The rapid development of mRNA vaccines to combat COVID-19 has highlighted the importance of this single-stranded molecule. Although there have been some costly developmental bumps along the way, interest in RNA-targeted therapies is once again surging.
RNA has “huge potential” as a therapeutic modality and is beginning to deliver on that potential. But “manufacturing RNA has issues in production, delivery and performance,” Thomas Barnes told BioWorld. Barnes is the CEO of startup Orna Therapeutics LLC, which has the goal of addressing those issues with oRNA, an engineered form of circular RNA.
By combining synthetic biology and RNA therapy, the team at startup Strand Therapeutics Inc. hopes to make mRNA therapy more effective. Strand recently announced an immuno-oncology deal with Beigene Ltd. that netted the company $5 million to begin with and could end up being worth more than $250 million. Beyond immuno-oncology, the company’s basic technology could be broadly useful for both mRNA- and cell-based therapies.
Pointing to his company’s work on “the next frontier in small molecules,” Ribometrix Inc. CEO Mike Solomon said the potential $1 billion-plus deal with Roche Holding AG offers evidence of big pharma’s mounting interest. Expertise in RNA folds helped Ribometrix nail down an agreement with Basel, Switzerland-based Roche’s Genentech arm that brings $25 million up front and potential milestone payments beyond $1 billion, as the pair discovers and develops RNA-targeted small-molecule therapeutics. Genentech gains exclusive rights to several predefined targets, including an exclusive global license for the development and commercialization of molecules.
Researchers at the University of California at San Diego have used RNA-targeted CRISPR to reverse symptoms in an animal model of myotonic dystrophy type 1 (DM1). They reported their findings in the Sept. 14, 2020, issue of Nature Biomedical Engineering.
Nessan Bermingham, Korro Bio Inc.’s co-founder and executive chair, told BioWorld that other groups are looking into adenosine deaminase acting on RNA (ADAR) – the body’s natural base editing system – but “we’re the first group, as far as I’m aware” to raise big money and bring together what could become the best technology for developing drugs based on an understanding of ADAR.
Chinese scientists have shown for the first time that the down-regulation of a single RNA-binding protein, polypyrimidine tract-binding protein 1 (Ptbp1), locally converted glial cells to neurons and showed promise for treating the symptoms of neurodegenerative diseases in mice.
Arrakis Therapeutics Inc.’s CEO, Michael Gilman, knew his 3-year-old company had a partnering tiger by the tail just by the amount of interest from companies who wanted to partner. He sat back and waited until the right offer came along and went with Roche Holding AG. The result is a massive collaboration and license agreement that could stretch into the billions of dollars. “We very deliberately stayed out of partnering discussions for the first couple years,” Gilman told BioWorld. “It took a while to figure out how to do this. We wanted to understand what we had before selling off parts of it.”
Boulder, Colo.-based startup Arpeggio Biosciences Inc. scooped up $3.2 million in seed funding in a round led by Khosla Ventures, with participation by Fundersclub, Fifty Years, TechU and Y Combinator.