Sources of funding:
Research and Mode-of-Action: European Research Council Advanced Grant MaCChines
Preclinical translation: European Research Council Proof-of-Concept
We performed advanced preclinical research on human cells with:
Ljubljana University Medical Centre, Department of Haematology
- assoc. prof. Matjaž Sever MD, PhD
- Veronika Mikolič
Looking for clinical-stage partners with secured CRISPR licensing in the field of oncology
About the condition
Chronic myeloid leukemia (CML) is a myeloproliferative neoplastic disease.
The diagnosis is based upon the genetic translocation between the t(9;22)(q34;q11.2), resulting in the formation of Philadelphia fusion chromosome, coding for BCR-ABL1 oncoprotein.
This condition occurs during the person’s life and is not inherited. It has three phases; chronic phase (the majority of diagnosed cases), accelerated phase and blast phase.
The life-long treatment relies on tyrosine kinase inhibitors (TKIs). Nearly 2% of patients develop point mutations, leading to the resistance to TKIs treatment. New solutions for treating cancer with genetic etiology are required.
The results and solution that Our Science offers
- Efficient application of CC-EXO to treat K562 tumor in an animal model targeting BCR-ABL translocation. Tumors in SCID mice were electroporated with plasmids carrying CC-EXO + gRNA. We observed the weight and the survival rate (increased).
Priority date EP patent application, September 2019
PCT phase, currently
Lainšček D, Forstnerič V, Malenšek Š, et al P09.06 ‘An enhanced CRISPR tool for treating chronic myelogenous leukemia’ Journal for ImmunoTherapy of Cancer 2020;8:doi: 10.1136/jitc-2020-ITOC7.106