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Archive for the Research Category

French Gene Therapy Company Advancing Three Programs for Retinal Diseases

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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.
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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: Port Delivery System Designed to Reduce Burden of Lucentis Injections for Wet AMD

Dr. Christopher Brittain, Genentech medical director, discusses his company’s port delivery system, a tiny capsule implanted into the eye, for delivery of Lucentis® over a period of a few months. The device is currently in a Phase 2 clinical trial.

The image on the right shows the port implanted into an eye.

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

ARVO 2018: Dr. Shannon Boye Reports on her Emerging Gene Therapy for LCA (GUCY2D)

At the annual ARVO research conference in Honolulu, I had an opportunity to talk with FFB-funded researcher Shannon Boye, PhD, University of Florida, about her advancement of gene therapy for Leber congenital amaurosis (GUCY2D mutations) toward a clinical trial.

ARVO 2018: World’s Largest Show and Tell for Innovations in Eye Research

arvo_post_042518In addition to funding sight-saving research, we at FFB work hard to tell the scientific world about it. That’s because knowledge sharing and collaboration are critical to accelerating the advancement of promising therapies. Progress in developing treatments and cures isn’t made in a vacuum.

The best opportunity for us to showcase FFB-funded research is at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), which is being held April 29 – May 3 this year in Honolulu. More than 11,000 eye researchers from around the world — including five intrepid members from FFB’s science team — will gather to participate in what is essentially a massive “show and tell” of the latest scientific advancements.
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Retinal Patch Performs Promisingly in Clinical Trial for Dry AMD Patients

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Regenerative Patch Technologies, a company developing stem-cell-derived treatments for people with retinal diseases, has reported encouraging results for the first five patients with advanced, dry age-related macular degeneration (AMD) participating in a Phase 1/2a clinical trial for its therapy – a patch comprised of a layer of retinal pigment epithelial (RPE) cells on a synthetic scaffold. The treatment is known as the California Project to Cure Blindness–Retinal Pigment Epithelium 1 (CPCB-RPE1).
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Study Suggests Vitamin A May Benefit Children with RP

Audio version:

An FFB-funded study at Massachusetts Eye and Ear Infirmary (MEEI) suggests that vitamin A palmitate supplementation may slow the decline of cone function by nearly 50 percent in children with retinitis pigmentosa (RP). Cones are the photoreceptors that normally provide daytime vision, and the ability to read, recognize faces, and perceive colors. Cone function was evaluated in the study by a full-field electroretinogram or ERG, which measures the retina’s electrical response to light.
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Vision Improvements Reported in Early Stem Cell Trial for Wet AMD

Audio version:

Two patients with advanced wet age-related macular degeneration (AMD) in a Phase I clinical trial demonstrated improved visual acuity sustained for one year after a sheet of retinal pigment epithelial (RPE) cells derived from embryonic stem cells was transplanted under their retinas. Each patient had one eye treated. Vision improvement for one patient was 29 letters or about 6 lines on an eye chart. The other had a gain of 21 letters or about 4 lines.
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