Myotonic dystrophy type 1 - Articles - Page 3

Avidity muscles into positive phase I/II data with lead candidate AOC-1001

Dec. 14, 2022

A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”

Neurology/Psychiatric

Arthex Biotech advances ATX-01 toward IND filing for DM1

Dec. 14, 2022

Arthex Biotech SA has achieved key regulatory milestones in its program to develop ATX-01 in myotonic dystrophy type 1 (DM1). Having held a pre-IND meeting with the FDA last year and received scientific advice from the EMA, Arthex plans to file an IND application in the U.S. and a clinical trial application (CTA) in Europe next year.

Neurology/Psychiatric

Vertex and Entrada establish collaboration focused on EEV therapeutics for DM1

Dec. 9, 2022

Vertex Pharmaceuticals Inc. and Entrada Therapeutics Inc. have entered into a global collaboration focused on discovering and developing intracellular Endosomal Escape Vehicle (EEV) therapeutics for myotonic dystrophy type 1 (DM1).

Neurology/Psychiatric

Pepgen reports new data from IND-enabling studies of PGN-EDODM1 for DM1

Dec. 9, 2022

Pepgen Inc. has announced new preclinical data supporting the progression into clinical trials of PGN-EDODM1, its product candidate in development for the treatment of myotonic dystrophy type 1 (DM1).

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.

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.

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.

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.

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.

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.