Therapies for Inherited Neuropathies team meets a key milestone and advances the development of therapeutics antibodies for the treatment of CMT1X.
The laboratories of Rudolf Martini and Richard Stanley have previously reported the discovery of a novel biological pathway that is part of the immune system response to tissue damage in the peripheral nervous system. A factor that is active in this pathway has proven to be a specific modulator of this response, and for the amplification of the tissue injury that results. This factor accelerates the disease pathology of CMT1X and other related CMT neuropathies. They have been joined by the laboratory of Jonathan Lai in a TIN-sponsored collaborative effort to design an antibody therapy that neutralizes the disease-amplifying factor.
Previous efforts by the team to isolate the factor, and engineer an animal model to test a potential antibody therapy, were successful and further enabled the creation of libraries of candidate antibodies and the selection of therapeutic candidates. The team now reports achieving a key milestone in proceeding to the selection of antibodies that meet the criteria of clinical developability.
This is a very exciting approach that takes advantage of biopharmaceutical engineering technologies that are currently responsible for 30% or more of drugs annually approved by the US FDA. Read more about the laboratories of Lai, Martini and Stanley.