RPGR

AGTC-501 was generally safe and well-tolerated and showed robust improvements in visual function at the 12-month analysis Pivotal Phase 2/3 VISTA trial for AGTC-501 in XLRP expected to begin in H1 2024 LONDON and CAMBRIDGE, Mass., Feb. 8, 2024 /PRNewswire/ -- Beacon Therapeutics Holdings Limited ('Beacon Therapeutics' or 'the Company'), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, yesterday announced the presentation of 12-month interim safety and efficacy results for the Phase 2 SKYLINE trial in patients with X-linked retinitis pigmentosa (XLRP) at the 47th Annual Macula Society Meeting in Palm Springs, California. Twelve-month data from males with XLRP demonstrated a response rate (defined as an improvement in retinal sensitivity, as assessed by microperimetry, of at least 7 decibels (dB) in at least 5 loci) of 63% in study eyes treated with a high dose (6.8 E+11 vg/eye) of AGTC-501. Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both). In addition, patients in the high dose cohort also demonstrated a robust improvement in visual function, including mean retinal sensitivity. AGTC-501 was well-tolerated and there were no clinically significant safety events associated with treatment, and any treatment-related adverse events were mostly nonserious and mild to moderate in severity. XLRP is an orphan disease predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, and it is therefore expected to rescue function of both rod and cone photoreceptors, making it uniquely well-suited as a potential treatment to improve the lives of patients with XLRP. Dr. Nadia Waheed, Chief Medical Officer of Beacon Therapeutics, said, "These data, which demonstrate a favorable safety profile and notable improvement in visual function, are another positive step in the development of AGTC-501 for XLRP, a blinding, orphan disease for which there is currently no approved treatment. We look forward to announcing the initiation of our Phase 2/3 VISTA trial in the first half of 2024." Presentation – Subretinal AGTC-501 Gene Therapy for XLRP: 12-Month Interim Safety & Efficacy Results of the Phase 2 SKYLINE Trial Presenter – Mark Pennesi, M.D., Ph.D., FARVO, Director, Ophthalmic Genetics at the Retina Foundation in Dallas, Texas; Professor of Ophthalmology and Professor of Molecular and Medical Genetics, Paul H. Casey Ophthalmic Genetics Division at the Casey Eye Institute, Oregon Health and Science University The presentation took place on Wednesday, February 7 at 6:25 p.m. PT. Contact: [email protected] Media: [email protected] About Beacon Therapeutics Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501 is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises. Find out more about Beacon Therapeutics at beacontx.com. SOURCE Beacon Therapeutics

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

PRESS RELEASE Beacon Therapeutics launches with £96 million ($120 million) to develop a new generation of gene therapies for retinal diseases resulting in blindness New leading gene therapy company created combining late-stage X-linked retinitis pigmentosa (XLRP) program from AGTC, a preclinical program spun out from the University of Oxford and a second preclinical program to deliver a broad ophthalmic development pipelineLed by a highly experienced leadership team including David Fellows, Dr. Nadia Waheed, and Dr. Abraham Scaria, each with decades of ophthalmic gene therapy experience£96m ($120 million) in funding from lead investor Syncona and additional investors including Oxford Science Enterprises (OSE) to progress pipeline to key value inflection points London and Oxford, UK, 12 June 2023 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a purpose to restore and improve the vision of patients with retinal diseases, launches today and will develop a new generation of gene therapies to treat a range of rare and prevalent retinal diseases that result in blindness. Syncona Limited, with additional investors including OSE, has provided £96 million ($120 million) to fund the acquisition of AGTC and provide capital to take each of Beacon Therapeutics’s development candidates through to value inflection points. Beacon Therapeutics’s lead clinical asset is AGTC-501, a gene therapy program currently in Phase II clinical trials for the treatment of XLRP that was acquired as part of Syncona’s acquisition of AGTC in November 2022. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Unlike other approaches in the space, AGTC-501 correctly expresses the full length RPGR protein, thereby addressing the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics’s first pre-clinical asset is an intravitreally (IVT) delivered novel AAV based program for dry Age-related Macular Degeneration (dry AMD). Dry AMD is a leading cause of irreversible vision loss in people over 60, if left untreated. Around 20 million people in the United States suffer from AMD. The second pre-clinical asset is targeting cone-rod dystrophy (CRD) which is caused by a null mutation in the Cadherin Related Family Member 1 (CDHR1) gene. The program has been licensed from the laboratory of Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford. Prof. MacLaren, a world leading retinal expert and an experienced biotech company founder and a Co-Founder of Beacon Therapeutics, will become a key scientific advisor to the company and will join the board of directors. To bolster its pipeline in the future, Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. Beacon Therapeutics will be led by David Fellows, the former Chief Executive of Nightstar Therapeutics with over 40 years’ experience in the ophthalmology field. He will be joined by retinal expert Dr. Nadia Waheed as Chief Medical Officer, formerly from Gyroscope Therapeutics. Dr. Abraham Scaria also joins the team from AGTC as Chief Scientific Officer bringing 25 years of experience in gene therapy, ranging from discovery research to early-stage clinical trials. David Fellows, Chief Executive Officer of Beacon Therapeutics commented: “Beacon Therapeutics combines a broad development pipeline, a deep scientific foundation, a strong clinical network, and a highly experienced management team to drive forward a unique late-stage clinical and pre-clinical pipeline. With the 12-month data from our Phase II SKYLINE trial for AGTC-501 expected shortly and two highly innovative and differentiated pipeline assets for prevalent and rare blinding diseases, we are excited to be building a new leader in the ophthalmic gene therapy space.” Chris Hollowood, CEO of Syncona Limited, the lead investor in Beacon Therapeutics commented: “By acquiring AGTC and combining its late-stage program with highly complementary and innovative science from the University of Oxford and elsewhere, we have created an exciting new gene therapy company in an area where we have a great breadth and depth of knowledge and expertise. We look forward to working closely with Beacon Therapeutics’ exceptional management team to advance these assets through the clinic and towards people suffering from debilitating retinal diseases.” Professor Robert MacLaren, Professor of Ophthalmology, University of Oxford, and Co-Founder of Beacon Therapeutics commented: “I have dedicated many years to translating pioneering gene therapies from the lab through to clinical trial phase with the support of my team in the Nuffield Laboratory of Ophthalmology and NIHR Oxford Biomedical Research Centre (BRC). Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data. The company has both highly innovative retinal gene therapy programmes and the means to target them to the main causes of blindness in both the young and old.” Katya Smirnyagina, Senior Partner, Life Sciences, Oxford Science Enterprises (OSE) added: “Beacon Therapeutics is truly an exciting new company built around an established scientific foundation and a wealth of experience in the gene therapy and ophthalmology space. We are delighted that expertise spun out from the University of Oxford will become a key component to drive the future success of the business that could bring significant change in the treatment of a broad range of retinal diseases that cause blindness.”Broad ophthalmic development pipelineThe company’s lead clinical gene therapy program AGTC-501 has a strong body of clinical evidence targeting XLRP, an inherited rare form of retinitis pigmentosa that causes progressive vision loss in boys and young men. AGTC-501 demonstrated meaningful efficacy and a good safety profile in the recent Phase I/II HORIZON trial and expects to be publishing 12-month data from its Phase II SKYLINE trial in the second half of 2023. In addition, Beacon Therapeutics is awaiting feedback from the US Food and Drug Administration (FDA) regarding the study design of its upcoming VISTA clinical trial, a Phase II/III study to assess the effect of AGTC-501 on the symptoms of retinitis pigmentosa in additional patients with XLRP. The first preclinical program features intravitreally (IVT) delivered gene therapy for dry AMD. IVT delivery provides access to a significantly greater number of patients than sub-retinal delivery as it can be administered within an outpatient clinic rather than requiring surgery. The second preclinical program, CRD, targets mutations in the CDHR1 gene, which may cause central vision loss similar to dry AMD in older people and severe sight loss in younger patients. The Company has a new location in London, UK, complementing existing facilities in Boston, Massachusetts and Alachua, Florida, US. Contacts:Beacon Therapeutics:David Fellows, CEOEmail: info@beacontx.com Consilium Strategic Communications:Alex Harrison, Ashley TappEmail: Beacon@consilium-comms.com Tel: +44 (0)20 3709 5700 About Beacon Therapeutics Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises. Find out more about us online at: www.beacontx.com.