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UniQure advances AMT-130 toward BLA with 2026 topline data and strong runway

Jul 29, 2026, 7:08 AM EDT1 sourcesAI-analyzed

Why it may matterVerify against the original reporting

Key near-term catalysts include BLA timing (Q3 2026) and September topline data for AMT-130, plus ongoing regulatory progress in the UK; strong cash runway reduces dilution risk and supports execution risk, supporting upside.

AI summary

What happened, with direct paths to the underlying reporting

uniQure disclosed progress on AMT-130, targeting a BLA submission in Q3 2026 with UK MHRA alignment and September four-year topline data. AMT-260 showed encouraging early signals and a solid safety profile. A $259 million follow-on offering extended cash runway into 2030, supporting anticipated commercial launch activities and pipeline investments.

AMT-130 BLA submission planned in Q3 2026 after FDA Type B alignment.
Topline four-year AMT-130 data expected September 2026.
AMT-260 Phase I/IIa shows early seizure reductions and favorable safety.
June 2026 follow-on offering raises $259M, extending runway to 2030.
Cash and investments: $810.3M as of 6/30/2026; funds support into 2030.

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