Cone-Rod Dystrophies

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

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

First iPSC therapy candidate to be licensed from the strategic R&D collaboration forged between BlueRock, FUJIFILM Cellular Dynamics, and Opsis Therapeutics in 2021 Primary photoreceptor diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in children and adults IND filing for OpCT-001 is planned for 2024 CAMBRIDGE, Mass., Jan. 23, 2024 /PRNewswire/ -- BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, announced today that it has exercised its option to exclusively license OpCT-001, an induced pluripotent stem cell (iPSC) derived cell therapy candidate for the treatment of primary photoreceptor diseases, from FUJIFILM Cellular Dynamics and Opsis Therapeutics. OpCT-001 is the lead cell therapy candidate being developed under the strategic R&D collaboration between BlueRock, FUJIFILM Cellular Dynamics, and Opsis Therapeutics that was forged in 2021. Under the terms of the agreement FUJIFILM Cellular Dynamics, and Opsis Therapeutics receive an undisclosed license fee and are eligible to receive payments upon achievement of certain development and commercial milestones. "We believe that cell therapy has great potential for restoring vision in patients who are living with retinal diseases," said Ahmed Enayetallah, Senior Vice President and Head of Development at BlueRock Therapeutics. "Our collaboration with the FUJIFILM Cellular Dynamics and Opsis Therapeutics team has allowed us to execute important IND-enabling activities, and we are excited to advance OpCT-001 toward the clinic, with an IND filing planned for this year." Primary photoreceptor diseases are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone rod dystrophies. These diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. No treatment options currently exist for this patient population. OpCT-001 aims to restore vision loss caused by these diseases by replacing degenerated tissue in the retina with functional cells. About BlueRock Therapeutics LP BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to reverse devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for patients suffering from neurological, cardiovascular, immunological, and ophthalmic diseases. Our lead clinical program, bemdaneprocel, (BRT-DA01) is in Phase I clinical trials for Parkinson's disease. We were founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to . Find more information at Follow us on Facebook: Forward-Looking Statements Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as "anticipate," "believe," "forecast," "estimate," "plan," and "intend," among others. These forward-looking statements are based on BlueRock's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the initiation, timing, progress, activities, goals and reporting of results of any preclinical programs and clinical trials and research and development programs, the potential benefits, timing and future operation of the agreements with FUJIFILM Cellular Dynamics and Opsis Therapeutics, the ability to advance therapies into, and successfully initiate, enroll and complete clinical trials, the potential clinical utility of product candidates, the regulatory pathway of, and the timing or likelihood of any regulatory filings and approvals for, any product candidates, and the ability to, and extent of, potentially commercializing any product candidate, are forward looking. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of BlueRock's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof. About BlueRock Therapeutics LP  BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to reverse devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for patients suffering from neurological, cardiovascular, immunological, and ophthalmic diseases. Our lead clinical program, bemdaneprocel, (BRT-DA01) is in Phase I clinical trials for Parkinson's disease. We were founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy Platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit      Follow us on LinkedIn: linkedin.com/company/bluerocktx About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to . Find more information at   Follow us on Facebook: Follow us on Twitter: @BayerPharma SOURCE BlueRock Therapeutics