uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

Publication date: Apr 09, 2019

uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease ~ On Track to Treat First Patient in Phase I/II Study of AMT-130 in 2H19 ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 08, 2019 (GLOBE NEWSWIRE) — uniQure N. V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U. S. Food and Drug Administration (FDA) has granted Fast Track designation for AMT-130, the Company’s gene therapy candidate for the treatment of Huntington’s disease.

“Achieving Fast Track Designation from the FDA underscores the high unmet medical need for patients suffering from Huntington’s disease, for which there are currently no approved, disease-modifying treatments,” stated Matt Kapusta, chief executive officer of uniQure.

“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. “

Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators’ clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading “Risk Factors” in uniQure’s Annual Report on Form 10-K filed on February 28, 2019.

Concepts Keywords
AMSTERDAM Frequent meetings communications
AMT Severe genetic diseases
Autosomal Dominant Neurodegenerative disorder
Brain Onset slow disease
Cassette Health
Chief Executive Officer Articles
Cognitive Fast track
Etiology Therapy
Exon Accelerated approval
Fast Track Designation
FDA
Gene
Genetic Diseases
Hemophilia
Huntingtin
Huntingtin Protein
Huntington
Intellectual Property
LEXINGTON
MicroRNA
Muscle
Mutant
NASDAQ
NDA
Netherlands
Neurodegenerative Disorder
Pipeline
Recombinant
United States
Vector

Semantics

Type Source Name
disease MESH abnormalities
disease MESH cognitive decline
disease MESH hemophilia
drug DRUGBANK Nonoxynol-9
gene UNIPROT TNFSF13
gene UNIPROT ANP32B
gene UNIPROT FBN1
gene UNIPROT AMT
drug DRUGBANK Alpha-methyltryptamine
disease MESH genetic diseases
drug DRUGBANK Coenzyme M
pathway BSID Release
gene UNIPROT SSRP1
disease MESH Risk Factors
gene UNIPROT FASTK
gene UNIPROT TMEM79
disease MESH neurodegenerative disorder
gene UNIPROT RNMT
gene UNIPROT MET
gene UNIPROT SLTM
disease MESH development
drug DRUGBANK Methionine
disease MESH suffering

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