On March 21, 2024 NeuExcell Therapeutics, a leading biotechnology company focused on in vivo neural regenerative therapies, reported the successful dosing of the first patient with the first-in-class NeuroD1 gene therapy product NXL-004 (Press release, NeuExcell Therapeutics, MAR 21, 2024, View Source [SID1234641362]). This milestone represents significant progress in the development of new treatment for malignant glioma patients.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Led by Professor Yulun Huang and his team at the Fourth Affiliated Hospital of Soochow University in Suzhou, China, the investigator-initiated trial (IIT) will evaluate the safety and tolerability of NeuExcell’s AAV-NeuroD1 gene therapy product NXL-004 in patients with glioblastoma. This clinical study marks the first clinical use of AAV-NeuroD1 gene therapy to treat patients worldwide.
Glioblastoma is a highly aggressive form of brain cancer with limited treatment options and poor prognosis. NXL-004, based on NeuExcell’s revolutionary in vivo trans-differentiation technology platform, aims to inhibit tumor cell proliferation and promote their transformation into neural cells through the overexpression of the NeuroD1 transcription factor. The advantage of this glioma cell trans-differentiation approach is minimal side-effect on other brain cells, which is distinct from radiation or chemotherapy that has collateral damage on healthy cells. Promising preclinical results from animal model studies have paved the way for this groundbreaking clinical trial, offering hope for many patients suffering from debilitating brain disorders such as malignant glioma.
"We are very excited about completing the dosing of the first patient using NeuroD1 gene therapy product NXL-004 in this exploratory clinical study," said Dr. Yulun Huang, the study’s lead investigator. "This milestone underscores the dedication of our team to try innovative approaches in addressing unmet medical needs such as glioblastoma. We are committed to closely monitor the patient and evaluate the safety and efficacy of NXL-004 in this trial."
Professor Gong Chen, the inventor of in vivo neural regenerative gene therapy and the founder of NeuExcell Therapeutics, expressed his gratitude: "We are grateful to Professor Yulun Huang and his team for their outstanding work. This first-in-human trial sets an important milestone for our first-in-class NeuroD1 gene therapy that has the potential to treat millions of patients suffering from severe neurological disorders. We are dedicated to developing game-changing treatments for neurodegenerative diseases using in vivo neural regenerative therapies."