May 30, 2024

Recording Available: Foundation Insights Forum – May 23, 2024

Insights Forum

The Foundation Fighting Blindness is pleased to provide a recording and full transcript of the Insights Forum, our quarterly conference call providing updates to the blinding diseases community. The call took place on May 23, 2024.

The guest speaker for this call included Rachelle Lin, OD, MS, FAAO, associate professor, Southern California College of Optometry at Marshall B. Ketchum University, where she teaches low vision rehabilitation and genetics.

Foundation Fighting Blindness
Insights Forum Transcript
May 23, 2024

Amanda Bement, Development Associate, Admin & Ops:

Thank you everyone for joining today's Insights Forum call. Before we get started, I would like to briefly review a few details for this call. Currently, all participant lines are muted and without video. Please be aware that the control bar is at the bottom of the Zoom window, but it may disappear when it is not in use. To prevent the control bar from hiding automatically, you can use the following keyboard shortcuts to always show the meeting controls. If you are using Windows commands, press the alt key, and if you are using a Mac keyboard, you can press command and backslash at the same time.

Today's presentation is being recorded and is available with closed captioning. To activate the closed captioning, you can select show captions in the control bar. Please note that on today's call, our speakers do have their video live. However, all of their comments will be provided verbally and there will be no slide. Throughout the call you'll be able to ask questions via the Q&A feature and the chat features, both of which are at the bottom of the Zoom window. We will address these questions towards the end of the call. If we do not get to your question live, we will follow up over the next week or so, so please do make sure to include your name with your question. You can also submit a question by sending an email to I would now like to turn the call over to our Chief Executive Officer, Jason Menzo. Jason.

Jason Menzo, Chief Executive Officer:

Very good. Thank you so much, Amanda, and good morning to everyone. Amanda lied just a second ago because she said that all of our speakers have their cameras on, but in fact, I do not have my camera on. We're having a technical glitch here today and it's just more proof as we've said in Insights Forums in the past that these are live not prerecorded and every once in a while things come up. But I am here live. I'm on my phone and my camera is off, but for those that have the Zoom interface, there's a picture of me so at least my smiling face is still on the interface, but it is all good.

We're super excited to have everyone here at our quarterly Insights Forum webcast. The purpose of these Forums is to provide our broader community with updates on strategic initiatives here at the Foundation Fighting Blindness and share research and development progress across the broader inherited retinal disease and age-related macular degeneration landscape.

First, let me tell you a little bit about what's on deck in the agenda for today. First up, we've got Chris Adams, our Vice President of Marketing and Communications, who will share highlights of our current outreach and engagement initiatives across the community. Then Peter Ginsberg, our Chief Operating Officer, will highlight a few notable corporate partnerships and developments in that field and review our fiscal year 2024 financial performance through the end of March. And then next, Dr. Todd Durham, who is our Senior Vice President of Clinical and Outcomes Research, will share some exciting recent news related to our My Retina Tracker Registry and genetic testing program, along with a snapshot of our ongoing natural history studies. And then I'll return to the call to provide updates on several key strategic initiatives and highlight the recent news and announcements that came out of the Association for Vision in Research in Ophthalmology meeting, which we call ARVO, which happened just a few weeks ago in Seattle.

And then finally to conclude our formal remarks, we are very excited to welcome our guest speaker, Dr. Rachelle Lin, who's Associate Professor at the Southern California College of Optometry, SCCO, at Marshall B. Ketchum University. Dr. Lin is an internationally renowned expert in low vision rehabilitation teaching on this important topic, along with conducting genetic testing for inherited eye conditions. Dr. Lin is going to share some of the various resources available to our community.

And then as we always do, following Dr. Lin's remarks, we will open the call up for your questions. And in addition to the speakers that I just mentioned, on our Q&A session, I'm pleased to invite Jeff Klaas, our Chief Strategy and Innovation Officer, and Dr. Amy Laster, our Senior Vice President of Science Strategy and Awards to join us for the Q&A session at the end of the call. So with all that said, I'd now like to turn the call over to our Vice President of Marketing and Communications, Mr. Chris Adams.

Chris Adams, Vice President, Marketing & Communications:

Thank you Jason, and good morning everyone. We're at the point of our fiscal year where we are wrapping up our spring activities and planning for the fall VisionWalks and initiatives. On today's call, I will highlight several of these recent activities.

Our professional outreach team recently launched the Envisioning a Path to Hope initiative to create awareness amongst eye care professionals about the resources available through the Foundation for patients and caregivers affected by blinding diseases. This initiative aims to provide eye care professionals with the knowledge and tools to offer support and hope to patients and their families. To join us in spreading awareness throughout the professional community, please visit Another way we leverage the power of our network is through our 46 regional chapters. We offer many ways to engage locally, such as chapter leadership, attending a speaker series, VisionWalks, special events, and DIY fundraising programs.

Today, I'd like to share one of our recent regional development activities. In April, we hosted our first international Vision Seminar in the United Kingdom to kick off our efforts in the region. Speakers from Moorfields Eye Hospital, Beacon Therapeutics and UCL Institute of Ophthalmology provided highlights on their groundbreaking research on blinding retinal diseases. There was also a panel discussion that included organizations such as the Royal National Institute of the Blind and Guide Dogs UK. This event was made possible by volunteer leadership in the UK, helping to build our global footprint for the Foundation.

And as of mid-May, we've completed 16 of our 20 VisionWalk events and have raised over $1.7 million. At this spring's Triangle VisionWalk, we were able to celebrate the Brindley family for their lifetime fundraising achievement of raising more than $1 million for VisionWalk. And kudos to all of the VisionWalk supporters of the recent walks.

In addition to the walks, there have been many special fundraising events throughout the year taking place across the country. Earlier this month our annual New York City Night for Sight Gala was a huge success, raising more than $725,000 and counting. More events are coming up this summer with Artistry of Wine in Chicago and golf tournaments in the Boston, Denver and New York City areas.

We're also excited about our upcoming VISIONS 2024 conference, which will be held next month from June 21 – 22 at the Marriott Magnificent mile in Chicago. VISIONS provides a unique opportunity to connect with others from the blind and low vision community and to learn about the latest research advancements, products and services. The conference sessions are shaping up quite nicely as well as the support from many exhibitors and sponsors. To get the latest information, visit our website at and click on the VISIONS 2024 tab at the top right of our home page. And if you haven't done so already, please be sure to reserve your hotel room by Tuesday, May 28, to receive the VISIONS 2024 discounted room rate.

And lastly, your commitment is truly vital to our cause and it extends far beyond fundraising. We're seeking dedicated volunteers and passionate leaders to propel our mission forward. There are many opportunities to engage such as serving as chapter volunteer leaders, committee chairs, organizers of community events, and contributors to local educational programs. To get involved today, visit Now with that said, I'd like to hand the program over to Peter Ginsberg, our chief operating officer. Peter.

Peter Ginsberg, Chief Operating Officer:

Chris, thank you very much. In addition to the essential funds we raised through VisionWalks and the other special events that Chris reviewed, we collaborate with corporate sponsors that provide critical funding for important Foundation initiatives. We're very pleased to highlight that our new partnership with BlueRock Therapeutics was launched in March. BlueRock is a clinical stage cell therapy company that's part of Bayer. And BlueRock recently signed on to support our Uni-Rare study. That funding will enable us to expand the number of affected individuals who can be part of Uni-Rare's four-year natural history cohorts. BlueRock is also supporting three of our other natural history studies and is a Silver Outreach partner. Our March 25th press release has further details on this collaboration.

Launched in 2022, the Uni-Rare study is a first of its kind natural history study for approximately 1,500 people with one or more of the 300 rare genes associated with IRDs. You'll hear more about the Uni-Rare study and our partnership with BlueRock from Todd next.

In addition to our Outreach partners, we have a strong lineup of corporate sponsors who support our key events such as our Innovation Summit held earlier this month for a sold-out crowd in Seattle. Thanks to our platinum partners, Johnson & Johnson and Spark Therapeutics, and our silver partners, Atsena Therapeutics, Ocuphire Pharma, Restore Vision and Sepul Bio. We're very grateful for the support of these partners enabling us to host these scientific community events, which in turn fuel research and innovation that's critical to our mission.

Now, before I cover the quarterly financials, I'd like to highlight a recent corporate development. During the recent ARVO meeting that Jason mentioned earlier, Johnson & Johnson, or J&J, highlighted several notable scientific data presentations. One of these presentations included a real-world analysis on the economic value of early genetic testing in patients with inherited retinal diseases, which further emphasized the importance of genetic testing. Todd will also speak later about the Foundation's continued support of genetic testing.

I'd like to conclude my remarks by providing our quarterly financial summary. The Foundation operates on a fiscal year that runs from July to June, and I'll report on our financials through March, so the first nine months of our fiscal year. For that period, our unrestricted revenue was $21.6 million versus operating of $13.8 million for a net fundraising surplus of $7.8 million. And that does place us on track for our fiscal 2024 budget, in which we're targeting $13.4 million in net fundraising surplus to support new research funding. I'll now turn the call over to Dr. Todd Durham to provide an update on our patient registry and also our ongoing natural history studies. Todd.

Dr. Todd Durham, Senior Vice President of Clinical and Outcomes Research

Thank you, Peter. I'm pleased to share with you this morning some recent progress and activities that we're working on to advance clinical outcomes and research activities. The Foundation hosts My Retina Tracker Registry, which is the largest retinal disease patient database connecting people to relevant clinical trials and natural history studies. Launched in 2017, our Registry and related genetic testing program aims to advance research focused on IRDs while addressing critical barriers to genetic testing and counseling for individuals with IRDs across the United States. Our Registry now has more than 30,000 participants, including more than 20,000 members who have genetic testing information in their profiles. Just last week, we announced exciting news related to My Retina Tracker Registry and the genetic testing program. We have formed a partnership with Prevention Genetics, a part of Exact Sciences to serve as the new genetic laboratory partner for the My Retina Tracker genetic testing program. We will continue to work with InformedDNA as a genetic counseling partner for the program.

As a result of these partnerships, genetic testing and genetic counseling through the My Retina Tracker Genetic testing program continues to be offered at no cost to patients with a clinical diagnosis of an IRD, who meet program eligibility criteria. The services are ordered online by a patient's eye doctor. To date, more than 20,000 people have received no cost genetic testing through the program. These collaborations are essential and they underscore the Foundation's commitment to improving patient access to high quality genetic testing, a critical component in accelerating the development of therapies for inherited retinal diseases.

One of the most important functions of genetic testing and My Retina Tracker Registry is to connect participants to relevant clinical trials and natural history studies. This enables the clinical research that is required in the process of finding treatments and cures.

It's critical that we understand the natural progression of the diseases we are targeting. In dealing with inherited retinal diseases, there are nearly 300 different conditions related to genetic variations. Natural history studies help us to evaluate how a disease progresses, estimate the rate of disease progression, and how variable that progression is among groups of affected people. Our ultimate goal is to boost and accelerate the development of therapies among both academic researchers and industry. Now, I'd like to provide a snapshot of the various natural history studies that the Foundation is funding.

The Uni-Rare study launched in 2022 is a first-of-its-kind natural history study for approximately 1,500 people with one of more than 300 rare genes associated with IRDs, including retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and a broad range of other conditions. The study is led by Dr. José-Alain Sahel, distinguished professor and chairman of the Department of Ophthalmology at the University of Pittsburgh School of Medicine.

As Peter mentioned, we have an exciting new update related to the Uni-Rare study. Based on our collaboration with BlueRock Therapeutics, we will expand the study to add a new cohort, which will include patients living with IRDs caused by mutations in multiple genes. In addition to this multi-gene cohort, this will provide more affected individuals in the IRD community with an opportunity to make a meaningful research impact. The data insights from the new study cohort will help inform future clinical trial designs and provide valuable information as BlueRock works to advance its pipeline of cell therapies for treating blindness.

We also have positive news to report on another of our sponsored natural history studies. Earlier this year, we launched GYROS, a four-year study of 45 participants affected with gyrate atrophy, which is associated with mutations in the OAT gene. Gyrate atrophy typically presents in childhood with night blindness and progresses with the loss of peripheral vision and may eventually lead to the loss of central vision.

The GYROS study is currently enrolling patients at 12 clinical research sites from the Foundation's Clinical Consortium and will help inform the design of clinical trials, including a gene therapy being developed by researchers at Johns Hopkins University. GYROS is being funded through a collaborative effort of three organizations, including the Foundation and Conquering Gyrate Atrophy, a nonprofit research and advocacy organization. This project is supported by the Food and Drug Administration of the US Department of Health and Human Services as part of a financial assistance award of $1.6 million. In total, 46% of the study is funded by FDA HHS and 54%, or $1.9 million, is funded by non-government sources.

In our Pro-EYS study, while follow-up visits continue, we recently shared two-year data with our investigators at our March annual study group meeting.

Our RUSH1F study, which is co-funded by Usher 1F Collaborative and Marjorie C. Adams Foundation has completed enrollment with 44 participants, and we have begun collecting data from some of those for their two-year follow-up visits.

The final study I'd like to highlight today is RUSH2A. This four-year study, which completed enrollment in 2019 studied retinal degeneration caused by mutations in the USH2A gene, which is a leading cause of Usher syndrome type 2A and autosomal recessive retinitis pigmentosa. We enrolled approximately 125 participants at 20 sites in the US, Canada and Europe.

At the recent ARVO conference, investigators from the Foundation's Clinical Consortium had a special session to present the four-year results of functional and structural data from the RUSH2A study. This session was very well attended by industry representatives, and it demonstrated the impact of the work we do to identify potential new endpoints that can then be used in clinical trials to show that an investigational treatment offers benefit to study participants.

We are very encouraged by the continued progress in learning more about IRD progression through our natural history studies and we believe these initiatives will have numerous benefits for our community. And with that summary, I'd like to hand the program back over to Jason.

Jason Menzo, Chief Executive Officer:

Thank you so much Todd, and again, thank you everyone for joining us today. In the interim, I actually was able to figure out our technical glitch and for those that are on the Zoom, I am now back on camera. So thank you to everyone and again, this is a terrific opportunity to engage with you all on a variety of different topics. And as we've discussed so many times on these calls, natural history studies really are critical to advancing the research in our field as Todd just said. One other important way that we are driving progress in retinal disease research is actually through our strategic partnerships and targeted funding initiatives. And on this note, just yesterday we announced the recipients of the first awards in our PRPH2 and Associated Retinal Diseases Program, or PARD for short. And following an extensive international call for proposals, the Foundation awarded nearly $1 million to two distinguished researchers. Dr. Andrew Goldberg of Oakland University in Rochester, Michigan, and Dr. Yoshikazu Imanishi from Indiana University. Each of them are working on cutting edge research aimed at understanding and ultimately treating PRPH2 related retinal diseases such as retinitis pigmentosa and of course certain macular dystrophies.

These awards are the first of what we anticipate to be six grants that will be distributed over the next three years through a collaboration that was established in 2023 between the Foundation Fighting Blindness and the Nixon Visions Foundation. And we're super grateful for this important partnership and excited about the impact this program will have on this critical area of research for years to come.

Another great example of collaboration in the research and scientific community is our Retinal Cell and Gene Therapy Innovation Summit, which is an event that we host every year in partnership with the Casey Eye Institute at Oregon Health and Science University in conjunction with the annual meeting of ARVO, which I referenced a little bit earlier in this call.

ARVO took place earlier this month in Seattle. And with more than 10,000 attendees, ARVO is the largest international eye research conference in the world. It's very active, informative and enriching on a multitude of fronts.

Prior to the start of ARVO, the Foundation Fighting Blindness with the Casey Eye Institute co-hosted our ninth annual Innovation Summit. And this event brought together representatives from the biopharmaceutical industry, physicians, researchers, and many other stakeholders who are so key to this field. The theme for this meeting was defining the preclinical to clinical roadmap for inherited retinal degenerations. We had over 400 global experts that were in attendance, which makes it our largest Innovation Summit yet, and there were more than 30 presentations covering topics ranging from preclinical models of disease, clinical trial design, as well as preclinical and clinical studies for gene and cell-based therapies.

One of the most important highlights and really one of the most touching aspects of the meeting was the patient perspective, which was shared by our Board Director, Martha Steele. And this was the first time we incorporated the patient voice into the agenda and wow, the feedback from the clinicians and the scientists was incredibly positive. Many attendees said that hearing Martha's story inspired them to connect the dots from what they do in the lab or in the clinic to the individuals who are eventually going to benefit from their work. And the Foundation will be issuing a summary of the entire day and all the presentations from the Innovation Summit on our website in the very near future.

But again, the Innovation Summit was just the preview, the outset of ARVO. And when we got into ARVO itself, there was a breadth of interesting and notable research presentations from our field. Importantly, there were at least 80 different research projects that were presented at ARVO that were funded by the Foundation Fighting Blindness. And I want to highlight a few of these presentations for you now and you're going to notice a theme that these are presentations focused on impact of research on people in the clinic. There was also a ton around animal models and preclinical, but these highlights I'm going to talk about now are really the highlights in the clinic, in people who are affected with these conditions who are benefiting from advancing treatments into the clinic.

The first is Atsena Therapeutics, a company developing innovative gene therapies for inherited retinal diseases, reported data from their Lighthouse phase I/II clinical trial, which is testing ATSN 201, which is a gene therapy for X-linked retinoschisis or XLRS. This emerging treatment was well tolerated for all three patients in the low-dose group and there were both structural and functional vision improvements for patients receiving this low-dose of the therapy. Lighthouse is now enrolling males with XLRS who are six years of age and older, and they're currently dosing patients in the mid-dose group, so a higher dose of the treatment. And the Foundation Fighting Blindness's RD Fund is a founding investor in this company, Atsena.

Next, Nanoscope announced that it met its primary and secondary endpoints for its RESTORE clinical trial of MCO-010. And this is an optogenetic therapy for restoring vision to people with advanced vision loss from retinitis pigmentosa regardless of the mutated gene that's causing their RP. Based on these results, Nanoscope has stated that they plan on submitting a biologic license application, or a BLA, to the US Food and Drug Administration in the second half of 2024. And if they're successful in this application and approved, then they could be on the market for this therapy in the not-too-distant future.

EyeDNA Therapeutics, which is a subsidiary of Paris-based Coave Therapeutics reported encouraging 24-month results for 17 adult patients with their phase 1/11 PDE6b gene therapy, which is a clinical trial underway in France. The results demonstrated vision improvements in patients who received the high and low dose of this gene therapy. EyeDNA is also evaluating this therapy in younger patients who are aged 13 to 17 and who also have earlier stage disease. Three younger patients are enrolled in this younger cohort so far.

There was also encouraging clinical data at ARVO from PYC Therapeutics, which is an Australian base developer of RNA therapies. They reported initial results from their Phase 1 PLATYPUS clinical trial in the US for an RNA therapy known as VP-001 for people with retinitis pigmentosa, which is caused by mutations in the gene PRPF31. PYC's results are early and preliminarily show relative safety, but the company is moving to a higher dose in patients with less advanced disease and so we're optimistic that this will lead to a potential efficacy signal.

There was another ARVO presentation that I want to mention today, and it relates to the Foundation's Elusive Genes Research Project. A team of genetic researchers from the UK, the US and Switzerland identified a mutation in the gene TMEM216. And it is a common cause of retinitis pigmentosa in people with African ancestry. The team estimates that approximately 20 to 30% of all RP patients of African descent are caused by this mutation. And this breakthrough genetic research was funded by the foundation's Elusive Genes initiative, which targets research primarily focused on identifying previously unrecognized genetic causes of IRDs, often referred to as elusive genes. And the specific genetic cause of disease remains elusive in approximately 40% of inherited retinal disease patients. And so work like this is really important to close that gap and particularly within this audience of individuals from African ancestry, this is a breakthrough and it's going to pay results as we move some of this knowledge into further research.

Finally, as Todd mentioned earlier, investigators from the foundation's Clinical Consortium presented results from the RUSH2A natural history study during ARVO. This special session included a comprehensive presentation of the advantages and disadvantages of various potential clinical trial endpoints, including a new endpoint using visual field data which will continue to develop and validate with additional work over the next several months. There was overwhelming positive feedback from attendees on the special session from academicians to industry representatives, and it really does affirm our belief that the Foundation is the cutting edge of this critical work needed for successful product development.

And then one final clinical research update that I'd like to mention today is from Opus Genetics, which we've talked about on our Insights Forums. Talked about Opus a bit over the last several years. Opus is a clinically staged gene therapy company for inherited retinal diseases with funding from the Foundation's venture arm, the RD Fund. And Opus recently announced data from the first three adult patients in their Phase 1/2 gene therapy for Leber's congenital amaurosis 5 or LCA5. This included positive safety data, which is the first thing you're looking for in these early-stage clinical trials, but they also showed vision improvements for patients in the trial so far to date. And based on these encouraging results, Opus plans to administer the next highest dose of this LCA5 gene therapy to the next cohort of adult patients.

There have been so many, many inspiring stories related to all of these clinical trial developments in this early clinical trial data. And these studies, as I said a few minutes ago, were no longer talking about the impact of a treatment on mice or in the lab or in a dish, but these are results in real people. And some of the patients who are in these clinical trials have been almost totally blind since birth and then they now can see and identify objects. They can read letters on an eye chart for the first time ever. And when I hear these stories, it reminds me of the life-changing impact of the work that we're doing and the importance of the involvement and support from all of you who are on this call today. It takes a while to take things from concept stage in the lab and into the clinic, but we're at this point right now in this field where more and more life-changing research is making it out of the lab and into the clinic and the results that we're seeing are really, really inspirational.

And as a reminder, you can find information and links to all of the ongoing clinical studies. I highlighted only a small few of them today. There are 50 that are ongoing around the globe today and all of these studies are available with all the clinical trial information on the clinical trial pipeline page of our website under the Research section. And again, our website is and this specific section is

Now I just want to switch gears briefly before we get to our very special guest speaker and provide a short update on one of our key strategic initiatives. As I mentioned in our prior calls, we are in the last year of our five-year Science Strategic Plan. We have been working with our global Scientific Advisory Board to develop a new plan which will span from fiscal year 2025, which starts on July 1st in two months through fiscal year 2029. And there are multiple steps underway in this process. Our scientific advisory board has reviewed the achievements in the field over the last five years and identified key knowledge gaps to inform the Foundation's research plans going forward. We have compiled all of these findings to provide the necessary context as our team sets the goals and investment levels for the next five years. We expect this new plan to be the most aggressive and robust funding plan with a single overarching goal to drive research projects and activities to overcome the barriers that exist when translating preclinical research towards the clinic. Of course, for those affected with inherited retinal diseases and dry age-related macular degeneration.

The plan will include very specific priorities, initiatives, and strategies to achieve this goal. And many of the Foundation's current programs such as our grants and awards, the Clinical Consortium, corporate and foundation partnerships, and importantly, the RD Fund facilitate our expected outcomes, highlighting their value to the Foundation's work and mission. The culmination of this process will come this summer with the rollout of our new five-year Science Strategic Plan, and I look forward to sharing more details with you as the process proceeds.

Now it is my absolute pleasure to introduce our key speaker today, Dr. Rachelle Lin, associate professor at the Southern California College of Optometry, also known as SCCO, at Marshall B. Ketchum University. Dr. Lin works with patients at the clinical departments of low vision rehabilitation and acquired brain injury at the University Eye Center in Anaheim, California. She also conducts genetic testing for inherited eye conditions and teaches low vision rehabilitation, genetics, clinical methods and nutrition. She received her Doctorate of Optometry and Master of Science in Vision Sciences from SCCO, and she completed her post-grad residency at the VA Long Beach Healthcare System in low vision, traumatic brain injury, and primary care. She speaks around the world on these important topics and we're thrilled to have her with us today and she's going to talk a little bit about some of the critical resources available to our community. Dr. Lin, I'll turn it over to you.

Dr. Rachelle Lin, Associate Professor, Southern California College of Optometry:

Thank you, Jason. Hello everyone and thank you FFB for having me here today to talk a little bit about low vision rehabilitation. I practice low vision optometry and I love being able to provide devices and resources to my patients. It's always wonderful when we are able to match up a patient with the devices they need to meet their visual goals, be it reading a book with magnification, using assistive technology to excel in school or at work or just to be more independent. Some of you may already be very familiar with low vision resources, but for those of you who are not, we need more awareness of low vision resources in the IRD community. I think about that a lot when patients tell me at the end of an exam that they wish that they knew about these resources sooner or they wish that these devices were available to them when they were struggling in school or at work.

So what is low vision rehabilitation? Low vision rehab encompasses a variety of resources and utilizes a team of specialists who work with patients to reach their individual goals. This team can include many members, often a low vision optometrist, but it can also include low vision ophthalmologists, occupational therapists, and many more. At the core of low vision rehabilitation is the low vision exam. The first part of the low vision exam is understanding the patient's visual needs and establishing their individualized goals. Next, we want to assess the vision. Even if the patient has limited vision, we want to use our specialized equipment and techniques such as trial frame refraction to maximize the use of any remaining vision. Even though glasses or contacts cannot return perfect vision back to patients with vision loss from an IRD, if the glasses or contacts can improve the vision, we want to check for that and prescribe it if it helps.

After we know the best vision we can get out of glasses or contacts, then we want to trial different low vision devices. These can be devices that provide magnification, increased contrast, better illumination, improved visual comfort, and much more. There are traditional devices such as magnifiers for near reading, telescopes for distance viewing and for driving and colored filters to improve visual comfort or enhance contrast. There are also a plethora of newer technologies such as digital portable devices, digital desktop magnification devices, head-borne wearable devices, assistive technology with text-to-speech software, cell phone apps, and now even integration of AI.

It is important to understand that different eye conditions affect vision in many different ways. Some people have central vision loss, others have peripheral side vision loss. Some people benefit from increased magnification and some need enhanced contrast. Likewise, there are so many different devices on the market. Therefore, our aim during the assessment is to help patients find the best devices for their vision and goals.

And last but not least, we want to make sure patients have all the resources they need. This can include connecting them with other members of this interdisciplinary team, such as driving specialists, occupational therapists, orientation and mobility instructors, guide dog schools, IRD specialists and genetic counselors, support groups, and so much more. Therefore, it is not uncommon for a patient with an IRD to have a low vision specialist in addition to their retinal specialist. You all on this call know how incredibly tough vision loss is and we want to make sure that you have all the resources available to you and that you have people advocating for you. For school-aged children, a low vision doctor can provide information and recommendations to the school district to help with the individualized education plan, IEP, and make sure that the kids get the devices that they need in school.

We also work a lot with state vocational rehabilitation agencies. For example, in California, our state agency is called the Department of Rehabilitation, and through this program, patients who are going to school or working are able to receive a low vision evaluation and also receive low vision devices free of cost.

So how can you find a low vision specialist? One of the resources I recommend is the AOA, American Optometric Association's Find a Doctor search webpage. You can search by location and specify that you are looking for a doctor with low vision services. There's also the Low Vision Prescriber Network, which has a very comprehensive list of providers across the nation. And because every state is a little different, I also think it is great to network with others locally, for example, at your local FFB events, to learn from others what great resources may be around you.

In summary, a low vision exam is not the same thing as your routine eye health examination. It is so great to hear about all these amazing updates from FFB and all the research going on which currently provides us hope for cures. Low vision rehabilitation also aims to give hope to patients. Hope that life continues on with an IRD. While we continue to fundraise and fight for cures, it is important that through low vision rehab patients living with IRD get the resources they need to live their lives to their fullest extent. Thank you very much and I'm happy to answer any questions.

Jason Menzo, Chief Executive Officer:

Thank you so much, Dr. Lin. This is Jason Menzo speaking again. We really do appreciate the work that you do and I think you said it perfectly just now. The importance of both sides of the coin while we're funding research and advancing potential treatments and cures that professionals like yourself are really working with the community today to ensure that everyone who's affected by these conditions have all the resources and tools they need to live the most fulfilling and impactful life they possibly can in the meantime. So appreciative of you being on a call and joining us on these calls with our community.

Now it's time to open it up our Q&A session, and so I'm going to ask Amanda to please go ahead and reread the instructions for how you can ask your questions. We already have eight or 10 that have been chatted in just during the call, so we've got a few that we'll start with. But Amanda, can you read the instructions?

Amanda Bement, Development Associate, Admin & Ops:

Thanks Jason. There are several methods that you can use to ask questions. You can submit them through the Q&A or chat functions, both of which are at the bottom of your Zoom screen and make sure that you include your name so that we can follow up afterwards. You can also send an email to and we'll follow up in the next week or so.

Jason Menzo, Chief Executive Officer:

Thank you, Amanda. We're going to start. Tyler Manning actually sent this question in earlier, prior to the actual call starting today a couple of days ago. We're going to direct this to you, Dr. Laster, our Senior Vice President of Science and Strategy. Tyler's question is over a decade ago, the first stem cell therapies were attempted and it seems like we're stuck in the early days of stem cell therapy without a ton of clinical proof yet that we can significantly restore vision in humans. Tyler shares that he's affected with Stargardt and just cares about the status of cell-based therapies and what's happening pre-clinically and then also in the clinic around cell-based therapies.

Dr. Amy Laster, Senior Vice President of Science Strategy and Awards:

Thank you, Jason. Again, this is Amy Laster. And thank you, Tyler, for your question. Stem cell therapy, it's among a class of therapeutics that can be classified as what we call restorative therapy, with these approaches really beneficial to late stage disease. There are a number of early phase clinical trials that are involving cell-based therapies including stem cell therapies specifically for retinal degenerative diseases. I will encourage you to go to the Foundation's website that was referenced earlier, under Research and Clinical Trial Pipelines so you can get more information on the different trials and where they're in their stages. Thank you.

Jason Menzo, Chief Executive Officer:

Thank you, Amy. Dr. Lin, I want to address this next question to you. We do hear this often. This question was also sent in before the call, but this comes up quite frequently out in the community. And this is around alternative approaches, alternative medicine I guess, as it relates to potentially treating these conditions. Things like acupuncture, hyperbaric oxygen chambers, other maybe less traditional modes. The question is, are you familiar with any well-controlled clinical trials in this arena or any experience you have with these types of approaches for RP?

Dr. Rachelle Lin, Associate Professor, Southern California College of Optometry:

Good question. There are some limited reports of cases regarding acupuncture and hyperbaric oxygen therapy on RP, but there are no current large-scale or substantial clinical studies demonstrating its effectiveness of either of those treatments for RP.

Jason Menzo, Chief Executive Officer:

Dr. Lin, there's two questions that were just chatted in. We are so fortunate to have you with us today. I want to make sure that we prioritize questions for you because we don't have you on every call. So let me ask you, when you're speaking or working with patients who have low vision, are there specific apps or tools that you like to recommend to your patients who have low vision? And also perhaps you can speak a little bit around the conversation when a low vision patient should not be driving anymore. Those two questions were just chatted in just in the last few minutes and I thought maybe we'd pose those to you.

Dr. Rachelle Lin, Associate Professor, Southern California College of Optometry:

The driving question, that comes up a lot, especially in the United States where we don't have excellent public transportation as maybe some places in the world. We do not go out of our way to stop patients from driving if they are good at driving and are capable of driving within the laws within their state. And that's oftentimes a question that comes up. You might be afraid to go to an eye exam if you think that that doctor's going to pull your license. That's not the case. Of course, you can always chat with them before hand about what their philosophy is. In our case, there are actually a lot of our patients who are excellent drivers and excellent candidates for driving. That is something you should speak about with your low vision provider. For example, patients who have a limited amount of vision loss that is relatively stable and is within the legal limits of driving, at least in California that's better than 20/200, they are able to do the driving tests within the state and it is low vision providers who are oftentimes filling out those application forms for those patients to make them eligible for those extended specialized driving tests.

If of course a patient is not safe for driving, if they have extremely limited vision, of course we can counsel them on that and give them our recommendations. And if someone's on the fence and they're not sure if they are going to be a good driver, we will oftentimes refer them to driving specialists to do driving assessments and to learn special skills to see if they can drive better and more safely, either with their vision or with bioptic telescopes. With regards to apps, one of the more common apps that is used is Seeing AI. That's available on iPhones. But there are many other apps out there. And with regards to the addition of artificial intelligence, a lot of current devices are now adding on AI features. So some of the wearable devices now have AI features embedded within them.

Jason Menzo, Chief Executive Officer:

Thank you so much. Let's shift gears. I'm going to pose this question to you, Todd. There's actually a couple of questions I'm going to pose to you, Todd. We have often a lot of questions around access or participating in My Retina Tracker and genetic testing outside the US, so that's question one. Question two is for parents who are considering research projects for their children. Is there anyone that can sit down with a family and discuss the pros and cons of eye research? We often get told to talk to the research companies, but worry that they only have a conflicted interest in this. And so maybe you can speak of the role of a genetic counselor and how they could participate in that. So both those questions to you.

Dr. Todd Durham, Senior Vice President of Clinical and Outcomes Research

Thank you. This is Todd Durham. On the first question, My Retina Tracker Registry, the registry itself is open to international involvement, so anyone around the world can sign on to create a profile. But where we do have a restriction is with the genetic testing program. The sponsored program is offered only in the United States. That has been over the years really a function of available funding to offer that program. So that's the answer to the first question. The second question I think is a great one. This parent is asking how can they learn more, how can they spend some more valuable time with someone who can give them information as parents as they ponder the possibility of having their child participate in a research study. A couple of ideas come to mind.

Genetic counselors, if you have access to one, can be excellent healthcare professionals and resources for this.

Any physician who's participating in a clinical trial, whether that's your own or your child's physician or the research physician, it is their job to help you think through the pros and cons of participating in that particular study.

We obviously encourage you to consult your own healthcare provider about your involvement in a research study, but it's the investigator in that study who really, it's their job to spend time with you to answer all your questions and make sure any of your concerns are addressed.

And the other thing that I can offer is here at the Foundation, we have a number of parents whose children have participated in clinical trials, so you could reach out to us and perhaps we could find a parent who would be willing to share their experiences with you as someone who's gone through that very same experience or understanding that thought process. Those are a few ideas I can think of.

Jason Menzo, Chief Executive Officer:

Thank you very much, Todd. Peter, Dan Day chatted in a question about Spark Therapeutics, and they have previously been offering genetic testing and partnership with Blueprint and curious about the impact of our new relationship we just spoke of earlier today and how that might affect Spark's program.

Peter Ginsberg, Chief Operating Officer:

Good morning. This is Peter Ginsberg and thanks for your question, Dan. Just as background for all on the call, Spark Therapeutics is the developer of LUXTURNA, the gene therapy that's available now. The Foundation Fighting Blindness supported a lot of the early research that led to the development and launch of LUXTURNA. Spark Therapeutics, to our knowledge, has not yet disclosed its plans going forward as it relates to genetic testing. So Dan, I'd recommend that you reach out to Spark for any questions regarding their plans going forward on genetic testing. But we feel really good about our ongoing support of genetic testing with Prevention Genetics. And Dan, also hope you'll be up in Chicago. Looking forward to seeing you and many of our participants at the VISIONS conference in June.

Jason Menzo, Chief Executive Officer:

Thank you, Peter. A few of you have chatted in specific ... Well actually, I should say many, many have chatted in very specific questions around a particular gene or what is the state of research in this particular very specific area. As I think everyone knows, there are over 300 different genes that can cause inherited retinal diseases and often in these calls we run up against the clock and we don't get to cover every single question, specifically if they're about a particular gene. What I always encourage is if you have any specific questions, to certainly send us an email at But everyone who's sent in a question on the chat, if we don't get to your question here on the call, we will follow up with you afterwards. So don't be alarmed if we don't get to every single question on the call because we probably won't, but we will follow up with all those specific questions.

We did have a couple specifically asking about LCA5 because I mentioned the Opus Genetics trial. Those of you have asked about LCA5 and the recruitment for the next cohort, we will get with you offline and so we have your information. Thank you for chatting in and we will connect you with the right individuals at Opus to make sure you're on their radar.

Dr. Laster, I want to come to you next. There was a question that was chatted in about the translational research award that we just announced with Odylia and then also a question around companies that are in clinical trials for gene agnostic approaches, whether the trial design allows for them to assess and understand, even though they're gene agnostic, what genetic mutations are incorporated in the clinical trial that the individual in the trial are affected with. So those two questions to you.

Dr. Amy Laster, Senior Vice President of Science Strategy and Awards:

Thank you. Again, this is Amy Laster. I'll take the first question. Our translational program, it's one of our funding mechanisms and really it's geared toward providing funding to either academic researchers, startups, or small companies that have a particular or promising therapeutic intervention for an inherited retinal degeneration or dry AMD. The funding is to help them get to the next stage of development in terms of getting to a clinical trial and so that could be what we call an IND enabling study. These are critical studies that the regulators will need to assess before they would approve the start of a clinical trial. We have just completed the 2024 awards for our translational program, and we'll do a full announcement on those in the coming weeks. But there was an award to Odylia Therapeutics to further their gene therapy program for a form of RP. RPGRIP1 is the gene.

The second question with regards to trials keeping track of the gene type for their patients. The genetic tracking, it varies. It's really dependent on the specific disease, the treatment, the research goals. There are some interventional trials, particularly gene agnostic where they're taking individuals with a clinical diagnosis, so the doctor has said you have RP, and it doesn't need to take into account what is the specific mutation that is contributing to your RP. There are other clinical trials where knowing the genetic diagnosis, so not just clinically what your diagnosis is, but specifically what your mutation is, is very important. In most cases, the gene therapies clinical trials or those that modify or edit like the RNA, the very specific precision medicine trials will need to be informed by the genetic diagnosis. There is a bit of variability in it, but there isn't a specific requirement that the genetic details are tracked for every single clinical trial. Thank you.

Jason Menzo, Chief Executive Officer:

Thank you, Dr. Laster. I recognize it is just about 12:00 on the East Coast, so I'm actually going to do a quick rapid fire answering of three or four questions just really quickly. A question was chatted in about how are we engaging regulators with the natural history and endpoint design data that we talked about in Todd's section. We are very active in engaging with both FDA and EMA in Europe, so I'll just leave it at that.

Someone asked about the name of the company that we referenced a little bit earlier in the call who we had stated that they have publicly stated that they plan on filing a gene agnostic optogenetic treatment with the FDA. That company is named Nanoscope. I had one other one I was going to try to get to just now. Oh, actually Dr. Lin, maybe we'll end on this note. Someone asked whether or not you have a feeling of the benefit versus cost of wearable devices for individuals with RP. Things that are assistive devices and if there's any quick tips that you have on that front.

Dr. Rachelle Lin, Associate Professor, Southern California College of Optometry:

Yes. Some of those newer technologies can be quite pricey. Oftentimes insurance doesn't cover, but some patients do have low vision benefits through their vision plan, so look carefully at your vision plan to see if you do have any low vision benefits. If not, I highly recommend going to check if your state agency allows you to have any services. As I mentioned, California, patients are able to get that low vision exam as well as devices completely covered if they are working, going to school or planning on going to school or working. It varies a little bit by state to state, but take a look at that and there are a lot of resources for getting devices that way. I also tell patients different devices work differently for different eyes, so always try out that device yourself before you drop a couple grand on a very pricey item. And for some patients it is very much worth it for their vision and for others, they might try it and say, "Oh, this isn't the one for me."

Jason Menzo, Chief Executive Officer:

Dr. Lin, how does someone in Ohio or someone not in southern California find someone like you local to them? Where should they go to try to find a professional like yourself?

Dr. Rachelle Lin, Associate Professor, Southern California College of Optometry:

I put two links into the chat box. There is the AOA Find a Doctor resource. And if you go to that resource, you can specify that you are looking for a low vision provider or someone who has low vision services within their office and you can search by zip code. And then there is also the Vision Council Foundation's Low Vision Provider Network, which is pretty comprehensive.

Jason Menzo, Chief Executive Officer:

Thank you so much. Unfortunately, as always is the case, we are up against the clock so we have reached the end of our time today. I do want everyone to know that our team is energized by the tremendous support from all of you in our community. We certainly encourage you to participate in as many Foundation activities as possible. Truly, it takes a village and together we are achieving great things. A great place to learn about what's happening in your local community is at our website, We certainly have chapters all over the country, expanding outside the country now as well. And VisionWalks, vision seminars, educational events, fundraisers, community events. There's just so much going on and our website's a one-stop shop to find those resources and what's happening in your local area. Certainly welcome any feedback or suggestions related to this webcast or the Foundation in general. And you can reach us anytime by emailing us at and of course you can always learn more at our website If you have any questions, don't hesitate to reach out. I'm going to turn the call over to Chris Adams to wrap things up.

Chris Adams, Vice President, Marketing & Communications:

Thanks Jason. Again, we'd like to thank everyone for participating in today's call and as a reminder, there will be a transcript and audio recording of today's call within the next week on our website, And as Jason mentioned, a good way to stay in connection with everything that's happening, connect with us on social. We're on Facebook, Twitter, LinkedIn, Instagram, TikTok and Threads. Thanks again for joining us today and you have any questions or need any updates, please send an email to Thank you and have a great day.