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....
Research Updates
BlueRock Photoreceptor Replacement Therapy Moving into a Clinical Trial
The development of gene therapies for inherited retinal diseases (IRDs) took off when young adults and children showed significant vision...
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 to people...
Théa Forms Sepul Bio to Advance RNA Therapies for LCA10 and USH2A into Clinical Trials
In December 2023, ProQR sold its sepofarsen (LCA10) and ultevursen (USH2A) programs to Théa, a large European biotechnology company focused...
2023 LCA Family Conference: Developing LCA Treatments
Luxturna®, the only approved treatment for one of 27 identified forms of Leber congenital amaurosis (LCA), cost $500 million to develop...
Let’s Chat About … RARE-X with Karmen Trzupek
Through global data sharing and analysis, the nonprofit RARE-X (the research arm of Global Genes) is working to accelerate treatments for rare...
Second Global RDH12 Scientific Conference Brings Clinical Trials Closer to Reality
The community of people living with Leber congenital amaurosis caused by mutations in the RDH12 gene moved closer to realizing the shared...
Editas Medicine’s EDIT-101 Clinical Trial Update Brings Mixed News on LCA10 (CEP290) Research
The trial update includes safety and efficacy data from all 14 patients treated to date in the study comprised of...
Let’s Chat About … Atsena Therapeutics’ LCA Research with Kara Fick and Shannon Boye
Atsena Therapeutics, a clinical-stage gene therapy company focused on reversing and preventing blindness, has an ongoing Phase 1/2 clinical trial...
Let’s Chat About…Advancing Treatments into Clinical Trials with Ben Shaberman
Innovative funding initiatives created by the Foundation Fighting Blindness are accelerating research advances to find treatments for Leber congenital amaurosis (LCA) and other rare inherited...