Therapeutic
Areas

People all over the world live with health conditions for which there are no treatments and no hope. We can do better. We must do better. By taking science in new directions and looking at old challenges in new ways, we can be of service to people by delivering options where none or few exist.Our new Cell Therapy Program is focused on utilizing the body’s own cells to create new cartilage as a means of repairing full-thickness, articular cartilage lesions in adult knees.

Therapeutic Areas

Ocugen, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one mutation-agnostic drug — “one to many,” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, Retinitis Pigmentosa, Dry Age-related macular degenerationand diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. and Canadian markets.

  • Eye Diseases
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    Ocugen’s focus centers on finding innovation that will make hard-to-treat blindness diseases a thing of the past. Below are areas our researchers and collaborators explore daily to help people and families in need.

    About Retinitis Pigmentosa (RP)

    Retinitis Pigmentosa (RP) is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina. According to the National Eye Institute, it is estimated that RP affects approximately 1 in 4,000 people (about 2 million patients), both in the U.S. and worldwide.

    More than 150 genetic mutations are known that cause RP and Leber Congenital Amaurosis (LCA). They represent 60% of the total RP population. The remaining 40% of people with RP cannot be genetically diagnosed making it difficult to develop individual treatments.

    There is currently no approved treatment which slows or stops the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Gene-replacement therapies are promising but are limited to treating just a single mutation and therefore cannot address the multiple mutations in RP. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect which may still cause stress and toxic effects. Therefore, the development of gene specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved.

    About Age-Related Macular Degeneration (AMD)

    Age-Related Macular Degeneration (AMD) is a prevalent eye disorder, characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in your direct line of sight.

    Dry AMD, which affects about nine to ten million Americans, involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. Dry AMD accounts for 85-90% of the total AMD population, and there is no approved treatment.

    How AMD causes blindness
    AMD is characterized by thickening and loss of normal architecture within Bruch’s membrane, lipofuscin accumulation in the retinal pigment epithelium (“RPE”), and drusen formation beneath the RPE in Bruch’s membrane. These deposits consist of complement components, other inflammatory molecules, lipids, lipoproteins B and E, and glycoproteins.

    About Diabetic Retinopathy and Diabetic Macular Edema

    Diabetic retinopathy (DR) and diabetic macular edema (DME) are the most common vision-threatening diseases occurring in people with diabetes. Approximately 7.7 million people are affected with DR and approximately 745,000 with DME in the United States. These numbers are expected to further increase as the number of people with diabetes increases.

    Options are limited
    There are limited treatment options available for people with DR and DME and there is a need for the development of safe and effective therapy. Currently, approved anti-VEGF therapy does not work effectively in approximately 50% of people with DME. These approved therapies target only one pathway associated with DR and DME, either angiogenesis with anti-VEGF therapy or inflammation in case of steroid therapy.

    About Wet-AMD

    About 10-15% of people with age-related macular degeneration (AMD) progress to the advanced “wet” form. It’s generally caused by abnormal blood vessels that leak fluid or blood into the macula. (The part of the retina that’s responsible for central vision.) The result can be irreversible damage to photoreceptor cells and rapid, severe vision loss, particularly in the center of the field of vision, causing significant functional impairment. Wet-AMD accounts for 90% of all AMD-related blindness.

  • Orthopedic Disease
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    Our new Cell Therapy Program is focused on utilizing the body’s own cells to create new cartilage as a means of repairing full-thickness, articular cartilage lesions in adult knees.

    Articular cartilage lesions are a serious and often mobility-limiting condition. When the cartilage is healthy, it makes movement easy, allowing the bones to glide over each other with very little friction, but it can be damaged by injury or normal wear and tear. Cartilage that is damaged can, over time, cause pain and reduce one’s ability to function. Small articular lesions have a limited capacity to self-repair, and full thickness injuries have no ability to naturally heal. There are no blood vessels or nerves to support healing, and as cartilage matures, chondrocytes have limited ability to replicate. Untreated damage eventually can lead to osteoarthritis.