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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:
Saturday, July 26, 2025

Vinay Prasad's Days as Head of CBER May Be Numbered

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Many industry insiders expected Sarepta CEO Doug Ingram to be out by now.  Instead, a new dynamic is gathering steam: Vinay Prasad, the rece...
2 comments:
Tuesday, June 17, 2025

CRISPR Stocks in Wake of Verve Therapeutics Acquisition by Eli Lilly

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  Last night, news broke that pharma giant Eli Lilly was in talks to acquire Verve Therapeutics.   After a 3-year lull in major CRISPR deal...
1 comment: