"RNA was long thought to be an 'undruggable' target for small molecules, because most cellular RNAs have extensive secondary structure, but only limited tertiary structure," Matthew Disney told BioWorld Science.

RNAi therapeutics specialist Sirnaomics Inc. has wrapped up a $105 million series D financing that will support ongoing clinical development of its cancer and fibrotic disease programs plus work on early stage programs targeting metabolic disease and viral infections. But with both new and existing investors jumping in, the company is also preparing for something bigger: its next phase of growth, including a near-term IPO.

RNAi therapeutics specialist Sirnaomics Inc. has wrapped up a $105 million series D financing that will support ongoing clinical development of its cancer and fibrotic disease programs plus work on early stage programs targeting metabolic disease and viral infections. But with both new and existing investors jumping in, the company is also preparing for something bigger: its next phase of growth, including a near-term IPO.

Nutcracker Therapeutics Inc., a startup developing a "push-button GMP-in-a-box" platform to manufacture mRNA therapeutics, has raised $60 million in series B financing led by Arch Venture Partners.

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.

DUBLIN – Mina Therapeutics Ltd. raised £23 million (US$29.9 million) in a series A round to take forward a pipeline and a platform based on a novel regulatory RNA species, small activating RNAs, which selectively activate gene expression.

CEO and co-founder Rosana Kapeller told BioWorld that Rome Therapeutics Inc.’s $50 million series A will fund early work in an “exploding” new area of biology: the repeatome – “the dark genome” or “junk DNA,” as many previously deemed the roughly 60% of the human genome that consists of repetitive sequences of nucleic acids.

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.”

HONG KONG – South Korean startup Rznomics Inc. has recently secured ₩12 billion (US$10 million) in a series A investment. The Gwangju-based company focuses on cancer treatment development based on RNA platform technology.