As previously reported, Craig-Hallum analyst Albert Lowe initiated coverage of Neurogene (NGNE) with a Buy rating and $50 price target. The firm notes the company is developing NGN-401, an AAV gene therapy for Rett syndrome, a rare and devastating neurodevelopmental disease that impacts girls beginning in infancy and is characterized by developmental regression and symptoms that require lifelong care. Craig-Hallum believes Neurogene is undervalued and that NGN-401 has an elegant design that gives it the upper hand over Taysha’s (TSHA) competing program TSHA-102. Looking ahead, the firm thinks NGN-401 is well-positioned to show efficacy advantages over TSHA-102 in the second half of 2025 clinical data update and be the leading gene therapy for Rett. This represents a significant commercial opportunity with pricing of over $2M in a U.S. population of about 6k patients, and Albert models WW NGN-401 sales reaching $1B in 2033, including U.S. sales of about $700M.
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