BioCentury | Oct 18, 2018
Distillery Techniques

Biomarkers; other

TECHNOLOGY: Tissue markers; computational models A computational model based on urine levels of 10 mRNA splice variants could help diagnose DM1. Transcriptomic analysis of cell-free RNA in urine samples from 36 DM1 patients, 28 healthy...
BioCentury | Mar 8, 2018
Emerging Company Profile

Splice time

Skyhawk Therapeutics Inc. is developing small molecules that manipulate the stability of the spliceosome during RNA processing to correct splicing defects that drive disease. Instead of focusing on genes in classic exon-skipping diseases, the newco...
BioCentury | Jan 3, 2018
Product R&D

Expanding small molecule horizons

With a platform boasting more than a decade of preclinical rigor and $55.3 million in series A funding, Expansion Therapeutics Inc. is the latest company to step into the RNA-binding small molecule arena. The newco...
BioCentury | Sep 14, 2017
Emerging Company Profile

RNA gets CRISPR

Two of the outstanding hurdles for CRISPR-based gene editing are the safety concerns of permanently changing the cell’s DNA and the efficacy limitations of relying on natural DNA repair mechanisms to fix the cuts. Locana...
BioCentury | Jun 29, 2017
Translation in Brief

New Therapeutic Targets and Biomarkers: June 2017

...Institute Distillery Therapeutics Liver cancer Muscleblind-like splicing regulator 3 (MBNL3) Patient sample, cell culture and mouse studies suggest inhibiting MBNL3...
BioCentury | Jun 7, 2017
Distillery Therapeutics

Cancer

...INDICATION: Liver cancer Patient sample, cell culture and mouse studies suggest inhibiting MBNL3 could help treat...
...apoptosis compared with normal MBNL3 expression. In two of the HCC cell lines, knockdown of MBNL3...
...could include identifying and testing MBNL3 inhibitors in models of HCC and liver cancer. TARGET/MARKER/PATHWAY: Muscleblind-like splicing regulator 3 (MBNL3)...
BioCentury | May 15, 2014
Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Dystrophia myotonica protein kinase (DMPK) In vitro and mouse studies identified a DMPK CUG repeat-binding inhibitor that could help treat myotonic dystrophy...
BioCentury | Oct 4, 2012
Distillery Techniques

Technology: Disease models

Approach Summary Licensing status Publication and contact information Disease models Mouse model for neurological features of myotonic dystrophy type 1 (DM1) A mouse model for DM1 could be useful for studying neurological features of the...
BioCentury | Oct 15, 2009
Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Muscleblind-like 1 (MBNL1); dystrophia myotonica protein kinase (DMPK) Studies in cell culture and in mice suggest that inhibitors of MBNL1 could help...
BioCentury | Sep 17, 2009
Distillery Therapeutics

Indication: Musculoskeletal disease

Indication Target/marker/ pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Dystrophia myotonica protein kinase (DMPK); muscleblind-like 1 (MBNL1) SAR studies identified inhibitors of RNA-protein interactions that could help treat myotonic dystrophy...
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