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Primary Ciliary Dyskinesia

1
Pipeline Programs
5
Companies
7
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Parion Sciences
Parion SciencesNC - Durham
1 program
1
VX-371Phase 21 trial
Active Trials
NCT02871778Completed123Est. Nov 2018
ReCode Therapeutics
ReCode TherapeuticsCA - Menlo Park
4 programs
Sano Genetics Testing KitN/A1 trial
SpirometryN/A1 trial
RCT1100PHASE_11 trial
RCT1100PHASE_11 trial
Active Trials
NCT06172374Active Not Recruiting150Est. Dec 2025
NCT05685186Active Not Recruiting50Est. Jun 2025
NCT06600425Completed7Est. Aug 2025
+1 more trials
Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
2 programs
Genetic Testing for PCD or PIDN/A1 trial
Longitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary N/A1 trial
Active Trials
NCT04702243Completed436Est. Aug 2025
NCT05161858Completed105Est. Sep 2025
Sano Genetics
Sano GeneticsUK - Cambridge
1 program
Sano Genetics Testing KitN/A
Vertex Pharmaceuticals
1 program
VX-371PHASE_2

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Parion SciencesVX-371
ReCode TherapeuticsRCT1100
ReCode TherapeuticsRCT1100
ReCode TherapeuticsSano Genetics Testing Kit
ReCode TherapeuticsSpirometry
Colorado TherapeuticsLongitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary
Colorado TherapeuticsGenetic Testing for PCD or PID

Clinical Trials (7)

Total enrollment: 880 patients across 7 trials

NCT02871778Parion SciencesVX-371

Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia

Start: Aug 2016Est. completion: Nov 2018123 patients
Phase 2Completed
NCT06600425ReCode TherapeuticsRCT1100

A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD

Start: Sep 2024Est. completion: Aug 20257 patients
Phase 1Completed
NCT05737485ReCode TherapeuticsRCT1100

Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects

Start: Feb 2023Est. completion: Jan 20259 patients
Phase 1Completed
NCT06172374ReCode TherapeuticsSano Genetics Testing Kit

A Study Providing Genetic Testing to Find Those Who May Have Primary Ciliary Dyskinesia for Potential Clinical Trials

Start: Aug 2023Est. completion: Dec 2025150 patients
N/AActive Not Recruiting
NCT05685186ReCode TherapeuticsSpirometry

A Longitudinal, Observational Study of Primary Ciliary Dyskinesia in Adults

Start: Mar 2023Est. completion: Jun 202550 patients
N/AActive Not Recruiting
NCT05161858Colorado TherapeuticsLongitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary

Longitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary Ciliary Dyskinesia

Start: Mar 2022Est. completion: Sep 2025105 patients
N/ACompleted
NCT04702243Colorado TherapeuticsGenetic Testing for PCD or PID

Defining the Genetic Etiology of Suppurative Lung Disease in Children and Adults

Start: Dec 2020Est. completion: Aug 2025436 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

5 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.