Canary / Canario

Cell Engineering and Molecular Pharming for Biopharmaceuticals

“…This may have potential application in treating disease states which require blocking of gene expression for curing effect [ 2 ]. However, antisense DNA technology has failed to live up to its early hype, as most standalone antisense companies folded [ 7 ]. The downfall of antisense has been attributed largely to its off-target effects, especially the tendency of nucleic acid sequences to induce generalized immune responses, as well as the difficulty of delivering the therapies to the right cell types.…”

“…Another nucleic acid-based therapy, short interfering RNA (siRNAs) or microRNAs (miRNAs) or RNAi therapies, have been hailed as the hallmark of new frontiers in biotechnology [ 7 ]. These are small noncoding RNAs that regulate gene expression by repressing translation of target cellular transcripts.…”

“…miRNAs can induce a cell to destroy complementary pieces of mRNA, preventing the target message from being transcribed. This RNA inhibition can be exploited to inhibit any gene of interest and may be particularly useful in gene therapies [ 2 , 7 , 87 ]. The major challenges with developing RNAi therapies also involve delivery to the target site, and cross-talk in signaling pathways.…”

“…The properties of miRNA-regulated vectors should allow for safer and more effective therapeutic applications [ 88 ]. This fast target validation and easy synthesis of miRNAs are attracting attention from drug developers, as a potential multi-billion dollar therapeutic platform in the next few years [ 7 ].…”

“…The growing confidence and interest in biopharmaceuticals has pushed big pharma companies to acquire technologies or invest in manufacturing facilities. Merck has bought RNAi developer, Sirna Therapeutics for $1.1 billion (RNAi being short interfering molecules to inhibit any gene of interest in any cells) [7]. Genentech has invested $140 million to set-up microbial-based manufacturing operations for biotherapeutics in Asia [8].…”