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- |||| delpacibart etedesiran (AOC 1001) / Avidity Biosci
Enrollment open: HARBOR: Global Study of Del-desiran for the Treatment of DM1 (clinicaltrials.gov) - Jun 12, 2024
P3, N=150, Recruiting, Sponsor: Avidity Biosciences, Inc.
Not yet recruiting --> Recruiting
- |||||||| delpacibart etedesiran (AOC 1001) / Avidity Biosci
New P3 trial: HARBOR: Global Study of Del-desiran for the Treatment of DM1 (clinicaltrials.gov) - May 13, 2024
P3, N=150, Not yet recruiting, Sponsor: Avidity Biosciences, Inc.
- Antibody Oligonucleotide Conjugates: Development of AOC 1001 for Myotonic Dystrophy () - Aug 9, 2023 - Abstract #ADCUSA2023ADC_USA_16;
Not yet recruiting --> Recruiting Introduction to oligonucleotides and AOC 1001; Exploring AOC 1001 Preclinical and Phase-1 clinical data; Discussing the next steps for oligonucleotide conjugates and AOC1001
- AOC 1001 / Avidity Biosci
Preliminary Assessment of the Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA) (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_4397;
P1/2
These results will be presented. Conclusions AOC 1001 represents a novel potential therapy addressing the underlying cause of DM1.
- || 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.
- | AOC 1001 / Avidity Biosci
Enrollment closed: MARINA: Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (clinicaltrials.gov) - Oct 17, 2022
P1/2, N=44, Active, not recruiting, Sponsor: Avidity Biosciences, Inc.
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. Recruiting --> Active, not recruiting
- AOC 1001 / Avidity Biosci
A phase 1/2 clinical trial evaluating the safety and pharmacokinetics of AOC 1001 in adults with myotonic dystrophy type 1: MARINA study design (Poster area - Ballroom B1-B2) - Aug 20, 2022 - Abstract #WMS2022WMS_446;
P1/2
The study will enroll 44 symptomatic adults aged 18 to 65 years with genetically confirmed DM1 (CTG repeat length ≥ 100). Eligible participants from parts A and B will have the option to participate in an open-label extension study.
- || AOC 1001 / Avidity Biosci
New P2 trial: MARINA-OLE: Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (clinicaltrials.gov) - Jul 29, 2022
P2, N=44, Enrolling by invitation, Sponsor: Avidity Biosciences, Inc.
- AOC 1001 / Avidity Biosci
Study Design of AOC 1001-CS1, a Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA) () - Mar 6, 2022 - Abstract #AAN2022AAN_2205;
P1/2
Eligible participants from parts A and B will have the option to participate in an open-label extension study. NA
- || AOC 1001 / Avidity Biosci
Enrollment open: MARINA: Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (clinicaltrials.gov) - Nov 10, 2021
P1/2, N=44, Recruiting, Sponsor: Avidity Biosciences, Inc.
NA Not yet recruiting --> Recruiting
- | AOC 1001 / Avidity Biosci
New P1/2 trial: MARINA: Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (clinicaltrials.gov) - Aug 30, 2021
P1/2, N=44, Not yet recruiting, Sponsor: Avidity Biosciences, Inc.
- AOC 1001 / Avidity Biosci
[VIRTUAL] Optimization of AOC-1001, an antibody-oligonucleotide conjugate targeting the underlying cause of myotonic dystrophy type 1 () - Apr 25, 2021 - Abstract #AAN2021AAN_3501;
Not yet recruiting --> Recruiting Based on these data, clinical investigation of AOC1001 in DM1 patients is planned.