Recording Available: Insights Forum | Thursday, September 24, 2026

Foundation Fighting Blindness
Insights Forum Transcript
September 24, 2026

Maddie Mossman:

Hello, everyone, and thank you for joining today's Insights Forum. My name is Maddie Mossman, and I'm the host of the Foundation Fighting Blindness' podcast, Eye on the Cure.

Before we get started, I would like to briefly review a few details for the call. Currently, all participant lines are muted and without video. Please be aware that the controls are at the bottom of the Zoom interface. This control bar may collapse when it is not in use. If you prefer to prevent the controls from auto hiding, you can use the following keyboard shortcuts to toggle the Always Show Meeting controls options. If you are using Windows commands, press the alt key. If you are using a Mac keyboard, press command and backslash at the same time. Today's presentation is being recorded and is available with closed captioning. To activate closed captioning, please select Show Captions at the bottom of the screen on the Zoom toolbar.

Please note that on today's call, our speakers do have their videos live. However, all of their comments will be provided verbally and there are no slides. Throughout the call, you'll be able to ask questions via the Q&A feature 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. So please make sure to include your name in your question. You can also submit a question by sending an email to info@fightingblindness.org. I would now like to turn the call over to our Chief Executive Officer Jason Menzo.

Jason Menzo, Chief Executive Officer:

Thank you very much, Maddie, and good morning, everyone. My name is Jason Menzo, and I am the CEO here at the Foundation Fighting Blindness. We're so glad to have you join us for our quarterly Insights Forum. These calls allow us to engage directly with you, our global Foundation Fighting Blindness community. We outline key strategies and priorities, we spotlight advances from across the field, and highlight the ways that we are advancing our mission to speed new treatments and cures for blinding retinal diseases to the patients who are waiting.

For our agenda today, we are going to start with Daniel Widner, who is our Vice President of Community Development. Daniel will be sharing his background and role within the Strategy and Innovation Office led by Jeff Klaas, who you all have met in prior Insights Forum calls. And then Peter Ginsberg, our Chief Operating Officer, will provide an update on recent corporate partnerships and industry developments. He will summarize our fiscal year 2026 financial performance and provide the financial outlook for what we can expect in the year ahead, fiscal 2027. And then after Peter, Dr. Amy Laster, who is our Chief Scientific Officer, will share a status update on our science grant programs and highlight recent developments on the clinical trial front, which I know is something that many of you are dialing into this call to hear specifically.

And then following Amy's presentation, we'll shift from our typical guest speaker format to a new format for the call today. We're going to have a panel discussion featuring Dr. Rusty Kelley, who's the Managing Director, and Dr. Gene de Juan, who is the Chair of our Gund Vision Fund, which previously, you all know, we called the Retinal Degeneration Fund or the RD Fund. Our venture fund is now called the Gund Vision Fund, and it's an honor of Gordon Gund and the Gund family for their more than 50 years of leadership, advocacy, and of course, philanthropic support for the mission of the Foundation Fighting Blindness.

In this conversational setting, Rusty and Gene are going to share their perspectives on the latest progress in the retinal degeneration space as it relates to research, emerging treatments, and what these advancements may mean for patients and families in the years ahead. And then following the panel discussion, we'll open the call up for Q&A as we typically do. In addition to the speakers that I just mentioned on the call, we also will be joined by Jeff Klaas, our Chief Strategy and Innovation Officer, Dr. Todd Durham, our Senior Vice President of Clinical and Outcomes Research, and Chris Adams, our Vice President of Marketing. They'll all be with us to participate in the Q&A session at the end of the call.

All right, so let's get started. I'm pleased to introduce one of our newest team members, Daniel Widner, who, as I just mentioned, serves as our Vice President of Community Development. Daniel brings significant experience in community-based fundraising and volunteer engagement with almost two decades of experience at the American Cancer Society. So with that, I'm going to turn the call over to Daniel.

Daniel Widner, Vice President, Community Development:

Thank you, Jason, and good morning, everyone. I'm Daniel Widner, and it's a pleasure to join you today on the Insights Forum. As I approach my first 100 days with the Foundation, I'm grateful for the opportunity to serve as Vice President of Community Development. I bring extensive development experience, as Jason mentioned, most recently with the American Cancer Society, where I spent the last 18 years. There, I led teams across market and regional roles with a focus on local boards and chapters, events, corporate engagement, and major gifts.

On a personal note, my family and I have been raising guide dogs for the past six years, and I'm excited to be a part of the Foundation Fighting Blindness team as this leadership role allows me to align my personal commitment to the community with my family, as well as my professional work, advancing its initiatives and raising funds, building resources, and accelerating research towards treatments and cures.

My team focuses on driving local engagement across the continental United States through our chapters. We engage local leaders to support the work in their markets, whether that's through events such as VisionWalks or signature events like our galas and golf tournaments. We also host Vision Connection events across the country, providing an opportunity for the Foundation to serve as a convener and community leader, helping connect members of the blind and low vision community with support and resources available locally.

Today, I'm thrilled to highlight an event that brings our entire community together in a brand new way. For the first time ever, the Foundation is hosting a National VisionWalk Day. You don't need to be in person to make an impact. This year, National VisionWalk Day is fully virtual, giving every one of us a chance to walk our own route at our own pace while standing shoulder to shoulder in one shared mission: advancing treatments and cures for blinding diseases.

On October 31st, 2026, we'll unite from coast to coast, families, friends, coworkers, classrooms, and supporters across the country all walking with purpose. At 10:00 AM Eastern, we'll kick off the day with a national livestream featuring exciting updates and latest breakthroughs in vision research. We're going to have a fun science trivia to test your knowledge and spark some friendly competition and meaningful opportunities to connect with walkers nationwide who are moving for the same cause. After the livestream, you can do your walk, whether it's on your neighborhood trail, a city sidewalk, a school track, or even your living room treadmill. Do it your way to be part of one shared mission.

Getting involved is simple. First, you can register your team. Invite your friends, family, coworkers, classmates, whether you're across town or across country. Second, kick off your fundraising. Share your page, send a few texts, make a few calls. Every dollar fuels progress, accelerates research, and strengthens hope for millions living with blinding retinal disease. And third, on October 31st, lace up your shoes and get moving. If you know a business or organization that may want to join us as a sponsor on National Walk Day or want to create a VisionWalk team, we'd love to connect with them. Partnerships make this movement stronger, and we're deeply grateful to our National VisionWalk partners, Samsung and Amgen, for their generous support.

This is our moment to show what a united community can do together, even when we're miles apart. Wherever you are, you have a place with VisionWalk. Register today, rally your community, and join us on October 31st as we close out Blinding Awareness Month by taking steps together toward a brighter future. Let's make National VisionWalk Day count. And costumes are encouraged. For more information, to learn more about our National VisionWalk Day, you can actually go to fightingblindness.org/nationalvisionwalkday, all one word. I would also encourage all of you to visit your local chapter page to learn more about engagement opportunities in your area, as we might already have a local VisionWalk for you to engage with it.

I want to finish by reiterating how excited I am to be leading our community development team, and I look forward to sharing updates on our initiatives and progress over the coming year. Now, I'm pleased to hand the program over to our Chief Operating Officer, Peter Ginsberg.

Peter Ginsberg, Chief Operating Officer:

Thanks, Daniel, and it's really great to have you on board. I'd like to begin our industry and financial summary by recognizing and thanking three of our Outreach partners, Belite Bio, Ray Therapeutics, and Merck.

Belite Bio is a new gold Outreach partner and recently filed for FDA approval of tinlarebant for the treatment of Stargardt disease. An FDA decision is expected by February. If approved, tinlarebant would be the first medication specifically approved for Stargardt disease. Tinlarebant is also being developed for the treatment of dry AMD.

Ray Therapeutics is a first-time Outreach partner and is a clinical stage company developing optogenetic therapies to restore vision. Its approach delivers a highly light sensitive protein to retinal cells, aiming to improve visual function regardless of the underlying genetic mutation. Ray's lead programs include RTx-015 for retinitis pigmentosa and RTx-021 for Stargardt disease and geographic atrophy.

Merck is another first-time Outreach partner. Merck, a household name, has more than 130 years of experience developing medicines and vaccines. Its ophthalmology pipeline targets retinal diseases associated with the vascular leakage and abnormal blood vessel growth, including diabetic macular edema, and Merck announced data on that drug today, and also wet AMD. We appreciate these companies' support of the Foundation's educational and outreach efforts.

Now, changing gears, one of the key roles of the Foundation is bringing the voices of our community directly to regulators such as the FDA. I'm pleased to report that next spring, on April 8th, to be specific, we'll host a patient-focused drug development meeting, or PFDD, focused on Usher syndrome. This is an opportunity for our community to help shape the future of treatments and ensure patient perspectives remain at the center of progress.

The goal of the meeting will be to educate FDA staff and other key stakeholders about what it's like to live with Usher syndrome, with perspectives from affected individuals and also caregivers and family members of affected individuals. Through patient stories, discussions, and feedback, we'll also create a Voice of the Patient report that will be hosted on the FDA website, which will help inform future Usher therapy development, influence regulatory review, and strengthen advocacy efforts for the Usher syndrome community. We're very grateful to AAVantgarde Bio, Atsena Therapeutics, Eli Lilly, The Fairfield Fund, and Sepul Bio By Théa for their generous support of this important initiative, and stay tuned for more details on this PFDD meeting and related activities in the coming months.

Now I'd like to highlight some recent news in our field. In Europe, Science Corporation has commercially launched PRIMA, which is a retinal implant designed to restore central vision in people with geographic atrophy or GA. Science Corp is also working with the FDA to bring PRIMA to the U.S. market as well.

In other approval news, the FDA approved LUMVOA, a new treatment for thyroid eye disease from Viridian. LUMVOA joins TEPEZZA as an FDA-approved treatment available for patients with TED, which is a rare autoimmune condition that affects the eyes and can worsen with time. Finally, Tarsus Pharmaceuticals completed its acquisition of Alkeus Pharmaceuticals, including Alkeus' emerging oral treatment for Stargardt disease known as gildeuretinol, which is now being evaluated in the largest Stargardt clinical trial ever. That's a Phase III study.

Moving on to our financial report, I'd like to provide a brief summary of our fiscal year 2026 financials and our budget outlook for fiscal 2027. You may recall that the Foundation operates on a fiscal year that runs from July to June, so we just completed our fiscal 2026 on June 30th. I'm pleased to report that based on our unaudited fiscal 2026 financial results, we achieved a $13.5 million net fundraising surplus, meeting our budget target for the year.

A key driver of our fiscal 2026 results was Legacy giving. Legacy giving plays a critical role in the Foundation's ability to fund impactful retinal disease research. If you're interested in learning more about the program, please go to fightingblindness.org/legacy-giving where you can find more details and also contact info for our Legacy giving team.

Overall, preliminarily in fiscal 2026, we invested almost $33 million in grants, clinical consortium funding, our My Retina Tracker Registry and genetic testing, and also public education. That does not include $8 million in fiscal year 2026 investments in promising emerging retinal disease companies through the Gund Vision Fund. Following our regular annual audit, which is ongoing now, our audited fiscal 2026 financial statements will be available.

And then upcoming for fiscal 2027, we're targeting $42.6 million in revenue against $25.4 million in operating expenses for a budgeted fundraising surplus of $17.2 million this year. And always, for more information on our financial reporting, you can go to the Foundation's website in the About Us section and navigate to Financials and Governance.

I'm now pleased to turn the call over to my friend and colleague, Dr. Amy Laster, our Chief Scientific Officer, for a research update. Amy?

Dr. Amy Laster, Chief Scientific Officer:

Thank you, Peter. I would like to provide a brief summary of our grant funding for fiscal years 2026 and 2027 and highlight recent progress in the clinical trial landscape. The Foundation Fighting Blindness currently funds 96 grants, and the research projects of these grants are conducted by over 120 research investigators at 86 institutions, eye hospitals, and universities. Beyond supporting US researchers, the Foundation fund laboratories in 18 countries worldwide, including ones in Australia, Canada, Germany, Japan, and the UK.

I am pleased to report that we invested approximately $11 million in new research projects in our fiscal year 2026, and we are targeting a similar level in fiscal year 2027. Our Science committee met recently to review the Foundation's five-year research investment outlook. The future investments will continue to support career development programs, a broad preclinical research portfolio spanning inherited retinal diseases and dry AMD, as well as translational programs that really emphasize therapeutic candidates with a clear path towards clinical development.

We have several grant programs with upcoming funding opportunities, and these include our traditional individual investigator research award and our postdoctoral fellowship award. The initial letters of intent are due in early October, and researchers that are interested in applying can go to our website, fightingblindness.org, and navigate to the research section for grants and awards. You can also visit the clinical trials pipeline page in the research section of our website. Here you can find a helpful list of ongoing clinical studies, and it really is encouraging to see that there are more than 60 clinical trials now underway.

I'd like to share a few recent updates from these studies that really give us reason for hope. First, Beacon Therapeutic, one of our Gund Vision Fund portfolio companies, reported positive pivotal VISTA trial results for laru-zova, its investigational one-time gene therapy for RPGR-associated X-linked retinitis pigmentosa. The study met its FDA-endorsed 12-month primary endpoint, so 31% of participants receiving the high dose and 24.1% receiving the low dose gained at least 15 letters in low-light visual acuity compared with none in the untreated group. Beacon reported a favorable safety profile and plans to begin regulatory discussions and share additional results at the upcoming American Academy of Ophthalmology annual meeting next month.

Also, to add onto what Peter has already stated, Belite Bio filed a new drug application with the FDA in June of this year for tinlarebant, which again is an oral treatment for Stargardt disease. Although accepted applications are typically reviewed within 10 months, the FDA granted priority review, and so moving the deadline for a decision to February 2027. If approved, tinlarebant could become the first treatment approved for Stargardt disease, and this is especially helpful milestone for this community.

Another Gund Vision Fund portfolio company, Opus Genetics, reported encouraging early results from the first low-dose cohort of its Phase 1/2 trial of OPGx-BEST1, and this is for BEST1-related retinal diseases. All five participants showed clinically meaningful improvement in visual function with no serious treatment-related safety concerns. OPUS plans to complete dosing in the second higher dose cohort in the fourth quarter of 2026 and could begin a Phase 3 trial in 2027. Opus also reached alignment with the FDA on Phase 3 trial design and commercial manufacturing requirements for both its BEST1 and LCA5 programs. Enrollment in the LCA5 Phase 3 trial is complete with dosing expected to begin in the fourth quarter of 2026.

Atsena Therapeutics, also a Gund Vision Fund portfolio company, received European orphan drug designations for ATSN-101, and this is for LCA1, and ATSN-201 for X-linked retinoschisis. These are important regulatory milestones as both programs move forward in pivotal trials.

And Sumitomo Pharma America treated the first participant in a Phase 1/2a clinical trial for DSP-3077, which is an emerging treatment for retinitis pigmentosa. This is a regenerative cell-based therapy that uses stem cells, which are master cells that can develop into many types of cells to create sheets of retinal cells.

Finally, Sepul Bio By Théa completed enrollment in its Phase 2 clinical trial of ultevursen. This is a potential treatment for USH2A-associated retinitis pigmentosa. With enrollment complete, the study will now evaluate the therapy safety and effectiveness over a planned two-year follow-up period.

In addition to following our clinical trial listing and recent research news, one important step that you can take is to join My Retina Tracker Registry. By enrolling, you or an affected family member may have an opportunity to be connected with clinical trials relevant to your retinal disease or mutation. So I hope you'll visit the research section of fightingblindness.org to learn more and consider joining. Staying informed and connected really is one way that we all can be a part of the progress ahead. I'd like to now turn the program back over to our CEO, Jason Menzo.

Jason Menzo, Chief Executive Officer:

Thank you very much, Amy. Gosh, what a great update. Whenever we get on our quarterly Insights Forum calls, your section, when you talk about the clinical progress that is taking place, always, I know for me and I know for many members of our audience, this is why we do what we do. This is really where all the effort really meets the moment. I love that not only today are there more promising treatments and therapies in the clinic than ever before, but several of the updates that you just shared, Amy, are really late-stage clinical programs that are close to potential FDA approval. I know that we talked about just now, we talked about the great news from Beacon, the great news from Belite Bio. NanoScope last week published that they've had their BLA accepted by the FDA.

So there's more and more programs that are not just entering the clinic or in the clinic, but are soon to be exiting the clinic and working with regulators to hopefully have more treatments approved and available to patients in the market. Of course, this is why we at the Foundation Fighting Blindness do what we do. We are at the center of supporting all of these advancements, and so thank you to everyone participating today for your support to help us drive the research to provide preventions, treatments, and cures for those affected by inherited retinal diseases.

Shifting gears now in the program, we wanted to provide a unique opportunity in today's program to give our audience a frontline view and perspective of the evolution within our industry, and importantly, the role that the Gund Vision Fund is serving to drive our mission from the lab to the clinic and eventually to patients. So with that, I'm going to invite Dr. Rusty Kelley and Dr. Gene de Juan to join in our virtual fireside chat.

Dr. Rusty Kelley, as I mentioned at the beginning of this call, is the Managing Director of the Gund Vision Fund, and Dr. Gene de Juan is the Chair of the Fund's Board of Directors. This is an especially timely conversation as Dr. de Juan just recently stepped into the Chair role, succeeding Dr. Adrienne Graves, who served as the Chair of the fund's Board since 2022. Really extend our sincere thanks to Dr. Graves for her exceptional leadership as Chair when the Fund was at this really critical stage having just launched, and guiding it from those early stages to where we are today. We're thrilled that she's going to continue to provide her expertise as a member of the Board.

For those who don't know Dr. de Juan, he is a pioneering vitreoretinal surgeon, an entrepreneur, and a leader in ophthalmic innovation. His perspective combines decades of clinical, academic, and industry experience with the commitment to accelerating new treatments and cures for retinal degenerative diseases. With that said, let's jump in.

My first question is to you, Gene, Dr. de Juan. We're living in really an extraordinary moment scientifically, but more promising approaches are moving into the clinic towards patients really more than ever before, but there's a constraint obviously around funding, and capital has been harder and harder to come by. So the first question is, what are you seeing in the funding environment for retinal degenerative diseases and where are the biggest gaps?

Dr. Eugene de Juan, Chair, Gund Vision Fund:

Thank you, Jason. It's a real honor to be part of the team effort here, particularly with Rusty at the Gund Vision Fund. So it's clear that all of the preparation, all the many years that I've been a part, I'm a practicing vitreoretinal surgeon, retinal specialist, and have been involved in the care of patients going blind from these diseases. So it's really a pleasure to be today seeing all of the changes, all of the efforts like Amy just had outlined, and Rusty will speak to more.

Every day we're learning new biology, new clues to the pathologies that are being developed, new ways of treating it. So investment, making sure that a company that has a new technology that, let's just say, could solve, I don't know, a kidney disease with this drug or retinal disease with this drug, that we can begin to encourage and share our extensive scientific expertise, extensive medical expertise, and partner with these companies, increase their likelihood of success, and increase their focus on retinal diseases. Not only does that give us access and encouragement, but it also allows us to benefit, and Rusty will talk more about that, how much we can benefit these dollars as we invest in these, and hopefully find the cures and demonstrate the cures, and participate, and then are rewarded back to allow us to do more for the community.

Jason Menzo, Chief Executive Officer:

That's great. Thanks, Gene. So Rusty, Gene's discussing the mechanism of how industry and venture investment and the mechanics of how treatments traditionally move from the lab to the clinic. But in many of our diseases, in many of our areas, the wins of industry don't necessarily naturally occur without an injection of a different thing called the Foundation Fighting Blindness or the venture philanthropy model, which sort of fills a gap. So maybe for those on the call who are less familiar with what venture philanthropy is, maybe you could just describe what is venture philanthropy and how does it fundamentally help us deliver our mission between the grant making mechanism and traditional industry?

Dr. Rusty Kelley, Managing Director, Gund Vision Fund

Thank you, Jason. Echoing Dr. Gene de Juan's sentiment, it's a real pleasure to be here with the community, the staff, and of course, Dr. de Juan himself. Gene mentioned something that's very special to us that speaks to our model of venture philanthropy, and that's the talent to the scientific and clinical governance, financial, investment banking, all of the talent that we've surrounded ourselves with at the level of the board that extends to a very significant Scientific Advisory Board at the Foundation, that we can leverage that expertise is very special, especially Gene de Juan who covers so many aspects of R&D. Of course, the clinical piece of it is the crux of it all.

But this term, venture philanthropy, it can be mysterious. It's used broadly or loosely to define a lot of different models. The way I like to say that the Gund Vision Fund model works is that we sit at this unique intersection of traditional mission-focused, traditional philanthropy, the Foundation Fighting Blindness, that supports discovery and scientific advancements. And then the other side of the intersection is this disciplined institutional investor arena that must prioritize providing financial returns to their investors. So we're doing both. The exception is that we don't have limited partners as investors that we're returning financially to. We're returning clinically and financially to the model, the Gund Vision Fund. And it's really the clinical priority that we are brutally focused on.

So the primary objective, again, is to channel these precious not-for-profit donation dollars when there's conviction into for-profit investments that turn science into therapeutic products. And then with clinical success comes financial returns. When we do have clinical financial success, those returns are recycled into new investments that further our mission. And that's fairly standard for therapeutically based and mission-based foundations that have venture arms. There are very few of them. There are even fewer that have been successful. It's a difficult business.

But another thing that makes our model so unique is that the Foundation has all of these wonderful resources. The granting programs that Amy discussed earlier, they're quite extensive both in the retinal space that includes retinitis pigmentosa, but also include dry age-related macular degeneration. So there's great breadth there in the granting programs. We have this beautiful patient registry that Todd Durham often speaks about, that's going to be covered on this call during the Q&A, that allows our companies to recruit clinical trial participants. Even further than that, it allows the industry to really understand the composition of these patients, both genetically and in terms of their natural history.

And then that brings us to another wonderful resource that's also under Todd Durham's leadership, is the natural history studies that we conduct that are standardized and allow us to really understand disease progression and the endpoints that are used to measure progression and also safety and efficacy in a clinical trial. It's those endpoints that the Foundation is doing such a great job, and our companies, to develop novel endpoints that are more accurate and that can predict success earlier and more accurately. Of course, I mentioned earlier, the Scientific Advisory Board, it's a real gift for those of us on the fund management side to be able to lean on that expertise.

Jason Menzo, Chief Executive Officer:

That's great. Thank you, Rusty. The simplest way for a donor who's thinking about, "Okay, well, how does a donation get used to deliver all of these wonderful results Rusty just spoke of?". Essentially it's a donation to the Foundation Fighting Blindness that can be restricted for the RD Fund to use the capital that's donated with the discipline of venture investing in leveraging all of the resources that Rusty just said. And the impact is undeniable. So we'll talk a little bit about that in just a minute. But first, I guess a basic question back to you, Gene. The Foundation Fighting Blindness has been around for now 55 years, and in that 55 years we've been delivering mission through a particular blueprint, set of priorities, set of objectives. Why was it important to create this new model? Why did we need to do this in the first place?

Dr. Eugene de Juan, Chair, Gund Vision Fund:

Well, I think as we changed from discovery and early laboratory demonstration of potential therapeutics to actually going into patients and participating in these companies all the way through, it's a much different financial burden, and we can't do it by ourselves. So if you like, the idea costs nothing, then the laboratory, let's just say it costs a dollar. Getting it into the clinic would cost more than $10. Getting it to patients after it's approved is again another order of magnitude. With the Gund Vision Fund, the way I like to think about it is, I give a dollar, and every year I can spend that dollar again and again and again and again. So it's very compelling as we all want to support this. We all want our family, our friends to get better, and we're willing to support it, but it's really great when we can support more and more.

That's what the Gund Vision Fund is trying to do. It's been very successful. I'm going to make it real simple, and I apologize for the financial people like Rusty that can correct me, but the way I see it is, we've spent $100 million already. We have a few million to continue to invest, but today we've spent $100 million. We've already gotten, in a short seven years, already gotten $30 million back that is coming so we're able to invest more. We have in the near term, one or two years, I'm hoping, another $30 million coming from those investments. So we're over half, that $1 is already 60 cents back. And then how we want to calculate it, after those two years, there's probably another $30 to $60 million more. So we are able to spend that dollar again and again and again. So wonderful, wonderful strategy. It takes a lot of people's work, a lot of effort, a lot of discipline, but we're doing it successfully, and I'm very happy to be a part.

Jason Menzo, Chief Executive Officer:

Not just the financial returns, but we're getting those financial returns while advancing the mission and getting clinical milestones achieved along the way. Gene, actually, I'm going to follow up with just one other aspect to it. We've invested $100 million, we've gotten $30 back, but the $100 million we've invested is leveraged, and I want to see if you could speak to this component of it, by attracting other investors when we put money to work in a company. Can you talk a little bit about the impact of our investment and how it extends way beyond just the dollar that we invest to bring other investors alongside us?

Dr. Eugene de Juan, Chair, Gund Vision Fund:

Yeah, just to comment and then I'll turn it to Rusty for more detail. For every dollar we put in, we get $20. Our impact is so great and so valuable that people follow behind us or partner with us 20 times. I don't know if that number is exactly right, but close to 20 times, every dollar is leveraged. So it's huge, very, very important. We couldn't do this by ourselves.

Jason Menzo, Chief Executive Officer:

That's right. Rusty, let me throw it to you really, I guess, two questions, and then we'll open up to the general Q&A in a second. But today we've got 20 companies that we've invested in. Maybe you could share with the audience what our portfolio looks like today, and then ultimately if you imagine what the next 5 to 10 years looks like, and what gets you so excited about the future holds and how the fund is going to be delivering mission.

Dr. Rusty Kelley, Managing Director, Gund Vision Fund

Thanks, Jason. I think this question is such an important illustration of how we bring together all of the expertise from the foundation, from the great minds of the board of the Gund Vision Fund. It is very intentional that we must diversify our portfolio, early, mid, late-stage intervention strategies across the spectrum of inherited retinal diseases, including retinitis pigmentosa, the spectrum of dry age-related macular degenerations, including its severe form, geographic atrophy, and then across the various therapeutic strategies or modalities that are being used to intervene.

In therapeutics, the failure rate is quite high, so you must cast a broad net. So when I mention across therapeutic strategies, we're talking about gene-specific therapies like DNA augmentation and RNA editing technologies. Importantly, the gene-agnostic interventions, small molecules, biologics, cell therapies that can either help delay progression and/or restore with a cell therapy. And of course, now we're in this new age, we're also investing in artificial intelligence-enabled platforms.

So the portfolio, as Jason mentioned earlier, the field is maturing, and it will continue with the discovery efforts, but we haven't seen this level of clinical intervention yet in our field. Our portfolio reflects that. As Gene said earlier, we're only seven years in, and we just experienced perhaps the worst biotech market in U.S. history, and here we are with a lot of clinically staged assets.

So we have, as Amy mentioned earlier, Opus Genetics. It has a number of programs that are in the clinic, several that are reading out positively. We have Atsena Therapeutics, which is really trailblazing along with Opus in terms of moving the regulators towards more progressive thinking. We have Beacon Therapeutics, as Amy mentioned earlier, that just read out its registrational trial for RPGR. So these are clinically staged leaders in our space. We also have the gene-agnostic clinical strategies that are in the clinic, including Nacuity Pharmaceuticals with a neuroprotectant that can slow progression, Sparing Vision that is using gene therapy to express neuroprotectants that are cone specific, very unique strategy that is beginning to see some interesting results.

And then we have the dry AMD strategies that are in the clinic, Perceive Bio and Osanni Bio. Osanni hasn't disclosed its technology, but Perceive Bio, which Gene de Juan is a founder of, is using gene therapy to express a complement factor that contains a protective allele. So, a very unique set of diversified clinically staged assets.

And then we have the non-clinical or earlier stage companies, including RNA-editing pioneer, Amber Bio. We have a very novel biologic, NVasc for dry AMD, and that's focused on creating a healthy vascular bed in these areas of atrophy in age-related macular degeneration. And then we've recently added another gene-agnostic or cell-based photoreceptor-specific-based therapy in Agnos Therapeutics, an early stage company, but it tells you that we are attempting to diversify our portfolio to late-stage intervention strategies.

And then recently, we invested in Salution Health. This is an AI-based ophthalmology services company, and I think we're really excited about what that company can do for the field as the artificial intelligence and machine learning technologies continue to improve. So these investments intentionally balance near term value-creating events with these longer-term discovery and breakthrough opportunities.

Jason Menzo, Chief Executive Officer:

That's great. Thank you, Rusty. And thank you, Gene. Fantastic. We could easily spend half a day just talking about the Gund Vision Fund, the portfolio, all the incredible technology that's being developed and delivered through the portfolio, and honestly, how the model is, what I'd like to think of, is the future of philanthropy, because this is a way where donated dollars are being maximized, leveraged, and then returned to play over and over and over again.

But we are going to shift. We've only got about 15 minutes left, so I'm going to ask Maddie in just a second to remind our audience how to ask questions. Before you do that, Maddie, though, I do want to address one thing that's very topical from my friend Eric Bernardo. So hi, Eric, just chatted in a question about the types of people who might be interested in donating or participating in the fund. I just want to speak to that because everyone who's listening to this might be thinking, "Gosh, this sounds incredible. How do I participate? What can I do?"

So first of all, individual donors or philanthropists, family offices, foundations, anyone who donates to the Foundation Fighting Blindness has the ability at a certain dollar amount, so it's a high-dollar type of gift that is eligible to be used in this fashion, but any of the audiences, individual donors or philanthropists, et cetera, can donate to the Foundation Fighting Blindness and have their donation restricted for use by the Gund Vision Fund. On top of that, if you happen to work in the venture capital space or healthcare investing, we would love to also connect with you, because in addition to bringing capital into the fund, we're always looking for other investors to co-invest alongside us in the companies that we're investing in. As Gene said, we're successful at leveraging our investment by bringing other investors alongside us. So if you're interested in learning more, certainly contact us offline at info@fightingblindness.org. All right, with that, Maddie, please remind folks how to ask questions.

Maddie Mossman:

Thank you, Jason. So there are several methods that you can use to ask questions. You can submit them through the Q&A function at the bottom of your Zoom screen. Please make sure that you include your name so that we can follow up with you afterward. You can also send an email to info@fightingblindness.org, and we will follow up with you in the next week.

Jason Menzo, Chief Executive Officer:

All right. Thank you, Maddie. I'm going to ask Amy, Jeff, Peter, all the team, rattling off everyone, but come off mute, turn your cameras on. First question I'm going to direct to you, Amy. There are way more questions than we're going to have the ability to get to today. As Maddie said, which is a good thing, we'll follow up with every single person. But one common theme, Amy, is around retinitis pigmentosa and the current landscape of advances both in the lab and in the clinic. Rusty mentioned the terms gene-agnostic versus gene-specific, and maybe you could give a quick primer on what's happening with RP.

Dr. Amy Laster, Chief Scientific Officer:

Thank you. This is Amy Laster. When we look at the Foundation Fighting Blindness clinical trial pipeline, it's really clear that we do see these two complementary strategies that are emerging for retinitis pigmentosa, so that's the gene-specific therapies and the gene-agnostic therapies. What's important is that they really are together creating the most diverse RP development in the landscape really than what we've ever had.

So on the gene-specific side, some of the most advanced programs are really targeting well-defined genetic causes of disease. Like earlier, we talked about the RPGR-associated X-linked RP from Beacon. There's also therapies from MeiraGTx that are in late-stage development. There are also clinical trials for genes such as CNGA1 or RDH12. These approaches, they really aim to replace or repair the specific gene-causing disease and represent a precision medicine at its best.

But at the same time, we're seeing a lot of exciting progress with gene-agnostic approaches, which really seek to benefit people regardless of their underlying mutation. Some of the more advanced examples of this is like Ocugen, their OCU400, which is a modifier. It's a gene therapy, but it's based on a molecule, if you will, that's currently in a Phase 3 trial. Unlike traditional gene replacement therapies, these programs are really designed to regulate multiple pathways that are involved in retinal health, particularly the OCU400. This is potentially making it relevant across many forms of RP.

So other notable gene-agnostic programs is like Sparing Vision, their RdCVF therapy, which is in a Phase 1/2/3. We're also seeing that the pipeline includes mutation-independent approaches like neuroprotective treatments like Nacuity or NAT, optogenetic therapies that we've heard about, Ray Therapeutics, NanoScope, and restorative strategies from BlueRock, which are cell-based strategies, or from JCyte or Sumitomo, as we mentioned earlier. So overall, the key message is that the RP field is not just relying on a single development strategy. There are advanced gene-specific programs as well as gene-agnostic therapies.

Jason Menzo, Chief Executive Officer:

Thanks, Amy. Questions like Richard Ingram just chatted in, and five minutes ago, Diana emailed in about this exact point. So what if I don't know my gene? What is available that could potentially benefit a broad range of audiences? This is the exact point.

The question around, which we get a lot, and Diana, this was your exact question, around how do I find out about clinical trials? How do I enroll in a clinical trial?

The absolute best resource that exists is going to fightingblindness.org and navigating to our Clinical Trial Pipeline. Chris, if you could put that in the chat, the direct link to it. Because at our Clinical Trial Pipeline, we not only segment, these are the treatments that are in development for gene-specific approaches, and these are the specific genes that are being targeted, but also, these are the gene-agnostic approaches that would potentially benefit individuals regardless of what gene is causing their condition. In each case, there's contact information for the sponsor so you can contact them and understand how to potentially get involved in a clinical trial. Of course, we always recommend talking to your ophthalmologist and getting clinical guidance from a medical professional before enrolling in any clinical trial.

With that said, Todd, I want to go to you next, Dr. Todd Durham, who leads our clinical, and a whole lot more about, our clinical programs. Todd, can you talk a little bit about genetic testing? We had a question from Steve earlier and his brother who is... Many people are thinking about this, like, "How do I get genetic testing? I may not be able to afford genetic testing." So perhaps you can talk a little bit about our program and how individuals can get no-cost genetic testing and what that looks like.

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

Thank you, Jason. This is Todd Durham. To answer that question, we have a program called the My Retina Tracker Genetic Testing Program. Chris Adams is going to paste the link here in the chat box for people who would like to access that information. But through this program and a provider who would order the test for you, you can access a no-cost genetic test. This is a clinical panel with 110 genes on it that we have developed with our lab partner, PreventionGenetics. So this does require a workup by a clinician to do that.

One of the great things about the program that we offer that addresses some challenges that have been around for a long time, we have hundreds of providers across the United States who are able to order through this program. It's not just a specialist in inherited retinal disease, so that's a great aspect of the program, but you do need to go see a medical provider for this. This is not a consumer-facing test, and we do recommend and require that you have genetic counseling once you complete one of these tests.

Participating in this program automatically enrolls you in My Retina Tracker Registry study itself. And that, as Rusty alluded to earlier on this call, is one of the ways that we can help match individuals in our registry with research opportunities, including clinical trials. We think it's a highly valuable program for that, and we look forward to hearing from you if you have questions. We have much more information on the website about that. As Jason said, we encourage you to continue to keep open the dialogue about changes in genetic testing, a clinical panel like ours and others that are available through these programs don't address all potential molecular changes that account for disease, and for that, there are more comprehensive tests and tools available.

Jason Menzo, Chief Executive Officer:

That's great. Thanks, Todd. All right, we've got time for probably two more questions. The first one came to us from Dan Day down in Florida. Hi, Dan. I'm going to throw it to you, Peter. Dan was asking, "What does it mean when we say that a company is an Outreach partner for the Foundation Fighting Blindness? What does that mean?" And then, Gene, I'm going to come to you at the end to talk a little bit about AMD.

Peter Ginsberg, Chief Operating Officer:

Hi, this is Peter Ginsberg. For Dan and the group, the Outreach partners play a critical role in funding the Foundation's educational and awareness building activities as well as other programs, so a really important part of the Foundation's activities. An Outreach partner is able to have its logo or name on our website, and you can see the list of all of our more than 20 outreach partners on the fightingblindness.org website in the About section, under Corporate Partners. Then also, their names are associated with our Vision Connection seminars and our quarterly vision webinars. And then companies are also able to send out a partner news email to the Foundation's community, perhaps to help to enroll a clinical trial to help you be aware of clinical trials that are ongoing that might be of interest to you or a family member or friend.

But we also have a lot of companies that are Outreach partners that use those outreach emails to develop a patient advisory board. We think this is really important that companies early on in their clinical development reach out to patients to hear from patients as to what's the most important thing about clinical trials, what's important to patients, how should these companies develop paperwork that is digestible for the patient community. So we have a lot of companies that use those outreach emails to connect directly with folks like you that can provide guidance as to how best to design their clinical trials and how best to develop their products. So these Outreach partnerships are a great tool for our community and for the Foundation. And again, you can see the list of Outreach partners on our fightingblindness.org website in the About section.

Jason Menzo, Chief Executive Officer:

That's great. Thanks, Peter. So to close today's call, Dr. de Juan, I want to come back to you. We had countless questions around age-related macular degeneration. So what I thought would be good is briefly if you could talk a little bit about, we hear geographic atrophy, GA, wet AMD, intermediate AMD, dry AMD, what do all these terms mean, and then also any thoughts you have on the treatments that are available in the market today. We've got complement inhibitors, we've got photo biomodulation treatments. So what are your thoughts on what's actually available today and what's in the pipeline?

Dr. Eugene de Juan, Chair, Gund Vision Fund:

So the treatments for macular degeneration have been, over the past 20 years, astounding. When I started, it was a disease of aging, and grandmother had it, and she was old, and she was going deaf, and she was going blind, but that was just because she was getting old. And then about 20 years ago, the anti-VEGFs were identified and made into drugs. The first one that was really effective was Lucentis, and it made patients significantly better and doubled their vision. I thought, "How's that possible?" I didn't even think it was possible, and it was really one of the personal events of my professional career that that I saw something that must have been like penicillin.

Now, wet macular degeneration is very well handled. It's not perfectly handled, but it's very well handled. It takes intense therapy. But for the geographic atrophy, which is the dry form, the severe form of the dry, which is really a progressing atrophy, there was really no treatment until two or three years ago when the anti-complements, we knew that there was a genetic risk. If you had this complement risk, that you were much more likely to get this disease. So the anti-complements were started really more than 10 years ago, but just three years ago, they became approved. Different than the treatment with Lucentis for the wet form, it just slows down the progression. So it's not immensely satisfying, but it is something that is good and is giving us a lot of hope that if we started earlier, could we slow it down enough to prevent the severe visual loss? And it's still the number one cause of visual loss in the United States and Europe.

Those are the treatments for the photo biomodulation. It's very interesting. It's for early stage. It has to be repeated. It seems to work, but it has to be repeated, and it has a modest effect early. So it's not quite the prevention that we would like, but all of these things are stepping up. In the future, almost everything that we're doing except exact gene therapies, having all the neuroprotection, hopefully it'll work for retinal degenerations and including geographic atrophy, also glaucoma actually, and maybe for other things like brain diseases. The therapies that are targeted towards end stage, returning vision for patients that have very advanced disease like optogenetics in both cases, in RP and also advanced GA, there's hope from these strategies. But there are many more.

My personal desire is that we prevent progression to the late stage of the disease, and that's where the focus on intermediate is so important. Intermediate is the stage of disease where you're at risk of either getting the wet form or the dry form and losing vision. Before that, you might have a sign or two of the disease, but you're not at risk.

Jason Menzo, Chief Executive Officer:

Yeah, that's great. What a great way to end a great call today. Dr. de Juan, thank you for joining us today. Dr. Kelley, thank you for joining us today and all of our guests.

As we wrap up today's call, I do want to remind you that this is, actually, not just yet, but next week, when it turns October 1st, October is Blindness Awareness Month, and there's a ton of content and educational forums and material that the Foundation Fighting Blindness puts out in the month of October associated with Blindness Awareness Month. So please, if you're not already following us on social media, please do so. Share, like, engage with us. There's a ton that we're going to be putting out next month. And of course, the best resource for all things related to this community is fightingblindness.org, where we provide an incredible amount of information, resources, including our mental health hub and access to mental health resources, which is super important.

So with that, I'm going to turn it over to Maddie to wrap things up for today's call.

Maddie Mossman:

Thanks, Jason. We would like to thank everyone for participating in today's call. As a reminder, there will be a transcript and audio recording of today's call within the next week on our website, www.fightingblindness.org. We invite you to follow us on Facebook, Twitter, LinkedIn, Instagram, TikTok, and Threads to stay informed on the latest news and activities from the foundation. You can like and share Foundation posts on your own social media channels to help spread word throughout the month. If there is any other information you need, please reach out to us by sending an email to info@fightingblindness.org. Thank you, and have a great day.

Jason Menzo, Chief Executive Officer:

Thanks, Maddie. Thanks, everyone.