Idera Pharmaceuticals recently presented pre-clinical data regarding the gene-silencing mechanisms of its third generation antisense (3GA) technology platform, a potential therapeutic for diseases that include, but are not limited to, facioscapulohumeral muscular dystrophy (FSHD).
The presentation, “Precise excision of targeted RNA by third generation antisense (3GA) oligonucleotides” was made at the recent Cold Springs Harbor Laboratory Conference on Regulatory & Non-Coding RNAs.
Diseases like FSHD are believed to be caused by impairments or mutations in certain genes. Correcting these mutations and the mechanisms of action involved in these processes are mostly still in development stages.
Antisense technology is a type of treatment for genetic disorders like FSHD. Because the genetic sequence of certain genes is known to cause these diseases, the synthesis of a strand of nucleic acids, such as DNA, RNA, or similar chemical agents, can turn off or inactivate defective genes. 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).
Idera researchers now have a better understanding of the mechanisms involved in the company’s antisense technology. In the presentation, they demonstrated that gene-silencing by 3GAs led to excision sites in targeted mRNA (messenger RNA, a conveyer of DNA information) in the region similar to that observed with siRNA (a type of RNA that silences, or shuts down, genes), but resulted in different products than those observed with earlier generations of antisense.
The company is also looking at the potential of 3GAs in targeting diseases caused by point mutations.
“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 Pharmaceuticals, 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.”
The company announced that it is investigating recently identified potential gene targets for FSHD and other diseases, including lupus nephritis, uveitis, and interstitial cystitis, with the intent of starting clinical studies in 2017. Idera said it also produced 3GA agents for a series of additional gene targets earlier this year.