Shanghai, China, and Lexington, MA., - October 24, 2023 - NeuShen Therapeutics (the “Company”), a global biotechnology company developing innovative treatments for patients with neurological and psychiatric disorders through a dual-platform strategy involving small molecule discovery and AAV-based gene therapy research, is pleased to announce the presentation of pre-clinical data from the collaborative NeuShen-UMass Chan Medical School ALS program. The data will be showcased in poster format at the forthcoming 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) taking place during October 24-27th, 2023 in Brussels, Belgium. Dr. Jun Xie, associate professor of microbiology & physiological systems at UMass Chan and the principal investigator of this program, will represent the team to introduce the data at the conference.

The joint research team behind this program employs recombinant adeno-associated virus serotype 9 (rAAV9) to systematically administer a single dose of optimized artificial microRNAs (amiRs) to target human SOD1 as a potential treatment for this specific subtype of familial ALS. The outcomes indicate that this treatment significantly delayed disease onset and limb weakness, improved motor function and breathing, and, most crucially, extended the survival of treated SOD1G93A mice by an average of 98.5 days compared to their untreated counterparts, demonstrating significant potential for clinical benefits.

The NeuShen-UMass Chan ALS program was established in January 2023 and is a three-year sponsored research initiative aiming to tackle ALS by adeno-associated virus (AAV) gene therapy, co-led by Dr. Jun Xie and Dr. Guangping Gao. Dr. Guangping Gao is professor of microbiology & physiological systems, Penelope Booth Rockwell Chair in biomedical research, director of the Horae Gene Therapy Center and Viral Vector Core, and co-director of the Li Weibo Institute for Rare Diseases Research at UMass Chan. The encouraging pre-clinical data serves as a testament to the synergistic capabilities and collaborative efforts fostered through this partnership, as both parties continue to work as a unified team to address ALS and explore the broader potential of gene therapy in treating various neurological and psychiatric disorders.

“We take immense pride in this significant progress, a direct result of our strong and very productive partnership with UMass Chan," stated Dr. Joan Shen, CEO and founder of NeuShen Therapeutics. "I hold great optimism for the ongoing advancement of this program, bringing us closer to providing treatment for our ALS patients."

“It is still early in development; however, the animal data are unprecedented. This could bring new hope to the ALS patients,” said Dr. Jun Xie. “The success of our optimized therapeutic strategy on the familial SOD1 mouse model encourages us to explore this approach on sporadic ALS.”

ALS is a rapidly progressive and fatal neurodegenerative disorder characterized by loss of motor neurons, which causes muscle weakness and paralysis, and leads eventually to early mortality typically 2-5 years after the onset of symptoms. As of today, there is neither a cure nor an effective treatment to reverse the progression of the disease. Nonetheless, numerous therapeutic approaches have emerged to treat ALS, focusing on ALS-linked genetic mutations, such as the superoxide dismutase 1 gene (SOD1). In this program, the research team aims to reduce mutant SOD1 using optimized artificial miRNAs (amiRs) through a single dosing approach, differentiating its strategy from traditional methods involving ASO or siRNA, which require repeated administration.

Presentation Details:

Presentation Title: Optimized Artificial miRNAs Delivered by AAV9 Dramatically Improve Survival, Respiratory and Motor Functions of SOD1G93A-ALS Mice

Presenting Author: Dr. Jun Xie, PhD, Associate Professor of Microbiology & Physiological Systems, UMass Chan Medical School

Poster Number: P398

Location: Poster Hall, Gare Maritime, Brussels, Belgium

Date & Time: October 25, 2023 18:15-19:30 and October 26, 2023 19:30-20:30

About NeuShen Therapeutics

NeuShen Therapeutics is a global 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.

About UMass Chan Medical School

UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chan School of Medicine; the Morningside Graduate School of Biomedical Sciences; the Tan Chingfen Graduate School of Nursing; Commonwealth Medicine, a public service consulting division; and MassBiologics, the only nonprofit, FDA-licensed manufacturer of vaccines, biologics and viral vector gene therapies in the United States. UMass Chan’s mission is to advance the health and wellness of our diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery and public service. In doing so, it has built a reputation as a world-class research institution and as a leader in primary care education, perennially ranked in the top 10 percent of medical schools for primary care by U.S. News and World Report. UMass Chan attracts more than $300 million annually in research funding, placing it among the top 50 medical schools in the nation. In 2021, the Medical School received a $175 million donation from The Morningside Foundation and was renamed UMass Chan Medical School.