1. Rocket Pharmaceuticals, Inc. announced the progress of phase 2 clinical trials for Fanconi Anemia treatment and Phase 1 clinical trials for Danaon Disease and PKD treatment at ASGCT. The two therapeutics are lentiviral vector and adenoviral vector-based gene therapy, respectively, and have shown positive results.

    Rocket Pharmaceuticals, Inc가 Fanconi Anemia 치료제 임상 2상, Danaon Disease와 PKD 치료제의 임상 1상 진행 현황을 ASGCT에서 발표했습니다. 두 가지 치료제는 각각 lentiviral 벡터와 adenoviral 벡터 기반 유전자 치료제며, 현재까지 긍정적인 결과를 보이고 있습니다.
    Original Article: Bakersfield. Rocket Pharmaceuticals Presents Positive Clinical Data from Danon Disease, Fanconi Anemia and Pyruvate Kinase Deficiency Programs at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

  2. Takeda eventually gave up developing a drug for Hunter syndrome. After the acquisition of Shire, the development of a treatment that Shire has been developing has been pursued with a target of market approval in 2022, but failed to gather sufficient data. The FDA recently rejected Takeda’s esophageal inflammatory disease treatment, TAK-994, and pevonedistat.

    Takeda가 헌터 증후군 치료제 개발을 결국 포기 했습니다. Shire가 개발하던 치료제를, Shire 인수 이후 2022년 시장 승인 목표로 개발을 추진했지만, 충분한 데이터를 마련하는 데 실패했습니다. FDA는 최근 다케다의 헌터 증후군 치료제뿐 아니라, esophageal inflammatory disease, TAK-994, pevonedistat 승인을 거절 한바 있습니다.
    Original Article: Fierce biotech. Takeda scraps dream of getting rare disease drug to market in latest setback to Wave 1 pipeline

Rare disease diagnostics & research

  1. 3billion was selected as a business operator for the national task of developing software for diagnosing rare pediatric diseases. 3billion plans to form a consortium with Seoul National University Hospital and Kakao Healthcare to develop an AI integrated software solution for multifaceted analysis of rare diseases in nine groups for three years from 2022 to 2024. 3billion will be in charge of developing WGS (Whole Genome Sequencing)-based diagnostic AI models and SW solutions.
    The hospitals in charge of R&D are 5 hospitals, including Seoul National University Hospital and Samsung Seoul Hospital. The SW to be developed will be verified in 13 hospitals, including 8 clinical verification network hospitals.

    쓰리빌리언이 소아희귀질환 진단 SW 개발 국가 과제 사업자로 선정 되었습니다. 쓰리빌리언은 서울대병원, 카카오헬스케어 등과 함께 컨소시엄을 이뤄, 2022년부터 2024년까지 3년간 9개 희귀질환군의 ‘소아희귀질환 다면분석 AI 통합 소프트웨어 솔루션’을 개발할 예정입니다. 쓰리빌리언은 WGS(Whole Genome Sequencing) 기반 진단 인공지능 모델 및 SW 솔루션을 개발하는 역할을 담당하게 되었습니다.
    연구개발 주관 병원은 서울대병원, 삼성서울병원등 5개 병원입니다. 개발된 SW는 임상 검증 네트워크 병원 8곳을 포함해 총 13개 병원에서 검증될 예정입니다.
    Original Article: Medigate News. 쓰리빌리언, 소아희귀질환 인공지능 SW 솔루션 개발 사업자 선정