Shanghai, China, and Lexington, MA., - April 18, 2024 - NeuShen Therapeutics (the “Company”), a global clinical-stage biotechnology company dedicated to developing innovative treatments for neurological and psychiatric disorders through a dual-platform strategy involving small molecule discovery and AAV-based gene therapy research, today announced that it will deliver four presentations at the American Society for Gene and Cell Therapy (ASGCT) annual meeting in Baltimore, Maryland, from May 7-11, 2024. One abstract produced by the NeuShen-UMass Chan joint ALS program is selected as an oral presentation, which also received the Excellence in Research Award. The other three presentations will be in poster format, showcasing the latest data from the Company’s AAV capsid engineering and ALS programs.

Gene therapy has been one of the key focus areas for the Company since its inception. The Company has established a proprietary and innovative AAV capsid engineering platform, enabling the discovery of novel capsids with high tropism for CNS and minimal targeting of peripheral tissues. Meanwhile, the Company has rapidly developed a strong portfolio through both independent research and academic collaboration. Beginning with genetic ALS and rare diseases, the Company plans to broaden its discovery scope to include common neurodegenerative diseases, such as Alzheimer's disease and Parkinson’s disease. The ALS program, co-developed with UMass Chan Medical School, has produced promising data, demonstrating compelling efficacy in an ALS mouse model. The results were presented at the European Society of Gene and Cell Therapy (ESGCT) annual meeting last year and will be updated with latest findings at the upcoming conference.

Dr. Huaqiong Shen, founder and CEO of the Company, highly praised the Company’s promising development in the field of gene therapy. She said, "We are immensely proud to bring 4 presentations to ASGCT, one of the most prestigious academic conferences in gene therapy, as we make our inaugural appearance. This underscores our profound scientific expertise and robust innovative capabilities in gene therapy. Our AAV capsid engineering platform continues to achieve scientific breakthroughs, and we're making steady progress within our ALS project. Our collaboration with world-class ALS researchers from UMass is also yielding positive results. I hope that our innovative delivery platform will accelerate CNS drug development and make therapeutic products available to patients with neurodegenerative diseases soon.”

Presentation details are as follows:

Oral Presentation:

Presentation Title: Optimized Artificial miRNAs Delivered by rAAV9 Dramatically Improve Survival, Respiratory and Motor Functions of SOD1(G93A)-ALS Mice

Presenting Author: Dr. Fang Wan, PhD, Postdoctoral Researcher, UMass Chan Medical School Gene Therapy Center

Date & Time: 5/10/2024 3:45PM-4PM

Abstract Number: 303

Session: Neurologic Diseases IV

Location: Room 307-308

Poster Presentations:

Presentation Title (1): Discovering rationally-engineered AAV5-derived capsid variants for superior CNS tropism and peripheral tissue de-targeting in non-human primate via systemic administration

Presenting Author: Dr. Sha Zhu, PhD, Associate Director of Discovery Biology, Gene Therapy, NeuShen Therapeutics

Date & Time: 5/8/2024 12PM

Poster Number: LBA-20

Session: Wednesday Posters: Late-Breaking Abstracts

Location: Exhibit Hall

Presentation Title (2): Directed Evolution of Rationally-Engineered AAV Capsid Variants with Enhanced Central Nervous System (CNS) Tropism Following Systematic Administration

Presenting Author: Dr. Hongxing Wang, PhD, Senior Director of Discovery Biology, Capsid Engineering, NeuShen Therapeutics

Date & Time: 5/9/2024 12PM

Poster Number: 986

Session: Thursday Posters: AAV Vectors - Capsid Engineering

Location: Exhibit Hall

Presentation Title (3): Identification of Artificial microRNAs Targeting C9orf72 Sense and Antisense Transcripts for ALS treatment

Presenting Author: Dr. Gabriela C. Toro, PhD, Senior Scientist of Discovery Biology, Gene Therapy, NeuShen Therapeutics

Date & Time: 5/10/2024 12PM

Poster Number: 1593

Session: Friday Posters: Neurologic Diseases

Location: Exhibit Hall

 

About NeuShen Therapeutics

NeuShen Therapeutics is a global clinical-stage biotechnology company focused on innovative drug research and development to address neurological and psychiatric disorders, using a dual-platform strategy consisting of small molecule discovery and AAV-based gene therapy. With operations in Shanghai, China and Lexington, MA., NeuShen boasts a world-class R&D team in CNS field and is honored to be advised by an outstanding board of directors and scientific advisory board.