CD59

– Significant advancements in engineering neutralizing antibody (NAb) evasion properties into receptor-targeted capsids, including novel TfR1-binding capsids, demonstrate increased opportunities to treat AAV seropositive patients – Disclosed multiple families of capsids targeting two novel proteins – CA4 and CD59; capsids binding human CA4 cross the blood-brain barrier in humanized mice, while capsids that cross react with human and non-human primate CD59 demonstrate enhanced tropism for the Central Nervous System (CNS) and skeletal muscle NEW YORK, May 10, 2024 (GLOBE NEWSWIRE) -- Apertura Gene Therapy, a biotechnology company widening the aperture of gene therapy opportunities for treating debilitating diseases, showcased significant advancements in its capsid engineering platform during several presentations at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Scientists from the lab of academic collaborator and scientific co-founder, Ben Deverman, Ph.D., Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard, presented new data showcasing the progress made in engineering neutralizing antibody (NAb) evasion attributes into the company’s novel, human-receptor targeted capsids and disclosed two newly identified families of capsids targeting human CA4 and CD59. “The progress regarding human CA4, CD59 and NAb-evading capsids presented by the lab of Apertura collaborator and scientific co-founder, Dr. Ben Deverman, support our position as the leader in receptor-mediated capsid engineering and validate our strategy to advance our internal CNS programs utilizing better gene therapy delivery vehicles that are human-centric blood brain barrier crossing capsids. We look forward to advancing our first program toward an IND submission next year,” said Joseph La Barge, Chief Executive Officer of Apertura. “Our proven ability to engineer NAb-evading attributes into our TfR1 and other receptor targeting capsids opens new opportunities to treat more patients who otherwise would not be eligible for gene therapy clinical trials or treatments.” The presence of anti-AAV NAbs at even low levels has been repeatedly shown to lower therapeutic levels of transgene expression and has long been a challenge in the gene therapy field. The presence of pre-existing NAbs across the population has limited the eligible patient population for clinical trials and addressable patient population for commercial products. Apertura’s capsid engine has yielded novel capsids that have demonstrated NAb-evading properties while maintaining the full engineered tropism of the capsid. Specifically, the data show that the latest TfR1-binding capsids are not neutralized below 50% activity (when diluted at 1:4) in a subset of sera that are neutralizing to AAV9, AAV5 and the TfR1 binding capsids without antibody evading mutations. Data from the company’s ongoing preclinical studies support the premise that subsets of patients with pre-existing NAbs, who would otherwise be excluded from treatment, could be dosed with its novel, NAb-evading capsids. The CD59 receptor is a GPI-anchored protein and member of the Ly6 family that is broadly expressed across the human body. Apertura’s novel capsids, engineered with the company’s proprietary, mechanism-of-action focused engine, have demonstrated through in-vitro and in-vivo studies efficient gene delivery to the brain, spinal cord and skeletal muscle, while de-targeting the liver, opening up significant opportunities for addressing neuromuscular and lysosomal storage disorders. These capsids have also shown to cross react with non-human primate CD59. Carbonic anhydrase IV (CA4) is a GPI-anchored enzyme highly expressed on endothelial cells across the blood-brain barrier. These proprietary, engineered capsids effectively cross the blood brain barrier in mice modified to express the human receptor and transduce multiple cell types across the CNS with high efficiency. The oral presentations and posters presented at ASGCT can be viewed here. Presentation details Presentation: Second Generation AAV Capsids Reprogrammed to Bind Human Transferrin Receptor are Targeted to the Brain and De-targeted from the Liver in human TFRC Knock-in Mice (#120)Presenter: Ken Chan, PhD, Broad Institute of MIT and Harvard Presentation: An AAV Capsid Programmed to Bind a Broadly Expressed Human Receptor Mediates Enhanced Gene Delivery to the CNS and Skeletal Muscle in vivo (#217)Presenter: Jason Wu, PhD, Broad Institute of MIT and Harvard Poster: Human CA4-Targeting AAV Capsids Efficiently Transduce the CNS in Humanized Mice (#991)Presenters: Nuria Roxana Botticello-Romero, Jason Wu, Qin Huang, Thomas Beddow, Ken Y. Chan, Pamela Brauer, Fatma Elzahraa Eid, Alina Chan, Benjamin E. Deverman Poster: Engineering Human CNS Receptor-Targeted Capsids for Improved Antibody Evasion (#1551)Presenters: Simon Pacouret, William Donahoe, John W. Harvey, Pamela P. Brauer, Qingxia Zheng, Chin-Yen Lin, Binhui Zhao, Gabrielle Clouse, Qin Huang, Jencilin Johnson, Casey Keyes, Catherine P. Pirtle, Jarrett Rios, Albert T. Chen, Fatma Elzahraa Eid, Andrew J. Barry, Yujia A. Chan, Ken Y. Chan, Benjamin E. Deverman, Poster: Benchmarking natural and synthetic AAV capsids for neutralizing antibody evasion (#1553)Presenters: William Donahoe, Simon Pacouret, Pamela P. Brauer, John W. Harvey, Catherine P. Pirtle, Ken Y. Chan, Yujia A. Chan, Benjamin E. Deverman About Apertura Gene TherapyApertura Gene Therapy is a biotechnology company unlocking new opportunities for treating currently intractable diseases. The company is uniquely positioned to develop genetic medicines by simultaneously engineering AAV capsids, genetic regulatory elements and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity and manufacturability. Apertura is advancing an internal pipeline of programs targeting undisclosed central nervous system disorders utilizing its novel capsids and plans to submit an Investigational New Drug (IND) application for its first product candidate in 2025. Founded in 2021 on technologies from the Broad Institute and Harvard Medical School, and with support from Deerfield Management Company, the company is based in New York City. For more information, please visit our website at www.aperturagtx.com and follow us on LinkedIn. Contacts: Investorsinvestors@aperturatx.com Mediamedia@aperturatx.com

Five abstracts to be presented, including new real-world research and data on Janssen's investigational gene therapy JNJ-1887 RARITAN, N.J., Oct. 3, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that five company-sponsored presentations will be featured during the European Society of Retina Specialists (EURETINA) 2023 annual meeting, taking place in Amsterdam, from October 5-8. The Company's presentations will include safety analysis data from two Phase 1 trials of the investigational gene therapy JNJ-81201887 (JNJ-1887): one trial in patients with geographic atrophy (GA), an advanced stage and severe form of age-related macular degeneration (AMD), and one trial in wet AMD (Abstracts #CA231214 and #CA231118).1,2 In addition, new research highlighting descriptive analyses of real-world GA trial populations, patients' perspectives on the symptoms and impact of X-linked retinitis pigmentosa (XLRP) and comorbidities among inherited retinal dystrophy patients will also be shared (Abstracts #CA231637, #CA231346 and #CA23429).3,4,5 Continue Reading Dare to Envision Courageous Science Normal Anatomy of the Retina Experience the full interactive Multichannel News Release here: "These data at EURETINA 2023 help bring us one step closer to a new era of innovation in retina," said James List, M.D., Ph.D., Global Therapeutic Area Head, whose team oversees a portfolio of programs including Retina at Janssen Research & Development, LLC. "We're committed to building on this momentum – and doing so with boldness and urgency – as we advance our mission to restore and preserve vision for those living with retinal diseases." GA is an advanced form of AMD.6 It affects more than five million individuals globally and is a leading cause of blindness in people over 65 years of age.5 It has a devastating impact on these patients and their quality of life, including their ability to read, drive and perform other day-to-day activities.5 XLRP is a rare inherited condition estimated to impact one in 40,000 people globally.7,8 People with XLRP have progressive vision loss, starting in childhood with night blindness.9 Over time, they lose their peripheral vision, and by approximately age 40, many are legally blind.8 Currently, there are no approved treatments for XLRP.8 A complete listing of the Company's abstracts being featured at the EURETINA Annual Meeting is provided below. Abstracts can also be found on the EURETINA website. Janssen will also have an interactive Retina Medical Affairs exhibit booth (location #12.115) at the EURETINA annual meeting. For more information about Janssen Retina's research and portfolio, please visit . About JNJ-1887 JNJ-81201887 (JNJ-1887), formerly referred to as AAVCAGsCD59, is an investigational one-time gene augmentation therapy for the treatment of people with geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD). JNJ-1887 is designed to increase local expression of a soluble form of CD59 (sCD59) to possibly reduce MAC formation and protect retinal cells, thus potentially slowing and preventing disease progression. The Phase 2b PARASOL clinical trial (NCT05811351) is actively enrolling patients to study JNJ-1887 for the treatment of adults 60 or older with advanced dry AMD with GA. JNJ-1887 has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) and Advanced Therapy Medicinal Product (ATMP) designation by the European Medicines Agency (EMA). About botaretigene sparoparvovec (bota-vec) Botaretigene sparoparvovec (bota-vec) is being investigated in collaboration with MeiraGTx Holdings plc for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye-specific form of the RPGR gene. Through a one-time administration, bota-vec is designed to deliver functional copies of the RPGR gene that may counteract the loss of retinal cells with the goal of preserving and potentially restoring vision for those living with XLRP. The Phase 3 LUMEOS clinical trial (NCT04671433) has surpassed enrollment and is evaluating participants to study bota-vec for the treatment of patients living with XLRP with disease-causing variants in the RPGR gene. Bota-vec has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME), Advanced Therapy Medicinal Product (ATMP) and Orphan designations by the European Medicines Agency (EMA). About the Janssen and MeiraGTx Strategic Collaboration In January 2019, Janssen Research & Development, LLC entered into a worldwide collaboration and license agreement with MeiraGTx Holdings plc, a clinical-stage gene therapy company, to develop, manufacture and commercialize its clinical-stage inherited retinal disease portfolio. Botaretigene sparoparvovec (bota-vec) is being developed as part of a collaboration and license agreement. In addition to forming a research collaboration to explore new targets for other inherited retinal diseases, Janssen is working with MeiraGTx to build core capabilities in viral vector design, optimization and manufacturing. About the Janssen Pharmaceutical Companies of Johnson & Johnson At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension. Learn more at . Follow us at @JNJInnovMed and @JanssenUS. Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding botaretigene sparoparvovec and JNJ-81201887. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended January 1, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. Neither Janssen Research & Development, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments. ________________________________ 1 Holz F et al. Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy (GA) Due to Age-related Macular Degeneration (AMD). Abstract #CA231214. European Society of Retina Specialists 2023 Annual Meeting. 2 Fischer, DM et al. Pooled Safety Analysis of a Single Intravitreal Injection of JNJ-1887 (Gene Therapy, AAVCAGsCD59) in Patients With Age-Related Macular Degeneration (AMD). Abstract #CA231118. European Society of Retina Specialists 2023 Annual Meeting. 3 Besirli, C et al. Concept elicitation interviews to obtain patients' perspectives on the symptoms and impacts of X-linked retinitis pigmentosa. Abstract #CA231346. European Society of Retina Specialists 2023 Annual Meeting. 4 Wennberg, A et al. Comorbidities Among Inherited Retinal Dystrophy Patients in Sweden: A National Patient Register Study. Abstract #CA23429. European Society of Retina Specialists 2023 Annual Meeting. 5 Gale, SL et al. Descriptive analyses of a real-world cohort of patients with geographic atrophy secondary to age-related macular degeneration. Abstract #CA231637. European Society of Retina Specialists 2023 Annual Meeting. 6 Cohen, MN et al. Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy (GA) Due to Age-related Macular Degeneration (AMD). Abstract #30071749. Presented at the 2022 American Academy of Ophthalmology Annual Meeting 7 Boughman JA, Conneally PM, Nance WE. Population genetic studies of retinitis pigmentosa. Am J Hum Genet. 1980;32(2):223–235. 8 Fishman GA. Retinitis pigmentosa. Genetic percentages. Arch Ophthalmol. 1978;96(5):822–826. 9 Wang DY, Chan WM, Tam PO, et al. Gene mutations in retinitis pigmentosa and their clinical implications. Clin Chim Acta. 2005;351(1-2):5-16. Me d i a Inquiries: Jennifer Silvent Mobile +1 973-479-9845 Rachael Jarnagin Mobile +1 415-705-9023 Investor Relations: Raychel Kruper Office +1 732-524-6164 [email protected] Click to Access Audio SOURCE Johnson & Johnson Innovative Medicine

SEATTLE--( BUSINESS WIRE )--Omeros Corporation (Nasdaq: OMER) today announced that the slides presented yesterday in the “late-breaker” session at the 2023 European Hematology Association (EHA) Congress in Frankfurt, Germany are now available on Omeros’ website . The presentation, which details data from a pre-specified interim analysis from the ongoing Phase 1b clinical trial of Omeros’ lead MASP-3 inhibitor OMS906 in complement-inhibitor-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH), was identified by EHA’s Scientific Program Committee as one of the top five late-breaking submissions and selected for oral presentation. The presentation, entitled OMS906, A mannan-binding lectin-associated serine protease-3 (MASP-3) Inhibitor, Normalizes Hemoglobin Levels in Treatment-naïve PNH Patients: Interim Data from a Proof-of-Concept Clinical Trial, was delivered to a large audience by Jens Panse, MD, Senior Physician and Deputy Director of the Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, and Managing Medical Director of the Center for Integrated Oncology, Aachen, Germany. Also available on Omeros’ website is a second presentation at EHA – Alternative Pathway MASP-3 Inhibitor OMS906: Results from a First-in-Man Phase 1 Study in Healthy Subjects and Study Design of Two Ongoing Clinical Trials in Patients with PNH – by Morag Griffin, MBChB, FRCPath, Consultant in Haematology of St. James University Teaching Hospital, Leeds, United Kingdom. This poster presentation describes findings from a single-ascending dose study evaluating OMS906 safety, pharmacokinetics and pharmacodynamics in healthy subjects. About PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening acquired disorder that causes production of red blood cells lacking certain surface proteins (CD55 and CD59), making them vulnerable to destruction by the complement system. This leads to intravascular hemolysis (destruction of red blood cells within blood vessels) and extravascular hemolysis (when damaged red blood cells are removed by macrophages in the spleen or liver). Left untreated, PNH is associated with debilitating anemia, a high risk of thrombosis, fatigue, and a severely reduced survival rate. There remains a significant unmet need for PNH therapies, as a large proportion of patients treated with C5 inhibitors continue to experience hemolysis and approximately one third of them still require red blood cell transfusions. About OMS906 OMS906 is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the complement system’s alternative pathway. The complement system plays a central role in inflammation and becomes activated as a result of tissue damage or microbial infection. Responsible for the conversion of pro-complement factor D to complement factor D, MASP-3 is believed to be the premier target in the alternative pathway. MASP-3 has the lowest native circulating level and low relative clearance compared to the other alternative pathway proteins and, unlike C5 and C3 blockers, MASP-3 inhibition leaves intact the lytic arm of the classical pathway, important for fighting infection. Also, unlike other components of the alternative pathway, MASP-3 is believed not to be an acute phase reactant, which could provide a significant advantage to MASP-3 inhibitors, like OMS906, over other alternative pathway inhibitors. MASP-3 inhibitors are thought to have preventive or therapeutic effects across a broad range of diseases including PNH, hemolytic uremic syndrome (HUS), atypical HUS, traumatic brain injury, arthritis, wet age-related macular degeneration, ischemia-reperfusion injury, transplant-related complications and other immune-related disorders. Through its growing and exclusive intellectual property position, Omeros controls the use of MASP-3 inhibitors across a wide range of alternative pathway-related and other diseases and disorders. About Omeros Corporation Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders including complement-mediated diseases, cancers, and addictive and compulsive disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application pending before FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, COVID-19, and atypical hemolytic uremic syndrome. Omeros’ long-acting MASP-2 inhibitor OMS1029 is currently in a Phase 1 clinical trial. OMS906, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is advancing across multiple clinical programs for alternative pathway-related diseases, including paroxysmal nocturnal hemoglobinuria (PNH) and complement 3 (C3) glomerulopathy. For more information about Omeros and its programs, visit www.omeros.com .