Ribonuclease H

Ionis announces that FDA accepts New Drug Application and grants Priority Review of tofersen for a rare, genetic form of ALS

Retrieved on: 
Tuesday, July 26, 2022
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The application has been granted priority review and given a Prescription Drug User Fee Act action date of Jan. 25, 2023.

Key Points: 
  • The application has been granted priority review and given a Prescription Drug User Fee Act action date of Jan. 25, 2023.
  • The FDA has noted that it is currently planning to hold an Advisory Committee meeting for this application.
  • "We also want to thank Biogen for their commitment to advancing tofersen, which, if approved, will be the first treatment that targets a genetic cause of ALS."
  • Dr. Bennett added, "Acceptance of the NDA for tofersen further strengthens Ionis' platform strategy to target all forms of ALS and central nervous system disorders more broadly."

Topline Results from Tofersen Phase 3 Study and its Open Label Extension in SOD1-ALS to be Presented at the American Neurological Association Annual Meeting

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Thursday, October 14, 2021
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Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H in an effort to reduce synthesis of SOD1 protein production.

Key Points: 
  • Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H in an effort to reduce synthesis of SOD1 protein production.
  • Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) under a collaborative development and license agreement.
  • Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.
  • To learn more, please visit www.biogen.com and follow us on social media Twitter , LinkedIn , Facebook , YouTube .

DGAP-News: Secarna publishes new scientific insights in Nucleic Acid Therapeutics on fundamental determinants of antisense oligonucleotide activity

Retrieved on: 
Thursday, August 5, 2021
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The article, "Investigation of the activity of antisense oligonucleotides targeting multiple genes by RNA sequencing" was published in Nucleic Acid Therapeutics was published in Nucleic Acid Therapeutics: https://www.liebertpub.com/doi/10.1089/nat.2020.0932

Key Points: 
  • The article, "Investigation of the activity of antisense oligonucleotides targeting multiple genes by RNA sequencing" was published in Nucleic Acid Therapeutics was published in Nucleic Acid Therapeutics: https://www.liebertpub.com/doi/10.1089/nat.2020.0932
    Standard ASO design is based on the sequence complementarity of the oligo to its target.
  • However, the degree of target knockdown that ASOs can achieve varies strongly between different ASOs having full complementarity to the target.
  • In a first step, Secarna investigated the ASO's effectivity against IDO1.
  • Alexander Gebauer, MD, PhD, CEO and Managing Director of Secarna Pharmaceuticals, added: "At Secarna, we are constantly working on improving our proprietary LNAplusTM ASO therapies platform.