Regenxbio (RGNX) Chardan's 8th Annual Genetic Medicines Conference summary

Company overview and pipeline highlights

• Over 15 years in gene therapy, with a shift from out-licensing to internal program development and significant growth in staff and capabilities.

• Focus on two main franchises: rare diseases (notably RGX-202 for Duchenne muscular dystrophy) and retinal diseases (notably RGX-314, partnered with AbbVie).

• Four programs advancing to late-stage development, with large commercial potential and strong execution in late-stage transitions.

RGX-202 (Duchenne muscular dystrophy) program

• RGX-202 features a differentiated microdystrophin construct with a human-like C-terminus, aiming for improved half-life and muscle repair.

• Safety profile is strong, with no serious adverse events and high microdystrophin levels in older patients, potentially enabling long-term functional benefits.

• Targeting a broad age range, including a new cohort for ages 1–3, addressing unmet needs and aligning with trends in newborn screening.

• Confident in accelerated approval pathway after positive FDA interactions and end-of-phase 2 meeting; pivotal study design aligns with regulatory expectations.

• Manufacturing capacity exceeds 2,000 doses/year, providing a cost advantage and supporting commercial scale.

RGX-314 (Retinal diseases) program

• Subretinal approach for wet AMD shows durable efficacy and safety, with some patients injection-free for 4–5 years.

• Suprachoroidal approach targets broader market access and is advancing through dose escalation, with strong safety enabling higher dosing.

• Two phase 3 studies (ATMOSPHERE in the US, ASCEND globally) compare RGX-314 to standard treatments, aiming for robust data at filing.

• Phase 2 AVIATE study in wet AMD is escalating to dose level 4, seeking further reductions in supplemental injections and improved vision outcomes.

• Prophylactic steroid regimen allows early tapering and maintains excellent tolerability at higher doses.