FDA Places Gene Therapy AMT-130 on Fast Track to Speed Development

FDA Places Gene Therapy AMT-130 on Fast Track to Speed Development

Publication date: Apr 11, 2019

This post was originally published on this site The U. S. Food and Drug Administration (FDA) has given fast track designation to AMT-130, uniQure’s gene therapy candidate for the treatment of Huntington’s disease. -We are nearing the initiation of a Phase I/II study of AMT-130, the first one-time administered AAV gene therapy to enter clinical testing for Huntington’s disease, and are on track to treat the first patient in the second half of 2019,” Kapusta said. AMT-130 is an experimental gene therapy designed to block production of the mutated form of the huntingtin (HTT) protein, the underlying cause of Huntington’s disease. The post FDA Places Gene Therapy AMT-130 on Fast Track to Speed Development appeared first on Huntington’s Disease News.

Concepts Keywords
AMT Antibodies
Antibodies Aminomethyltransferase
Brain Huntington’s disease
Cerebrospinal Fluid Fast track
Chief Executive Officer Viral vector
Clinical Trial Huntingtin
Fast Track Designation Branches of biology
FDA Gene delivery
Genetic Life sciences
Genetic Material Huntington s disease
Hippocampus
Huntingtin
Huntingtin Protein
Huntington
Memory
MicroRNA
MiRNA
Molecule
Mutant
Nerve
Protein
Resonance
Spinal Cord
Spinal Fluid
Striatum
Vector
Virus

Semantics

Type Source Name
gene UNIPROT CYREN
gene UNIPROT PIGS
gene UNIPROT SLC6A4
gene UNIPROT HTT
gene UNIPROT SET
pathway BSID Release
gene UNIPROT TMEM79
disease MESH suffering
gene UNIPROT SLC35G1
drug DRUGBANK Alpha-methyltryptamine
gene UNIPROT AMT
gene UNIPROT FASTK
disease MESH Development

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