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Arcturus emerging as a strong player in RNA therapeutics space

With a fast-growing pipeline of siRNA and mRNA drugs, San Diego startup Arcturus Therapeutics is poised join the ranks of more established RNA-based drug companies like Alnylam and Moderna Therapeutics. It’s welcome timing, as the market’s cast a keen eye on RNA therapies. Once a biotech underdog, scientists are finding that the gene modifying potential of these RNA therapeutics can alter the course of disease […]

With a fast-growing pipeline of siRNA and mRNA drugs, San Diego startup Arcturus Therapeutics is poised join the ranks of more established RNA-based drug companies like Alnylam and Moderna Therapeutics.

It’s welcome timing, as the market’s cast a keen eye on RNA therapies. Once a biotech underdog, scientists are finding that the gene modifying potential of these RNA therapeutics can alter the course of disease at its root.

Arcturus’ core platform, called LUNAR, uses fat molecules as vessels to transport its RNA-based drugs – primarily using small interfering RNA, or siRNA. They make it to their requisite genetic destination because this platform incorporates Unlocked Nucleic Acid (UNA) chemistry into the drug design, CEO Joseph Payne said.

The company’s data has advanced to show that this siRNA approach not only works in primates, but has demonstrated longevity in its efficacy. Here’s how Payne describes how it works:

 

“Imagine a spigot spinning off mutated proteins it’s not supposed to – that’s what happens in many genetic defects,” Payne said. “siRNAs can turn that off.”

Acturus’ lead candidate, LUNAR-101, is going after TTR-limited amyloidosis (so’s Alnylam, actually), using an siRNA-based mechanism.

“Our technologies provide us the freedom to operate on the 20,000 genes in the body,” Payne said. “We can go after these genes with a knockdown technology, or suppress the activity of these genes with siRNA. Our platform can increase the gene expression of weak, or missing, genes.”

The company was previously incubated at J-Labs, but recently graduated to a new lab on Pfizer’s La Jolla campus. On the books, it’s raised about $7 million, Payne said – but private investments into the company bring Arcturus’ funding past $10 million. And it’s getting some industry cred: The two-year-old company was, for instance, one of just 30 private companies to present at this year’s J.P. Morgan healthcare investment conference.

Outside of siRNA, Arcturus is working on ways to evolve messenger RNA (mRNA), antisense and microRNA into oligotherapeutics.

“The holy grail of pharma companies is to be able to control gene expression,” Payne said.