Idera Pharmaceuticals Presents Novel Mechanism of Action of its Third- Generation Antisense Technology

Idera Pharmaceuticals, Inc. recently announced the presentation of new preclinical data that demonstrates the novel gene-silencing mechanism of action of the third-generation antisense (3GA) technology platform.

In the presentation, titled Precise Excision of Targeted RNA by Third-Generation Antisense (3GA) Oligonucleotides, Idera scientists presented data that demonstrated that gene-silencing by 3GAs led to excision sites in the targeted mRNA in the region similar to that observed with siRNA. These excision products are different from those observed with earlier generations of antisense. This presentation also provided a demonstration of 3GA’s specificity by showing that the incorporation of a mismatch at the region of excision led to the loss of gene-silencing activity. Based on these studies, the company is also conducting studies to further the potential applications of 3GAs in targeting diseases caused by point mutations. Data from these studies is expected to be presented in the second half of 2016.

This presentation is currently available on Idera’s website at http://www.iderapharma.com/our-science/key-presentations-and-publications.

“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,” said Sudhir Agrawal, DPhil, President of Research at Idera Pharmaceuticals. “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.”

Previously the company has announced the identification of NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4) as initial gene targets to advance into IND-enabling activities, which will occur throughout 2016. Potential disease indications related to these targets include, but are not limited to, interstitial cystitis, lupus nephritis, uveitis, and facioscapulohumeral muscular dystrophy (FSHD). The company is currently conducting clinical, regulatory, and commercial analysis activities and conducting IND-enabling studies with the plan to enter the clinic in 2017 for the first clinical development program. In addition to these activities, over the first half of 2016, Idera generated 3GA compounds for a series of additional gene targets. These will enable the company to continue to expand its future pipeline opportunities for both internal development as well as partnerships in areas outside of Idera’s focus. Additionally, Idera is party to a collaboration and license agreement with GSK to research, develop, and commercialize compounds from its 3GA technology for the treatment of undisclosed, selected renal targets.

Idera’s proprietary third-generation antisense (3GA) platform technology is focused on silencing the mRNA associated with disease-causing genes. Idera has designed 3GA oligonucleotides to overcome specific challenges associated with earlier generation antisense technologies and RNAi technologies.

Idera Pharmaceuticals is a clinical-stage biopharmaceutical company developing novel nucleic acid-based therapies for the treatment of certain cancers and rare diseases. Idera’s proprietary technology involves using a TLR-targeting technology, to design synthetic oligonucleotide-based drug candidates to act by modulating the activity of specific TLRs. In addition to its TLR programs, Idera has created a third-generation antisense technology platform using its proprietary technology to inhibit the production of disease-associated proteins by targeting RNA. For more information, visit www.iderapharma.com.