Last update 08 Mar 2025
Cure Rare Disease, Inc.

Cure Rare Disease, Inc.

Startups
|
Private Company|
2017|
Massachusetts, United States
|
< 10
|
www.cureraredisease.org

Overview

Tags

Nervous System Diseases
Skin and Musculoskeletal Diseases
Other Diseases
CRISPR/Cas9
Gene editing

Disease domain score
A glimpse into the focused therapeutic areas
Technology Platform
Most used technologies in drug development
Targets
Most frequently developed targets
Disease DomainCount
Nervous System Diseases18
Top 5 Drug TypeCount
CRISPR/Cas913
Gene editing5
Top 5 TargetCount
ATXN3(ataxin 3)1
FKRP(Fukutin-related protein)1
DYSF(Dysferlin)1
telethonin(titin-cap)1
ADSS1(adenylosuccinate synthase 1)1

Related

18
Drugs associated with Cure Rare Disease, Inc.
Spinocerebellar Ataxia Type 3(CRD)
Target
ATXN3
Mechanism
ATXN3 gene modulators
Active Org.
Cure Rare Disease, Inc.Cure Rare Disease, Inc.Startup
Originator Org.
Cure Rare Disease, Inc.Cure Rare Disease, Inc.Startup
Active Indication
Spinocerebellar Ataxias
Inactive Indication-
Drug Highest PhasePreclinical
First Approval Ctry. / Loc.-
First Approval Date-
Exon 44 Duplication(CRD)
Target-
Mechanism-
Active Org.
Cure Rare Disease, Inc.Cure Rare Disease, Inc.Startup
Originator Org.
Cure Rare Disease, Inc.Cure Rare Disease, Inc.Startup
Active Indication
Muscular Dystrophies
Inactive Indication-
Drug Highest PhasePreclinical
First Approval Ctry. / Loc.-
First Approval Date-
Limb Girdle Muscular Dystrophy 2i(CRD)
Target
FKRP
Mechanism
FKRP stimulants
Active Org.
Cure Rare Disease, Inc.Cure Rare Disease, Inc.Startup
Originator Org.
Cure Rare Disease, Inc.Cure Rare Disease, Inc.Startup
Active Indication
Muscular Dystrophy, Limb-Girdle, Type 2I
Inactive Indication-
Drug Highest PhasePreclinical
First Approval Ctry. / Loc.-
First Approval Date-
1
Clinical Trials associated with Cure Rare Disease, Inc.
NCT05514249
/ Unknown statusPhase 1
Treatment of a Single Patient With CRD-TMH-001
100 Clinical Results associated with Cure Rare Disease, Inc.
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0 Patents (Medical) associated with Cure Rare Disease, Inc.
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1
Literatures (Medical) associated with Cure Rare Disease, Inc.
01 Oct 2023Human gene therapy
Cure Rare Disease: An Initiative to Enable N of 1 Gene Editing.
100 Deals associated with Cure Rare Disease, Inc.
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100 Translational Medicine associated with Cure Rare Disease, Inc.
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Pipeline

Pipeline Snapshot as of 04 Apr 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3
Preclinical
18
1
Other
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