Weekly Highlights | Gene Therapy News Digest Issue 121

Sep 23, 2024 18:23 CST Updated 18:23

Ribo Life Science

Small Nucleic Acid Drug Developer

BridgeBio

Pharmaceutical R&D Developer

Industry Dynamics Overview

Focus

Warm congratulations to SINEUGENE's SNUG01 for achieving positive clinical results in the IIT study at Peking University Third Hospital

On September 18, 2024, SINEUGENE announced that the investigator-initiated clinical study (IIT) of its self-developed innovative gene therapy drug SNUG01 for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease) conducted at Peking University Third Hospital has completed the enrollment of all subjects and achieved positive clinical outcomes. The study, led by renowned ALS expert Professor Dongsheng Fan from Peking University Third Hospital, aims to evaluate the safety and preliminary efficacy of SNUG01. As SINEUGENE's strategic partner, PackGene BioTech extends sincere congratulations on this significant progress!

Warm congratulations to Ruifeng Bio on the FDA approval of its world-first IND for Usher Syndrome ocular drug

On September 17, 2024, local time in the United States, the U.S. Food and Drug Administration (FDA) officially approved the IND application for RM-101, an innovative drug developed by Ruifeng Bio for Usher syndrome. This approval marks a milestone breakthrough and progress in the world's first AAV-based gene editing drug development for Usher syndrome. As a strategic partner of Ruifeng Bio, PackGene Biotherapeutics extends its heartfelt congratulations on this significant achievement!

Ribo Life Science's Self-Developed Small Nucleic Acid Drug for Treating IgA Nephropathy Receives First Clinical Trial Approval from the EU

On September 15, 2024, during the annual meeting of the 2024 European Society for Medical Oncology (ESMO), the latest clinical research data on WGc-043 injection, an mRNA vaccine for EB virus-positive tumors independently developed by Chengdu Westgene Biomedical Technology Co., Ltd., was released.

New data shows that WGc-043 has achieved breakthrough results in safety, immunogenicity, and anti-tumor activity.

One Step Forward, One Step Back, BridgeBio Advances AVV9 Gene Therapy Development for Canavan Disease

Recently, BridgeBio announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to the investigational gene therapy BBP-812 for the treatment of Canavan disease. This designation is based on the positive results from the Phase 1/2 clinical trial CANaspire, where all treated patients showed functional improvements, indicating that BBP-812 has the potential to become the first therapy to address the unmet needs of patients with Canavan disease.

CXO Sees Positive Developments! Ministry of Industry and Information Technology Issues Statement: Focus on Innovative Fields Such as Cell and Gene Therapy, Peptide Drugs, and Novel Antibody Drugs to Accelerate Layout

Recently, the Ministry of Industry and Information Technology (MIIT) issued a notice to accelerate the layout and construction of pilot platforms for the manufacturing industry, aiming to speed up the establishment of a number of urgently needed pilot verification platforms for high-quality development in manufacturing.

The "Guidelines for the Construction of Pilot Platforms in the Manufacturing Industry (2024 Edition)" mentions that in fields with high quality and safety requirements, such as pharmaceuticals, efforts should be accelerated to build intelligent and green pilot-scale platforms. It encourages the development of models like Contract Research Organization (CRO) and Contract Development and Manufacturing Organization (CDMO), enhances the public service efficiency of leading enterprises' pilot platforms, and lowers the threshold for start-up companies.

Shanghai Releases "Science and Technology Innovation Action Plan" Cell and Gene Therapy Special Project Application Guidelines

In order to further implement the innovation-driven development strategy and accelerate the construction of a globally influential science and technology innovation center, based on the "14th Five-Year Plan for the Construction of a Globally Influential Science and Technology Innovation Center in Shanghai," the "Action Plan for Promoting Cell Therapy Scientific and Technological Innovation and Industrial Development in Shanghai (2022-2024)," and the "Action Plan for Promoting Gene Therapy Scientific and Technological Innovation and Industrial Development in Shanghai (2023-2025)," the Shanghai Science and Technology Commission has specially released the 2024 "Science and Technology Innovation Action Plan" Cell and Gene Therapy Special Project Application Guidelines.

Innovation Breakthrough

Shu Yilai/He Yingzi Team Develops Multi-Site Expression Gene Replacement Therapy to Restore Hearing in DFNB111 Deaf Mouse Model

Recently, the team of Shu Yilai and He Yingzi from the Eye, Ear, Nose and Throat Hospital of Fudan University published a research paper titled "Hearing restoration by gene replacement therapy for a multisite-expressed gene in a mouse model of human DFNB111 deafness" in The American Journal of Human Genetics, a journal under Cell Press.

This study explored the efficacy of AAV gene therapy for Mpzl2-related hearing loss. The research team established a mouse model of autosomal recessive non-syndromic deafness (DFNB111) and designed an AAV-ie delivery system-based gene replacement strategy, which effectively delivered to various types of cochlear cells. Administration of AAV-ie-Mpzl2 successfully restored auditory function in Mpzl2-/- mice and recovered the structural integrity and survival rate of outer hair cells (OHCs) and Deiter’s cells (DCs).

Nature Sub-Journal: Zhejiang University's Yao Yuan Team Discovers Ultra-Small Cas13j Protein, Achieving Highly Efficient RNA Editing In Vivo

On September 19, 2024, the Yao Yuan team from Hangzhou International Science and Technology Innovation Center, Zhejiang University, published a research paper titled: Compact RNA editors with natural miniature Cas13j nucleases in the journal Nature Chemical Biology.

Data-Driven Protease Mining Can Leverage Species Diversity to Discover "Novel Proteins." This Study Employs BT-IT Fusion Technology, Using Intelligent Mining Algorithms to Perform Large-Scale Mining of Microbial Metagenomic Data, Leading to the Discovery of the Ultra-Compact Cas13j Protein and Its Efficient In Vivo RNA Editing.

Shanghai Institute of Materia Medica and Zhejiang University Team Collaboratively Develop an Efficient Delivery System, Providing a New Strategy for Gene Therapy

Recently, a research team led by Gan Yong, a researcher at the Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Yu Miaorong, an associate researcher, and Professor Hu Guoqing from Zhejiang University, published a research paper titled "Direct Cytosolic Delivery of siRNA via Cell Membrane Fusion Using Cholesterol-Enriched Exosomes" in Nature Nanotechnology. The study combines theoretical modeling and experimental research to deeply reveal the critical role and underlying mechanism of cholesterol in regulating exosome-mediated RNA drug delivery and develops an efficient engineered exosome RNA delivery system, providing a safe and effective innovative delivery strategy for gene therapy.

Cell.【Frontier】 | Novel Proteolipid Carriers for Safe and Efficient In Vivo Nucleic Acid Delivery

Safe and effective in vivo delivery of DNA and RNA using proteolipid vehicles

Capital Express

George Church's New Company! $65 Million Series A Funding to Develop Next-Generation Cell Therapy

On September 19, in Cambridge, Massachusetts, GC Therapeutics (GCTx) announced the completion of a $65 million Series A financing round to expand and unlock next-generation cell therapies. GCTx's induced pluripotent stem cell (iPSC) programming platform TFome™ (tee-eff-ome), invented and developed by a world-class team of scientists from Dr. George Church’s lab at Harvard Medical School and the Wyss Institute, integrates the latest advances in synthetic biology, gene editing, cell engineering, and machine learning to overcome development and scaling complexities associated with cell therapy and improve patient access across a wide range of disease areas. The financing was led by Cormorant Asset Management, with participation from Mubadala Capital, Andreessen Horowitz (a16z) Bio+Health, Medical Excellence Capital, Cercano Management, and Pear VC, bringing GCTx’s total funding to $75 million since its founding in 2019.

Novo Nordisk Reaches $600 Million Collaboration to Develop Liver-Targeted LNP Delivery Technology

NanoVation Therapeutics today announced a multi-year research and development partnership with Novo Nordisk to advance the development of innovative genetic medicines for cardiometabolic diseases and rare disorders.

$40 Billion Mainly Invested in Cell Gene Therapy, Medical Devices, and Other Fields: Jiangsu Wuxi Establishes a Special Biomedical Industry Mother Fund

Recently, the Wuxi Biomedical Industry Special Fund (Limited Partnership) in Jiangsu was established, with Wuxi Zhanxin Private Equity Fund Management Co., Ltd. as the general partner, and a capital contribution of 4 billion yuan.

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