BioCentury | Jun 17, 2020
Product Development

June 16 Quick Takes: Takeda out-licenses neurology pipeline to Neurocrine; plus Illumina challenge blocks BGI, FDA approves a pair of firsts and more

...should lead to better patient outcomes than its predecessor. Amicus Therapeutics Inc. (NASDAQ:FOLD), which has AT-GAA...
...RNA polymerase II BioCentury Staff teplizumab (MGA031, PRV-031) Ilaris, canakinumab (ACZ885) Zepsyre, lurbinectedin (PM01183, PM1183, tryptamicidin) avalglucosidase alfa, NeoGAA, Neo-GAA...
BioCentury | Mar 21, 2020
Finance

Design debuts with $45M series A, portfolio against repeat expansions

...an inherited nucleotide repeat mutation, Shah told BioCentury. In Friedreich’s ataxia, patients have hundreds of GAA...
...normal levels, Shah said. For Design's lead compound, one end recognizes and binds to a GAA...
...transcription elongation factor that links a polyamide compound with high affinity for large numbers of GAA...
BioCentury | May 29, 2019
Company News

Amicus bolsters rare disease gene therapy pipeline with expanded UPenn deal

...a mouse model of Pompe disease, an adeno-associated viral vector gene therapy encoding an engineered GAA...
...$0.16 to $12.15 on Wednesday. Targets: CDKL5 - Cyclin dependent kinase like 5; GAA - Acid alpha glucosidase Elizabeth...
BioCentury | Sep 14, 2018
Targets & Mechanisms

Repeat offenders

...ALS), Friedreich ataxia and early infantile epileptic encephalopathy, which result from excessive repeats of GGGGCC, GAA...
BioCentury | Feb 2, 2018
Company News

Amicus to build U.S. biologics facility

...therapy ATB200/ATT221 is in Phase I/II testing. The co-administered therapy comprises ATB200, a recombinant human GAA...
...AT2221, a pharmacological chaperone. Amicus Therapeutics Inc. (NASDAQ:FOLD), Cranbury, N.J. Business: Endocrine/Metabolic Chris Lieu ATB200/AT2221 Amicus Therapeutics Inc. Acid alpha glucosidase (GAA...
BioCentury | Jan 27, 2018
Company News

AbbVie gains on updated 2018 EPS guidance

...therapy ATB200/ATT221 is in Phase I/II testing. The co-administered therapy comprises ATB200, a recombinant human GAA...
BioCentury | Jan 19, 2018
Tools & Techniques

Synthetic expressions

...GAA repeats (dark circles) in the FXN gene that underlie the pathogenesis of Friedreich’s ataxia. GAA...
...concept could work in Friedreich’s ataxia, by reactivating RNA polymerase after it halts at the GAA...
...expression at GAA sites with paused RNA polymerases. “The molecule goes to many places with GAA...
BioCentury | Jan 12, 2018
Company News

Spark unveils gene therapy program for Pompe's

...using its liver-targeting adeno-associated virus (AAV) platform. The therapy, SPK-GAA, will use a novel acid alpha glucosidase (GAA...
...continuous secretion of transgene-derived GAA from the liver. Spark expects to complete IND-enabling experiments for SPK-GAA...
...Therapeutics Inc. (NASDAQ:ONCE), Philadelphia, Pa. Business: Endocrine/Metabolic Allison Johnson AT982 ATB200/AT2221 AVD-RD-03 Lumizyme SPK-GAA Amicus Therapeutics Inc. Audentes Therapeutics Inc. Avrobio Inc. Sanofi Spark Therapeutics Inc. Acid alpha glucosidase (GAA) Pompe's...
BioCentury | Jan 9, 2018
Company News

Spark to develop gene therapy for Pompe's

...using its liver-targeting adeno-associated virus (AAV) platform. The therapy, SPK-GAA, will use a novel acid alpha glucosidase (GAA...
...continuous secretion of transgene-derived GAA from the liver. Spark expects to complete IND-enabling experiments for SPK-GAA...
...on Monday and regained $0.25 to $14.21 on Tuesday. Allison Johnson ATB200 Myozyme neoGAA Amicus Therapeutics Inc. Genethon Sanofi Spark Therapeutics Inc. Acid alpha glucosidase (GAA) Pompe's disease SPK-GAA...
BioCentury | Jan 9, 2018
Distillery Therapeutics

Neurology

...containing 4 (BRD4) ligand tool compound conjugated to a polyamine that binds disease-associated, transcriptionally repressive GAA...
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