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.
Biotech companies posted Q2 2026 results: REGENXBIO's Duchenne trial met its primary endpoint, Compass Pathways' COMP360 NDA submission is on track for Q4, Puma raised full-year guidance on NERLYNX demand, and Evotec cut its 2026 outlook.
Bristol Myers Squibb, Sanofi, and Pfizer have filed new patent infringement suits over drugs including Camzyos, Lantus, and abrocitinib, while the Federal Circuit denied Sarepta's rehearing bid in a Regenxbio DNA patent case.
A securities class action lawsuit alleges REGENXBIO misled investors about the safety of its gene therapy RGX-111. Following an FDA clinical hold due to a patient tumor, the stock fell 17.8% in one day. Affected shareholders have until April 14, 2026 to seek lead plaintiff status.
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 |
|---|---|---|---|
| NCT07652606 |
Expanded Access to RGX-202 |
TEMPORARILY_NOT_AVAILABLE | |
| NCT07236606 |
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) |
ACTIVE_NOT_RECRUITING | 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) |
ACTIVE_NOT_RECRUITING | PHASE2/PHASE3 |
| NCT05683379 |
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD |
ACTIVE_NOT_RECRUITING | |
| NCT05407636 |
Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05296447 |
Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With and Without Diabetic Retinopathy |
ENROLLING_BY_INVITATION | PHASE2 |
| NCT05210803 |
Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD |
ENROLLING_BY_INVITATION | PHASE2 |
