Innovir And SKI To Develop Leukemia Drugs

21 August 1994

Innovir Laboratories, a US biotechnology company formed in 1989 to develop ribozyme-based drugs for various disorders, has entered into an agreement with the Sloan Kettering Institute for Cancer Research for the preclinical evaluation of a new approach to treating acute promyelocytic leukemia.

The goal of the collaboration is to develop a ribozyme-based therapeutic to induce the maturation and differentiation of leukemic white blood cells, thereby restoring them to normal functioning. Under the terms of the deal, SKI researchers will evaluate the in vitro and in vivo efficacy of certain proprietary, site-specific segments developed by Innovir, which are known as external guide sequences. Innovir's founding scientists have discovered that the binding of EGS's to ribozymes could redirect the ribozymes to cleave target disease-causing RNA sequences and render them inactive, restoring the cancerous cells to a benign, non-proliferative state.

APL has been linked to a chromosomal translocation that leads to overproduction of abnormal immature leukocytes. Conventional treatments have relied on cytotoxic chemotherapy or retinoic acid, a therapy which induces differentiation in immature cells. However, patients nearly always develop resistance to retinoic acid and relapse. Innovir and SKI hope that Innovir's ribozyme technology will provide an alternative to chemotherapy and retinoic acid for APL and possibly other types of cancer.

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