Going Toward the Light: Nobel Prize Winners Discovered Optogenetics for Restoring Vision
Hope in Focus is delighted with the announcement that the Nobel Prize in Physiology or Medicine 2026 has been awarded...
Research Updates
Opus Genetics in Alignment with FDA for Phase 3 LCA5 Gene Therapy Clinical Trial
Opus Genetics has reached alignment with the US Food and Drug Administration (FDA) on the Phase 3 clinical trial design...
Report from 2026 ARVO: Several Emerging LCA Gene Therapies are Restoring Vision
In reporting on emerging therapies for LCA and other retinal diseases, my mantra has always been “just the facts.” If...
LCA5 Gene Therapy Development Could be Accelerated Thanks to New FDA Rare Disease Review Process
Opus Genetics’ emerging LCA5 gene therapy, OPGx-LCA5, has been accepted into the US Food & Drug Administration’s (FDA) new Rare...
Light at the End of the Tunnel: LCA6 Gene Therapy Advances Toward Clinical Trial
I am excited to report on impressive progress in the development of Odylia Therapeutics’ emerging gene therapy for retinal degeneration...
A Vision-Restoring XLRP Gene Therapy Gets New Life: MeiraGTx to Seek FDA Approval
In May 2025, Johnson & Johnson (J&J) announced that though its emerging gene therapy bota-vec for X-linked retinitis pigmentosa (XLRP)...
LCA5 Gene Therapy Provides Vision Improvements in Clinical Trial
Nothing is more hopeful or gratifying for the retinal disease community than an emerging or FDA-approved therapy that provides vision...
Promising RNA Therapies for LCA10 and USH2A Move Back into Clinical Trials
Moving emerging therapies through clinical trials and across the finish line is often challenging—and in some cases, harrowing. Many treatments...
Reports on Gene Therapy Advances: A Highlight from the 2025 Hope in Focus Conference in Minneapolis
Gene therapy is unequivocally the most advanced approach for treating retinal diseases like Leber congenital amaurosis (LCA). Of course, there’s LUXTURNA® which is...
Diverse Emerging Therapies Featured at the 2025 Hope in Focus Conference in Minneapolis
The development of diverse treatment approaches for Leber congenital amaurosis (LCA) is necessary because no single modality will work for everyone affected....