Recently, the first patient dosing of NXL-004, an AAV-NeuroD1 gene therapy product developed by NeuExcell Therapeutics, was successfully completed at the Fourth Affiliated Hospital of Soochow University (Suzhou Dushu Lake Hospital). The procedure was carried out by Professor Huang Yulun’s team, comprising experts in neurosurgery. This achievement marks a significant milestone in the advancement of gene therapy for neurological disorders.

NXL-004 is an innovative treatment approach developed based on Professor Chen Gong’s pioneering in situ differentiation technology platform. It is notably the first AAV gene therapy product for malignant glioblastoma to receive FDA orphan drug designation. The therapy targets glioblastoma cells by overexpressing the neurogenic transcription factor NeuroD1, inhibiting tumor cell proliferation while promoting their differentiation into neurons. This mechanism offers potential advantages over conventional treatments such as radiotherapy and chemotherapy, with minimal side effects on surrounding tissues.

The initial patient, suffering from recurrent glioblastoma, underwent surgery and dosing without complications. Postoperative observations revealed a stable condition with no significant adverse reactions, culminating in a smooth recovery and discharge within a week. This successful administration of AAV-NeuroD1 gene therapy represents a groundbreaking leap from preclinical research to clinical application, offering hope for improved treatment outcomes in glioblastoma patients.

Professor Huang Yulun expressed optimism regarding the therapy’s safety and efficacy, citing promising preclinical data and the favorable safety profile observed in the first clinical trial subject. Likewise, Professor Chen Gong, the founder of NeuExcell Therapeutics, commended Professor Huang Yulun’s team for their collaboration and surgical expertise, underscoring the significance of this milestone in advancing neurological disease treatments.

The successful dosing of NXL-004 at Suzhou Dushu Lake Hospital underscores the potential of gene therapy in revolutionizing the management of malignant glioblastoma, addressing the urgent need for more effective treatment options in neuro-oncology. This achievement serves as a testament to the collaborative efforts of researchers and medical professionals in pushing the boundaries of medical science for the benefit of patients worldwide.

Check out our AAV CDMO service to expedite your gene therapy research
About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Scientists develop potential stealth cancer therapy

Credit: ACS Central Science (2024). DOI: 10.1021/acscentsci.4c00559Sneaking by cancer's defenses, by disguising tumor-fighting antibodies inside the molecules cancer uses to nourish tumor growth, is the basis of a novel therapy from Yale Cancer Center researchers at...

read more

Related Services

Plasmids GMP Services

Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.

AAV GMP Services

Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
aav icon

Technology Platforms

PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.