Weekly Highlights | Gene Therapy News Digest Issue 148

Apr 28, 2025 18:39 CST Updated 18:39

Novabio Therapeutics

Developer of Multi-Modal Treg Cell Therapy

Gritgen

Gene Therapy Drug Developer

Industry News FlashView

Focus on Hotspots

Wuhan University's Zhang Xianzheng Team Achieves Chemoimmunotherapy for Colorectal Cancer Using Orally Administered Gene-Editing Nanoparticles

On April 23, 2025, the Zhang Xianzheng team at Wuhan UniversityNature Nanotechnology(IF=38.1) Published online a research paper titled "An orally administered gene editing nanoparticle boosts chemo-immunotherapy in colorectal cancer," which shows that disruption of the tumor necrosis factor receptor-associated protein 1 (TRAP1) gene leads to the translocation of cyclophilin D in tumor cells, a gene encoding the mitochondrial chaperone protein in tumor cells.

China Approves Clinical Trial for World's First Intrathecal Cell Drug to Treat ALS

On April 24, 2024, the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) officially approved the clinical trial application (acceptance number: CXSL2500094) for "Human Autologous Polyclonal Regulatory T-Cell Injection" by Shanghai Saierxin Biomedical Technology Co., Ltd. The indication is Amyotrophic Lateral Sclerosis (ALS, commonly known as Lou Gehrig's disease). As the world’s first innovative therapy using intrathecal injection of Treg cells to treat ALS, this breakthrough offers hope to potentially transform the survival prospects of hundreds of thousands of ALS patients globally.

Gritgen Therapeutics' First AAV Gene Therapy Drug Granted Breakthrough Status by CDE

On April 24, 2025, the breakthrough therapy designation announced on the official website of the Center for Drug Evaluation (CDE) under the China National Medical Products Administration showed that GS1191-0445 Injection, a Class 1 innovative drug independently developed by Gritgen Therapeutics Co., Ltd., has been included as a breakthrough therapy. The public comment period for this designation is from April 16 to April 23, 2025. This marks a high level of recognition for the company's continuous innovation capabilities in the field of gene therapy and also signifies that China’s research and development in hemophilia gene therapy has reached an advanced global level.

Provides New Insights for the Development of Novel RNAi Tools and Gene Therapy Drugs: The Westlake University Team Reveals for the First Time the Dynamic Cutting Mechanism of Human AGO-siRNA "Scissors"

Beijing time, April 16, 2025, the team of Enzhi Shen from the School of Life Sciences at Westlake University collaborated with the team of Jianping Wu inCell ResearchThe research成果 titled "Mechanistic Insights into RNA Cleavage by Human Argonaute2-siRNA Complex" was published online in the journal.

This article reveals for the first time the unique molecular mechanism of the human AGO2 (hAGO2)-siRNA complex in cleaving target RNA, indicating that the AGO-siRNA complex needs to undergo precise and strictly regulated conformational changes to mediate efficient gene silencing.

Professor Zhong Guisheng's team from Weimei Gene establishes in vivo transcriptional enhancer reconstruction technology, providing a novel solution for genetic deafness gene therapy

Recently, Professor Zhong Guisheng's team published in the international academic journal Neuron (Neuron) published research titled "Deciphering enhancers of hearing loss genes for efficient and targeted gene therapy of hereditary deafness," proposing and validating an innovative in vivo transcriptional enhancer reconstruction technology—ARBITER (AAV reporter-based in vivo transcriptional enhancer reconstruction). This breakthrough not only opens new avenues in cochlear basic research but also demonstrates significant potential in the study of underlying mechanisms of gene expression regulation and precise gene therapy for hereditary deafness.

Mol Ther | Gao Guangping Team Summarizes the Complexity and Challenges of AAV Immunology

On March 28, 2025, the team led by Professor Guangping Gao from the Horae Gene Therapy Center at UMass Chan Medical SchoolMolecular TherapyThe journal published a review paper titled "The curious case of AAV immunology," which mainly explores the complexities of adeno-associated virus (AAV) immunology, including the interaction between AAV and the host immune system, immune challenges in clinical applications, and future research directions.

Innovative Breakthrough

Novel mRNA Delivery System: Molecular Therapy Reveals New Avenues for mRNA Tumor Antigen Delivery Systems

On February 27, 2025, the related research findings were published in an international authoritative journal under the title "A Universal Viral Capsid Protein Based One Step RNA Synthesis and Packaging System for Rapid and Efficient mRNA Vaccine Development."Molecular TherapyUp.

Mol Ther | Recent Advances in Gene Editing Technologies for Disease Treatment

On March 21, 2025, the team of Professor Renzhi Han from the Departments of Pediatrics and Molecular Medicine and Genetics at Indiana University School of Medicine published a review article titled "Recent advances in therapeutic gene-editing technologies" in the journal Molecular Therapy. The review primarily focuses on the latest advancements in gene-editing technologies, especially the CRISPR system, for therapeutic applications, including the development of next-generation gene-editing tools such as Base Editors and Prime Editors and their applications in preclinical and clinical research. It also explores the potential and challenges of these technologies in the field of gene therapy.

Cell Sub-Journal: Nobel Prize Team Unveils the Mystery of CRISPR Gene Editing Efficiency

Recently,Molecular CellThe journal published the latest research revealing the core mechanism behind it. This study, led by Nobel laureate Professor Jennifer Doudna (with postdoctoral researcher Shi Honglue as the first author of the paper), uncovered the fundamental trade-off between broad PAM recognition and genome editing efficiency. It proposed that efficient RNA-guided genome editing relies on an optimized two-step target capture process, where selective but low-affinity PAM binding takes precedence over rapid DNA unwinding. This not only unravels the efficiency code of CRISPR-Cas9 gene editing but also lays the foundation for designing more effective CRISPR-Cas and related RNA-guided genome editors.

Nat Commun | Dose-dependent adverse events triggered by AAV gene therapy in the CNS are associated with DNA damage response-dependent pro-inflammatory signaling

On April 18, 2025, the team led by Professor Anna Kajaste-Rudnitski at the Gene Therapy Center of the San Raffaele Telethon Institute for Gene Therapy in Milan, ItalyNature CommunicationsJournal article titled "AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain". The study mainly explores the DNA damage response-dependent pro-inflammatory signaling pathways triggered by adeno-associated virus (AAV) vectors in human induced pluripotent stem cell (iPSC)-derived central nervous system (CNS) models and mouse brains, revealing the intrinsic immune mechanisms of neurotoxicity potentially caused by AAV vector gene therapy, and proposes potential intervention strategies to improve the safety of CNS-targeted AAV gene therapy.

Capital Express

U.S. Department of Health and Human Services Plans to Pay Moderna $176 Million for mRNA Avian Influenza Vaccine

According to the Associated Press, the U.S. Department of Health and Human Services announced on April 22, 2025, that the government will invest $176 million in Moderna to accelerate the development of a pandemic influenza vaccine.

Total investment of 300 million yuan: A cell medical industry innovation base opens in China

In response to the national strategy for the coordinated development of the Beijing-Tianjin-Hebei region and following the clarion call of the "Healthy China 2030" era, Zhongrui Detai Biotechnology Group, under the investment promotion efforts of the Dingzhou Municipal People's Government, has moved from Beijing to Dingzhou. With an investment of 300 million yuan, the group is constructing the Beijing Contemporary Innovation Research Biomedical Industrial Park. Spanning 33,000 square meters, it aims to build China's leading cell medical industry innovation base. The park focuses on three core industrial sectors: "medical device intelligent manufacturing, cell technology research and development, and cell medical innovation," forming a closed-loop ecosystem for the biopharmaceutical industry that spans from basic research to industrial application. As the core entity of the industrial park, Ochuang Keyi Cell Medical Group has emerged accordingly.

Pudong Establishes 1 Billion Yuan Biomedical Fund

Recently, China Life Leading (Shanghai) Private Equity Investment Fund (hereinafter referred to as "China Life Leading Fund") was officially inaugurated in Pudong. Pudong Venture Capital Group, China Biologic Products, SDIC Xiantao, Foteng Capital, and Hangzhou Cobetter Filtration joined as fund partners. The fund also signed agreements with industrial partners such as LaNova Medicines, Eternity Bioscience, and Insilico Medicine.

Recommended Reading:Pudong Establishes 1 Billion Yuan Biomedical Fund

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