Therapeutic development for treatment of Myeloproliferative Neoplasms

  • Research type

    Research Study

  • Full title

    Development of therapeutics for the treatment of myeloproliferative neoplasms

  • IRAS ID

    359666

  • Contact name

    Edward Ainscow

  • Contact email

    ed.ainscow@alethiotx.com

  • Sponsor organisation

    Alethio Therapeutics Ltd

  • Duration of Study in the UK

    3 years, 0 months, 1 days

  • Research summary

    Alethio Therapeutics is seeking to develop therapeutics for the treatment of a set of blood cancers known as myeloproliferative neoplasms (MPN). These set of diseases are characterised by mutations in the JAK2, CALR and MPL genes in haematopoietic stem cells which lead to the overproduction of myeloid cells (platelets, red blood cells, megakaryocytes etc.)
    Alethio Therapeutics have identified specific markers of stem cells carrying the disease causing mutations and are using these markers as a means of selectively killing those cells. We are using antibodies and antibody drug conjugates in order to achieve this.
    The current project is to develop and optimise these therapeutics so that they can be considered as candidates for a clinical trial. In order to demonstrate efficacy and safety of our molecules we plan to test on primary human tissues, in addition to derived cell lines. Included in this will be blood derived material such as platelets and mononuclear cells from which we will test the mechanism of action of our molecules. Additional testing will include samples taken from patients (sourced from commercial or academic research project with REC approval covering their use) which will be cultured in vitro during testing.

  • REC name

    North West - Preston Research Ethics Committee

  • REC reference

    26/NW/0075

  • Date of REC Opinion

    2 Apr 2026

  • REC opinion

    Favourable Opinion