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Strong Rare Disease Pipeline Drives Favorable Sentiment for Ultragenyx (RARE)

Ultragenyx Pharmaceutical (NASDAQ:RARE) is one of the 17 biotechnology stocks with more than 50% upside.

On February 3, the price target on Ultragenyx Pharmaceutical (NASDAQ:RARE) was set to $60 by H.C. Wainwright analyst Ram Selvaraju, who maintained a Buy rating on the stock. The analyst’s estimate leads to a promising upside potential of more than 155% from the prevailing level.

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Selvaraju attributed his views to the company’s impressive rare disease pipeline and expected regulatory milestones. He also highlighted on-schedule BLA resubmission for UX111, which includes extended clinical data showing sustained neurologic benefits and biomarker improvements. The filing also includes an adequate safety profile for Sanfilippo syndrome type A, which does not have any approved therapies. The analyst is optimistic about the company achieving profitability as early as 2027, which reflects favorably on its attractive risk-reward profile.

On January 28, Ultragenyx Pharmaceutical (NASDAQ:RARE) was assigned an Overweight rating by Barclays analyst Eliana Merle. She estimated a target price of $44, which yields more than 87% upside potential. Merle anticipates a more favorable outlook for biotech stocks, supported by robust fundamentals and forecasted continued mergers and acquisitions activity.

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

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: 15 Most Promising Mid-Cap Healthcare Stocks Under $50 and 11 Most Promising Small-Cap Industrial Stocks Under $50.

Disclosure: None. This article is originally published at Insider Monkey.

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.

And this breakthrough has already set off a frenzy among hedge funds and Wall Street’s top investors.

What most investors don’t realize is that one under-owned company holds the key to this $250 trillion revolution.

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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This prediction might not be bold at all:

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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