NXL-001, developed using this innovative platform, has demonstrated remarkable progress in treating stroke, offering new hope for patients with this debilitating condition. The therapy works by overexpressing the NeuroD1 neurogenic transcription factor in glial cells, promoting their transdifferentiation into neurons. This approach has shown promise in treating various neurological injuries and degenerative diseases, presenting a new frontier in central nervous system disorder treatment.
NeuExcell Therapeutics’ collaboration with Spark Therapeutics, a subsidiary of Roche, has also played a crucial role in advancing gene therapy. The collaboration, initiated in 2021 and valued at over $190 million, focuses on developing gene therapy for Huntington’s disease using the in situ neural regeneration technology platform.
At the ASGCT meeting, NeuExcell Therapeutics presented the latest progress of NXL-001 in treating a monkey model of stroke, highlighting the therapy’s potential to drive functional recovery in severe stroke cases. The data presented showed significant improvements in motor function, sparking excitement among experts and paving the way for future clinical trials.
Dr. Shen Jian, speaking about the potential of gene therapy, stated, “The rapid development of cell and gene therapy technology has overcome the medical challenge of neuronal regeneration. This opens up possibilities for fundamentally treating diseases related to neuronal death, such as stroke, Huntington’s disease, ALS, Alzheimer’s disease, Parkinson’s disease, and other neurodegenerative diseases.”
NeuExcell Therapeutics’ NXL-001 gene therapy for stroke is poised to enter clinical trials soon, offering a ray of hope for patients and families affected by this condition. The company plans to submit new drug clinical trial applications (IND) in the United States and China, followed by Phase 1/2 clinical studies, marking a significant milestone in the field of gene therapy for neurological disorders.
https://mp.weixin.qq.com/s?__biz=MzA3MTYzMzMxMg==&mid=2454536336&idx=1&sn=a73404491a03f900885d3dffaaa7ceb1
Check out our AAV CDMO service to expedite your gene therapy research
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
[2024/12/06] Gene and Cell Therapy- weekly digest from PackGene
FeaturedNewsArticlesPackGene's NewsletterReceive the latest news and insights to your inbox.About PackGenePackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span...
Prime Editing: Precision Gene Therapy on the Horizon
The world of gene editing is evolving rapidly, and prime editing is emerging as a standout innovation. Known for its precision and versatility, this technique has taken center stage in therapeutic research, promising transformative advancements for genetic diseases. A...
Rgenta Therapeutics and GSK Forge Strategic Alliance to Advance RNA-Targeted Small Molecule Medicines
Woburn, Mass. – December 4, 2024 – Rgenta Therapeutics, a clinical-stage biotechnology company specializing in RNA-targeted small molecule therapies, has announced a multi-year strategic research alliance with pharmaceutical giant GSK. The partnership will focus on...
RNA-targeting molecule from Arrakis allows muscles to relax in mice with myotonic dystrophy
"Dune"-inspired Arrakis Therapeutics has tested its spice—an RNA-targeting small molecule—in a mouse model of myotonic dystrophy type 1 (DM1), with the drug reversing the prolonged muscle contraction that is characteristic of the disease. Arrakis, based not on a...
Related Services