Last update 25 Apr 2026

Rare Trait Hope Fund

Private Company|2013|
www.raretrait.com

Overview

Tags

Other Diseases
Congenital Disorders
Endocrinology and Metabolic Disease
AAV based gene therapy

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
Endocrinology and Metabolic Disease1
Top 5 Drug TypeCount
AAV based gene therapy1
Top 5 TargetCount
AGA(aspartylglucosaminidase)1

Related

1
Drugs associated with Rare Trait Hope Fund
AAV9-hAGA (Rare Trait Hope Fund)
Target
AGA
Mechanism
AGA modulators
Active Org.
Rare Trait Hope Fund
Originator Org.
Rare Trait Hope Fund
Active Indication
Aspartylglucosaminuria
Inactive Indication-
Drug Highest PhasePhase 1/2
First Approval Ctry. / Loc.-
First Approval Date-
1
Clinical Trials associated with Rare Trait Hope Fund
NCT07530796
/ Not yet recruitingPhase 1/2
An Open-Label, Single Center, Phase 1/2 Study to Evaluate the Safety and Efficacy of DANAGALEX (scAAV9/AGA) in Participants With Aspartylglucosaminuria (AGU)
100 Clinical Results associated with Rare Trait Hope Fund
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0 Patents (Medical) associated with Rare Trait Hope Fund
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100 Deals associated with Rare Trait Hope Fund
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100 Translational Medicine associated with Rare Trait Hope Fund
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Pipeline

Pipeline Snapshot as of 29 Apr 2026

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
Phase 2
1
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Current Projects

Drug(Targets)IndicationsGlobal Highest Phase
AAV9-hAGA (Rare Trait Hope Fund)
 ( AGA )		Aspartylglucosaminuria
More
Phase 1/2
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Translational Medicine

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Profit

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