The biopharmaceutical company Apellis has treated the first patient in its Phase 3 clinical trial program for APL-2, a compound designed to slow the progression of advanced dry age-related macular degeneration (AMD) also known as geographic atrophy (GA).
More than two decades ago, the Foundation Fighting Blindness (FFB) began funding RPE65 gene therapy research that led in late 2017 to LUXTURNA™, the first FDA-approved gene therapy for the eye or an inherited condition. Ultimately, the Foundation provided more than $10 million in funding for the groundbreaking effort.
ProQR, a biotech company in the Netherlands, has reported vision improvements for patients in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.
The company reported that 60 percent of subjects in the trial demonstrated improvements in visual acuity and their ability to navigate a mobility course. The treatment was also safe for patients.
Ophthotech is a biopharmaceutical company committed to developing therapeutics and gene therapy solutions to treat retinal diseases. The company is aggressively pursuing therapies for orphan conditions like retinitis pigmentosa (RP), Stargardt disease, and Leber congenital amaurosis (LCA), as well as common indications such as wet and dry age-related macular degeneration (AMD).
“The first time I examined a person’s retina as a junior resident, something clicked. All the things I found interesting came together — surgery, patient care, genetics, regenerative medicine, and cell biology,” says Mandeep Singh, MD, PhD, assistant professor in ophthalmology, Wilmer Eye Institute, Johns Hopkins Medicine. “I knew retina was what I wanted to focus on for my career.”
Dr. Singh is one of four new recipients of career development awards (CDAs) from the Foundation Fighting Blindness. Each recipient will receive a total of $375,000 over five years to help build an independent research program in addition to their clinical practices. FFB currently funds 12 CDAs for up-and-coming clinicians to advance their experience and expertise in research for retinal degenerative diseases. Since its inception, FFB has given CDAs to more than 100 clinical investigators.
Hosted by the Foundation Fighting Blindness and Casey Eye Institute at Oregon Health & Science University, the Innovation Summit for Retinal Cell and Gene Therapy has emerged as one of the most essential events for researchers and companies developing treatments and cures for retinal degenerative diseases.
In its fifth year, the Innovation Summit featured 40 presentations from industry experts from around the world. More than 250 people were in attendance. The event was held on April 27, the day before the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Honolulu.
Get the inside scoop on this year’s top research advances and what’s next in retinal disease science by watching VISIONS 2018 LIVE!
We will be streaming several of our annual VISIONS conference sessions live on Friday, June 22 and Saturday, June 23. The sessions will be live streamed here on our blog, and our national Facebook as well.
Here’s the full schedule of live sessions:
Friday, June 22, 2018
- 9:00 a.m. PDT – 10:15 a.m. PDT– Opening Session: Mission Possible! The Year’s Top Advances and What’s Next in Retinal Disease Science
- 12:30 p.m. PDT – 2:00 p.m. PDT – Keynote Luncheon: Think Outside our Perceived Limitations
- 3:10 p.m. PDT – 4:15 p.m. PDT – What You Need to Know About Genetics, Genetic Testing, and Patient Registries
- 4:20 p.m. PDT – 5:25 p.m. PDT – Clinical Trials Update and Is a Clinical Trial Right for You?
Saturday, June 23, 2018
- 9:00 a.m. PDT – 10:25 a.m. PDT – Retinitis Pigmentosa (RP) Research and Clinical Updates
- 10:30 a.m. PDT – 11:40 a.m. PDT– Stress Management and Self-Care for People with Degenerative Diseases
- 1:00 p.m. PDT – 2:35 p.m. PDT – Adapting and Thriving: Find Your Product – Tips and Tools for Peak Personal Performance
- 3:05 p.m. PDT – 4:30 p.m. PDT – Closing Session: Featuring Biotech Executive Emmett Cunningham and Chapter Awards
The full list of the many sessions that will be live on the blog, with additional details, is also on our website here.
We hope you’ll tune in, share it with your friends, and learn more about the promising new advances in retinal research.
Horama, a French biotech developing gene therapies for rare eye diseases, was established in 2014 as a spin-off of INSERM, France’s public scientific and technology institute. Today, the company has three gene-therapy development programs underway for rare inherited retinal diseases, targeting people with mutations in the genes PDE6B, RPE65, and RLBP1.