Ocugen, Inc., a biotechnology company, focuses on discovering, developing, and commercializing novel gene and cell therapies, biologics and vaccines that improve health and offers hope for patients across the globe.
The company’s technology pipeline includes:
Modifier Gene Therapy Platform — Based on the use of nuclear hormone receptors (‘NHRs’), the company’s modifier gene therapy platform has the potential to address many retinal diseases, including rare genetic diseases, such as RP (OCU400)...
Ocugen, Inc., a biotechnology company, focuses on discovering, developing, and commercializing novel gene and cell therapies, biologics and vaccines that improve health and offers hope for patients across the globe.
The company’s technology pipeline includes:
Modifier Gene Therapy Platform — Based on the use of nuclear hormone receptors (‘NHRs’), the company’s modifier gene therapy platform has the potential to address many retinal diseases, including rare genetic diseases, such as RP (OCU400), with a gene-agnostic approach. The company is actively recruiting patients in the United States and Canada in the Phase 3 liMeliGhT clinical trial for OCU400 for the treatment of RP and is on track to complete enrollment in the first half of 2025. In January 2025, the company announced positive two-year safety and efficacy data across multiple mutations from the Phase 1/2 clinical trial of OCU400. In February 2025, the company announced that the European Commission (EC) has provided a positive opinion from the European Medicines Agency (EMA) Committee for Advanced Therapies for OCU400 Advanced Therapy Medicinal Product (ATMP) classification. OCU410 completed dosing in Phase 2 of the Phase 1/2 ArMaDa clinical trial for the treatment of geographic atrophy (‘GA’), an advanced form of dAMD. Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the OCU410 Phase 1/2 ArMaDa clinical trial included: no drug-related serious adverse events (‘SAEs’), reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (‘LLVA’). In October 2024, the Data and Safety Monitoring Board (‘DSMB’) for the OCU410ST GARDian clinical trial approved enrollment for the second phase of the Phase 1/2 clinical trial. In February 2025, the company announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission. Additionally, the European Medicines Agency (‘EMA’) granted orphan medicinal product designation (‘OMPD’) for OCU410ST for the treatment of ABCA4-associated retinopathies, including Stargardt disease, RP 19 (‘RP19’), and cone-rod dystrophy 3 (‘CORD3’). Both OCU410 and OCU410ST studies have completed the low, medium, and high dose cohorts in the Phase 1 part of the Phase 1/2 trials to date, with OCU410 completing dosing in the Phase 2 part of the Phase 1/2 trials.
Novel Biologic Therapy for Retinal Diseases — OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin. OCU200 possesses unique features, which potentially enable it to treat vascular complications of diabetic macular edema (‘DME’), diabetic retinopathy (‘DR’), and wet age-related macular degeneration (‘AMD’). 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. The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and the company is actively recruiting patients.
Regenerative Medicine Cell Therapy Platform — The company’s Phase 3-ready regenerative cell therapy platform technology, which includes NeoCart (autologous chondrocyte-derived neocartilage), is being developed for the repair of knee cartilage injuries in adults. The company received concurrence from the FDA on the confirmatory Phase 3 trial design and has completed renovating an existing facility into a current GMP facility to support clinical study and initial commercial launch. This facility is needed to generate patient-specific NeoCart implants from chondrocytes derived from knee biopsy. The company intends to initiate the Phase 3 trial contingent on adequate availability of funding.
Inhaled Mucosal Vaccine Platform — The company’s next-generation, inhaled mucosal vaccine platform includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. The company has completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. In January 2025, the company announced that the Investigational New Drug (IND) application is in effect and the National Institute of Allergy and Infectious Diseases (‘NIAID’) intends to initiate a Phase 1 clinical trial in the second quarter of 2025. The company is continuing discussions with relevant government agencies, as well as strategic partners, regarding developmental funding for its OCU510 and OCU520 platforms.
Modifier Gene Therapy Platform
The company is developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including inherited retinal diseases (‘IRDs’), such as RP, Stargardt disease; and multifactorial diseases, such as dAMD. The company’s modifier gene therapy platform is based on the use of NHRs, which have the potential to achieve homeostasis—the basic biological processes in the retina to restore a healthy state from a diseased state. Unlike single gene replacement therapies, which only target one genetic mutation, the company’s modifier gene therapy platform, through its use of NHRs, represents a unique, gene-agnostic approach designed to address not just the mutated gene but provide a molecular ‘reset’ of health and survival of gene networks. OCU400, the company’s lead product candidate in its modifier gene therapy platform, has received Orphan Drug Designation (‘ODD’) from the FDA for RP and LCA, a regenerative medicine advanced therapy (‘RMAT’) designation for the treatment of RP associated with NR2E3 and rhodopsin (‘RHO’) mutations from the FDA, and OMPD from the European Commission (‘EC’), based on the recommendation of the EMA, for RP and LCA. These broad ODD, RMAT, and OMPD designations further support the broad (gene-agnostic) therapeutic potential of OCU400 to treat RP associated with mutations in multiple genes.
The OCU400 Phase 3 liMeliGhT clinical trial is currently underway, with enrollment on track to meet the company’s target submission of a Biologics License Application (‘BLA’) and Marketing Authorization Application (‘MAA’) in mid-2026.
In August 2024, the company received notification from the FDA that it could begin its expanded access program (‘EAP’) for the treatment of adult patients with RP with OCU400. This program is available for patients with early, intermediate to advanced RP with at least minimal retinal preservation who may benefit from the mechanism of action of OCU400 prior to approval of the BLA.
The company also received approval from Health Canada to initiate a Phase 3 trial for OCU400 for the treatment of RP. The Health Canada trial will run in parallel with the United States FDA trial, expediting the ability to potentially provide a gene-agnostic treatment option to approximately 110,000 patients in the United States and Canada.
In January 2025, the company announced positive two-year long-term data across multiple mutations from the Phase 1/2 clinical trial of OCU400, which demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects at two years when compared to untreated eyes. 100% (10/10) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes. Also, treated eyes with multiple mutations and RHO subjects demonstrated a statistically significant (p=0.005) improvement in visual function when compared to untreated eyes. Treated eyes in RHO subjects (who meet Intent-to-treat criteria) for OCU400 Phase 3 trial demonstrated a statistically significant improvement (p=0.01, N=4) with around two-line gain in visual function (LLVA) at two years when compared to untreated eyes.
In February 2025, the company announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU400 ATMP classification. ATMP classification is granted to medicines that can offer groundbreaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential one-time gene therapy for life.
OCU410 and OCU410ST are being developed utilizing the nuclear receptor gene RAR-related orphan receptor A (‘RORA’), for the treatment of GA secondary to dAMD and Stargardt disease, respectively. OCU410 is a potential one-time, curative therapy with a single sub-retinal injection. OCU410 targets multiple pathways associated with AMD pathogenesis, in contrast to products currently approved or under development that treat only one cause of GA, require multiple injections per year, and have safety considerations. OCU410ST has received ODD from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease. OCU410ST has also received OMPD from the EMA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, RP19, and CORD. In March 2025, the company announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU410 and OCU410ST ATMP classification.
OCU410 has completed dosing in the Phase 2 of the Phase 1/2 ArMaDa clinical trial. OCU410ST has completed Phase 1 of the Phase 1/2 GARDian clinical trial, and the DSMB has approved enrollment for the second phase of the Phase 1/2 GARDian clinical trial.
In November 2024, the company announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 ArMaDa clinical trial for GA. The key findings included: no drug-related SAEs, reduced lesion growth, preservation of retinal tissue, and a positive effect on the functional visual measure of LLVA. In February 2025, the company announced dosing has been completed in Phase 2 of the OCU410 ArMaDa clinical trial. Phase 2 was a randomized expansion phase in which 51 subjects were randomized in a 1:1:1 ratio to either one of two OCU410 dose groups or to an untreated control group.
In October 2024, the company announced that the DSMB had approved proceeding to Phase 2 of the OCU410ST GARDian trial using high and medium doses. In November 2024, the company announced preliminary efficacy and safety data from evaluable subjects at six months from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410ST GARDian clinical trial, which demonstrated an 84% reduction in atrophic lesion growth in treated eyes versus the corresponding untreated eyes.
In February 2025, the company announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission. The Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-µL injection of OCU410ST at a concentration of 1.5 x 1011 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity, as measured by best corrected visual acuity (‘BCVA’), and LLVA compared to untreated controls. One-year data will be utilized for the BLA filing.
Novel Biologic Therapy for Retinal Diseases
The company is developing OCU200, which is a novel fusion protein containing parts of human transferrin and tumstatin. OCU200 is designed to treat DME, DR, and wet AMD. The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and the company is actively recruiting patients for the Phase 1 clinical trial. The OCU200 Phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). All subjects will receive a total of two intravitreal injections of OCU200 six weeks apart. Patient follow-up will take place up to three months after the last injection.
Regenerative Medicine Cell Therapy Platform
NeoCart is a Phase 3-ready, regenerative cell therapy technology that combines breakthroughs in bioengineering and cell processing to enhance the autologous cartilage repair process. NeoCart is a three-dimensional tissue-engineered disc of new cartilage that is manufactured by growing the patient's own chondrocytes, the cells responsible for maintaining cartilage health. Current surgical and nonsurgical treatment options for knee cartilage injuries in adults are limited in their efficacy and durability. In prior clinical studies, Phase 2 and Phase 3, NeoCart has shown potential to accelerate healing, reduce pain, and provide regenerative native-like cartilage strength with durable benefits post transplantation. NeoCart was shown to be generally well-tolerated and demonstrated greater clinical efficacy than microfracture surgery at two years after treatment. Based on this clinical benefit, the FDA granted a RMAT designation to NeoCart for the repair of full-thickness lesions of knee cartilage injuries in adults. Additionally, the company received concurrence from the FDA on the confirmatory Phase 3 trial design where chondroplasty will be used as a control group. The company has completed renovating an existing facility into a GMP facility in accordance with the FDA's regulations in support of NeoCart manufacturing for personalized Phase 3 trial material. The company intends to initiate the Phase 3 trial contingent on adequate availability of funding.
Inhaled Mucosal Vaccine Platform
The company is party to an exclusive license agreement (as amended, ‘WU License Agreement’) with The Washington University in St. Louis (‘Washington University’), pursuant to which the company licensed the rights to develop, manufacture, and commercialize a mucosal COVID-19 vaccine for the prevention of COVID-19 in the United States, Europe, Japan, South Korea, Australia, China, and Hong Kong (the ‘Mucosal Vaccine Territory’). In addition, the company internally developed technology related to the flu and COVID-19's vaccine design and filed intellectual property. The company is developing a next-generation, inhalation-based mucosal vaccine platform based on a novel ChAd vector, which includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. The company’s inhaled mucosal vaccine platform is driven by its conviction to serve a major public health concern, which requires the endorsement and support of government funding in order to develop and ultimately commercialize its vaccine candidates. As these vaccine candidates are being developed to be administered via inhalation, they have the potential to generate rapid local immune response in the upper airways and lungs, where viruses enter and infect the body. In October 2023, OCU500 was selected by the NIAID Project NextGen for inclusion in clinical trials. OCU500 will be tested via two different mucosal routes, inhalation and intranasal delivery. In January 2025, the company announced that the IND application is in effect to initiate the Phase 1 clinical trial of OCU500. The company is continuing discussions with relevant government agencies, as well as strategic partners, regarding developmental funding for its OCU510 and OCU520 platforms.
Strategy
Key elements of the company’s strategy are continuing to advance its modifier gene therapy platform into and through clinical development; expanding and exploring partnerships with current and future key collaborators and commercial partners to maximize patient access, global reach, and the value of the company’s product candidates; obtaining government funding to advance its vaccine programs towards commercialization; and advancing the clinical development of its regenerative medicine platform towards market authorization and developing in-house manufacturing capability.
Modifier Gene Therapy Platform and Gene Therapy Product Candidates
The company is developing its modifier gene therapy platform, inclusive of OCU400, OCU410, and OCU410ST for the treatment of multiple IRDs, such as RP, Stargardt disease, and multifactorial diseases, such as dAMD and GA. The company’s modifier gene therapy platform is a cutting-edge technology licensed from SERI, an affiliate of Harvard Medical School, and involves the targeted delivery and expression of one or more NHRs in the disease tissues and is designed to introduce a functional gene to modify the expression of multiple genes and gene networks, which potentially enables it to address multiple retinal diseases with one product.
OCU400 for IRDs
OCU400 is the company’s first product candidate being developed with its modifier gene therapy platform. OCU400 has the potential to restore retinal integrity and function across a range of genetically diverse IRDs. OCU400 consists of a functional copy of the retina-specific NHR gene, NR2E3, delivered to target cells in the retina using an AAV5 vector that has the potential to be used as a gene therapeutic not only for the treatment of retinal diseases associated with mutations in genes, such as NR2E3, RHO, and PDE6ß, but also other gene mutations associated with RP. As a potent modifier gene, expression of NR2E3 may help reset retinal cell homeostasis, metabolism, and visual cycle function (Figure 9). OCU400 has received ODD for RP and LCA, a RMAT designation to OCU400 for the treatment of RP associated with NR2E3 and RHO, and OMPD from the EC, based on the recommendation of the EMA, for RP and LCA. These ODD, RMAT, and OMPD designations represent gene-agnostic broad coverage for RP and are not mutation-specific designations.
The first patient has been dosed in the company’s Phase 3 clinical trial for OCU400 for the treatment of RP. The company received notification from the FDA that it could begin its EAP for the treatment of adult patients with RP with OCU400. The company also received approval from Health Canada to initiate a Phase 3 trial for OCU400 for the treatment of RP. In February 2025, the company announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU400 ATMP classification. The Phase 3 clinical trial is on track to complete enrollment in the first half of 2025, file the BLA and MAA in mid-2026, and pursue commercialization in 2027, if approved.
Modifier Gene Therapy Platform Based on the Use of NHRs
The company’s modifier gene therapy platform is designed to target NHRs to potentially provide therapeutic benefit to patients suffering from genetically diverse IRDs. The use of genetic modifiers represents a broadened means of potentially treating a variety of retinal degenerative diseases, as compared to single-gene replacement therapy. While single-gene replacement therapies have shown tremendous promise in rare retinal diseases, they are highly specific and cannot improve a multitude of disease-causing genetic defects. The company’s modifier gene therapy platform has the potential to restore retinal integrity and function across a range of genetically diverse IRDs and other degenerative retinal diseases, providing the company with significant potential long-term value.
The company’s modifier gene therapy platform encompasses the targeted delivery and expression of certain NHRs that are expressed naturally in retinal tissue. Preclinical studies have shown that NR2E3, a member of the NHR family, is a dual activator and repressor that, with other transcription factors, modulates cell fate and differentiation of rod and cone photoreceptor cells, specialized cells for detecting light, in the eye. Disease outcome is a result of a primary mutation, as well as modifier alleles. NR2E3 is a master regulator of several key pathways in retinal development and function. NR2E3 potentially prevents and rescues degenerating retina by resetting the homeostatic state of key gene networks in the presence of a primary mutation.
The delivery of Nr2e3 in a mouse lacking a functional Nr2e3 gene restored the retina structure and function. It was also demonstrated preclinically that RORA offers a protective allele in AMD, where the loss of photoreceptor cells leads to blindness. NR2E3 regulates the expression of both Nuclear Receptor Subfamily 1 Group D Member 1 (‘NR1D1’) and RORA. Thus, the nuclear receptors work in overlapping networks to modulate normal retinal development and function. These receptors impact gene expression of hundreds of genes and numerous networks and, as such, may be potent modifiers of retinal disease and degeneration.
OCU400 Phase 3 Clinical Study Overview
In April 2024, the FDA cleared the company’s IND amendment to initiate a Phase 3 liMeliGhT clinical trial of OCU400 for RP. The company is actively recruiting patients in the United States and Canada in the Phase 3 liMeliGhT clinical trial for OCU400 for the treatment of RP and is on track to complete enrollment in the first half of 2025. OCU400 is the first gene therapy program to enter Phase 3 with a broad RP indication. This Phase 3 trial will enroll 150 subjects, distributed 1:1 into two separate arms (RHO: N=75, and Gene Agnostic: N=75). In each arm, subjects will be further randomized into a 2:1 ratio to treated and untreated control groups. Subjects will be followed for a year after dosing for primary endpoint analyses. In the Phase 1/2 OCU400 clinical trial, a MLMT scale was the primary functional endpoint. For the Phase 3 OCU400 clinical trial, an updated mobility course will be used, Luminance Dependent Navigation Assessment (‘LDNA’), that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity. More than 60% of intent-to-treat (ITT) patients from the Phase 1/2 clinical trial (RHO and NR2E3) meet the responder criteria (Responder = 2 Lux level improvement) for Phase 3.
OCU400 Phase 1/2 Clinical Study Results
In January 2025, the company announced positive two-year long-term data across multiple mutations from the Phase 1/2 clinical trial of OCU400, which demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects at two years when compared to untreated eyes. 100% (10/10) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes. Also, treated eyes with multiple mutations and RHO subjects demonstrated a statistically significant (p=0.005) improvement in visual function when compared to untreated eyes. Treated eyes in RHO subjects (who meet Intent-to-treat criteria) for OCU400 Phase 3 trial demonstrated a statistically significant improvement (p=0.01, N=4) with around two-line gain in visual function (LLVA) at two years when compared to untreated eyes.
In the Phase 1/2 OCU400-101 study, a total of 22 subjects aged nine to 77 years, male and female, received OCU400 subretinal injection in three dose levels of up to 300 µL. All subjects had a confirmed molecular diagnosis of either biallelic autosomal recessive NR2E3 mutations, or autosomal dominant NR2E3 mutation, or autosomal dominant RHO mutations, or CEP290 mutation. In continuation of the preliminary analyses update, the company announced an update for 18 participants. The positive trial update demonstrated that OCU400 continued to be generally safe and well-tolerated in subjects across different mutations and dose levels. 89% of participants demonstrated preservation or improvement in the treated eye, either on BCVA or LLVA or MLMT scores from baseline. 78% of participants demonstrated preservation or improvement in the treated eyes in MLMT scores from baseline. 80% of RHO mutation subjects experienced either preservation or improvement in MLMT scores from baseline.
OCU410 and OCU410ST for the Treatment of Dry AMD and Stargardt Disease
The company is developing OCU410 and OCU410ST for the treatment of dAMD and Stargardt disease, respectively. OCU410 and OCU410ST utilize an AAV delivery platform for the retinal delivery of the RORA gene. RORA regulated gene networks are relevant in the treatment of dAMD and Stargardt disease. RORA reduces oxidative stress, limits lipofuscin deposits, reduces chronic inflammation, regulates complement activation, and improves choroidal blood flow. Gene variants of the ABCA4 gene are associated with both AMD and Stargardt disease. Stargardt disease is usually caused by mutations in the ABCA4 gene. This gene transports oxidized retinol compounds from photoreceptors to RPE cells for detoxification. In mouse models, ABCA4 -/- displayed low levels of CD59. A cell-surface glycoprotein, CD59, prevents the formation of the complement membrane attack complex. The company’s OCU410 and OCU410ST programs have completed the low, medium, and high dose cohorts in the Phase 1 part of the Phase 1/2 trials to date. OCU410 completed dosing in the Phase 2 part of the Phase 1/2 ArMaDa clinical trial. OCU410ST has completed Phase 1 of the Phase 1/2 GARDian clinical trial, and the DSMB has approved enrollment for the second phase of the trial.
In February 2025, the company announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a BLA submission. The Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-µL injection of OCU410ST at a concentration of 1.5 x 10^11 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity, as measured by best corrected visual acuity (BCVA), and LLVA compared to untreated controls. One-year data will be utilized for the BLA filing. In March 2025, the company announced that the EC has provided a positive opinion from the EMA Committee for Advanced Therapies for OCU410 and OCU410ST ATMP classification.
Novel Biologic Product Candidate for Retinal Diseases
The company is developing OCU200, which is a novel fusion protein containing parts of human transferrin and tumstatin. OCU200 is designed to treat DME, DR, and wet AMD. The company has completed the technology transfer of manufacturing processes to its CDMO and has produced trial materials to initiate a Phase 1 trial. The Phase 1 clinical trial will assess the unilateral intravitreal administration of OCU200 alone or in combination with an approved anti-VEGF therapy in participants with DME. This is a multicenter, open-label, dose-ranging trial with three cohorts in the dose-escalation portion of the trial. The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and the company is actively recruiting patients.
OCU200 for the Treatment of DR, DME, and Wet AMD
OCU200 is a novel fusion protein containing parts of human transferrin and tumstatin, which are already present normally in retinal tissues. Patients affected by these diseases share common symptoms, such as blurriness in vision and continued vision loss through disease progression. The formation of fragile and leaky new abnormal blood vessels leads to fluid accumulation in and around the retina, causing vision damage.
Tumstatin, which acts as an anti-angiogenic, anti-inflammatory, and anti-oxidative agent, is the active component of OCU200. It binds to integrin receptors, which play a crucial role in disease pathogenesis. Transferrin is expected to enhance the delivery of fused proteins across cellular barriers, including retinal barriers. OCU200 is designed to address the limitations of current therapies by targeting multiple mechanisms associated with ocular neovascularization and inflammation, specifically focusing on non-responders to currently available treatment options.
A proof-of-concept study involving different animal models demonstrated the therapeutic potential of OCU200 in the treatment of DR, DME, and Wet AMD. In an animal model for DME and DR (oxygen-induced retinopathy in mice), OCU200, at a significantly lower dose (10 micrograms per eye), was comparable to existing approved anti-VEGF therapy (Eylea, 20 micrograms per eye) in preventing disease manifestation and progression. Studies in animal models for Wet AMD (laser-induced choroidal neovascularization in mice and rats) suggest that OCU200 may possess comparable or slightly better activity compared to anti-VEGF control groups in preventing the formation and growth of new leaky blood vessels and subsequently disease symptoms.
NeoCart (Autologous Chondrocyte-Derived Neocartilage) Cell Therapy Platform
The company diversified its innovative pipeline in 2022 by introducing NeoCart (autologous chondrocyte-derived neocartilage), a Phase 3-ready, regenerative medicine cell therapy technology that combines breakthroughs in bioengineering and cell processing to enhance the autologous cartilage repair process. In May 2022, the FDA granted an RMAT designation to NeoCart for the repair of full-thickness lesions of knee cartilage injuries in adults.
NeoCart was acquired in the company’s reverse merger in 2019 with Histogenics Corporation (‘Histogenics’). Prior to 2019, Phase 1 and Phase 2 clinical trials were conducted to demonstrate the safety and efficacy of NeoCart. These clinical trials reported a decrease in pain and improved function of the knee. Additionally, per the results of the Phase 2 clinical trial, more patients responded to NeoCart than microfracture surgery. No SAEs were reported. A Phase 3 clinical trial was conducted to demonstrate the safety and effectiveness of NeoCart as compared to microfracture surgery to treat cartilage defects in the knee. This clinical trial enrolled 249 subjects between the ages of 18 and 59. The Phase 3 clinical trial of NeoCart narrowly missed the primary endpoint of a statistically significant improvement in pain and function in a dual threshold responder analysis one year after the treatment as compared to microfracture surgery.
The company has received concurrence from the FDA on the confirmatory Phase 3 clinical trial design. This study will be a randomized, controlled clinical trial designed to evaluate the efficacy and safety of NeoCart in comparison to the current standard of care, chondroplasty, in subjects with articular cartilage defects. The company intends to initiate the Phase 3 trial contingent on adequate availability of funding. The company’s Phase 3 clinical trial will use chondroplasty as the control instead of microfracture, which was used in the Phase 3 clinical trial conducted by Histogenics. Additionally, the Phase 3 clinical trial conducted by Histogenics used a responder analysis for the co-primary endpoint (as opposed to microfracture) that included an improvement of at least 12 points in outcome compared to baseline at one year on the knee injury and OA outcome score pain assessment test and an improvement of at least 20 points in outcome compared to baseline on the International Knee Documentation Committee subjective test. In contrast, its Phase 3 clinical trial will use a co-primary efficacy endpoint defined as the mean change from baseline (as opposed to chondroplasty) to two years for the patients' Knee Injury and Osteoarthritis (‘OA’) Outcome Score Pain and Function (Activities of Daily Living) subscale scales. Additionally, the Phase 3 clinical trial conducted by Histogenics enrolled patients with a total lesion size of less than six cm², while its Phase 3 clinical trial will enroll patients with total lesion sizes between one to three cm².
The cartilage is a complex tissue that protects the various joints and bones in the human body. It acts as a shock absorber throughout the body, withstanding significant pressure and allowing for joints to glide smoothly with minimal friction. Cartilage damage can be caused by acute trauma, such as a bad fall or a sports-related injury, or by repetitive trauma, such as general wear over time. Unlike other tissues in the body, cartilage in the joints has no innate ability to repair itself, making any injury permanent. If left untreated, even a small defect can expand in size and progress to debilitating OA, ultimately requiring a joint replacement procedure. Over 528 million individuals worldwide are diagnosed with OA. This number is expected to increase as the population of aging yet active individuals and the rates of obesity increase.
NeoCart is designed to treat pain at the source, improve function, and potentially prevent a patient's progression to OA. NeoCart is a three-dimensional tissue-engineered disc of new cartilage that is manufactured by growing chondrocytes, the cells responsible for maintaining cartilage health. The chondrocytes are derived from the patient on a unique scaffold. In this therapy, the patient's cells are separated from a tissue biopsy specimen and multiplied in a manufacturing facility. The cells are then infused into the scaffold, which is a three-dimensional structure that enables the proper delivery, distribution, and organization of cells in their natural environment to support tissue formation. Before NeoCart is implanted in a patient, the patient's cells and the scaffold undergo a bioengineering process in a Tissue Engineering Processor (‘TEP’). The TEP is designed to mimic the conditions inside a functional joint so that the tissue is prepared to begin functioning like normal healthy cartilage prior to implantation. Once NeoCart is ready to be implanted, a bioadhesive is used to anchor NeoCart at the site of cartilage injury and seal the implant to the surrounding native cartilage. The bioadhesive is a natural, biocompatible material that acts as adhesives for biological tissue, thereby eliminating the need for complicated suturing.
Inhaled Mucosal Vaccine Platform
The company is developing a next-generation, inhalation-based mucosal vaccine platform based on a novel ChAd vector, which includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine.
Novel Inhaled Mucosal Vaccine Platform for the Prevention of COVID-19 and the Seasonal Flu
The company is developing a next-generation, inhalation-based mucosal vaccine platform based on a novel ChAd vector, which includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine.
The company’s novel inhaled mucosal vaccine platform is specifically designed to generate local mucosal immunity in the nasopharyngeal region. The mucosal vaccination method has demonstrated potent induction of both mucosal and systemic immune responses, which prevents infection and spread, thereby limiting the origins of new variants. The advantages of these inhaled mucosal vaccines include needle-free administration, the potential for increased compliance, scalable manufacturing, storage and shipping at standard refrigerated conditions, and the potential to develop multi-strain and variant-specific versions.
Pursuant to the WU License Agreement, the company obtained the rights to develop, manufacture, and commercialize a mucosal COVID-19 vaccine in the Mucosal Vaccine Territory. In addition, the company internally developed technology related to the flu and COVID-19's vaccine design and filed intellectual property. In October 2023, OCU500 was selected by the NIAID Project NextGen for inclusion in clinical trials. OCU500 will be tested via two different mucosal routes, inhalation and intranasal delivery. NIAID intends to initiate a Phase 1 clinical trial in the second quarter of 2025. The company is continuing discussions with relevant government agencies, as well as strategic partners, regarding developmental funding for its OCU510 and OCU520 platforms.
Competition
Other competitors for the company’s novel inhaled mucosal vaccine platform include CyanVac LLC, Meissa Vaccines, Inc., Codagenix, Inc., Intravacc B.V., McMaster University, and Tetherex Pharmaceuticals Corporation.
Clinical Supply of the company’s Modifier Gene Therapy Platform
The company has a co-development and commercialization agreement with CanSinoBIO with respect to the development and commercialization of the company’s modifier gene therapy platform, including OCU400, OCU410, and OCU410ST. CanSinoBIO is responsible for the CMC development and manufacture of clinical supplies of such product candidates and is responsible for the costs associated with such activities. CanSinoBIO has an exclusive license to develop, manufacture, and commercialize its modifier gene therapy platform in and for China, Hong Kong, Macau, and Taiwan (the ‘CanSinoBIO Territory’), and the company maintains exclusive development, manufacturing, and commercialization rights with respect to its modifier gene therapy platform outside the CanSinoBIO Territory (the ‘Company Territory’).
The company partners with CanSinoBIO for the process development, manufacturing, testing, and release of drug product candidates for use in IND-enabling studies and clinical trials. The company performs discovery and analytical development activities in its research and development lab. The partnership with CanSinoBIO enables the company to complete manufacturing, with the release of clinical trial materials in an expedited manner, and helps in mitigating the risk of delay that can be associated when working with highly competitive CDMOs that have long wait times with regard to gene therapy manufacturing. Although the company relies on its partnership for manufacturing, it has personnel with extensive experience in gene therapy manufacturing to oversee and guide the process and analytical development, scale-up, release, and stability testing at its partner site. The company performs periodic audits of its manufacturing partner to confirm compliance with applicable regulations.
Clinical and Commercial Supply of NeoCart
The company has completed renovating an existing facility into a current GMP facility in accordance with the FDA's regulations in support of NeoCart manufacturing for Phase 3 clinical trial material.
Clinical Supply of Inhaled Mucosal Vaccine Platform
In October 2023, OCU500 was selected by the NIAID Project NextGen for inclusion in clinical trials. Project NextGen is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID, with funding from Project NextGen, will cover the full cost of the clinical trials, including operations and related analysis.
Clinical Supply of OCU200
In October 2020, the company entered into a manufacturing agreement with a CDMO for the manufacture of OCU200. Under the manufacturing agreement, the company’s CDMO will manage all CMC and clinical manufacturing activities for OCU200. The company has completed the technology transfer of manufacturing processes to its CDMO and has produced clinical trial materials to initiate the planned Phase 1 clinical trial. In April 2023, the FDA placed the company’s IND application to initiate a Phase 1 trial targeting DME on clinical hold, as part of the FDA's request for additional information related to CMC. The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and the company is actively recruiting patients.
License and Development Agreements
Modifier Gene Therapy Program
Exclusive License Agreement with SERI
In December 2017, the company entered into an exclusive license agreement with SERI, which was amended in January 2021 (as amended, the ‘SERI Agreement’). The SERI Agreement gives the company an exclusive, worldwide, sublicensable license to patent rights, biological materials, and technical information for NHR genes, such as NR1D1, NR2E3 (OCU400), RORA (OCU410 and OCU410ST), Nuclear Protein 1, Transcriptional Regulator (‘NUPR1’), and Nuclear Receptor Subfamily 2 Group C Member 1 (‘NR2C1’). The January 2021 amendment to the SERI Agreement additionally granted the company rights in co-owned intellectual property pursuant to certain patent applications and provisional patent applications at the time of the amendment. Under the SERI Agreement, the company may make, have made, use, offer to sell, and import licensed products, and must use commercially reasonable efforts to bring one or more licensed products to market as soon as reasonably practicable.
Co-Development and Commercialization Agreement with CanSinoBIO
The company entered into the CanSinoBIO Agreement with CanSinoBIO with respect to the development and commercialization of its modifier gene therapy product candidates, OCU400, OCU410, and OCU410ST. The CanSinoBIO Agreement was originally entered into in September 2019 with regards to OCU400 and was subsequently amended in September 2021 and November 2022 to include OCU410 and OCU410ST, respectively. Pursuant to the CanSinoBIO Agreement, the company is collaborating with CanSinoBIO on the development of its modifier gene therapy platform. CanSinoBIO is responsible for the CMC development and manufacture of clinical supplies of such product candidates and is responsible for the costs associated with such activities. CanSinoBIO has an exclusive license to develop, manufacture, and commercialize its modifier gene therapy platform in and for the CanSinoBIO Territory, and the company maintains exclusive development, manufacturing, and commercialization rights with respect to its modifier gene therapy platform in the Company Territory.
NeoCart
License Agreement with Purpose
In December 2005, Histogenics entered into an exclusive agreement (the ‘Purpose Agreement’) to sublicense certain technology from Purpose, which the company assumed as a result of its reverse merger with Histogenics. Purpose entered into the original license agreement (‘BWH-Purpose Agreement’) with Brigham and Women’s Hospital, Inc. (‘BWH’) in August 2001. The BWH-Purpose Agreement granted Purpose an exclusive, royalty-bearing, worldwide, sublicensable license, under its rights in licensed patents and patent applications co-owned by BWH.
Vaccines
Exclusive License Agreement with Washington University
In September 2022, the company entered into the WU License Agreement with Washington University, pursuant to which the company was granted an exclusive, sublicensable, royalty-bearing license to patent rights for a mucosal COVID-19 vaccine, as well as a license to certain tangible research property and technical information necessary to exploit the patent rights within the United States, Europe, and Japan. In January 2023, the company amended the WU License Agreement to add the countries of South Korea, Australia, and China to the Mucosal Vaccine Territory, and in November 2023, the company further amended the WU License Agreement to add Hong Kong to the Mucosal Vaccine Territory.
NIAID Project NextGen Clinical Trial Support
In October 2023, OCU500 was selected by the NIAID Project NextGen for inclusion in clinical trials. Project NextGen is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID, with funding from Project NextGen, will cover the full cost of the clinical trials, including operations and related analysis.
Novel Biologic Therapy for Retinal Diseases
Exclusive License Agreement with the University of Colorado
In March 2014, the company entered into an exclusive license agreement with CU, which was amended in January 2017 and clarified by a letter of understanding in November 2017 (as amended and clarified, the ‘CU Agreement’). The CU Agreement gives the company an exclusive, worldwide, sublicensable license to patents for OCU200 to make, have made, use, import, offer to sell, sell, have sold, and practice the licensed products in all therapeutic applications. Under the CU Agreement, the company must use commercially reasonable efforts to develop, manufacture, sublicense, market, and sell the licensed products and has assumed primary responsibility for preparing, filing, and prosecuting broad patent claims for OCU200 for CU's benefit. Further, the company has assumed primary responsibility for all patent activities, including all costs associated with the perfection and maintenance of the patents for OCU200.
Intellectual Property
As of February 24, 2025, the company’s patent portfolio for its product candidates included a total of 18 issued patents in the United States, 45 issued or registered patents in foreign countries, 10 pending patent applications in the United States, and 20 pending patent applications in foreign countries. The company’s issued or registered patents and pending patent applications include those licensed from SERI, Purpose, Washington University, and CU. Certain issued patents and pending patent applications cover multiple of its product candidates. The company’s intellectual property includes compositions of matter, methods of use, product candidates, and other proprietary technology. As of February 24, 2025, the company had exclusive rights or owned rights to: two issued United States patents, one pending United States patent application, and one pending foreign patent application related to OCU400; one pending United States patent application, and three pending foreign patent applications related to OCU410 and OCU410ST; 15 issued United States patents; six pending United States patent applications, 20 issued or registered foreign patents, and nine pending foreign patent applications related to NeoCart; two pending United States patent applications and seven pending foreign patent applications related to OCU500, OCU510, and OCU520; and one issued United States patent and 25 issued or registered foreign patents related to OCU200. The company’s current portfolio of issued patents in the United States and issued or registered patents in foreign countries related to its product candidates expire between 2025 and 2038.
Government Regulation and Product Approval
The company’s business activities, including but not limited to, research, marketing, sales, promotion, distribution, medical education, and other activities following product approval, are subject to laws and regulations by numerous federal and state regulatory and law enforcement authorities in the United States, in addition to the FDA, including potentially the Department of Justice, the Department of Health and Human Services and its various divisions, including the Centers for Medicare and Medicaid Services (‘CMS’) and the Health Resources and Services Administration, the Department of Veterans Affairs, the Department of Defense, and state and local governments. The company’s business activities must comply with numerous healthcare laws, including but not limited to, the federal civil FCA, the federal Anti-Kickback statute, and other laws and regulations, as well as data privacy and security laws and regulations, as well as state and federal consumer protection and unfair competition laws.
History
Ocugen, Inc. was founded in 2013.
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