Last update 04 Jun 2025

CRISPR-Cas9 ABE

Overview

Basic Info

Drug Type
Transformer Base Editor
Synonyms
k-abe
Target
CPS1
Action
activators
Mechanism
CPS1 activators(Carbamoyl phosphate synthetase 1 activators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Carbamoyl-Phosphate Synthase I Deficiency Disease
Inactive Indication-
Originator Organization
Acuitas Therapeutics, Inc.Acuitas Therapeutics, Inc.Danaher Corp.Danaher Corp.
Active Organization
Danaher Corp.Danaher Corp.Acuitas Therapeutics, Inc.Acuitas Therapeutics, Inc.
Inactive Organization-
License Organization-
Drug Highest PhaseClinical
First Approval Date-
Regulation-

Related

100 Clinical Results associated with CRISPR-Cas9 ABE
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100 Translational Medicine associated with CRISPR-Cas9 ABE
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100 Patents (Medical) associated with CRISPR-Cas9 ABE
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100 Deals associated with CRISPR-Cas9 ABE
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Carbamoyl-Phosphate Synthase I Deficiency DiseaseClinical
United States
Danaher Corp.Danaher Corp.
15 May 2025
Carbamoyl-Phosphate Synthase I Deficiency DiseaseClinical
Canada
Acuitas Therapeutics, Inc.Acuitas Therapeutics, Inc.
15 May 2025
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

No Data

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Translational Medicine

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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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