William Blair initiated research coverage of Neurogene Inc. (NGNE $45.06), a genetic medicine company focused on the development of AAV-based therapies for the treatment of rare neurological diseases.

“Neurogene is a clinical-stage biotechnology company that was founded in 2018 with the purpose of addressing complex and devasting neurological diseases with high unmet need,” said analyst Sami Corwin, Ph.D. “We hold the view that Neurogene’s lead candidate, NGN-401, could be a first-in-class and best-in-class gene therapy for Rett syndrome, which is a rare genetic neurological disorder that leads to severe neurological and physical impairment and premature death and for which there are no disease-modifying treatments available. NGN-401 is equipped with a full-length human MECP2 transgene and Neurogene’s EXACT technology, enabling regulated and more consistent gene expression between cells, while minimizing gene overexpression and related toxicities. NGN-401 is being evaluated in a Phase I/II trial at clinically meaningful doses and with a direct administration method, which we believe will lead to greater biodistribution throughout the brain and better clinical outcomes than with other approaches. Initial data from the Phase I/II trial of NGN-401 is expected in fourth quarter 2024 and will be a key catalyst for the stock, in our view.

“The company is also developing NGN-101, an AAV9 gene therapy designed to deliver the gene encoding CLN5 to halt or reduce the progression of CLN5 Batten disease–related neurodegeneration and vision loss,” added Dr. Corwin. “NGN-101 is being investigated in a Phase I/II, open-label, non-randomized, dose-escalation study, with NGN-101 administered through a combination of ICV and intravitreal injections to address the neurodegeneration and vision loss aspects of the disease, respectively. The company is enrolling patients in the third dose escalation cohort, and initial data from the trial are expected to be presented in the second half of 2024.”

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