Gene Therapy Candidate AMT-130 Shows Promise in Preclinical Findings

Gene Therapy Candidate AMT-130 Shows Promise in Preclinical Findings

Publication date: Mar 07, 2019

This post was originally published on this site Encouraging new preclinical data on AMT-130, uniQure’s experimental gene therapy candidate for Huntington’s disease, were presented at the 14th Annual Huntington’s disease Therapeutics Conference. -Findings from our preclinical studies illustrate the therapeutic potential of AMT-130 in restoring function to damaged brain cells in Huntington’s disease and providing a safe and sustained reduction of mutant huntingtin protein with a gene therapy candidate that could be applicable to a broad patient population,” Sander van Deventer, MD, PhD, chief scientific officer at uniQure, said in a press release. -uniQure’s neuroimaging translational studies in non-human primates and Huntington’s disease patients shows the importance of careful, individualized neurosurgical planning and meticulous dosing parameters in delivering gene therapy products to the brain,” said Joseph J. Higgins, MD, vice president, clinical development at uniQure. In another poster titled -Virtual neurosurgical planning demonstrates the safety of deep brain gene delivery in patients with early manifest HD,” researchers used high-resolution magnetic resonance imaging (MRI) to identify the correct path through which they could guide micro-catheters for the targeted delivery of AMT-130 in affected regions of the brain. Scientific presentations by uniQure also included a talk by Pavlina Konstantinova, PhD, vice president, new therapeutic target discovery at uniQure, titled -Gene Therapy for Huntington’s Disease: Silencing the Villain,” as well as two more posters -Sustained mutant huntingtin lowering in the brain and cerebrospinal fluid of Huntington disease minipigs mediated by AAV5-miHTT gene therapy” and -Transfer of therapeutic miRNAs within extracellular vesicles secreted from Huntington’s disease iPSC-derived neurons. ” The post Gene Therapy Candidate AMT-130 Shows Promise in Preclinical Findings appeared first on Huntington’s Disease News.

Concepts Keywords
AMT MRI
Antibodies Antibodies
Biomarkers Spectroscopy
Brain Monoclonal antibody
California Gene Therapy
Catheters Magnetic resonance imaging
Cerebrospinal Fluid Antibody
Convection Huntingtin
Deventer Therapy
Extracellular Vesicles Immunology
FDA Immune system
Hippocampus Biotechnology
Huntingtin Clinical medicine
Huntington Biomarkers
Magnetic Resonance Spectroscopy Branches of biology
Memory Medicine
MicroRNA Experimental therapy
Molecule Catheters
MRI Disease
MRNA Gene Therapy
Mutant
Mutation
Nerve Cells
Neurodegenerative Condition
Neuroimaging
Neurons
Neurosurgical
Orphan Drug Status
Palm Springs
PhD
Primates
Protein
Resonance
Serum
Spinal Cord
Spinal Fluid
Striatum
Vector
Virus

Semantics

Type Source Name
drug DRUGBANK Alpha-methyltryptamine
gene UNIPROT AMT
gene UNIPROT SLC35G1
gene UNIPROT ARID1A
gene UNIPROT PALM
gene UNIPROT TNIP1
gene UNIPROT PDC
pathway BSID Release
gene UNIPROT HTT
gene UNIPROT SLC6A4
gene UNIPROT PIGS
gene UNIPROT SAMD11
gene UNIPROT MARS
gene UNIPROT SMIM10L2B
gene UNIPROT SMIM10L2A
gene UNIPROT CSF2
gene UNIPROT LAMC2
disease MESH development
gene UNIPROT CYREN
disease MESH Huntington disease
disease DOID Huntington disease

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