Ocugen, Inc. Common Stock Canaccord Genuity's 46th Annual Growth Conference

Thank you for having me.

Great question. I think, we, as Ocugen today, I believe we lead ophthalmology gene therapies. Why? We have three programs in late stage. If you take the vision loss diseases today with significant unmet medical needs, IRDs, which are inherited diseases, retinitis pigmentosa and Stargardt, and on the age-related macular degeneration, dry AMD is much bigger, right? It is 90% of the AMD compared to wet AMD is 10%. All these diseases have significant unmet medical needs, and these are blindness diseases today with very limited therapies or no therapies across the globe. Today, I am proud to say Ocugen is in late stages, all three of them. We are targeting two BLAs next year, for both retinitis pigmentosa and Stargardt and AMD in 2028. So that is what Ocugen is.

If we are able to get the approvals in all the major markets, U.S. and EU, including Japan, in the next 3 years and launch these therapies, that will be a monumental task because that can change the paradigm of all these unmet medical needs across the globe for blindness diseases. Millions of patients can get benefited. Today, as we are talking, a lot of the patients across the globe are becoming legally blind, right? That's what Ocugen wants to do. Not only take these gene therapies in the next 2 years, work hard to file those BLAs or market authors in Europe, across the globe, get them approvals, but we want to work even harder to provide market access to patients who need them globally.

Not after 5 or 10 years, in a parallel process.

Yeah. Nuclear hormone receptors, these are modifier genes. Simple concept is genes don't work in isolation. They work as a network. When somebody has a genetic defect, it's there from birth. So you have the misfolded or toxic protein it's producing, right? And that has an impact on network of genes. So there are genes which are transcription factors responsible in retina for key functions, phototransduction, cell development, metabolisms, cell survival. Some of these key functioning genes are impacted by defective gene, irrespective of what defect it is, right? The technology, it identified the missing link. For something like retinitis pigmentosa, mostly a disease that impacts phototransduction pathways. There's a modifier gene, NR2E3, that's invented by Dr. Neena Haider at Harvard, and she invented these modifier genes. Why? She is a genetics person. She worked on human genome before.

She spent about 20 years inventing these genes. The invention was, you take RP, you take multiple mutations. Every time there is a mutation, she is tracking it back. Oh, these transcription factors are getting impacted. They provide key functions. Then, NR2E3 is a link. How about we upregulate NR2E3? Everything comes to sync in a cellular homeostasis. She also worked on RORA, which is another modifier gene, because when you take diseases such as Stargardt or AMD, one is inherited, another one is age-related. But they both have same complex pathways for progression of the disease, right? Oxidative stress, lipid metabolism, inflammation, complement system. This other gene regulates all those pathways. It also creates a healthy environment for retinal cells, including RP, to survive, which is very important in the case of this. RP has to be protected.

The technology came from Harvard, from Dr. Neena Haider's lab. She did the original work. Then, of course, Ocugen took it, and they exponentially grew that technology platform. Today, not only the IP covers the ability of our modified genes in ophthalmology space, we also cover neurospace. That's the future. Got it.

Yeah. In Stargardt and GA, geographic atrophy, which is a late stage of dry age-related macular degeneration, in both the cases, of course, you have the macular central vision issues, you have lesion. There are complex pathways. If you take current therapies, the two products approved for GA in U.S., they target complement system. Right? Many companies worked on that. If you take that pathway, that's a final frontier you can call it. It's the last pathway, but the origination is caused by oxidative stress and drusen formation, which is lipid metabolism. Then you got inflammation. A lot of these are important too. Very important. This RORA regulates all these pathways, and we have data in one of those nature publications.

We demonstrated that. Not only it regulates all those pathways, it also has ability to reset cellular homeostasis and create a healthy environment for these cells to survive.

There are certain cells in our human system, right?