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Home » myotonic dystrophy type 1

Articles Tagged with ''myotonic dystrophy type 1''

Limb exam

Avidity searches for cause of SAE as FDA puts partial hold on dystrophy study

Sep. 27, 2022
By Lee Landenberger
A serious adverse event (SAE) in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s lead program. The action is centered on the phase I/II Marina study of AOC-1001, an antibody oligonucleotide conjugate for treating myotonic dystrophy type 1, the most common form of muscular dystrophy in adults.
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Hand holding dollar sign

Expansion raises $80M to develop drugs for neurodegenerative disease

Sep. 29, 2021
By Michael Fitzhugh
Expansion Therapeutics Inc., a company developing new drugs for severe RNA-mediated diseases based on the work of its scientific founder, Scripps Research chemistry professor Matthew Disney, has raised $80 million in a series B financing to identify and advance oral small-molecule candidates for the potential treatment of myotonic dystrophy type 1, amyotrophic lateral sclerosis and tauopathies.
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RNA

Small molecule targets same repeat expansion in distinct diseases

Nov. 11, 2020
By Subhasree Nag
"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.
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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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Building on previous AAV deals, Vertex partners with Affinia in a $1.6B agreement

April 27, 2020
By Lee Landenberger
Weeks after raising an oversubscribed $60 million series A, Affinia Therapeutics Inc., of Waltham, Mass., is collaborating with Vertex Pharmaceuticals Inc. in a far larger deal, one potentially worth more than $1.6 billion.
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AOC can you say? $100M flag flying proud: Avidity series C for muscle bids

Nov. 14, 2019
By Randy Osborne
Avidity Biosciences Inc. CEO Sarah Boyce told BioWorld it's "a little too soon" to say when the company's lead program in myotonic dystrophy type I might reach the clinic, but the firm's $100 million series C financing will help with that effort and the portfolio in rare muscle diseases, as the antibody-oligonucleotide conjugate (AOC) platform undergoes further development.
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