We all know well that liver is *cleaning* organ, prone to suck everything bad in circulation. IF one design pro-drug/drug with ligand that can be recognized by cell membrane transporters....liver targeting molecule is not far away. ALNY capitalize on this approach, with chem-modified siRNA.
Will this work on CNS and/or eye cells? REGN/ALNY have sufficient scientific knowledge to help each others in drug design/discovery.
However, this does not negate gene-approach in tretman of the rare/specific eye conditions. If one have missing/faulty gene, correction by gene-therapy approach is valid one. Also, if secreted protein (by gene insertion) is antagonist already in use. So, REGN/ADVM collaboration stand until is terminated. Also, REGN may be interested by RGNX efforts,....as DEW indicated, they do not want be captured/stuck in limbo. They need to growth organically revenue outside eylea.
Because wast majority of this inside-cell targets can not be approached by Abs, they need small molecule/others collaborations to capitalize on RGC efforts/data.
PS:REGN escaped Avalance failure with initial IR-drug, for new candidate they are on *watch* mode, IMO. |
