• Therapeutics
  • Immune-Oncology

Chronic myeloid leukemia (CML)

  • Discovery
  • Mode-of-Action
  • Preclinical
  • IND-Application
  • Phase 1
  • Phase 2
  • Phase 3

Sources of funding:

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Research and Mode-of-Action: European Research Council Advanced Grant MaCChines 

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Preclinical translation: European Research Council Proof-of-Concept

External Collaborations:

We performed advanced preclinical research on human cells with: 

Ljubljana University Medical Centre, Department of Haematology

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  • 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.  

Bcr Abl1

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). 

Survival Rate
H4 Smalensek

Intellectual property

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

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