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Neurogene Announces Expansion and Plans for More Rapid Patient Enrollment of Rett Syndrome Gene Therapy Clinical Trial
Ongoing Phase 1/2 clinical trial for NGN-401 to include additional patients in Cohort 1 and a dose-escalation cohort Both previously planned updates are
About this update from Neurogene Inc.
[{"type":"text","content":"\nOngoing Phase 1/2 clinical trial for NGN-401 to include additional patients in Cohort 1 and a dose-escalation cohort\n\n\nBoth previously planned updates are expected to provide a more robust dataset to inform future registrational trial design\n\n\nRemoval of staggered dosing in Cohort 1 expected to enable the anticipated completion of Cohort 1 dosing in the second half of 2024\n\n\nThird patient dosed in Cohort 1\n\n\n NEW YORK--(BUSINESS WIRE)--\nNeurogene Inc. (NASDAQ: NGNE) (“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.\n\n\n“We are excited to share that we have met our first 2024 program milestones, including dosing the third patient in the NGN-401 Phase 1/2 trial for Rett syndrome and expansion of the trial to include more patients in the current dosing cohort and the addition of a high dose cohort,” said Founder and Chief Executive Officer, Rachel McMinn, Ph.D. “Our clinical development strategy has been to build flexibility and optionality early in the program with two concurrent dose cohorts designed to generate a more complete data package, which we expect will inform future registration discussions with global health authorities. We expect that expansion of the clinical trial and the removal of staggered dosing in Cohort 1 will enable us to treat more patients in a shorter period of time. Based on this update, we expect to complete enrollment of Cohort 1 in the second half of 2024.”\n\n\nRett Syndrome Program Update\n\n\nThe U.S. Phase 1/2 clinical protocol for NGN-401 has been amended as follows:\n\n\n\nCohort 1, which specifies a total dose of 1×1015 total vector genomes delivered via intracerebroventricular (ICV) administration, was expanded from five patients to eight patients. The dosing stagger has been removed from Cohort 1, enabling the remaining patients to be dosed in parallel.\n\n\n\nCohort 2, which specifies a total dose of 3×1015 total vector genomes delivered via ICV administration, was added and is expected to include a total of eight patients.\n\n\n\nThe first three patients in C...