September 2014- Volume 10, Issue 9

September 2014

In this Issue

Research & Development

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

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

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

Library renewal

Library renewal

Collaboration renewal maintains collection of more than 200,000 compounds for investigation

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

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

Preclinical

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

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

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
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