Recording Available: Insights Forum | Wednesday, August 27, 2025

Foundation Fighting Blindness
Insights Forum Transcript
August 27, 2025

Amanda Bement, Development Operations Specialist:

Hello, everyone, and thank you for joining today's Insights Forum. 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 controls are at the bottom of the Zoom window. This control bar may collapse when it is not in use. If you would like to prevent the controls from hiding automatically, you can use the following keyboard shortcuts to switch between the Always Show Meeting Controls options. If you are using Windows commands, press the Alt key, and 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, which is also on the Zoom toolbar. 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 few weeks, 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:

Well, thank you very much, Amanda, and good morning everyone. Thank you for joining us today. My name is Jason Menzo, and I am the CEO here at the Foundation Fighting Blindness. And of course, we're so glad to welcome you to our quarterly Insights Forum. These calls are designed to provide you, our global community, updates on key strategies and initiatives, highlights of the progress taking place in our field, and of course, to report out our performance against our mission of advancing more treatments and cures for blinding retinal diseases.

With that in mind, our agenda today is going to start with Mr. Jeff Klaas, who is our Chief Strategy and Innovation Officer. Jeff is going to highlight our many ongoing outreach and community engagement initiatives occurring across the country, and really across the globe. And then Mr. Peter Ginsberg, our Chief Operating Officer, will highlight several new corporate partnerships. He will review our full fiscal year 2025 financial performance and then provide a snapshot of the fiscal year 2026 budget plans and projections for the year ahead.

And as a reminder, the Foundation operates on a fiscal year that goes from July 1st through the end of June, and so this will be our first time sharing our full-year fiscal 2025 results, and of course, a preview for the year that we just started a month or so ago. And then after Peter, Dr. Amy Laster, who is our Chief Scientific Officer, will report on our newly announced science awards, key takeaways from our Innovation Summit, and recent progress in the clinical trial landscape. And then finally, after Amy, I'll come back to the call and share some of our accomplishments from this past year and the aggressive goals that we have for the year ahead. And of course, as always, despite the fact we have a full agenda, we will dedicate a significant amount of time at the end of the call for questions and answers. And Amanda just shared the Q&A process to type in your questions, and we'll review that again before that session starts.

In addition to the speakers that I just mentioned, we have a few additional members of the Foundation's leadership team that are joining us for the Q&A session. So if you have questions, either as it relates to clinical and outcomes research, we're going to be joined by Dr. Todd Durham, who's our Senior Vice President of Clinical and Outcomes Research. And if you have thoughts or questions about the RD Fund, we're going to be joined in the Q&A session by the Managing Director of the RD Fund, Dr. Rusty Kelley. So with all that said, I'd like to kick things off officially, and turn the call over to our Chief Strategy and Innovation Officer, Mr. Jeff Klaas.

Jeff Klaas, Chief Strategy and Innovation Officer:

Thank you, Jason, and good morning everyone. I'm pleased to join you today on the Insights Forum. Our Strategy and Innovation team is focused on expanding awareness of our mission, growing community engagement, and strengthening fundraising. For these efforts to succeed, we need your participation. I'd like to share a range of opportunities for you to get involved. First, on a local basis, we're committed to recruiting and retaining community-based grassroots chapter leadership and membership. Our chapter network has three areas of focus: education, resources, and revenue. Chapters hold seminars and meetings that provide research information, low-vision resources, and other industry topics, and provide outreach to retinal practices and eye care professionals in local communities. Through our VisionWalks, dinners, sporting events, and do-it-yourself, DIY, fundraisers, our 45 chapters help accelerate our mission by raising revenue to fund critical research.

2025 marks the 20th year of VisionWalk, our flagship fundraising program. And in the past two decades, VisionWalk has grown from 11 events in its first year, to a robust nationwide program with 33 annual events across the United States. There's still time to help us and honor this milestone, by participating in an upcoming VisionWalk, forming a team, or simply donating. We have 13 walks scheduled through the end of this year, across the country, from Boston to San Diego, so to learn more or find a VisionWalk near you, please visit visionwalk.org.

We're also proud of our signature fundraising events, including Scramble for Sight golf tournaments and Night for Sight dining events. These gatherings combine fun, community, and philanthropy, raising significant funds for sight-saving research. Beyond fundraising, we're hosting more than 60 Vision Connection events nationwide, featuring speakers, discussions, and networking. Details are on the events page at fightingblindness.org. Online, we also offer free quarterly vision webinars with expert speakers. The next session is Saturday, September 20th, on thyroid eye disease, so register at our events page.

Another way to contribute is through our Vision Warrior Program, formerly known as Raising our Sights. This program enables individuals to transform their passion into purpose, with fundraising events, from concerts, fitness challenges, to birthdays and barbecues. Vision Warriors are change-makers across the country. We provide event guides, and you can design your own campaign by visiting the Get Involved tab on our homepage.

Looking ahead, our United in Vision global community conference will take place June 12th and 13th of next year, in Fort Worth, Texas. This landmark event merges the Foundation's Visions Conference with the Retina International World Congress, creating a global forum that will unite patients, families, researchers, clinicians, and industry leaders, featuring sessions, exhibits, and new technologies. Our keynote speaker is Paralympic champion, Anastasia Pagonis, a powerful advocate for resilience and inclusion. Registration's now open at fightingblindness.org/Visions-2026.

In closing, from VisionWalks and signature events, to Vision Warriors, webinars, conferences, there are countless ways to be part of our mission. I encourage each of you to take action today, attend, volunteer, fundraise, donate. Please go to our website, fightingblindness.org, to find the opportunity that works best for you. Together we can accelerate research and change lives. Your involvement makes all the difference. With that, I'll turn the program over to Peter Ginsberg, our Chief Operating Officer.

Peter Ginsberg, Chief Operating Officer:

Thanks, Jeff, and good morning everyone. First, I'd like to highlight several new corporate partnerships that help us fund important Foundation programs. We recently welcomed two partners that are supporting our mental health initiative, and that includes Astellas Pharma, a global life sciences company, and maker of IZERVAY for the treatment of geographic atrophy. And also Genentech, a member of the Roche Group, which has a broad portfolio of treatments, such as LUXTURNA, LUCENTIS, SUSVIMO, and VABYSMO for vision-threatening conditions such as inherited retinal diseases, wet age-related macular degeneration, and diabetic eye diseases.

In May, we launched our Mental Health Resource Center, that's online, for the blind and low-vision community. This site offers tools, resources, and connections to help individuals and families manage the unique emotional complexities associated with vision changes, this includes informational articles, videos, and infographics.

Importantly, through our partnership with ALMA, which is a national network of mental health service providers, we provide a list of licensed therapists who have completed training provided by our external experts, and have received certification for supporting blind or low-vision patients. This list is growing as more therapists complete the training.

In conjunction with the launch of our Mental Health Initiative, we also hosted a quarterly Vision Webinar on the topic of mental health solutions for our community. That webinar replay, and the other sources of information I just mentioned, are available in the Mental Health Resource Center section of our website, which you can navigate to from our homepage by clicking on Living with Vision Loss.

I'm also pleased to report that the Foundation recently announced a partnership with Clinical Enrollment, which is a firm providing novel clinical trial recruitment resources for retinal diseases, among other areas. This collaboration is designed to optimize the trial recruitment process by providing an excellent experience for potential trial participants and reducing recruitment timelines for trial sponsors so they can get their therapeutics to patients quicker. By pairing the Foundation's 42,000-plus member My Retina Tracker Registry database with the services offered by Clinical Enrollment, individuals with retinal diseases will have an easier path to participating in clinical trials. By the way, if you or your family member has an inherited retinal disease and is not already part of our registry, you can find more information on our website, under My Retina Tracker Registry.

By being a part of our registry, you, or your affected family member, may have opportunities to be connected with upcoming clinical trials. And at last count, there are more than 50 clinical trials being conducted globally, and with the Foundation's continued research funding, we expect that number to grow in the coming years. If you haven't already completed genetic testing, we encourage you to talk with your doctor about the Foundation's genetic testing program, which offers individuals with a clinical diagnosis of an IRD, access to high quality genetic testing, genetic counseling, and connection to the registry, importantly, at no cost to the patient.

Now, I'd like to provide a brief summary of our fiscal year 2025 financials and our budget outlook for fiscal 2026. The Foundation operates on a fiscal year that runs from July to June, so we just completed our fiscal 2025 on June 30th. I'm pleased to report for fiscal 2025, our preliminary unaudited financial results indicate that we exceeded our budgeted $13.5 million net fundraising surplus for the year. Now, a key driver of our FY '25 results was legacy giving, and since August is national Make-A-Will month, it's worth highlighting that legacy giving plays a critical role in the Foundation's ability to fund impactful retinal disease research each year. If you're interested in leaving a legacy gift to Foundation Fighting Blindness, please go to fightingblindness.org/legacy-giving. Again, that's fightingblindness.org/legacy-giving.

Overall, preliminarily, we invested more than $31 million in retinal disease research and education in fiscal 2025, and that does not include $8 million in fiscal 2025 investments in promising emerging retinal disease companies through the RD Fund.

Now, following our regular annual audit, our audited fiscal 2025 financial statements will be available this fall. Finally, for fiscal 2026, which is the current fiscal year we just started, we're targeting $37.9 million in unrestricted revenue against $24.4 million in operating expenses, for a $13.5 million net fundraising surplus. I'd like to note that in addition to individual donations, which make up the majority of our revenue, corporate sponsor revenue is expected to grow further in fiscal 2026, to exceed 10% of our overall unrestricted revenue compared to less than 2% five years ago, and that's a positive sign of the increasing number of organizations committed to our cause.

For more information on our financial reporting, you can go to the Foundation's website About Us section and navigate to Financials and Governance. I'm now pleased to turn the call over to Dr. Amy Laster, our Chief Scientific Officer, for a research update. Amy.

Dr. Amy Laster, Chief Scientific Officer:

Thank you, Peter. Today, I'd like to cover three topics: newly announced science awards, some key takeaways from our Innovation Summit, and recent progress in the clinical trial landscape. I'm pleased to report that for our individual investigator research award program, last year, we received 77 letters of intent, or pre-proposals, and this led to 23 applications that were reviewed by our Scientific Advisory Board, who is comprised of more than 60 top clinicians and scientists from around the globe. And from the applications review, there were seven newly-funded research programs really poised to advance the field of retinal disease and therapy development. I want to highlight what these programs will add to our research landscape. These awards will deepen understanding of genetic mechanisms underlying retinal diseases, specifically those caused by RP1 and TMEM216. This really helps to clarify disease pathways and inform target intervention. Also, to accelerate therapeutic development for conditions linked to EYS mutations, potentially paving the way for novel treatment strategies. Identify robust biomarkers, which helps to monitor disease progression and evaluate treatment efficacy, enhancing clinical trial design and personalized care.

Improving our gene classification tools, which will enable faster and more accurate identification of disease-causing variants, and also uncover rare genetic contributors to retinal diseases, expanding the number of individuals who receive a confirmed genetic diagnosis, and informing future therapeutic targets. Together, these projects address critical gaps in retinal disease research, and lay the groundwork for transformative advances in diagnosis, monitoring, and treatment. In upcoming weeks, we will publish to our website, and on our social channels, these, and all newly funded grants from our past fiscal year. I also want to share that in addition to approving these awards, our science committee of the board spent some time discussing the known and potential impact to federal funding for research in our field. The committee agreed to remain flexible in current and future budget allocations, and to hear recommendations by staff, as we learn more to adopt either new or temporary funding mechanisms that will support sustaining promising research with urgent needs, to avoid any permanent consequences.

With that said, I am very pleased that we anticipate funding more than $15 million in new preclinical awards alone over this current fiscal year. Also, I want to highlight that in May, the Foundation hosted our 10th annual Retinal Therapy Innovation Summit, this is alongside Casey Institute at the Oregon Health and Science University. The Summit is held prior to the annual meeting of the Association of Research and Vision and Ophthalmology, you may hear this as ARVO. With more than 400 industry professionals in attendance for this sold-out event, the Summit provided a special focus on clinical development and trials for retinal disease therapy, and have broad agenda, featuring 28 presentations by retinal experts from around the world, on topics ranging from retinal gene and RNA editing, natural history studies, endpoints, stem cell therapies, gene therapy, artificial vision and for the first time, the Summit included presentations on emerging small-molecule treatments. A summary of the presentations can be found on the Foundation's website.

I'll wrap up my remarks today with some noteworthy clinical development from the past several months. The highlights include Atsena Therapeutics, which reported positive results from Part A of their Lighthouse Phase 1/2 clinical trial for its X-linked retinoschisis, or XLRS gene therapy. Atsena has received agreement from the FDA to expand the current study into a continuous Phase 1/2/3 combined clinical trial, and this may reduce the time to potential approval. Also,

Beacon Therapeutics reported vision improvements for patients receiving the company's X-linked retinitis pigmentosa, XLRP, gene therapy, and its Phase 2 DAWN clinical trial. Beacon is currently enrolling approximately 75 male XLRP patients for its Phase 2/3 VISTA clinical trial.

Johnson & Johnson reported results from its Phase 3 LUMEOS clinical trial of bota-vec, a gene therapy for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. And while the study did not show a statistically significant improvement in the primary endpoint, many treated patients showed improvements in vision, as measured by a low-luminance visual acuity testing, as well as several other imaging testing, like static perimetry and visually-guided mobility assessment.

PYC Therapeutics reported positive results from two Phase 1/2 clinical trials in the US and Australia, for its emerging RNA therapy for people with retinitis pigmentosa-11 caused by mutations in the gene PRPF31. The company is planning to launch a Phase 2/3 clinical trial later in this year.

Opus Genetics reported positive one-year results from adult patients treated in the ongoing Phase 1/2 study of its lead gene therapy candidate for LCA5. The company expects to report three-month results from pediatric patients in the third quarter of this year.

Opus also received clearance to launch a clinical trial for a gene therapy for the treatment of Best disease, or vitelliform macular dystrophy. This is a rare inherited retinal condition causing macular degenerations due to mutations in the BEST-I gene. With this IND clearance, Opus Genetics plan to initiate a Phase 1/2 clinical trial later on this year.

jCyte has begun dosing patients in a Phase 2, sham-controlled, clinical trial for its emerging, gene-agnostic cell therapy designed to improve vision in patients with retinitis pigmentosa (RP). All trial participants will be dosed at the UC Irvine Gavin Herbert Eye Institute.

Ocugen has dosed the first patient in its Phase 2/3 GARDian3 clinical trial for its emerging gene-modifier therapy for people with Stargardt disease caused by mutations in the gene ABCA4.

And finally, Nanoscope Therapeutics submitted the first modules of a biologics licensing application to the FDA. This is for approval of its emerging optogenetic therapy for restoring some vision to people with advanced retinitis pigmentosa.

This wraps up my remarks today. If you want more information on these announcements, we provide details in the News section of our website, FightingBlindness.org, along with a comprehensive list of ongoing IRD-related clinical trials, which is available in the Research section of our website, under Clinical Trial Pipeline. As you can tell, there are new developments happening frequently, so please check our website often. I'd now like to turn the program over to our CEO, Jason Menzo.

Jason Menzo, Chief Executive Officer:

Well, thank you so much, Dr. Laster. It is always so exciting to track the momentum that continues to build from the labs, and all the work that we fund through our grants and awards, and all of our research projects, all the way through into the clinic, where real individuals and patients are being dosed with these groundbreaking and innovative therapies all over the world. And this is why we do what we do, for these great scientific innovations and incredible science fiction discoveries to translate from the lab to the clinic, and to the individuals who are affected by these blinding retinal diseases. I want to start my comments by thanking everyone who's here on the call today. Of course, the many researchers, the physicians, the donors, and all of our partners across the globe. The momentum that we're seeing in this field is all thanks to this togetherness, and this community working together to make meaningful progress every day towards accomplishing the mission of advancing treatments and cures for blinding retinal diseases.

Since our last Insights Forum back in April, it's been a very busy few months here at the Foundation. Spring and summer are always full of fundraising and community events, grant reviews for new research projects, due diligence in the RD Fund on potential investments in companies that are advancing our mission, research conferences where the global research community gets together, and of course, our internal staff drive to push to finish the fiscal year as strong as we possibly can. And what I wanted to do was spend a few minutes today talking a little bit about what we achieved last year. And again, as Peter mentioned, our fiscal year just ended at the end of June. So we're going to spend a few minutes talking about what we achieved last year, and then also looking ahead to what we expect to achieve in the year ahead, in fiscal year 2026.

And so what I want to start with is the resources that we have to drive our mission which all come down to the funds that we can raise and limiting how much we spend on things that are unrelated to the mission. When it comes to our organizational goal setting, we really start the process by looking at our financial goals. We then have science goals. We have engagement goals, to engage our community. We have operational goals, to make sure that our operations are as efficient and impactful as possible. And then we have culture goals. So those five areas are really the key areas that we assess when looking back at what we achieved in a prior year, or how we envision moving the organization forward in the year ahead. With that in mind, I want to share a little bit of a finer point on the information that Peter shared a few minutes ago on the financial front.

As Peter had shared, we significantly exceeded both our budget goal for revenue and stayed below the budget for expenses, which resulted in a robust surplus, and that is how we put resources, and even more money, to fuel the mission in the years ahead. Another key financial metric that we closely track is what percentage of our overall expenditures do we direct to our mission as compared to overhead or other expenses? For background, as a key rule, non-for-profits should direct at least 65% of all of their expenses towards their mission. Of course, here at the Foundation Fighting Blindness, we aim for a bar much higher than that. And in fact, when we look back at fiscal year 2025, greater than 76% of our expenses and all of our spending went directly to the Foundation's mission related to programs, things like funding research, or our no-cost genetic testing program, or investments that move the mission forward through investments made by the RD Fund.

This is a metric that we're very proud of, it speaks to how laser-focused we are at driving the mission forward, and really sets us as a model for organizations that do the type of work that we do. We want to continue to push that closer and closer to 80%, and perhaps north of 80%, but 76% is a good metric. As I mentioned, the floor is 65%, and we're well north of that.

To that end, we also had a strong year against our science goals. For example, our research funding led to nearly 20 specific research programs advancing to the next stage of clinical development, and that included seven programs gaining clearance to enter the clinic for the first time. That is why we do what we do. This is one of the most important goals that we track every year. Advancing programs that we've been actively funding or engaged with, to the next stage of development, into the clinic, through the clinic, through regulatory review, and ultimately, into the market.

And so that is a really important goal, and I'm really proud to repeat that seven programs gained clearance to enter the clinic for the first time this past year. That really does speak to the impact that we're all having together as a community, to rapidly move the mission forward.

One of the other accomplishments that I wanted to highlight from this past year is really around how we engage our community. Last year, we had set a goal to boost our online engagement by about 12%, and the results were really incredible, and I wanted to highlight that this past year, we exceeded that target by 134%, with online engagements with the Foundation reaching nearly 3 million. We recognize that people are engaging with organizations like the Foundation Fighting Blindness differently than they have in the past. And for that reason, we continue to seek new ways to engage with you, whether it be on our website, through social media, or forums like we are on right now, like the Insights Forum, or other webinars.

Now that said, I am still a very firm believer in the power of real live and in-person connection, which is why all the efforts that we have in engaging our community online are in addition to our in-person community events, like those that Jeff Klaas mentioned earlier in this call, things like VisionWalks and Vision seminars, and signature events like our Night For Sight dinner events and our Scramble For Sight golf events. We've invested in building communities across the nation, and now expanding outside the U.S., and for those reasons, we really do believe that in-person engagement is as important as ever before. But 3 million online engagements is pretty impressive.

One of the other areas that we were very active last year, and those who are close with the Foundation are not going to be surprised by this, because I feel like we've been putting a lot of communications out as it relates to our advocacy efforts related to federal policy affecting vision research.

As a team, we spent a lot of time on the Hill this past year, advocating for our mission, advocating for the importance of vision research. We even provided expert witness testimony to the U.S. Senate Subcommittee on Labor Health and Human Services, the subcommittee within the Senate Appropriations Committee, and we have been really actively engaged directly with many congressional leaders to highlight the importance of vision research as a federal priority. The testimony that we provided highlighted the strong partnership that we have with the National Eye Institute, and that together, we have led to transformative discoveries, including the first FDA-approved gene therapy for any inherited disease. For many, many years, the Foundation Fighting Blindness and the National Eye Institute have shared a close public-private collaboration to advance high-impact scientific discoveries and bring forth new treatments faster to patients. Vision loss from diseases such as retinitis pigmentosa, macular degeneration, and Stargardt disease affect tens of millions across this country, and cost the U.S. economy an estimated $134 billion annually.

One of the things that we've been working really hard to achieve on the Hill is making sure that policymakers understand how important our community is to this great nation. And so as we make this broader case for investment in vision research, we also believe that it's important to translate early-stage biomedical research into things that advance into the clinic, and ultimately, to the market, because one of the facts that is so important to recognize is that biomedical research is one of the highest-yielding commitments that our nation makes. It is estimated that for every dollar that the federal budget invests in the National Institutes of Health-funded research, $2.56 in new economic activity is created. For many regions of our country, National Eye Institute-supported projects also represent a major anchor in the local economy, at the local public or private university research institution.

We've been spending a lot of time on the Hill advocating for the importance of vision research and the independence of the National Eye Institute. In future Insights Forums we'll talk a little bit about new policies that we're creating and advancing on the Hill as well. And of course, we'll keep our community updated as this federal funding process proceeds. It is a challenging environment, I think everyone would recognize that, but we continue to do all that we can to impact the outcomes. Our ability to fund critical research is more important now than ever before. And as Dr. Laster mentioned, we are prepared to be flexible and action-oriented to address gaps as they arise, as the political landscape continues to evolve.

A few other highlights from last year: we implemented a new internal customer service function, which I was very proud of. That was under Peter Ginsberg's leadership. The aim there was to improve your experience when you call the Foundation on the phone. And of course, as we've previously discussed on these calls, this past year, a real highlight as it relates to partnering with an agency in the federal government, was the Advanced Research Projects Agency for Health, or ARPA-H, on a project called The Whole Eye Transplant. As a reminder, this past year, we announced that we partnered with several of the leading research institutions here in the U.S., to develop a roadmap to perform successful human whole eye transplants in the next six years.

These are all really great accomplishments, but the most important thing is how we are working together as an organization, and with our community, to advance this mission as quickly and efficiently as possible.

When we look ahead and we think about how do we maintain the momentum from last year and really build on that for fiscal 2026, we've established very aggressive goals across those same five dimensions. We've created very aggressive financial goals for the year ahead, very aggressive science goals to advance even more projects into the clinic and through the clinic, very aggressive goals around how we engage our community, how we streamline and make our operations as efficient as possible. And of course, continue to build on a world-class culture so the best and most talented people that are in the workforce want to work at the Foundation Fighting Blindness, so that we have the best and brightest working on this mission with us. We must continue the focus on all the positive momentum of the research we're funding, and then of course, celebrate the successes along the way.

And as we wrap up our formal remarks and move towards the question and answer period for today's call, I do want to take a moment to recognize a real incredible achievement by one of the world's most revered and respected experts in the retinal disease research field. I'm very pleased to report that one of our previous Insights Forum speakers, and exceptionally close collaborator here at the Foundation, Dr. Jose Sahel, was recently awarded the 2026 Proctor Medal from the Association for Research in Vision and Ophthalmology, which we always refer to as ARVO. This award, established in 1949, is a highly prestigious award, and honors outstanding research in basic or clinical ophthalmological sciences. Among his many achievements, Dr. Sahel established two of the foremost retinal research centers in the world, the Institut de la Vision in Paris and the University of Pittsburgh Medical Center Vision Institute in Pittsburgh, Pennsylvania. He currently serves as the endowed distinguished professor and chair of ophthalmology at UPMC, University of Pittsburgh Medical Center.

Dr. Sahel has been an active and insightful member of the Foundation's Scientific Advisory Board since 2001. He currently serves as the vice chair on our Scientific Advisory Board. He is also on the board for the RD Fund. We're so proud, and congratulate our friend and colleague, Dr. Sahel, on this well-deserved honor, and thank him for the many contributions he's made to the field, and for many bright years of collaboration ahead. With that said, that brings an end to our formal remarks. One of the things important to us, as we designed today's agenda, was to make sure that we had a good chunk of time for question and answer. The last couple of Insights Forums, we pressed right up close to the beginning of the hour, and didn't have as much time for Q&A. With that in mind, we've got about 20 minutes today for Q&A, which is more time than usual, which is great. I'm going to turn it over to Amanda to remind you how you can ask questions, and then we'll jump right in.

Amanda Bement, Development Operations Specialist:

Thanks, Jason. 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, or you can also send them in through chat. Please make sure that you include your name so that we can follow up afterwards. And you can also send an email to info@fightingblindness.org, and we'll follow up in the next few weeks. Thank you.

Jason Menzo, Chief Executive Officer:

Thank you, Amanda. We do have a number of questions that have been chatted in already, earlier today, or have been emailed in even before the session started, and so why don't we kick things off with a few of those before we get to those that are chatted in here in real time. Dr. Laster, one of the questions that we often hear from the community, and was emailed in from Shane Doucet, I think is how you say his last name, was an update on the NAC Attack clinical trial. I know many folks on the phone are probably curious about that as well, so maybe you can share an update on NAC Attack.

Dr. Amy Laster, Chief Scientific Officer:

Thank you, Jason. This is Amy Laster. So N-acetylcysteine, or NAC, is a drug that was studied at Johns Hopkins University, and they have launched a Phase 3 clinical trial, which is called NAC Attack, and it evaluates NAC for treating retinitis pigmentosa. The Phase 3 trial spans 45 months. It has completed enrollment actually, for over 400 patients across the United States, also in Canada, Mexico, and in Europe. NAC is an FDA-approved antioxidant that's used for other conditions, and researchers believe that it can slow vision loss by basically reducing some of the harmful reactive oxygen species in the retina. This is independent of the RP-causing gene mutation. The early trial showed that NAC was well tolerated and offered some small vision improvements.

There is a caution in taking NAC outside of the trial. NAC is available on most shelves of pharmacies, but there are unknown long-term effects and risks. As always, we advise that you talk to your retinal specialist and clinician before taking NAC. There is a related drug, NACA, being tested by Nacuity for Usher syndrome, and this is with support from the Foundation Fighting Blindness.

Jason Menzo, Chief Executive Officer:

Thank you, Amy. Another question that is somewhat related, and we get this question all the time, but specifically, the question is around what's the latest in cell therapies for RP generally. I think about NAC and NACA as being two examples of gene-agnostic approaches, cell therapy being another example of gene-agnostic approaches, and we have other gene-agnostic approaches as well. Perhaps you can speak to just the latest in the landscape on gene-agnostic approaches, including, and especially, cell therapy.

Dr. Amy Laster, Chief Scientific Officer:

Again, this is Amy. I'll start with stem cell therapy. These treatment approaches for retinal disease are really considered a potential therapeutic method, aiming to replace lost cells in the retina with stem cells. This is mainly for individuals with retinal diseases who retain some of their useful retinal ganglion cells. There are several types of stem cells, like retinal progenitor cells, human embryonic stem cells, and induced pluripotent stem cells. I mentioned earlier that Jcyte was launching a Phase 2 trial, so they use the stem cell-derived RPE cells. There's also induced pluripotent stem cells deriving RPE used in trials sponsored by the National Eye Institute, which have examined efficacy and safety in patients using, and during clinical trials.

Blue Rock Therapeutics has the first photoreceptor replacement therapy using an induced pluripotent stem cell-derived photoreceptor that's currently in a Phase 1/2 clinical trial. As Jason mentioned, stem cell, or cell-based therapies, is a type of mutation-agnostic therapy. And these are treatments that don't target a specific genetic mutation, instead, they really aim to restore or to preserve vision, generally addressing a common pathway of retinal degeneration, or by reprogramming some of the surviving retinal cells that make them responsive to light, like optogenetics. When we talk about Nanoscope or Ray Therapeutics, these are some companies developing optogenetic approaches. And then as we've already discussed, some of the small molecules, like NAC, or Nacuity, and Ocugen has a clinical trial with the gene modifier that is also gene-agnostic. These are some examples, in addition to cell-based therapies, of mutation, or gene-agnostic approaches.

Jason Menzo, Chief Executive Officer:

Thank you very much, Dr. Laster. And I guess I neglected to request that all of my other panelists come off of mute and turn their cameras on, because I'm going to start directing questions to each of you. Sorry about that, I should have made that call out. Everyone is here for the Q&A now, Jeff Klaas, Peter Ginsberg, Dr. Todd Durham, Dr. Rusty Kelley, and of course, Dr. Laster, who we just heard from. Dr. Kelley, Rusty, I want to direct a question to you. We often get questions about how does the RD Fund continue to push the mission forward from the Foundation Fighting Blindness? And maybe if I'm a participant on this call, I'm thinking, "Okay, so we hear about these investments that the RD Fund's making, but what are one or two meaningful advances that were made possible because of the RD Fund that have occurred maybe in the last couple months?"

Dr. Rusty Kelley, Managing Director of the RD Fund:

Thank you, Jason. It's always great to participate in the Insights Forum. I'll just remind the audience, particularly any new visitors to the Insights Forum, that the RD Fund is the venture arm of the Foundation, that leverages philanthropic capital to invest in early-stage biotech companies that are developing therapies for blinding retinal diseases, and specific to the FFB mission, inherited retinal degenerations and macular degenerations, including age-related AMD. The RD Fund has grown to more than $120 million in assets under management. It's invested in 16 biotech companies that have now attracted over a billion dollars in follow-on capital from more than 50 institutional investors, or co-investors of the RD Fund. These ventures span a broad range of therapeutics that Amy has discussed today and previously, including our hope to diversify our portfolio into these later-stage interventional strategies, like cell therapies that Amy just discussed. But our goal is to diversify a portfolio, and we've done a great early job of doing so with genetic medicines, and also protective approaches for treating these classes of diseases that we just mentioned.

As we approach our seventh anniversary as a fund, we have launched, financed, and accelerated some of the most promising ventures in retinal science. And these companies are not just advancing therapies, they're really reshaping what's possible for patients, and I'll give you a few examples of how that's occurring. A great example, and again, Amy mentioned earlier the recent news of both Opus and Atsena with various regulatory milestones, but also clinical success. Both of those companies, Opus Genetics and Atsena Therapeutics, the RD Fund helped launch, finance, and/or accelerate the building of a wonderful pipeline of inherited retinal disease gene therapy assets. As Amy mentioned earlier, Opus Genetics, now a publicly traded clinically-staged biotech company, is developing gene therapies for inherited retinal diseases, including its lead candidates, LCA5 and BEST1. The FDA acceptance of the BEST1 IND just a few days ago, is great news for the company, great news for the fund, the Foundation, and the field, as it allows the company, Opus Genetics, to bring its portfolio into the clinic more meaningfully, and especially for a disease like BEST1, which we think is highly addressable by gene therapy.

Another great example is Atsena Therapeutics, as Amy and others have mentioned previously. Really, the news that Amy shared regarding their FDA approval to expand their ongoing Phase 1/2 Lighthouse study for XRS into a continuous Phase 1/2/3 trial is monumental for the field. It shaves years off the development strategy and tens of millions of dollars. This is really important for the field. It sets the stage for orphan disease, especially when, in an early Phase 1/2 or a first in-human trial, that you demonstrate a well-tolerated approach to gene therapy that shows encouraging improvements in both retinal structure and visual function, then you have a much greater chance at these regulatory interactions that allow you to accelerate your programs. And the last thing I'll say, and happy to take additional questions on these topics, is that the RD Fund model leverages the Foundation in so many ways, and including its ability to invest in technologies that the Foundation funded through its granting mechanisms many years prior to the formation of these companies, including Opus Genetics and Atsena Therapeutics.

It's especially rewarding to us, the staff, and the RD Fund, and of course, we represent many others, including two wonderful board of directors that enabled us to do so. But to see some of the patient testimonies from some of these initial trials that enable these patients to do things that they could not do before is incredibly encouraging. And I think it speaks to the model that the Foundation was so wise to create this venture philanthropy model of the RD Fund, that really enables us to achieve clinical mission with our many co-investors, and of course, the capital that we need to advance these expensive therapies. I think the last thing that I will say is that the RD Fund has a dual mission, it's clinical and financial. And it's clinical first, of course. And if we are successful, or win, and we have been successful, that we have the ability to roll these financial returns back into new investments, is very, very powerful. So thanks for the question, Jason, and happy to take any additional questions.

Jason Menzo, Chief Executive Officer:

Great, thank you, Rusty. One question that actually was chatted in just a minute ago, and I'll take it, on the RD Fund, is a question which I love. Thank you Charlie for the question. Charlie chatted in "How do I donate directly to the RD fund?" I think that we all are appreciative of that question. The way that the funding of the RD Fund is set up is that ultimately, donations are realized as a donation to the Foundation Fighting Blindness. And in certain circumstances, if the donation is of a certain size, it can be restricted for use by the RD Fund. We'll get with you offline Charlie, and we can talk to you more specifically about that. We are in the middle of launching a lot of activities to raise more funds for the RD Fund specifically, because as Rusty said, this is a very expensive proposition to fund companies that are advancing clinical stage treatments and cures, so every dollar is really important to that end.

All right, we've got just a few minutes left, and I want to try to get to a handful of other topics. Dr. Durham, a couple questions for you. One, we always get a lot of questions around the registry, genetic testing, how do I get no-cost genetic testing via the registry, when should I retest if my positive gene wasn't identified five, seven, eight years ago? That's part one. The other part is maybe you can speak a little bit to the work that you and your team are doing around identifying appropriate endpoints for the clinical trials. We've spent a lot of time on this call talking about all these clinical trials that are occurring, how do we make sure that the field is using the right endpoints to measure the impact, so that way regulators know what programs they are actually working, and vice versa. So those all to you, Dr. Durham.

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

Thanks, Jason. And thanks for the question about the endpoints. First, I'll talk about the Registry. In Peter's prepared comments at the beginning of the call, he mentioned My Retina Tracker Registry, which is a study that we run in-house. And the primary benefit for people who are affected with inherited retinal degenerations of participating in the Registry is to be informed of clinical trial, and other research opportunities, as our partners engage us in assistance with that activity, and to keep up to date with research updates that will be relevant for you. And the more we know about your inherited retinal degeneration through your sharing your information with us, the more specific can those updates be. So we encourage people, if you have an inherited retinal degeneration, to sign up with the Registry. This is a study. We take your privacy seriously. You'll sign an informed consent, and fill out an information form about your condition. And you can register for that directly through our website, by looking for My Retina Tracker Registry.

And on that website there's also an email address. If you should have any questions or problems registering yourself for the study, you can contact the coordinator at coordinator@myretinatracker.org. There's a link there for you to click to send an email, and our staff is happy to assist your initial registration and any other questions you might have. Another route that people join the Registry is through the sponsored genetic testing program that we have. This would be another way to join the Registry. If you have not had a confirmatory genetic test or diagnosis in the last several years, and with a large panel, you may be eligible to participate in this program. And the best way to do that is to speak with your healthcare provider. This panel test has to be ordered by a provider here, in the United States, and you have to have had an initial diagnosis of inherited retinal disease to qualify for that.

The majority of people who go through that program, and if they have an IRD, they will get a confirmation genetically. There are a fair number of people who will come back with the information that indicates that they can't make a conclusive diagnosis from that test, and so we do receive questions from a number of people about what are my next steps as far as that goes. And the reason for those indeterminate, or null results, have multiple ways that can come about. Depending on what your result is, probably the best single recommendation I can make is to work with your genetic counselor if you have one, or your healthcare provider. Sometimes the panels are updated. Sometimes the database that's used to define what is a pathogenic variant can be updated, so that your diagnosis can be updated later even without doing another test. I'll just take one minute, Jason, in the interest of time, to address the other question, which was about what is the Foundation doing to encourage the development of novel endpoints to make sure that clinical trials are more successful.

We have a very large portfolio of studies that we're conducting through the Foundation Fighting Blindness Clinical Consortium. And that is their primary goal, which is to get a better understanding of disease progression so we can understand when should companies intervene in the disease process that matches the way that they believe their medication works. And how do you measure, using an endpoint in a trial, whether that new therapy is having the beneficial effect that you hope it does. We evaluate through our natural history studies, a number of measurement characteristics, like how reliable they are, how consistent, and whether they change over certain period of time. And we have been really pleased with the impact these studies have had thus far. There are at least three companies I know of who have made extensive use of the data from the RUSH2A study in particular, and that, I think, is a huge benefit to the community it's serving, to make sure those trial designs have much better information behind them, and therefore, more likely to succeed. Thanks for the question.

Jason Menzo, Chief Executive Officer:

Awesome. Thank you, Dr. Durham. We are just about out of time, but I do want to give Dr. Laster two final questions, a couple of final remarks. I do want to, before doing so, remind everyone that every question that gets chatted in, and there are many that we weren't able to get to, but every single question that gets chatted in or put into the chat, or sent in via the info@fightingblindness.org, we will follow up offline. There are some times where the questions are very specific or personal, and so we don't answer them live on this forum, and we follow up with you offline. If we didn't get to your question, don't worry, it's not that we didn't catch it, or that we ignored it, it's just that we're going to follow up with you offline in some instances.

With that in mind, we do have two final questions I want to pose to you, Dr. Laster. Our friend, Terry Pink Alexander, hi, Terry, thank you for the question, asked about what's the latest as it relates to Usher Syndrome Type 3. And then we had, and I apologize, I don't remember who it was at chatted it in, but maybe a little bit more color behind the activities around the whole eye transplant. I'll pose you those two final questions, and then we'll wrap up the call.

Dr. Amy Laster, Chief Scientific Officer:

Thank you, Jason. Again, this is Amy Laster. Related to Usher three, and specifically what's happening in the clinical trial, the Foundation funded some work with the Usher 3 initiative several years ago, to develop a small molecule that may prevent the progression of Usher 3. They have taken that asset, and they're working with a company in Australia and have approval to launch a clinical trial. It will be a 15-month clinical trial. It has not yet started, and so that is upcoming, to evaluate the safety and tolerability of this small molecule specifically for Usher 3. As we learn more about the launch of the trial, as well as the data from the trial, we will certainly put that out on our website and socials, and talk about it in forums like this.

And the next question, with regards to the whole eye transplant. There is a federal program, Jason mentioned a federal agency, the Advanced Research Projects Agency for Health, ARPA-H. They have funded several teams, including one led by the University of Colorado and Dr. Nia Washington, for a total human eye, what we allotransplantation, so THEA. This, again, established by ARPA-H, and the research goal for this is to develop an optic nerve regeneration strategy to do a complete whole eye transplantation. And this would serve as a cure for blindness in people with any ocular-based disease or damage. The Foundation has played a critical role in working with this team. There are nine research sites that are trying to understand how do you get the optic nerve, which typically does not regenerate, how to coerce it to regenerate, how to successfully get an eye from a donor and have it sustainable and healthy to be transplanted, and then the actual surgical technique of transplanting of the eye.

Many of these stages have been done before, but it's the optic nerve regeneration that is the challenge. And the team has been working on this project for about a year now. It's an aggressive project to have, within six years, this whole eye transplant successful in a human.

Jason Menzo, Chief Executive Officer:

That's a great way to end the call. Thank you, Dr. Laster. We often say here, in the Foundation, sometimes the work seems like it's science fiction becoming science fact, and this is a prime example of that. We'll end on that note. We have reached end of our time together today. I thought we definitely covered more ground in 20 minutes of Q&A than we typically do, but there should probably even be more time allocated to question and answer, because there's so many great questions from all of you in our community. With that said, I'm going to turn it back over to Amanda to wrap up the call.

Amanda Bement, Development Operations Specialist:

Thank you, everybody, for joining. Again, if you have any questions, you can submit them at info@fightingblindness.org. There will be a transcript and audio recording for today's call within the next week or so, on our website, www.fightingblindness.org. And also, please be sure 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 the word throughout the month. If there's any other information you need, please again, reach out to us at info@Fightingblindness.org. Thank you, and have a great rest of your day.

Jason Menzo, Chief Executive Officer:

Great. Thanks, everyone.