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.
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.
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
Rare disease pharmaceutical industry trendsIonis Pharmaceutical and AstraZeneca announced that they had succeeded in Phase 3 clinical trial of co-developing transthyretin amyloidosis treatment(eplontersen). However, the…