BioCentury | Apr 25, 2019
Translation in Brief

Risking CRISPR correction before birth

...4-hydroxyphenylpyruvate dioxygenase; SP-C (SFTPC) - surfactant protein C Mary Romeo, Staff Writer Children's Hospital of Philadelphia University of Pennsylvania 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Oct 11, 2018
Distillery Therapeutics

Endocrine/metabolic

...base editing system in models of tyrosinemia type I using clinically relevant delivery vectors. TARGET/MARKER/PATHWAY: 4-hydroxyphenylpyruvate dioxygenase...
...H. Peranteau, Children’s Hospital of Philadelphia, Philadelphia, Pa. email: peranteauw@email.chop.edu Mark Zipkin Children's Hospital of Philadelphia University of Pennsylvania 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Oct 10, 2018
Preclinical News

In utero base editing corrects metabolic birth defect

...Hereditary tyrosinemia type I is treated with Orfadin nitisinone, a small molecule competitive inhibitor of 4-hydroxyphenylpyruvate dioxygenase...
...diseases that lack an established therapeutic avenue. Mark Zipkin Orfadin, nitisinone oral suspension Children's Hospital of Philadelphia University of Pennsylvania 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Oct 13, 2017
Clinical News

FDA approves once-daily dosing for Orfadin

...for the indication with recommended twice-daily dosing. Orfadin is a synthetic reversible competitive inhibitor of 4-hydroxyphenylpyruvate dioxygenase...
...SIX:SYNN; NYSE:SYT), Basel, Switzerland Product: Orfadin nitisinone oral suspension Business: Endocrine/Metabolic Alicia Parker Swedish Orphan Biovitrum AB Syngenta AG 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Jul 21, 2016
Translation in Brief

Survival of the modified

...involves blocking metabolite accumulation by using Orfadin nitisinone - an inhibitor of the upstream enzyme 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Jun 30, 2016
Distillery Techniques

Techniques: In vivo selection of genetically modified hepatocytes to improve efficacy of hepatocyte-targeting gene therapies

...co-administering a liver-targeted, genome-integrating vector encoding the therapeutic gene of interest and an shRNA targeting 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | May 2, 2016
Clinical News

Nitisinone oral suspension regulatory update

...company plans to launch the new formulation of Orfadin, a synthetic reversible competitive inhibitor of 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Sep 21, 2015
Clinical News

Oral suspension Orfadin nitisinone regulatory update

...and neurological problems. The company said it expects a decision from FDA in 2Q16. The 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Jul 13, 2015
Clinical News

Oral suspension Orfadin nitisinone regulatory update

...that can cause liver failure, kidney dysfunction and neurological problems. The company will launch the 4-hydroxyphenylpyruvate dioxygenase...
BioCentury | Jan 6, 2014
Company News

Swedish Orphan Biovitrum, Syngenta, Rare Disease Therapeutics sales and marketing update

...for Latin America. Swedish Orphan said financial terms were not disclosed. Swedish Orphan markets the 4-hydroxyphenylpyruvate dioxygenase...
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