BC Extra | Jun 4, 2019
Tools & Techniques

OHSU's Mitalipov proposes path forward for germline gene editing

Germline gene editing pioneer Shoukhrat Mitalipov has weighed in on therapeutic use of the technology, writing in a Nature Medicine perspective that it should only be applied when it can convert a mutated gene to...
BC Innovations | Apr 25, 2019
Translation in Brief

Risking CRISPR correction before birth

A team from CHOP and UPenn has published its second study showing CRISPR-based gene editing can treat deadly diseases in the womb but has yet to address the safety repercussions for the mother and fetus....
BioCentury | Jan 26, 2019
Product Development

Target wide, test narrow in lupus

While last year’s spate of clinical misses in lupus leave the field still in search of optimal targets, the 2019 outlook isn’t all doom. Companies with shots on goal are using sharper endpoints and aiming...
BioCentury | Dec 1, 2018
Politics, Policy & Law

China’s germline growing pain

How Chinese regulatory authorities respond over the long term to the CRISPR baby furor could influence its industry’s success on the global stage, as domestic and international stakeholders seek reassurance that the authorities are on...
BioCentury | Nov 17, 2018
Product Development

MyoKardia’s myosin mechanism deconvolution

With preclinical proof of concept that its lead candidate mavacamten acts via two distinct mechanisms to treat cardiomyopathy, MyoKardia Inc. is ready to accelerate a pipeline of preclinical programs that exploit the mechanisms separately for...
BC Innovations | Oct 25, 2018
Targets & Mechanisms

Blueprint for selectivity

Blueprint Medicines Corp. is making the jump from cancer to rare disease with an inhibitor for the rare pediatric bone disease FOP that sets the company up against at least two competitors further ahead in...
BC Innovations | Sep 26, 2018
Distillery Techniques

Assays and screens; other

TECHNOLOGY: Cell-free assays; computational models A computational model based on mutations in plasma cell-free DNA (cfDNA) could help predict the time to progression in mCRC patients treated with anti-EGFR therapies. Construction of the model involves...
BC Innovations | Sep 10, 2018
Distillery Techniques

Disease models

TECHNOLOGY: Transgenics and knockouts PGK1-knockout mice expressing an SP-C variant could be used to study the biology of idiopathic pulmonary fibrosis (IPF) and screen for therapies. Mice with inducible knockout of PGK1 that systemically expressed...
BC Innovations | Aug 22, 2018
Translation in Brief

Confirming CRISPR

Two critiques of a 2017 human embryo gene editing study, including one suggesting large deletions at the target site misled researchers into thinking CRISPR-Cas9 corrected a genetic mutation, raise questions about what assays and screening...
BC Innovations | Mar 7, 2018
Distillery Therapeutics

Infectious disease

INDICATION: Coronavirus In vitro and cell culture studies identified a peptide inhibitor of coronavirus S protein that could help treat Middle East respiratory syndrome (MERS) coronavirus infection. Chemical synthesis and testing in HEK cell-based viral...
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