Gene Therapy Weekly Digest No.173 Highlights Major Advances in CAR-T Reimbursement, AAV Clinical Milestones, and Financing

Nov 10, 2025 19:08 CST Updated 19:08

Juventas

Innovative Cell Therapy Drug Developer

Opus Genetics

Gene Therapy Developer

Industry News FlashView

Focus on Hotspots

Negotiation Successful! Gene Cell Therapy Costing Millions Per Dose May Be Included in Commercial Insurance

On the afternoon of November 2, 2025, at around 3 PM, at the scene of the National Medical Insurance Catalog negotiations and the commercial insurance innovative drug price consultation, the CEO of Juventas led the negotiation team out of the venue. Facing reporters' inquiries, she smiled and said: "We've reached an agreement!" Although the final result still awaits the official announcement by the National Healthcare Security Administration, this positive signal undoubtedly brings a historic turning point for the accessibility of the "astronomically priced" CAR-T gene-cell therapy drugs.

World's First Angelman Syndrome AAV Gene Therapy Completes Dosing of First Participant

Recently, MavriX Bio announced that the first patient has been dosed in the phase 1/2 clinical trial ASCEND-AS (NCT07181837) for its investigational AAV gene therapy MVX-220, which is being developed to treat Angelman syndrome. This milestone not only marks the official entry of the world’s first gene therapy targeting Angelman syndrome into human clinical evaluation but also brings new hope to the entire field of neurodevelopmental disorders.

Recommended Reading：World’s First Angelman Syndrome AAV Gene Therapy Completes Dosing of First Participant

An ophthalmology AAV gene therapy receives positive feedback from the U.S. FDA, set to launch pivotal Phase III clinical trials

Recently, Opus Genetics announced that the clinical development program for its investigational AAV8 gene therapy, OPGx-LCA5, has received positive support from the U.S. FDA and is about to enter the pivotal Phase III clinical research stage, bringing new hope for the treatment of this ultra-rare inherited retinal disease.

$141 Million Series B Financing! Development of Ophthalmic Gene Therapies

On November 3, 2025, AAVantgarde Bio, a clinical-stage biotechnology company focused on treating inherited retinal diseases (IRDs), announced the successful completion of a $141 million Series B financing round. The round was led by Schroders Capital as the new lead investor, alongside existing investors Atlas Venture and Forbion. Other new investors included Amgen Ventures, Athos Capital, CDP Venture Capital (through its large venture fund), Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture, and Willett Advisors. The company also received continued strong support from existing investors Longwood Fund and Sofinnova Partners.

Giant Falls! James Watson, Discoverer of the DNA Double Helix, Passes Away

2On November 6, 2025, renowned American molecular biologist James Watson passed away at the age of 97. As one of the co-discoverers of the DNA double helix structure, Watson's name has long been engraved on the monument of modern life sciences.

Innovative Breakthrough

Nat Biotechnol | Site-specific DNA insertion in the human genome using engineered recombinases

On November 6, 2025, Professor Patrick D. Hsu's team from the Arc Institute in the United States published a research article titled "Site-specific DNA insertion into the human genome with engineered recombinases" online in the journal Nature Biotechnology. This study established a systematic recombinase engineering framework, achieving highly efficient optimization of large serine recombinases (LSR) through strategies such as directed evolution, structural modeling, machine learning, and dCas9 fusion. Using Dn29 as a model, the research team obtained several high-performance variants (superDn29, goldDn29, and hifiDn29), achieving integration efficiencies of up to 53% and specificities of 97% at endogenous human genomic sites. The optimized system enables efficient integration of DNA sequences up to 12 kb in length in non-dividing cells, stem cells, and primary T cells, maintaining stable expression with almost no transcriptomic perturbation.

Nat Commun | Zidong Qiu's team from Shanghai Jiao Tong University collaborates to report engineered APOBEC cytidine deaminase fused with PUF protein for efficient in vivo RNA base editing

On November 4, 2025, Professor Zilong Qiu from Shanghai Jiao Tong University School of Medicine and Professor Zefeng Wang's team from Southern University of Science and Technology published a research paper in Nature Communications titled “Effective in vivo RNA base editing via engineered cytidine deaminase APOBECs fused with PUF proteins”. This study utilized AI-assisted structural design to optimize APOBEC family proteins, constructing an efficient and specific RNA base editing system named CU-REWIRE5, capable of achieving C-to-U editing in vivo.

Through AAV-mediated delivery, researchers significantly reduced cholesterol levels by targeting Pcsk9 mRNA in the mouse liver and repaired Mef2c mutations, improving social behavior in an autism model. This work provides a novel precision editing tool for treating genetic diseases at the RNA level.

Nat Biomed Eng | A Modified LNP-mRNA Complex for Enhanced Gene Editing in the Heart

On November 3, 2025, Professor Kevin E. Healy's team from the University of California, Berkeley, published a research paper titled "A microphysiological system for screening lipid nanoparticle–mRNA complexes predicts in vivo heart transfection efficacy" in the journal Nature Biomedical Engineering. This study utilized a human-derived three-dimensional cardiac microphysiological system to screen for acid-sensitive degradable PEG lipid-modified LNPs, which significantly improved mRNA delivery and gene editing efficiency in the heart. The optimized LNPs exhibited a higher proportion of cardiac transfection and lower liver and spleen uptake in vivo without affecting cardiac function.

This study provides a predictable in vitro screening platform for cardiac mRNA therapy and lays the foundation for the application of non-viral delivery systems in heart diseases.

NEJM丨CAR-T Therapy Resolves Multidrug-Resistant Ulcerative Colitis Dilemma

Recently, Fabian Müller and colleagues from the University of Erlangen-Nuremberg in Germany published an article titled "CD19 CAR T-Cell Therapy in Multidrug Resistant Ulcerative Colitis" in the NEJM. Starting from the significant infiltration of B cells and plasma cells in UC mucosal inflammation, and targeting the gap in existing therapies that do not address the activated B-cell system, they treated a patient with multidrug-resistant UC using CD19 CAR-T cell therapy, achieving remarkable efficacy.

Capital Express

A Gene Therapy Company in China Completes Series C Financing

Recently, Shanghai Guotou XianDao Private Equity Fund Management Co., Ltd. (hereinafter referred to as "Guotou XianDao") announced that, with the support of the Shanghai Science and Technology Commission and the Municipal Financial Office, Guotou XianDao, as the lead investor, joined forces with renowned investment institutions such as Loyal Valley Capital to complete the Series C financing round for Shanghai Tianze Yuntai Biopharmaceutical Co., Ltd. (hereinafter referred to as "Tianze Yuntai" or "the Company").

A Chinese Cell Therapy Company Completes Over 100 Million Yuan in Angel Financing

Recently, Vito Biotech (Shanghai) Co., Ltd. ("Vito Biotech" or "the Company"), a company focused on in vivo cell therapy, announced the completion of an angel round of financing exceeding RMB 100 million. The financing was led by Qiming Venture Partners, with participation from B Capital, Apricot Capital, and Shunxi Fund. The proceeds will mainly be used to advance the iterative upgrade of its core technology platform and accelerate the research and development process of its first candidate product for autoimmune disease treatment.

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