INT41

INT41 is an experimental gene therapy and intrabody candidate developed by Vybion for the treatment of Huntington's Disease. It consists of a humanized single-chain variable fragment (scFv) antibody, or intrabody, that is designed to be expressed within the cell or delivered via exosomes. INT41 specifically binds to the N-terminal fragments of the mutant Huntingtin (mHtt) protein, particularly the proline-rich region. By binding to these toxic fragments, INT41 prevents the formation of protein aggregates and facilitates the clearance of mHtt from the cytoplasm and nucleus of neurons. The therapy is delivered using viral vectors such as adeno-associated virus (AAV) or through exosome-mediated delivery systems to achieve effective intracellular targeting in the central nervous system.

Other names

INT41 intrabodyINT-41 intrabodyINT 41 intrabody