CHARLESTON, S.C. – Neuroene Therapeutics announced that the FDA has granted orphan drug designation for the company’s proprietary compound: NT102. NT102 was developed as part of a novel patented platform of mitochondrial and neuroprotective chemicals for the treatment of neurological diseases such as pharmacoresistant epilepsies. In preclinical studies funded by...
treatment News
NEW YORK – Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) for patients...
NEW YORK, NY — Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome, which showed that NGN-401 was...
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...
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...
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...
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...
New Rochelle, NY – Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy. They also showed that after single dose inhaled delivery of SP-101, SP-101 vector genomes were detected throughout the respiratory tract...
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...