Weekly Highlights | Gene Therapy News Brief No.152

Jun 09, 2025 18:01 CST Updated 18:01

NeoCura

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Qiming Venture Partners Founder: The "DeepSeek Moment" for China's Innovative Drugs

Gary Rieschel, founding managing partner of Qiming Venture Partners, stated that 10 years ago, China lagged behind the United States by 20 years in the development of first-in-class drugs. Today, it is only two to three years behind. Within a short decade, this represents an astonishing compression of time. China’s biotechnology ecosystem is becoming increasingly sophisticated, and the “DeepSeek moment” may occur when Akeso’s bispecific antibody Ivonescimab (AK112) is launched with efficacy twice that of Keytruda.

AAV Gene Therapy: Illuminating Hope for Children with Inherited Blindness

A study published in the prestigious international medical journal *The Lancet* has brought new hope to these children. The study shows that the AAV gene therapy rAAV8.hRKp.AIPL1 can safely and effectively improve the vision of children with rare hereditary eye diseases caused by AIPL1 gene mutations, significantly enhancing their quality of life. It also paves a new way for the treatment of hereditary eye diseases.

Shenxi Bio: Positive Results of the World's First AAV-Based Glioma Clinical Study Announced

Recently, the IIT clinical study of NXL-004 for the treatment of malignant glioma, conducted collaboratively by the Fourth Affiliated Hospital of Soochow University and NeuExcell Therapeutics, has successfully completed patient enrollment. Preliminary analysis indicates positive outcomes. The study, led by Professor Huang Yulun from the Department of Neurosurgery at the Fourth Affiliated Hospital of Soochow University, aims to evaluate the safety and preliminary efficacy of NXL-004 in patients with recurrent or progressive malignant glioma. This research represents the first-in-human study based on in-situ transdifferentiation technology and is also the world's first AAV-based clinical study for glioma.

USTC Collaborates with Xingmu Team to Achieve Breakthrough Progress in AAV Gene Therapy for nAMD

Recently, the University of Science and Technology of China (hereinafter referred to as "USTC"), the First Affiliated Hospital of USTC, and StarMab Bio, among other teams, published a groundbreaking research achievement in the authoritative journal *Research*. They developed an adeno-associated virus (AAV) vector that specifically targets retinal pigment epithelial cells (RPE). Through intravitreal injection, it can express a bispecific antibody that simultaneously targets VEGF-A and angiopoietin-2 (ANG-2), successfully treating nAMD in animal models and patients.

AI Empowers Vaccine Development! NeoCura’s mRNA Personalized Tumor Neoantigen Vaccine XH001 Enters Clinical Trials

On June 4, the Center for Drug Evaluation (CDE) of the National Medical Products Administration officially approved the clinical research of NeoCura's self-developed mRNA personalized tumor neoantigen vaccine XH001. This breakthrough not only marks a new phase in AI + healthcare but also signifies China's entry into the first tier in the global race for tumor immunotherapy, accelerating the industry into a "golden age."

An AAV Gene Therapy for Neurodegenerative Diseases Is About to Be Submitted for Market Approval

Recently, uniQure, a gene therapy company, announced significant progress in the regulatory advancement of its gene therapy candidate AMT-130 for Huntington's disease. The company has reached an agreement with the U.S. Food and Drug Administration (FDA) on key elements of the statistical analysis plan and Chemistry, Manufacturing, and Controls (CMC) information related to the drug. uniQure plans to submit a Biologics License Application (BLA) in the first quarter of 2026. Previously, the drug received designations such as Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track.

Innovative Breakthrough

STTT: AAV Gene Therapy for Alzheimer's Disease

Recently, researchers at the University of California, San Diego, published a research paper titled "Neuron-targeted caveolin-1 overexpression attenuates cognitive loss and pathological transcriptome changes in symptomatic Alzheimer’s disease models" in the journal Signal Transduction and Targeted Therapy.

The study shows that using AAV9 to deliver Caveolin-1 to neurons can alleviate cognitive loss and pathological transcriptomic changes in a mouse model of Alzheimer's disease with existing symptoms.

Recommended Reading:STTT: AAV Gene Therapy for Alzheimer's Disease

Nat Commun | Engineered U7 Small Nuclear RNA Scaffold Enhances ADAR-Mediated Programmable RNA Base Editing with a 25-Fold Increase in DMD

On May 26, 2025, Adrian W. Briggs' team from Shape Therapeutics published a research article titled "An engineered U7 small nuclear RNA scaffold greatly increases ADAR-mediated programmable RNA base editing" in the journal Nature Communications. The study developed an engineered U7 small nuclear RNA (snRNA) scaffold that significantly enhances RNA editing efficiency using endogenous ADAR enzymes.

Nat Commun | Pre-mixing enables encapsulation of long-chain RNA into cubic phase lipid nanoparticles

On May 30, 2025, a research team led by Professor Hojun Kim and Professor Ji-Hoon Kim from the Korea Institute of Science and Technology (KIST) published a research paper titled “Premixing enables loading of long RNA in cubic phase lipid nanoparticles” in the journal Nature Communications. The study mainly proposes a method to successfully encapsulate long-chain RNA (up to 4000 bases) into cubic phase lipid nanoparticles (cubosomes) through a pre-mixing strategy.

The study found that the pre-mixing strategy effectively maintained the cubic structure of cubosomes and significantly enhanced their ability to encapsulate long-chain RNA. Moreover, the encapsulated cubosomes retained excellent delivery efficiency and stability after being stored at room temperature for 24 days. This achievement not only provides new insights into the development of mRNA vaccines that do not require a cold chain but also offers important perspectives on integrating macromolecular cargoes (such as peptides or polymeric materials) into other liquid crystal systems.

Nat Biotechnol | Dual SORT LNPs for Multi-Organ Base Editing

On June 2, 2025, Professor Daniel J. Siegwart's team from the University of Texas Southwestern Medical Center published a research paper titled "Dual SORT LNPs for multi-organ base editing" in the journal Nature Biotechnology. The study primarily developed dual-targeting lipid nanoparticles (Dual SORT LNPs) to simultaneously deliver base editors to the liver and lungs, correcting the genetic mutation that causes alpha-1 antitrypsin deficiency (AATD). Stable gene editing effects lasting up to 32 weeks were achieved in mouse models, providing a new strategy for gene therapy of multi-organ diseases.

Nat Commun | A research team led by Xianjin Xiao from Huazhong University of Science and Technology has developed a novel small-molecule detection method based on a split crRNA Cas12a system.

On May 30, 2025, Professor Xiao Xianjin and Professor Cheng Liming from the Department of Laboratory Medicine at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, published a research article titled "Spatially blocked split CRISPR-Cas12a system for ultra-sensitive and versatile small molecule activation and detection" in Nature Communications. The study introduces an SBS-Cas system where small molecules are modified on the scaffold strand. Spatial hindrance is introduced through binding reactions between small and large molecules, masking the trans-cleavage activity of Cas12a. When the target small molecule competitively binds to the large molecule with the scaffold, this spatial hindrance is removed, activating the trans-activity. The system demonstrates excellent sensitivity and versatility, successfully achieving signal response in the complex intracellular environment. This innovative CRISPR-Cas12a detection platform provides a new solution for small molecule detection.

Nat Biotechnol | Li Dali/Chen Liang Collaborate to Report Base Editor for Modeling and Correcting Mitochondrial Diseases

On June 3, 2025, the research team led by Li Dali from East China Normal University and the research team led by Chen Liang from Lingang Laboratory published two research papers on the construction and correction of mitochondrial disease models in the journal Nature Biotechnology.

Capital Express

$160 Million: An Ophthalmology AAV Gene Therapy Drug Reaches Licensing Agreement

On June 6, 2025, Ocugen (NASDAQ: OCGN) announced that it will sign a licensing agreement with a leading pharmaceutical and healthcare company in South Korea. Under the agreement, Ocugen will receive upfront license fees and near-term development milestone payments totaling up to $11 million. Within the first 10 years of commercialization in the South Korean market, Ocugen is eligible to receive additional sales milestone payments of at least $150 million, along with a 25% royalty on net sales. Furthermore, Ocugen will be responsible for the commercial manufacturing of OCU400.

160 Million Yuan! Southeast University Completes the Transformation of "New Type Circular RNA Drugs" Achievements

Southeast University Completed the Technology Transfer Agreement for "Novel Circular RNA Drugs," Injecting Cutting-Edge Research Achievements into Industrial Development with a High Conversion Amount of 160 Million Yuan, Marking a New Journey in the Industrialization of Circular RNA-Based Stroke Drug Treatment.

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