Last update 09 Aug 2025

JLK-247

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
AAV-TT coHGSNAT gene therapy (Phoenix Nest)
Target
HGSNAT
Action
modulators
Mechanism
HGSNAT modulators(heparan-alpha-glucosaminide N-acetyltransferase modulators), Gene transference(Gene transference)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseSkin and Musculoskeletal Diseases+ [1]
Active Indication
Mucopolysaccharidosis III
Inactive Indication-
Originator Organization
The University of ManchesterThe University of Manchester
Active Organization
UT Southwestern Medical CenterUT Southwestern Medical CenterPhoenix Nest, Inc.Phoenix Nest, Inc.
Inactive Organization-
License Organization
Phoenix Nest, Inc.Phoenix Nest, Inc.The University of ManchesterThe University of Manchester
Drug Highest PhasePreclinical
First Approval Date-
Regulation-

Related

100 Clinical Results associated with JLK-247
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100 Translational Medicine associated with JLK-247
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100 Patents (Medical) associated with JLK-247
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100 Deals associated with JLK-247
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Mucopolysaccharidosis IIIPreclinical
United States
UT Southwestern Medical CenterUT Southwestern Medical Center
13 May 2025
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Clinical Result

Indication
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Approval

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Regulation

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