Regenxbio

REGENXBIO Inc. announces participation in UBS Global Healthcare Conference (Nov 13, Rancho Palos Verdes), Stifel 2024 Healthcare Conference (Nov 19, New York), and Piper Sandler 36th Annual Healthcare Conference (Dec 5, New York). Webcasts available at www.regenxbio.com.

Global exosome market to grow at ~20% CAGR by 2030, driven by chronic disease incidence, diagnostic improvements, and personalized medicine interest. North America leads, with key players like Danaher and Thermo Fisher Scientific. Notable developments include Aruna Bio's US patent for neural exosomes and EXO Biologics' EUR 16 million Series A funding.

Christine N. Kay, MD, presented TEASE clinical trial findings on gildeuretinol (ALK-001) for Stargardt disease at the AAO Meeting, showing a 21% reduction in atrophic lesion growth. Gildeuretinol, an oral vitamin A-derived drug, aims to slow disease progression and has FDA orphan drug and breakthrough designations, with further data expected in 2025.

DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.

Phase 2 sub-study at AAO Meeting shows ABBV-RGX-314 gene therapy reduces annualized injection needs by 97% for wet AMD, with 78% of patients injection-free at 9 months, and no new safety signals.

REGENXBIO's ABBV-RGX-314 gene therapy for wet AMD reduced antiVEGF injections by 97% in 9 patients with bilateral disease, with 78% injection-free post-treatment. Safety was well-tolerated, with no serious adverse events. This marks the first bilateral treatment for wet AMD, potentially offering a one-time effective treatment.

The gene therapy market is rapidly expanding, with over 180 companies developing more than 200 gene therapies. Key players include Ultragenyx Pharmaceutical Inc, Rocket Pharmaceuticals, and Adverum Biotechnologies. Prominent therapies in the pipeline include DTX401, RP-L102, and ADVM-022. Gene therapy aims to treat diseases by modifying genes, offering potential long-lasting effects with fewer side effects. Challenges include immune reactions, long-term effects, and high costs.

Regenxbio announced positive Phase II data for ABBV-RGX-314 in wet AMD, showing 97% reduction in anti-VEGF treatment burden, 100% of patients needing zero or one supplemental injection, and 78% injection-free. Best-corrected visual acuity and central retinal thickness were sustained, with no drug-related serious adverse events as of September 11, 2024.

ABBV-RGX-314 shows 97% reduction in treatment burden at nine months, favorable safety and efficacy, and potential for bilateral wet AMD treatment.