Sarepta Therapeutics (SRPT): A Billionaire’s High-Stakes Bet on Gene Therapy’s Future

In the high-stakes world of biotechnology investing, few bets are as bold as billionaire Glenn Russell Dubin’s multi-year stake in Sarepta Therapeutics (NASDAQ:SRPT). Dubin, co-founder of Highbridge Capital Management and a contrarian investor known for his macro-driven strategies, has positioned Sarepta as one of his top picks with a projected 226% upside potential as of late 2024. This article dissects the rationale behind his conviction, the company’s progress, and the risks that could upend its trajectory.

Dubin’s Investment Thesis: Gene Therapy as a Game-Changer

Dubin’s 12.4% stake in Sarepta as of Q3 2023 reflects his belief in the company’s ability to transform genetic medicine. His bullishness centers on two pillars:
1. Pipeline Depth: Sarepta’s expanding portfolio of gene therapies targeting rare neuromuscular disorders, including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD).
2. Regulatory Momentum: Progress in securing FDA approvals for therapies like ELEVIDYS, which treats DMD, and its siRNA programs for facioscapulohumeral muscular dystrophy (FSHD1) and myotonic dystrophy (DM1).

Dubin has consistently emphasized that Sarepta’s asymmetric risk-reward profile—high risk due to clinical trial uncertainty but massive rewards if therapies gain traction—aligns with his strategy of concentrating capital in undervalued, high-impact names.

Financial Performance: Growth Amid Growing Pains

Sarepta’s Q1 2025 results underscore its promise and challenges:
- Revenue Surge: Net product revenue hit $612 million, a 70% year-over-year increase, driven by ELEVIDYS’s 180% revenue growth.
- Operational Hurdles: Supply chain bottlenecks and a patient safety incident (a single case of liver failure) forced temporary delays, prompting Sarepta to lower its 2025 revenue guidance to $2.3–$2.6 billion from earlier expectations.

The stock’s volatility mirrors its dual narrative: breakthroughs in clinical trials drive optimism, while execution risks keep skeptics on edge.

Pipeline Progress: Key Catalysts Ahead

Sarepta’s near-term catalysts include:
1. Regulatory Submissions: 2025 filings for SRP-9003 and SRP-9004 (LGMD therapies) could unlock new markets.
2. Clinical Readouts: Anticipated data from FSHD1 and DM1 trials could validate the company’s siRNA platform, expanding its addressable patient population.

Dubin has noted that Sarepta’s non-GAAP operating profit of $334 million (excluding collaboration expenses) signals financial resilience, even amid R&D-heavy spending.

Risks: Supply Chains, Payouts, and Competition

• Supply Chain Constraints: ELEVIDYS’s revenue is concentrated in top-tier sites at full capacity, risking scalability.
• Pricing Pressure: Payer resistance to high-cost gene therapies (ELEVIDYS costs ~$4.5 million per patient) could limit adoption.
• Regulatory Scrutiny: The FDA’s stance on long-term efficacy and safety data remains a wildcard.

Conclusion: A High-Conviction, High-Risk Play

Glenn Dubin’s Sarepta stake epitomizes his “bet the farm” approach to investing. With a 226% upside target, the stock’s potential hinges on executing on its pipeline, resolving supply chain issues, and navigating regulatory hurdles.

Key Data Points to Back the Bull Case:
- Sarepta’s Q1 2025 revenue growth of 70% YoY suggests strong demand for its therapies.
- 2.37% portfolio allocation by Dubin (as of Q4 2024) reflects his long-term confidence, despite short-term volatility.
- A $11.98 billion market cap as of late 2024 leaves room for expansion if therapies gain broader adoption.

For investors, Sarepta is a high-risk, high-reward proposition. While the company’s science is groundbreaking, its success depends on operational execution and reimbursement models. Dubin’s sustained advocacy since 2023 signals a belief that these hurdles are surmountable—a bet worth watching closely in 2025 and beyond.