16:04 , Jul 16, 2018 |  BC Extra  |  Preclinical News

On-target mutations latest worry for CRISPR stocks

On the heels of preclinical studies evaluating off-target mutations driven by CRISPR-Cas9 gene editing, geneticist Allan Bradley’s group from the Wellcome Sanger Institute shifted the spotlight to on-target mutations that could result from the technology....
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...
17:54 , Nov 1, 2017 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: October 2017

New Therapeutic Targets and Biomarkers: October 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during October 2017. Therapeutic targets are defined as any protein, gene or other molecule...
19:44 , Oct 3, 2017 |  BC Innovations  |  Distillery Therapeutics

Cancer

INDICATION: Breast cancer In vitro, cell culture and mouse studies identified a thiophenecarboxamide-based PI4K2A inhibitor that could help treat breast cancer. In silico screening of a small molecule library for molecules that bound PI4K2A and in...
23:03 , Jun 29, 2017 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: June 2017

New Therapeutic Targets and Biomarkers: June 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during June 2017. Therapeutic targets are defined as any protein, gene or other molecule...
16:05 , Jun 27, 2017 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Alzheimer's disease (AD) Fruit fly studies suggest inhibiting EFR3A or PI4KA could help treat AD. In a Drosophila model of AD, knockout of the fruit fly homolog of EFR3A, knockout of PI4KA or a PI4KA...
13:23 , May 31, 2017 |  BC Innovations  |  Distillery Therapeutics

Musculoskeletal

INDICATION: Muscular dystrophy Patient sample, zebrafish and dog studies suggest inhibiting PITPNA could help treat Duchenne muscular dystrophy (DMD). In primary myoblasts derived from DMD patients, PITPNA knockdown increased fusion index in muscle fibers, a measure...
07:00 , Mar 24, 2016 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Phosphatidylinositol transfer protein cytoplasmic 1 (PITPNC1)

Cancer INDICATION: Cancer Patient sample and mouse studies suggest inhibiting PITPNC1 could help treat metastatic cancers. In patients with breast cancer, colon cancer or melanoma, levels of PITPNC1 were higher in primary tumors than in the corresponding...
07:00 , Sep 10, 2015 |  BC Innovations  |  Tools & Techniques

Undiscovered country

Although total RNA sequencing can identify oncogenes caused by the fusion of two genes, the method is inefficient and costly because the transcripts from the abnormal genes are often rare. NuGen Technologies Inc. has developed...
07:00 , May 14, 2015 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Phosphatidylinositol 4-kinase catalytic β (PI4KB; PI4KIIIβ)

Infectious disease INDICATION: Viral infection In vitro studies suggest inhibitors of PI4KB could help treat RNA virus infections. Chemical synthesis and in vitro testing of pyrazolopyrimidine analogs identified multiple compounds that selectively inhibited PI4KB with IC50 values...