>>Kaycee: Hello. Welcome, everybody. We will wait just a couple of minutes to get started. While you're waiting, feel free to set your chat from all panelists to "all panelists and attendees." That way when you have a question or comment everyone can see what you are sharing in the chat box. Again, we'll get started in just a couple of minutes. You can go ahead and change your chat from "all panelists" to "all panelists and attendees" while you wait. We love to see where you're coming from, so feel free to put that in the chat while we are waiting. We're going to wait just a couple of minutes to get started and while we're waiting, feel free to change your chat from all panelists to "all panelists and attendees." You can share where you're tuning in from in the chat. We've got someone from San Francisco. Welcome. We're glad you're here.  Wichita Falls. Copperas Cove, Mansfield, Texas. Abilene, Bakersfield, California. Glad you're here, Chris. All right. Houston. Welcome. Well, I'm going to go ahead and get us started with a few announcements. If you have a question or a comment during the time our presenters are speaking, please post that in the chat box and make sure that it says all panelists and attendees. Your microphones and cameras are automatically muted so you don't need to worry about that. The handout for today's session will be shared in the chat for immediate viewing and will also be available for later viewing along with the recording of this and past Coffee Hour sessions shared through a link on tsbvi.edu/coffeehour. Once you're on that page, you can scroll down under the list of sessions where it says visit the new TSBVI outreach Coffee Hour archives. I believe it's also at the top now. That's the link that will take you to the recordings, handouts, transcripts and chat session. To obtain your CEUs, respond to the evaluation that will be e-mailed to you. You will enter the code given at the end of today's session and the CEU certificate will automatically generate upon completion of that evaluation. There's no opening code only a closing code and it will be given at the end of today's presentation. We'll stop the presentation at 3:55 to give you your code and announcements. I'm happy to present Ben Shaberman and Michelle Glaze. 

>>Michelle: Thank you for having us. Welcome, everyone. We are excited to be here with you today.  My name is Michelle Glaze. I'm the associate director of professional outreach for the foundation Fighting Blindness. I joined with Ben Shaberman two years ago to build a professional outreach department but I've been connected with the foundation Fighting Blindness since 2011. I was diagnosed with retinitis pigmentosa in 2004 and understand the journey that individuals go through when they're diagnosed with a blinding condition like retinitis pigmentosa or many others. I am just elated to be a part of this team to help raise awareness with the professional communities about resources that are available to individuals with retinal degenerative disease through the foundation.  Today we're going to highlight some of those resources. We're going to talk a lot about genetic testing and dive into clinical trials and research that's underway. Before we dive into the presentation, I do want to give Ben Shaberman, senior director of scientific outreach the opportunity to introduce himself and then we will go ahead and get started. So, Ben. 

>>Ben: Well, like Michelle said, thank you very much for having us as presenters. We're excited to tell you about our programs and the great research underway. My role at the foundation is to communicate the science to both our constituents and eye care professionals. And, again, I am privileged to be here. Michelle, take it away. 

>>Michelle: All right. There you go. Thank you.  So I'm going to start just by giving you a little bit of an overview about the foundation Fighting Blindness, who we are and what we do. The foundation was established in 1971 by a group of individuals who either were impacted by a blinding retinal disease or they had a loved one who was.  We've been celebrating 50 years of driving research and the mission is to drive research to find treatments and cures for blinding retinal diseases.  Over the last 50 years the foundation has developed into the largest private funder of retinal degenerative disease research and the organization has raised over $116 million towards this mission.  Really our goal is to eradicate the entire spectrum of retinal degenerative diseases. The research we fund is for diseases like Stargardt disease, Usher Syndrome and other related conditions. The foundation funds lab translational research, clinical trials. We do offer some career development awards, and we also have a patient registry as well as a genetic testing program. We fund some natural history studies. So there's a variety of research that we back and support. We have a variety of resources available not only to individuals who are impacted by these diseases but also to the professional community. You know, that work with patients and individuals as they're walking through their journey with vision loss. Our team really seeks to not only provide information and support, but also to provide a lot of hope for patients and that's what our organization is all about. So our website is really full of information for patients when they're newly diagnosed all the way through looking at the clinical trial pipeline that's underway and our genetic testing program.  There is just a vast amount of information available on our website and it's a great resource for individuals as they're learning about their retinal disease and also trying to figure out how to move forward and what's going on in the research and clinical trial world currently for inherited retinal diseases. We do have a variety of patient support services besides our website. Individuals can call into our patient support line or e-mail info@fightingblindness.org if more specific information is of interest. We do have some opportunities for folks to get involved and to learn about who we are and what we do. We have chapters in over 40 markets and these are volunteer led.  This is a real sense of community and support so it's an opportunity for people to connect with others who have an inherited retinal disease or age related macular degenerative. I didn't know any individuals that were impacted by these diseases, maybe one that I could think of but I didn't know much so connecting with others is really important and the foundation does a great job of creating opportunities for individuals to do so. With the world that we live in now, many of our chapter events and organizational events are virtual so it really provides an opportunity for anyone anywhere, either within the United States or even internationally to tap in and hear about what's going on. In fact, we have a webinar this weekend, the 22nd that's focused on clinical trials and that is open to individuals, patients, families, professionals. If you are interested in tapping into that or listening in, you can go to our website to register and RSVP. We do host several events as well. These are predominantly fundraising opportunities. We have a nonprofit organization and to drive research it's expensive so, you know, we do have our flagship fundraiser vision loss, which will be virtual in June. We have some other events. We hosted an event called Hope from Home to celebrate our 50th anniversary and then there are different little regional activities and programs that are set up across the U.S. Dining in the Dark is one that has been done for quite a while through the foundation. For the eye care professional community we do offer continuing education courses. We now have three that we have hosted that are enduring on our professional outreach web page, excuse me. It's for eye care professionals on our website and those are available for one CME or one cope credit or eye care professionals that are interested in that. And then we have our My Retina Tracker or open access genetic testing program and I'd like to spend a little bit of time today talking about that so that everyone is aware of this resource that is available for the foundation. Before I dive into the program, I want to talk a little bit about why genetic testing is important. It's really necessary to understand that even if you've been or if an individual has been clinically diagnosed with an inherited retinal disease, genetic testing can really solidify that prognosis. People can go and get a genetic test to confirm a clinical diagnosis and in many cases that can change. It does provide a more accurate overview of what an individual might expect in an inherited retinal disease. We actually work with our partner Informed DNA and there was a study done that showed that a clinical diagnosis changed for IRD patients in about 13% of cases. And that's because many of the diseases can look alike.  I myself, when I was first diagnosed, it was thought that I may have Stargardt disease because I have a lot of central vision loss. Whenever I did genetic testing it was confirmed my mutations were RP1, which is retinitis pigmentosa. Mine is much different than the typical or, you know, classic form of RP. So it is important to know for sure.  It can also -- genetic testing can also confirm inheritance patterns and it can help individuals and families to understand risk for other family members. For those of us that are parents, I know that's really important. I wanted to know is there any chance that my son will be affected. And so genetic testing and genetic counseling did help me to understand that. And people might want to know as well will other children be impacted, could my siblings be impacted or other family members. So genetic testing can help with all of this. There are over 40 clinical trials underway right now in the inherited retinal disease space and Ben is going to talk about those in more detail. There's also an FDA-approved gene therapy called LUXTURNA that's specifically for mutations and RPE65. In order for individuals to qualify for most clinical trials and to qualify for LUXTURNA, genetic testing is often required. Another important reason to get genetically tested. So genetic testing can be expensive and so the foundation has come up with a no-cost genetic testing option. This is a best in class partnership between Blueprint Genetics, Informed DNA, and the Foundation Fighting Blindness.  It is really a holistic approach to understanding not only the genetic diagnosis but understanding how the inherited retinal disease may impact you moving forward. The registry is an opportunity for individuals to get involved in clinical trials, if interested. And so we'll go through kind of the specifics around this program as we move forward in these slides. But I think it's important to note that the My Retina Tracker program is an open access program through the Foundation Fighting Blindness really is a way to help patients and families manage their inherited retinal disease. It helps to drive clinical research, to find treatments and cures, and it puts patients on the right path moving forward and walking through their journey with vision loss and potentially to treatments down the road. The open access genetic testing program or the My Retina Tracker program is open to all patients in the United States who have been clinically diagnosed with inherited retinal disease. If an individual has received genetic testing with a panel of more than 32 genes over the last four years, they wouldn't qualify for the no-cost option at this time but certainly in the future individuals may. So, you know, if there was a test that was already done, you know, hold tight. There may be some opportunities moving forward for the no-cost option through the Foundation. We have had more than 8,000 people order tests since 2017. And this is a test that has to be ordered through a physician. If an individual wants the genetic testing, they need to go to their eye care professional. The eye doctor will have to register through blueprint genetics and the test is ordered through blueprint online. It takes four weeks to get the test results back.  These tests are usually done via a saliva test or a buckle swab. It's rare that eye care professionals utilize the blood option but that is an option as well. Patients in this program are required to receive genetic counseling. We do offer that at no cost to patients through Informed DNA. But if an eye doctor that's ordering the test has a genetic counselor on site they may use their own counselor and we highly encourage individuals, once they have received genetic testing, to register with the My Retina Tracker Registry I'll talk about later. So I think it's really important to note that patient privacy is something that we really are concerned about. We want to make sure that an individual's demographic information and medical history is not shared with any third parties but we also want to make this information available to researchers and biotech companies that are looking for individuals for clinical trials. So what we do is we make sure that all information is de-identified. We do not share any personal information with third parties at all. Patient privacy is very important to the foundation and a very important aspect to this program. It is important to note that not all genetic testing options or genetic testing programs out there adhere to this type of privacy so it's something to keep in mind if you are seeking genetic testing. So I want to take a minute to dive into the three different aspects of the My Retina Tracker open access program. I mentioned that blueprint genetics is our partners for the genetic testing portion. This is a very, very broad panel that they use. Blueprint looks at 322 genes in the My Retina Tracker panel and it is a way for physicians to really get a clear picture of what's going on genetically. The blueprint panel does also include mutations in RPGR, which is the leading cause of retinitis pigmentosa and we want to make sure we highlight that because there are some other genetic test options out there that do not. There was a study done that showed that about 6% of patients had RPGR mutations if they had an inherited retinal disease and while RP is usually a disease that we see in males, they found that 24% of RPGR patients were actually female. Females are usually carriers but they can be impacted so it is important that this gene is looked at and that is part of the blueprint panel. There's some other information on this slide that goes into further detail. I think it's just really important to note that the Blueprint panel is going to give you a very clear view of what's going on genetically because it is very broad and very deep. I mentioned -- I'm sorry.  Ben, can you go back? 

>>Ben: Whoops. Sorry. 

>>Michelle: There we go. Can you go to Informed DNA? There you go. Okay. So I mentioned Informed DNA for our no-cost genetic counseling option. And it is very important that when you receive genetic testing you also receive genetic counseling. These tests can be very, very complicated even for eye care professionals sometimes to interpret. Our partner Informed DNA has licensed counselors in every state in the U.S. and over the last three years they've counseled more than 3,000 IRD patients. These individuals are very, very knowledgeable and inherited retinal disease and in fact more than 90% of doctors that utilize the My Retina Tracker open access program also utilize Informed DNA for genetic counseling. The counselors have an average of about seven years of genetic counseling experience. These folks are very patient-centric. They take a lot of time with patients and they want to make sure that patients clearly understand not only their genetic test results but also implications for the future. Now while the Informed DNA option is no cost to individuals that are utilizing the My Retina Tracker program, it also can be available to patients if they've already received genetic testing from another program. For myself, in 2015, I received genetic test results from a completely different program. It wasn't at all related to the Foundation's My Retina Tracker program but I did not understand my genetic test results clearly. I had mutations in RP1. One was clearly pathogenic and the other was called a variant of unknown significance. And what that meant for me is it was unclear as to whether or not my retinitis pigmentosa was dominant or recessive. That was very important to me because I have a little boy and I worried about whether or not he would be impacted by the disease as he grows up. And so I actually reached out after joining the team and being encouraged by Ben, reached out to Informed DNA and I was able to get counseling through Informed DNA. I only paid a $50 co-pay which was covered by my insurance. I can't tell you how wonderful my counselor was. She spent an hour and a half on the phone with me during the first visit. She went through my family history, asked a ton of questions. She went through my genetic test results, which I had sent to her, and then she asked some questions that uncovered that both my mom and dad had been genetically tested. She wanted to review those results. She wanted to take a look at the variant of unknown significance. She took about a month to go through and do that further research and then she scheduled a second consultation for me. And at that time she was able to share with me that my variant of unknown significance had been reclassified to likely pathogenic, taking a look at that re-classification and the genetic test results that I had provided to her for myself, my mom, and my dad and a family history, she was able to tell me that my RP is recessive, and that was a huge relief for me and I would have never known that had I not really dug in deep with a genetic counselor. I just wanted to highlight that story because I can't say enough about Informed DNA and also they provide a telephone-based or telemedicine-type of approach.  You know, an individual doesn't have to worry about getting a ride to a facility or finding an office.  All you have to do is answer the phone when they call you, the consultation occurs in the privacy of your own home and so I really highly recommend this option for individuals that are going through genetic testing with our program or even outside of our program, if you haven't received it. And lastly I wanted to highlight the My Retina Tracker patient registry. Now, this is a part of the genetic testing program. It is not required, however, for patients to have genetic testing in order to participate in the patient registry. The registry is open to any individual who has been clinically diagnosed with an inherited retinal disease. It is a global platform. It's free. It is secure and it's very easy to use. It is patient controlled.  It's a way for patients to really raise their hand and be seen by the research community. You enter some demographic information. You answer some questions. You can upload genetic test results if you would like, or any other information that you want to include but it's not required. The more information you provide the more valuable your record. But this is a way for researchers, biotech and pharmaceutical companies who are looking at either starting a clinical trial or trying to recruit patients for clinical trials to find individuals to participate. Without patients and individuals in the clinical trials, the trials can't occur and without trials we will never find treatments and cures. This is the foundation's way of helping to drive research and a very important aspect of the program. At this time we have over 16,000 active or useful registrants in the My Retina Tracker Registry. We have received inquiries from over 150 researchers or biotech companies. Again, this data is de-identified. If a researcher is looking into the registry, they're not going to see Michelle Glaze. They're going to see an alphanumeric code like A 12345. The researcher will reach out to the Foundation Fighting Blindness, let them know they are interested in recruiting that patient for a clinical trial, then the foundation reaches out to the patient and provides all the information that the patient will need in order to self-advocate and/or reach out to that researcher to be involved in the trial if they're interested. So once again this is all in the patient's hands. It is confidential, it is private. If you would like more information about the My Retina Tracker Registry, you can go to myretinatracker.org and you can access anything we talk about today from www.fightingblindness.org. I am going to turn the presentation over to Ben. I want to highlight as we're sharing this information our goal is really to make sure that everyone is aware of all of the resources, the information, the support, the guidance, and most importantly hope that is available and out there for individuals with a blinding retinal disease. And Ben is going to talk a lot about that hope piece now by highlighting all of the clinical trials that are underway. So, Ben. 

>>Ben: Okay. Thank you for that nice overview, Michelle. As Michelle said at the beginning of our talk, the Foundation has been around for about 50 years and during much of that time the research we were funding was research in labs and academic research centers. But over the past about 12, 13 years the research began moving into human studies, into clinical trials. We're at a juncture now where we have more than 40 trials underway for emergent therapies. There's been this tremendous surge in human research and we are of course very excited about that. The Foundation still funds a lot of lab research but one of our focuses is getting research to the point, maybe get it into an early stage clinical trial where we can attract outside investments from biotechs, pharmaceuticals who can then take the emerging therapy and move it through the clinical trial and hopefully get FDA approval.  So, again, that's an important focus for us is getting research to the point where we can attract these outside commercial investments. And our portfolio, today we have about 90 projects. Many are in the U.S. and Europe but we have projects funded all over the world. Now in December of 2017, we really had a game changer for our field, really the field of gene therapy in general. With the FDA's approval of the first gene therapy for the eye or an inherited condition. And that gene therapy is called LUXTURNA. And in case you didn't know, what gene therapy is you're delivering a new gene to the retina to replace the mutated gene that's causing the disease. So in the case of LUXTURNA, that's specifically for people who have mutations in one gene called RPE65. And this work came out of the children's hospital of Philadelphia, that's where the clinical trial started and actually there's a lot of lab research that proceeded that. But early on in the clinical trial, these kids and young adults with RPE65 mutations, which caused a condition called labor congenital amaurosis or RP, kids and young adults were having dramatic vision improvements. Before treatment they were virtual blind, after treatment they were putting away their canes, they could see the faces of their parents and their loved ones, some could even see stars in the sky for the first time. Very dramatic improvements.  As the trial went along, the reports continued to be good. In 2013 children's hospital of Philadelphia spun out a company called Spark Therapeutics to raise the money to get this treatment across the finish line, which as I indicated, they did in 2017.  And so this therapy is applicable to people with RPE65 mutations. That's just one gene. But what's so nice about the success of this project is that it's opened the door for other companies and researchers. It's really paved the path for them to gene therapies for retinal diseases. What has happened subsequent to this FDA approval is many of the companies that are listed on this particular slide have developed gene therapies which are either in clinical trials or moving toward clinical trials, so this field is just exploding. There are gene therapies underway for retinitis pigmentosa. A few companies have had promising results, for achromatopsia, for other forms of RP. Another form of LCA. I'm not going to go through this whole list because it is a long list but it's not a complete list. There are many other gene therapy projects in the pipeline that are approaching clinical trials. Again, the thing to keep in mind about these particular gene therapies is each is targeting a specific gene. And that really harkens back to why it's important to get genetically tested, as Michelle was saying, so you can figure out what gene and gene therapy is appropriate for you. So gene therapy, again, is an approach used to replace the bad gene with a good gene but there are other ways that this approach is being used to restore vision in people. And on the right side of this slide I have a side view image of the retina. And toward the top of this layered image are the photoreceptor cells, the rods and cones that make vision possible.  And those are kind of vertical cells. The cones are in red, green, and blue. The rods are just longer dark cells. But for those of you that can see it, I have this ominous black X going through the photoreceptors and that's to signify somebody who's lost all their photoreceptors and is therefore completely blind due to a retinal disease, maybe RP, maybe AMD. And because of that, gene replacement isn't going to help these folks because there are no photoreceptors to receive the new gene to replace the bad gene. But what's some very smart researchers realized is there are other cells in the retina that survive after photoreceptors are lost, and many people with RP and other retinal diseases.  We call these cells toward the bottom of this diagram ganglion cells, and I have a circle going around those cells. And what these very clever researchers have done is they put a gene in these cells that enables these cells to become light sensitive. Ganglion cells have an important function but they're not the cells that respond to light normally but when you put special genes in them that are light sensitive or make light sensitive proteins, they do bestow light sensitivity to the ganglion cells. The short explanation is that they're using these ganglion cells for people as a backup system for people who have lost all their photoreceptors. And there are a few human studies of this approach underway. And it's actually bionic sight, a new company, that has had some encouraging early results for this approach in the first four, four people in the clinical trial.  These are people that had basically light perception or worse and after receiving this gene therapy they can now see shapes and movement. As an anecdote, people who practice martial arts could see their opponent against the mat. They could see the contrast for the first time. And so those folks are getting low doses of the therapy. The hope is as they go to higher doses it will restore even more vision. The Bionic Sight trial is still at an early stage. And then there are other companies not listed here who have other approaches that are moving toward clinical trials. So what's nice about this approach, it's designed for people who have lost really all their vision and it doesn't require somebody to know what their mutated gene is. It's a gene agnostic approach. Some other projects that the foundation is funding that I wanted to mention include Nacuity, which is a new company out of Dallas that has this very strong antioxidant that one would take orally and the hope is that this antioxidant would slow vision loss. What researchers have learned is that oxidative stress plays a major role in photoreceptor loss in people with RP, Usher Syndrome, and other conditions. So the hope is if you can get this antioxidant and mitigate oxidative stress you can slow down the vision loss. This is in a phase one 2 trial for people with Usher Syndrome but the company is working to move this trial into the U.S. perhaps later this year, early next year. Another company I wanted to mention that we are funding is called Sparing Vision. They're out of France. They are developing a therapy that protects cones. So cones are the photoreceptors in the macular region, in the center of the retina. They give us the ability to read, recognize faces. They give us central vision, color vision. They're really the cells that are most important to our activities of daily living. So this is a one-time therapy that would express a protein to preserve cones. And this is also a gene agnostic approach. This has been in development now for about two decades, actually, but it's gotten to the point where hopefully they will launch a clinical trial later this year or next year. This type of treatment would be appropriate for people with RP, perhaps Usher Syndrome, and perhaps some other retinal conditions. And then finally on this slide I wanted to mention one other company that we're supporting called ProQR out of Netherlands.  Their approach is to correct mutations in our genetic messaging system. So DNA is the genetic information that's stored in our cells. RNA is the messaging system that sends that information back and forth so the cells can function properly. And what ProQR is doing is their treatment can mask mutations in the RNA, in the genetic messaging system and they do this through a tiny piece of genetic material with a long very impressive name called an antisense oligonucleotide. Really what it does is it works like a little mask to cover up the mutation. This approach has worked well in USH2A and LCA clinical trial. The LCA now is in Phase 2/3. The USH2A is moving toward that and they're conducting a trial. That is at an earlier stage and they haven't reported results for that yet. But some good news coming from that human research. So finally to close things out, I wanted to talk about one of the many emerging stem cell treatments. This happens to be in a phase two clinical trial and it's being developed by a company called Reneuron out of the U.K. A few sites in the U.S., the main one is up in Boston. What they're doing is they're taking stem cells that have almost developed into photoreceptors, we call these retinal progenitors and they're projecting them underneath the retina.  Just to back up for a moment, stem cells, the reason we get so excited about stem cells is you can use them to make just about any cell type in the body you might need for a therapy. So in our world we're using these to create new photoreceptors and perhaps some other types. So anyway, in this reneuron trial, they are injecting these retinal progenitors underneath the retina and the hope is they integrate with the existing retina and place the photoreceptors that have been lost to disease. The goal is to help them integrate so that they can restore vision. So this is in a Phase 2 trial and most of the patients in this Phase 2 trial have had about three lines of vision improvement, which is very encouraging. Actually, this vision improvement has been durable out to about 18 months. That's really important. We obviously want this to work for many years. And if the Phase 2 continues to go well, hopefully Reneuron will pursue a Phase 3 trial, which will be the last step before seeking FDA approval. This too is targeting multiple retinal diseases, it's general agnostic for RP, Usher Syndrome, and potentially other clinical trials. I have a picture of this smiling gentleman on the right. That's Jason Comander. He's the lead investigator. We have provided him with career development funding and we've funded a lot of work that helped make this clinical trial possible. And, again, this is just one of the stem cell trials underway. The good news is there's so much great research underway we don't have time to cover it all. So that concludes our talks. I wanted to let people know if you want more information on the research that we've talked about or the genetic testing program, you can go to fightingblindness.org where there are articles on most of the research and much more that we didn't have a chance to talk about. For all the clinical trials that are underway, you can go to clinicaltrials.gov and get information on what those trials are doing, what patients they're recruiting, and potentially you can reach out to these trial sites to see if you qualify. And then, finally, as Michelle implored, it's great if you are a patient with a retinal condition, a retinal degenerative disease that you get into our My Retina Tracker Registry at myretinatracker.org and that way you can get on the radar of those conducting clinical trials. With that, I'm going to open up the chat and -- 

>>Kaycee: We have a few questions. Do you want me to read them to you guys? 

>>Ben: Sure, you can read them. I was going to -- sure. Go ahead. 

>>Kaycee: Okay. Is there any relationship between cortical visual impairment and [Indiscernible] genetics? 

>>Ben: That is an interesting question. Let me preface the answer by saying cortical vision impairment is really at the bottom line a brain issue. Your visual cortex is in the back of your head and your retina and your brain work in partnership to make vision possible. Your retina sends information to your brain and your brain takes that information to create the images that we see.  And to my understanding for the most part there is not a relationship between cortical vision impairment and retinal genetics because the diseases that were involved in the genetic conditions are all causing the retina to degenerate. They don't have an effect on the brain. But it's a really interesting question. I don't know that I've ever gotten it before. I'm impressed. 

>>Kaycee: Can you talk a little bit about who can order genetic testing? 

>>Ben: Sure. So this is a test that your doctor needs to order and any ophthalmologist or retinal specialist in the U.S. can order the test. To be very honest with you, many may not know about it so you may have to do a little education. But they order it online directly from Blueprint. Most optometrists can order the tests. There are a few states in the U.S. where the optometrist has to get approval from an MD but for the most part optometrists can. And so if you are interested, reach out to your doctor. If you're having some issues and your doctor is not receptive to it or they're not willing to set up an account with Blueprint, you can reach out to us at info@fightingblindness.org, and I should have listed that on our slides. And just let us know and we can try to connect you with a doctor who is ordering tests. 

>>Michelle: I will just add, if I may, our website, there is a section where we list retina doctors across the nation. Most of those physicians that are listed on our website do utilize our open access or My Retina Tracker program so that's another resource if you're looking for a physician who might be able to help. 

>>Ben: Right. One of the challenges is if you have one of these rare diseases. A lot of doctors just don't know about them and many are just not willing to put in the effort to learn about them and learn about the resources available. So, anyway. 

>>Kaycee: Our next question says would any of the current research address RP and optic atrophy? 

>>Ben: Okay. So much of the research we talked about does address RP. So optic atrophy involves the optic nerve and the optic nerve, continuing on from my discussion from the cortex, the optic nerve connects the retina to the brain. It's like a cable. And even though it's very close to the retina it's sort of a different world and the projects I talked about really don't involve the optic nerve. With that said, there are researchers and companies that are pursuing research for the optic nerve. I apologize. I'm just not as knowledgeable about those. What I would suggest is if you go on to clinicaltrials.gov you can search conditions like optic neuropathy, optic atrophy and see what clinical trials are ongoing. 

>>Kaycee: We have another question with several words I don't know how to pronounce. I just heard about a treatment medication for IHON. Could you talk about that one? And then it has the drug name Idebenone and then it says lebbers hereditary optic neuropathy. 

>>Lebers hereditary optic neuropathy, we're going to call it LHON. So there is a company called gen sight that has conducted clinical trials for gene therapy for LHON and they are continuing to pursue advancement of that gene therapy. Very interestingly what happened, which may sound like good news but it created a problem for them, is they treated one eye and they left the other eye untreated. And we often do this in trials so you compare the treated eye with the untreated eye. The untreated eye is a control. But what happened in a lot of these patients is the untreated eye improved and the regulators had issues with that because they're wondering if there was some placebo effect or whatever. But the company is trying to figure out why that happened because there were vision improvements, which is a positive thing. And I will say I have heard of -- I'm not sure how to pronounce it correctly, but I will admit I'm not that knowledgeable about it. I think if you go to clinicaltrials.gov and search on it, you can see what's happening in the clinic for that treatment, that potential treatment. 

>>Kaycee: Those are all the questions that are currently in the chat. 

>>Ben: Well, those were excellent questions. Thank you. And, again, if folks out there think of something or they want more information, I saw that  Kaycee, you put our e-mail address in the chat, info@fightingblindness.org. Yeah, we're having educational events all the time, as Michelle said.  We have a clinical trials webinar this Saturday from noon to 2:00 eastern time. And it's free, well-attended and we have some excellent speakers and if you want to hear me talk again, I'm one of the speakers as well. 

>>Kaycee: Ben, one more question just came in.  What about other genes in LCA? Anything in the pipeline? 

>>Ben: Wow, do you have another half hour to talk about LCA research? [ Laughter ] But that isn't a facetious comment. I'm saying that because there is a lot of good LCA research in the pipeline. A company called aCena has a trial for something we funded. That's getting some encouraging early results. There are treatments in the pipeline for the LCA gene CRB1, RP grip 1, NAT1, LCA5, and I'm sure I'm not covering them all. One of the challenges with LCA, for those of you who don't know, LCA causes very significant vision loss very early in life. It's really the most severe retinal disease. And the challenging thing beyond causing significant vision loss is there are more than two dozen genes, each of which when mutated can cause LCA. So we're going to need -- if we're going to address LCA genetically, we're going to need a lot of therapies. But we're working on it and you're going to be hearing more and more about these LCA therapies as they move through the pipeline. 

>>Kaycee: Someone shared that their children have an LCA diagnosis but their type of LCA has not been identified. Do you have suggestions on what they should do for that? 

>>Ben: So the first question I have -- and actually what I would suggest because I think this is a dialogue that needs to occur. I don't have just a pat answer. But if this person wants to reach out to me or Michelle, Cynthia put our e-mail addresses in the chat and we can start having a dialogue. The first question I have is when was this person genetically tested last and what kind of testing did they get? We can talk further. And if you send an e-mail to us, we will also send you a link for the webinar on Saturday. Since I don't have it handy.  Sure. You're welcome. To the people who are saying thank you. It is. It's 11:00 a.m. central time, noon eastern. And just to give you a little preview, I will provide an introduction to clinical trials. It will be different from what I talked about today. But then we have Paul Yang from Oregon health and science university. He's an outstanding clinical researcher. And also representatives from the companies Biogen and Janssen who will be talking about their clinical trials. Noah, thank you for finding the link to our webinar and posting it in the chat. 

>>Michelle: Very interactive group. Wonderful. 

>>Kaycee: All right. If there aren't any more questions, I will go ahead and give us our closing announcements. Thank you so much, Michelle and Ben for being with us today. 

>>Ben: Our pleasure. 

>>Michelle: Thank you for having us. 

>>Kaycee: Absolutely. On May 20 we have Dr. Christine Roman coming to do a Q&A and then on May 24 we have occupational therapy review of sensory processing with Lisa ricket so check our Coffee Hour website, tsbvi.edu/coffeehour for the upcoming registration information. Again, to obtain your CEUs today, respond to the evaluation that will be e-mailed to you from escWorks. You'll enter the code and the CEU certificate will automatically generate upon completion of that evaluation. Also the handouts and recordings from this and past sessions are available through a link on our Coffee Hour page, tsbvi.edu/coffeehour. Once you're on that page, you'll see a link for the top that says visit the new TSBVI outreach Coffee Hour archives. That is a link that will take you to recordings, handouts, and chat information. On the evaluation from escWorks there will be two boxes, No. 10 and 11 that say additional comments you would like to share with the presenters and additional comments you would like to share with the event planning committee. Please let us know if you have any ideas for future Coffee Hours. We're currently planning for next school year right now, so we would love to hear from you. Thanks again to Michelle and Ben and thank you all so much for attending. 

>>Ben: Thank you. 

>>Michelle: Thank you.