RHO

MALVERN, Pa., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Clinical Showcase webcast from the February 21, 2024 live event is now available in the Events & Presentations section of the Ocugen website. The showcase replay includes more detail on the Phase 3 study design for OCU400, as well as market potential for this first-in-class product candidate. The panel discussion offers first-hand experience from an OCU400 Phase 1/2 clinical trial patient with the RHO mutation who has completed 12 months of therapy and lead investigator, Dr. Byron Lam, Professor of Ophthalmology, Mark J. Daily, Endowed Chair in Ophthalmology at the University of Miami. Dr. Lejla Vajzovic, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine, provides insight on the disease burden and unmet medical need associated with retinitis pigmentosa. Finally, the inventor of modifier gene therapy, Dr. Neena Haider, CEO & CSO Shifa Precision, faculty at Harvard Medical School, gives commentary throughout on the genesis of modifier gene therapy and how this treatment approach has evolved from the lab into the clinic. Please direct any questions related to the webcast to Tiffany Hamilton, Head of Communications, at Tiffany.Hamilton@ocugen.com. About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at and follow us on X and LinkedIn. Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact: Tiffany Hamilton Head of Communications IR@ocugen.com

Molecules that are induced by light to rotate bulky groups around central bonds could be developed into photo-activated bioactive systems, molecular switches, and more. Molecules that are induced by light to rotate bulky groups around central bonds could be developed into photo-activated bioactive systems, molecular switches, and more. Researchers at Hokkaido University, led by Assistant Professor Akira Katsuyama and Professor Satoshi Ichikawa at the Faculty of Pharmaceutical Sciences, have extended the toolkit of synthetic chemistry by making a new category of molecules that can be induced to undergo an internal rotation on interaction with light. Similar processes are believed to be important in some natural biological systems. Synthetic versions might be exploited to perform photochemical switching functions in molecular computing and sensing technologies, or in bioactive molecules including drugs. They report their findings in Nature Chemistry. "Achieving a system like ours has been a significant challenge in photochemistry," says Katsuyama. "The work makes an important contribution to an emerging field in molecular manipulation." Insights into the possibilities for light to significantly alter molecular conformations have come from examining some natural proteins. These include the rhodopsin molecules in the retina of the eye, which play a crucial role in converting light into the electrical signals that create our sense of vision in the brain. Details are emerging of how the absorption of light energy can induce a twisting rearrangement of part of the rhodopsin molecule, required for it to perform its biological function. "Mimicking this in synthetic systems might create molecular-level switches with a variety of potential applications," Katsuyama explains. A key innovation by the Hokkaido team was to achieve photo-induced (i.e., light-driven) rotation of molecular groups around a series of chemical bonds that incorporate a nitrogen atom together with other bonded carbon atoms. The rotational properties were enabled by adding molecular components that contained an atom from the 'chalcogen' group of elements in the periodic table, specifically sulfur or selenium, to a simple organic molecule: an amide compound. This brought a new level of control and versatility to synthetic photo-induced rotational systems. Some of the chemical groups that rotate around the central bonds were relatively large, based on rings of six bonded carbon atoms. This facilitated the large-scale molecular changes that might be required for practical use in molecular switching systems. In addition to demonstrating the photo-induced changes, the team also performed theoretical calculations that gave insights into the likely mechanisms by which the rearrangements proceeded. The team also explored the effects of temperature on the transformations. The combination of theoretical and experimental work should help guide future research towards exploring and controlling modifications to the systems already achieved. "Our next research priority is focused on the potential of our methods for making new bioactive molecules activated by light. These could be applied in biological research or possibly developed as drugs," Ichikawa concludes. Using light to activate the conformational changes allows control over where and when the changes occur. This could be vital for precisely targeted applications in biological systems, including eventual therapeutic possibilities.

SparingVision Reaches Final Dose Escalation Step in PRODYGY Trial with SPVN06 for retinitis pigmentosa Positive recommendation received from the Data Safety Monitoring Board (DSMB) enabling progression to highest dose of Part 1 of the PRODYGY trialFavorable safety and tolerability profiles observed with first two SPVN06 doses across six patients (three patients per cohort)Exploration of SPVN06 in geographic atrophy to begin in 2024 Paris, 24 January 2024 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, today announces a key advancement in its Phase I/II clinical trial (PRODYGY, NCT05748873) for SPVN06, the Company’s lead gene-agnostic investigational gene therapy for the treatment of retinitis pigmentosa (RP). The trial has now progressed to the final dose cohort of the dose-escalation phase (Part 1). This positive recommendation from the Data Safety Monitoring Board (DSMB) represents a further step towards the commencement of the controlled part of the study (Part 2) anticipated to start in Q2 2024, in line with study development timelines. SPVN06 is a breakthrough gene therapy approach aimed at stopping or slowing disease progression in patients affected by rod-cone dystrophy, (RCD), regardless of their genetic background. SparingVision is initially focusing on mid-stage RP, one of the leading inherited causes of blindness globally. This progress of the PRODYGY trial to the third cohort at the highest dose of SPVN06 follows an earlier positive recommendation from the DSMB to dose the second cohort at the medium dose in August 2023. The two positive recommendations received to date from the DSMB were based on the favorable safety and tolerability profiles observed in patients treated with the lower (first cohort) and medium (second cohort) doses of SPVN06. After completion of the third and final cohort in Part 1, the Company plans to advance the PRODYGY trial to Part 2, a three-arm controlled, double-masked, randomized extension phase investigating two doses of SPVN06 and one untreated arm, with additional clinical sites opening in the United States. The primary endpoint of the PRODYGY Phase I/II trial is expected to be reached in the second half of 2025, twelve months after the last patient in Part 2 is dosed. Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: “We are encouraged by the strong safety and tolerability profile of SPVN06 demonstrated so far. This milestone not only allows us to progress to the highest dose and, pending further DSMB positive recommendation, start Part 2 in Q2 2024; it also gives us the added confidence to expand the evaluation of SPVN06 to geographic atrophy (GA), another significant blinding condition. We are excited about the future as we continue to advance our portfolio of pioneering genomic medicines to save sight.” With the encouraging safety data gathered in PRODYGY so far and supported by a body of evidence and KOL insights, SparingVision believes in the strong potential of SPVN06 in other retinal diseases for which cone preservation is the landmark for stopping disease progression. The Company intends to explore the benefits of its gene therapy for the treatment of geographic atrophy (GA), with a view to potentially initiating Investigational New Drug (IND)-enabling studies for GA in 2024. PRODYGY trial PRODYGY (Promising ROd-cone DYstrophy Gene therapY) is a multicentric Phase I/II trial to assess the safety, tolerability as well as preliminary efficacy and quality-of-life following a single subretinal injection of SPVN06 in the worst-seeing eye of adult patients with RCD due to a mutation in the RHO, PDE6A, or PDE6B gene. The study aims to recruit a total of 33 patients in two parts: Part 1: open-label, dose-escalation phase including three cohorts of three patients with severe advanced RCD, to determine two recommended best-tolerated doses for Part Two.Part 2: controlled, double-masked, randomized extension phase including 24 patients with intermediate advanced RP, divided into three cohorts: six untreated subjects and 18 patients receiving one of the two recommended doses determined in Part One. Further information on the trial can be found on www.ClinicalTrials.gov (CT identifier: NCT05748873). **ENDS** Contacts: SparingVisionStéphane BoisselPresident and CEONathalie TrepoInvestor Relations nathalie.trepo@sparingvision.comICR ConsiliumAmber Fennell, Namrata Taak, Davide Salvi+44 (0)20 3709 5700sparingvision@consilium-comms.com NOTES TO EDITORS: About SparingVision SparingVision is a global ophthalmology leader bringing new hope to millions affected by retinal diseases, for which there are currently no viable treatments. The Company has assembled a suite of cutting-edge technologies from gene therapy to CRISPR, enabling it to deploy the right technology to the right disease and ensure the delivery of breakthrough treatments to millions of patients. Both of its products, SPVN06 and SPVN20 go beyond single gene correction therapies to deliver new gene-agnostic treatments for Retinitis Pigmentosa, a group of inherited retinal diseases which are the leading cause of blindness globally. The Company also has a strategic collaboration with Intellia Therapeutics (NASDAQ:NTLA) to develop novel genome editing-based treatments for ocular disease utilizing CRISPR-Cas9 technology. SparingVision is a spin-off from the Paris Vision Institute and backed by high-quality international investors including 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, Intellia Therapeutics, UPMC Enterprises, Jeito Capital and Ysios Capital.Visit www.sparingvision.com for more and follow us on LinkedIn and X (formerly Twitter) @SparingVision About SPVN06 SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor (Rod derived Cone Viability Factor, RdCVF) and one enzyme reducing oxidative stress (Rod derived Cone Viability Factor Long form, RdCVFL). Acting synergistically, RdCVF and RdCVFL aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVision’s primary disease targets are retinitis pigmentosa (RP), one of the most common inherited retinal diseases that affects an estimated two million patients worldwide and dry Age-related Macular Degeneration (AMD). There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases, where the loss of rods is known to be an early signal of the disease. SPVN06 is the result of decades of world-leading ophthalmology research by SparingVision founders José-Alain Sahel and Thierry Léveillard at the Paris Vision Institute