September 2014- Volume 10, Issue 9
September 2014
In this Issue
Research & Development
A toolkit for the genome
A toolkit for the genome
Horizon Discovery Group licenses Stanford University technology to enhance gene editing
A deal on peptides
A deal on peptides
Zealand Pharma and Boehringer Ingelheim sign second collaboration agreement to advance novel peptide medicines
Understanding the ways of pathogenic harm
Understanding the ways of pathogenic harm
NIAID grant supports infectious disease research at the Institute for Genome Sciences to the tune of $15.2 million
Interferon may be key to fighting Ebola
Interferon may be key to fighting Ebola
Finding provides framework for new drug development efforts
Delivering the genes
Delivering the genes
Scripps Research Institute scientists find that cancer drug rapamycin may help bypass blood stem cell defenses
Discovery
Sanford- Burnham sets sights on MEF2
Sanford- Burnham sets sights on MEF2
Transcription factor thought to be a therapeutic target for Alzheimer’s, Parkinson’s disease
Less pain, more gain
Less pain, more gain
Scripps scientists in Florida discover new targets to reduce side effects in treating breast cancer
Library renewal
Library renewal
Collaboration renewal maintains collection of more than 200,000 compounds for investigation
Clinical Trials
Easy to take
Easy to take
Weekly, ready-to-use drug could be good news for type 2 diabetics
Xention commences first Phase 2 study of XEN-D0103 in atrial fibrillation
Xention commences first Phase 2 study of XEN-D0103 in atrial fibrillation
Xention Ltd. recently announced that the Phase 2 development of its lead atrial fibrillation (AF) program is underway
Exciting results from exon-skipping
Exciting results from exon-skipping
Sarepta’s eteplirsen stabilizes lung function, slows mobility loss in Duchenne patients
Dupilumab’s skin story
Dupilumab’s skin story
Investigative monoclonal antibody shows potential for eczema relief
Ohr presents two Squalamine Phase 2 data sets
Ohr presents two Squalamine Phase 2 data sets
Trial data for retinal vein occlusion and wet age-related macular degeneration detailed at 2014 ASRS meeting
RedHill options Phase 2 pancreatic cancer drug
RedHill options Phase 2 pancreatic cancer drug
RP101 has been granted Orphan Drug designation for the adjunct treatment of pancreatic cancer by FDA and EMA
Business & Government Policy
FDA approves Ryanodex for treatment of malignant hyperthermia
FDA approves Ryanodex for treatment of malignant hyperthermia
Eagle Pharmaceuticals Inc. announced this summer that the U.S. Food and Drug Administration had approved Ryanodex
A deep breath for an $8.3-billion plunge
A deep breath for an $8.3-billion plunge
Roche and InterMune enter merger agreement aimed at expanding Roche’s respiratory portfolio
Roche snaps up Santaris
Roche snaps up Santaris
Deal gains Roche access to Santaris’ platform for RNA-targeted therapeutics
Venter gains a key human asset
Venter gains a key human asset
Google’s Franz Och joins Human Longevity as chief data scientist
Three-in-one combo gets two green lights
Three-in-one combo gets two green lights
ViiV Healthcare receives FDA approval and EU marketing authorization for Triumeq in treatment of HIV-1 infection
Diagnostics
Hope springs eternal
Hope springs eternal
NIH names new clinical sites for Undiagnosed Diseases Network and provides $43 million
Tapping CDx expertise
Tapping CDx expertise
Ventana signs companion diagnostic test agreements with Merck KGaA and Quintiles
Flushing out the fatty fog
Flushing out the fatty fog
Researchers build off CLARITY technique to make bodies ‘transparent’ for 3D imaging
FDA green-lights DoD Ebola diagnostic
FDA green-lights DoD Ebola diagnostic
Assay will aid in detecting infection with Ebola Zaire virus in individuals at risk
Feature
ASHG 2014 Preview: Shining a light on human genetics in sunny San Diego
ASHG 2014 Preview: Shining a light on human genetics in sunny San Diego
Reportedly the largest human genetics meeting and exposition in the world, the ASHG gathering from Oct. 18 to 22 will cover ethics in genomics, advances in sequencing and much else, attracting more than 6,500 scientific attendees and featuring well over 200 exhibiting companies
Editor's Focus
Much ado about Ebola
Much ado about Ebola
There is no doubt that Ebola is a monumental problem, and it's a notable part of our September issue coverage. But there's also been a lot of excessive fear and hype as well, which has perhaps made the disease look like something out of an apocalyptic novel or film. On the bright side, the increased attention seems to have mobilized many pharmas, biotechs, government agencies and others to increase the pace of Ebola diagnostics and therapies, which could be welcome relief to the people most at risk of the disease.
Commentary
Out of Order: The undiscovered country
Out of Order: The undiscovered country
Off- label prescribing of pharmaceutical products by physicians has a long history and even some notable successes, but the practice also poses many challenges and risks.
Patent Docs: FDA releases draft guidances on biosimilars
Patent Docs: FDA releases draft guidances on biosimilars
The U.S. Food and Drug Administration was been busy in spring 2014, releasing two new guidances (in May and August) relating to its evolving standards for satisfying the biosimilarity requirements of the Biologics Price Competition and Innovation Act.
Q&A
Q&A: Taking the measure of ‘omics results in health science and personalized medicine
Q&A: Taking the measure of ‘omics results in health science and personalized medicine
DDNews asks Dr. Rohit Khanna, vice president of worldwide marketing for the Waters Division of Waters Corp., to share his views on the advances in mass spectrometry and how they are impacting healthcare delivery
Preclinical
Roche puts its back into fighting SMA
Roche puts its back into fighting SMA
Oral treatment for spinal muscular atrophy appears effective in mice
Finding the right channel
Finding the right channel
Xention’s atrial fibrillation research receives funding from British government
Smoke stopper
Smoke stopper
Addex drug achieves positive results in preclinical model of nicotine addiction
Epizyme debuts preclinical EZH2 inhibitor data at ASH meeting
Epizyme debuts preclinical EZH2 inhibitor data at ASH meeting
EPZ-6438 has engendered partial response in two non- Hodgkin lymphoma patients; maximum tolerated dose not yet reached
GSOs and TLRs make antisense work
GSOs and TLRs make antisense work
Idera Pharmaceuticals and collaborators publish promising preclinical data targeting microRNA with gene-silencing oligonucleotide technology
