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- || DYNE-251 / Dyne Therap
Trial completion date, Trial primary completion date: DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov) - Oct 23, 2024
P1/2, N=88, Recruiting, Sponsor: Dyne Therapeutics
Trial completion date: Nov 2026 --> Nov 2029 | Trial primary completion date: Nov 2026 --> Nov 2029
- ||| DYNE-101 / Dyne Therap
Enrollment change: ACHIEVE: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Sep 25, 2024
P1/2, N=104, Recruiting, Sponsor: Dyne Therapeutics
Trial completion date: Nov 2026 --> Nov 2029 | Trial primary completion date: Nov 2026 --> Nov 2029 N=72 --> 104
- DYNE-251 / Dyne Therap
Initial Data from the DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping (Colorado Convention Center | Bluebird 1A) - Mar 8, 2024 - Abstract #AAN2024AAN_2660;
N=72 --> 104 BackgroundObjectiveDesign/MethodsConclusions
- ||| DYNE-251 / Dyne Therap
Enrollment change: DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov) - Mar 7, 2024
P1/2, N=88, Recruiting, Sponsor: Dyne Therapeutics
BackgroundObjectiveDesign/MethodsConclusions N=48 --> 88
- DYNE-101 / Dyne Therap
ACHIEVE, a Randomized, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-101 in Individuals with DM1 (ePosters Virtual) - Apr 21, 2023 - Abstract #EAN2023EAN_284;
- DYNE-101 / Dyne Therap
ACHIEVE Trial, a Randomized, Placebo-Controlled, Multiple Ascending Dose Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1 (DM1) (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_3154;
N=48 --> 88 Background Objective Design/Methods Results Conclusions
- || Review, Journal: The myotonic dystrophy type 1 drug development pipeline: 2022 edition. (Pubmed Central) - Mar 2, 2023
Three interventional first-in-human clinical trials got underway with distinct drug classes, namely AOC 1001 and DYNE-101 nucleic acid-based therapies, and the small molecule pitolisant, which joins the race toward market authorization with other repurposed drugs, including tideglusib, metformin, or mexiletine, already in clinical evaluation. Furthermore, newly disclosed promising preclinical data for several additional nucleic-acid therapeutic candidates and a CRISPR-based approach, as well as the advent into the pipeline of novel therapeutic programs, increase the plausibility of success in the demanding task of providing valid treatments to patients with DM1.
- ||| DYNE-101 / Dyne Therap
Enrollment open: ACHIEVE: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Sep 2, 2022
P1/2, N=64, Recruiting, Sponsor: Dyne Therapeutics
Furthermore, newly disclosed promising preclinical data for several additional nucleic-acid therapeutic candidates and a CRISPR-based approach, as well as the advent into the pipeline of novel therapeutic programs, increase the plausibility of success in the demanding task of providing valid treatments to patients with DM1. Not yet recruiting --> Recruiting
- || DYNE-251 / Dyne Therap
New P1/2 trial: DELIVER: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (clinicaltrials.gov) - Sep 1, 2022
P1/2, N=46, Recruiting, Sponsor: Dyne Therapeutics
- Building a FORCETM platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping (Poster area - Ballroom B1-B2) - Aug 20, 2022 - Abstract #WMS2022WMS_306;
FORCE-M23D is a mouse-specific Fab-PMO conjugate designed to target TfR1 and skip exon 23 of the murine Dmd transcript to restore dystrophin expression in the mdx mouse model of DMD...Additionally, Dyne's clinical candidate, DYNE-251, a Fab-conjugated PMO designed to skip DMD exon 51, was evaluated in non-human primates (NHPs)...Lastly, we have begun development of FORCE conjugates for the treatment of exon 53 and exon 45 skipping amenable patients...This conjugate resulted in superior skipping of exon 53 compared to unconjugated PMO. Collectively, these data support utilizing the FORCE platform for the development of therapies for individuals with DMD mutations amenable to exon skipping.
- | FORCE-M23D / Dyne Therap
Preclinical, Journal: Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice. (Pubmed Central) - Aug 10, 2022
Importantly, FORCE-M23D treatment resulted in improved functional outcomes compared with administration of unconjugated M23D. Our results suggest that FORCE conjugates are a potentially effective approach for the treatment of DMD.
- | DYNE-101 / Dyne Therap
New P1/2 trial: ACHIEVE: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (clinicaltrials.gov) - Aug 1, 2022
P1/2, N=64, Not yet recruiting, Sponsor: Dyne Therapeutics