ABCB5

Very low risk for drug-drug interactions (DDI) with Mazindol ER based upon results from in vitro metabolism and transporter studies Demonstrated pharmacological differentiation versus currently available medications used to treat narcolepsy which can have significant DDI effects preventing co-administration or requiring dose adjustments ZURICH, SWITZERLAND / ACCESSWIRE / June 14, 2023 / NLS Pharmaceutics Ltd. (Nasdaq:NLSP, NLSPW) ("NLS" or the "Company"), a Swiss clinical-stage biopharmaceutical company focused on the discovery and development of innovative therapies for patients with rare and complex central nervous system disorders, today announced positive data from five recently completed in vitro drug-drug interaction studies investigating the potential for DDI of mazindol, a triple monoamine reuptake inhibitor and partial orexin-2 agonist, as well as its hydrolyzed metabolite (M6). Typical DDI studies include Cytochrome P450 (CYP), UDP glucuronosyltransferase (UGT) and transporter inhibition and induction studies to evaluate whether mazindol affects the pharmacokinetic (PK) effect of other drugs (mazindol as a "perpetrator"). In addition, in vitro studies are conducted with CYP and transporter substrates to evaluate whether other drugs can affect mazindol PK (mazindol as a "victim"). Following U.S. Food and Drug Administration (FDA) guidelines, the in vitro DDI studies were designed to evaluate the drug interaction potential across a wide range of metabolic enzymes and transporters when co-administered with mazindol. The mazindol in vitro studies examined the following: CYP Inhibition in Human Liver Microsomes CYP Induction in Cultured Human Hepatocytes CYP and UGT Reaction Phenotyping UGT Inhibition in Human Liver Microsomes ATP-binding case (ABC) and solute carrier (SLC) Transporter Inhibition and Substrate Potential in Cells and Vesicles Results of the in vitro perpetrator studies showed that mazindol and its hydrolysis metabolite do not have the potential to inhibit or induce the metabolism/transport of concomitant medications. Based on these in vitro data, it can be concluded that mazindol and the hydrolysis metabolite are not inhibitors of CYPs, UGTs, or transporters (including P-glycoprotein, breast cancer resistance protein [BCRP], organic anion transporter [OAT]1, OAT3 and organic cation transporter [OCT]2 and mazindol does not induce metabolism. Therefore, mazindol does not affect the PK of concomitant medications. Results of the in vitro victim studies showed that mazindol is not metabolized by CYPs or UGTs and is not transported by BCRP, OAT1, OAT3 and OCT2. Only the hydrolysis metabolite (but not mazindol) is a P-glycoprotein (Pgp) substrate; thus a clinical DDI study of mazindol is planned to quantify the potential for an increase in the hydrolyzed metabolite (M6) exposure in the presence of a Pgp inhibitor (e.g., Itraconazole). Otherwise, concomitant medications are not expected to affect the PK of mazindol. "We are pleased to have seen these positive results from our DDI in vitro study with mazindol, which further demonstrated its differentiated pharmacological and safety profile. This latest set of data reinforce that mazindol may have the potential to provide an advantage over other current treatments for patients with narcolepsy taking multiple drugs, including hormonal contraceptives, proton-pump inhibitors and anti-epileptic drugs, who are at higher risk of experiencing adverse drug interactions or even discontinuation of their medications due to those interactions," said George Apostol, MD, MS, Chief Medical Officer of NLS. "Based on these data, once-daily Mazindol ER shows potential to meet yet another unmet need of narcolepsy patients, in addition to its strong efficacy, fast onset of action, well demonstrated long-term safety profile, low scheduling and its unique mechanism of actions." Alex Zwyer, Chief Executive Officer added, "Discussions with both physicians as well as patient advocacy groups at SLEEP 2023, the annual meeting of the American Academy of Sleep Medicine (AASM) and the Sleep Research Society (SRS), held on June 3 - 7, 2023, in Indianapolis, reinforced the importance a mazindol's DDI drug pro realize an unmet need for patients." Beginning in July 2023, NLS will initiate the AMAZE development program for Mazindol ER, consisting of two pivotal Phase 3, randomized, double-blind, placebo-controlled, multicenter studies in the U.S. designed to assess the safety and efficacy of mazindol in patients with narcolepsy type 1 (NT1) compared to placebo. These two studies will enroll a total of 100 NT1 patients. Mazindol ER will be administered at fixed doses of 3 mg once daily for a total of eight weeks. A 52-week open-label extension will further evaluate the long-term safety and tolerability of Mazindol ER in NT1 patients. Topline data from the double-blind studies are expected in late 2024. About DDI in vitro studies DDI studies are a standard and necessary step required by the FDA and other regulatory agencies globally for approving a new drug to market. DDI studies help identify potential adverse reactions that may be caused by interactions between multiple drugs, leading to unintended reactions, toxic side effects, or in some cases, a lack of therapeutic efficacy. With the rise in polypharmacy to treat comorbidities, alongside prevalent substance abuse, drug-drug interactions have become a critical factor to consider when treating narcolepsy. Approximately 50% of prescribed drugs currently on the market are known to cause drug interactions with CYP3A4 inhibitors and can lead to side effects. Findings from DDI studies help to inform drug labeling that is then used by healthcare providers to aid in therapeutic decision-making. Currently available medications used to treat narcolepsy can have significant DDI effects which prevent co-administration or require dose adjustments of drugs like hormonal contraceptives, proton-pump inhibitors or anti-epileptic drugs. About Mazindol ER The Company's lead product candidate, Mazindol ER, is a proprietary extended-release formulation of mazindol and is being developed for the treatment of narcolepsy, and potentially other sleep-wake disorders such as Idiopathic Hypersomnia (IH), for which NLS recently obtained Orphan Disease Designation (ODD) from the FDA and the European Medicines Agency (EMA). Mazindol is a triple monoamine reuptake inhibitor and partial Orexin-2 Receptor agonist, which was used for many years to treat patients diagnosed with narcolepsy in compassionate use programs. A Phase 2 multi-center U.S. clinical study evaluating Mazindol ER in adult patients suffering from narcolepsy met its primary endpoint with high statistical significance and demonstrated a favorable safety and tolerability profile. NLS also successfully completed a Phase 2 study in the U.S. evaluating Mazindol ER in adult patients suffering from Attention Deficit/Hyperactivity Disorder (ADHD). The study met all primary and secondary endpoints and well-tolerated. Mazindol ER has received ODD both in the U.S. and in Europe for the treatment of narcolepsy. Up to 1/3 of narcoleptic patients are also diagnosed with ADHD. The Phase 3 program for Mazindol ER, AMAZE, will commence in July 2023. More information on these studies can be found at or . About NLS Pharmaceutics Ltd. NLS Pharmaceutics Ltd. (Nasdaq:NLSP) is a global development-stage biopharmaceutical company, working with a network of world-class partners and internationally recognized scientists, focused on the discovery and development of innovative therapies for patients with rare and complex central nervous system disorders who have unmet medical needs. Headquartered in Switzerland and founded in 2015, NLS is led by an experienced management team with a track record of developing and commercializing product candidates. For more information, please visit . Safe Harbor Statement This press release contains expressed or implied forward-looking statements pursuant to U.S. federal securities laws. For example, NLS is using forward-looking statements when it discusses the potential benefits of Mazindol ER and the timing and expected format of the AMAZE Phase 3 clinical program. These forward-looking statements and their implications are based on the current expectations of the management of NLS only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; NLS may encounter delays or obstacles in launching and/or successfully completing its clinical trials; NLS' products may not be approved by regulatory agencies, NLS' technology may not be validated as it progresses further and its methods may not be accepted by the scientific community; NLS may be unable to retain or attract key employees whose knowledge is essential to the development of its products; unforeseen scientific difficulties may develop with NLS' process; NLS' products may wind up being more expensive than it anticipates; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; NLS' patents may not be sufficient; NLS' products may harm recipients; changes in legislation may adversely impact NLS; inability to timely develop and introduce new technologies, products and applications; and loss of market share and pressure on pricing resulting from competition, which could cause the actual results or performance of NLS to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, NLS undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. More detailed information about the risks and uncertainties affecting NLS is contained under the heading "Risk Factors" in NLS' annual report on Form 20-F for the year ended December 31, 2022 filed with the Securities and Exchange Commission (SEC), which is available on the SEC's website, , and in subsequent filings made by NLS with the SEC. For additional information: Marianne Lambertson (investors & media) NLS Pharmaceutics Ltd. +1 239.682.8500 ml@nls-pharma.com SOURCE: NLS Pharmaceutics AG View source version on accesswire.com:

The dynamics of the cell and gene therapies market in rare disorders are anticipated to change as companies across the globe are thoroughly working toward developing new therapeutic options to treat a wide array of indications. LAS VEGAS, May 9, 2023 /PRNewswire/ -- DelveInsight's Cell and Gene Therapies in Rare Disorders Market Insights report includes a comprehensive understanding of current treatment practices, emerging cell and gene therapies for various rare disorders, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Key Takeaways from the Cell and Gene Therapies in Rare Disorders Market Report As per DelveInsight analysis, the cell and gene therapies in rare disorders market size in the 7MM was approximately USD 1.5 billion in 2022. According to the assessment done by DelveInsight, the estimated total prevalent cases of selected indications for cell and gene therapies in rare disorders in the 7MM were approximately 900K in 2022. Leading companies developing novel cell and gene therapies for various rare disorders, such as Hemophilia A, Hemophilia B, Fabry disease, Retinitis Pigmentosa, Amyotrophic Lateral Sclerosis, Duchenne Muscular Dystrophy, and others include Roche, Freeline Therapeutics, Spark Therapeutics, Astellas Gene Therapies, Actus Therapeutics, GenSight Biologics, Coave Therapeutics, Johnson & Johnson, MeiraGTx, Applied Genetic Technologies Corporation, GenSight Biologics, Nanoscope Therapeutics, 4D Molecular Therapeutics, Ocugen, jCyte, ReNeuron, REGENXBIO, Amicus Therapeutics, Pfizer, Sarepta Therapeutics, Capricor Therapeutics, Nippon Shinyaku, Brainstorm Cell Therapeutics, CRISPR Therapeutics, Vertex Pharmaceuticals, Editas Medicine, Sangamo Therapeutics, Krystal Biotech, Abeona Therapeutics, Castle Creek Biosciences, Holostem Terapie Avanzate S.r.l., RHEACELL, Ishin Pharma, Anterogen, Ultragenyx Pharmaceutical, and others. The promising cell and gene therapies for various rare disorders in the pipeline include Giroctocogene fitelparvovec, Dirloctocogene samoparvovec, Fidanacogene elaparvovec, Verbrinacogene setparvovec (FLT-180a), FLT190, Isaralgagene civaparvovec (ST-920), 4D-310, SPK-3006, AT845, ACTUS-101, LUMEVOQ (lenadogene nolparvovec), CTx-PDE6b, Botaretigene sparoparvovec, ATGC-501 (laruparetigene zosaparvovec), GS030, MCO-010 (sonpiretigene isteparvovec), 4D-125, OCU400, jCell, RGX-121, AT-GTX-502 (scAAV9.P546.CLN3), PF-06939926, SRP-9001, CAP-1002, NurOwn (MSC-NTF cells), Exagamglogene autotemcel, EDIT-301, BIVV003, VYJUVEK (beremagene geperpavec), EB-101, D-Fi (dabocemagene autoficel), RV-LAMB3-transduced epidermal stem cells, Allogeneic ABCB5-positive Stem Cells, ISN001, ALLO-ASC-SHEET, DTX301, UX111 (ABO-102), DTX401 (AAV8G6PC), and others. In July 2022, the Institute for Clinical and Economic Review (ICER) released a final evidence report assessing the comparative clinical effectiveness and value of ZYNTEGLO (betibeglogene autotemcel) for treating beta-thalassemia. In May 2022, Orchard Therapeutics reached an agreement with Gesetzliche Krankenversicherung Spitzenverband (GKV-SV), which will result in reimbursed access to LIBMELDY (atidarsagene autotemcel) for all metachromatic leukodystrophy (MLD) patients in Germany who fall within the scope of the European marketing authorization. Discover which therapies are expected to grab the major cell and gene therapies in rare disorders market share @ Cell and Gene Therapies in Rare Disorders Market Report Cell and Gene Therapies in Rare Disorders Overview Cell and gene therapies use genes and cells to treat disease. A gene is a unit of DNA containing genetic information passed down from generation to generation. The genome comprises all genes; genes may contain information on observable features such as height or eye color. Many genes contain instructions for RNA or protein molecules that are not visible from the outside but serve crucial tasks in the body's cells. Cells are the building blocks of plants and animals (including humans); they are small functional units that work together to generate organs and tissues. Cell and gene therapy technology is quickly evolving for many different diseases. However, cell and gene treatments are still experimental drugs, and much more study is required before many of these therapies are available to patients worldwide. Cell and Gene Therapies in Rare Disorders Epidemiology Segmentation DelveInsight estimates that there were approximately 900K prevalent cases of selected indications for cell and gene therapies in rare disorders in the 7MM in 2022. As per our analysis, the highest prevalent cases from the selected indications for cell and gene therapies in rare disorders were for Retinitis Pigmentosa in the United States, whereas the least cases were reported for Hunter Syndrome in 2022. The cell and gene therapies in rare disorders market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into: Total Prevalent Cases of Shortlisted Indications for Cell and Gene Therapies in Rare Disorders Total Indication Eligible-wise Cases for Cell and Gene Therapies in Rare Disorders Indication-wise Treated Cases of Cell and Gene Therapies Cell and Gene Therapies in Rare Disorders Market Insights Numerous cell and gene therapies for rare diseases are currently approved in the 7MM, including retinitis pigmentosa ( LUXTURNA), beta-thalassemia ( ZYNTEGLO), epidermolysis bullosa ( JACE), limbal stem cell deficiency ( OCURAL), and many others. As per Delveinsight analysis, the total cell and gene therapies in rare disorders market size was around USD 1.5 billion in 2022. According to predictions, the United States will have the largest cell and gene therapies in rare disorders market. ZOLGENSMA produced the highest revenue of roughly USD 1 billion among the 7MM in 2022, while ROCTAVIAN is predicted to take the highest market share by 2032. ROCTAVIAN has received conditional approval in Europe for the treatment of severe hemophilia A. In addition to this approval, BioMarin Pharmaceutical is working to approve the drug in the United States, with a PDUFA target action date of June 30, 2023. Hemophilia A is predicted to produce the most revenue among the selected indications by 2032, owing to the precedence of existing high treatment cost and expected high cost for emerging therapies along with significant residual unmet need. Gene treatments for Hemophilia A are estimated to earn around USD 6 billion in sales revenue by 2032 in the 7MM. The field of cell and gene therapies for rare indications is expected to rapidly expand in the coming years, as an increasing number of companies submit investigational new drug applications for these treatments each year, along with rising regulatory approval in the United States and Europe. In terms of manufacturing aspects of cell and gene therapies, there will be more competition for contract manufacturing businesses and pharmaceutical/biotechnology firms. The competition for contract manufacturing organization's production capacity will intensify as more companies enter the cell and gene therapy market, possibly driving up manufacturing costs. To ensure pharmaceutical/biotechnology firms can compete or obtain an advantage over competitors, companies may need to invest in manufacturing technologies or acquire companies with manufacturing expertise. To know more about cell and gene therapies in rare disorders treatment guidelines, visit @ Cell and Gene Therapy Insights Emerging Cell and Gene Therapies for Hemophilia A and Key Companies Giroctocogene fitelparvovec: Pfizer/Sangamo Therapeutics Dirloctocogene samoparvovec: Roche Emerging Cell and Gene Therapies for Hemophilia B and Key Companies Fidanacogene elaparvovec: Pfizer/Spark Therapeutics Verbrinacogene setparvovec (FLT-180a): Freeline Therapeutics Emerging Cell and Gene Therapies for Fabry Disease and Key Companies FLT190: Freeline Therapeutics Isaralgagene civaparvovec (ST-920): Sangamo Therapeutics 4D-310: 4D Molecular Therapeutics Emerging Cell and Gene Therapies for Pompe Disease and Key Companies SPK-3006: Spark Therapeutics AT845: Astellas Gene Therapies ACTUS-101: Actus Therapeutics Emerging Cell and Gene Therapies for Leber Hereditary Optic Neuropathy and Key Companies LUMEVOQ (lenadogene nolparvovec): GenSight Biologics Emerging Cell and Gene Therapies for Retinitis Pigmentosa and Key Companies CTx-PDE6b: Coave Therapeutics Botaretigene sparoparvovec: Johnson & Johnson/ MeiraGTx ATGC-501 (laruparetigene zosaparvovec): Applied Genetic Technologies Corporation GS030: GenSight Biologics MCO-010 (sonpiretigene isteparvovec): Nanoscope Therapeutics 4D-125: 4D Molecular Therapeutics OCU400: Ocugen jCell: jCyte Emerging Cell and Gene Therapies for Hunter Syndrome and Key Companies RGX-121: REGENXBIO Emerging Cell and Gene Therapies for Batten Disease and Key Companies AT-GTX-502 (scAAV9.P546.CLN3): Amicus Therapeutics Emerging Cell and Gene Therapies for Duchenne Muscular Dystrophy (DMD) and Key Companies PF-06939926: Pfizer SRP-9001: Sarepta Therapeutics CAP-1002: Capricor Therapeutics/Nippon Shinyaku Emerging Cell and Gene Therapies for Amyotrophic Lateral Sclerosis (ALS) and Key Companies NurOwn (MSC-NTF cells): Brainstorm Cell Therapeutics Emerging Cell and Gene Therapies for Beta Thalassemia and Sickle Cell Anemia and Key Companies Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals EDIT-301: Editas Medicine BIVV003: Sangamo Therapeutics Emerging Cell and Gene Therapies for Dystrophic Epidermolysis Bullosa and Key Companies VYJUVEK (beremagene geperpavec): Krystal Biotech EB-101: Abeona Therapeutics D-Fi (dabocemagene autoficel): Castle Creek Biosciences RV-LAMB3-transduced epidermal stem cells: Holostem Terapie Avanzate S.r.l. Allogeneic ABCB5-positive Stem Cells: RHEACELL ISN001: Ishin Pharma ALLO-ASC-SHEET: Anterogen Emerging Cell and Gene Therapies for Ornithine Transcarbamylase Deficiency and Key Companies DTX301: Ultragenyx Pharmaceutical Emerging Cell and Gene Therapies for Sanfilippo Syndrome Type A and Key Companies UX111 (ABO-102): Ultragenyx Pharmaceutical Emerging Cell and Gene Therapies for Glycogen Storage Disease Type IA and Key Companies DTX401 (AAV8G6PC): Ultragenyx Pharmaceutical Learn more about the FDA-approved cell and gene therapies for rare disorders @ Approved Cell and Gene Therapies in Rare Disorders Treatment Cell and Gene Therapies in Rare Disorders Market Dynamics The cell and gene therapies in rare disorders market is predicted to grow positively due to an increase in the approval of a growing number of gene therapies and their ease of adoption following approval, the ability to treat a wide range of conditions, an increase in the number of cases, an expected one-time dosing approach, and curative treatment options. The approval of LIBMELDY, SKYSONA, HOLOCLAR, UPSTAZA, ROCTAVIAN, and other medicines has successfully created regulatory channels for the development of further cell and gene therapies. Companies around the world are working hard to develop new cell and gene therapies options to treat a wide range of indications, such as hemophilia A and B, lysosomal storage disorder (Fabry, Pompe Disease, Danon Disease, MPS I, MPS II, MPS III), neurological disorders (Batten, Parkinson), musculoskeletal disorders (DMD, myotubular myopathy), eye diseases (achromatopsia, limbal stem cell deficiency, retinitis pigmentosa, retinoschisis, age-related macular degeneration, Leber's hereditary optic neuropathy), and other indications such as diabetic macular edema, inborn metabolism disorder (Wilson's disease, Phenylketonuria, OTC deficiency/urea cycle disorders), dystrophic epidermolysis bullosa, gangliosidosis, and xerostomia. Many diseases' treatment landscapes have drastically evolved in the last few years. Companies are now developing cell and gene therapies that will play an important role in the future, particularly in the treatment of rare genetic disorders. The process of defining ideal candidates for given gene therapy and cell therapy will have to wait for the enrolment and long-term follow-up of a sufficient number of study subjects to provide satisfactory clarity regarding its safety and efficacy. In conclusion, the future of cell and gene therapy looks optimistic. Several clinical trials have yielded favorable results in terms of safety and efficacy. The findings of these studies motivate additional research into many indications, and the current scenario predicts a positive shift in the cell and gene therapies in rare disorders market for the forecast period. However, several factors may impede the growth of cell and gene therapies in rare disorders market in the coming years. Despite advances since the enactment of the Orphan Drug Act, people in the United States with rare diseases continue to face challenges to diagnosis, care, and treatment. Moreover, in Europe, a possible crisis with gene treatments for rare diseases is developing, and few companies have withdrawn the drug after failing to get reimbursement in the EU. Scope of the Cell and Gene Therapies in Rare Disorders Market Report Therapeutic Assessment: Cell and Gene Therapies in Rare Disorders current marketed and emerging therapies Cell and Gene Therapies in Rare Disorders Market Dynamics: Conjoint Analysis of Emerging Cell and Gene Therapies in Rare Disorders Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's Views, Analyst's Views, Cell and Gene Therapies in Rare Disorders Market Access and Reimbursement Discover more about cell and gene therapies for rare disorders in development @ Cell and Gene Therapy Clinical Trials Table of Contents Related Reports Gene and Cell Therapies in Rare Disorder Epidemiology Forecast Gene and Cell Therapies in Rare Disorder Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted gene and cell therapies in rare disorder epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Cell And Gene Therapy For Multiple Myeloma Market Cell And Gene Therapy For Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cell and gene therapy for multiple myeloma companies, including Pfizer, MorphoSys, Biokine Therapeutics, Arcellx, Rapa Therapeutics, among others. Gene Therapy For Ocular Rare Disease Pipeline Gene Therapy For Ocular Rare Disease Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gene therapy for ocular rare disease companies, including Roche Holding, Novartis, Ocugen, among others. Adeno-Associated Virus Vectors in Gene Therapy Market Adeno-Associated Virus Vectors in Gene Therapy Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key adeno-associated virus vectors in gene therapy companies, including Pfizer, CSL Behring, Spark Therapeutics, Freeline Therapeutics, RegenxBio, Amicus Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

MALVERN, Pa., April 27, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the FDA has granted Orphan Drug Designation for its OCU410ST (AAV5-hRORA), an adeno-associated virus serotype 5 capsid protein containing gene construct encoding human retinoic acid receptor-related orphan receptor alpha, for the “treatment of ABCA4-associated retinopathies” including Stargardt, Retinitis Pigmentosa 19 (RP19), and Cone-rod dystrophy 3 (CORD3) diseases. “There are approximately 44,000 patients in the U.S. living with ABCA4-associated retinal diseases for whom no treatment options exist,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “This designation acknowledges the potential for OCU410ST to fulfill a significant unmet medical need and represents an important milestone in our effort to develop innovative treatments for inherited retinal diseases.” Orphan drug designation is granted by the FDA to certain products that show promise in the treatment, prevention, or diagnosis of rare and serious diseases affecting fewer than 200,000 people in the United States. OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. The ABCA4 gene produces an ATP-binding cassette (ABC) superfamily transmembrane protein and expressed exclusively in retinal photoreceptors. It is involved in clearance of all-trans-retinal aldehyde, a byproduct of the retinoid cycle, from photoreceptor cells. Mutation in ABCA4 gene results in Stargardt disease, a rare genetically inherited disease that directly affects the retina of the eye, often resulting in the slow progression of vision loss in children. In addition, different ABCA4 alleles have been identified to cause other retinopathies such as CORD3 and RP19. Ocugen plans to submit an IND in 2Q 2023 to initiate a Phase 1/2 clinical trial. About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, 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 Twitter 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 Tiffany.Hamilton@ocugen.com