3 min read April 2024 — In an interview with Invest:, Shankar Musunuri, chairman, CEO and co-founder of biotech company Ocugen, shared how it continues to grow as a dynamic and innovative company, focused on revolutionizing medical treatments through gene and cell therapies, and its contribution to global public health through cutting-edge vaccine solutions.
How has Ocugen grown in the past year and how does this growth reflect on the broader dynamics of the Greater Philadelphia region?
Over the past 12 months, our modifier gene therapy platform has experienced significant advancements, with our phenomenal team successfully advancing three gene therapies through to clinical trials. In September 2023, we announced the most recent positive clinical study update from the Phase 1/2 trial of OCU400 — a modifier gene therapy product candidate for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). OCU400 has the potential to be a gene-agnostic, one-time treatment for life for patients with RP and LCA — conditions for which there are more than 125 mutated genes associated. In the U.S., RP and LCA affect 110,000 and 15,000 people, respectively.
Additionally, we embarked on two pivotal clinical trials addressing rare disease as well as a blindness disease affecting millions. One of these trials focuses on age-related macular degeneration (dAMD), specifically targeting late-stage patients with geographic atrophy (GA). This trial, initiated at Phase 1/2, is a significant undertaking, with 1 million people affected by GA in the U.S. alone. Simultaneously, another Phase 1/2 trial was initiated for Stargardt disease, a genetic eye disorder that causes retinal degeneration and vision loss in approximately 41,000 Americans for which no treatment options exist.
We look forward to continuing our scientific contributions to the biotech and pharma hub in the greater Philadelphia region and making a positive economic impact at the same time.
What makes Philadelphia a great place to be?
Philadelphia boasts an excellent ecosystem, with top-tier medical schools and universities driving cutting-edge research. The existing healthcare ecosystem, complemented by recent graduates along with pharma and biotech veterans, positions the region uniquely.
As biotech companies experience success and growth, a concerted effort is required to transform the funding landscape in Philadelphia. The aim is to emulate the environment in Boston, where there has been an influx of funding and established public/private partnerships to ensure stability and longevity.
This shift in paradigm is essential to nurture a vibrant startup culture, allowing emerging companies to grow and become pioneers in delivering critically needed medicines that have the potential to transform and save lives globally.
What are the main areas of focus for Ocugen in its current research, and which areas do you consider to have the highest potential or opportunities?
Ocugen is at the forefront of transformative medical advancements, with a primary focus on our first-in-class gene therapies to address hard to treat blindness diseases.
The uniqueness of our modifier gene therapy lies in its exceptional ability to reset homeostasis, offering the potential for a one-time treatment to restore function in non-dividing cells, such as photoreceptors. This approach represents a paradigm shift in treating degenerative conditions, demonstrating our commitment to ushering in a new era of medical interventions.
Expanding our gene therapy endeavors beyond rare disease, Ocugen has introduced the OCU410 program targeting geographic atrophy, which is a late-stage manifestation of dry age-related macular degeneration (dAMD). With a staggering 10 million patients in the United States and 266 million globally affected, this program utilizes our unique technology to address four crucial disease pathways, differentiating itself from current treatments that focus on just one pathway. The potential impact of the OCU410 program extends far beyond the numbers, offering hope to individuals grappling with the debilitating effects of AMD.
Complementing our gene therapy focus, is our regenerative cell therapy to treat articular cartilage lesions – NeoCart®. NeoCart’s 3D technology for cartilage repair is a groundbreaking approach with the potential to help more than one million patients in the United States facing limited treatment options. The revolutionary nature of our technology extends beyond the current cell therapy option that is only 2D.
In vaccines, Ocugen is pursuing inhalation technology, marking an evolution in vaccine delivery. Originating from our early response to provide a non-mRNA option to fight COVID-19, this platform presents a distinctive solution to potentially provide protection against severe disease, increase durability and prevent transmission of viral threats. While our near-term focus is on COVID-19 and flu, the long-term vision encompasses a broader range of respiratory diseases This forward-looking approach positions Ocugen as a trailblazer in addressing public health challenges.
As we navigate the frontiers of medical innovation, Ocugen remains resolute in its pursuit of excellence and its mission to redefine the landscape of healthcare. Our commitment to research, innovation, and patient-centric solutions positions us as a beacon of hope for those in need. As Ocugen continues to grow as a dynamic and innovative company, our focus remains on revolutionizing medical treatments and contributing to global health through cutting-edge solutions in gene and cell therapies and vaccines. Our mission is anchored in making a substantial and lasting difference in the lives of patients worldwide.
What is your outlook for Ocugen over the next two to three years, and how does this inform your priorities for this time frame?
In the next three years, our ambitious goal at Ocugen is to launch major gene therapy programs. Our focus extends beyond becoming a cutting-edge biotech leader in the Philadelphia area as we are dedicated to bringing game-changing gene and cell therapies and vaccines to market globally.
Our strategy involves being opportunistic in exploring vaccines and cell therapies, and conserving capital to advance our gene therapies. We are deeply committed to helping our patients and at the same time making the right business decisions to create value for our shareholders.
The next critical phase for Ocugen involves moving our lead candidate—OCU400—into Phase 3 clinical trials in early 2024. OCU400, along with OCU410 and OCU410ST, have the potential to provide one-time, curative therapies for life-redefining the landscape of gene therapies.
As we anticipate the economic realities associated with gene therapies, acknowledging the current pricing models of $1 to $3 million-plus per patient in the United States, we are dedicated to working with payers and government agencies to help make our gene therapies more accessible.
In navigating the cutting edge of technology, we recognize our moral and ethical obligations. Our commitment extends to finding ways to ensure market access for patients, thus making a positive impact on their lives and fostering a more inclusive healthcare landscape.
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