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…
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)…
Hope in Focus Invites Community to Join the Walk to Fight Rare Diseases
Hope in Focus, a nonprofit 501(c)3 rare disease advocacy organization based in Ledyard, Connecticut, is partnering with the Denise D’Ascenzo…
Content Editor – Part-Time, Remote
Hope in Focus | Part-time | Remote Hope in Focus is a 501(c)(3) rare disease advocacy organization supporting a global…
Community Story Writer – Part-Time, Remote
Hope in Focus | Part-time | Remote Hope in Focus is a 501(c)(3) rare disease advocacy organization supporting a global…
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…
Celebrating Community and Impact — Dinner in the Dark 2025
This year marked our 10th Dinner in the Dark, held on November 1 at Foxwoods Resort Casino — a milestone…
Hope in Focus to Host Signature Fundraiser, Dinner in the Dark, Nov. 1 at Foxwoods
Hope in Focus, a nonprofit organization dedicated to advancing treatments and support for Leber congenital amaurosis (LCA), will host its…
Volunteer Opportunity: Writer/Editor for Short-Term Project
We’re in the process of relaunching the Hope in Focus website, and we need the help of a professional writer/editor…