Keio University School of Medicine

RSS Otsuka Pharmaceutical Co., Ltd. (Otsuka) announces that the hematological tumor gene panel test HemeSite®, the first-in-Japan, comprehensive genomic profiling assay for hematological malignancies, is covered by Japan's national health insurance system, effective March 1. Sales have commenced today. In recent years, classification and diagnostic criteria for hematological malignancies from the World Health Organization (WHO), as well as clinical treatment guidelines from medical societies around the world, highlight the critical importance of medical treatment based on genome information, making this information essential for the latest medical treatment. In Japan, the usefulness of genomic information for diagnosis, treatment selection, and prognosis prediction is described in the Japanese Society of Hematology Genome Guidelines*1. Insurance coverage for the cancer gene panel test for solid tumors exists in Japan, but until now insurance reimbursement has not been provided to any gene panel test for hematological malignancies. HemeSight was developed jointly by Otsuka and the National Cancer Center Japan. Its performance has been verified by a research consortium*2 comprising the National Cancer Center, Kyushu University, Kyoto University, Nagoya Medical Center, the Institute of Medical Science at the University of Tokyo, and Keio University School of Medicine. In hematological malignancies, genomic information is useful for treatment selection and for diagnosis and prognosis prediction, therefore the use of gene panel testing at the time of initial diagnosis is recommended in the Genome Medicine Promotion Act. From now on, the initial diagnosis of leukemia such as acute myeloid leukemia, malignant lymphoma, and multiple myeloma is covered by insurance. Accordingly, in the case of acute myeloid leukemia, HemeSight can be used for prognosis prediction based on genetic abnormalities and in the case of malignant lymphoma and multiple myeloma, it can be used for diagnosis when conventional methods cannot be used or when it is difficult to distinguish from other diseases. In addition, diseases such as acute myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia are also covered by insurance when they recur or are refractory. Also, the start of insurance coverage may be beneficial to patients with unexplained, significant decreases in blood cells, for whom it has been difficult to determine treatment plans because definitive diagnoses could not be made. The launch of the hematological tumor gene panel test HemeSight is expected to advance the social implementation of personalized medicine for hematological tumors in Japan. Product Overview The hematological tumor gene panel test HemeSight is composed of the in vitro diagnostic drug HemeSight Diagnostic Drug and the medical device program HemeSight Analysis Program. *1 Japanese Society of Hematology Guidelines for Genomic Testing of Hematological Malignancies, 2021 Partially Revised Edition　（http://www.jshem.or.jp/genomgl/home.html） *2 Consists of an in vitro diagnostic product for hematological malignancies gene panel testing and its program.

TOKYO, Japan I February 13, 2025 I Restore Vision Inc., a clinical-stage biotechnology company advancing gene therapies for retinal disorders, today announced that the first patient in its Phase I/II clinical trial with RV-001 has been dosed at Keio University Hospital. RV-001 utilizes adeno-associated virus (AAV) vectors to deliver a unique functional gene encoding “Chimeric Rhodopsin”, a light-driven G-protein activation mechanism to re-establish light activation in retinal interneurons. The goal is to expand light sensitivity to retinal interneurons via an intravitreal injection of RV-001. This milestone marks the initiation of the world’s first clinical trial of optogenetic gene therapy, which leverages a proprietary “Chimeric Rhodopsin” for patients with retinitis pigmentosa (RP). “Over 2 million people worldwide suffer photoreceptor loss in retinitis pigmentosa, resulting in severe vision loss and eventual blindness” said, CEO and Ophthalmologist Yusaku Katada, MD, PhD. “Our “chimeric rhodopsin” approach has broad potential to alter light sensitivity across multiple subtypes of RP patients. [1] We are continuing to advance the Phase I/II trial to assess safety, light-activation and light-sensitivity in these patients, post-administration of our lead gene therapy product RV-001.” Phase I/II Safety, Tolerability, and Exploratory Efficacy Trial Highlights Connecting Innovation, Academia, and Investment Restore Vision, Inc., a spin-out startup from Keio University, bridges cutting-edge academic innovation with entrepreneurial application to drive progress in retinal therapies. Developed through collaborations with Keio University School of Medicine and Nagoya Institute of Technology, RV-001’s optogenetic technology addresses the limitations of traditional approaches by combining high light sensitivity with a unique G-protein activation mechanism. [2],[3] This groundbreaking technology has the potential to be expanded to other retinal diseases, paving the way for broader applications in vision restoration. The Japan Agency for Medical Research and Development (AMED) and other academic grants, with key contributions from Keio University and Nagoya Institute of Technology, supported RV-001 development. About Restore Vision Restore Vision Inc., a clinical-stage gene therapy company, specializes in developing transformative gene therapies for inherited retinal disorders. By integrating academic excellence with entrepreneurial vision, the company is committed to pioneering treatments that address critical unmet medical needs and improve patient outcomes worldwide. [1] Roberts et al. Arch Ophthalmol. 2010 [2] Sasaki et al. PLoS One. 2014 [3] Katada et al. iScience. 2023 SOURCE: Restore Vision

The first optogenetic "Chimeric Rhodopsin" gene therapy tested in humans. RV-001 starting dose was generally well tolerated in this open-label trial. RV-001 a gene-agnostic approach for RP patients. TOKYO, Feb. 13, 2025 /PRNewswire/ -- Restore Vision Inc., a clinical-stage biotechnology company advancing gene therapies for retinal disorders, today announced that the first patient in its Phase I/II clinical trial with RV-001 has been dosed at Keio University Hospital. RV-001 utilizes adeno-associated virus (AAV) vectors to deliver a unique functional gene encoding "Chimeric Rhodopsin", a light-driven G-protein activation mechanism to re-establish light activation in retinal interneurons. The goal is to expand light sensitivity to retinal interneurons via an intravitreal injection of RV-001. This milestone marks the initiation of the world's first clinical trial of optogenetic gene therapy, which leverages a proprietary "Chimeric Rhodopsin" for patients with retinitis pigmentosa (RP). "Over 2 million people worldwide suffer photoreceptor loss in retinitis pigmentosa, resulting in severe vision loss and eventual blindness" said, CEO and Ophthalmologist Yusaku Katada, MD, PhD. "Our "chimeric rhodopsin" approach has broad potential to alter light sensitivity across multiple subtypes of RP patients.[1] We are continuing to advance the Phase I/II trial to assess safety, light-activation and light-sensitivity in these patients, post-administration of our lead gene therapy product RV-001." Phase I/II Safety, Tolerability, and Exploratory Efficacy Trial Highlights Patient Criteria: Participants with advanced RP, agnostic to genotype, and characterized by severe vision loss, no- to limited vision. Safety and Efficacy: In addition to safety, exploratory efficacy endpoints include improvements in light sensitivity and visual function. Connecting Innovation, Academia, and Investment Restore Vision, Inc., a spin-out startup from Keio University, bridges cutting-edge academic innovation with entrepreneurial application to drive progress in retinal therapies. Developed through collaborations with Keio University School of Medicine and Nagoya Institute of Technology, RV-001's optogenetic technology addresses the limitations of traditional approaches by combining high light sensitivity with a unique G-protein activation mechanism.[2],[3] This groundbreaking technology has the potential to be expanded to other retinal diseases, paving the way for broader applications in vision restoration. The Japan Agency for Medical Research and Development (AMED) and other academic grants, with key contributions from Keio University and Nagoya Institute of Technology, supported RV-001 development. About Restore Vision Restore Vision Inc., a clinical-stage gene therapy company, specializes in developing transformative gene therapies for inherited retinal disorders. By integrating academic excellence with entrepreneurial vision, the company is committed to pioneering treatments that address critical unmet medical needs and improve patient outcomes worldwide. Media Contact Hikaru Miyazaki COO, Restore Vision Inc. Email: [email protected] SOURCE Restore Vision Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED