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.

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.

PARIS – Curadigm SAS has raised $1 million in non-dilutive funding from Bpifrance (Banque publique d’investissement in France), for development of its Nanoprimer technology. Bpifrance’s Deep Tech program in France recognizes health tech companies with breakthrough innovation and strong commercial potential.

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.