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On December 29, 2021, Ionis Pharmaceuticals announced the completion of a collaboration agreement with AstraZeneca on eplontersen
In ATTR patients, mutant and wild-type TTR proteins form fibrils in human tissues, which in turn disrupt normal tissue function
Globally, there are an estimated 300,000 to 500,000 ATTR-CM patients, and 1 to 40,000 ATTR-PN patients
The single-stranded DNA molecule of ASO drugs can lead to the degradation of mRNA or change the splicing of mRNA by binding to a specific RNA sequence, so as to achieve the effect of targeting the underlying disease mechanism at the mRNA level and treating the disease
▲Ionis' new generation LICA technology can deliver ASO to various organs and tissues of the human body by coupling ASO with different ligands (Image source: Ionis official website)
In addition, Ionis also expects hereditary ATTR-PN to be the first indication for eplontersen and is expected to submit a New Drug Application (NDA) to the US FDA by the end of 2022
References:
[1] Ionis And Astrazeneca Close Deal To Develop And Commercialize Eplontersen.
[2] Astrazeneca And Ionis Close Agreement To Develop And Commercialise Eplontersen.
[3] Astrazeneca And Ionis Sign Deal To Develop And Commercialise Eplontersen.