Therapeutic target identification for Myeloproliferative Neoplasms
Research type
Research Study
Full title
Identification of targets for the treatment of myeloproliferative neoplasms and other haematological diseases
IRAS ID
369058
Contact name
Edward Ainscow
Contact email
Sponsor organisation
Alethio Therapeutics Ltd.
Duration of Study in the UK
2 years, 11 months, 30 days
Research summary
Alethio Therapeutics is developing novel therapeutics for the treatment of myeloproliferative neoplasms (MPN), a group of blood cancers driven by acquired genetic alterations in haematopoietic stem and progenitor cells. While recurrent driver mutations are well characterised, there remains substantial genetic and molecular heterogeneity between patients, indicating the presence of additional disease-relevant targets.
The aim of this project is to identify and prioritise new therapeutic targets through the analysis of genomic data generated from patient-derived samples from individuals with MPN. This will include the analysis of DNA and/or RNA using approaches such as whole exome sequencing, targeted sequencing, and transcriptomic profiling. These analyses will be used to identify recurrent genetic alterations, dysregulated pathways, and molecular dependencies that may inform future drug discovery efforts. Genes or pathways identified through sequencing analyses will be validated using independent confirmation methods, such as quantitative PCR, flow cytometry, or immunohistochemistry, to confirm findings at the RNA and/or protein level.
Alethio Therapeutics will not recruit patients or collect biological samples directly. Patient-derived samples will be obtained from academic collaborators, biobanks, or commercial providers, where samples and associated data have been collected under appropriate ethical approval and informed consent permitting research use by commercial entities. A sufficient number of samples will be used to support both discovery and confirmation experiments.
Patient-derived cells may be maintained in vitro using appropriate culture systems, including conditions designed to model aspects of bone marrow or disease biology. Cells may be exposed to defined stimuli, treatments, or experimental conditions prior to molecular analysis. Genomic profiling may include bulk or single-cell sequencing approaches.
The outputs of this research will be aggregate and hypothesis-generating, intended to guide downstream laboratory validation and therapeutic development. This work will not involve clinical decision-making, return of individual results to patients, or any direct impact on patient care.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
26/NW/0178
Date of REC Opinion
17 Jun 2026
REC opinion
Favourable Opinion