REGENXBIO Inc. (RGNX): A Bull Case Theory

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We came across a bullish thesis on REGENXBIO Inc. on Valueinvestorsclub.com by mtmutombo. In this article, we will summarize the bulls’ thesis on RGNX. REGENXBIO Inc.'s share was trading at $8.50 as of July 16^th.

REGENXBIO (RGNX) is a gene therapy pioneer with commercialized technology and multiple late-stage programs, yet trades at a distressed valuation with an enterprise value below $100 million despite over $420 million in pro forma cash. The stock’s weakness is largely due to broader biotech malaise and heightened uncertainty under the Trump administration's FDA appointments, especially around DMD therapy approvals.

RGNX's DMD program, RGX-202, has strong biomarker and functional data and superior safety versus competitors, but the company’s valuation is compelling even when discounting DMD. The core investment thesis centers on RGNX’s partnership with AbbVie on RGX-314 for wet AMD, which recently expanded into diabetic retinopathy, triggering a $200 million milestone. Positive Phase II data showed a 97% reduction in rescue treatments, with pivotal data due in 2026.

Separately, RGX-121 for MPS II, now partnered with Nippon Shinyaku, could earn a $150 million priority review voucher (PRV) upon likely approval in late 2025. The company also monetized Zolgensma royalties for $150 million upfront and $100 million in milestones, reinforcing its cash runway through 2027. Zolgensma, an approved SMA therapy marketed by Novartis, is forecast to exceed $3 billion in peak sales.

Despite these high-quality assets and revenue streams, RGNX trades as if its programs are deeply impaired. Even assigning only a 10% probability of success to the DMD program, a conservative sum-of-the-parts model yields a valuation far above current levels. RGNX represents a deeply undervalued platform with credible late-stage assets, strong partners, and meaningful near-term catalysts, mispriced due to FDA uncertainty rather than clinical fundamentals.

Previously we covered a bullish thesis on Rocket Pharmaceuticals, Inc. (RCKT) by sharkbiotech in March 2025, which highlighted strong Danon disease data, a large market opportunity, and solid financial runway. The company’s stock price has depreciated approximately by 66.97% since our coverage. This is because the thesis has not yet played out. mtmutombo shares a similar perspective but emphasizes REGENXBIO’s diversified pipeline and near-term catalysts.

REGENXBIO Inc. is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 35 hedge fund portfolios held RGNX at the end of the first quarter which was 35 in the previous quarter. While we acknowledge the potential of RGNX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.

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Disclosure: None.