Regenxbio (RGNX) Goldman Sachs 45th Annual Global Healthcare Conference summary

Leadership transition and strategic direction

• Announced transition of Curran Simpson to President and CEO effective July 1, with Ken Mills moving to non-executive Chairman of the Board.

• Leadership change is part of a planned process to guide the company into commercialization.

• No major strategic shifts expected; focus remains on late-stage assets RGX-202, RGX-314, and RGX-121.

• 2024 plans and late-stage execution to continue as previously outlined.

Pipeline focus and technology platform

• Core focus on AAV gene therapy in retina, muscle, and neurodegeneration.

• Strategic resource allocation to late-stage assets, aiming for all pipeline programs to reach late-stage or BLA filing by year-end.

• Major partnership with AbbVie in retina, targeting wet AMD and diabetic retinopathy.

• Duchenne muscular dystrophy program aims for best-in-class status with novel biology.

• Hunter syndrome program nearing BLA submission, supported by strong manufacturing capabilities.

Clinical data and product differentiation

• RGX-314 shows durable vision improvement in wet AMD, with up to 4 years of maintained or improved outcomes.

• Targeting at least 50% of wet AMD patients to maintain vision without further injections.

• Suprachoroidal delivery being developed for broader, in-office use.

• Diabetic retinopathy represents a unique, high-need market with little competition for one-time gene therapy.