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Home » Topics » Science » RNA

RNA
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RNA

Promising RNA-targeted therapies emerging

March 17, 2021
By Peter Winter
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.
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RNA illustration

For therapeutic RNA, chasing its tail may be the way to go

Feb. 24, 2021
By Anette Breindl
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.
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Strand Therapeutics weaves together synthetic biology, mRNA

Feb. 1, 2021
By Anette Breindl
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.
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RNA strand

Ribometrix in potential $1B-plus Roche deal as more enter RNA fold

Jan. 6, 2021
By Randy Osborne
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.
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Dollar sign in piggy bank

Remix raises $65M series A to develop RNA modulators

Dec. 8, 2020
By Michael Fitzhugh
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.
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Muscle fibers

RNA-targeted CRISPR reverses myotonic dystrophy symptoms

Sep. 17, 2020
By Anette Breindl
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.
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Muscle fibers

RNA-targeted CRISPR reverses myotonic dystrophy symptoms

Sep. 15, 2020
By Anette Breindl
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.
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RNA illustration

A knight at the OPERA? Kraken the RNA code, Korro on (r)ADAR with $91.5M series A

Sep. 10, 2020
By Randy Osborne
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.
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Illustration of liver, intravenous therapeutics

Curadigm awarded $1.2M to support development of its nanotechnology platform

Aug. 25, 2020
By Bernard Banga
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.
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CRISPR Cas9 illustration

Making neurons from glia alleviates neurodegenerative diseases

April 17, 2020
By John Fox
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.
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