancer treatment, with 30 of them accounting for a high proportion of 77%. Among them, there are more projects for the treatment of lung cancer, breast cancer, and colon/colorectal cancer.
 

Industry insiders pointed out that, with the implementation of the breakthrough therapy drug work program, an increasing number of new drugs have received recognition and encouragement from regulatory authorities. By accelerating the R&D process of new drugs and shortening the time to market, these innovative drugs are expected to be applied in clinical settings earlier.
 

Disclaimer: In no event shall the information or opinions expressed in this article constitute investment advice to any person.