gene therapy groundbreaking

On January 22, 2024, the Dushu Lake Hospital affiliated with Soochow University and NeuExcell Therapeutics commenced an exploratory clinical study to evaluate the safety, tolerability, and preliminary effectiveness of NXL-004 injections in treating patients with malignant glioma.

The gene therapy drug NXL-004, central to the study, was developed by NeuExcell Therapeutics. Professor Huang Yulun of Dushu Lake Hospital, an expert in neurosurgery, is the principal investigator. The project also received congratulations from Patheon Biologics, a partner of NeuExcell Therapeutics, for its initiation.

The launch event was attended by Professor Huang Yulun and his team, NeuExcell Therapeutics co-founders Professor Chen Gong and Ms. Xu Jie, CEO Dr. Sheng Jian, and their R&D team, along with representatives from contract research organizations.

Professor Huang Yulun acknowledged the collaboration with NeuExcell Therapeutics, noting that NXL-004 has demonstrated promising efficacy and safety in preclinical studies. Professor Chen Gong expressed appreciation for the collaborative efforts and high hopes for the drug, developed using neural transcription factors, targeting malignant gliomas.

The meeting covered the development background, product details, and research methodology, emphasizing adherence to Good Clinical Practice (GCP) and relevant legal regulations to protect participant rights and privacy, aiming for quality research results.

Dr. Sheng Jian, CEO of NeuExcell Therapeutics, expressed gratitude for the support from Dushu Lake Hospital and anticipated the prompt commencement of patient enrollment and the potential positive impact on patients, hopeful for the clinical trial’s success.

The article also highlights the high mortality rate of malignant gliomas, particularly glioblastomas (GBM), and the current limitations in their treatment, which includes surgery, radiation therapy, and chemotherapy. It mentions the inevitability of recurrence in these patients and the need for better treatment options.

NXL-004, based on Professor Chen Gong’s team’s innovative cell transdifferentiation technology, is presented as an innovative gene therapy drug for gliomas. It received Orphan Drug Designation from the FDA in December 2023 for treating malignant brain gliomas, the first such designation for an AAV gene therapy product in this category.

The initiation of this study signifies the entry of NXL-004 into clinical development, offering potential new treatment options for patients with malignant gliomas.

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PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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