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Rare Diseases

  • Clinical Trials,Drug Development,Rare Diseases

    Cereno Scientific’s CS014 shows encouraging phase 1 results in idiopathic pulmonary fibrosis

    Cereno Scientific, a biotechnology company pioneering treatments for rare cardiovascular [...]

    July 17, 2025
  • Artificial Intelligence,Clinical Development,Clinical Trials,Drug Development,Rare Diseases

    AI validation of rentosertib efficacy supports global trial expansion in IPF

    Qureight’s deep-learning biomarkers confirm representativeness of Insilico’s Phase 2a IPF [...]

    July 16, 2025
  • Clinical Development,Patient Centricity,Rare Diseases

    OMED Health launches new gastrointestinal treatments and services through personalized health plans

    OMED Health, a UK-based company developing non-invasive breath analysis tools [...]

    July 15, 2025
  • Clinical Development,Clinical Trials,Drug Development,Rare Diseases

    Heidelberg Pharma advances dual ATAC pipeline, secures $20M payment and eyes 2027 funding runway

    Lead multiple myeloma candidate enters Cohort 8; second asset dosed [...]

    July 14, 2025
  • Clinical Trials,Drug Development,Rare Diseases

    Cereno taps global CRO for next big step in pulmonary hypertension drug trial

    Selection of CRO marks key milestone in advancing CS1 as [...]

    July 7, 2025
  • Biologics & Biosimilars,Clinical Trials,Drug Development,Rare Diseases

    Eleva doses first volunteers with new therapy targeting rare kidney disease

    Dosing of CPV-104 in healthy volunteers marks key milestone in [...]

    July 7, 2025
  • Cell & Gene Therapy,Clinical Trials,Rare Diseases,Regulatory Affairs

    First-in-class cell therapy for rare mitochondrial disorder gains FDA fast track

    FDA recognition accelerates development of first-in-class mitochondrial cell therapy for [...]

    July 4, 2025
  • Clinical Development,Drug Development,Precision medicine,Rare Diseases,Technology and platforms

    Rewind Therapeutics strengthens Scientific Advisory Board with leading neurology experts as it advances remyelination therapy

    Appointments enhance clinical expertise as GPR17 modulator progresses toward trials [...]

    July 2, 2025
  • CDMOs & Manufacturing,Drug Development,Partnerships & Funding,Rare Diseases

    Shionogi and BioVersys agree €498M licensing deal for NTM drug candidates

    Deal gives Shionogi access to BioVersys’ BV500 program targeting serious [...]

    July 2, 2025
  • Biologics & Biosimilars,Clinical Development,Drug Development,Rare Diseases

    Takeda’s mezagitamab shows rapid, durable platelet response in Phase 2 ITP trial

    Data presented at ISTH 2025 highlight potential for improved outcomes [...]

    July 3, 2025
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