Download New Platform Technology for the Development of Therapeutic

New Platform Technology for the Development of Therapeutic Proteins
and Antibodies
Yong-Sung Kim
Dept. of Molecular Science and Technology, Ajou University, Suwon 443-749, Korea.
Tel: +82-31-219-2662; Fax: +82-31-219-2394; E-mail: [email protected].
In this talk, I would like to introduce our efforts to establish new platform technology
for the development of therapeutic proteins and antibodies, which have been established in our
group for the last several years. Those are 1) Novel protein scaffold development as an
alternative to antibody and 2) Interfering transbody, which can penetrate into living cells and
selectively degrade targeted mRNA to resulting in gene-silencing effect.
For the first topic, I will introduce a novel non-antibody protein scaffold based on
human Kringle domain.
Due to the limits of mAbs in terms of stability, solubility,
manufacturing cost, intellectual property, etc., alternative non-antibody scaffolds have been
developed to have ability to specifically bind to given targets, like antibody, but overcome the
limits of antibody. We have developed Kringle domain variants to function as agonists against
TRAIL receptors, death receptor 5 (DR5) and 4 (DR4) as well as antagonists against tumor
necrosis factor-alpha. I will deliver the biochemical/biophysical properties of Kringle domain
variants and the in vitro and in vivo biological efficacy.
For the second topic, I will introduce a novel proof-of-concept technology, we termed
as 'interfering transbody', in which a cell-penetrating antibody (transbody) specifically
hydrolyzes targeted mRNAs in the cellular cytosol and leads to targeted gene silencing.
Targeting particular mRNAs for degradation is a fascinating approach to achieve gene silencing.
I will convince you that interfering transbody could have potential applications in anti-cancer or
anti-viral therapies.