RegenXBio Earnings Call Balances Gene Therapy Risks

RegenXBio reported that dosing is complete in the AFFINITY pivotal Duchenne trial, with robust enrollment in the confirmatory study and encouraging Phase 1/2 data. At 18 months, treated boys showed a ...
Healio

Woman presents with visual disturbances upon awakening

A 65-year-old white woman presented with 9 months of intermittent “kaleidoscopic” visual disturbances and blurred vision upon awakening.Her medical history was significant for hypertension, ...

REGENXBIO Inc. (NASDAQ:RGNX) Q4 2025 earnings call transcript

REGENXBIO Inc. (NASDAQ:RGNX) Q4 2025 Earnings Call Transcript March 5, 2026 REGENXBIO Inc. misses on earnings expectations. Reported EPS is $-1.3 EPS, expectations were $-1.01. Operator: Thank you for ...
The Malaysian Reserve

REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights

Late-stage gene therapy pipeline for rare and retinal diseases advancing toward key catalysts RGX-202 for Duchenne muscular dystrophy: New Phase I/II data ...
Ophthalmology Times

Real-world approaches to managing DME: Improving durability, drying, and patient burden

Severe, long-standing DME with cystoid changes, subretinal detachment, and high inflammatory cytokine burden tends to respond better to dual ANG2/VEGF-A inhibition than VEGF monotherapy. Faricimab ...
Science Daily

Wireless retinal implant helps blind patients see again

A tiny wireless implant is giving new hope to people blinded by advanced age-related macular degeneration. In a major ...

Opus Genetics, Inc.: Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting

Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or dose-limiting toxicities at three months Early signals of functional and str ...

Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting

Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or dose-limiting ...
Ophthalmology Times

Rare Disease Day: An abundance of research Is underway to address rare eye diseases

Stargardt programs address ABCA4 payload constraints via dual-AAV platforms (VG801, AAVB-039) and explore first ...
MedPage Today

Retinal Implant-Eyeglasses Combination Improved Vision in Geographic Atrophy

ORLANDO -- The PRIMA system combining a subretinal photovoltaic implant and eyeglasses that project near-infrared light to the implant significantly improved vision in patients with geographic atrophy ...
bjo.bmj

Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients

2 Development Division, CHA Biotech Company, Ltd, Seoul, Republic of Korea 3 Division of Rheumatology, Department of Internal Medicine, CHA Bundang Medical Center, Seongnam, Republic of Korea 4 ...
Medscape

AMD Gene Therapy Sustains Efficacy in First Two-Eye Study

The first study to evaluate both eyes treated with a gene therapy targeting neovascular, or wet, age-related macular degeneration (AMD) has found the treatment as safe and effective in the second eye ...