Recording Available: Insights Forum | Thursday, April 24, 2025

Guest Speaker

David Gamm, MD, PhD

Dr. David Gamm’s lab is at the forefront in developing cell-based therapies to combat retinal degenerative diseases. As the director of the McPherson Eye Research Institute and a member of the Waisman Center Stem Cell Research Program, the UW Stem Cell and Regenerative Medicine Center, and the American Society for Clinical Investigation, his efforts are directed toward basic and translational retinal stem cell research. 

Play video:

Foundation Fighting Blindness
Insights Forum Transcript
April 24, 2025

Amanda Bement, Development Operations Specialist:

Thank you everyone for joining today’s Insights Forum with Foundation Fighting Blindness. 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 prefer to prevent the controls from hiding automatically, you can use the following keyboard shortcuts to change your always show meeting controls option. If you are using Windows Commands, you can 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 will be no PowerPoint slides. Throughout the call, you will be able to ask questions via the Q&A feature on the bottom of the Zoom window. We will address these questions towards the end of the call. If we do not get to your question live, we will follow up over the next week or so, so please make sure to include your name within 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, very good, and thank you very much, Amanda. Good morning everyone. Thank you for joining us today. As always, I’m thrilled when we start these calls and I see the chats from all over the place at people saying, “Good morning. Good morning, morning, morning.” It’s always so exciting to have our community together here for our quarterly Insights Forum.

My name is Jason Menzo. I am the CEO here at the Foundation Fighting Blindness, and we really do appreciate you joining us for our first Insights Forum here in 2025. We actually have quite a few updates to share today and an outstanding guest speaker, so let’s go ahead and get into it.

For our agenda today, first up, we are going to introduce Jen Nielsen, who’s our Vice President of Field Operations, and Jen is going to share highlights on several programs designed to expand awareness of the foundation and our mission, increase community engagement and strengthen our fundraising capabilities in communities across the country. And then Peter Ginsberg, our Chief Operating Officer, will provide an update on notable corporate partnerships and announcements, and he will also review our financial performance for fiscal year 2025 through March. And then after Peter, Dr. Amy Laster, our Chief Scientific Officer, will summarize three categories of newly announced science awards and provide a snapshot of recent clinical program developments.

Then after Amy, I’ll come back on, I’ll share updates on two very important topics. Number one is our soon to launch mental health initiative that we’re very excited about. And then after that, I’ll provide an update on important federal policies that are either being proposed or are in action right now and their impact on our mission. And then to wrap up our formal remarks, we are honored to have Dr. David Gamm here today as our guest speaker. Dr. Gamm will discuss his innovative work in stem cell therapy in development for retinal diseases, including his research at the University of Wisconsin in Madison.

And then as always, after Dr. Gamm’s remarks, we will open the call up for Q&A, which is always one of the most exciting parts of this call. We’ve already got eight or 10 questions that have been sent in, and in addition, when we get to the Q&A session, in addition to today’s speakers that I’ve already mentioned, we will have several additional members of the foundation team joining us, including Dr. Todd Durham, our Senior Vice President of Clinical and Outcomes Research, Dr. Rusty Kelly, the Managing Director for the RD Fund, Chris Adams, our Vice President of Marketing Communications, and Jeff Klaas, our Chief of Strategy and Innovations. With that said, I’d like to kick things off and turn the call over to our VP of Field operations, Jen Nielsen.

Jen Nielsen, Vice President of Field Operations:

Thank you, Jason, and good morning everyone. I’m pleased to join you on this Insights Forum. In my role as VP of Field Operations, I lead the performance, growth and engagement of our chapter network, ensuring alignment with our organizational goals and empowering staff to execute high-impact fundraising initiatives and events. Today, I’ll provide a snapshot of two key programs that are designed to expand awareness of our mission, increase community engagement, and strengthen our fundraising capabilities.

I’ll start by highlighting a major milestone that the Foundation is celebrating this year. 2025 marks the 20th anniversary of VisionWalk, our flagship fundraising event dedicated to advancing treatments and cures for blinding diseases. In the past two decades, VisionWalk has grown from 11 events in its first year to a robust nationwide program with 33 annual events. Over the years, VisionWalk has brought together more than 235,000 walkers, donors and sponsors raising over $71 million to fund groundbreaking research. These collective contributions have helped fuel nearly 50 potential treatments into the clinical trial pipeline, offering real hope to individuals and families affected by vision loss.

Each VisionWalk event includes family-friendly activities and educational resource fair and a non-competitive, accessible walk route. Beyond fundraising, VisionWalk events create a strong community where individuals and families impacted by blinding diseases can connect, share their journeys, and learn from one another. We want to express our tremendous appreciation for the many people on our call today who have been involved in VisionWalk throughout the years. If you’ve participated or donated to a VisionWalk, please feel free to raise your hand. It’s amazing to see all those hands raised.

As we celebrate two decades of progress, we are also looking ahead. There’s still work to be done, and your continued support is essential to moving research forward. Join us in honoring this milestone by participating in an upcoming VisionWalk, forming a team, or simply donating. To learn more or to find a VisionWalk event near you, please visit VisionWalk.org.

Today I would also like to highlight a new initiative that we began last year called Vision Connection. Throughout the year, we host in-person events designed to inform and connect individuals affected by retinal diseases and age-related macular degeneration. Organized and hosted by our dedicated local chapter leaders in collaboration with the local community manager, these sessions feature exceptional programming, engaging Q&A discussions and opportunities to connect with others in the community. Distinguished guest speakers provide education on the latest advances in research. Topics include clinical trials, genetic testing, inherited retinal diseases, AMD, low vision and vision rehabilitation, and coping with progressive vision loss.

Each presentation is followed by an interactive Q&A session offering attendees the opportunity to ask questions and engage directly with experts in the field. We are committed to fostering a grassroots movement that engages volunteers in local markets. Our Vision Connection plays a crucial role in many chapters, serving as the kickoff for our VisionWalks and other events, and encouraging the community to raise funds to support critical research.

We will host approximately 60 Vision Connection events in 2025. To find an event near you, please go to the events page of our website, FightingBlindness.org. Whether you’re looking to attend an event, volunteer, or support our cause, we welcome your involvement. Join our efforts to drive vital research and make a difference in the lives of those affected by retinal diseases. Your participation makes a huge difference. Thank you. With that, I’ll now turn the program over to Peter Ginsberg, our Chief Operating Officer.

Peter Ginsberg, Chief Operating Officer:

Hey, thanks Jen, and good morning everyone. My name is Peter Ginsberg and I wanted to talk about some of our new efforts around corporate sponsoring. So to complement our community-based fundraising and engagement activities that Jen just described, we continue to expand our partnerships with companies - large and small. These collaborations enable the Foundation to fund more retinal disease research while providing sponsoring companies with an opportunity to engage with patients, clinicians, and other companies and experts in our field.

I’d like to highlight that Amgen has become a visionary partner of the foundation building on its prior outreach, partnership and support of our VisionWalk program. Amgen, which is the largest biotech company in the world, markets the thyroid eye disease drug, TEPEZZA. Our expanded partnership with Amgen will enable us to provide additional education and networking opportunities and other resources for the thyroid eye disease community. We greatly value Amgen’s commitment and involvement in supporting the Foundation and reaching the broader patient population dealing with serious eye diseases.

I’m also pleased to report that we have three new sponsors for our My Retina Tracker Registry and genetic testing program. Beacon Therapeutics is one of those sponsors, and it’s an ophthalmic gene therapy company targeting X-linked retinitis pigmentosa, cone-rod dystrophy and dry AMD. We also have as a new sponsor, PYC Therapeutics, which is a developer of RNA therapies targeting retinitis pigmentosa and autosomal dominant optic atrophy. Also, Théa, which is a leading eye care company based in France, is using RNA therapy to develop treatments for IRDs, including Leber congenital amaurosis and Usher Syndrome. We really appreciate these three new My Retina Tracker Registry and genetic testing program sponsors.

We also have tremendous corporate support for events that the Foundation hosts throughout the year. We have a strong lineup of corporate sponsors supporting our upcoming Retinal Therapeutics Innovation Summit, which is being held May 2nd in Salt Lake City, prior to the annual meeting of the Association for Research in Vision and Ophthalmology, better known as ARVO. This important industry event is jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University Casey Eye Institute. Casey Eye Institute is also the Summit’s Premier Sponsor. We greatly appreciate Casey Eye’s partnership and support for this great event.

The summit brings together members of the medical and research communities with representatives from the biotech and pharma industries, and we discuss rapidly emerging ocular therapies and strategize how to remove barriers facing the development of retinal disease therapies. This year, our sponsors at the Innovator level are Alkeus Pharmaceuticals, Belite Bio, Restore Vision, Spark Therapeutics, and Thea. For a full list of all of our Retinal Therapeutics Innovation Summit sponsors, please check our website in the about section under corporate partners.

Moving on from our important corporate partnerships to other industry news and investments, I’d like to highlight three recent developments related to the RD Fund, which is the Foundation’s venture capital arm. Earlier this month, RD Fund portfolio company, Atsena Therapeutics, raised $150 million in new Series C financing to advance its gene therapies for inherited retinal diseases, including X-linked retinoschisis (XLRS) and Leber congenital amaurosis. This is a major accomplishment with new funding provided by two new highly respected healthcare investors, Bain Capital and Wellington Management. In addition, all existing investors participated in the financing, including the RD fund.

I’m pleased to also report that one of our other RD fund portfolio companies, Opus Genetics, was successful in raising funds to fuel its gene therapy clinical pipeline focused on rare IRDs. Opus raised over $20 million and has the potential to receive another $21 million in additional proceeds upon exercise of warrants issued in the transaction. The public offering was led by healthcare focused funds, Perceptive Advisors and Nantahala Capital, with participation from other new institutional biotech investors.

One final announcement I wanted to highlight is that in February of this year, the RD fund invested in Perceive Pharma, alongside other industry leading investors such as Deerfield Management and J&J innovation. Perceive Pharma was spun out from Perceive Biotherapeutics to accelerate the development of neuroprotective therapies for people with glaucoma and potentially additional ocular indications, including retinitis pigmentosa. Neuroprotection is an approach to slower halt degeneration of neural cells, including those with a retina, independently of the genetic mutations causing disease. The RD Fund is also an ongoing investor in Perceive Biotherapeutics, which is developing a gene therapy targeting the complement system for treatment of dry age-related macular degeneration.

Overall, it’s really encouraging for these companies to be making such progress across these important retinal diseases, and we’re excited that these companies are gaining support from key industry players in raising capital and advancing their clinical programs.

I’ll wrap up my comments today with our quarterly financial update. The Foundation operates on a fiscal year that runs from July to June. So we’ve completed three quarters of our fiscal 2025, and for the first nine months ended March 31, our unrestricted fundraising revenue was approximately $23.4 million against operating expenses of $15.1 million for a net fundraising surplus of $8.3 million. And these results placed us on track for our fiscal 2025 budget in which we’re targeting revenue of $36.4 million against operating expenses of $22.9 million for a $13.5 million net fundraising surplus. With that financial and corporate update, I’m now pleased to turn the call over to my colleague, Dr. Amy Laster, our Chief Scientific Officer for a research update. Amy?

Dr. Amy Laster, Chief Scientific Officer:

Thank you, Peter. Today I’m pleased to share with you that the Foundation just announced five new awards for our fiscal year 2025 and three of our programs, which include the Brett Family Translational Awards, our PRPH2-associated Retinal Diseases Award, and the Free Family AMD Initiative Award.

The Foundation has a very rigorous review process, which is led by our esteemed Scientific Advisory Board of over 60 top scientists and clinicians from around the globe, which ensures that each funded project has the potential to deliver transformative insights and advancements in retinal disease treatments.

Let’s start with the Brett Family Translational Research Award. In this program, we received 58 letters of intent, which led to inviting 19 applications reviewed by our Scientific Advisory Board.

From those applications, two awards were given. One to Dr. Sylvain Chemtob, from the Research Center of Hospital Maisonneuve Rosemont in Montreal, Canada, for work to develop a new treatment for dry age-related macular degeneration. This research is focused on a specific part of the immune system which can damage the cells in the retina. And by targeting a key factor in this immune pathway, the researchers hope to reduce inflammation and protect the retina from damage.

The second award was to Dr. Daniel Paull from the New York Stem Cell Foundation in New York City to find a precise drug to treat dry age-related macular degeneration using induced pluripotent stem cell modeling, which we’ll hear about a little bit later from Dr. Gamm. Dr. Paul’s lab intended by studying these lab-grown cells, they can understand the disease better and test new drugs to see which ones might work best. And this technique, once they optimize this, can be used for other eye diseases.

Moving on to our PRPH2-associated Retinal Disease Awards. In this program, we received 22 letters of intent, which led to the review of nine applications and two awards were given. First to Dr. Jason Comander at Mass Eye and Ear in Boston to study how changes in the PRPH2 gene cause retinal degeneration. Dr. Comander uses this very detailed approach to understand exactly how these genetic changes lead to disease. And by understanding the genetic causes, they hope to find better ways to not only diagnose, but also treat people affected by mutations in this gene. And the second award was to Dr. Frauke Coppietersm of Ghent University in Belgium for research to understand why PRPH2 disease can look different in different people, which we call disease heterogeneity, and they’re also working on new ways to treat this disease.

And then lastly, I want to talk about our Free Family Age-Related Macular Degeneration Research Award. We received 16 letters of intent, which led to the review of four applications, and one award was given to Doctors Glenn Yiu and Rui Chen of UC Davis Health and University of California at Irvine, respectively, for studying how drusen develops in non-human primates. And they’re using very advanced novel techniques to understand the cellular location and features of the contributing factor of this early hallmark of dry age-related macular degeneration.

As these awards indicate, there is a diverse range of research that’s taking place in both academic settings and in the clinic, and we are excited to fund these compelling projects. But as you can tell from the number of applications and letters of intent that were received, there are many worthy projects and researchers that still need funding, and that’s why we continue to have a strong sense of urgency in our fundraising efforts to expand our impact.

There have been many recent announcements of new clinical trials starting and data being reported. The Foundation lists these announcements in the News section of our website, but I want to take a moment to highlight a few of the noteworthy clinical developments since our last Insights Forum, which was in December.

I’ll start with one from Luxa Biotechnology, who reported vision improvements in their trials of an RPE stem cell therapy for dry age-related macular degeneration. And also MeiraGTx announced the publication of data showing that its LCA4 gene therapy restored meaningful vision for blind children. Atsena Therapeutics launched Part B of its Phase ½ clinical trial for X-link retinoschisis gene therapy and ViGeneron received FDA clearance to launch a clinical trial for its RNA trans-splicing gene therapy for Stargardt disease. SpliceBio received FDA clearance to launch a trial for its emerging protein-splicing therapy for Stargardt Disease.

And two more I want to highlight. Sepul Bio began enrolling patients in its Phase 2 clinical trial for ultevursen, which is an emerging RNA therapy for people with retinitis pigmentosa or Usher syndrome caused by mutations in exon 13 of the USH2A gene. And then lastly, Neurotech Encapsulated Cell Technology received FDA approval for the treatment of macular telangiectasia type 2 or MacTel.

It’s very encouraging to monitor the tremendous progress being made across research institutes and companies really dedicated to finding treatments and cures for blinding diseases. Stay tuned and check out our website often. With that, I’d like to turn the program over to our CEO, Jason Menzo.

Jason Menzo, Chief Executive Officer:

Thank you Amy, and I could not agree with you more. If there’s one takeaway for everyone who’s on this call is that last comment that Amy just made. Check our website often, FightingBlindness.org. We have a section of the website called the Clinical Trial Pipeline. And every one of the advancements that Amy just described and many, many more are all updated regularly on our website. It’s a great resource for information like that.

And also I do want to mention for those who don’t know, Dr. Laster, who was just speaking a moment ago, was recently appointed as our Chief Scientific Officer just last month. And over the last 16 years, Amy’s leadership and expertise has really been a pivotal force in advancing so many of our scientific initiatives, whether it’s spearheading our five-year scientific strategic plan that we’ve discussed on this call many times over the last year, to strengthening collaborations with key partners, she really has been a catalyst for so much of our progress and I’m thrilled to be able to appoint her as our new Chief Scientific Officer and congratulate her. And Amy, thank you for your many years of service and commitment to this mission. So that’s awesome and I’m just really, really happy to be able to share that with all of you today.

All right, so let’s shift gears. For several years we’ve heard from many members of our community about the lack of quality mental health resources available specifically for the blind and low vision community. In response, back in 2023, we established an internal task force to explore and drive solutions specifically tailored to the needs of our community. This task force that we formed was guided by an external Mental Health Advisory Council that we established, which includes five expert licensed therapists, four of whom are blind themselves or have low vision, a retinal specialist who has treated patients facing depression and anxiety associated with their vision loss, and a member of our Foundation Fighting Blindness Board of Directors, who herself is affected by vision loss and took the action to organize a local mental health support group in Southern California.

With the guidance from this Council and the feedback from members of our community, I’m thrilled to announce today a suite of new mental health resources specifically designed for the blind and low vision community. These resources will be available on our website, FightingBlindness.org, and also rolling out through our national network of chapters and on other platforms. To house these resources, we’re launching a specialized Mental Health Resource Center on the website offering resources, support, community connections designed specifically for individuals who are navigating vision challenges. The Resource Center will launch in just a few weeks during Mental Health Awareness Month, which is in May. And even more so, I’m really excited to share that as part of this initiative, we have also formed a strategic partnership with an organization called ALMA.

And if you’ve never heard of ALMA, I’m going to share a little bit about who they are and why this partnership is so critical. ALMA is a network of 25,000 mental health professionals dedicated to simplifying access to quality, affordable mental healthcare. And as part of our collaboration with ALMA, the Foundation, along with two very special subject matter experts, Rebecca Alexander and Charlie Kramer and our new partner ALMA have co-developed specialized training for mental health professionals focused on the unique experiences and needs of individuals who are blind or have low vision. A curated directory of the licensed therapists who have completed this certified training will be available in this new mental health resource center on our website. And in addition, there will be webinars and podcasts and articles and expert interviews and personal stories for individuals and families navigating vision loss. It’s a really important initiative and we’re thrilled to be able to share this with you today.

As part of this launch, on May 17th, we are going to host our quarterly vision webinar on the topic of mental health solutions for our community. Dr. Elizabeth Morray, who’s a licensed psychologist and the Vice President of Clinical Operations for ALMA, our new partner I was just describing, she’ll be joining us along with Rebecca Alexander, who is a nationally recognized expert in this space. And they’re going to share what resources can help those in the low vision community, including access to the ALMA network of experienced clinicians. So to everyone on this call, please mark your calendars as this will be a great discussion. And for more information and to register and any other information related to any of our events, please go to the events page at our website at FindingBlindness.org.

Now I’d like to move on to a topic that is front and center in the news every single day around the globe, actually, even though it relates specifically to us here in the United States, I doubt there’s a corner of the globe that isn’t aware of some of the policy changes that are taking place here in the states. As you know, we’ve been monitoring and reporting on actual and proposed federal policy actions that touch our mission. It’s not about politics. This is truly about policy. And the Foundation has taken a proactive position in advocating to prioritize vision research within the federal policy landscape, whether it is related to funding, regulations, the regulatory process, or even staffing at many of our federal institutions.

The Foundation’s funding for research comes primarily from private donations and from supporters like you. In fact, it’s important to note, that greater than 98% of the Foundation’s funding comes from donations, and less than 2% of our funding comes from federal grants or contracts. So changes that are happening at the federal level do not directly impact the operations of the Foundation Fighting Blindness for the most part. But that said, the treatments and cures for blinding diseases that are in development rely heavily upon the collaboration between the Foundation Fighting Blindness, academic institutions across the nation, and really across the globe, industry, and especially the federal government. It is an ecosystem of each of these parties that really do work together in collaboration to advance treatments and cures. We can’t and don’t do this alone.

The National Institutes of Health, the National Eye Institute, the Food and Drug Administration, the Advanced Research Projects Agency for Health, which is ARPA-H, are just a few of the very important institutions that we work closely with that contribute to this ecosystem. And if they are disrupted, whether it be related to funding, staffing or otherwise, it can have a dramatic impact on our mission.

Let’s take the National Eye Institute, for example. The National Eye Institute currently supports vision research through more than 2,100 research grants made to scientists at medical centers, universities, and other institutions. Well, unfortunately, just last week, the White House shared their proposed budget, we want to emphasize this is their proposed budget, for fiscal year 2026. And the Foundation Fighting Blindness, along with many others in the research community, were alarmed because that proposed budget called for a 40% reduction in research funding for the National Institutes of Health, where the National Eye Institute currently sits. And it also called for the flat-out elimination of the National Eye Institute by folding it into a broader neuroscience institute, which would be problematic because the National Eye Institute is a dedicated, focused entity exclusively pursuing issues related to the eye, and if it is folded into a broader, less specific institute, there’s obviously some challenges that we would anticipate that would come from it. But the bigger headline is the 40% proposed reduction in funding for research at the National Institutes of Health.

So obviously these proposed policy changes, while not directly impacting the operation of the Foundation Fighting Blindness, would have a dramatic impact on the advancement of treatments and cures for retinal diseases and really all conditions that affect vision, whether it be glaucoma, cornea or other ocular, cataract, etc, ocular issues. So funding cuts at the National Eye Institute would severely impact nearly all of the researchers that we fund, because again, we’re funding folks like Dr. Gamm, and Dr. Gamm is also receiving funding from the National Eye Institute, for example. Cuts at the National Eye Institute would significantly impact nearly all of the researchers that we fund, and that’s why we’re concerned.

As the federal administration considers policy and funding changes, the Foundation is actively working in Washington to urge policymakers to prioritize vision research and protect these critical partners to our mission. And in fact, I sent an open letter to the Health and Human Services secretary RFK, Robert F. Kennedy Jr. advocating to prioritize vision research as part of the administration’s Make America Healthy Again agenda. Earlier this month, I was in Washington DC meeting with congressional offices explaining why vision research is so important to the health of this nation, and I’ll be back on the Hill again in a few weeks.

As advocates, we must ensure continued federal investment, maintaining the National Eye Institute’s independence, strengthening the FDA’s regulatory infrastructure because all of these institutes play a critical role in advancing our mission and preserving America’s leadership in vision science.

While we are taking action on the Hill, we urgently need you members of our community, especially those here in the U.S., to also engage by contacting Congress directly to advocate for vision research. And what you can do is call or email your members of Congress, in particular your senators, let them know that we need Congress to preserve and fully fund the National Eye Institute as a priority to help advance science, treat and cure vision loss, and maintain America’s global leadership. Eliminating of the National Eye Institute would absolutely have a devastating impact on the advancement of treatments and cures for blinding diseases.

Most importantly, we ask you to share your personal stories, tell your member of Congress why prioritizing vision research is important to you, why it matters to your family. Many members of Congress don’t fully understand the burden of vision loss and how it impacts the people within their constituency. Your story can really help strengthen the voice and impact of our community. And to help along with this, we’ve now added on our home page at the website, FightingBlindness.org, a feature about how to contact Congress, links to a page with sample letters and links with information about how to reach out to Congress. If you haven’t seen it yet or haven’t gone there yet, go to FightingBlindness.org and on our home page there are these additional resources. And of course, if you need assistance with outreach efforts or you have feedback for us or any questions for us, you can always contact us anytime at Info@FightingBlindness.org or of course on any of our social media channels.

With that, let’s shift gears. When we talk about cutting edge research in our field, one of the most prominent and well-respected names mentioned is Dr. David Gamm, and I’m thrilled to have Dr. Gamm join us today as our guest speaker. He’s a pediatric ophthalmologist and distinguished professor in the Department of Ophthalmology and Vision Sciences at the University of Wisconsin in Madison, and he’s also the director of the McPherson Eye Research Institute. His work focuses on stem cell-based therapies for retinal degenerative diseases, including retinitis pigmentosa and age-related macular degeneration. Over the last 10 years, Dr. Gamm and his team have received several Foundation grants, including the Gunn-Harrington Award for the development of retinal and photoreceptor cell therapies. The Foundation providing funding of Dr. Gamm’s early research led to enhanced funding from the National Eye Institute, which in turn led to the licensing of some of this technology to an industry player. And this is a perfect example of what I was speaking about a minute ago. This critical ecosystem of the Foundation, the National Eye Institute, academia, like the University of Wisconsin, and industry, we all work together in this ecosystem to advance treatments and cures.

Dr. Gamm founded a company called Opsis Therapeutics to advance this technology. And then Opsis entered into a strategic R&D and clinical manufacturing collaboration with BlueRock Therapeutics, which is a cell therapy company and wholly owned subsidiary of Bayer AG, huge multinational pharmaceutical company. BlueRock is now conducting a Phase ½ clinical trial in the U.S. for photoreceptor cell therapy for people with inherited retinal diseases, such as retinitis pigmentosa and cone rod dystrophy. Dr. Gamm, we are thrilled to have you join us today on the Insights Forum and I will turn the call over to you.

Dr. David Gamm, Professor, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison:

All right, well thank you very much and it’s great to talk to this group and just a little bit of background, I started working with the Foundation Fighting Blindness, or actually they reached out to me shortly after I finished my fellowship. So we’re talking a little over 20 years ago and it was really one of the most important interactions that I made early in my career and really gave me the ability to move forward with these early concepts, which at the time, it was a brand new field. So here at the University of Wisconsin, Jamie Thompson had just published the ability to generate human embryonic stem cells, which opened up this whole entire field, but it was still very fledgling. The FFB saw the promise of it, saw that I was interested, helped back my early work and continue to do so over the next couple of decades leading to where we are today. I’m very grateful for them. I’ve served on their Advisory board for a number of years too, so I feel it’s only right for me to… They said so many nice things about me, I should say a lot of nice things about them too and very deserving.

So the time that I have with you, I think we want to spend a lot of time with Q&A, but I wanted to give some background too is just to give you a little bit of a primer on what are pluripotent stem cells, what makes them different from other maybe stem cells that you hear about, especially the ones that are perhaps kind of more snake oil or the ones that they try and sell to you on the internet that really don’t do anything at all. And then talk about where stem cells are in the grand landscape of potential therapies and how we use them to advance not just cell therapies, which is what we’re excited about starting here in the next month, but also how we use them as a tool to advance gene therapies, genome therapies, and really all the other types of approaches to treating inherited degenerative diseases of the retina that you heard about just now.

To start with then, so what is a pluripotent stem cell? Really a pluripotent stem cell has two really important features to it. One is that it can self-renew indefinitely. What does that mean? That means that once you have it, it will continue making more of itself. You have to treat it just right. These are very finicky diva-like cells, but if you treat it just right, they will continue to make more of themselves forever and always. Which means that you have an unlimited supply of them and that’s important. Secondly, when instructed to do so, they have the potential to stop making more of themselves and go down specific pathways to make, at least hypothetically, every single cell type in your body, which is an amazing property, but also a very daunting one too because if you can imagine if you’re starting with this set of cells that can make all the different cell types in the body, well, if you have blindness, you don’t want liver cells in your eye, you don’t want bone, you don’t want these other things. So what the charge was for us in different fields was how do we harness that and get them to go directly down a pathway we’re interested in? In this case retina. And then ultimately not just the entire retina, but maybe just photoreceptors or cones or rods or retinal pigment epithelium, the cells that are lost in so many of these diseases that we’re talking about.

My lab was in a great position here at UW where all this technology was founded to be able to embark on that, on those studies to be able to harness and figure out the different cues and things that we could do to get them to go down the pathway we wanted them to go down. And ultimately we were able to do that and not only with embryonic stem cells, but later on in the mid-2000s, the technology to develop induced pluripotent stem cells came on board also here at UW, as well as in Japan.

In many respects, we were on the forefront of all this simply by where I’m at here in Madison, Wisconsin. So induced pluripotent stem cells are different than embryonic stem cells in that they’re taken from adults and they’re from a skin sample or now we use blood samples and we can convert genetically the normal cells that you have populating your body right now back to a state that is essentially identical to an embryonic stem cell. That’s the source now that we use, that my company uses, that my laboratory uses predominantly are these induced pluripotent stem cells. They’re truly pluripotent stem cells, but they’re induced to become that from adult cells as opposed to starting off that way. Everything I’m going to talk about from here on out really is about iPS cells or induced pluripotent stem cells. They can be derived from any person on the planet and that gives us a lot of flexibility in terms of how we use them.

In 2009 we finally developed a way in which we could specifically grow retina, pull that out from all the other things that these cells might want to make and use them to study and then hopefully, ultimately develop therapeutics from. That led to patents, and again, ultimately I’ll talk about the company that we started.

Once we had all that in place, then the question was, “Okay, what are the applications?” You can take a blood sample from somebody and you can generate an unlimited supply of their retina in a dish in the laboratory. Wonderful. What can you do with it? There are actually a number of applications, not just cell therapies, which I’ll finish my discussion concentrating on, but one that people don’t know about. And all of these by the way have been funded and supported by the Foundation Fighting Blindness.

But one, and we worked with Eric Pierce at Harvard to do this is gene diagnosis. There are a lot of individuals who clinically know they have or a loved one has retinitis pigmentosa and they go through all of the genetic testing, but it comes up kind of empty. They say, “Well, we know you have it, but we can’t tell you what exactly you have.” And that’s important because it tells you whether or not you might be eligible for different trials.

One of the things that we do in medicine when we try to diagnose somebody is we take a biopsy. We take a piece of your liver or whatever, and your liver is huge and you can do without a little bit of that, but you can’t biopsy your retina. That’s a bad thing. But we can produce your retina in a dish. Using a blood sample, we can make an unlimited supply. And that allows us essentially to have a roundabout biopsy of the person’s retina who has the disease.

We’ve used that in combination with genetic testing to uncover some unknown cryptic mutations that have led to families diagnosis of RP. In at least one of those cases, it led to their ability to be enrolled into a clinical trial. That’s one exciting way that we can use the technology that isn’t often discussed. The other one is, because we can grow these retinas in a dish, we can study why it is that that particular genetic mutation leads to deficits or dysfunction of the cells that they’re expressed in. What do I mean by that? Well, we do know a lot of the genes that cause ultimately blindness in these disorders, but in many respects we don’t know exactly why.

We can say, “Well, it’s this gene and this gene isn’t expressed or it’s expressed in an incorrect way, and ultimately that goes from A to Z and we have blindness.” But why? Why is it that gene, what role does it play that ultimately over time causes the cell that is expressed in either a photoreceptor or a retinal pigment epithelial cell to become so dysfunctional that it ultimately degenerates and dies? And if we understand the path by which the gene mutation leads to that problem, then we may have more places where we can intervene therapeutically, and at the very least we’ll be able to develop better assays or tests to see whether or not specific therapeutics are more likely to help those patients or not, as opposed to just having a black and white, A or Z. If we can follow them along the way, then we can develop better therapeutics too.

We’ve used this technology then to develop model systems of retina in a dish with different diseases and then tested gene therapies, genome therapies, RNA-based therapies to see whether or not they affect change within these cells that suggest to us that they’re overcoming the basic underlying problems in the cells that ultimately lead to their demise.

Those are a couple of situations where we use our technology an awful lot in my lab every day that don’t go back into the patient, they’re done in the lab, but they’re helping as a model system to move along the types of technologies that you heard about earlier in today’s Insights.

Okay, so what everybody wants to talk about then is the last application or not last, there’s others as well, and that’s cell replacement. If in fact we can make these cells and they serve as a source of spare parts, then can we put them back into patients who’ve lost them and get them to hook back up and restore function?

And we’re not talking just the eye. People are doing the liver, cartilage, the brain, all sorts of different areas. It’s not just the eye, although the eye does tend to lead the way in these types of experimental therapies for a lot of reasons that we can discuss if people are interested. To do that then, it’s a whole other level of effort because now we’re not just talking about testing something in a dish, we’re talking about developing a cell therapy that’s going to go into a human. That raises the bar in terms of your safety and testing because now things are getting real serious, right? Now you’re talking about taking something that we have to manufacture and placing that into a loved one.

To do that then, it requires an awful lot of teamwork, a lot of funding to be able to take what we’ve learned how to do in our lab and to expand that, make it cryopreservable, meaning we can freeze it down, we can transport it, we can make it over and over again on a billion cell scale so that patient 1 gets the same product that patient 3,332 gets. And that sounds kind of trivial, but it actually takes years and an incredible amount of expertise to be able to accomplish. We approached that by starting a company called, Opsis Therapeutics. Our company is a subsidiary of Fujifilm, which you think of as a film company, but actually is a holdings company and has a lot of therapeutics associated with it as well.

They actually bought out Jamie Thompson’s company here in Madison and now it’s called Fujifilm Cellular Dynamics. They have an incredible amount of patent suite as well as know-how to take cell therapeutics and make them expanded and what we call GMP or capable of being put into patients. I entered my company with them and we were able to, from 2016 to 2022, so we’re talking a good five to six years, to take all of the processes that we developed in our lab and ultimately get them to the point where we could put them into patients.

We then entered into a partnership with Bayer and BlueRock to go ahead and move forward with a clinical trial. At that point, we were confident that we could make the product, but we had to produce what’s called an investigational new drug product application. We worked for a couple of years for that. We ended up submitting that application to the FDA last summer and then a month later we were approved. In the last few months then, we’ve been preparing to start that clinical trial, which we hope will be very, very soon to start first in Miami. That’ll be the first site.

Where in that spectrum, and I’m wrapping up here because I’m probably getting close to my 10 minutes and I tend to talk too long, but where in that spectrum might you or your loved one, where in there should you be thinking - should you be thinking cell therapies, gene therapies? I mean, on the one hand, it’s great that we hear about all these technologies being developed. On the other hand, it can make your head swim because, “Do I want all of them? Do I want three of them? Do I want that plus half of that?”

The bottom line is that for early stage therapeutics, you can be in an early Stage 1 / Stage 2 therapy or experimental clinical trial, kind of 1 for the most part. And you want to know where you’re at.

That is a subject of two variables. One is what is the exact disease that you have? Do you have macular degeneration? Do you have LCA? Do you have RP? What’s the gene that you’re working with or that you’re dealing with in your family? That’s one part. The other part is where are you on that course of the disease? Are you young and just starting to lose vision and noticing problems and obviously worried? Are you midway through, muddling through doing okay, or are you at the end stage? And so that you have just maybe just a little bit of vision left or maybe no vision at all.

If you’re in that early stage, then it’s probably not worth the risk of doing something that is more geared towards late stage treatments. You really want to preserve or keep what you’ve got. There are gene therapies, genome therapies, RNA-based therapies, when the cells are still alive, the photoreceptors or the RPE, they’re still there, they’re just not functioning right and they’re on their way out. But there you want to preserve things or maybe pull back the hands of time and just preserve what you’ve already got.

If those cells though are already gone, then you have to either bypass them with things like optogenetics or prosthetics or replace them like cell therapies. Then you kind of move over into a different category of potential therapeutics. At that stage, gene therapy, genome therapy, that’s not going to help you. You have to look for something that’s going to be a little more universally restorative. And then at the very end, if you’ve got nothing working in that eyeball, then you might have to look at something like a whole eye transplant, which sounds crazy, but actually there’s a lot of work being done on that. And my laboratory is part of a large multi-institutional national consortium to look into that possibility as well. But if you’ve got vision and you’re doing okay, you don’t want a whole other eyeball that brings in all sorts of other problems.

You have to size the risk and benefit of the different therapies. And that’s again, getting back and finishing up here where FFB comes in because they have such an extensive knowledge of the entire cohort of therapies that are available or even on the horizon that they can be a wonderful source of information for folks, not only about what their disease is, but what might be most applicable for their or their family’s condition. With that, I’ll wrap it up and just move it over to Q&A.

Jason Menzo, Chief Executive Officer:

All right, thank you very much. Dr. Gamm, that was awesome. Always love getting to hear your words of wisdom and I know many folks in the community feel the same way as evidenced by the bazillion questions and chat that have come in so far. Thank you very much for being on the call today. We are ready to switch to our Q&A session. I’m keeping an eye on the clock. We can go maybe a few minutes past the top of the hour this morning to make sure we can cover at least a handful of questions, but I do want to make sure everyone knows that everyone who chatted in a question or put it in the Q&A or sent a note to Info@FightingBlindness.org, every single question that comes to us, we will respond to. If we don’t get to it here on the call, we will follow up with you offline. So don’t be alarmed if we didn’t get to your question here on the call. We’ll make sure we cover every single question that comes in. So that is my commitment to you. With that, let me turn it over to Amanda to just repeat the instructions for asking questions and then we’ll dive in.

Amanda Bement, Development Operations Specialist:

As Jason just mentioned, there are several methods that you can use to ask these questions. You can either submit them through the Q&A feature, which is at the bottom of your Zoom screen and do please include your name so that we can follow up afterwards if we do not get to it within these next few minutes. You can also send an email to Info@FightingBlindness.org and we will make sure to follow up as Jason mentioned.

Jason Menzo, Chief Executive Officer:

Great, thank you. Let me invite the other members of our team to come off mute, turn your camera on. Julie Hill just chatted in a question two minutes ago, which is funny because it is the exact same question that was queued up from an email we received beforehand. And I’m going to direct this question to you, Amy, and this is about Nanoscope. Many folks in the community have heard public announcements from Nanoscope and are curious about what the status is, what we can share, what we know, and maybe even just briefly what the technology is so that folks who aren’t familiar with it can become familiar with it.

Dr. Amy Laster, Chief Scientific Officer:

Thanks, Jason. Again, this is Amy Laster. Nanoscope is a clinically-staged company and they have developed an optogenetic strategy. They’ve created an opsin which allows for light-sensing abilities in cells. They’re using this essentially to restore vision in patients with severe retinal degenerations. Last fall, they announced that after their Phase 2b clinical trial that they are applying for what is called a biologic license application (BLA) to the FDA. This is required to have any drug approved for market availability and being available for physicians to prescribe for treatment.

Last month, they actually published a paper, and I know there were some questions about it on their very early first Phase 1/2a trial of four patients with retinitis pigmentosa with at least one variant in the ABCA4 gene. The opsin or the technology that they have, this is delivered to the eye via a gene therapy vector. It’s an intravitreal injection, it’s a single injection. And they’ve shown their published paper that there were some patients that had improvement in their visual acuity, shape, discrimination, so being able to see differences in shape, as well as their mobility over a one-year follow-up with no significant safety issues that were reported or described. We are continuing to follow up on Nanoscope and where they are moving forward with a BLA, which they anticipate applying for in this calendar year. So stay tuned.

Jason Menzo, Chief Executive Officer:

Yeah, that’s great. And as we say, anything related to news in our space will end up on our website. So if you don’t already, make sure you check our website often because there’s great information, headlines, new news when it becomes available, podcasts, the clinical trial pipeline. I know many of you are familiar with their website, probably go there regularly, but if you don’t, it’s a great resource.

Dr. Gamm, are you still there? I see that you’re muted, but we’ve got a couple questions I want to make sure I send your way. Good. Okay, I see you now.

Related to Nanoscope, there was actually a question that came in about whether or not Opsis or BlueRock would explore the usage of the stem cell treatment that they’re developing in combination with the product from Nanoscope and the concept of, I use the term polypharmacy, multiple treatments in the same person, and maybe you could speak just to in a clinical trial stage of developing a potential treatment as compared to what might be the case in a future state after FDA approval and it’s in the market, and not just as it relates to these two products, but just generally how those interact with each other.

Dr. David Gamm, Professor, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison:

Great questions. It’s a nice segue you talked about BLA. You have to think about it prior to FDA approval for public use or public consumption and then afterwards. Prior to that, it’s part of trials and trials are necessarily and importantly regulated by the FDA. So you are not able to carte blanche do whatever the heck you want because you haven’t established yet that you have safety and efficacy. In those cases, the FDA has to approve every aspect of what you’re doing. And in many respects, you want to know what your product is doing. And so having it be as clean as possible so that I know that therapeutic A, what does it do on its own as opposed to A plus B or B alone? In most cases that are, in almost all cases that I know of, it’s really just a single product that’s being tested, not two separate products.

That doesn’t even get into all of the industry-wide complexities that would be in place that probably aren’t as concerning to patients, but certainly as industry goes, you want to have clean lines of ownership and things like that too. Once things are approved, then your provider can use them in multiple different ways.

Now specifically with Nanoscope and cell therapy, those two I would not mix because they’re essentially kind of doing similar things. In the case of Nanoscope, they’re basically saying, “Photoreceptors are gone, we’re not going to use them anymore. We’re going to get cells that are still in the retina that normally don’t respond to light. We’re going to get them to do the job as best they can.” The benefit of that is that the cells that they’re working with are already plugged into the system. They’re not designed to detect light or to see, but they can be coerced to do that at some level and they’re already plugged in.

With cell therapy, with photoreceptor replacement, we’re actually replacing the cells that are lost. So these have a high ceiling, they have all the ability to see like you and I see, but they’re not plugged in yet. We’re asking these cells that have all the bells and whistles necessary for vision, but now we’re putting them into an environment where they have to find their partners and establish those connections.

Like many approaches, they all have their pluses and minuses, they all have their strengths and potential weaknesses. And that’s why it’s important to have a very broad portfolio because not every person is going to respond the same way to every therapy. There could be folks that Nanoscope might be the best thing for them. There might be other folks that cell therapy is going to be the best thing for them. There is, while we talk about these as being agnostic, we don’t particularly care about what your gene mutation is, nothing is truly agnostic. There’s not a silver bullet for whatever ails you and knowing where you are in that disease process and what the state of your retina is important in making those decisions.

Jason Menzo, Chief Executive Officer:

That’s great. I’ve got to keep in mind the clock, it’s 12:03. I’m going to just move to the lightning round, I guess, maybe kind of rapid fire. Dr. Gamm, a couple questions more for you. One, which I think is very important. Many people in this country are taking statins, atorvastatin, Lipitor, Zocor, so on, and these are commonly prescribed drugs. And this question is the relationship between taking a statin and age-related macular degeneration. I guess there was a book that was published, I’m not familiar with it, but that implied that there might be a negative relationship between these two things. I’ve always heard the opposite, but curious what your thoughts are on that. And then I’ll fire a couple other questions to you.

Dr. David Gamm, Professor, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison:

Real quick. But I will preface by, I’m a pediatric ophthalmologist, so my clientele don’t tend to take a lot of statins, but I’m aware of the studies and this is one of those situations where again, it seems like I’m a shill for FFB, but it’s just the truth. That’s where working through all of this data… You can find data that will say coffee will kill you or coffee will save your life. There’s just tons of studies out there and some of them are good, some of them are bad, some of them are big, some of them are small. And so you can find anything to fit whatever belief system you might have or might want.

So stepping back and being scientific about it, there have been studies that were done mostly in the mid-2010s using hundreds of thousands of patients that showed no effect or maybe a negative effect. And then there are other studies that looked at it that saw maybe a positive effect. Bottom line is if you do a meta-analysis on all of that, the jury is out. And I would not avoid taking them because I’m worried about it and I would not take them by virtue of my AMD. There just isn’t enough data out there. And the fact that hundreds of thousands of patients have been looked at and it’s still a question probably means that there’s not a strong association.

Jason Menzo, Chief Executive Officer:

Question from an eye care professional was sent in, from an optometrist. But I know that this relates not just to eye care professionals, but probably everyone who’s on this call, everyone has been exposed to e-blasts or social media posts or ads in the paper about, “Come to my clinic and get cell therapy.” And there’s a million different, and there’s a million different ways that this shows up. So let me ask you this. What should our audience know about the difference between cell therapy that’s being developed in an academic, rigorous clinical environment as compared to the things that you might see on Facebook. How does an eye care professional talk about that to their patients?

Dr. David Gamm, Professor, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison:

Very simple. We are at a point at which anything legitimate and is being going through legitimate trials, we’re paying your transportation. We’re asking you to participate in something that is valuable to the scientific medical community. We are not charging you for that. If they ask for money, if there’s a stem cell based therapy that requires you to pay, it is snake oil until proven otherwise. They’re profiteering. There is a conflict of interest because they’re making a livelihood off of that. All the stem cell clinics, basically, most of them are taking blood from inside of you, calling them stem cells, squirting them wherever, everywhere else in the body. They last for maybe a day and then they’re gone. And then you pay them thousands of dollars. Those are just, for lack of a better word, shysters.

Jason Menzo, Chief Executive Officer:

That’s the technical term. I’m going to fire one more question to you, Dr. Gamm, and that may be all we have time for today, everyone. So again, if we haven’t gotten to your question, we will certainly follow up with you offline. In the chat, there’s a lot about this and there’s obviously a lot of sensitivity to the point that we spent time on this call talking about, which is this ecosystem of the Foundation Fighting Blindness, in your case, Wisconsin, the National Eye Institute, the FDA, BlueRock Therapeutics, how each of these parties work together and the role that they all play in advancing potential treatment. We spent some time talking about what our community can do to advocate for vision research. I’m just curious if you could speak from the perspective of a researcher and clinician, how you sit as it relates to things that are happening at the federal government from a policy standpoint and the disruption that is occurring and may even continue to occur as it relates to this ecosystem.

Dr. David Gamm, Professor, Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison:

Yeah, I concur with all the comments that you made earlier and just how critical this is. I’ll add one perspective to all this and that I think is really important and is often overlooked. We in academic labs work very leanly. We don’t sit on a trust fund. We don’t have money behind us that we can just use. We are required by all of our funders to use our funds as we are provided them. So we don’t have rainy day funds, we don’t have any funds that we can rely on. Why is this important? It keeps us very lean, it keeps us very focused. We don’t have silver spoons in our mouths. We actually oftentimes operate just at or a little below what we’re funded for. But what that means is that if there’s even a pause, if even the federal government just decide they want to screw around for six months and then figure it out later, during that six months, you can’t pay people.

And so if you had a factory that makes cars and say, “You know what? We’re going to figure this out, but I think we want to make some changes here. We’re going to stop doing stuff.” And then the factory turns around and say, “Well, I can’t pay you for six months.” They’re going to go, they’re going to leave. You can’t just pause and restart. That will destroy the labs. The labs will go away. There won’t be anything to return to. You’ll also lose a generation of trainees going into these fields. And so even a short pause, even a kind of, “Eh, let’s try to figure things out.” Or, “Let’s message different things every other week,” causes enough uncertainty in an already very lean system where it could be devastating and you won’t be able to just snap your fingers and pick things back up where you left. You will lose the people and the talent that’s necessary to move these therapies forward and they’ll just go somewhere else.

Jason Menzo, Chief Executive Officer:

And so with that cheery, rainbow filled optimism, no, obviously I’m kidding, but it is an important reminder of I guess the state of affairs. And again, I just want to reemphasize to everyone in our community why now is a great time for you to get involved, reach out to your senators in particular, but certainly your Congresspeople as well. Make your voice heard and really share. This is not about politics. This is really about policies and how they could impact this mission. And telling your story and emphasizing why vision research is important, why it matters to you and why it matters to your family.

So on that note, we are going to conclude today’s call. Thank you all so much for spending time with us today. Of course, as always, there’s a ton of information on our website, FightingBlindness.org, and if you have any feedback, suggestions, questions, you can always reach us on all the social media platforms and directly by emailing us at Info@FightingBlindness.org. Thank you very much and I’ll turn it over to Chris to wrap things up for the call.

Chris Adams, Vice President, Marketing & Communications:

Thank you for joining us on today’s call. If you have any questions, again, send us an email at Info@FightingBlindness.org. All the latest research updates and news can be found on our website at FightingBlindness.org, and please follow us on social media. We’re on all the major handles. Again, thank you for joining today’s call and have a great afternoon.

There will be a question and answer session at the end of the formal remarks; however, if you would like to send in questions prior to the call, please send an email to membership@FightingBlindness.org.

After registering, you will receive a confirmation email containing a link to the online webinar so that you may join the call from any device – computer, phone, or tablet. Please plan to connect a few minutes in advance to make sure your device is linked to the Zoom webinar platform.

If you are a screen reader user or need assistance registering, please reply to this email and provide your name, email address, and phone number so we can register you in the Zoom platform.

We appreciate your support and hope you can join us on Thursday, April 24, 2025! A recording and transcript will be available a week after the event.

ZOOM MEETING TIPS

If you’re new to Zoom or want to learn more about phone and keyboard shortcuts that allow you to navigate Zoom settings without using your mouse, go to our Zoom Meeting Tips section on the Foundation website.