Weekly Highlights | Gene Therapy News Digest Issue 176

Dec 01, 2025 07:30 CST Updated 07:30

Zhongmou Therapeutics

Gene Therapy Product Developer

Juventas

Innovative Cell Therapy Drug Developer

BRL Medicine

Cell and Gene Therapy Drug Developer

Industry News FlashView

Focus

Congratulations on Zhongmou Therapeutics' ZM-02 Optogenetic Gene Therapy Project receiving U.S. IND approval in just 20 days!

On November 28, 2025, Zhongmou Therapeutics (referred to as "Zhongmou"), a global clinical-stage gene and optogenetics therapy company, announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for its innovative optogenetic gene therapy product, ZM-02. As Zhongmou's partner, PackGene Biotech extends its warmest congratulations!

FDA Approves Novartis' Innovative Gene Therapy

On November 24, 2025, the U.S. FDA approved the AAV gene therapy Itvisma (onasemnogene abeparvovec-brve) for the treatment of children, adolescents, and adults aged 2 years and older with spinal muscular atrophy (SMA) who have been confirmed to carry SMN1 gene mutations. According to the press release, this approval makes Itvisma the first and only gene replacement therapy widely available for the treatment of SMA patients.

Recommended Reading：Just Now! FDA Approves Novartis Innovative Gene Therapy

Niraleucel Injection (Yuanruida®) Approved for Lymphoma, China's Indigenous CAR-T Therapy Enters a New Era with Dual Indications

On November 28, 2025, Juventas announced that its self-developed CAR-T cell therapy product, Nalkylcel Injection (brand name: Yuanruida®), has been approved for marketing by the China National Medical Products Administration (NMPA) for the treatment of relapsed or refractory large B-cell lymphoma (r/r LBCL) after two or more lines of systemic therapy. This marks the second marketing approval in China for Nalkylcel Injection, following its initial approval in November 2023 for treating adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). It represents the first CD19 CAR-T product in China with full independent intellectual property rights and is currently the only CAR-T cell therapy product in the country covering two major hematological malignancy indications—leukemia and lymphoma.

Gene Therapy Lights Up Hope for Life! BRL Medicine's BRL-101 Once Again Helps Thalassemia Patients Gain a New Lease on Life

On November 27, 2025, BRL Medicine Inc. (hereinafter referred to as "BRL Medicine"), a company focused on gene and cell therapy, announced another successful case of a patient achieving "poverty alleviation." This successful treatment is not only a miracle for one family but also reaffirms the safety and efficacy of BRL-101 gene therapy developed by BRL Medicine, offering a safer treatment option for thalassemia patients worldwide.

Tianjin FTZ takes the lead in introducing standardized guidelines for the classification of gene and cell therapies

On November 24, Tianjin Free Trade Zone released the "Classification and Grading Standards for Clinical Research and Clinical Translation of Gene and Cell Therapy New Technologies (Trial)", providing institutional support for the entire chain transformation in this field from research and development to clinical application. Based on international common rules, the specification scientifically categorizes gene and cell therapy products and establishes a differentiated regulatory system of "risk grading and access classification".

Innovation Breakthrough

Fudan University Develops a Stronger Fanzor Gene Editing System, Single AAV Delivery Enables Efficient In Vivo Gene Editing

The research team led by Zhou Xingtao, Huang Jinhai, and Zhao Guoli from the Eye, Ear, Nose, and Throat Hospital of Fudan University has developed an engineered ultra-compact Fanzor-ωRNA system with enhanced genome editing activity. The editing efficiency has been improved 6 to 129 times, making it the most active RNA-guided eukaryotic endonuclease in the current Fanzor1 family. It can also be developed into a base editor. More importantly, this system is compact and can be delivered in vivo via a single AAV, achieving robust in vivo genome editing. It holds promise for applications in basic research and disease treatment.

Novel CAR-T Cell Therapy Reverses and Prevents Aging-Related Intestinal Issues

Researchers at Cold Spring Harbor Laboratory have developed a CAR-T cell therapy targeting uPAR, and confirmed that it can reverse and prevent aging-related intestinal regeneration and health defects.

World's First! Positive Progress in Ex Vivo Gene Therapy for Hunter Syndrome!

Recently, the University of Manchester, UK, announced that a 3-year-old patient with Mucopolysaccharidosis Type II (MPS II) has shown comprehensive recovery in both physical and cognitive functions months after receiving a novel autologous hematopoietic stem cell gene therapy (AVR-RD-05) in a Phase I/II clinical trial. This positive development not only brings new hope to patients with MPS II but also sets a new benchmark for the application of hematopoietic stem cell-based gene therapies in neurometabolic disorders.

Capital Express

Nearly 100 Million in Financing Secured! Vico Biotech Boosts CGT Globalization, Strengthens "Safety Line" for Innovative Therapies

On November 27, 2025, Shanghai Vico Biotechnology Co., Ltd. (hereinafter referred to as "Vico Bio") announced the completion of a nearly RMB 100 million Series A financing round. This round was led by Shenzhen Capital Group, with Wuxi Capital following on. Fenghe Capital served as the exclusive financial advisor. The proceeds from this financing will help Vico Bio accelerate the establishment of a U.S.-based safety evaluation testing laboratory for cell and gene therapy (CGT), CGT safety evaluation services, CGT clinical services, and the overseas expansion of biobreeding services.

Total investment exceeds 2 billion yuan, a cell therapy industrial base in China to be commissioned soon

The Cell Therapy Incubation and Acceleration Park (formerly known as "CDT Biotechnology Industrial Park") located in the Jinniu High-tech Industrial Development Zone of Chengdu City has successfully passed the preliminary completion acceptance by the five responsible parties. It is reported that this major project, with a total investment of over 2 billion yuan, will be put into use in early 2026.

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