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Fabry disease

Biomarin
Biotech

BioMarin inks up to $475M deal to challenge Fabry ERT incumbents

BioMarin is paying $70M upfront to license a next-generation Fabry disease therapy, expanding its franchise following the $4.8B Amicus takeover.
Nick Paul Taylor Oct 29, 2026 10:13am
sold

PTC bets $211M on ‘special opportunity’ to enter Fabry market

Aug 13, 2026 8:15am
pause button on blue background

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

Feb 6, 2026 10:56am
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
water tap outside

4DMT restructures pipeline, extending cash runway through 2028

Jan 10, 2025 2:00pm

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