It might be difficult to view the past year through anything other than a COVID-shaped hole. But 2020 brought some remarkable and impactful news for the biopharma sector that had little to do with the novel coronavirus. In this end-of-year recap, BioWorld takes a look at some of achievements and trends affecting the industry that were completely unrelated to – or, in some cases, in spite of – the COVID-19 pandemic.
Skyhawk Therapeutics Inc., one of the early leaders in developing small-molecule drugs that target RNA, closed out the year with yet another big pharma partnering deal, an alliance with Vertex Pharmaceuticals Inc., in which it is getting $40 million up front plus up to $2.2 billion more in milestones and royalties on product sales.
Remix Therapeutics Inc., a Cambridge, Mass.-based startup developing small-molecule RNA modulators, said it has raised $65 million in series A financing led by Foresite Capital.
A Polish/Dutch team of investigators has reported new insights into the structure of the SARS-CoV-2 genome that suggests it could be amenable to both small-molecule and oligonucleotide drugs.
"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.
