21:50 , May 7, 2019 |  BC Extra  |  Tools & Techniques

Imaging data boosts accuracy of 3-D chromosome models

The latest advance in mapping 3-D genomic structures has improved modeling by integrating an imaging technique with sequencing technology. 3-D chromatin architectures, which help regulate gene expression, are emerging targets for cancer and developmental disorders....
20:17 , May 3, 2019 |  BC Extra  |  Preclinical News

May 3 Preclinical Quick Takes: CRISPR, Cincinnati Children's, Chromatin

Small molecule CRISPR inhibitors are here  Two groups have reported small molecule inhibitors this week that could be used to control CRISPR-based gene editing, as the U.S. Defense Advanced Research Projects Agency looks for ways...
13:52 , Jan 29, 2019 |  BC Innovations  |  Distillery Therapeutics

Cancer

INDICATION: Melanoma Patient sample and mouse studies suggest promoting UCP2 expression could help treat melanoma. In patients, high tumor levels of UCP2 mRNA were associated with survival. In two mouse models of melanoma, tumor-specific overexpression...
18:51 , May 30, 2018 |  BC Innovations  |  Distillery Therapeutics

Neurology

INDICATION: Spinal muscular atrophy (SMA) In silico , in vitro , cell culture and Drosophila studies identified a small molecule targeting SMN2 mRNA that could help treat SMA by increasing levels of full length SMN2...
18:00 , Aug 24, 2017 |  BC Innovations  |  Emerging Company Profile

Heartfelt WISPER

HaYa Therapeutics is using antisense oligonucleotides to target the lncRNA WISPER to treat cardiac fibrosis caused by cardiovascular diseases such as myocardial infarction. Because WISPER expression is confined to the heart, the newco is betting...
21:34 , Aug 3, 2017 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: July 2017

New Therapeutic Targets and Biomarkers: July 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during July 2017. Therapeutic targets are defined as any protein, gene or other...
18:09 , Jul 10, 2017 |  BC Innovations  |  Distillery Therapeutics

Cardiovascular

INDICATION: Myocardial infarction (MI) Patient sample and mouse studies suggest antisense oligonucleotides (ASOs) targeting the long non-coding RNA (lncRNA) WISPER could help treat MI and MI-associated cardiac fibrosis. In intraventricular septum samples from aortic stenosis...
22:23 , Apr 6, 2017 |  BC Innovations  |  Targets & Mechanisms

Competing for growth

With little in the clinical pipeline for achondroplasia beyond BioMarin Pharmaceutical Inc. ’s late-stage vosoritide, startups Therachon AG and BioClin Therapeutics Inc. are rethinking how to tackle the disease. By targeting FGFR3 directly, the two...
07:00 , Sep 22, 2016 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Protein kinase B (AKT; AKT1; PKB; PKBA); microRNA-155 (miR-155)

Infectious disease INDICATION: Leishmaniasis Cell culture and mouse studies suggest inhibiting the miR-155 /AKT signaling pathway could help treat a virulent subtype of leishmaniasis. In mouse bone marrow macrophages, a strain of Leishmania guyanensis that...
07:00 , Jun 9, 2016 |  BC Innovations  |  Distillery Therapeutics

Therapeutics: Metabotropic glutamate receptor subtype 1 (mGluR1; GRM1); SH3 and multiple ankyrin repeat domains 3 (SHANK3; PROSAP2; SPANK-2)

Neurology INDICATION: Autism Mouse studies suggest positive allosteric modulators (PAMs) of mGluR1 could help treat autism spectrum disorders caused by SHANK3 haploinsufficiency. In a mouse model of SHANK3-deficient autism, an mGluR1 PAM increased synaptic transmissions...