Calrecticulin R (CalR) is a highly conserved intracellular protein with multiple functions in human cells. Mutations in exon 9 of the CalR gene is seen in a significant proportion of patients with Myeloproliferative Neoplasms (MPN), either alone or in combination with mutations in the Janus Kinase 2 (JAK2) gene. Prevention of subsequent malignancies through the elimination of prefiltration myeloid cells with the CalR mutation is believed to be an efficient treatment strategy in MPN.
Our proprietary IO160 program is a first-in-class vaccine containing a single CalR-derived peptide designed to engage cytotoxic T-cells specific for the mutation in exon 9 of CalR.
IO160 is being tested in a single clinical trial:
- A single arm phase I clinical trial in MPV patients with CalR exon 9 mutations (NCT03566446).
The calreticulin (CALR) exon 9 mutations are promising targets for cancer immune therapy.
Leukemia, February 2018