NeuExCell Therapeutics, Inc.

SUZHOU, China, March 21, 2024 /PRNewswire/ -- NeuExcell Therapeutics, a leading biotechnology company focused on in vivo neural regenerative therapies, announces the successful dosing of the first patient with the first-in-class NeuroD1 gene therapy product NXL-004. This milestone represents significant progress in the development of new treatment for malignant glioma patients. 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." About NeuExcell Therapeutics: NeuExcell Therapeutics is a leading biotechnology company focused on developing innovative neural regenerative therapies to address unmet medical needs in the field of neurodegenerative diseases and cancers. NeuExcell is committed to revolutionizing the field of CNS drug development and improving outcomes for patients worldwide.

PHILADELPHIA, Dec. 18, 2023 /PRNewswire/ -- On December 7, 2023, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation ("ODD") to NXL-004, an investigational AAV gene therapy product being developed by NeuExcell Therapeutics Inc. ("NeuExcell"), for the treatment of malignant glioma. This is the world's first AAV gene therapy product for malignant glioma to receive FDA ODD designation. Malignant gliomas are the most common primary malignant brain tumors in adults, about 60% of which are glioblastoma (GBM). Characterized by high malignancy and mortality, the current standard-of-care of GBM, encompassing surgery, radiotherapy, and chemotherapy, results in a median overall survival of merely 15-18 months and a 5-year survival rate below 10%. Recurrence is virtually inevitable for malignant glioma and there is no clear standard salvage therapy. NXL-004 is an innovative treatment based on the groundbreaking astrocyte-to-neuron ("AtN") conversion platform developed by Professor Gong Chen. It has shown good efficacy and safety in preclinical studies and will enter first-in-human clinical trials in early 2024. According to the U.S. Orphan Drug Act, drugs that have received ODD in the U.S. can enjoy a series of regulatory support, including but not limited to tax credits for clinical trial expenses, exemption of new drug application fees, and 7-year market exclusivity after product approval. ODD can help accelerate development of NLX-004 and provide new hope for many GBM patients around the world. NeuExcell is committed to restore health and improve the quality of life for millions of patients worldwide suffering from neurodegenerative diseases and neurological injuries. NeuExcell's disruptive AtN technology has the potential to regenerate neuronal tissues and restore neurological functions, with robust preclinical efficacy data in a wide variety of disease models including non-human primates. NeuExcell's lead clinical programs include Alzheimer's disease, ischemic stroke and GBM, all expected to enter the clinic in 2024, with pipeline expansion opportunities in numerous indications such as Huntington's disease and Amyotrophic Lateral Sclerosis. SOURCE NeuExcell Therapeutics

KING OF PRUSSIA, Pa., Sept. 13, 2022 /PRNewswire/ -- NeuExcell Therapeutics, Inc., a preclinical gene therapy company focusing on improving the lives of patients suffering from neurodegenerative diseases, today announced that Ronald Lorijn, MD, MBA, Chief Executive Officer of NeuExcell Therapeutics, will participate in the upcoming virtual UBS Biotechnology Private Company Symposium. Dr. Lorijn will provide a corporate overview followed by a moderated Q&A at the upcoming Symposium on September 21-22, 2022. UBS Virtual Biotechnology Private Company Symposium Continue Reading NeuExcell Therapeutics has been invited to participate in the upcoming UBS Biotechnology Private Company Symposium. Tweet this Date: Wednesday September 21, 2022 Time: 12:30 p.m. EST About NeuExcell Therapeutics NeuExcell is a privately held early-stage gene technology company headquartered in King of Prussia, Pennsylvania. Its mission is to improve the lives of patients suffering from neurodegenerative diseases and CNS injuries. Based upon the scientific work of Prof. Gong Chen, the Company has developed a potentially disruptive neural repair technology through astrocyte-to-neuron conversion in-vivo by introducing neural transcription factor(s) through adeno-associated virus (AAV)-based gene therapy. NeuExcell's pipeline covers major neurodegenerative diseases such as Stroke, Huntington's Disease, Amyotrophic Lateral Sclerosis, Alzheimer's Disease, Parkinson's Disease, Traumatic Brain Injury, Spinal Cord Injury, and Glioma. For more information, visit . SOURCE NeuExcell Therapeutics