Fabry disease

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Biotech

UniQure halts mid- to high-dosing for AAV gene therapy

Two patients receiving uniQure’s gene therapy at a mid-level dose have experienced asymptomatic Grade 3 liver enzyme elevations.
Sanofi

Sanofi GCS inhibitor fails ph. 3 Fabry trial but wins in Gaucher

Feb 2, 2026 5:46am
Fabry disease

Cash-strapped Sangamo seeks partner for new gene therapy

Jun 24, 2025 11:53am
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4DMT restructures pipeline, extending cash runway through 2028

Jan 10, 2025 2:00pm
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Sangamo slashes time to market for Fabry gene therapy

Oct 22, 2024 12:57pm
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Relay loses interest in SHP2 inhibitor after Genentech leaves

Aug 7, 2024 6:00am