A gene therapy company developing one-time treatments for rare diseases, uniQure produced HEMGENIX, the first FDA-approved gene therapy for hemophilia B, now sold worldwide by partner CSL Behring. Founded in 1998 as Amsterdam Molecular Therapeutics by scientists from Amsterdam's Academic Medical Center, it renamed itself uniQure—a play on "unique cures." It also developed Glybera, the first gene therapy approved in the Western world, and its lead experimental candidate targets Huntington's disease.
FDA now says a BLA for AMT-130 is reasonable, reversing its prior rejection, and uniQure plans to submit in Q3 2026.
The FDA reversed course on AMT-130, now agreeing a under is reasonable based on existing data. rose 127% to $3.6 million on higher HEMGENIX royalties, but a $16.0 million non-cash loss on pushed the net loss to $53.5 million. The path to market for the lead asset is back on, but the company must align with the FDA on a confirmatory study design before submission.
Key takeaways
The FDA indicated a for AMT-130 under is reasonable based on existing Phase I/II data, a reversal from the prior quarter when the agency said those data were unlikely to support a submission.
The FDA wants alignment on a confirmatory study design before the submission, and the study may need to be well underway or fully enrolled at the time of .
rose 127% to $3.6 million, driven by higher HEMGENIX royalty payments from CSL Behring.
Section summaries
Management's Discussion and Analysis
Net loss widened to $81.1M in Q2 2026 driven by a $16.0M non-cash loss on pre-funded warrants and a $5.9M intangible write-down.
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Total revenues rose to $5.8M from $5.3M, driven by higher HEMGENIX royalty payments from CSL Behring.
The net loss widened to $53.5 million from $37.7 million a year ago, primarily due to a $16.0 million non-cash loss on the change in fair value of and a $20.4 million swing in foreign currency effects.
R&D expenses were essentially flat at $34.0 million, as lower AMT-130 manufacturing and clinical costs were offset by higher spending on the AMT-260 and AMT-162 programs.
Cash and investments reached $810.3 million at quarter-end after a June 2026 follow-on offering raised $242.7 million in net proceeds, extending the projected runway into 2030.
What changed
The FDA's position on AMT-130 shifted: in Q3 2025 and Q1 2026 the agency said Phase I/II data were unlikely to support a and recommended a sham-controlled trial; now the FDA says a BLA under is reasonable based on existing data, though it still requires alignment on a confirmatory study design.
The cash runway extended into 2030 from the second half of 2029, after a $242.7 million follow-on offering in June 2026 lifted cash and investments to $810.3 million.
SG&A expenses rose to $17.4 million from $13.5 million a year ago, as the commercial headcount build flagged in Q1 2026 continued despite the prior regulatory uncertainty.
A $5.9 million of a favorable supply intangible asset was recorded following the termination of the Genezen CSA, a new charge not present in prior quarters.
What to watch
Whether the FDA and uniQure reach alignment on the AMT-130 confirmatory study design before the planned Q3 2026 submission, and what the trial's size, cost, and timeline will be.
The outcome of the defendants' motion to dismiss the amended securities class action complaint, due September 14, 2026, and whether a loss contingency becomes probable.
Quarterly SG&A expense trajectory now that the commercial headcount has been built, to assess whether these costs can be reduced if the AMT-130 regulatory path faces further delays.
Whether HEMGENIX royalty from CSL Behring continues to grow, providing non- cash while the AMT-130 program advances toward submission.
R&D expenses decreased slightly to $34.0M from $35.4M, as lower AMT-130 manufacturing and clinical costs were partly offset by higher spending on AMT-260 and AMT-162.
SG&A expenses increased to $17.4M from $13.5M, primarily due to higher headcount to support potential commercial launches of AMT-130.
Other expense included a $5.9M of a favorable supply intangible asset following the termination of the Genezen CSA.
Non-operating loss of $27.0M compared to a $6.6M gain last year, mainly from a $16.0M loss on pre-funded warrant fair value and a $20.4M swing in foreign currency effects.
Cash and investments totaled $810.3M at quarter-end, bolstered by $242.7M in net proceeds from a June 2026 follow-on offering, and are expected to fund operations into 2030.
Quantitative and Qualitative Disclosures About Market Risk
We are exposed to a variety of financial risks in the normal course of our business, including market risk (including currency, price, and interest rate risk), credit risk and liquidity risk. Our overall risk management program focuses on the preservation of capital and the unpr…
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We are exposed to a variety of financial risks in the normal course of our business, including market risk (including currency, price, and interest rate risk), credit risk and liquidity risk. Our overall risk management program focuses on the preservation of capital and the unpredictability of financial markets and has sought to minimize potential adverse effects on our financial performance and position.
Our market risks and exposures to such market risks during the three months ended June 30, 2026, have not materially changed from our market risks and our exposure to market risk discussed in Part II, Item 7A of our Annual Report.
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Table of Contents
uniQure faces a securities class action over statements about its AMT-130 gene therapy study and regulatory pathway.
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A action was filed in February 2026 alleging false or misleading statements about the Phase I/II study of AMT-130 and the timing of a potential filing.
The claims are brought under Sections 10(b) and 20(a) of the Exchange Act on behalf of investors who purchased shares between September 24 and October 31, 2025.
The Oklahoma Firefighters Pension and Retirement System was appointed lead plaintiff and filed an amended complaint in July 2026.
The amended complaint focuses on alleged misstatements regarding the import and viability of Phase I/II data and available regulatory approval pathways.
Defendants' motion to dismiss the amended complaint is due September 14, 2026, and management intends to vigorously defend the action.
The company has not recorded any liability, stating that a loss is not probable and reasonably estimable at this time.
AMT-130 BLA submission is planned for Q3 2026 under accelerated approval, but confirmatory study design and FDA alignment remain key risks.
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The FDA agreed a for AMT-130 under is reasonable based on existing data, but wants alignment on a design before submission.
The FDA indicated the should be well underway or fully enrolled at the time of , and may require a randomized standard-of-care control instead of a sham procedure.
Failure to align with the FDA on design, or any requirement for additional studies, could delay or prevent AMT-130 approval and commercialization.
A UK regulatory submission for AMT-130 is also planned for Q3 2026 following a successful pre-submission meeting with the MHRA, but approval is not assured.
Risks are heightened by the challenges of developing a novel gene therapy for a rare disease, including reliance on non-traditional study designs and small patient populations.