Neurogene focused on advancing Phase 1/2 trial for NGN-401, a differentiated clinical stage gene therapy to treat Rett syndrome using its EXACT technology; interim clinical data expected in 4Q24
Two patients successfully dosed with NGN-401, which has been well tolerated to date with no treatment-emergent or procedure-related serious adverse events, or transgene-related overexpression toxicity
Post transaction cash, cash equivalents, and investments of approximately $200 million expected to fund advancement of Neurogene’s EXACT gene therapy portfolio into 2H26
Shares to trade on NASDAQ under the new ticker “NGNE”
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