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Department of Gene Therapy
Outline of the Department of Gene Therapy
The Department of Gene Therapy is a social cooperative course established on February 1, 2021 in the Graduate School of Medicine, Nippon Medical School with the support of an American company, Aruvant Sciences.
As the name of the laboratory indicates, we are engaged in research and development of gene therapy, and are currently developing new therapies (regenerative medicine product: gene therapy drugs) for hypophosphatasia.
Topics & News
December 5, 2023 " Development of adeno-associated virus vector-mediated gene therapy for inherited diseases " was presented at the SNBL Science Seminar.
September 11-13, 2023 "Efficacy and Safety of a Novel Therapeutic Agent (Gene Therapy drug: ARU-2801) for the Treatment of Hypophosphatasia" was presented at the 29th Annual Meeting of the Japan Society of Gene and Cell Therapy.
August 1, 2023 The paper " Gene Therapy for Hypophosphatasia in Alpl-/- mice (infantile HPP model)―Toward Clinical Application" was published.
July 8, 2023 "Efficacy of a Novel Therapeutic Agent (Gene Therapy drug: ARU-2801) for the Treatment of Hypophosphatasia" was presented at the 15th ALPS Research Conference.
July 8, 2023 "Safety of a Novel Therapeutic Agent (Gene Therapy drug: ARU-2801) for the Treatment of Hypophosphatasia" was presented at the 15th ALPS Research Conference.
July 7, 2023 Our project was featured in the British Open Journal "Open Access Government".(doi: 10.56367/OAG-039-10951)
June 2-4, 2023 "Development of gene therapy drug (ARU-2801) for HPP" was presented at 2nd Soft Bones International Scientific Meeting 2023.
June 2-4, 2023 "Possibility of prenatal gene therapy for lethal HPP model mice" was presented at 2nd Soft Bones International Scientific Meeting 2023.
May 16-20, 2023 " Efficacy and safety of preclinical gene therapy for hypophosphatasia using ARU-2801 (AAV8 vector expressing TNALP-D10)" was presented at The American Society for Gene and Cell Therapy (ASGCT) 26th Annual Meeting.
April 30, 2023 Handbook for Gene Therapy Research and Development, 2nd Edition, "Hypophosphatasia" was published.
April 1, 2023 The paper "Development of gene therapy for hypophosphatasia" was published. (doi: 10.21820/23987073.2023.2.59)
March 25, 2023 The paper "Gene therapy for Hypophosphatasia" was published.
January 10, 2023 The paper "Development of a novel gene therapy drug for Hypophosphatasia" was published.
November 15, 2022 The paper " Gene Therapy Using Recombinant AAV Type 8 Vector Encoding TNAP-D10 Improves the Skeletal Phenotypes in Murine Models of Osteomalacia" was published. (doi: 10.1002/jbm4.10709)
December 14-17, 2022 " Development of effective and safe gene therapy drug (ARU-2801) for hypophosphatasia" was presented at 67th Annual Meeting of the Japan Society of Human Genetics.
November 30, 2022 The paper "Development of a novel gene therapy drug for hypophosphatasemia" was published.
November 14-16, 2022 " Evaluation of efficacy and safety of a novel gene therapy drug (ARU-2801) for hypophosphatasia in non-human primates " was presented at The Society for Endocrinology BES 2022. (SFE BES 2022).
November 1-3, 2022 " Development of an adeno-associated viral gene therapy drug (ARU-2801) intended for hypophospnatasia" was presented at The 55th Annual Scientific Meeting of the Japanese Society for Pediatric Endocrinology. (JSPE 2022)
October 28, 2022 " Current Status and Issues in Gene Therapy Drug Development - Focusing on Toxicity-" was presented at The 8th Annual Meeting of HAMAMATSU TOXICOLOGY STUDY FORUM.
October 11-14, 2022 " Validity and safety preclinical in vivo study of gene therapy for hypophosphatasia using ARU-2801 (AAV8 vector expressing TNALP-D10)" was presented at The 29th Annual Meeting of the European Society of Gene & Cell Therapy. (ESGCT 2022)
September 25, 2022 The paper "Development of a novel gene therapy drug (ARU-2801) for hypophosphatasemia" was published.
September 9-12, 2022 "Non-human primate in vivo study of ARU-2801, an investigational adeno-associated viral gene therapy intended for hypophosphatasia, shows durable transgenic plasma alkaline phosphatase levels are achievable without any toxicities" was presented at the The American Society for Bone and Mineral Research (ASBMR) 2022 Annual Meeting. (ASBMR 2022)
September 9-12, 2022 " Gene therapy using recombinant AAV serotype 8 encoding TNAP-D10 improves the skeletal phenotype in a late-onset HPP mouse model" was presented at the The American Society for Bone and Mineral Research (ASBMR) 2022 Annual Meeting. (ASBMR 2022)
September 9-12, 2022 " Gene therapy using recombinant AAV serotype 8 encoding TNAP-D10 improves the skeletal phenotype in the PHOSPHO1 deficient mouse model" was presented at the The American Society for Bone and Mineral Research (ASBMR) 2022 Annual Meeting. (ASBMR 2022)
July 14-16, 2022 "Investigation of the Efficacy and Safety of a Gene Therapy Drug (ARU-2801) for Hypophosphatasia" was presented at the 28th Annual Meeting of the Japan Society of Gene and Cell Therapy.
June 30-July 2, 2022 "Current Status and Issues of Safety Evaluation of Gene Therapy Drugs (Symposium: Development of Gene/Cell Therapy Drugs and Their Safety Evaluation)" was presented at the 49th Annual Meeting of the Japanese Society of Toxicology.
June 10, 2022 The paper "Development of a gene therapeutic drug (ARU-2801) for hypophosphatasemia" was published.
November 8-10, 2021 "Successful adeno-associated virus mediated neonatal gene therapy treatment of hypophosphatasia murine model resulted in bone maturation and increased survival to at least 18 months" was presented at the 28th Annual Meeting of the the Society for Endocrinology BES 2021 (SfE BES 2021).
October 1-4, 2021 "Successful adeno-associated virus mediated neonatal gene therapy treatment of a murine model of infantile hypophosphatasia resulted in bone maturation and increased survival to at least 18 months" was presented at the The American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting. (ASBMR 2021)
October 15, 2021 The paper "Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A" was published.（doi: 10.1038/s41598-021-99979-2）
June 15, 2021 The paper " Gene Therapy Using Adeno-Associated Virus Serotype 8 Encoding TNAP-D 10 Improves the Skeletal and Dentoalveolar Phenotypes in Alpl -/- Mice" was published.（doi: 10.1002/jbmr.4382）
June 15, 2021 The paper " Treatment with bone maturation and average lifespan of HPP model mice by AAV8-mediated neonatal gene therapy via single muscle injection" was published. (doi: 10.1016/j.omtm.2021.06.006)
February 1, 2021 The Department of Gene Therapy was established.