NEW YORK – Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced results of a well-controlled non-human primate study, which demonstrate that route of adeno-associated virus (AAV) administration plays a critical role in optimizing gene therapy delivery to key...
Latest News
SAN MATEO, Calif. — NeurogesX, Inc. (Nasdaq: NGSX), a biopharmaceutical company focused on developing and commercializing novel pain management therapies, announced today that the Office of Orphan Product Development (OOPD) of the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Qutenza(TM) (formerly NGX-4010), a high concentration...
Indianapolis, Ind. – Alois Alzheimer recognized more than a century ago the role of neuroinflammation in the development of the disease that would bear his name. “If you actually go back to his original drawings, he was drawing the cells, which today we know are microglia,” immune cells of the...
SAN FRANCISCO, Calif. — Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, today announced the successful completion of a $120 million financing co-led by Viking Global Investors and Cormorant Asset Management with participation from new and existing investors, including The Column...
Meaningful efficacy observed in slow-progressor subgroup; NfL biomarker improvements support potential for accelerated regulatory pathway SEOUL, South Korea — Neuronata-R®, an autologous bone marrow-derived mesenchymal stem cell (MSC) therapy for amyotrophic lateral sclerosis (ALS), has demonstrated meaningful efficacy signals in a subgroup of participants with slow disease progression in its recently...
The FDA designation marks a significant milestone in the development of a novel treatment for a rare neurodevelopmental disorder Plan to initiate first-in-human U.S. clinical trials of lead drug candidate for treating ASD in 2026 BOSTON, Mass. — NeuroNOS, a biopharmaceutical company focused on developing treatments for autism and Alzheimer disorders, and a subsidiary of Beyond Air (NASDAQ: XAIR), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation...
WUHAN, China and SAN DIEGO, Calif. — Neurophth Therapeutics, Inc. announced today that the last patient has been enrolled in Phase I/II clinical trial of Opvika® (Esonadogene Imvoparvovec) for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON). Neurophth is conducting a Phase I/II, single-arm, multi-center study aimed...
Jerusalem, Israel – In a compelling Genomic Press Interview published in Brain Medicine on September 25, 2024, Professor Hermona Soreq of the Hebrew University of Jerusalem in Israel unveils the profound implications of her groundbreaking research on the cholinergic system and small RNA regulators in brain-body communication. Prof. Soreq, holder...
HERZLIYA, Israel – NeuroSense Therapeutics, a biotechnology company developing treatments for neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS), based on a novel combination drug therapy aiming at slowing or halting the disease progression, announces the successful completion of its first Phase IIa study. The study, NST002, aimed at establishing various safety...
CAMBRIDGE, Mass. — NeuroSense Therapeutics, Ltd. (NASDAQ: NRSN), a leading clinical-stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, today announced that two members of its Scientific Advisory Board will present new data from the Company’s Phase 2b trial during the General Neurology and Late Breaker sessions at the 77th Annual American...