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Home » myotonic dystrophy type 1

Articles Tagged with ''myotonic dystrophy type 1''

Delivery of therapeutic oligonucleotide cargoes into tissues and cells

Pepgen rallies as early data suggest sporting chance in DM1

Feb. 24, 2025
By Randy Osborne
Pepgen Inc. seems to have gained a leg up on competitors in early data with PGN-EDODM1 in myotonic dystrophy type 1 (DM1), and shares of the Boston-based firm (NASDAQ:PEPG) closed Feb. 24 at $2.29, up 92 cents, or about 67%. The company unveiled initial positive data from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I study with PGN-EDODM1, which deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein.
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Dyne seeks accelerated path on positive dystrophy data; shares down

Jan. 10, 2025
By Lee Landenberger
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.
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3D cross-section illustration of muscle anatomy
Neurology/psychiatric

Arrakis reports preclinical data from RNA-targeted small-molecule drug program for DM1

Dec. 5, 2024
Arrakis Therapeutics Inc. has presented data on its RNA-targeted small-molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1). The company’s proprietary RNA‐specific chemical, biological and structural methods and RNA-directed medicinal chemistry enabled structure-based small-molecule drug design targeting the trinucleotide (CUG) repeat expansion in the mRNA of DMPK (myotonic dystrophy protein kinase) that drives DM1 pathology.
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Novartis acquires gene therapy developer Kate Therapeutics for $1.1B

Nov. 21, 2024
By Karen Carey
Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
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Person sitting in wheelchair

Dyne in: Wall Street sits down as phase I/II DM1, DMD table set

May 20, 2024
By Randy Osborne
Shares of Dyne Therapeutics Inc. (NASDAQ:DYN) closed May 20 at $35.38, up $7.70, or 28%, on word of positive data from the phase I/II Achieve trial of DYNE-101 in myotonic dystrophy type 1 (DM1) and the phase I/II Deliver effort with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. CEO John Cox, who joined Waltham, Mass.-based Dyne eight weeks ago, said he “couldn’t be more proud to be part of this team.” Studies are ongoing, but new data with regard to DM1 as well as DMD showed a “compelling” impact, Dyne said, plus satisfying safety profiles.
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3D cross-section illustration of muscle anatomy
Neurology/Psychiatric

Arthex’s ATX-01 receives FDA clearance for first-in-human study in myotonic dystrophy type 1

Feb. 28, 2024
Arthex Biotech SL has received FDA clearance to initiate the phase I/IIa Arthemir study of ATX-01 for the treatment of myotonic dystrophy type 1 (DM1).
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Neurology/Psychiatric

Juvena’s JUV-161 awarded US orphan drug designation for myotonic dystrophy type 1

Jan. 24, 2024
The FDA has awarded orphan drug designation to Juvena Therapeutics Inc.’s JUV-161, an...
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Dyne proves Force to be reckoned with in DM1, DMD as phase I/II validate platform

Jan. 3, 2024
By Randy Osborne
With positive initial phase I/II data in hand from two trials, Dyne Therapeutics Inc. plans to report more findings and start enrolling registrational cohorts in both studies by the end of this year for DYNE-101 in myotonic dystrophy type 1 (DM1) and a study called Deliver with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
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Limb exam
Neurology/Psychiatric

Pepgen’s PGN-EDODM1 cleared to enter clinic in Canada for DM1, receives US orphan drug designation

Sep. 7, 2023
Pepgen Inc. has received a no objection letter from Health Canada for its clinical trial application (CTA) to initiate a phase I study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Initial data from the study are expected next year.
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Astellas returns two gene therapies to Modalis

Aug. 15, 2023
By Marian (YoonJee) Chu
Modalis Therapeutics Corp. – previously Edigene Corp. – has regained full rights to two gene therapy candidates for muscle disorders, MDL-201 and MDL-202, that were co-developed with Astellas Pharmaceutical Inc. since 2019.
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