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...
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...
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...
TORONTO, Canada – A new round of clinical trials supported by the Ontario Institute for Cancer Research (OICR) will harness a unique opportunity to test some of the newest treatment strategies for a range of different cancers. OICR has announced two new clinical trials as part of its Window-of-Opportunity (WOO)...
SOUTH SAN FRANCISCO, Calif. – Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new exploratory 2-year longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi™ (risdiplam) in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular...
DUBLIN – Horizon Therapeutics plc (Nasdaq: HZNP) today announced the publication of a new analysis of the pivotal Phase 2/3 N-MOmentum trial for UPLIZNA (inebilizumab-cdon), assessing the potential for reduced risk of worsening disability in those living with NMOSD. These data are published in the May issue of Neurology Neuroimmunology...
DUBLIN – Horizon Therapeutics plc (Nasdaq: HZNP) today announced that a new post hoc analysis of the UPLIZNA N-MOmentum Phase 2/3 pivotal trial has been published in Multiple Sclerosis and Related Disorders showing that prior rituximab exposure did not impact the efficacy of UPLIZNA, and that UPLIZNA demonstrated comparable efficacy to trial participants...