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Wednesday, October 9, 2024

Genome Editing for Hereditary Angioedema Promises a Life Free from Disease Burden

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Intellia Therapeutics is about to unveil phase II data on NTLA-2002 at the end of the month.  NTLA-2002 aims to be a one-time treatment for...
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Thursday, August 24, 2023

The Nucleic Acid Therapeutics Race to Revolutionize Alpha-1-Antitrypsin

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Alpha-1-antitrypsin disease (AATD) is caused by mutations in the SERPINA1 gene coding for alpha-1-antitrypsin (AAT).   There are an estimate...
Monday, July 24, 2023

Lightning Fast Wave Life Sciences Demonstrates High ADAR Editing Rates Across Targets

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There was a time, not that long ago, when 1-3% ADAR editing rates in tissue culture cells were typically reported in the field.  The hope th...
Friday, July 14, 2023

Korro Bio To Become Third Publicly Listed ADAR Editing Company

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Today, pure-play ADAR Editing Korro Bio announced that it will reverse merge into biotech shell Frequency Therapeutics (current ticker: FRE...
Saturday, April 29, 2023

Roche Impresses with Effective RNA Editing of Polyglutamine Repeat mRNA

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Roche has shown interest in RNA Editing through its 2021 partnership with Shape Therapeutics.   The goal of this partnership was to use Sha...
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Wednesday, April 26, 2023

RNAi Also Conquers the Central Nervous System

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In the span of a day, RNAi Therapeutics have gone from a mechanism widely viewed as being constrained to the liver only, to a major therapeu...
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Tuesday, April 25, 2023

Oligonucleotides Break Through to the Lung

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It is days like today that I live for .  Days when new platform technology data is revealed that will change the practice of medicine and be...
3 comments: