The Boston R&D Center and GeneLeap Biotech, two subsidiaries of Luye Life Sciences Group, took part in the 24th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, presenting the progress of four of the company's latest preclinical developments in gene and cell therapy to the global biological community.

ASGCT is one of the world's most influential academic conferences in the area of gene and cell therapy. More than 3,500 scholars, clinical investigators, physicians and other professionals attend the meeting every year. The ASCCT annual meeting is held as a virtual event from May 11 to 14 this year.

Dr. Sean Fu, President of Luye Life Sciences Boston R&D Center, and CEO of GeneLeap Biotech, said, "This is the first time that we have presented information on the progress of our scientific research in gene and cell therapy at the ASGCT annual meeting. This reflects the strength of our R&D team's innovation and drug development in this field. We look forward to working with global scientists to expand our scientific progress for translational science and provide greater momentum of innovation for the development of the company in the future."

Details of the latest scientific progress presented by Luye Life Sciences are as follows:

• Immune Suppression with Rapamycin May Cause Prolonged IgM Formation Against AAV Vector

In collaboration with the Indiana University School of medicine, Luye Life Sciences Boston R&D Center and GeneLeap Biotech leveraged the strength of proprietary nanoparticle formulation technology to encapsulate immune suppressive drug rapamycin and compared it with its free form. Studies showed that rapamycin might be a more effective strategy to achieve second dose of AAV gene therapy but induced prolonged IgM responses.

• Strategies in AAV Upstream and Downstream Purification for High Yield Separation of Empty/Full Capsids

Luye Life Sciences Boston R&D Center conducted a series of studies on both the AAV upstream and downstream processes that offers greater feasibility to yield high viral genome and achieve excellent high purity with full AAV capsid. Such successful process will enable the AAV production to meet the demand for supporting both clinical and commercial efforts.

• Optimization of Human FIX Expression Cassettes for Superior Liver-targeted Gene Therapy of Hemophilia B

Together with Luye Life Sciences Boston R&D Center and the Indiana University School of medicine, GeneLeap Biotech identified a candidate drug AAV8.pXLLY027 with superior hFIX expression and improved potency, which holds the great potential to significantly reduce the administration dosage, safety concern and cost burden in clinical application.

• Therapeutic Efficacy of An Investigational AAV Gene Therapy in Alleviating Choroidal Vascularization in Rat Laser-induced CNV Model

GeneLeap Biotech generated an AAV gene therapy clinical lead AAV8.pTHVE002 expressing aflibercept was successfully selected with superior expression and efficacy, which lays a solid foundation for the researchers to advance clinical development of this investigational gene therapy for treating millions of wet AMD patients over the world with one-time administration. 

Dr. Jason Zhang, CSO of GeneLeap Biotech, said, "We are glad to have this opportunity to share our research in gene and cell therapy, including our clinical leads in hemophilia B and wet-AMD AAV gene therapy, as well as progress in AAV process development and immune tolerance. We look forward to continued collaborations with scholars worldwide in our efforts to address the unmet needs of patients."

About Luye Life Sciences Boston R&D Center

The R&D center at Boston, Woburn, MA is a subsidiary of Luye Life Sciences, developing novel drugs for cancer and genetic disorders, including gene therapy, immune checkpoint inhibitory antibodies, CAR-T cells therapy, and novel drug delivery mechanisms. At present, the AAV platform for gene therapy program developed by the Boston R&D center has several candidates in the pipeline.

About GeneLeap Biotech

GeneLeap Bio., a subsidiary of Luye Life Sciences, specializes in developing gene therapy drugs for cancer and severe infectious diseases, including mRNA and oligonucleotides. Currently, GeneLeap Biotech has R&D operations in both the USA (Boston) and China (Nanjing, Yantai).