Last update 11 Sep 2025

CRD-003

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Limb Girdle Muscular Dystrophy 2i(CRD), CRD 003, CRD003
Target
FKRP
Action
stimulants
Mechanism
FKRP stimulants(Fukutin-related protein stimulants)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]
Active Indication
Muscular Dystrophy, Limb-Girdle, Type 2I
Inactive Indication-
Originator Organization
Cure Rare Disease, Inc.Cure Rare Disease, Inc.
Active Organization
Cure Rare Disease, Inc.Cure Rare Disease, Inc.
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with CRD-003
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100 Translational Medicine associated with CRD-003
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100 Patents (Medical) associated with CRD-003
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100 Deals associated with CRD-003
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, Limb-Girdle, Type 2IPreclinical
United States
Cure Rare Disease, Inc.Cure Rare Disease, Inc.
11 Apr 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
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Deal

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Clinical Trial

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Approval

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Regulation

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