Regenxbio (NasdaqGS:RGNX) FY Conference Transcript

Summary of Regenxbio FY Conference Call (January 14, 2026) Company Overview - Company: Regenxbio (NasdaqGS: RGNX) - Key Speakers: Curran Simpson (CEO), Mitch Chan (CFO), Dr. Steve Pakola (CMO) [1] Industry and Technology - Industry: Gene Therapy - Technology: AAV (Adeno-Associated Virus) gene therapy with over 5,000 patients dosed [2][3] - Focus: Development of new capsids to enhance therapeutic efficacy and safety [5] Core Points and Arguments Pipeline and Product Development - BLA Review: The company has a Biologics License Application (BLA) under review for the Hunter program, with a PDUFA date of February 8, 2026 [8][22]. - Late-Stage Programs: Top-line readouts expected for two late-stage programs: Duchenne program (RGX-202) in Q2 2026 and wet AMD program (RGX-314) with AbbVie later in 2026 [4][8]. - Duchenne Program: Aiming to provide functional benefits to children with Duchenne muscular dystrophy, with pivotal data showing significant improvements in patient outcomes [10][11][19]. - Wet AMD Program: RGX-314 is positioned as a potential first non-rare gene therapy approved, with significant commercial readiness efforts in collaboration with AbbVie [9][25]. Manufacturing and Scalability - Manufacturing Capabilities: The company has advanced manufacturing processes, capable of producing 2,500 doses per year for RGX-202 and up to 350,000 doses for RGX-314 [28][29]. - Quality Control: Achieved an 80% full capsid level in batches, which is critical for safety and efficacy [29]. Safety and Efficacy - Immune Suppression Regimen: A proactive immune suppression strategy has been implemented to enhance safety and efficacy, allowing for higher dosing without significant adverse effects [36][37]. - Clinical Outcomes: Positive safety profiles and functional benefits observed in patients, with significant improvements in NSAA scores [19][17]. Additional Important Content - Global Expansion: Plans for expanding clinical studies outside the U.S. to address broader patient needs [21][39]. - Partnerships: Collaboration with AbbVie for the wet AMD program, leveraging their existing sales force and expertise in ophthalmology [43][44]. - Market Potential: The company is targeting significant unmet needs in rare diseases and chronic retinal diseases, with a focus on long-term patient outcomes and reducing treatment burdens [31][27]. Conclusion - Future Outlook: Regenxbio is positioned for a transformative year with multiple late-stage catalysts, a strong manufacturing base, and a commitment to patient-centric outcomes in gene therapy [31].