Property of Pepscan Systems BV
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A Novel Class of Transport Peptides for Enhanced Drug Delivery
Pepscan Systems' XMTM
A major challenge in today's biotechnological drug development is the fact that, on one
hand, many recombinant products such as bioactive proteins, enzymes, and antibodies,
and on the other hand, antisense oligonucleotides and gene constructs, have inherent
unfavorable pharmacokinetic properties due to their physicochemical nature. None of
these molecules have the ability to readily penetrate cell membranes and the body's
physiological barriers to reach their pharmacological targets in the body. This problem
also applies to many conventional "small molecule" drug candidates. The phenomenon
usually leads to low in vivo "effective availability", that is the amount of drug that
eventually reaches its site of action, a problem that either makes otherwise promising
molecules unsuitable for drug therapy or, alternatively, leads to approaches that try to
circumvent the problem by using uneconomically high dose levels (with an inherent
safety and tolerability risk) often combined with patient unfriendly routes of
administration and complicated therapeutic regimens.
Pepscan's novel delivery system XMTM consists of a modified and optimized peptide,
that originates from the E
rns
viral surface protein to which Pepscan's proprietary peptide
optimization technology was applied. We have demonstrated in several experimental
models that the XMTM molecule is able to translocate a conjugate consisting of XMTM
peptide and molecular cargo such as a large protein or oligomer across cell membranes.
Figure 1 depicts the in vitro uptake of functional
-Galactosidase tetramer, a protein
complex with a total molecular weight of 550 kDa, which is otherwise unable to
penetrate cell membranes due to its size and hydrophilic nature.
