uniQure ($QURE): Gene Therapy's Boldest Bet?

Welcome back to The Omaha Verdict where I unpack stocks in plain English (no financial degree needed).

Today’s pick is not a typical compounder. It’s volatile. It’s pre-revenue. It burns cash. But it also might cure one of the most brutal neurological diseases known to science. We’re looking at uniQure ($QURE), a small-cap biotech trying to do something massive: cure Huntington’s disease with a one-time brain-delivered gene therapy. Yep. Cure. Not treat. Not manage. Cure.

uniQure ($QURE) is a $15 stock with billion-dollar upside potential. The company is pioneering one-time gene therapies that could become the standard of care -- if the FDA gives it the green light.

Most analysts see $QURE’s Huntington’s therapy hitting the market in 2028. But FDA minutes expected any day might confirm an accelerated path for an accelerated launch. If that happens, this stock could reprice in days, not years.

This is a classic high-risk, high-reward with:
➔ Solid institutional backing
➔ A strong cash position
➔ Multiple upcoming clinical and regulatory catalysts
➔ A clear path to commercial scale if they execute

Let’s dive in.

Omaha Verdict Score™: 10 / 15

What does uniQure do?

uniQure is a Netherlands-based gene therapy company. Its focus is on using viral vectors (AAVs) to deliver one-time treatments for devastating neurological diseases. Think Huntington’s, epilepsy, ALS.

Its business model:
➔ Build once → Treat once → Get paid like a pharma company for a lifetime drug.
➔ Already validated with Hemgenix (Hemophilia B therapy, now commercialized by CSL Behring).
➔ Now aiming for a repeat, but even bigger.

Key Programs:

➔ AMT-130 – Huntington’s disease (flagship)
First-ever gene therapy in human trials for HD. Delivered via brain surgery. Shows early promise in slowing disease. Just got Breakthrough Therapy designation from the FDA. A BLA (Biologics License Application) filing could happen as soon as late 2025. Approval maybe 2026.

➔ AMT-260 – Epilepsy
One-time gene therapy to reduce seizures in drug-resistant temporal lobe epilepsy. First human data just dropped: 92% fewer seizures in the first patient. Big if it holds.

➔ AMT-191 – Fabry disease
Competing in a crowded space but early signs suggest it's safe and effective. Interim data due in H2 2025.

➔ AMT-162 – ALS (SOD1 variant)
Trying to beat Biogen’s Tofersen with a one-shot therapy. Too early to judge but promising.

Durable Moat ➔ 3 / 3

➔ They’ve built a solid gene delivery system. They use a special virus (called AAV) to deliver medicine directly into cells. It’s like a safe, targeted shuttle system; and they’ve spent years perfecting it.
➔ They own valuable patents
➔ First-mover in HD gene therapy
➔ Retained full rights to AMT-130 (potentially huge upside)

They’ve carved out a rare position: a CNS-focused (Central Nervous System) gene therapy pure-play with advanced programs, cash, and real momentum. If AMT-130 works, this could be the standard of care.

Management Quality ➔ 4 / 5

➔ Locked in $375M by selling Hemgenix royalties at the top (that money now funds the next-gen pipeline)
➔ Cut costs and refocused early — now funded through 2027
➔ Navigated FDA like pros (Breakthrough status + accelerated path)
➔ Backed by top biotech funds (EcoR1, Avoro, RTW) who bought even more shares in the last funding round (a strong vote of confidence in the team)

Only reason it’s not a 5/5? They’ve never launched a product themselves. Commercial execution is still unproven — but so far, they’ve made all the right moves.

Business Fundamentals ➔ 2 / 5

Let’s be real. It’s pre-revenue. Cash burn is high. Metrics look scary.

➔ Revenue: $1.6M (Q1 2025 — mostly interest income)
➔ Net Loss: -$239.5M
➔ Free Cash Flow: -$186M
➔ Net Profit Margin: -883.35% (not a typo)
➔ Operating Margin: -679.58%

BUT…
➔ Cash: $409M (enough runway through mid-2027)
➔ Debt: Low (~$50M true debt; rest is non-recourse royalty liability)

This is a binary biotech play. Either one of their therapies gets approved — and revenue explodes — or they burn through cash and raise again. That’s the game.

Valuation ➔ 1 / 2

Current price: ~$15
Market Cap: ~$800M
EV: ~$420M

$QURE has $409M in cash. Enterprise value is just $420M. You're almost getting a pipeline of gene therapies for free.

Analysts project AMT-130 could generate $2B+ at peak. Even a 25–50% shot at that outcome makes the risk/reward compelling. Institutions seem to agree — 92% of shares are held by top biotech funds (EcoR1, Avoro, RTW, BlackRock, etc.).

This stock trades like a “maybe someday”, but if the FDA greenlights an Accelerated Approval path this year, sentiment could flip fast.

⚠️ Key Risks

➔ Clinical trial failure – If AMT-130 flops, the whole thesis cracks
➔ Safety – Brain surgery + viral vectors = non-trivial risk
➔ Regulatory delays – FDA could slow things down
➔ Commercial hurdles – Launching an invasive therapy is not like selling pills
➔ Better rivals – ASOs (like Wave or Ionis) might offer similar benefits with easier delivery
➔ Still pre-revenue – Any hiccup in cash flow or trial timing could require dilution

Bottom Line

uniQure is not for the faint of heart. But for long-term investors willing to bet on biotech disruption, it’s one of the more asymmetric plays out there.

➔ First gene therapy for Huntington’s now at the gates of approval
➔ Multiple clinical shots on goal (epilepsy, ALS, Fabry)
➔ Fully funded through at least mid-2027
➔ High institutional conviction

If AMT-130 gets approved and performs as hoped, $QURE won’t stay a $15 stock. And with FDA minutes + regulatory clarity expected imminently, this might be the calm before the storm.

In my view, $QURE deserves a closer look from long-term investors who can stomach volatility in exchange for potentially life-changing upside.

Disclosure: I hold a long position in $QURE.

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Financial Disclaimer

Disclaimer: This newsletter is for informational purposes only and is not financial advice. I’m not a financial advisor, and nothing here should be taken as a recommendation to buy or sell any stock. Always do your own research and consult with a professional before making any investment decisions.