Listen to this page using ReadSpeaker
Posts tagged retinitis pigmentosa

FFB Funding More than $2 Million in New Research

The Foundation Fighting Blindness has announced funding for seven new research projects to advance the development of treatments and cures for retinal degenerative diseases. Each project will receive a total of $300,000 over a three-year period.

The grants were selected through FFB’s annual call for research proposals from individual investigators. Seventy scientists submitted requests for funding. Applications were reviewed by FFB’s Scientific Advisory Board, which is comprised of the world’s leading retinal experts.
Continue Reading…

Watch VISIONS 2018 LIVE on June 22 & 23

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.

 

French Gene Therapy Company Advancing Three Programs for Retinal Diseases

Audio version:

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.
Continue Reading…

ARVO 2018: Dr. Henry Klassen Provides Update on jCyte Stem Cell Trials

Dr. Henry Klassen, jCyte co-founder and investigator at UC Irvine, provides an update in the video below on the clinical trials for an RP therapy derived from stem cells.

ARVO 2018: Dr. Stephen Daiger Reports on the State of Genetic Testing for Inherited Retinal Diseases

After presenting a poster on a new mutation in the RP gene KIF3B at the ARVO meeting in Honolulu, FFB-funded geneticist Dr. Stephen Daiger discusses the progress that’s been made in genetic testing for people with inherited retinal conditions.

ARVO 2018: Dr. Steve Rose Reports on CRISPR/Cas9 for Inherited Retinal Diseases

FFB’s own Dr. Steve Rose, chief scientific officer, reviews our commitment to funding and exploring CRISPR/Cas9 gene editing for inherited retinal disease in the video below.

FFB currently funds CRISPR/Cas9 projects at four institutions:

  • Johns Hopkins University (retinitis pigmentosa caused by the P23H mutation in RHO)
  • Columbia University (RP caused by the D190N mutation in RHO)
  • Massachusetts Eye and Ear Infirmary (RP caused by a mutation in RP1)
  • UCLA (Usher syndrome 1B caused by a mutation in MYO7A)

Want to learn more about the benefits of CRISPR/Cas9? Check out: A Cut-and-Paste Approach to Fixing Retinal-Disease Genes

Clinical Trial to Launch for System Combining Optogenetics and Eyewear

Audio Version:

gensightThe French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1/2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation. The system is designed to restore vision for people who are blind from retinitis pigmentosa (RP) and potentially other retinal conditions such as: Usher syndrome, Stargardt disease, and dry age-related macular degeneration.

The GS030 will be evaluated in 18 people with RP who can see no better than counting fingers. Additional trial details are available at the clinical trials Web site hosted by the National Institutes of Health.
Continue Reading…

MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial

MeiraGTx, a gene-therapy company in London and New York City, has treated its first patient in a gene-therapy clinical trial for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. The Phase I/II study is taking place at Moorfields Eye Hospital in London. The safety-oriented trial will enroll 36 participants. Three dose levels of the therapy will be evaluated.

XLRP is a leading cause of inherited, progressive retinal degeneration and vision loss. The condition usually affects males, but is also diagnosed occasionally in females. Mutations in the gene RPGR cause about 70 percent of XLRP cases. RPGR mutations affect about 15,000 people in the United States and tens of thousands more around the world.
Continue Reading…

FFB-Funded Scientists Report on Nine Promising Translational Research Efforts

Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.

The Foundation Fighting Blindness has taken the translational challenge head on by investing more than $75 million in therapy-development projects with strong clinical-trial potential through its Translational Research Acceleration Program (TRAP), which includes Gund-Harrington Scholar Awards.
Continue Reading…

SparingVision Formed to Advance Sight-Saving Protein for RP

L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, and Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance

L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance

The development of a vision-saving treatment for people with retinitis pigmentosa (RP) is getting a major boost thanks to the formation of the French biotech SparingVision to move it into a clinical trial and out to the international marketplace.

A spin-off of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone-viability factor (RdCVF). The emerging therapy performed well in several previous lab studies funded by the Foundation Fighting Blindness. SparingVision’s goal is to launch a clinical trial for the protein in 2019.
Continue Reading…