Also known as: Regenxbio, REGENXBIO
REGENXBIO is a biotechnology company focused on gene therapies and the development of adeno-associated virus (AAV) technologies. It advances internal and partnered programs across retinal, neurodegenerative, and rare disease indications.
A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
Ocular Therapeutix's experimental drug Axpaxli showed superior vision maintenance compared to Regeneron's Eylea in a late-stage wet AMD trial. Meanwhile, Ocugen reported 12-month data showing its gene therapy OCU410 reduced geographic atrophy lesions by 31%, though less than earlier interim results. Both companies are advancing toward regulatory submissions and further clinical development.
A class action lawsuit was filed against REGENXBIO covering the period from February 9, 2022 to January 27, 2026, following FDA clinical holds on gene therapies RGX-111 and RGX-121 after a CNS tumor was discovered in a trial participant.
Global orphan drug sales are forecast to reach $409 billion by 2032, representing one-fifth of prescription drug sales, with Johnson & Johnson and Argenx leading the market amid regulatory uncertainty.
The FDA has denied or discouraged at least eight drug applications in the past year, including treatments for rare diseases, raising concerns about regulatory consistency and the future of experimental therapies.
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The FDA placed clinical holds on Regenxbio's RGX-111 and RGX-121 gene therapies following a tumor case in an MPS I patient. The company's stock fell nearly 18% on the news.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07236606 |
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) |
SUSPENDED | PHASE3 |
| NCT06942520 |
Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME) |
RECRUITING | PHASE2 |
| NCT06491927 |
Long Term Follow-up for RGX-202 |
ENROLLING_BY_INVITATION | |
| NCT06103487 |
Long Term Follow-Up for RGX-111 |
ENROLLING_BY_INVITATION | |
| NCT05693142 |
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) |
RECRUITING | PHASE2/PHASE3 |
| NCT05683379 |
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD |
RECRUITING | |
| NCT05407636 |
Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD |
RECRUITING | PHASE3 |
| NCT04704921 |
Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD |
ACTIVE_NOT_RECRUITING | PHASE2/PHASE3 |
| NCT04597385 |
Long-term Follow-Up for RGX-121 |
UNKNOWN | |
| NCT04591834 |
Mucopolysaccharidosis Type II Observational |
WITHDRAWN |
