Science and Innovation
RNA-based therapeutics hold immense promise to precisely regulate genes and cellular pathways in unprecedented ways. Since the inception of siRNA technology, two decades of dedicated research have culminated in the approval of numerous therapeutic programs, heralding a transformative era of therapeutic possibilities.
We aspire to make meaningful contributions, and actively engage in this emerging era of RNA therapeutics.
RNAi History
1998
RNA interference (RNAi), a remarkable gene regulation mechanism, was first described by Andrew Fire and Craig Mello.
2006
Andrew Fire and Craig Mello, win the Nobel Prize in Physiology or Medicine.
2018
First RNAi-based therapeutic, Onpattro, approved for the treatment of hereditary amyloidogenic transthyretin (hATTR) amyloidosis with polyneuropathy in adults.
Since then
A number of other programs have received approval, and a wide array of companies are actively progressing clinical RNAi programs across diseases and therapeutic areas.
siRNA Mechanism of Action
RNAi therapeutics, also known as siRNA, employ double-stranded RNA constructs to precisely decrease the expression of target genes through sequence-specific recognition. These siRNAs associate with a protein complex called the RNA-Induced Silencing Complex (RISC), which unwinds the siRNA and utilizes one strand as a guide to selectively identify and bind to complementary mRNA molecules. Once recognized, the RISC complex cleaves and degrades the targeted mRNA, thereby halting the production of the corresponding protein and effectively silencing the gene of interest.
GalNAc
N-Acetylgalactosamine (GalNAc), an amino sugar derivative of galactose, is conjugated to RNAi molecules, facilitating highly precise delivery to hepatocytes through asialoglycoprotein receptors. A straightforward subcutaneous injection of GalNAc-conjugated siRNA results in rapid homing and uptake of therapeutic agents in the liver within just a few hours. This effect is sustained, with a duration of effect lasting several months, due to the storage within hepatocyte endosomes.
The GalNAc targeting approach has been clinically validated and is incorporated in multiple marketed RNA therapeutics.
Our focus
We are committed to advancing the field of RNA therapeutics by harnessing the potential of RNA interference (RNAi) and other innovative RNA-based technologies.
Our dedicated research teams collaborate on the development of cutting-edge siRNA therapeutics, with the goal of addressing various diseases at the genetic level. By staying at the forefront of this rapidly evolving field, we aim to bring forth transformative treatments that can significantly improve patients' lives.
In addition to our focus on liver targeted therapeutics, our objective is to pioneer innovative technologies geared towards previously unexplored cell types and tissues, thereby broadening the scope of RNA therapeutics to tackle new disease areas.
