Replimune’s 70% Single-Day Gain And uniQure’s Simultaneous 20% Surge Tell The Same Story That The Departure Of FDA Commissioner Marty Makary Has Unlocked A Sentiment Rally Across The Entire Gene Therapy And Oncolytic Immunotherapy Sector
According to Investing.com, CNBC and SEC filings, uniQure N.V. (NASDAQ: QURE) shares surged over 20% on Friday May 29, while peer Replimune Group (NASDAQ: REPL) rocketed 70% triggered by Replimune’s announcement that the FDA had aligned with the company on a pathway to resubmit its Biologics License Application for RP1, an oncolytic immunotherapy for advanced melanoma, after the drug had previously been rejected by the agency.
uniQure had no new clinical data. No pipeline update. No corporate announcement. Its stock moved because the market concluded that the FDA reversal on Replimune’s RP1; a decision explicitly linked to the departure of former FDA Commissioner Marty Makary earlier in May signals a fundamentally more permissive regulatory environment for biotechnology companies navigating the agency. Understanding why requires understanding both companies.
Who uniQure is and what it is fighting for. uniQure is a Dutch-American gene therapy company headquartered in Lexington, Massachusetts and Amsterdam, listed on NASDAQ under the ticker QURE. Founded in 1998, it is among the oldest and most technically credentialed gene therapy companies in the world.
Its most significant commercial achievement is the approval of etranacogene dezaparvovec; marketed as Hemgenix: a one-time gene therapy for haemophilia B that achieved the status of the most expensive drug ever approved upon its 2022 launch at $3.5 million per patient. Hemgenix is commercialised by CSL Behring under a landmark 2020 licensing and commercialisation agreement.
The drug represents over a decade of research into adeno-associated virus (AAV5) vector delivery of functional Factor IX gene sequences directly into hepatocytes, achieving sustained clotting factor production from a single infusion.
Beyond haemophilia, uniQure’s pipeline is anchored by AMT-130, an investigational gene therapy for Huntington’s disease; one of medicine’s most devastating and previously untreatable neurodegenerative disorders.
In March 2026, the FDA informed uniQure it could not agree that data from the Phase I/II studies, compared to an external control, were sufficient to support a marketing application for AMT-130, strongly recommending uniQure conduct a prospective, randomised, double-blind, sham surgery-controlled study instead.
That guidance; effectively a rejection of uniQure’s accelerated approval approach was among the most consequential setbacks in the company’s recent regulatory history, and it remains unresolved. uniQure is simultaneously planning a Marketing Authorisation Application submission to the UK’s MHRA in the third quarter of 2026, pursuing international regulatory pathways as a hedge against continued FDA friction.
Why Replimune’s FDA news moved uniQure’s stock. The connection is structural, not literal. Replimune announced it would resubmit the RP1 Biologics License Application in the coming days after collaborative communications with the FDA.
The development represents a reversal for the drug, which had been rejected by the agency under former FDA Commissioner Marty Makary’s leadership. The FDA’s shift in position follows Makary’s departure earlier this month, signalling changing priorities at the agency. The rejection had been among the most contentious decisions during Makary’s tenure.
The market read this as a sector-wide signal. If the FDA is now willing to reverse contentious rejections issued under Makary’s tenure, then companies with drugs that were blocked, delayed, or subjected to unusually demanding evidence standards during that period have just seen their probability-of-approval distributions shift meaningfully upward in both directions.
uniQure with AMT-130 stalled at the FDA after the March guidance, and with the haemophilia B approval precedent demonstrating that the company can achieve regulatory success with complex gene therapy, is precisely the type of company a rational biotech investor revalues on this kind of signal.
The broader post-Makary regulatory trade. Makary’s tenure at the FDA was characterised by a more conservative posture toward accelerated and adaptive approval pathways, particularly for gene therapies and oncology biologics where surrogate endpoint data and external control comparisons have been the basis for regulatory filings.
Several high-profile rejections and requests for larger, more burdensome confirmatory trials came during his leadership. His departure has been interpreted by biotech investors as a potential unwinding of that posture; not uniformly across all products, but across the subset of drugs whose rejections were specifically tied to the elevated evidence standards he championed.
The Replimune reversal is the first concrete, documented confirmation that the unwinding is real and happening at the level of individual BLA reconsiderations.
The financial position and the pipeline risk. In 2025, uniQure raised aggregate net proceeds of $404.2 million through follow-on public offerings, issuing 11.8 million ordinary shares and pre-funded warrants, strengthening its balance sheet significantly. That cash runway gives the company time to navigate the AMT-130 regulatory impasse.
However, the pipeline carries meaningful risk on multiple fronts. AMT-162, the company’s investigational gene therapy for SOD1-ALS, has been discontinued following a dose-limiting toxicity in the Phase I/II EPISOD1 study. The Huntington’s programme remains the value driver and it is currently stalled. The 20% move on Friday reflects not a resolution of that stall, but a change in the probability that the stall is temporary rather than permanent.
What the move means for the sector. Friday’s simultaneous surge in QURE and REPL was not unique to those two tickers. The broader gene therapy and oncolytic immunotherapy sector moved in sympathy, as investors recalibrated regulatory risk premiums across the cohort of companies with drugs in advanced regulatory stages.
The pattern is familiar from previous episodes of FDA leadership transitions; approval rates, review timelines, and standards of evidence are demonstrably sensitive to the philosophy of the person at the top of the agency.
When that person changes, the market does not wait for the next approval or rejection to update. It updates immediately, in the prices of every company whose pipeline is in regulatory play.
The verdict. uniQure’s 20% gain on Friday was not about gene therapy science. It was about the probability that the regulatory environment that blocked AMT-130’s accelerated approval pathway has itself shifted.
Whether that probability assessment is correct will be determined by the FDA’s actual conduct over the next three to six months, specifically, whether the agency engages constructively with uniQure’s AMT-130 programme in the way it has now engaged with Replimune’s RP1. The stock price is pricing optimism. The clinical and regulatory calendar will price the truth.
To check out our previous coverage on biotech, FDA approvals, and the gene therapy sector, read our articles here.
