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Thursday, May 21, 2026

From ZZ to SZ to MZ to potentially MM! The New State of Play for RNA Editing in Alpha-1 Antitrypsin Disease

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The last few days and weeks delivered critical datapoints in the race to develop RNA Editing oligonucleotides for the treatment of AATD.  Th...
Tuesday, May 19, 2026

Wave Life Sciences Sets 30% RNA Editing Bar for AATD

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Wave Life Sciences ( here ) and Beam Therapeutics ( and here ) just presented important updates on their alpha-1 antitrypsin disease (AATD) ...
Monday, April 13, 2026

FDA Plays Cynical Political Games to Keep Effective Cancer Drug from Patients

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Under the current FDA, patients don't tire of losing . This time, the FDA re-rejected a drug which, when given together with anti-PD1 t...
1 comment:
Sunday, March 29, 2026

An Explanation for the Disconnect between Z-AAT Editing Efficiency and Alpha-1 Antitrypsin Output

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The commonly stated goal of alpha-1 antitrypsin editing approaches in addressing AATD is to achieve 50% editing of the mutant Z-allele.  Thi...
1 comment:
Tuesday, March 17, 2026

Approving AMT-130 Now Will Incentivize Better Huntington's Drugs and Speed Access

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To be clear: the FDA has just violated the trust of investors willing to risk significant capital throughout the ups and downs of the market...
1 comment:
Tuesday, September 30, 2025

Plea to Sarepta and Arrowhead: Just Focus on Aberrant Exon 1 Transcript in Huntington’s Disease

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Last week, uniQure achieved a breakthrough in developing disease-modifying therapies for Huntington’s Disease by showing that AMT-130 slowe...
12 comments:
Thursday, September 25, 2025

PepGen Disrupts Myotonic Dystrophy Type I

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Yesterday was a memorable day in the development of drugs for two of the main severe monogenic diseases without treatments to change disease...
1 comment: