Neurogene Inc. is a clinical-stage biotechnology company. The Company, through its proprietary transgene regulation technology, Expression Attenuation via Construct Tuning (EXACT), is building a product portfolio of genetic medicines for rare neurological diseases with high unmet needs not otherwise addressable by conventional gene therapy. Its clinical-stage program, NGN-401, utilize the EXACT platform, which is in development for the treatment of Rett syndrome, a disease with a patient population that has a significant unmet need, and that ultimately progresses to substantial neurological and physical impairment and premature death. NGN-401 is being evaluated in female patients with Rett syndrome in a Phase 1/2 open-label, multi-center clinical trial that assesses the safety, tolerability, and efficacy of two doses of NGN-401 delivered using a one-time intracerebral ventricular (ICV) procedure. It is also developing NGN-101 for the treatment of CLN5 Batten disease.
High Profit Growth
The company's net income leads the industry, with the latest annual income totaling USD 0.00.
Undervalued
The company’s latest PE is -7.78, at a low 3-year percentile range.
Institutional Selling
The latest institutional holdings are 17.70M shares, decreasing 5.56% quarter-over-quarter.
Held by Bill Nygren | OAKMX
Star Investor Bill Nygren | OAKMX holds 1.31K shares of this stock.
Higher Market Activity
The company has more investor interest, with a 20-day turnover ratio of 0.02.