Rare Diseases

Rare Diseases
1
Pipeline Programs
6
Companies
6
Clinical Trials
1 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
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

6 companies ranked by most advanced pipeline stage

Swedish Orphan Biovitrum
1 program
1
NI-0501Phase 11 trial
Active Trials
NCT04765553CompletedEst. Jul 2021
Genomics
GenomicsUK - Oxford
2 programs
Trio Whole Genome Sequencing and Participant-Specific ResearchN/A
clinical whole genome sequencingN/A1 trial
Active Trials
NCT03290469Completed355Est. Jan 2020
Recordati
RecordatiFrance - Saint-Victor
1 program
Colombia National Porphyria RegistryN/A1 trial
Active Trials
NCT05496933Unknown100Est. Dec 2022
AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
1 program
Epidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in RussiaN/A1 trial
Active Trials
NCT07247292Suspended100Est. Dec 2029
Precision BioSciences
1 program
Trio Whole Genome Sequencing and Participant-Specific ResearchN/A1 trial
Active Trials
NCT04586075Recruiting500Est. Oct 2030
Plus Therapeutics
1 program
dual expert guidance structureN/A1 trial
Active Trials
NCT03563677CompletedEst. Sep 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Swedish Orphan BiovitrumNI-0501
AstraZenecaEpidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia
Precision BioSciencesTrio Whole Genome Sequencing and Participant-Specific Research
RecordatiColombia National Porphyria Registry
Plus Therapeuticsdual expert guidance structure
Genomicsclinical whole genome sequencing

Clinical Trials (6)

Total enrollment: 1,055 patients across 6 trials

NCT04765553Swedish Orphan BiovitrumNI-0501

A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.

Start: Jan 2021Est. completion: Jul 2021
Phase 1Completed
NCT07247292AstraZenecaEpidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia

Epidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia

Start: Dec 2025Est. completion: Dec 2029100 patients
N/ASuspended
NCT04586075Precision BioSciencesTrio Whole Genome Sequencing and Participant-Specific Research

UW Undiagnosed Genetic Diseases Program

Start: Jul 2021Est. completion: Oct 2030500 patients
N/ARecruiting
NCT05496933RecordatiColombia National Porphyria Registry

Colombia National Porphyria Registry

Start: Apr 2021Est. completion: Dec 2022100 patients
N/AUnknown
NCT03563677Plus Therapeuticsdual expert guidance structure

Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases

Start: Oct 2018Est. completion: Sep 2022
N/ACompleted
NCT03290469Genomicsclinical whole genome sequencing

NICUSeq: A Trial to Evaluate the Clinical Utility of Human Whole Genome Sequencing (WGS) Compared to Standard of Care in Acute Care Neonates and Infants

Start: Sep 2017Est. completion: Jan 2020355 patients
N/ACompleted

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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

1 actively recruiting trials targeting 1,055 patients
6 companies competing in this space

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