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Foundation News » Retinitis Pigmentosa
Enhanced Imaging Technique May Help Identify Clinical Trial Participants
Advances in AOSLO technology are increasing the likelihood of finding the best candidates.
QLT’s Drug for RP and LCA Improves Vision in 11 Patients
In the biotech company’s clinical trial, participants have been able to navigate without a cane and see objects for the first time.
Foundation Honors Visionary Leaders at Annual Conference
Awards were presented to three people who have made significant, long-lasting contributions to the fight against blindness.
ARVO Research Highlights from Foundation’s Scientific Advisory Board
Among the SAB’s picks are emerging treatments for choroideremia, XLRP, dominant RP and AMD.
Foundation Invests $1.5 Million to Find Elusive RP Genes
Two leading retinal experts will use the techniques they have developed to help track down ultra-rare mutations.
FFB-HHMI Fellowships Promote Retinal Research Careers to Medical Students
This year’s awards go to students at Case Western Reserve and Columbia University who will conduct lab research for retinitis pigmentosa.
Companies Collaborate to Advance Dominant RP Gene Therapy
The American and Irish outfits have teamed up to increase the treatment’s chances of making it to the marketplace.
Second Sight Reports Future Plans for Bionic Retinas
Its Argus II system will be tested for use in patients with dry AMD and upgraded to provide more detailed vision.
Synthetic Form of Active Marijuana Ingredient Preserves Vision in Rats with RP
The treatment has also produced positive results in other disease studies, but would need to be refined before it’s tested in humans.
First Patients Receive Commercial Implant of Argus II Bionic Retina
The device, which received FDA approval last year, is able to restore limited vision to people with advanced RP.
RP Gene Therapy Improves Vision After Significant Retinal Degeneration

Tested in a mouse model by a Foundation-funded team, the treatment shows promise for targeting a specific form of retinitis pigmentosa.

GenSight’s Optogenetic Therapy Advancing Toward Human Study

Targeting various retinal diseases, the French company hopes to launch a clinical trial in 2015.

Tackling the Next Gene Therapy Challenge: Autosomal Dominant Diseases

Backed by the Foundation, Dr. Al Lewin is spearheading a ‘knock-down and replace’ strategy targeting RP caused by the rhodopsin gene.

Gene Linked to Rare Metabolic Disorder also Associated with RP

The discovery, made by a Dutch research team, shows that various mutations in one gene can affect different parts of the body.

Cone-Rod Dystrophy Gene Therapy Rescues Vision in Canines

Developed by a research team in France, the treatment is now being adapted for use in human studies.

NEK2 Identified as New Retinitis Pigmentosa Gene

The discovery was made by a Foundation-funded team of researchers employing a state-of-the-art technique.

Orphan Designation Boosts Clinical Development of RP Stem Cell Therapy

The company ReNeuron plans to launch a clinical trial of the treatment in mid-2014.

Medicare to Provide Payment for the Argus II Retinal Prosthesis System

Approved by the FDA in February, the device is capable of restoring some vision in people with advanced RP.

The Foundation Invests $2.1 Million in Seven New Research Efforts

Among the projects, awarded by the Scientific Advisory Board, are those targeting specific as well as multiple diseases.

Foundation Funding Two New Optogenetics Projects at UC Berkeley

The Foundation is bolstering its commitment to restoring vision for people with the most advanced retinal diseases by investing $800,000 in the development of two emerging optogenetics therapies at the University of California, Berkeley.

Alpha IMS Becomes Second Bionic Retina Approved in Europe

The device’s creator, Retina Implant AG, of Germany, is planning to launch clinical trials in the United States.

Argus II Retinal Prosthesis to be Offered at 12 U.S. Clinical Centers

Also known as the “bionic retina,” the FDA-approved device has been shown to help people with advanced retinitis pigmentosa.

Genetic Study of Chinese Patients with Recessive RP Provides Global Benefits
A Foundation-funded team focused on one of the world’s largest retinitis pigmentosa populations finds new genetic mutations.
Researchers Identify Better Virus for Retinal Gene Delivery
The new virus can penetrate most cells in the retina through a less-invasive injection.
New Animal Model Improves Understanding of XLRP Due to RP2 Mutations

The Foundation-funded advancement brings researchers closer to developing treatments for humans.

Retinal Symposium Focuses on Research and Financial Partnerships for Advancing Therapies

The Foundation-hosted event brought together 150 venture capitalists, drug company representatives, researchers and people affected by retinal diseases.

Bone Marrow Stem Cells in Clinical Trial for Retinal Diseases
Extracted from the hip bone, the cells may be able to heal tissue in people who have lost vision due to loss of blood flow. 

GenSight Receives $41.3 Million for Ocular Gene Therapy Development

Provided by venture capitalists, the funding helps to move forward a project supported by the Foundation Fighting Blindness.

Stem Cell Pioneers Creating Retinal Patch to Restore Vision
The Foundation-funded project has the potential to help people affected by a variety of retinal diseases.

XLRS Natural History Study Beginning in Portland, Oregon

The study will help researchers choose participants for clinical trials and measure the effectiveness of potential treatments.

Six Emerging Retinal Therapies Receive $3.1 Million in Foundation Funding

Backed by a truly collaborative effort, each of the cutting-edge projects has the potential to eventually move into a clinical trial.

German Company’s Bionic Retina Restores Some Vision in Clinical Trial

Retina Implant AG is testing its device in three countries and hoping to expand its human study to the United States.

Vision-Restoring Retinal Prosthesis Receives FDA Approval

Designed for people with advanced retinitis pigmentosa, the “Argus II” will be available in the United States later this year.

X-Linked RP Sometimes Misdiagnosed as Autosomal Dominant RP

Results from a Foundation-funded study involving more than 250 affected families underscore the importance of genetic testing and counseling.

Saving Vision by Turning Rods into Cones

Working with an animal model, a research team reduced the rate of retinal degeneration using a gene therapy that shows promise for treatment in people.

Increasing Blood Vessel Permeability to Get Treatments to the Retina

In a lab study, Foundation-funded researchers identified a potential way to help vision-saving treatments reach their intended destinations.

Scientists Report Significant Advancements in Clinically Focused Retinal Research

Gene therapies, stem cell treatments and vision-preserving plant extracts were among the subjects covered by participants in the Foundation’s annual TRAP meeting in Las Vegas.

New Gene Linked to X-Linked Retinitis Pigmentosa

The discovery, made by a research team based in England, will help with future diagnoses and potential treatments for the condition.

Retinal Gene Therapy Developer AGTC Receives $37.5 Million in Venture Capital

Leveraging the Foundation’s support, Applied Genetic Technologies Corporation is developing gene therapies for achromatopsia and X-linked retinoschisis.

New Information on Vitamin A Treatment Regimen Now Available

Based on Foundation-funded clinical studies, the regimen may add two decades’ worth of useful vision for people affected by retinitis pigmentosa.

Researchers Measure Retinal Function Through Vision Map of the Brain

In the future, the technique could help determine changes in vision and the effects of treatments.

Reducing Inflammation to Save Vision

Two recent studies identify the role chronic inflammation plays in retinitis pigmentosa and a potential method of treatment.

FDA Grant Expands Foundation-Funded Study of Retinal Imaging Technology

A closer look at the effects of potential treatments could lead to quicker determinations of their effectiveness.

$17.3 Million Invested in Stem Cell Treatment for RP

The California Institute of Regenerative Medicine awards a grant to a researcher aiming to reactivate inactive cones in patients’ retinas.

Simpler Gene Therapy Envisioned for Some Forms of Dominant RP

A recent discovery made by a Foundation-funded team may help expedite getting treatments into clinical trials.

 

Emerging Recessive RP Gene Therapy Moves Closer to Human Study
Tests in an animal model showed that delivery of a healthy version of a specific gene preserved vision for 18 months.

Foundation Invests $2.4 Million in Eight New Sight-Saving Research Projects

Aimed at providing treatments and identifying causes, the projects have the potential to help those with LCA, AMD, RP and Usher syndrome, among other diseases.

Chemical Restores Vision in Lab Study

In the early stages of a study involving an animal model, small amounts of what is known as AAQ enabled subjects to respond to light.

Making Genetic Counseling More Accessible

Knowing how to identify a disease-causing gene or what to do with that information isn’t always easy. A new phone-based counseling service is helping to simplify the process.

Stem Cell Clinical Trial for RP Planned for Late 2013
A British company recently announced that it intends to launch a clinical trial that would provide those affected with healthy retinal cells.

New Study Reveals That One Exposure to Nicotine Reduces Retinal Function

Researchers found that the equivalent of one or two cigarettes’ worth can adversely affect otherwise healthy eyes.

FDA Meeting Helps Pave the Way for Clinical Trial of Cross-Cutting Gene Therapy

The Foundation and its partner RetroSense Therapeutics continue to push an optogenetic approach to treating various diseases.

ARVO Delivered Good News from Several Retinal Treatment Clinical Trials

More than 12,000 eye experts attended the Association for Research in Vision and Ophthalmology’s annual conference in Fort Lauderdale.

Part of the Retina May Have the Potential for Self Repair

A recent study shows that, for some retinal diseases, the retina may have the potential to heal itself.

Healthy, Happy Living May Slow Retinal Degeneration

A recent study suggests that exercise, stress reduction and social activity have a positive effect on vision.

Charles Bonnet Syndrome Causes Blind People to "See Things"

A recent report in an ophthalmology journal highlights a surprising, and sometimes unsettling, experience for people who have lost their vision.

Generous Foundation Supporters Fuel the Fight Against Blindness
A handful of recent contributions showcase donors’ commitment to funding research focused on saving and restoring sight.

Vision Testing and Retinal Imaging in Clinical Trials: What Patients Can Expect

As more people are recruited for human studies, the demand for measuring the ways in which the eyes respond to treatments increases.

Drug for RP and LCA Continues to Perform Well in Clinical Trial

Many of the patients in the study, being conducted by QLT, Inc., report improvement in visual acuity and the size of their visual fields.

Foundation Commits $2 Million to Development of a Cross-Cutting Drug Treatment

The start-up company MitoChem Therapeutics has identified three compounds that could benefit people affected by a variety of retinal diseases.

Valproic Acid Clinical Trial for RP Expanding to Boost Participant Enrollment

The Foundation-funded study, which began last year, is for the treatment of autosomal dominant retinitis pigmentosa.

Omega-3 Rich Diet Combined with Vitamin A Slows Visual Acuity Decline in Patients with RP
A report published by a Foundation-funded research team concludes that the diet could translate into 18 additional years of preserved central vision.

Foundation Funding Developer of “Slimy” Gene Therapy for Restoring Sight

In pre-clinical studies, genes from algae are being used in a cutting-edge therapy known as ‘optogenetics.’

Success in a Canine Study Moves Gene Therapy for X-linked RP Closer to a Clinical Trial

The treatment restored cells in degenerated retinas and was able to improve vision dramatically, according to investigators.

Researchers Find Further Evidence That Bile Acid Slows Vision Loss in Mice

A Foundation-funded team at the University of Iowa concludes that TUDCA shows promise for treatments of certain kinds of RP and Bardet-Biedl syndrome.

First Gene Therapy Clinical Trial for Recessive RP is Underway

Previous Foundation-funded projects helped make possible the Phase I human study, which is taking place in Saudi Arabia.

Scientists Report Outstanding Progress in FFB-Funded Translational Research

An international team of retinal researchers funded by the Foundation's TRAP program report excellent progress in moving their emerging treatments and cures – including gene therapies, stem cell treatments and pharmacological compounds – toward human studies.

Genable Receives $6.8 Million Investment to Develop Gene Therapy for Dominant RP

Building on previous Foundation funding, the company is planning a clinical trial aimed at treating a form of the disease that’s affected families for generations.

Argus II Retinal Prosthesis Implanted for the First Time Commercially in Europe

Several months after winning marketing approval in Europe, the Argus II, a prosthetic device known as “the bionic retina,” is implanted for the first time commercially in Italy

Researchers Discover Retinal Disease in Which New Photoreceptors are Generated
A Foundation-funded researcher team has discovered a canine retinal disease with an amazing, unexpected effect. While the condition acts like a typical retinal disease and causes the loss of photoreceptors, it also leads to the proliferation of new ones.

Gene Therapy Revives Cones Long After They Stop Working

If you could do one thing for a person losing their vision to a retinal disease, saving their cones would be near the top of the list. That’s because cones are the retinal cells that allow people to see color and fine detail, enabling them to drive, read and see the faces of loved ones.

QLT Receives FDA Fast Track Designations for RP and LCA Treatment

September 14, 2011 - The FDA has granted the biopharmaceutical company QLT with Fast Track Designations for its synthetic retinoid treatment for people with Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) caused by variations in the RPE65 and LRAT genes. Individuals with these genetic variations do not produce a retinoid critical for vision; QLT’s treatment serves as a replacement for that missing retinoid.

Foundation Receives $300K Grant for Recessive RP, LCA Gene Therapy Research
August 29, 2011 - The Foundation Fighting Blindness announces a $300,000 grant from the M. House Family Fund to support gene therapy research related to autosomal recessive retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). The funding will span three years to help researchers develop an AAV gene therapy treatment that can safely and effectively deliver a healthy gene to the inner cells in the retina that are affected by variations in the CRB1 gene.

Newly Found Gene is Common Cause of RP in People of Jewish Descent

August 11, 2011 - A Foundation-funded research team, led by the University of Iowa, has identified a defect in the gene MAK as a prevalent cause of autosomal recessive retinitis pigmentosa (arRP) in people of Ashkenazi (Eastern European) Jewish descent.

Foundation Fighting Blindness to Fund Nine New Research Grants

July 28, 2011 - The Foundation Fighting Blindness has announced funding for nine new 3-year grants in support of a wide range of innovative retinal research projects including the development of: enhanced genetic screening strategies, new gene therapies, vision-preserving drugs, and advanced retinal imaging techniques.

Looking for Genes in All the Right Places

July 20, 2011 - An innovative, targeted search technique known as whole-exome sequencing is dramatically reducing the time, effort and expense it takes to discover genetic defects that cause retinal disease. While the increasing raw power of genetic-screening technologies is continually accelerating the pace of gene discoveries, the whole-exome search strategy is a major leap forward.

Gene Discovery Enhanced by Quickly Advancing Technologies

Thanks to accelerating advances in computing power and lab instruments, discovering the genetic defect that causes a person’s retinal degenerative disease is continually getting quicker and less expensive.

Sharing With Congress How High-Tech Drug Screening Speeds Research

 

One reason it takes so long to develop a drug that serves as either a treatment or a cure for a specific disease is that it often takes researchers years, even decades, to screen the innumerable compounds that go into making the drug. Or at least it used to. These days, thanks to recent advances in computer, robotic and data-processing technologies, such tests can take only weeks, as long as researchers have the right resources, which can be very costly and difficult to access.

Harnessing Inner Retinal Cells to Restore Vision in Advanced Disease

Dr. John Flannery, a Foundation-funded researcher, recently restored some visual ability in mice with retinitis pigmentosa by injecting a gene therapy that helps make up for the loss of photoreceptors.

Faster-Acting, Longer-Lasting Gene Therapy for RP Shows Promise

Long before the launch of gene therapy studies that have restored vision for patients with Leber congenital amaurosis (LCA), Foundation-funded researchers were hard at work refining their gene delivery technologies for future therapies to treat a broad spectrum of retinal degenerative diseases. They recognized that for certain retinal conditions, they needed to develop gene delivery technologies that: 1) worked more quickly; 2) provided a more sustained effect; and 3) worked more efficiently and effectively in photoreceptors.

Emerging Treatment “Gobbles Up” Culprit in Many Retinal Diseases

Foundation-funded researchers are developing an elegant approach to wiping out the toxic, vision-robbing molecules common to many retinal degenerative diseases. They hope to turn their emerging research into a cross-cutting treatment that has the potential to preserve vision in people with a broad range of retinal conditions.

Researchers Take another Critical Step toward Using Skin Cells to Treat Retinal Disease

A research team funded by the Foundation Fighting Blindness used an innovative repair technique to correct the disease-causing genetic defect in stem cells derived from a person’s skin — stem cells that hold the potential to treat their retinal degenerative condition.

Researchers Use Stem Cells to Build New Retina

A research team from Kobe, Japan has taken a major step forward in developing a functional retina from embryonic stem cells.

Gene Therapy Preserves Vision in Lab Study of Bardet-Biedl Syndrome

In a groundbreaking effort funded in part by the Foundation Fighting Blindness, scientists at Baylor College of Medicine used gene therapy to preserve vision in mice affected with a form of Bardet-Biedl syndrome (BBS), a devastating, multi-faceted disorder that causes significant vision loss from retinitis pigmentosa (RP). This research advancement represents the greatest success thus far in the development of a treatment for RP associated with BBS.

A Valuable Opportunity Exists in Federally Funded Research
An opinion editorial, penned by Foundation chief science staff members Drs. Steven Bramer and Stephen Rose, advocating for increased federal support of embryonic stem cell research was published on The Hill’s Congress Blog. Embryonic stem cells hold incredible promise for the development of treatments for all retinal degenerative diseases and the furthering of other areas in retinal research.

UPDATED: Participants Recruited for Valproic Acid Clinical Trial
The National Eye Evaluation and Research network (NEER), the clinical trial network of the Foundation Fighting Blindness, is now recruiting participants for a Phase II clinical trial of valproic acid to evaluate the drug’s effectiveness in slowing vision loss for people with autosomal dominant forms of retinitis pigmentosa (adRP).
The X-Link Factor
Consortium Makes Major Strides in the Development of Treatments for X-Linked RP

After three years of intensive research on multiple fronts, a consortium funded by the Foundation Fighting Blindness is reporting significant progress in the development of vision-saving  treatments for X-linked retinitis pigmentosa (XLRP).
The Three Major Inheritance Patterns
Autosomal Recessive, Autosomal Dominant, and X-Linked
Most retinal degenerative diseases — including retinitis pigmentosa, Stargardt disease, and Usher syndrome — are inherited. They are caused primarily by genetic variations that are passed down from one or both parents, through one of the three inheritance patterns reviewed in this article. (The following discussion on inheritance patterns is not relevant for age-related macular degeneration (AMD), because risk of AMD is associated with a complex combination of genetic and lifestyle factors.)
Clinical Trials are Expensive
Labor Intensive Clinical Trials Require Time, Effort and Financial Resources

We are no doubt in an exciting time as more clinical trials of promising treatments come to fruition. But as we have been reporting in recent years, clinical research brings a whole new financial challenge to the Foundation and our supporters. Human studies cost several million dollars each and are many times more expensive than laboratory studies.
Pivotal Advancement in Gene Therapy for Dominant RP

Genable, a Foundation-funded biotechnology company in Ireland, has used an innovative gene therapy to slow the progression of vision loss in mice affected by a dominant form of retinitis pigmentosa. The company is working toward a study of the treatment in larger animal models with the goal of launching a clinical trial thereafter.

Q&A with Dr. Steve Bramer: A Pivotal Role
Foundation Brings Knowledge and Expertise to Clinical Studies

Potential RP Treatment Based on Ancient Chinese Medicine
The Foundation Fighting Blindness is working toward launching a clinical trial in 2011 of a vision-preserving agent known as TUDCA. Based on a traditional Chinese medicine, TUDCA is a synthetically manufactured bile acid that has demonstrated safety and efficacy in animal models of retinal degeneration. Foundation-funded animal studies are taking place at the Oregon Health & Science University to determine optimal TUDCA dosing for humans.

Stem Cell Research: Recent Legal Decisions and Unauthorized Treatments

Update, September 9, 2010: A panel of the U.S. Court of Appeals ruled that the federal government, including the NIH, can resume funding of embryonic stem cell research while the court reviews the original order that banned funding.

On August 23, national media began focusing much attention on embryonic stem cells, because a judge from the U.S. District Court in Washington, D.C. issued a preliminary injunction banning federal funding for virtually all embryonic stem cell research.

FDA-Approved Drug Moving into Clinical Trial for Treatment of RP
Valproic acid, a drug which has shown promise for preserving vision in people affected by autosomal dominant forms of retinitis pigmentosa (adRP), is moving into a Foundation-funded multicenter clinical trial this fall. 

Gene Therapy Reactivates Retina, Restores Vision

An international research team, funded in part by the Foundation Fighting Blindness, has used gene therapy to reactivate retinal cells that were previously unresponsive to light. The treatment was delivered to cones, the retinal cells that provide central and daytime vision. Investigators evaluated the gene therapy in two mouse models of retinal degenerative disease, as well as cultures of human retinal tissue. Using a variety of tests, the researchers demonstrated that the treated cells restored functional vision, and that the restoration of vision persisted over a long period of time.

French Researchers Planning Clinical Study of Vision-Preserving Protein

A promising treatment aimed at preserving cones, the retinal cells that provide central and daytime vision, is poised to move into a Phase I clinical trial within the next year. Known as rod-derived cone viability factor (RdCVF), the therapeutic protein has consistently preserved vision in several preclinical studies.

Congressional Briefing Highlights Research Funding Need for Artificial Retina
Nearly 25 years ago, when Dr. Mark Humayun first envisioned the development of a bionic retina to enable the blind to see, scientific pundits equated the undertaking to a “moon shot,” implying that the project might be overly ambitious. However, thanks to sustained funding and innovative engineering by a team of engineers, scientists, and clinicians led by Dr. Humayun, 38 people with advanced retinitis pigmentosa, a vision-robbing retinal degenerative disease, are now successfully using implanted artificial retinas. Many of these individuals are participating in a clinical evaluation of the device led by Second Sight Medical Products, Inc.

New Study Paves Way for Treating Second Eye in Gene Therapy Clinical Trial
All 12 participants in the gene therapy clinical trial for Leber congenital amaurosis (LCA) at the Children’s Hospital of Philadelphia (CHOP) have been so delighted with the vision restoration from treatment to one eye, they have been anxiously awaiting the go-ahead to have their second eye treated. Thanks to results from a Foundation-funded lab study in large animals, that will likely happen soon.

Why It’s Important to Stay in Touch with Your Retinal Specialist

For many years, people with retinal degenerative diseases such as retinitis pigmentosa (RP), Usher syndrome, and Stargardt disease have been frequently told by their eye care professionals that “there’s nothing we can do.”

Researchers funded by the Foundation Fighting Blindness are moving promising treatments and cures into human studies, and in the not-too-distant future, there are likely to be therapies that will enable eye care professionals to better treat many retinal degenerative diseases.

Is a Genetic Test Right for You?

If you have an inherited retinal degenerative condition such as retinitis pigmentosa, Usher syndrome or Stargardt disease, should you get genetically tested? Is there a benefit in trying to determine the genetic variation that is causing your condition?

Gene Therapy Clinical Trial for Retinitis Pigmentosa
Researchers from the United States, China, and Saudi Arabia are planning to launch a gene therapy clinical trial in 2010 for a recessive form of retinitis pigmentosa caused by variations in the gene MERTK.

Latest Update: Gene Therapy Restores Significant Vision to Children with LCA
Gene therapy has taken another major step forward as a treatment for retinal diseases. Researchers conducting the landmark gene therapy clinical trial at The Children’s Hospital of Philadelphia (CHOP) have just reported that 12 people with Leber congenital amaurosis have now been successfully treated and are experiencing sustained, and in many cases significant, improvement in their vision.

Latest Update: Childhood Rediscovered: Gene Therapy Restores Vision for 9-year old with LCA
When it was his turn to take the stage at a press conference at The Children’s Hospital of Philadelphia (CHOP), Corey Haas got up from his seat in the auditorium and walked effortlessly up the four steps in front of him. Without hesitation, he crossed the stage, stepped around the table and took his seat next to Dr. Jean Bennett. His white cane was nowhere to be seen.

Saving Vision with Skin Cells
Thanks to Foundation-funded researchers at the University of Wisconsin-Madison, skin cells might someday restore vision lost to retinal degenerative diseases.

Vision Improvements from Gene Therapy Persist After One Year
Researchers from the Universities of Pennsylvania and Florida have reported that vision improvement for three individuals treated with gene therapy in a Phase I clinical trial has persisted for one year. The trial participants, all in their early 20s and with severe vision loss from Leber congenital amaurosis (LCA), continue to experience significant increases in night and day vision. Most importantly, the safety of the treatment, which is the primary goal of this and all Phase I clinical trials, is now extended to one year.

International Team Planning New Gene Therapy Clinical Trial for RP
Researchers from the United States, China, and Saudi Arabia are planning to launch a gene therapy clinical trial in 2010 for a recessive form of retinitis pigmentosa caused by variations in the gene MERTK.

Doheny Recognized for Development of Retinal Prosthesis
The Doheny Eye Institute at the Keck School of Medicine of the University of Southern California has won a prestigious 2009 R&D 100 Award for development of its retinal prosthesis, which gives people blind from retinitis pigmentosa the ability to identify shapes and objects, and navigate the environment. The award is given by R&D Magazine in recognition of technologically significant products.

Q&A on Modifier Genes with Dr. Stephen Daiger
Funded by the Foundation Fighting Blindness since 1985, Dr. Stephen Daiger, of the University of Texas—Houston, is a world-renowned expert in identifying and understanding the genes and related mutations that cause retinal degenerative diseases.

A Vision for Gene Therapy
FFB Partnership Accelerates Advancement of Gene Therapies into the Clinic

A decade ago, five scientific leaders with expertise in gene therapy formed a company to commercialize their promising innovations for the treatment of incurable genetic diseases. They named the company Applied Genetic Technologies Corporation, with their acronym, AGTC, cleverly representing the four organic compounds — adenine, guanine, thymine, and cytosine — that make up the genetic code found in virtually every cell on the planet.

FFB Partnering with AGTC to Accelerate Advancement of Gene Therapies into the Clinic
The Foundation Fighting Blindness (FFB) has announced that it is collaborating with Applied Genetic Technologies Corporation (AGTC), a leading gene therapy development company, to advance gene therapies for retinal degenerative diseases into clinical trials.

Neurotech Treatment Shows Biological Effect in RP Clinical Studies
Neurotech’s innovative treatment employing Encapsulated Cell Technology (ECT) demonstrated a biological effect after 12 months for people with retinitis pigmentosa (RP), Usher syndrome, and choroideremia participating in two Phase II/III clinical trials. Both trials are being funded in-part by the Foundation Fighting Blindness.

Promising Research Presented at 2009 ARVO Annual Meeting
Every year, thousands of eye researchers from around the world convene at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting to share the latest advances and breakthroughs in eye and vision research. ARVO’s Annual Meeting is regarded as the premier annual research conference for the ophthalmological community.

Dr. Dean Bok Receives Foundation Fighting Blindness Career Achievement Award
The Foundation Fighting Blindness honored Dr. Dean Bok, of the Jules Stein Eye Institute of UCLA, with the prestigious Llura Liggett Gund Award Career Achievement Award on May 7.  The highly coveted award was presented to Dr. Bok in recognition of his outstanding dedication and commitment to retinal science and his extraordinary career achievements that have significantly advanced the research and development of preventions, treatments and cures for retinal degenerative diseases that affect more than 10 million Americans. Dr. Bok received the award before an audience of his peers at a Foundation dinner following the Association for Research in Vision and Ophthalmology Annual Meeting in Ft. Lauderdale.

Second Sight Recruiting More Participants for U.S. Retinal Prosthesis Study

Retinal implant offers partially restored vision for people with retinitis pigmentosa

*This product now has approval. Click here for an update.

Second Sight Medical Products, Inc., developer of the retinal prosthesis known as the Argus II, is seeking 10 additional participants for its feasibility clinical study in the United States.  A total of 22 people have already participated in the three-year study, which is underway in the U.S., Europe, and Mexico.

Emerging Treatment Stabilizes Vision in People with Dry AMD

Owings Mills, MD - March 26, 2009 — An innovative technology, employing a tiny capsule implanted in the eye, is stabilizing vision in people suffering from dry age-related macular degeneration (AMD). Encapsulated Cell Technology (ECT), developed by Rhode Island-based Neurotech, preserved vision in a majority of the 51 people who participated in a Phase II clinical trial.

Foundation Appoints Four New National Trustees to Advance Mission

The Foundation Fighting Blindness has appointed four new national trustees to further advance the organization’s fundraising, organizational development, and volunteer recruitment efforts.

2009 Day of Science: Cures Coming into Sight

Highlights from the 2009 Day of Science Meeting Hosted by the Foundation Fighting Blindness

Dr. Al Maguire captivated the Day of Science audience when he played a video of an eight-year-old boy, who after being treated with a landmark gene therapy, was able to quickly navigate an obstacle course he was previously unable to follow. The audience erupted in loud applause when Dr. Maguire added, “That boy has put away his blind cane and is now enrolled in a mainstream school.”

Retinal Nourishment is Key in Preventing Vision Loss
Nourish the retina. That's the concept behind two newly identified strategies for keeping the retina healthy and preventing vision loss. They were the findings of Foundation-funded researcher Constance Cepko, Ph.D., of Harvard Medical School, who studies the degeneration of photoreceptors in animal models of retinal disease.

Partnership Aims at Treatment for Dominant RP

FFB is partnering with the Irish biopharmaceutical company Genable to develop an innovative gene therapy for people affected with autosomal dominant forms of retinitis pigmentosa or adRP.

Gene Therapy Shows Promise for Curing Day Blindness

A new advance in gene therapy is providing hope for people with a debilitating retinal disease known as achromatopsia or day blindness. Researchers from the University of Pennsylvania have used gene therapy to successfully treat dogs with the same condition.

Light of Day

Gene Therapy Shows Promise for Curing Day Blindness

A new advance in gene therapy is providing hope for people with a debilitating retinal disease known as achromatopsia or day blindness. Researchers from the University of Pennsylvania have used gene therapy to successfully treat dogs with the same condition.

Two New Research Collaborations to Evaluate Potential Retinal Treatments

The National Neurovision Research Institute (NNRI), the clinical support organization of the Foundation Fighting Blindness, recently established research collaborations with two pharmaceutical companies to evaluate potential treatments for retinal degenerative diseases.

Neurotech Capsule Receives Fast Track Status from FDA

Neurotech’s innovative encapsulated cell technology (ECT), which shows promise for treating a wide range of retinal diseases, has received Fast Track status from the FDA. Fast Track status is given to therapies that address an unmet medical need. Also, the FDA will review an application for marketing approval of a Fast Track treatment within six months. Neurotech plans to release results from their three Phase II/III clinical trials in spring 2009. At that point the company may seek FDA marketing approval for the treatment to make it generally available and/or conduct additional clinical studies.

Emerging Treatment Provides Crucial Biochemical for Restoring Vision
People with certain forms of Leber congenital amaurosis and retinitis pigmentosa have new hope for vision restoration thanks to a treatment being developed by QLT, Inc., a biopharmaceutical company in Vancouver, Canada.
Results from Universities of Pennsylvania and Florida Study Reaffirm LCA Gene Therapy Potential

Three young adults with Leber congenital amaurosis (LCA) receiving gene therapy in a Phase I clinical study at the University of Florida and University of Pennsylvania demonstrated improved vision in brightly and dimly lit settings. The gene therapy was also shown to be safe for all three participants, who were ages 21-24.

Initial results of the study were published today in an online edition of the journal Human Gene Therapy. Additional findings from the study will be published shortly in the Proceedings of the National Academy of Sciences.

Glaucoma drug brimonidine moving into clinical trials for RP, AMD

Allergan is launching two clinical trials to evaluate the safety and effectiveness of the drug brimonidine in slowing the progression of retinitis pigmentosa and the dry form of age-related macular degeneration (AMD).

Hill Briefing Highlights Research Effort that is Helping the Blind to See

Two of the world’s top retinal researchers came to Capitol Hill on June 24 to educate congressional staff about their landmark clinical trial in which they used gene therapy to restore some vision in three young adults who were virtually blind from a severe form of retinitis pigmentosa known as Leber congenital amaurosis (LCA). Seventy people attended the briefing, which was hosted by the Alliance for Eye and Vision Research and the Foundation Fighting Blindness.

New Model of Artificial Retina Moves into Human Studies

For people with the most advanced retinal degenerative disease - those who are blind or have only light perception - artificial retinas are providing promise for restored vision.

Second Sight® Medical Products is now conducting a Phase II human study of its second-generation artificial retina - the Argus II - in clinical centers in the U.S., Mexico, and Europe (England, France, and Switzerland).

Congressional Briefing Underscores the Importance of Vision Research Funding
Top leaders from the vision research community came to Capitol Hill on October 31 to brief 70 congressional staff and guests about the critical need for research funding to eradicate vision-robbing retinal diseases that affect more than nine million Americans. Representatives from the Foundation Fighting Blindness, National Eye Institute and Alliance for Eye and Vision Research held the briefing to highlight the public importance of funding for eye research.
Now They See - Breaking News from the Foundation Fighting Blindness

Three young adults with virtually no vision can now read several lines on an eye chart and see better in dimly lit settings thanks to an innovative gene therapy aiming to reverse blindness in a severe form of retinitis pigmentosa known as Leber congenital amaurosis or LCA. One person was even able to better navigate an obstacle course several weeks after receiving the therapy.

Paris Research Center Focused on Moving Promising Treatment into Clinical Trials

In 2005, French researchers José Sahel, M.D., and Thierry Léveillard, Ph.D., won the Foundation’s Annual Trustee Award for the most promising advance of the year: Identifying a protein that shows great potential for preserving vision in people affected by a variety of retinal degenerative diseases. Sahel and Léveillard, both with the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris, are now part of a new FFB-funded research center chartered with moving this promising protein into clinical trials.

Nanoparticles Show Promise for Delivering Vision-Restoring Genes to the Retina
Nanoparticles are a rapidly emerging technology that is appearing in emerging treatments for retinal diseases, cancer, HIV, and a variety of other conditions. These microscopic particles — which are like tiny rocks as small as one fifty-thousandth of an inch — are uniquely able to penetrate cells and tissue for the delivery of therapeutic genes and molecules.
FFB Receives $1 Million Gift to Advance Research for XLRP

Thanks to a $1 million gift to the Foundation Fighting Blindness, researchers are one step closer to curing a severe, vision-robbing retinal disease known as X-linked retinitis pigmentosa (XLRP).

Genetic Breakthrough Gives New Hope to Family’s Fight Against Blindness

Shortly after Alan Brint was born in 1997, his parents David and Betsy realized that his vision was not right. They were devastated to learn from retinal experts that young Alan was nearly blind because of a genetic disease called Leber congenital amaurosis (LCA). “It’s every parents’ worst nightmare,” says David, “to find out that something is tremendously wrong with your child. Blindness never even occured to us until the doctor said, “Your child is blind and there is nothing we can do at this time.”

Human Neural Stem Cells Show Potential for Vision Rescue

An FFB-funded research team from the University of Wisconsin and the University of Utah used human neural stem cells — cells derived from the brain — to rescue vision in a rodent model of retinal degenerative disease.

Potential Cure for Blind Children Moves into a Human Study

Children born blind from a devastating retinal condition called Leber congenital amaurosis (LCA) may soon be able to see thanks to an innovative gene replacement therapy that has just moved into a clinical trial.

Umbilical Cord Tissue Shows Promise as Retinal Disease Treatment

The umbilical cord is not only the lifeline for a developing baby, but it might also save sight for people affected by a variety of retinal degenerative diseases.

Drug Reduces Swelling of Central Retina in Patients with RP

Cystoid macula edema (CME) is a complication in approximately 10-15 percent of people affected by retinitis pigmentosa (RP). CME can cause increased vision loss and retinal deterioration beyond what occurs as a result of RP.

Neurotech begins Phase II/III human studies of treatment for retinitis pigmentosa

Neurotech Pharmaceuticals, Inc. has begun enrollment of participants in two multicenter Phase II/III human clinical trials for an innovative treatment to slow the progression of a group of blinding diseases known as retinitis pigmentosa. Both studies are partially funded by the Foundation Fighting Blindness.

DHA to be Studied as Treatment for X-Linked RP

An FFB-funded investigative team from the Retina Foundation of the Southwest is conducting a Phase II clinical trial of docosahexaenoic acid (DHA) for the treatment of X-linked retinitis pigmentosa (XLRP).

Dr. Matthew LaVail Receives Llura Liggett Gund Award for Excellence in Retinal Degeneration Research

Dr. Matthew LaVail was awarded the Llura Liggett Gund Award—the most prestigious honor awarded by The Foundation Fighting Blindness (FFB)—for lifetime achievement in retinal disease research.

Moderate light exposure causes retinal damage in dogs with RP gene

FFB-funded researchers from the University of Pennsylvania, along with investigators from Cornell University, have discovered that dogs with mutations in their rhodopsin gene experienced retinal damage when they were exposed to moderate amounts of light.

Retinal Chip Study Expanded

A 10-person evaluation of the Artificial Silicon Retina (ASR) microchip, developed by Optobionics (Naperville, Illinois), is being expanded to 20 patients at three sites: Johns Hopkins School of Medicine (Wilmer Eye Institute), Emory University School of Medicine/Atlanta Veterans Administration Medical Center, and Rush University Medical Center.

Genetics in RP and Retinal Degenerative Diseases: Discoveries, Breakthroughs, and Challenges

During the 1970s, when the Foundation first began funding retinal disease research, scientists suspected that genetics played a key role in the development of many retinal degenerative diseases (RDDs). In the early 1980s, researchers reported the first gene linkage to an RDD. And, in the late 1980s, the first RDD gene, rhodopsin, was discovered. Today, thanks to significant financial investment, hard work, and innovations in genetic technologies, 158 RDD genes have been identified. Most excitingly, as a result of the highly successful, sight-giving gene therapy tested on 40 blind dogs- one of whom was the famous Briard named Lancelot- we are about to embark on the first human trial of gene therapy to treat the RDD known as Leber's congenital amaurosis (LCA). This landmark Phase I clinical trial is scheduled to begin in fall 2005. FFB is delighted by the significant progress we've helped make possible through genetic research funding- an endeavor that is critical to the treatment and cure of RDDs.

FFB Research Center: University of Iowa’s Genetic Testing Initiative will Bolster Progress in Treating RDDs

Ed Stone, Val Sheffield, Tom Casavant, and their colleagues at the University of Iowa’s Center for Macular Degeneration and Allied Retinal Diseases see no reason why there shouldn’t be cost-effective, efficient, and accurate genetic testing available for every person who has an inherited retinal degenerative disease (RDD). They’re certain that more and better treatments will be attainable, if genetic testing — and the information gleaned from it — is more accessible to researchers, ophthalmologists, and patients. With support from The Foundation Fighting Blindness and other organizations, they are well poised to make this plan for ubiquitous testing a reality.

 
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