Ocugen is a biotechnology company that focuses on innovative solutions in the fields of gene and cell therapies, as well as vaccine development. Their primary scientific platforms include a modifier gene therapy platform designed to address inherited retinal diseases, such as retinitis pigmentosa (RP) and geographic atrophy associated with dry age-related macular degeneration (dAMD). The OCU400 gene therapy utilizes an adeno-associated virus (AAV) vector to deliver the NR2E3 gene to the retina, aiming to preserve and restore retinal function. Clinical studies have shown promising results, indicating that OCU400 is generally safe and well-tolerated, with a significant percentage of treated subjects demonstrating stabilization or improvement in visual function.

In addition to OCU400, Ocugen is developing OCU410, another gene therapy targeting geographic atrophy, which employs the RORA gene to address multiple pathways involved in the disease's progression. This therapy aims to provide a one-time treatment option through subretinal injection, potentially reducing the need for frequent interventions associated with current therapies. Furthermore, Ocugen is also advancing OCU410ST for Stargardt disease, which has received Orphan Drug Designation from the FDA. Overall, Ocugen's commitment to addressing unmet medical needs through its innovative gene and cell therapy platforms reflects its dedication to improving patient outcomes in the realm of ocular diseases.

United States

Ocugen, Inc. is a 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 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, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market.

Endocrine System Diseases

Eye Diseases

Genetic Diseases

Infectious Diseases

Metabolic Diseases

Musculoskeletal Diseases

Ocugen

Retinitis Pigmentosa

OCU400&nbsp;(AAV-NR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases (“IRDs”). It consists of a functional copy of a nuclear hormone receptor (“NHR”) gene,&nbsp;NR2E3, delivered to target cells in the retina using an adeno-associated viral (“AAV”) vector.&nbsp; As a potent modifier gene, expression of&nbsp;NR2E3&nbsp;within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration.
Gene therapy

OCU400

Articular cartilage defects in the knee

NeoCart® combines breakthroughs in bioengineering and cell processing to enhance the autologous cartilage repair process by merging a patient’s own cells with a fortified 3-D scaffold designed to accelerate healing and reduce pain.

NeoCart® (Autologous chondrocyte-derived neocartilage)

Rare genetic disease

Stargardt disease

OCU410ST&nbsp;(AAV5-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease. OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.

OCU410ST

Age-related macular degeneration

Geographic atrophy

About OCU410 
OCU410 utilizes an AAV delivery platform for the retinal delivery of the&nbsp;RORA&nbsp;(ROR Related Orphan Receptor A) gene. The&nbsp;RORA&nbsp;protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually.

OCU410

Eye disease

Leber congenital amaurosis

Diabetic macular edema

OCU200, a recombinant fusion protein consisting of tumstatin and transferrin, for treating diabetic macular edema (DME). OCU200 possesses unique features that potentially enable it to treat vascular complications of DME. Tumstatin is the active component of OCU200 and binds to integrin receptors, which play a crucial role in disease pathogenesis. Transferrin is expected to facilitate the targeted delivery of tumstatin into the retina and choroid and potentially help increase the interaction between tumstatin and integrin receptors.

OCU200

COVID-19

OCU500

Infectious disease

COVAXIN is a highly purified and inactivated vaccine that is manufactured using a vero cell manufacturing platform with an excellent safety track record of more than 300 million doses supplied.
<a href="https://ir.ocugen.com/news-releases/news-release-details/ocugens-covid-19-vaccine-co-development-partner-bharat-biotech-0">SOURCE</a>

Ocugen

Company Details