Idera Pharmaceuticals recently presented pre-clinical data on the mechanisms of action behind its antisense (3GA) technology platform, a gene silencing technology whose potential disease indications include, but are not limited to, lupus nephritis.
Idera’s 3GA technology specifically silences disease-causing genes using small molecules called oligonucleotides, which offer an improvement over a similar technique called inhibitory RNA (RNAi). In the presentation, “Precise excision of targeted RNA by third generation antisense (3GA) oligonucleotides,” Idera scientists released data showing that gene-silencing by 3GAs led to excision sites in targeted mRNA in the region similar to that observed with siRNA, but resulted in different products than those observed with earlier generations of antisense.
The presentation, made at the recent Cold Spring Harbor Laboratory meeting on Regulatory and Non-Coding RNAs, also showed that 3GA acts very specifically to silence genes.
Based on their results, Idera researchers are currently working to further understand how 3GA can be used to target specific diseases, focusing on what are known as point mutations — mutations that influence only a very limited portion of a gene. The data will be available in the second half of 2016.
“Our in-depth understanding from our pioneering work in antisense technology, along with our insights into the interaction of nucleic acids with Toll-like receptors, has allowed us to design this very unique technology platform to fully realize the potential of antisense technology,” Sudhir Agrawal, president of Research at Idera, said in a press release. “We are continuing to conduct preclinical studies with multiple 3GA candidates in house and with our collaborators, with a goal of advancing this technology to clinical development.”
Candidate gene targets that Idera has identified include NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4). Potential diseases that could be treated using 3GA technology include, interstitial cystitis, lupus nephritis, uveitis, and facioscapulohumeral muscular dystrophy (FSHD). The technology potentially could also be used to treat several other diseases.