Calimmune has become the latest RNAi drug developer to move into the clinic, recently initiating a phase I/II trial of its HIV therapy Cal-1.
The therapy is based on the work of UCLA AIDS Center and California Institute of Technology researchers, who discovered that the chemokine receptor CCR5 was used by the virus to infect cells.
With the approach, patients’ T cells and stem cells are isolated from peripheral blood, then treated with the HIV-1 fusion inhibitor C46, as well as a self-inactivating lentiviral vector encoding short hairpin RNAs against CCR5. The cells are then re-infused, with the treated T cells providing immediate protection against the virus and the stem cells offering long-term protection.
The clinical study is expected to enroll 12 patients, who will be divided into three cohorts. The first group will receive Cal-1 without any preconditioning with busulfan, a chemotherapeutic used during bone marrow transplantation procedures.
The second and third cohorts will receive either one or two initial doses of busulfan prior to Cal-1 treatment.
The phase I/II trial is slated to wrap up in mid-2015.