BIO 2014: Why San Diego is a Hub for RNA R&D and Drug Development

6/23/14

Editor’s note: Arcturus CEO Joe Payne is set to give a company presentation Wednesday at the BIO 2014 convention in San Diego. —BVB

Medicinal chemistry has come a long way since the first opiates were “prescribed” in 3500 B.C. Since then, we have seen advancements of science usher in over 1,100 FDA-approved drugs—with a substantial number of additional breakthroughs on the horizon. We now are witnessing the chemical development of three major classes of drugs: Small molecules; Antibodies; (and more recently) RNA therapeutics.

Cambridge, MA, and the San Francisco Bay Area might lay claim to leading the efforts toward the first two classes of drug development, but San Diego is undeniably a growing hub for RNA research and development.

San Diego biotechnology companies are actively engaged in the research and development of every type of RNA medicine—Antisense, siRNA, UNA oligomers, messenger RNA, microRNA—and pursuing every RNA delivery technology, including lipid-enabled delivery, conjugate chemistry, and self-delivery.

Combine this with the world-class RNA research taking place at the surrounding academic institutions in San Diego, and it leads me to conclude that San Diego is a leading hub for RNA medicine companies, laying the foundation for substantial growth over the next few decades.

Table Comparing RNA Capabilities (Courtesy Arcturus Therapeutics)

Table Comparing RNA Capabilities (Courtesy Arcturus Therapeutics)

While it’s debatable whether San Diego or Boston ranks as the world’s leading hub for RNA medicine, San Diego clearly has more companies focused on emerging RNA technologies than the Bay Area. In any case, consider the pioneering work done by San Diego’s RNA companies:

—Isis Pharmaceuticals (NASDAQ: ISIS) received FDA approval in early 2013 for mipomersen (Kynamro), developed with Genzyme (NASDAQ: GENZ), the first systemic antisense drug to reach the market. They recently landed a $100 million deal with Biogen Idec to support the development of RNA medicines to address significant brain diseases.

—Regulus Therapeutics (NASDAQ: RGLS), a world leader in microRNA therapeutics, has advanced their first RNA medicine into the clinic for Hepatitis C.

—Arcturus Therapeutics, founded in 2013, discovered Lipid Unlocked Nucleic Acid RNA (LUNAR) delivery technology—a premier solution for messenger RNA and siRNA. Arcturus combines its delivery technology with so-called unlocked nucleic acid chemistry to pursue superior RNA medicines for rare diseases.

San Diego is not just a world-leading biotech center of innovation, it garners 266 days of sunshine each year. So, while the debate might continue as to which city can hold the title as the leading RNA hub, there is less uncertainty with respect to the climate—the US Weather Bureau recently described San Diego as “the closest thing to perfect in America.”

Joseph Payne is the CEO of San Diego-based Arcturus Therapeutics, which is advancing proprietary technology for developing RNAi-based drugs targeting orphan diseases. He has over 20 years of successfully drug discovery experience at Merck Research Labs, DuPont Pharmaceuticals, Bristol-Myers Squibb, Kalypsys, and Nitto Denko. Follow @

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  • Billiam

    Only Harvard and MIT in Boston? No BU, BC, Northestern, … I realize this is a cheerleading opinion, but come on – don’t be this blatant – it seriously detracts from your credibility.

  • Scien-Toast

    Thank you Xconomy for getting a someone to write an advertisement about his own company. As a long time San Diego resident biologist: (1) no San Diego is not an RNA therapeutics hub because as you can see from the list, it’s just the author’s own company name listed multiple times. He just needed an excuse to do that. Boston is. (2) any person who lists USD as an “academic” university makes it abundantly clear he/she has no sense of what h/she is saying. From Wikipedia: The University of San Diego (USD) is a private Roman Catholic liberal arts college in San Diego, California. The university offers 40 baccalaureate degrees, and several degrees in law, masters, and doctorate programs. The university comprises seven different academic colleges. (3)

    • ABC RNA

      Don’t drop your beaker! I personally liked the tone. A young
      company promoting itself without taking pot shots at its competitors. Keep in mind that this is coming out at the start of Bio 2014… which is in fact in San Diego.

      The table is informative too.