RGNX

RGNX is a clinical-stage AAV gene-therapy company whose $685M market cap is a bet on pipeline optionality, not the reported financials. FY2025 revenue of $170M (+104.5%) looks like growth but is milestone/collaboration income — the five-year history ($470M→$113M→$90M→$83M→$170M) is lumpy partnership recognition, not a durable commercial ramp. Underneath, the business burns cash relentlessly: operating income -$161M, net loss -$194M, R&D $228M, and -$124M operating cash flow. The balance sheet is the loudest signal — cash & equivalents fell 40% to $34.5M, equity dropped 60% to $103M, liabilities jumped 70% (3.41x leverage), and the accumulated deficit is now $1.13B. Management explicitly flags 'our ability to continue as a going concern,' so a dilutive raise on top of the +3% share growth is a live risk.

The bull case rests almost entirely on ABBV-RGX-314 (sura-vec), partnered with AbbVie for wet AMD and diabetic retinopathy. Enrollment in the pivotal ATMOSPHERE and ASCENT trials completed in October 2025, with topline data expected Q4 2026 — a genuine, near-dated, value-defining catalyst backed by a deep-pocketed partner sharing cost and commercialization. A second partnership (Nippon Shinyaku on RGX-121/RGX-111) adds non-dilutive validation.