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REGENXBIO Publishes Preclinical Data Highlighting RGX-202’s Potential for Duchenne Muscular Dystrophy

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO announced the publication of preclinical results for its investigational gene therapy, called RGX-202, for Duchenne Muscular Dystrophy. The findings, published in the peer-reviewed journal Molecular Therapy Methods and Clinical Development, showed that a microdystrophin gene therapy construct including the C-terminal/CT domain showed improved functional benefits compared to a construct without it.

RGX-202 is unique among investigational or approved microdystrophin gene therapies for Duchenne in that it includes this crucial CT domain, making it the closest to naturally occurring dystrophin. The preclinical study evaluated two AAV vectors in mdx mice, a model for Duchenne. One vector encoded a microdystrophin protein with the CT domain, while the other encoded an equivalent microdystrophin without it.

A medical professional holding a branded prescription drug manufactured by the pharmaceutical company.

The results showed that the microdystrophin with the CT domain was maintained at higher levels in transduced muscles, more effectively recruited the dystrophin-associated protein complex to the muscle membrane, and led to increased muscle force and resistance to damage in the dystrophin-deficient mice. These findings suggest that the CT domain enhances the microdystrophin design by promoting higher protein accumulation due to a longer half-life, which can lead to improved functional benefits.

REGENXBIO Inc. (NASDAQ:RGNX) is a clinical-stage biotechnology company that provides gene therapies that deliver functional genes to cells with genetic defects in the US.

While we acknowledge the potential of RGNX to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than RGNX and that has 100x upside potential, check out our report about this cheapest AI stock.

READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now.

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.

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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?

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  • 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.

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Dr. Inan Dogan

Dr. Ian Dogan

Co-Founder and Research Director at Insider Monkey

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