In which context was Ocugen founded and what is its core purpose in the biopharmaceutical market?
As an entrepreneur and a scientist, I am always open to new business opportunities in healthcare that can help address unmet medical needs. I was extremely impressed by the ophthalmology research our Co-founder, Uday Kompela, PhD, was conducting and began Ocugen to further develop his work in blindness diseases. Subsequently, we began working with Dr. Neena Haider at Harvard Medical School, who discovered a modifier gene therapy approach to treating retinal disease. In addition to eye diseases, we are focused on vaccines to positively impact public health. Finally, we have a Phase 3-ready regenerative cell therapy for cartilage repair, NeoCart®. Our passion for patients and unwavering commitment to bring new treatments to people who currently have no hope, is what drives the work we do every day. Our tagline "Courageous Innovation" means that we are not afraid to push boundaries in pursuit of discoveries that could help millions of patients.
Accessibility is also critically important. It’s not good enough to bring breakthrough gene therapies to market if patients cannot afford them. We must be diligent in pricing so that even the most vulnerable populations can have access to treatment. We are very much aware that this is a monumental task, and that funding is what usually stands in the way, but we are dedicated to treating all patients.
How did you decide to move from ophthalmology to the broader set of therapy areas you are covering, what is the connection point?
There are a lot of commonalities between gene and cell therapies and vaccines—the science behind the way they work, the laboratory process, and in manufacturing. While they seem like very disparate medical endeavors, the shared scientific platform makes complete sense for us to work in all three.
Getting our hands into COVID-19 vaccines came as a natural decision considering our team's “Big Pharma” experience working on one of the world’s most needed and widely distributed pneumococcal vaccines. We are also committed to bringing new options to the COVID-19 landscape to offer choice and deliver next generation technology as the virus evolves. Similarly, as there is currently only one regenerative medicine for cartilage repair on the market, we think it is beneficial to offer an enhanced alternative. As a result, we are pursuing the development of NeoCart®, which is a three-dimensional tissue-engineered disc of new cartilage manufactured by growing the patients' own cells.
When do you think the shift from traditional treatments to gene and cell therapy will finally occur?
Gene and cell therapy in Europe and the U.S. has gained a lot of traction, but most companies are taking the path of least resistance and targeting only a few thousand patients per treatment option. Moreover, the customary method is a general one rather than individualized. With gene therapy, there can be different mutations for the same gene and an all-around approach is not necessarily the best. Instead, we believe we have a potential solution: modifier genes that operate on a broader level and reset homeostasis of the retina overall without removing or altering bad genes. In this way, we could potentially control disease progression. Regulators usually subsidize treatments for rare diseases that affect a small number of patients, but we intend to direct our gene therapies towards much larger groups. As a result, we need to find the best way to sponsor our treatments because it is not feasible to ask for millions of dollars from our patients.
We are not only working on rare diseases but eye diseases that affect much larger populations. There are 10 million patients who are suffering from Dry Age-Related Macular Degeneration in the U.S. We have encouraging data based on the preclinical work for OCU410 and next year we will go into the clinic targeting late-stage, geographic atrophy patients.
We are very excited about our work in gene and cell therapies and look forward to seeing other biotech companies emerge alongside us. Pharma industry trends indicate that within the next five to ten years there will be significantly more gene and cell therapies commercially available.
What makes the COVID-19 vaccines Ocugen is working on different to existing options?
As we have seen, the virus is continually mutating and the vaccines that are available often cannot offer enough protection to new variants. COVAXIN™, developed by our partner Bharat Biotech, is built on a similar platform to the polio vaccine. COVAXIN™ uses a novel adjuvant (Algel-IMDG) that works together with the inactivated virus to generate an immune response against several antigens. Our second vaccine in development, OCU500, generates local mucosal immunity in the nasopharynx and can generate T-cell responses, which are essential for reducing infection and transmission of COVID-19. OCU500 is unique and could potentially be used as a universal booster.
