This section highlights FDA-related milestones and regulatory updates for drugs developed by Ocugen (OCGN).
Over the past two years, Ocugen has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
OCU200, OCU400, OCU410, OCU410ST, and OCU500. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
OCU200 - FDA Regulatory Timeline and Events
OCU200 is a drug developed by Ocugen for the following indication: Wet-AMD (Age-related Macular Degenerations) and diabetic macular edema (DME).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- OCU200
- Announced Date:
- March 18, 2025
- Indication:
- Wet-AMD (Age-related Macular Degenerations) and diabetic macular edema (DME)
Announcement
Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial recently convened and reviewed safety data following dosing of the first cohort in the dose-escalation portion of the Phase 1 study and approved continuation of dosing in the second cohort.
AI Summary
Ocugen, Inc. announced a positive update on the Phase 1 OCU200 clinical trial. The Data and Safety Monitoring Board (DSMB) recently met to review safety data after the first cohort was dosed during the dose-escalation phase. Based on the review, the DSMB approved proceeding to dose the second cohort. This decision indicates that the OCU200 treatment, which is administered through intravitreal injection, has a favorable safety and tolerability profile with no serious adverse events reported so far.
OCU200 is a novel fusion protein designed to treat diabetic macular edema, and it may benefit patients with other retinal diseases as well. The trial is being carefully conducted in different dosing groups, with patients receiving two doses six weeks apart and followed for up to six months. This milestone supports the continued development of innovative therapies for vision-threatening conditions.
Read Announcement- Drug:
- OCU200
- Announced Date:
- January 16, 2025
- Indication:
- Wet-AMD (Age-related Macular Degenerations) and diabetic macular edema (DME)
Announcement
Ocugen, Inc announced that the first patient has been dosed in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME).
AI Summary
Ocugen, Inc. has reached an important milestone by dosing the first patient in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME). The trial is a multicenter, open-label study designed to test the safety of OCU200, a drug that works differently from current treatments. It uses a unique approach by combining tumstatin and transferrin to target the retina and choroid, which may help patients who do not respond to standard anti-VEGF therapies. In this study, patients receive two intravitreal injections six weeks apart at low, medium, or high doses to assess the best amount for future use. The initial steps of this trial are key to understanding how effectively OCU200 can reduce inflammation, vascular permeability, and other issues that lead to vision loss in DME and related diseases.
Read Announcement- Drug:
- OCU200
- Announced Date:
- October 9, 2024
- Indication:
- Wet-AMD (Age-related Macular Degenerations) and diabetic macular edema (DME)
Announcement
Ocugen, Inc announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the investigational new drug application for the Phase 1 clinical trial evaluating OCU200, a recombinant fusion protein consisting of tumstatin and transferrin, for treating diabetic macular edema (DME).
AI Summary
Ocugen, Inc. announced that the FDA has lifted the clinical hold on the investigational new drug application for its Phase 1 clinical trial evaluating OCU200. This trial will test OCU200, a recombinant fusion protein made of tumstatin and transferrin, aimed at treating diabetic macular edema (DME). OCU200 works by using tumstatin to target the underlying disease mechanisms through integrin receptors, while transferrin assists in delivering tumstatin to the retina and choroid. The company hopes that this new approach will benefit DME patients, including the 30 to 40% who do not respond to current anti-VEGF therapies. With nearly 746,000 people in the U.S. affected by DME, this study is an important step toward finding new treatment options for those at risk of vision loss.
The trial will focus on establishing the safety and optimal dosing levels of OCU200, potentially paving the way for additional developments in treating other diabetic eye diseases.
Read Announcement
OCU400 (AAV5-hNR2E3) - FDA Regulatory Timeline and Events
OCU400 (AAV5-hNR2E3) is a drug developed by Ocugen for the following indication: Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- OCU400 (AAV5-hNR2E3)
- Announced Date:
- February 3, 2025
- Indication:
- Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
Announcement
Ocugen, Inc announced that the European Commission has provided a positive opinion from the European Medicines Agency's (EMA) Committee for Advanced Therapies (CAT) for OCU400 Advanced Therapy Medicinal Product (ATMP) classification.
AI Summary
Ocugen, Inc. announced a significant development as the European Commission, through the EMA’s Committee for Advanced Therapies (CAT), provided a positive opinion for classifying OCU400 as an Advanced Therapy Medicinal Product (ATMP). This designation marks an important milestone for the company’s first gene therapy aimed at treating retinitis pigmentosa (RP) and supports its ongoing Phase 3 clinical trial. The ATMP classification can accelerate the regulatory review timeline and allows Ocugen to seek more frequent scientific advice and protocol assistance from the EMA. This clear regulatory pathway is expected to keep the company's clinical and commercial strategies on track, targeting market availability in both Europe and the United States by 2027. The positive opinion reinforces confidence in the therapy’s potential, which could offer a one-time gene treatment for RP patients suffering from multiple gene mutations.
Read Announcement- Drug:
- OCU400 (AAV5-hNR2E3)
- Announced Date:
- January 13, 2025
- Indication:
- Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
Announcement
Ocugen, Inc. announced a positive 2-year safety and efficacy update for the Phase 1/2 OCU400 clinical trial.
AI Summary
Ocugen, Inc. announced positive two-year safety and efficacy results for its Phase 1/2 OCU400 clinical trial, aimed at treating retinitis pigmentosa. The update shows that 100% (9/9) of treated evaluable subjects exhibited improvement or preservation in visual function compared to untreated eyes, with statistically significant improvements (p=0.01) regardless of the specific mutation. In addition to visual acuity gains—measured by a 2-line improvement in low-luminance visual acuity—patients also demonstrated stabilization or improvement in mobility during the testing period. The trial reported a favorable long-term safety profile with no serious adverse events related to OCU400. These findings underline the potential of OCU400 as a mutation-agnostic one-time therapy for a condition that could lead to legal blindness, marking a promising step forward in the treatment of retinal disorders.
Read Announcement- Drug:
- OCU400 (AAV5-hNR2E3)
- Announced Date:
- August 5, 2024
- Indication:
- Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
Announcement
Ocugen, Inc. announced that it has received notification from FDA to begin its expanded access program (EAP) for the treatment of adult patients, aged 18 and older, with retinitis pigmentosa (RP) with OCU400—a modifier gene therapy product candidate.
AI Summary
Ocugen, Inc. has received notification from the FDA to begin its expanded access program (EAP) for treating adult patients with retinitis pigmentosa (RP) using its modifier gene therapy candidate, OCU400. The EAP is designed for patients aged 18 and older who have early, intermediate, or advanced RP with at least minimal retinal preservation. This program gives eligible patients access to OCU400 outside of clinical trials, potentially offering a one-time treatment that could improve vision by resetting dysfunctional gene networks in the retina.
The initiative underscores Ocugen’s commitment to the RP community, aiming to serve the 300,000 patients in the U.S. and Europe—and 1.6 million patients globally—who currently have few therapeutic options. With ongoing clinical trials complementing the EAP, Ocugen seeks to expand enrollment to patients with a diverse range of RP gene mutations as it works toward a biologics license application approval.
Read Announcement- Drug:
- OCU400 (AAV5-hNR2E3)
- Announced Date:
- June 20, 2024
- Indication:
- Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
Announcement
Ocugen, Inc announced that the first patient has been dosed in its Phase 3 liMeliGhT clinical trial for OCU400—a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).
AI Summary
Ocugen, Inc. recently reached an important milestone in its pursuit to treat retinitis pigmentosa (RP) by dosing the first patient in its Phase 3 liMeliGhT clinical trial for OCU400, a modifier gene therapy product candidate. This trial builds on promising results from earlier Phase 1/2 studies that showed improvements in vision-related outcomes, such as Best-Corrected Visual Acuity and other mobility tests. The study, set to last one year, will enroll 150 participants, including individuals both with and without specific gene mutations, to assess the treatment’s impact on preserving or improving vision. Dosing the first patient underlines the company’s commitment to delivering a one-time, long-lasting treatment option for the estimated 1.6 million people affected by RP worldwide. The progress of this trial brings hope for a new therapeutic approach to managing RP and improving patients’ quality of life.
Read Announcement- Drug:
- OCU400 (AAV5-hNR2E3)
- Announced Date:
- April 12, 2024
- Indication:
- Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
Announcement
Ocugen, Inc announced that Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder will participate in a fireside chat during the Emerging Growth Virtual Healthcare Equity Conference, presented by Noble Capital Markets, taking place from April 17 – 18, 2024.
AI Summary
Ocugen, Inc. announced that Dr. Shankar Musunuri, the company’s Chairman, CEO, and Co-Founder, will participate in a fireside chat during the Emerging Growth Virtual Healthcare Equity Conference. The event is hosted by Noble Capital Markets and will take place from April 17 to 18, 2024. Dr. Musunuri’s session is scheduled for Wednesday, April 17, 2024 at 10 a.m. ET on Presentation Track 1, and it will be available as a live webcast through Ocugen’s website.
During the chat and one-on-one sessions, Dr. Musunuri will highlight the company’s recent progress in business and clinical development, including FDA clearance to start a Phase 3 clinical trial for its gene therapy candidate, OCU400, which aims to treat retinitis pigmentosa. The webcast will be archived on the company’s website for 90 days after the event.
Read Announcement- Drug:
- OCU400 (AAV5-hNR2E3)
- Announced Date:
- April 10, 2024
- Indication:
- Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
Announcement
Ocugen, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA).
AI Summary
Ocugen, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has reviewed the study design, endpoints, and planned statistical analysis for its pivotal OCU400 Phase 3 liMeliGhT clinical trial. This review focused on the trial’s ability to support a Marketing Authorization Application (MAA) in the European Union. The decision was based on the safety and tolerability data from the earlier Phase 1/2 study of OCU400, a gene therapy aimed at treating retinitis pigmentosa (RP). The Phase 3 trial will enroll 150 participants in the U.S., dividing them into two groups: one with 75 participants carrying RHO gene mutations and another with 75 participants representing various gene mutations related to RP. The favorable opinion from EMA could potentially reduce both the time and cost necessary for marketing authorization in the EU.
Read Announcement- Drug:
- OCU400 (AAV5-hNR2E3)
- Announced Date:
- April 8, 2024
- Indication:
- Retinitis pigmentosa (RP) and Leber Congenital amaurosis (LCA).
Announcement
Ocugen, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) amendment to initiate a Phase 3 clinical trial of OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).
AI Summary
Ocugen, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) amendment, allowing the company to start a Phase 3 clinical trial for OCU400, a modifier gene therapy candidate for retinitis pigmentosa (RP). This milestone represents a significant step forward for patients with RP, as OCU400 is the first gene therapy to enter Phase 3 trials targeting a broad RP indication. The study will include 150 participants split into two arms: one for patients with the RHO gene mutation and another that is gene agnostic, ensuring a diverse group of patients have the opportunity to benefit from the therapy.
The trial will use a newly developed mobility course called the Luminance Dependent Navigation Assessment (LDNA), which is designed in collaboration with the FDA to measure patient improvement across a range of light intensities. This innovative approach aims to show a meaningful improvement in vision, offering new hope for patients with RP who currently have limited treatment options.
Read Announcement
OCU410 - FDA Regulatory Timeline and Events
OCU410 is a drug developed by Ocugen for the following indication: Dry Age-related Macular Degeneration (Dry AMD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- OCU410
- Announced Date:
- March 3, 2025
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc. announced that the European Commission has provided a positive opinion from the European Medicines Agency's (EMA) Committee for Advanced Therapies (CAT) for OCU410 and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification.
AI Summary
Ocugen, Inc. announced a significant regulatory milestone as the European Commission issued a positive opinion from the EMA’s Committee for Advanced Therapies (CAT) for its gene therapy candidates, OCU410 and OCU410ST. The ATMP classification supports fast-tracking these innovative treatments, which target unmet needs in vision loss disorders. OCU410 is designed for geographic atrophy in dry age-related macular degeneration, while OCU410ST targets Stargardt disease caused by ABCA4-related retinopathies. This decision permits more frequent interactions with the EMA for scientific advice and protocol assistance, paving the way for future clinical trials and potential marketing approvals. The classification reinforces the promise of these one-time gene therapies to improve patient outcomes.Read Announcement
- Drug:
- OCU410
- Announced Date:
- February 12, 2025
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc. announced that dosing is complete, ahead of schedule in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410—a novel multifunctional modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD).
AI Summary
Ocugen, Inc. announced that dosing is complete ahead of schedule in the Phase 2 portion of its Phase 1/2 ArMaDa clinical trial for OCU410, a novel multifunctional modifier gene therapy candidate for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). The trial, conducted at top retinal surgery centers across the United States, enrolled 51 subjects who were randomized into treatment and control arms. Early study results from Phase 1/2 showed a favorable safety and tolerability profile, with no serious adverse events related to OCU410. Initial data also indicated promising improvements, such as slower lesion growth and enhanced visual function. Designed as a one-time treatment, OCU410 has the potential to reduce the burden of frequent injections required by current therapies. These encouraging outcomes support plans for a future pivotal Phase 3 trial in 2026, aiming to advance the commercial strategy and regulatory filings by 2028.
Read Announcement- Drug:
- OCU410
- Announced Date:
- December 19, 2024
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study.
AI Summary
Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for its OCU410 ArMaDa clinical trial recently convened and approved moving forward with the second phase of the Phase 1/2 study. The DSMB carefully reviewed safety data from 15 subjects in Phase 2, finding that OCU410 has a favorable safety and tolerability profile. Importantly, no serious adverse events related to the study drug have been reported, which supports further testing.
OCU410, a novel gene therapy for geographic atrophy secondary to dry age-related macular degeneration, is administered via a single unilateral subretinal injection. This positive DSMB decision paves the way for additional evaluation of the therapy’s safety and efficacy, underscoring its potential to offer a one-time treatment alternative to the frequent injections required by current therapies.
Read Announcement- Drug:
- OCU410
- Announced Date:
- December 19, 2024
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study.
AI Summary
Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently met and approved the continuation of Phase 2 in its Phase 1/2 study. The board reviewed safety data from 15 subjects and found that OCU410, an AAV5-hRORA gene therapy candidate, appears to be safe and well tolerated, with no serious adverse events reported. The study focuses on treating geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD), a major cause of vision loss. In Phase 2, 45 subjects will be randomized into different dosage groups or an untreated control group to assess not only safety but also efficacy.
This positive decision by the DSMB supports further investigation of OCU410 as a potential one-time treatment, which may reduce the need for regular injections currently required by existing therapies.
Read Announcement- Drug:
- OCU410
- Announced Date:
- November 19, 2024
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD).
AI Summary
Ocugen, Inc. announced positive preliminary results from the Phase 1 dose-escalation portion of its Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), a condition secondary to dry age-related macular degeneration. In this trial, the company studied nine patients across three dose groups and found no drug-related serious adverse events. Treated eyes showed slower lesion growth, preservation of retinal tissue, and a notable improvement in low luminance visual acuity—an important measure of visual function. These findings suggest that OCU410 may not only slow disease progression but also improve vision, offering a potential one-time treatment option that addresses more than one aspect of the disease.
The results are encouraging compared to existing anti-complement therapies, which require repeated injections and do not improve visual function significantly. The positive safety and efficacy data support further development of OCU410 as a novel treatment option for patients with GA.
Read Announcement- Drug:
- OCU410
- Announced Date:
- July 25, 2024
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc announced that the company has Initiated Phase 2 clinical trial
AI Summary
Ocugen, Inc., a biotechnology company dedicated to gene and cell therapies, recently announced the initiation of a Phase 2 clinical trial for its promising modifier gene therapy, OCU410. This trial aims to assess both the safety and efficacy of OCU410 in a broader patient population suffering from geographic atrophy, an advanced form of dry age-related macular degeneration. Researchers will randomize patients into one of three groups: two treatment groups receiving either a medium or high dose, and a control group. The strategy of using a single treatment dose could offer a simpler and potentially more effective alternative to current therapies that require multiple injections over time. The Phase 2 trial follows positive early-phase results, and Ocugen plans to continue sharing clinical updates as it works to advance this innovative approach for reducing vision loss in patients with geographic atrophy.
Read Announcement- Drug:
- OCU410
- Announced Date:
- July 25, 2024
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc. announced that dosing is complete in the third cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a novel modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 1 million people in the United States.
AI Summary
Ocugen, Inc. announced that dosing is complete in the third cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA), a promising modifier gene therapy candidate for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects about one million people in the United States. In this part of the trial, three subjects received a single subretinal injection of the highest dose being tested. The study is being conducted at 14 leading retinal surgery centers across the country and is designed to assess safety and proper dosing levels.
The completion of dosing in this cohort is an important milestone for evaluating OCU410, which has the potential to offer a one-time treatment option for GA. Future trial phases will further explore the safety and effectiveness of this gene therapy approach as a transformative option for managing GA.
Read Announcement- Drug:
- OCU410
- Announced Date:
- April 19, 2024
- Indication:
- Dry Age-related Macular Degeneration (Dry AMD)
Announcement
Ocugen, Inc announced that dosing is complete in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD).
AI Summary
Ocugen, Inc. announced that dosing is complete in the second cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA), a modifier gene therapy candidate aimed at treating geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dAMD). In this trial, 3 subjects received a single subretinal injection of the medium dose (5x10¹⁰ vg/mL) of OCU410. The therapy is designed as a one-time treatment that targets multiple disease pathways, including lipid metabolism, inflammation, oxidative stress, and the complement system. Up to 13 leading retinal surgery centers across the United States are involved in conducting the trial, which seeks to demonstrate not only the safety but also the potential for transforming GA treatment compared to current therapies that require repeated injections over many years.
Read Announcement
OCU410ST - FDA Regulatory Timeline and Events
OCU410ST is a drug developed by Ocugen for the following indication: For Stargardt Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- OCU410ST
- Announced Date:
- June 16, 2025
- Indication:
- For Stargardt Disease
Announcement
Ocugen, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies).
AI Summary
Ocugen, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment, allowing the company to begin a Phase 2/3 pivotal confirmatory trial of its modifier gene therapy candidate, OCU410ST. This trial will focus on treating all forms of Stargardt disease, a condition linked to ABCA4-associated retinopathies. The study will enroll 51 participants, with 34 receiving a one-time subretinal injection of OCU410ST and 17 serving as controls. The primary goal is to evaluate the therapy's ability to reduce the size of atrophic lesions, while secondary measures will assess improvements in visual acuity. The FDA clearance paves the way for a more rapid clinical development process, potentially making the treatment available to patients sooner and addressing an important unmet need for those with Stargardt disease.
Read Announcement- Drug:
- OCU410ST
- Announced Date:
- May 27, 2025
- Indication:
- For Stargardt Disease
Announcement
Ocugen, Inc. announced that the United States Food and Drug Administration (U.S. FDA) has granted Rare Pediatric Disease Designation (RPDD) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.
AI Summary
Ocugen, Inc. announced that the U.S. FDA has granted Rare Pediatric Disease Designation (RPDD) for its gene therapy candidate, OCU410ST. This designation is aimed at treating ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.
The RPDD is given for serious and life-threatening diseases that mainly affect children under 18 years old and have a limited number of affected individuals in the U.S. This recognition highlights the urgent need for treatments for these inherited retinal diseases, which often begin in childhood and affect not only the patients but also their families.
This milestone supports Ocugen’s commitment to developing new therapies for rare pediatric conditions and may help accelerate the clinical development of OCU410ST, offering hope to those impacted by these vision conditions.
Read Announcement- Drug:
- OCU410ST
- Announced Date:
- March 5, 2025
- Indication:
- For Stargardt Disease
Announcement
Ocugen, Inc. announced general business update.
AI Summary
Ocugen, Inc. announced a general business update highlighting an important milestone in its effort to address Stargardt disease. The company has achieved alignment with the FDA to launch a Phase 2/3 pivotal confirmatory clinical trial for its gene therapy candidate, OCU410ST. This trial, focused on a condition that affects around 100,000 Stargardt patients in the U.S. and Europe who currently have no treatment options, is expected to accelerate clinical development by two to three years. In the study, 51 subjects will be enrolled, with 34 receiving a single subretinal injection and 17 serving as untreated controls. Early data from prior trials showed promising results, including slowed lesion growth and meaningful improvements in visual function. Ocugen’s update reflects its strategic drive to fast-track new therapies and further its mission to deliver innovative treatments for blindness-related diseases.
Read Announcement- Drug:
- OCU410ST
- Announced Date:
- February 27, 2025
- Indication:
- For Stargardt Disease
Announcement
Ocugen, Inc. announced that alignment has been reached with the U.S. Food and Drug Administration (FDA) to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a biologics license application (BLA) submission.
AI Summary
Ocugen, Inc. announced that it has reached an agreement with the U.S. Food and Drug Administration (FDA) to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST. This important trial is designed to confirm the therapy’s benefits and safety, and if the results are positive, they could support a future biologics license application (BLA) submission. The planned trial will evaluate the effect of a one-time, subretinal injection in patients with Stargardt disease, a condition that has no current treatment. The study will compare treated patients with a control group by measuring changes in atrophic lesion size and visual acuity improvements. Ocugen’s advancement to this trial phase highlights the potential of OCU410ST to address a critical unmet need in patients suffering from Stargardt disease and promises new hope for those affected by this vision-impairing condition.
Read Announcement- Drug:
- OCU410ST
- Announced Date:
- November 20, 2024
- Indication:
- For Stargardt Disease
Announcement
- Ocugen, Inc. announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3).
AI Summary
Ocugen, Inc. announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for its gene therapy candidate, OCU410ST. This designation is for the treatment of rare ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). The orphan designation acknowledges that these conditions affect fewer than 5 in 10,000 patients in the European Union and provides incentives such as reduced regulatory fees, protocol assistance, research grants, and up to 10 years of market exclusivity after approval.
Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen, expressed that receiving this designation is an honor and a significant step towards offering a much-needed treatment option for patients battling these challenging retinal diseases. This progress underscores Ocugen’s commitment to advancing innovative therapies for rare conditions.
Read Announcement- Drug:
- OCU410ST
- Announced Date:
- August 28, 2024
- Indication:
- For Stargardt Disease
Announcement
Ocugen, Inc announced that dosing is complete in the third cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease affects approximately 100,000 people in the United States (U.S.) and Europe.
AI Summary
Ocugen, Inc. announced that it has completed dosing in the third cohort (high dose) of its Phase 1/2 GARDian clinical trial for its gene therapy candidate OCU410ST (AAV-hRORA) intended for treating Stargardt disease. This phase of the trial involved three patients who received a single subretinal injection of the highest tested dose (2.25×10^11 vg/mL). Stargardt disease affects around 100,000 people in the United States and Europe, and currently, there are no FDA-approved treatments for the condition.
The study is being conducted at six leading retinal surgery centers across the U.S., and the high dose cohort marks an important milestone in evaluating both the safety and potential efficacy of OCU410ST. Ocugen remains committed to advancing innovative gene therapy solutions to address unmet needs for patients suffering from this genetic eye disorder.
Read Announcement- Drug:
- OCU410ST
- Announced Date:
- June 21, 2024
- Indication:
- For Stargardt Disease
Announcement
Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial recently convened and approved to proceed with dosing the high dose of OCU410ST in the dose-escalation phase of the study.
AI Summary
Ocugen, Inc. announced a significant advancement in their OCU410ST GARDian clinical trial for Stargardt disease. The Data and Safety Monitoring Board (DSMB) recently met and approved moving forward with dosing the high dose of OCU410ST during the dose-escalation phase. This decision comes after the medium dose was established as a safe and tolerable option, supporting the progress of this gene therapy candidate.
The DSMB’s recommendation signals an important step towards a potential one-time treatment for patients with Stargardt disease, an inherited retinal disorder that currently lacks FDA-approved therapies. With six patients already dosed in the low and medium cohorts, the move to include the high dose is seen as a promising development. Ocugen is optimistic that this phase in the trial will further demonstrate the safety and potential long-term benefits of OCU410ST.
Read Announcement- Drug:
- OCU410ST
- Announced Date:
- May 15, 2024
- Indication:
- For Stargardt Disease
Announcement
Ocugen, Inc announced that dosing is complete in the second cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease as a one-time treatment for life.
AI Summary
Ocugen, Inc. announced a key milestone in its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA), a promising modifier gene therapy candidate for Stargardt disease. The dosing for the second cohort has now been completed, marking an important step in the study of this one-time, lifetime treatment. In this gene therapy approach, researchers are focusing on addressing the underlying causes related to Stargardt disease, which affects vision. The trial is designed to assess the safety, tolerability, and potential effectiveness of OCU410ST. Completing dosing in the second group suggests progress in understanding how the treatment works and its possible benefits for patients with this condition.
Results from the trial will help determine if this gene therapy can slow or prevent vision loss, offering hope to those affected by Stargardt disease.
Read Announcement
OCU500 - FDA Regulatory Timeline and Events
OCU500 is a drug developed by Ocugen for the following indication: OCU500 is based on a novel chimpanzee adenovirus-vectored (ChAd) technology. Earlier clinical studies to prevent COVID-19 employing a similar vector administered vi.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- OCU500
- Announced Date:
- January 27, 2025
- Indication:
- OCU500 is based on a novel chimpanzee adenovirus-vectored (ChAd) technology. Earlier clinical studies to prevent COVID-19 employing a similar vector administered vi
Announcement
Ocugen, Inc. announced that the U.S. Food and Drug Administration (FDA) has reviewed the Company's Investigational New Drug (IND) application and it is in effect.
AI Summary
Ocugen, Inc. announced a significant milestone in its COVID-19 vaccine development. The U.S. Food and Drug Administration (FDA) has reviewed the company’s Investigational New Drug (IND) application and confirmed it is in effect. This decision clears the path for the next steps in testing their new vaccine candidate, OCU500, which is designed to be inhaled into the lungs or delivered as a nasal spray.
The IND approval means that Ocugen can now move forward with a Phase 1 clinical trial to evaluate the vaccine’s safety and its ability to trigger an immune response. The trial, scheduled to begin in the second quarter of 2025 and sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), marks an important step in developing a potentially improved method to help prevent COVID-19 infections and reduce transmission.
Read Announcement- Drug:
- OCU500
- Announced Date:
- January 27, 2025
- Estimated Event Date Range:
- April 1, 2025 - June 30, 2025
- Target Action Date:
- Q2 2025
- Indication:
- OCU500 is based on a novel chimpanzee adenovirus-vectored (ChAd) technology. Earlier clinical studies to prevent COVID-19 employing a similar vector administered vi
Announcement
Ocugen, Inc. announced that Phase 1 clinical trial is anticipated to start in 2Q 2025
AI Summary
Ocugen, Inc. announced that its Phase 1 clinical trial for OCU500, a novel COVID-19 vaccine, is expected to start in the second quarter of 2025. The vaccine, designed to be delivered as both an inhaled solution and a nasal spray, aims to target the virus at its point of entry in the body. This trial marks an important step forward, as the U.S. Food and Drug Administration has approved the company’s Investigational New Drug application, allowing the study to move ahead.
Sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), the study will include 80 adult participants split into two groups with different dosages and delivery methods. The trial will primarily focus on assessing the vaccine’s safety, while also evaluating the body’s immune response. Ocugen hopes that this innovative approach will offer a safer and more effective option for preventing COVID-19 infections and easing its impact on public health.
Read Announcement
