Uniqure (NASDAQ: QURE) shares opened nearly 200% up on Wednesday – marking one of the most dramatic single-day moves in biotech this year.

The surge follows a trifecta of bullish development: a breakthrough in Huntington’s disease, a $175 million capital infusion, and a reaffirmed “buy” rating from Stifel.

These tailwinds made investors recalibrate the company’s long-term potential, especially in gene therapy, where QURE has long been a pioneer.

Versus its year-to-date low in April, Uniqure stock is up more than 350% at the time of writing.

Unique stock soars on Huntington’s disease breakthrough

Uniqure shares soared this morning primarily because the company announced promising data for “AMT-130” – it’s candidate therapy for Huntington’s disease.

According to QURE’s press release, patients treated with the therapy showed sustained reductions in mutant huntingtin protein levels – a key driver of disease progression.

This marks the first time a gene therapy has shown such a durable biomarker impact in Huntington’s, a devastating neurodegenerative disorder with no cure.

“We are encouraged by the consistency and magnitude of the effect,” said Ricardo Dometsch, the firm’s chief medical officer.

QURE shares are rallying today as investors bet on AMT-130 becoming a first-in-class treatment, unlocking a multi-billion-dollar market and positioning Uniqure as a leader in CNS gene therapy.

QURE shares rally as fresh capital boosts balance sheet

Investors are cheering Uniqure stock because the clinical-stage biotechnology firm announced a $175 million non-dilutive funding through a strategic royalty monetisation deal with Hercules Capital on Wednesday.

This agreement, which involves selling a portion of future royalties from its already approved and commercialised haemophilia B therapy (Hemgenix), strengthens QURE’s cash position without the need to issue new shares – preserving shareholder value.

As Matt Kapusta, the firm’s chief executive, noted, “This transaction provides us with substantial  capital to advance our pipeline without compromising ownership.”

In short, this financing will help QURE stock via accelerating the development of AMT-130 and other candidates while avoiding the dilution that often plagues small-cap biotech stocks.

Stifel maintains QURE stock at ‘buy’

Part of the surge in Uniqure shares at writing is related to a bullish note from Stifel analysts.

On Wednesday, the investment firm reiterated its “buy” rating on QURE stock, citing the improved financial flexibility and promising Huntington’s data.

According to the analysts, the biotech firm’s risk-reward profile has shifted dramatically – with AMT-130 now showing real promise and the company’s cash runway extended well into 2027.

“We believe QURE is entering a new phase of value creation,” Stifel experts wrote in their research note.

This endorsement from a respected Wall Street firm helped validate investor enthusiasm and likely contributed to the stock’s parabolic move.

All in all, with analyst support and fresh capital, Amsterdam-headquartered Uniqure now has the momentum to pursue aggressive clinical and commercial expansion.

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