BioCentury | Feb 24, 2020
Distillery Therapeutics

DICER1 gene therapy for choroidal neovascularization

DISEASE CATEGORY: Ophthalmic INDICATION: Choroidal neovascularization (CNV) An adeno-associated viral serotype 2 (AAV2)-based gene therapy delivering the RNase DICER1 could treat CNV. In mice with spontaneous CNV, subretinal injection of an AAV2 vector encoding a...
BioCentury | Feb 8, 2020
Tools & Techniques

Beyond PROTACs and the proteasome: broadening the TAC toolbox

...despite the name, RIBOTACs were more inspired by antisense oligos, which trigger RNA degradation via RNAse H...
BioCentury | Dec 6, 2019
Targets & Mechanisms

A view of immune reprogramming from ASH

ASH presentations on immune reprogramming point to new targets and mechanisms that control macrophage and T cell phenotype switching, and highlight the phenomenon's translational relevance in diseases beyond solid tumors. Strategies to convert immunosuppressive macrophage...
BioCentury | Jun 14, 2019
Clinical News

June 13 Clinical Quick Takes: Roche's Gazyva meets in Phase II for lupus; plus Boehringer/Lilly, Savara and more

Roche's Gazyva clears Phase II hurdle in lupus nephritis Gazyva obinutuzumab from Roche (SIX:ROG; OTCQX:RHHBY) met the primary endpoint in the Phase II NOBILITY trial to treat lupus nephritis. Given in combination with standard of...
BioCentury | Aug 23, 2018
Distillery Therapeutics

Cancer

INDICATION: Breast cancer Cell culture and mouse studies suggest inhibiting IRE1 or its RNase activity could help treat triple-negative breast cancer (TNBC). In a panel of human breast cancer cell lines, levels of IRE1 RNase...
BioCentury | Jul 11, 2018
Translation in Brief

The RNA Renaissance continues

Two papers from Matthew Disney’s lab at The Scripps Research Institute herald a pair of RNA-related discoveries: a method for engineering small molecules that target and cleave oncogenic microRNAs, and a platform that identified RNA-targeting...
BioCentury | Mar 29, 2018
Tools & Techniques

Cutting-edge diagnostics

Following the splash that CRISPR has made in the world of therapeutics, academics are expanding the tool kit of Cas enzymes and finding new uses in diagnostics. Early hints suggest the technology could become competitive,...
BioCentury | Dec 13, 2017
Preclinical News

Synthetic virus-like protein complexes could improve drug delivery

In a paper published in Nature , researchers at the University of Washington designed a synthetic RNA-protein complex that, like a virus, can package and protect its own RNA genome, suggesting a novel approach to targeted...
BioCentury | Sep 28, 2017
Product R&D

Wave’s purity progress

...The company described stereopure oligos with heightened stability and defined a stereochemical code for boosting RNAse H...
...has Kynamro approved for hypercholesterolemia. The ASO targets apolipoprotein B-100 (APOB-100) for degradation via an RNAse H-dependent...
...increase efficacy and defined a stereochemical code for engineering ASOs with both increased stability and RNAse H-dependent...
BioCentury | Aug 24, 2017
Emerging Company Profile

Heartfelt WISPER

...In the MI mouse model, chimeric DNA/locked nucleic acid (LNA) antisense oligonucleotides (ASOs) that induced RNase H...
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