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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
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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/A1 trial
NESTN/A1 trial
Active Trials
NCT00359580Completed157
NCT03420274Completed691Est. Jun 2023
Genome & Company
Genome & CompanyKorea - Suwon
2 programs
Genetic Studies in the Amish and MennonitesN/A
NESTN/A
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
Human BioSciencesNEST
Autonomous TherapeuticsGenomic Sequencing and Personalized Treatment for Birth Defects in Neonatal Intensive Care Units
Human BioSciencesGenetic 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
NCT03420274Human BioSciencesNEST

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
NCT00359580Human BioSciencesGenetic 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.