Degeneración macular relacionada con la edad

Kapil Bharti, PhD, a senior investigator and director of the Intramural Research Program at the National Eye Institute (NEI), talks with host Ben Shaberman about his clinical trial underway for an innovative cell therapy for geographic atrophy (advanced dry age-related macular degeneration) as well as a clinical trial of the drug metformin for Stargardt disease.

Hema Ramkumar, MD, a retinal specialist, surgeon, and founder of the company Oculogenex, talks with host Ben Shaberman about her emerging therapy that targets mitchondria to slow the progression of intermediate dry age-related macular degeneration and potentially other retinal diseases. She also discusses her experiment that will put mice in space so researchers can gain a better understanding of retinal degeneration.

The free, online event will feature a lecture from David Boyer, MD, a Senior Partner at Retina-Vitreous Associates Medical Group and dry AMD expert.

The investment supports the clinical development of two novel programs, both with the aim of being protective against vision loss.

On World Sight Day 2022, Retina International is presenting data from a report on its study into the Socio-economic Impact of late-stage age-related macular degeneration (AMD) in Bulgaria, Germany, and USA.

The Foundation Fighting Blindness is pleased to provide an audio recording and full transcript of the Insights Forum, our quarterly conference call providing updates to the inherited retinal disease community. The call took place on July 30, 2020.

The Foundation Fighting Blindness is closely monitoring the COVID-19 situation and its impact on the IRD community.

The Foundation Fighting Blindness is pleased to provide an audio recording and full transcripts of the Insights Forum, our quarterly conference call providing updates to the inherited retinal disease community. The call took place on January 31, 2020.

My Retina Tracker Program is the highest volume IRD genetic testing program in the U.S.

The Foundation Fighting Blindness is pleased to provide an audio recording and full transcript of the Insights Forum, our quarterly conference call providing updates to the inherited retinal disease community. The call took place on October 30, 2019.

The program will offer patients with inherited retinal disease no-cost genetic testing and genetic counseling in the United States. Look for updated information on how to participate to be posted in mid-October, with program registration starting shortly thereafter.

The newly funded research efforts include several therapies that have strong potential to treat a wide range of inherited retinal diseases.

The results of the LADDER trial demonstrate a major step forward but introduce many questions.

After a series of failed trials, current research suggests that the next generation of gene therapies for wet age-related macular degeneration holds promise. Learn how investigators are inducing the body’s cells to administer their own therapeutic response to disease activity.

Bipartisan Bill Will Stimulate Up to $1 Billion in New Funding for Blindness Research

Age-related macular degeneration (AMD) is the leading cause of blindness for people over 50 years of age in developed countries.

Bill Introduced in U.S. House Would Speed Up Cures for Blindness

The Foundation Fighting Blindness (the Foundation) and CheckedUp have formed a collaborative partnership to deliver patient-friendly diagnostic and disease-management information to people with retinal diseases such as age-related macular degeneration, retinitis pigmentosa, and Stargardt disease during their visits to eye doctors.

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.

Recent developments in research on age-related macular degeneration.

New, higher treatment dose reduces frequency of eye injections for patients

In two Phase 3 clinical trials, IZERVAY slowed the growth rate of lesions, the regions of cell loss in the central retina, associated with geographic atrophy (GA)

The company’s emerging, oral treatment is designed to slow vision loss

An emerging XLRP gene therapy acquired from AGTC is the company’s lead clinical program

Geographic atrophy causes significant central vision loss and remains an unmet medical need.

Geographic atrophy (GA) is the advanced form of dry age-related macular degeneration (AMD) which causes blindness in one million people in the United States and 5 million worldwide.

This is the first time that a treatment has been approved for this leading cause of blindness.

The company plans to seek FDA approval for the emerging therapy in first quarter of 2023

There are currently no FDA-approved therapies for geographic atrophy (GA), the advanced form of dry AMD.

The device is designed for people with late-stage AMD who have no treatment options

This new treatment can reduce the frequency of injections into the eye

The Foundation Fighting Blindness is funding Bärb Rohrer, PhD, Medical University of South Carolina, to evaluate safety and efficacy of the gene therapy in a large animal model

Known as APL-2, the drug met its primary endpoint in the OAKS clinical trial but not the DERBY trial

Early, encouraging results from two human studies — trials launched by Bionic Sight and GenSight — are putting optogenetic therapies in the spotlight for patients with advanced vision loss from retinal conditions.

Projects target a variety of conditions including: age-related macular degeneration, Stargardt disease, retinitis pigmentosa, and Usher syndrome type 3A

Company will seek marketing approval for the emerging therapy

In a Phase ½ clinical trial, the emerging treatment reduced the burden of regular anti-VEGF injections

Tiny implant provides continual delivery of anti-VEGF treatment for six months

The gene therapy is designed to reduce the treatment burden for patients

A healthy diet is also important for people with retinal conditions

The benefits of genetic testing for IRD patients, how to participate in the Foundation’s Open Access program, and what to expect from the genetic testing process.

Currently there are no treatments for Advanced Dry AMD, also known as GA, which can lead to significant central vision loss

Dual-vector delivery system designed to deliver the large ABCA4 gene

Zimura inhibits a complement protein known as C5, a component of the immune system that, when overactive, can cause retinal degeneration.

Beovu from Novartis reduces treatment burden for wet AMD patients

Retinal research paves the way for new treatments for the entire neurological system.

Dr. Zack is a member of the Foundation’s Scientific Advisory Board and chairs its Cellular Molecular Mechanisms of Disease study section.

Eye Bonds provide the opportunity to advance, and accelerate development for, more promising treatments into and through clinical trials and out to the people who need them.

A video from a clinical trial of robot-assisted surgery demonstrates the potential benefits of robot-assisted subretinal injections.

Unlike current treatments requiring multiple injections, REGENXBIO’s gene therapy is administered as a one-time subretinal injection to the affected eye.

Gyroscope Therapeutics, a gene-therapy development company in the UK, has launched the first gene therapy clinical trial for the dry form of age-related macular degeneration (AMD).

Sofia Sees Hope, a nonprofit dedicated to finding treatments and cures for people with Leber congenital amaurosis (LCA) and other inherited retinal diseases (IRDs), has made a $100,000 donation to the Foundation Fighting Blindness to support therapy development and genetic testing.

The French bioelectronics company Pixium Vision has reported that its PRIMA bionic vision system has restored some central vision in patients with advanced dry age-related macular degeneration (AMD) participating in a clinical feasibility trial.

The Seattle biotech Acucela is now enrolling participants in its Phase 3 clinical trial for emixustat hydrochloride, an emerging oral drug for slowing vision loss in people with Stargardt disease, an inherited form of a macular degeneration.

Since 1989 genetic researchers, many funded by the Foundation, have identified approximately 270 genes linked to IRDs. In most cases, defects in a single gene can cause a retinal disease and vision loss.

Trial participants will receive intravitreal injections of APL-2, or a sham procedure (placebo), monthly or every other month. The injections are made into the vitreous, the soft gel in the middle of the eye.

On September 4, 2018, seven researchers, including six previously funded by the Foundation, were recognized with the prestigious 2018 Antonio Champalimaud Vision Award for their contributions to the advancement of blindness-reversing RPE65 gene therapies.

The company is taking on a multi-track strategy that includes retinal gene-therapy development, including delivery of over-sized genes and design of a two-step process of gene knockdown and replacement for autosomal dominant conditions.

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.

Seventy scientists submitted requests for funding.

Call to Action: Ask Congress to Support $1 Billion in Eye Research

Many research groups from around the world are investigating ways to create new photoreceptors from stem cells for transplantation into the retina for vision restoration.

Watch recorded sessions from VISIONS2018.

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.

ARVO 2018: Dr. Stephen Daiger Reports on the State of Genetic Testing 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.

Several research studies have suggested that the Mediterranean diet (MeDi) - which is rich in fruits, vegetables, legumes, cereals, and fish - may be good for your health, including lowering your risk of an early visit from the grim reaper.

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.

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 I/IIa clinical trial for its therapy – a patch comprised of a layer of retinal pigment epithelial (RPE) cells on a synthetic scaffold.

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.

The 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.

An exciting year in fighting blindness.

Known as LUXTURNA™ (voretigene neparvovec), the gene therapy restored vision in a clinical trial for people between the ages of 4 and 44 with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65.

If a clinic is charging for a stem-cell treatment or procedure for an IRD, it is probably not legit. The expense to the patient is a major red flag.

An advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval.

In addition to funding promising biotech start-ups, the Foundation Fighting Blindness has played a critical role in developing research talent.

The high-tech, vision-restoring system interfaces with the visual cortex, the back of the brain where visual input is processed to create the images we see.

The partnership will help companies and researchers quickly obtain and implement high-quality vectors for their retinal gene-therapy development efforts.

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.

In its fourth year, the meeting is becoming the world’s most comprehensive overview of the promising research underway for emerging IRD treatments.

How the Foundation Fighting Blindness (FFB) provided timely funding of $155,000 to help a lab at the University of California, San Diego (UCSD), leverage a $2 million retinal-gene discovery project.

Dr. Berson dedicated himself to clinical care and vision-saving research for people with inherited retinal diseases for five decades.

“…participation in a study for an emerging therapy that is not regulated by the FDA or another well-recognized regulatory agency like the European Medicines Agency in Europe, is fraught with dangers and can lead to unexpected serious consequences.”

Company Builds on FFB’s Initial Investment to Garner $265 Million in Therapy Development Funding

Improved outcome measures will make clinical trials for degenerative retinal diseases — including age-related macular degeneration (AMD), the world’s leading cause of blindness in seniors, and inherited retinal conditions such as RP and Stargardt disease — less expensive to conduct and able to deliver more precise results.

Understanding the pathways of the retinal neural network — and how they are rewired with aging and disease — is helpful in trying to save and restore vision.

Dr. Shinya Yamanka discussed his early clinical trial for iPSC-derived retinal pigment epithelial (RPE) cells for a 78-year-old woman with advanced wet age-related macular degeneration (AMD).

The complex and elusive nature of these conditions can also extend to the way they are passed down in families, making diagnosis and prognosis quite challenging.

RetroSense’s optogenetic therapy is designed to restore vision to people who are completely blind from retinal degenerative diseases such as retinitis pigmentosa by bestowing light sensitivity to retinal ganglion cells, which survive after photoreceptors, the cells that make vision possible, are lost.

Both approaches show strong, near-term potential for providing meaningful vision to people who are otherwise blind from retinal diseases such as retinitis pigmentosa and age-related macular degeneration (AMD).

At VISIONS 2016, FFB’s national conference, the Foundation honored him with its Ed Gollob Board of Directors Award for breakthrough research conducted within the past year.

Dr. Weleber became the 10th recipient of the Foundation’s highest honor, named after FFB co-founder Lulie Gund, during the opening lunch of the VISIONS 2016 conference.

Subretinal injection is the most common form of delivery for gene therapies currently in clinical trials.

Researchers from the University of Cologne in Germany and Radboud University in the Netherlands found that increased sunlight exposure during working life significantly increases risk of AMD later in life.

A pioneering, 80-year-old named Ray Flynn is the first AMD patient to receive the Argus II, and he is already reporting that he can see things better in his garden.

For those of us supporting the drive for vision-saving treatments and cures, he’s exactly the type of person we want on our team.

Dr. Boye received the Foundation’s Board of Director’s Award, which was presented at VISIONS 2015, FFB’s annual conference, for achievements in retinal research.

The mission of the program—to regenerate the neurons and neural connections in the eye and visual system—is synonymous with the Foundation’s mission to eradicate retinal diseases.

One of the hot topics at ARVO this year is a rapidly advancing gene-therapy approach called clustered regularly interspaced short palindromic repeats, or CRISPR.

An innovative genetic-engineering approach called “directed evolution” to find optimal gene-delivery systems based on adeno-associated viruses (AAVs).

The powerful and secure system enables patients to keep track of their clinical care and vision changes. At the same time, it enables scientists to search the “de-identified” (i.e., anonymous) patient information to study conditions and identify targets for treatments, preventions and cures.

Dr. Boye and her research team received a $900,000 grant for a gene therapy project targeting Leber congenital amaurosis.

Simply put, they’re creating therapies that can save vision in as many people as possible, independent of the genetic cause of disease.

Non-24 is a very rare condition affecting many (but not all) people who are totally blind and have absolutely no light perception. Their circadian clocks become out of sync as a result.

The French retinal implant developer Pixium quietly launched a clinical trial for its Intelligent Retinal Implant System 1 (IRIS1) in France, Austria and Germany.

Identifying the genes and proteins that play a major role in retinal health and vision is an important step in finding preventions and cures for degenerative diseases.

Many registries enable patients to collect and track information about their health, so they can take an active role in managing their care.

There’s hope for retinal regeneration for humans, thanks to Foundation-funded researcher Dr. Thomas Reh, who is investigating how to derive new photoreceptors from retinal cells called Muller glia.

The goal, “to regenerate the neurons and neural connections in the eye and visual system,” is exactly what people with retinal diseases need to save and restore their vision.

A profile on Dr. Robert Langer, a medical researcher who has received dozens of awards, accolades and honorary degrees, including, recently, FFB’s Visionary Award.

Sometimes, saving vision simply comes down to keeping retinal cells alive, or at least slowing their degeneration.

More good news about treatments and technological advances for restoring vision for people with retinal diseases.

Diseases such as age-related macular degeneration, cone-rod dystrophy and Stargardt disease cause scotomas, or blind spots, which often have devastating effects on central vision. They cause gaps in a person’s visual field, making it difficult to see words in a book, images on a computer monitor or TV and the features of someone’s face.

Great progress is being made in identifying targets for therapies, including using the body’s own protective systems to keep the photoreceptors alive.

Known as the implantable miniature telescope (IMT), the device is surgically placed in the eye, where it focuses images onto the area of the patient’s retina that is still functional.

How do you know if a treatment is legit? There should be preclinical and clinical trial data published in a peer-reviewed journal on research for the treatment.

Athletes around the world navigate the challenges of daily life and rigorous training, but what if a factor came into play that you had less control over? What if, one day, something changed in your vision?

Sean was diagnosed with dry age-related macular degeneration (AMD) in his 40s. Now Sean is using his AMD as an opportunity to educate others about the spectrum of blinding diseases while fundraising for the Foundation Fighting Blindness.

Sean Teare has completed a marathon and a triathlon as DIY fundraisers for the Foundation Fighting Blindness.

Acquisition significantly expands Janssen’s eye disease portfolio and strengthens its gene therapy capabilities. Late-stage AMD affects millions of people with no effective treatments currently available .

Al Linde has learned to “just roll with it” throughout his life to overcome many challenges.