$2.50
$5.00
bear $2.50
base $3.50
bull $5.00
margin of safety $1.75
market price* $23.25
Neurogene is a clinical-stage gene therapy company. Its lead candidate, NGN-401, is a one-time treatment for Rett syndrome, currently in the Embolden registrational trial (20 girls and women, single-arm, dosing to finish in the second quarter of 2026). It received FDA Breakthrough Therapy designation in February 2026. A second program, for CLN5 Batten disease, is on hold after the FDA denied a streamlined-pathway designation. The company has no product revenue (about $0.9 million of licence-type revenue in 2024, none in 2025) and does not generate earnings: operating cash burn was about $70.6 million in 2024 and $77.2 million in 2025. After subtracting stock compensation (a real cost, $14.2 million in 2025), owner earnings were roughly minus $74 million in 2024 and minus $93 million in 2025, or about minus $4.20 and minus $4.35 per share. We measure only from 2024 because the December 2023 merger created the current business. How we valued it. Our approach credits only what is already secured or a proven, repeatable process. A drug that has not yet been approved, has no signed partner, and has no sales does not meet that bar, however encouraging the early data. Four of five patients with 12 months of follow-up met the trial's responder definition, and the trial succeeds at 7 of 20 responders, but that is a small open-label sample and the final readout, expected after dosing completes, has not happened. We therefore assign the drug candidates no value in any case. What remains is the cash. The company held about $269 million of cash and investments at the end of 2025 and says this funds operations into the first quarter of 2028. Cash spent on trials, manufacturing qualification and staff is what the company is choosing to use up, so each case estimates how much of it is left when the story reaches a decision point, rather than treating today's balance as fully available to shareholders. Owner earnings are after cash interest, and the company has no meaningful debt, so no debt adjustment applies. Roughly 23 million shares plus about 6.8 million pre-funded warrants (which behave like shares) are outstanding; we use about 23 million weighted shares for per-share figures, so if the warrants are counted separately the per-share numbers would be about 20 percent lower. Bear case, about $2.50 per share. The Embolden trial misses its 35 percent responder threshold or safety problems appear (the filing notes serious adverse events in one Phase 1/2 patient and the known class risks of AAV gene therapy). The company stops in 2027 with roughly $90 million of cash left, of which wind-down, leases and severance absorb part, leaving about $60 million. Base case, about $3.50 per share. Spending continues at the present pace of roughly $100 to $120 million a year through the readout in 2027, and about $80 million of cash is left at that point, with no value assigned to the therapy itself, and modest costs for wind-down or transition. Not credited: approval, launch, sales, the Breakthrough designation, and any priority review voucher, none of which is yet secured. Bull case, about $5.00 per share. Spending stays at the low end of the pace shown in 2025 to mid-2026 (about $50 million in the first half of 2026) and manufacturing at the company's own Houston facility, already at commercial scale per the filing, avoids extra build-out cost, leaving more cash at the readout. Even here we assign no value to the therapy itself. What would make this wrong. This valuation is a floor-style estimate of the cash that will be left; it deliberately leaves out the possibility that NGN-401 is approved. If the Rett trial succeeds and the therapy is approved and sold, the shares could be worth far more than these figures. The company also has a recurring need to raise money, which would dilute holders, and more spending or a delayed readout would lower the cash-based values. We could not use the second-quarter 2026 filing text to confirm cash beyond what the cash-flow data show; the 2026 cash figures come from that filing's financial data and the operating cash burn of about $50 million in the first half of 2026 is in line with our estimates. Because the company has more risk of cash consumption than a stable business, the margin-of-safety price is set well below the downside case.