Targeting Mononuclear & CD34+ cells by in vitro generated platelets
Research type
Research Study
Full title
Targeting commercial, fully anonymised Mononuclear and CD34+ cells for modification and assessing efficacy of in vitro generated platelets.
IRAS ID
342312
Contact name
Aurore Saudemont
Contact email
Sponsor organisation
Xap Therapeutics Ltd
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
Current biological therapies present safety challenges such as adverse reactions and off-target effects including unintended immune responses. Xap Therapeutics aim to mitigate this with targeted and personalised treatment of various disorders, using engineered platelets. Studies have already shown potential for engineering platelets to carry therapeutic agents. Xap have engineered platelets in vitro from induced pluripotent stem cells (iPSCs) and have engineered these cells to be capable of conditional activation to a specific target and subsequent delivery of a variety of therapeutic cargo. To develop this therapeutic platform further, Xap require the use of relevant target cells including haematopoietic stem cells (HSCs) and Mononuclear cells (MCs) with haemopoietic lineage.
Haematopoietic stem cells produce all blood cell components. By modifying HSCs, Xap Therapeutics plan to measure the success of their therapeutic platform using techniques such as gene editing analysis and cellular assays. In addition, the use of Mononuclear cells (such as Peripheral blood mononuclear cells, PBMCs) will focus on i) demonstrating our engineered platelets to deliver a variety of therapeutic strategies to Mononuclear cells, and ii) using these haemopoietic-lineage cells as a positive control against the engineered platelets in the various assays undertaken.
Xap Therapeutics is therefore applying for the use of commercially sought HSCs (CD34+ cells) and Mononuclear cells (MCs) such as Peripheral blood mononuclear cells (PBMCs), peripheral monocyte or CD4+ cells, for this purpose. Strict adherence to protocols will be ensured for sample handling, storage, and data protection, to maintain confidentiality and comply with regulations. Once satisfied with the efficacy level in vitro, Xap Therapeutics plan to test their strategy in vivo (animal models), for example, by using murine immunodeficient mice. Collaboration with Contract Research Organisations (CROs) will occur for such in vivo studies.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
24/SC/0163
Date of REC Opinion
5 Jun 2024
REC opinion
Further Information Favourable Opinion