Dicerna Announces Boehringer Ingelheim’s Acceptance of Candidate for Development Under RNAi Research Collaboration and License Agreement
May 24, 2021
Development Candidate Will Be Evaluated for Treatment of Nonalcoholic Steatohepatitis
Candidate Selection Triggers Single-Digit Multimillion USD Milestone Payment to Dicerna
“Advancing this selected target to development demonstrates the commitment of our team and our collaborative partners at
Under the terms of the existing agreement between Dicerna and
About Nonalcoholic Steatohepatitis (NASH)
Nonalcoholic steatohepatitis (NASH) is a condition characterized by the buildup of fat in the liver, potentially leading to liver fibrosis, cirrhosis, liver failure or cancer, and has an especially high prevalence among people who are obese or have type 2 diabetes. NASH is estimated to affect approximately 1.5% to 6.5% of adults in the
About RNAi and Dicerna’s GalXC™ RNAi Platform Technologies
Ribonucleic acid interference, or RNAi, provides a unique advantage to other disease inhibitor technologies, like small-molecule pharmaceuticals or monoclonal antibodies: instead of targeting proteins after they have been produced and released, RNAi silences the genes themselves via the targeted destruction of the messenger RNA (mRNA) made from the gene. Rather than seeking to inhibit a protein directly, the RNAi approach can prevent a disease-causing protein’s creation, directly impacting disease manifestation.
Dicerna’s proprietary GalXC™ RNAi platform aims to advance the development of next-generation RNAi-based therapies. Investigational therapeutics developed using our flagship GalXC technology utilize a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules that are designed to bind specifically to receptors on liver cells, leading to selective hepatocyte internalization and access to the RNAi machinery within the cells. Dicerna is continuously innovating and exploring new applications for RNAi technology beyond GalNAc-mediated delivery to the liver, including alternative RNA structures and fully synthetic ligands that target other tissues and enable new therapeutic applications, referred to as GalXC-Plus™.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding the collaboration agreement with
GalXC™ and GalXC-Plus™ are trademarks of