Weekly Highlights | Gene Therapy News Digest No.117

Aug 26, 2024 18:42 CST Updated 18:42

StarryGene

Gene Therapy Drug Developer

Industry Dynamics Overview

Focus on Hotspots

Warm congratulations to StarryGene on the successful clinical approval of the IND for the expanded indication DME of "XMVA09 Injection"!

On August 19, 2024, StarryGene announced that the second Investigational New Drug (IND) application for its self-developed gene therapy Class I innovative drug "XMVA09 Injection" has been approved by the National Medical Products Administration (NMPA). In March this year, the IND application for XMVA09 Injection for the treatment of wet age-related macular degeneration (wAMD) received clinical tacit approval from the CDE. The newly approved indication is diabetic macular edema (DME).As a strategic partner of StarryGene, PackGene warmly congratulates this successful approval! We look forward to continuing our collaboration with StarryGene to accelerate the clinical application and commercialization process of XMVA09 injection, benefiting more patients.。

How a Patent War Profoundly Impacts the Entire Gene Therapy Field！

Recently, professionals in the gene therapy-related industries are attempting to block a ruling from taking effect. They believe that a recent decision by the U.S. District Court for the District of Delaware could potentially harm the interests of the entire AAV gene therapy sector.

Nature Sub-Journal: Harvard Chinese Scholar's Research Finds That Eating More Pork, Beef, and Lamb May Increase Diabetes Risk

On August 13, 2024, Professor Hu Bingchang's team from the Harvard T.H. Chan School of Public Health published a research paper titled: Integration of epidemiological and blood biomarker analysis links haem iron intake to increased type 2 diabetes risk in the Nature Metabolism, a sub-journal of Nature.

The study shows that heme iron intake from red meat is associated with an increased risk of type 2 diabetes (T2D). Increased heme iron intake correlates with adverse plasma profiles of insulinemia, blood lipids, inflammation, and T2D-related metabolites. The study also found that metabolites, including L-valine and uric acid, may mediate the relationship between heme iron and type 2 diabetes, highlighting their critical role in the pathogenesis of type 2 diabetes.

Nanjing Chengshi Biomedical Technology Co., Ltd.'s First Personalized Tumor mRNA Vaccine Clinical Trial Launched at Nanjing University Suzhou Hospital

Recently, the first clinical trial of personalized tumor mRNA vaccines has officially started at Suzhou Hospital Affiliated to Nanjing University Medical School. This research is an investigator-initiated clinical trial (IIT) led by Professor Zhang Junfeng. Its main purpose is to evaluate the safety and efficacy of personalized neoantigen vaccines combined with PD-1 inhibitors for adjuvant treatment after radical surgery for colorectal cancer, lung cancer, and gastric cancer. Professor Zhang Junfeng, former dean of Nanjing University Medical School, is a recipient of the National Science Fund for Distinguished Young Scholars and a Changjiang Scholar under the Ministry of Education. He has long been engaged in research on nucleic acid drug delivery systems and biotherapy.

Opus Genetics Gene Therapy Receives FDA Rare Pediatric Disease Designation

On August 20 local time, Opus Genetics announced that the FDA had granted its ocular gene therapy OPGx-LCA5 Rare Pediatric Disease (RPD) designation for the treatment of the inherited retinal disease LCA5. OPGx-LCA5 is an adeno-associated virus 8 (AAV8) vector designed to precisely deliver the functional LCA5 gene to the outer retina of patients with Leber congenital amaurosis (LCA) caused by biallelic mutations in the LCA5 gene. With this designation, OPGx-LCA5 will be eligible for a priority review voucher upon approval, which can be used for any subsequent marketing application that is saleable or transferable to other companies.

Lion Biotech: IND Application for mRNA TCR-T Injection Accepted by CDE, for Treatment of HPV-Related Hepatocellular Carcinoma

On August 21, 2024, according to the CDE official website, the new drug clinical trial application (IND) for "GZL-016 Injection" by Guangzhou Liven Biotech Co., Ltd. ("Liven Biotech" for short) was accepted. According to Liven Biotech, the indication for GZL-016 Injection is hepatitis B virus-related hepatocellular carcinoma. Liven Biotech owns the First-in-Class, world's first T-cell receptor therapy (TCR-T) targeting the hepatitis B virus surface antigen.

Innovation Breakthrough

Advantages and Potential of Miniature Base Editing Tools in AAV-Based Gene Therapy Applications

On August 15, 2024, the research team of Huihua (Shanghai) Biotechnology Co., Ltd., Huang Jinhai's team from the Eye, Ear, Nose and Throat Hospital of Fudan University, and Yang Hui's team from the Center for Excellence in Brain Science and Intelligence Technology of the Chinese Academy of Sciences collaborated to publish a research paper titled: Engineered IscB–ωRNA system with expanded target range for base editing in the journal Nature Chemical Biology.

Through metagenomic data mining, the study identified 19 novel IscB-ωRNA systems with varying Target Adjacent Motif (TAM) ranges. By integrating RNA structure optimization, protein engineering, flow cytometry, and off-target detection techniques, the researchers successfully developed an IscB system (IscB.m16*) with a broad target recognition range and higher editing efficiency in human cells.

New Progress in Brain-Computer Interface Enhancement Technology Based on Gene Editing

Research teams led by Fang Ying and Tian Huihui from the National Center for Nanoscience and Technology have made new progress in brain-computer interface enhancement technology based on bio-editing techniques. The related results were published in Advanced Materials under the title "Spatially Precise Genetic Engineering at the Electrode-Tissue Interface."

Nature Sub-Journal: Peking University's Zhang Ying Team Reveals Early Tumor Editing Mechanism and Its Potential Application in Tumor Immunotherapy

2On August 21, 2024, the research group of Zhang Ying from the School of Life Sciences at Peking University and the Peking-Tsinghua Center for Life Sciences, in collaboration with Harvard Medical School, published a research paper titled: Tumor editing suppresses innate and adaptive anti-tumor immunity and is reversed by inhibiting DNA methylation in the journal Nature Immunology.

This study utilized a transgenic mouse model capable of inducing breast cancer to collect very early and late-stage tumors for comprehensive single-cell transcriptome sequencing (scRNA-seq). It revealed the preferential silencing of key innate and adaptive immune pathways in early tumor editing, as well as significant impacts on TIL differentiation, function, and tumor-immune cell communication. Furthermore, it elucidated the important efficacy of DNA demethylation therapy in reversing tumor immune editing, promoting inflammatory cancer cell death, enhancing tumor immunogenicity, and boosting anti-tumor immunity.

Zhejiang University's Tang Jianbin/Sun Wenjing Develop Mildronate-Derived LNP with Extremely Low CL Ratio for Efficient mRNA Vaccine Delivery, Minimizing Inflammatory Side Effects

On August 16, 2024, the research group of Professor Tang Jianbin from the College of Chemical and Biological Engineering at Zhejiang University, in collaboration with the research group of Sun Wenjing from the Zhejiang University-Hangzhou Global Scientific Innovation Center, published a research paper titled “Low-Dose Mildronate-Derived Lipidoids for Efficient mRNA Vaccine Delivery with Minimal Inflammation Side Effects” in the journal ACS Nano. The research team considered that LNPs developed with minimal cationic lipid ratios might be the optimal solution to address safety concerns; thus, they developed mLNPs based on mildronate (mildronate), with a cationic lipid ratio of only 1%. These mLNPs were verified to possess highly efficient in vivo transfection efficacy and minimal inflammatory responses, showing promise in broadening the application prospects of mRNA in the prevention and treatment of various diseases.

Capital Express

Raised $150 Million! Novartis and Versant Collaborate Again to Establish xRNA Therapeutics Company

Novartis and venture capital firm Versant Ventures jointly announced on Thursday that Borealis Biosciences has emerged from stealth mode, securing a $150 million Series A funding round from founding investors Versant Ventures and Novartis, as well as strategic research collaboration funding committed by Novartis for the development of next-generation RNA therapeutics targeting kidney diseases.

Tome Biosciences Reported in Trouble, Founded by Zhang Feng’s Student, Completed Over $200 Million Financing 9 Months Ago

According to multiple informed sources, gene-editing startup Tome Biosciences is facing difficulties, even though the company completed a $213 million financing round just nine months ago.

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