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Genetic Disease

0
Pipeline Programs
7
Companies
9
Clinical Trials
4 recruiting
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
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

6 companies ranked by most advanced pipeline stage

Genomics
GenomicsUK - Oxford
3 programs
BeginNGS TestN/A1 trial
Genetic EnrolleesN/A1 trial
Whole genome sequencingN/A1 trial
Active Trials
NCT06306521Recruiting10,000Est. Feb 2029
NCT03458962Recruiting1,000Est. Mar 2070
NCT06276348Completed120Est. Nov 2024
Human BioSciences
Human BioSciencesWV - Martinsburg
2 programs
Genetic Studies in the Amish and MennonitesN/A
NESTN/A
Genome & Company
Genome & CompanyKorea - Suwon
2 programs
Genetic Studies in the Amish and MennonitesN/A1 trial
NESTN/A1 trial
Active Trials
NCT00359580Completed157
NCT03420274Completed691Est. Jun 2023
Eloxx Pharmaceuticals
2 programs
ELX-02PHASE_11 trial
ELX-02PHASE_21 trial
Active Trials
NCT03309605Completed62Est. Jul 2019
NCT04069260Terminated3Est. Dec 2019
Autonomous Therapeutics
1 program
Genomic Sequencing and Personalized Treatment for Birth Defects in Neonatal Intensive Care UnitsN/A1 trial
Active Trials
NCT02551081Recruiting2,000Est. Dec 2025
Imagine Pharma
Imagine PharmaPA - Pittsburgh
1 program
Natural History of Type 1 Interferonopathies: Insights From a European CohortN/A1 trial
Active Trials
NCT07040774Recruiting500Est. Oct 2045

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Eloxx PharmaceuticalsELX-02
Eloxx PharmaceuticalsELX-02
Imagine PharmaNatural History of Type 1 Interferonopathies: Insights From a European Cohort
GenomicsBeginNGS Test
GenomicsWhole genome sequencing
GenomicsGenetic Enrollees
Genome & CompanyNEST
Autonomous TherapeuticsGenomic Sequencing and Personalized Treatment for Birth Defects in Neonatal Intensive Care Units
Genome & CompanyGenetic Studies in the Amish and Mennonites

Clinical Trials (9)

Total enrollment: 14,533 patients across 9 trials

NCT04069260Eloxx PharmaceuticalsELX-02

A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis

Start: Aug 2019Est. completion: Dec 20193 patients
Phase 2Terminated
NCT03309605Eloxx PharmaceuticalsELX-02

Phase 1 Study of ELX-02 in Healthy Adult Subjects

Start: Oct 2017Est. completion: Jul 201962 patients
Phase 1Completed
NCT07040774Imagine PharmaNatural History of Type 1 Interferonopathies: Insights From a European Cohort

Natural History of Type 1 Interferonopathies: Insights From a European Cohort

Start: Oct 2025Est. completion: Oct 2045500 patients
N/ARecruiting
NCT06306521GenomicsBeginNGS Test

An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing

Start: Feb 2024Est. completion: Feb 202910,000 patients
N/ARecruiting
NCT06276348GenomicsWhole genome sequencing

Newborn Genomic Sequencing Pilot Study

Start: Mar 2023Est. completion: Nov 2024120 patients
N/ACompleted
NCT03458962GenomicsGenetic Enrollees

Diagnostic Odyssey: Whole Genome Sequencing (WGS)

Start: Feb 2018Est. completion: Mar 20701,000 patients
N/ARecruiting
NCT03420274Genome & CompanyNEST

Ensuring Patients' Informed Access to Noninvasive Prenatal Testing

Start: Feb 2018Est. completion: Jun 2023691 patients
N/ACompleted
NCT02551081Autonomous TherapeuticsGenomic Sequencing and Personalized Treatment for Birth Defects in Neonatal Intensive Care Units

Genomic Sequencing and Personalized Treatment for Birth Defects in Neonatal Intensive Care Units

Start: Oct 2015Est. completion: Dec 20252,000 patients
N/ARecruiting
NCT00359580Genome & CompanyGenetic Studies in the Amish and Mennonites

Genetic Studies in the Amish and Mennonites

Start: Feb 2004157 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

4 actively recruiting trials targeting 14,533 patients
7 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.