01:50 , Sep 14, 2018 |  BC Innovations  |  Targets & Mechanisms

Repeat offenders

Dozens of intractable DNA repeat-driven diseases could boil down to a common problem: disrupted boundaries between chromatin domains, according to a study from the University of Pennsylvania. The results present new opportunities for diseases like...
20:21 , Feb 2, 2018 |  BC Week In Review  |  Company News

Amicus to build U.S. biologics facility

Amicus Therapeutics Inc. (NASDAQ:FOLD) plans to spend up to $200 million to build a new biologics facility in the U.S. for its Pompe's disease program, CEO and Chairman John Crowley told BioCentury. The company said...
00:07 , Jan 27, 2018 |  BC Extra  |  Company News

AbbVie gains on updated 2018 EPS guidance

AbbVie Inc. (NYSE:ABBV) gained $14.91 (14%) to $123.21 on Friday after increasing its full-year 2018 non-GAAP EPS guidance to $7.33-$7.43 from $6.37-$6.57. The pharma said the adjusted range reflects "the impact of U.S. tax reform...
03:28 , Jan 19, 2018 |  BC Innovations  |  Tools & Techniques

Synthetic expressions

Bringing synthetic biology to transcriptional regulation could catalyze a new generation of gene therapies that are more tunable, disease-specific and easy to deliver than the products dominating the field. The new constructs include synthetic promoters that...
16:28 , Jan 12, 2018 |  BC Week In Review  |  Company News

Spark unveils gene therapy program for Pompe's

Spark Therapeutics Inc. (NASDAQ:ONCE) said it is developing a preclinical gene therapy for Pompe's disease using its liver-targeting adeno-associated virus (AAV) platform. The therapy, SPK-GAA, will use a novel acid alpha glucosidase (GAA) transgene in-licensed...
22:47 , Jan 9, 2018 |  BC Extra  |  Company News

Spark to develop gene therapy for Pompe's

Spark Therapeutics Inc. (NASDAQ:ONCE) said it is developing a preclinical gene therapy for Pompe's disease using its liver-targeting adeno-associated virus (AAV) platform. The therapy, SPK-GAA, will use a novel acid alpha glucosidase (GAA) transgene in-licensed...
22:06 , Jan 9, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Ataxia Patient sample and cell culture studies suggest a synthetic transcription elongation factor that induces FXN expression could help treat Friedreich’s ataxia. The synthetic molecule consists of a small molecule bromodomain containing 4 (BRD4) ligand...
19:00 , Jan 4, 2018 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: December 2017

New Therapeutic Targets and Biomarkers: December 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during December 2017. Therapeutic targets are defined as any protein, gene or other molecule...
01:09 , Dec 13, 2017 |  BC Innovations  |  Distillery Therapeutics

Endocrine / Metabolic

INDICATION: Pompe's disease Mouse studies suggest gene therapies delivering secretion-optimized GAA variants could help treat Pompe’s disease. The gene therapies consist of a liver-tropic adeno-associated viral serotype 8 (AAV8) vector encoding one of two GAA variants...
22:59 , Nov 14, 2017 |  BC Innovations  |  Distillery Therapeutics

Endocrine / Metabolic

INDICATION: Pompe's disease Mouse studies suggest GAA-loaded nanoparticles could help treat Pompe’s disease without generating anti-GAA antibodies associated with resistance to enzyme replacement therapy. The nanoparticles consist of low-immunogenic anionic phosphatidylinositol liposomes loaded with GAA. In...