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Ultragenyx Pharmaceutical (RARE) Garnering Bullish Sentiment With Forthcoming GTX-102 Phase 3 Angelman Trial

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 10 best biotech stocks with highest upside potential.

On April 16, Morgan Stanley increased the price target for Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) from $50 to $67, resulting in an adjusted upside potential in excess of 178% at the prevailing level. The firm also reiterated its Outperform rating on the stock.

Nestor Rizhniak/Shutterstock.com

Morgan Stanley revised the price target upward based on a strict new probability-of-success approach to the forthcoming GTX-102 Phase 3 Angelman trial. This major clinical readout is anticipated in the second half of 2026. The resulting risk profile is rather asymmetrical. The firm has projected a 50% to 70% upside, which significantly offsets a 20% downside risk of a clinical failure.

Back on March 30, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced clearance from the US Food & Drug Administration for the Investigational New Drug Application for UX016. It is a prodrug of sialic acid under investigation for use as a substrate replacement therapy for GNE Myopathy. GNEM is a rare neuromuscular disease characterized by severe disability due to mutations in the GNE gene, which impairs sialic acid biosynthesis.

The UX016 program is supported externally via proof-of-concept clinical trials, which include a Phase 1/2 trial scheduled for initiation in the latter half of 2026. Commenting on the progress of UX016, CEO Emil Kakkis observed that moving the drug into the clinic is a significant step for the GNE myopathy community, and it demonstrates the company’s effort to create a novel method for improving sialic acid utilization by the muscles. The first-in-human study will involve around 24 patients between 18 and 55 years of age suffering from GNEM, who will be recruited in the US.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops novel therapies, with a focus on identifying, acquiring, and commercializing products for rare and ultra-rare genetic diseases. It has a strong emphasis on gene therapy and covers various stages of clinical trials. The company is currently going through high cash burn with the aim of turning profitable from 2027 onwards.

While we acknowledge the risk and potential of RARE as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than RARE and that has 10,000% upside potential, check out our report about this cheapest AI stock.

READ NEXT: 33 Stocks That Should Double in 3 Years and 15 Stocks That Will Make You Rich in 10 Years.

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The $250 Trillion AI Hype is Real. A few years from now, you’ll probably wish you’d bought this stock.

Dr. Inan Dogan

Dr. Ian Dogan

Co-Founder and Research Director at Insider Monkey

When Jeff Bezos said that one breakthrough technology would shape Amazon’s destiny, even Wall Street’s biggest analysts were caught off guard.

Fast forward a year and Amazon’s new CEO Andy Jassy described generative AI as a “once-in-a-lifetime” technology that is already being used across Amazon to reinvent customer experiences.

At the 8th Future Investment Initiative conference, Elon Musk predicted that by 2040 there would be at least 10 billion humanoid robots, with each priced between $20,000 and $25,000.

Do the math. According to Musk, this technology could be worth $250 trillion by 2040.

Put another way, that’s roughly equal to:

  • 175 Teslas
  • 107 Amazons
  • 140 Metas
  • 84 Googles
  • 65 Microsofts
  • And 55 Nvidias

And here’s the wild part — this $250 trillion wave isn’t tied to one company, but to an entire ecosystem of AI innovators set to reshape the global economy.

It’s a leap so massive, it could reshape how businesses, governments, and consumers operate worldwide.

Even if that $250 trillion figure sounds ambitious, major firms like PwC and McKinsey still see AI unlocking multi-trillion-dollar potential.

How could anything be worth that much?

The answer lies in a breakthrough so powerful it’s redefining how humanity works, learns, and creates.

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In fact, Verge argues this company’s supercheap AI technology should concern rivals.

Before I reveal the details, let’s talk about how some of the richest people on the planet are positioning themselves.

  • Bill Gates sees artificial intelligence as the “biggest technological advance in my lifetime,” more transformative than the internet or personal computer, capable of improving healthcare, education, and addressing climate change.
  • Larry Ellison — through Oracle, is spending billions on Nvidia chips and partnering with Cohere to embed generative AI across Oracle’s cloud and apps.
  • Warren Buffett — not known for tech hype — says this breakthrough could have a ‘hugely beneficial social impact.

When billionaires from Silicon Valley to Wall Street line up behind the same idea — you know it’s worth paying attention to.

Even as we admire what Tesla, Nvidia, Alphabet, and Microsoft have built, we believe an even greater opportunity lies elsewhere…

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A few years from now, you’ll wish you’d owned this stock.

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Dr. Inan Dogan

Dr. Ian Dogan

Co-Founder and Research Director at Insider Monkey

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