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Friday, March 14, 2025

Hepcidin Agents Set to Improve Polycythemia Vera Care

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Protagonist Therapeutics earlier this month reported positive results from its phase III VERIFY study of hepcidin mimetic rusfertide for th...
Monday, March 10, 2025

Base Editor Beam Therapeutics Sets New Record in Alpha-1-Antitrypsin Correction Race

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Today Beam Therapeutics reported initial data for BEAM-302, a CRISPR-based base editor for the correction of the Z mutant form of alpha-1-a...
2 comments:
Tuesday, February 25, 2025

Clinical Data Support PepGen’s Potentially Disruptive Myotonic Dystrophy Type 1 Approach

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Yesterday, PepGen showed first clinical data for PGN-EDODM1 in Myotonic Dystrophy Type 1 (DM1).   Although it is years behind the DMPK knoc...
Tuesday, February 18, 2025

Increasing the Reach of ADAR Therapeutics by Protein Structure Prediction

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'Only' being able to convert an ‘A’ to an ‘I’ when genome editing can seemingly re-write the code at will had been seen as a major l...
2 comments:
Wednesday, February 12, 2025

Why I Am Taking My HAE Cure Idea to My Grave

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Last year, as I was researching the hereditary angioedema (HAE) therapeutics landscape, I came across data in the C1 inhibitor gene regulati...
Thursday, February 6, 2025

The Lp(a)-Lowering Drug Race

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Cardiovascular disease (CVD) remains a leading cause of death and morbidity worldwide.  According to the WHO , 18 million of the 68 million ...
1 comment:
Wednesday, January 29, 2025

Huntington’s Disease Therapeutics Breaking Through (Finally!)

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Huntington’s is one of the large devastating monogenic diseases for which disease-modifying therapies have yet to materialize.  This is abou...
4 comments: