BioCentury | Sep 7, 2019
Product Development

Regeneron’s evinacumab data could make it the therapy of choice in HoFH

With a cleaner safety profile and better efficacy than marketed agents, Regeneron’s first-in-class evinacumab is poised to become the standard of care for homozygous familial hypercholesterolemia and other rare lipid disorders, including for the hardest...
BioCentury | May 11, 2018

Amplifying oligos

The last five years have seen a steady stream of preclinical deals for antisense, siRNA and mRNA therapies that suggests pharmas are taking the modalities seriously as alternatives to traditional small molecules and antibodies. But...
BioCentury | Sep 28, 2017
Product R&D

Wave’s purity progress

Wave Life Sciences Ltd. ’s latest data releases provide the fullest view to date into how the company synthesizes its stereopure oligos and the benefits that can be derived from the chemistry. But whether the...
BioCentury | Feb 15, 2016
Product Development

High fidelity

Wave Life Sciences Ltd. has set its sights on rare neuromuscular and CNS diseases where its stereopure nucleic acid technology could confer safety and efficacy advantages over competing programs. Wave was formed in 2012 from...
BioCentury | Aug 10, 2015
Clinical News

Kynamro mipomersen: Phase III data

The double-blind, international Phase III FOCUS FH trial in 310 patients with HeFH showed that once-weekly 200 mg subcutaneous Kynamro met the primary endpoint of reducing LDL-C from baseline to week 61 in cohort 1...
BioCentury | Oct 16, 2014
Distillery Therapeutics

Indication: Endocrine/metabolic disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Endocrine/metabolic disease Mucopolysaccharidosis N-acetylglucosaminidase-a (NAGLU); insulin-like growth factor-2 (IGF-2); mannose-6-phosphate receptor (M6P receptor) Mouse studies suggest a NAGLU-IGF-2 fusion protein could help treat mucopolysaccharidosis IIIB and...
BioCentury | Jul 24, 2014
Cover Story

It is an RNA world

RNA-based molecules have redefined the universe of tractable targets by putting virtually anything that is gene encoded within reach of a disease-modifying agent. This redefinition has launched RNA as the biotech industry's third drug modality....
BioCentury | Apr 14, 2014
Product Development

Family resemblance

The early Phase III data appear similar for competing anti- PCSK9 mAbs from Amgen Inc. on one end and partners Regeneron Pharmaceuticals Inc. and Sanofi on the other. But starting mid-year, additional data in the...
BioCentury | Nov 11, 2013
Company News

Isis Pharmaceuticals, SQI Diagnostics deal

SQI will use its Ig Plex multiplexing immunogenicity technology to develop a multiplex anti-drug antibody assay for Isis. Ig Plex allows for simultaneous quantification of Ig isotype and subclass for multiple therapeutic proteins in the...
BioCentury | Oct 24, 2013
Cover Story

A platform for RNA

An information explosion on the number of functional RNA molecules expressed in cells and the mechanisms by which they control gene expression 1,2 is driving the formation and funding of biotechs developing oligonucleotide-based therapeutics. Pharmas...
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