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 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to NGN-401, an investigational gene therapy in late-stage clinical...
treatment News
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 that the first participant was dosed in the Embolden™ registrational clinical trial of NGN-401, a gene therapy in development for the treatment...
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 provided an update on recent achievements for NGN-401 gene therapy for the treatment of Rett syndrome and plans for multiple value-creating catalysts anticipated...
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 details of Embolden™, the Company’s registrational clinical trial designed to evaluate NGN-401 gene therapy for the treatment of females with Rett syndrome...
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...
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 positive, updated interim clinical data in the pediatric cohort (ages 4-10 cohort) from its ongoing Phase 1/2 trial evaluating NGN-401 gene therapy...
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 the Company has completed discussions with the U.S. Food and Drug Administration (FDA) on its Embolden™ registrational trial protocol and plans to...
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...
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...