Articles from Neurogene Inc.

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 third quarter 2024 financial results and highlighted recent corporate updates.

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 an update on its ongoing Phase 1/2 open-label clinical trial evaluating NGN-401 gene therapy for the treatment of Rett syndrome.

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 interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase 1/2 open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. Low-dose NGN-401 has demonstrated a favorable safety profile.

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 it has entered into a securities purchase agreement for a private investment in public equity (“PIPE”) financing that is expected to result in gross proceeds of approximately $200 million to Neurogene, before placement agent fees and offering expenses. The oversubscribed PIPE financing included participation from a U.S.-based healthcare focused investor, RTW Investments, Casdin Capital, EcoR1 Capital, Redmile Group, Great Point Partners, LLC, Commodore Capital and Samsara BioCapital.

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 second quarter 2024 financial results and highlighted recent corporate updates.

Neurogene Inc. (NASDAQNGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that NGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The designation was based on preliminary clinical evidence from the ongoing Phase 1/2 clinical trial with NGN-401 that shows potential to address unmet medical needs in this disease.

Neurogene Inc. (NASDAQNGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the BMO 2024 Genetic Medicines Summit.

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 Neurogene will be added to the Russell 3000® Index, effective at the open of U.S. equity markets today, Monday, July 1, 2024.

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 patient in Cohort 2 received high-dose NGN-401 gene therapy in the Phase 1/2 trial for female pediatric patients with Rett syndrome, and high-dose NGN-401 has been well-tolerated following dosing in May 2024. The Company also provided an update on interim safety data on the first three patients in the low-dose cohort during the IRSF ASCEND 2024 Rett Syndrome National Summit, which shows that NGN-401 has continued to have a favorable safety profile with no new treatment-related adverse events (AEs).

Neurogene Inc. (NASDAQNGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the following conferences:

Neurogene Inc. (NASDAQNGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that its NGN-401 gene therapy for Rett syndrome has been selected to participate in the U.S. Food and Drug Administration (FDA) Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. As part of the START Program, Neurogene will have opportunities for enhanced communications with the FDA, with the aim to further accelerate the pace of NGN-401’s development. These opportunities are designed to provide frequent advice and regular ad-hoc conversations to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population.

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 first quarter 2024 financial results and highlighted recent corporate updates.

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 generally well-tolerated by all three patients dosed, with follow-up of approximately nine, six and three months post-dosing. These data were presented during the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

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 initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome will be presented at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting. The data show that the NGN-401 gene therapy candidate has been generally well-tolerated, and there have been no treatment-emergent or procedure-related serious adverse events or signs of MeCP2 overexpression-related toxicity observed in any patient, including a patient with a mild variant predicted to result in residual MeCP2 function.

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 fourth quarter and full year 2023 financial results and highlighted recent corporate updates.

Neurogene Inc. (NASDAQNGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the expansion of its ongoing Phase 1/2 gene therapy clinical trial for NGN-401 for female pediatric patients with Rett syndrome and updates to enable more rapid enrollment in the trial.

Neurogene Inc. (NASDAQNGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that Company management will participate in the following investor conferences:

Neurogene Inc. (NASDAQNGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the appointment of Julie Jordan, M.D., as Chief Medical Officer. Dr. Jordan brings to Neurogene over 20 years of industry and clinical expertise involving the design and execution of global clinical trials across multiple development areas, including gene therapy and central nervous system disorders. She most recently served as Chief Medical Officer of Homology Medicines, a clinical-stage company advancing gene therapies for the treatment of rare diseases.

Neurogene Inc. (NASDAQNGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced today a business update and provided its outlook for 2024. Key milestones for the Company’s clinical-stage gene therapy product candidates remain on track for 2024, while the Company continues to advance its discovery-stage transgene regulation portfolio.

Neurogene Inc. (NASDAQNGNE) (“Neurogene”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the closing of its merger with Neoleukin Therapeutics, Inc. (“Neoleukin”). Neurogene shares are expected to begin trading on the NASDAQ Global Market under the ticker “NGNE” beginning today at the market open.

Neurogene Inc., a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced today the dosing of the first two female pediatric patients with Rett syndrome in its ongoing Phase 1/2 trial of NGN-401. Rett syndrome is a debilitating, X-linked, neurodevelopmental disorder with significant unmet medical need. To date, NGN-401 has been well-tolerated with no treatment-emergent or procedure-related SAEs, and no signs of transgene-related overexpression toxicity.

Neurogene Inc., 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 cleared the Company’s Investigational New Drug (IND) application for NGN-401 for the treatment of Rett syndrome.

Neurogene Inc., a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, announced the addition of NGN-401 for Rett syndrome to the Company’s development pipeline. NGN-401 is Neurogene’s first product candidate using the Expression Attenuation via Construct Tuning (EXACT) gene regulation platform technology, and has shown in preclinical models to deliver efficacious levels of MeCP2, while avoiding toxicity observed with conventional MECP2 gene therapy. EXACT is a self-regulating gene therapy platform technology that can be tuned to deliver a desired level of transgene expression within a narrow range, and is compatible with viral and non-viral delivery platforms.

Neurogene Inc., a 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 cleared the Company’s Investigational New Drug (IND) application for NGN-101. NGN-101, Neurogene’s lead product candidate in its Batten disease franchise, is an investigational adeno-associated virus (AAV) gene therapy for the treatment of CLN5 Batten disease, a rare and fatal neurodegenerative disorder with no approved disease-modifying therapies available.

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 diagnosed with CLN5, a subtype of Batten disease.

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 regions of the central nervous system. These results underscore that the route of delivery is an essential factor in developing effective treatments for genetic neurological diseases. Data were presented virtually today at the 24th annual meeting of American Society of Gene & Cell Therapy.