Genetic Disease
2
Pipeline Programs
2
Companies
5
Clinical Trials
2 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
1
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
EP
Eloxx PharmaceuticalsIsrael - Nes Tziona
2 programs1
1
ELX-02Phase 21 trial
ELX-02Phase 11 trial
Active Trials
GenomicsUK - Oxford
3 programsBeginNGS TestN/A1 trial
Genetic EnrolleesN/A1 trial
Whole genome sequencingN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Eloxx PharmaceuticalsELX-02
Eloxx PharmaceuticalsELX-02
GenomicsBeginNGS Test
GenomicsWhole genome sequencing
GenomicsGenetic Enrollees
Clinical Trials (5)
Total enrollment: 11,120 patients across 5 trials
A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
Start: Aug 2019Est. completion: Dec 2019
Phase 2Terminated
Phase 1 Study of ELX-02 in Healthy Adult Subjects
Start: Oct 2017Est. completion: Jul 2019
Phase 1Completed
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
Newborn Genomic Sequencing Pilot Study
Start: Mar 2023Est. completion: Nov 2024120 patients
N/ACompleted
Diagnostic Odyssey: Whole Genome Sequencing (WGS)
Start: Feb 2018Est. completion: Mar 20701,000 patients
N/ARecruiting
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
2 actively recruiting trials targeting 11,120 patients
2 companies competing in this space