Novel Platform Technology for Developing Therapeutic Human mAbs

DESCRIPTION (provided by applicant): Despite significant advances in treatment and early detection, cancer remains a significant disease threat to society. Therapeutic monoclonal antibodies are effective new treatments that have recently been developed as targeted therapies against many types of cancer. Over 17 monoclonal antibodies have been approved in the US for treating a variety of diseases. The advantages are high specificity, predictable side effects, and more rapid and less expensive advancement through clinical trials. Significant hurdles remain, however, including the low efficiency of antibody development, instability of hybridoma cell lines, adverse patient immune responses to foreign protein or carbohydrate sequences, and commercially, royalty stacking issues from the need to access the multiple technologies required to develop an efficacious antibody therapeutic. There is currently no technology platform that allows one to develop human monoclonal antibody producing cell lines by direct transformation of human immune cells. Such antibodies can be tested for specific therapeutic characteristics and the cell lines directly scaled for larger antibody manufacturing without the use of additional platform technologies. The approach described herein is one that allows for more rapid discovery of high value therapeutic products, that will both personalize and revolutionize the therapeutic human monoclonal antibody as the new era of genomics and proteomics continues to provide enormous opportunity for the discovery of druggable targets. The objective of this Phase I proposal is utilize the ABL-MYC retroviral transformation technology for the development of fully human monoclonal antibodies. NeoClone has utilized this approach commercially since 1999 to develop murine antibodies for research and diagnostic use. If successful in this proposal, the advantages of our murine antibody technology would hold true: rapid, efficient, cost effective development of high affinity monoclonal antibodies. Additional advantages for therapeutics would be fully human antibody sequence, stable cell lines, accurate post-translational modifications and no need for further maturation to improve affinity. Additionally, this technology would have the potential to use the patient's own cells for antibody development. A more rapid and efficacious therapeutic antibody technology would be a welcome addition to the arsenal of approaches to combat cancer and other deadly diseases. Therapeutic monoclonal antibodies are emerging as one of the most effective targeted drug approaches for cancer. NeoClone is proposing to adapt it's successful monoclonal antibody technology to enable rapid development of fully human therapeutic monoclonal antibodies. The potential advantages of this approach are more rapid and efficient drug development with less potential for deleterious side effects.