About
Oligonucleotide Therapy
Areas of Focus
Categories
Gene Therapy, RNA & Peptide TherapyBoston & New England
Clinical Trials (7)
View allTotal Trials
7
As Lead Sponsor
7
As Collaborator
0
Total Enrollment
170
By Phase
PHASE1: 2PHASE2/PHASE3: 2PHASE1/PHASE2: 3
By Status
COMPLETED: 3ACTIVE_NOT_RECRUITING: 2WITHDRAWN: 1UNKNOWN: 1
Clinical Trials Pipeline7 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
2014
2015
2016
2017
2018
2019
2020
2021
2022
2023
2024
Today
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Recent Trials
NCT05052554Lead Sponsor
Study With QR-504a to Evaluate Safety, Tolerability & Corneal Endothelium Molecular Biomarker(s) in Subjects With FECD3
PHASE1Withdrawn
Started: Aug 10, 2021 · Completed: Apr 12, 2022
NCT04855045Lead Sponsor
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
PHASE2/PHASE3Unknown
15 participants
Started: Mar 23, 2021 · Completed: Dec 31, 2023
NCT04123626Lead Sponsor
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
PHASE1/PHASE2Active, not recruiting
11 participants
Started: Oct 7, 2019 · Completed: Jun 7, 2022
NCT03913143Lead Sponsor
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
PHASE2/PHASE3Active, not recruiting
36 participants
Started: Apr 4, 2019 · Completed: Mar 31, 2023
NCT03780257Lead Sponsor
Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene
PHASE1/PHASE2Completed
20 participants
Started: Mar 6, 2019 · Completed: Oct 14, 2021
Locations (1)
Headquarters
Cambridge, MA, United States
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