June 2015- Volume 11, Issue 6

June 2015

In this Issue

Clinical Trials

Merger boosts development of Sanfilippo treatment

Merger boosts development of Sanfilippo treatment

Following acquisition of Abeona, PlasmaTech plans to push company’s pipeline forward with two Phase 1/2 clinical trials in 2015

Envisioning a better future for wet AMD patients

Envisioning a better future for wet AMD patients

Ohr Pharmaceutical to launch Phase 3 study of OHR-102 eye drop therapy to treat wet AMD

In pursuit of predictive biomarkers

In pursuit of predictive biomarkers

NanoString, CITN to seek out biomarkers to predict outcomes for cancer immunotherapies

AbbVie shares data on Humira in non-infectious uveitis

AbbVie shares data on Humira in non-infectious uveitis

Results of VISUAL-I show Humira meets primary endpoint of prolonging time to treatment failure

Preclinical

Eyes on the (glaucoma) prize

Eyes on the (glaucoma) prize

Isarna Therapeutics demonstrates capability of antisense oligonucleotide to treat glaucoma

Novel pancreatic cancer drug may also combat malignant ascites

Novel pancreatic cancer drug may also combat malignant ascites

Treatment being studied involves a combo of low-dose cancer prodrug ifosfamide and Cell-in-a-Box capsules

Bald but beautiful

Bald but beautiful

Huntingdon Life Sciences and Harlan Laboratories announce the launch of the SHrN novel hairless NOD.SCID model at AACR 2015

Peering into the preclinical realm

Peering into the preclinical realm

Here’s a brief roundup of some additional news items related to preclinical research that you might find of interest.

Some ocular help from man’s best friend

Some ocular help from man’s best friend

Novel gene-based therapy for achromatopsia demonstrates functional rescue of cone cells

Research & Development

Skin as good as the real thing?

Skin as good as the real thing?

Synthetic membrane from Millipore reportedly works as well as real skin in transdermal diffusion studies

Popping the Cork on a microbiome alliance

Popping the Cork on a microbiome alliance

Janssen Biotech, APC Microbiome Institute unite to investigate microbiome’s potential in IBD

Speeding single-cell genomics

Speeding single-cell genomics

WaferGen and Genentech to explore SmartChip platform in sequencing applications

Pulling the genomic puppetmaster’s strings

Pulling the genomic puppetmaster’s strings

Ability to modify epigenome very specifically prevents ‘scorched earth’ approach

Labguru embraces ChemAxon’s Marvin JS

Labguru embraces ChemAxon’s Marvin JS

Integration of web-based electronic lab notebook with chemistry software tool seeks to enhance functionality for chemists

Discovery

TSRI scientists link GIRK3 to binge drinking

TSRI scientists link GIRK3 to binge drinking

Study reveals molecular mechanisms of binge drinking and may reveal avenues for treating substance abuse

Cystic fibrosis deal focused on ‘adding tomorrows’

Cystic fibrosis deal focused on ‘adding tomorrows’

Cystic Fibrosis Foundation Therapeutics announces $14-million research expansion agreement with Genzyme

First and PhoreMost

First and PhoreMost

Newly founded company will apply Site-Seeker platform to identify drug targets considered ‘undruggable’

Two for FSHD

Two for FSHD

Facio partners with Evotec to initiate a FSHD drug discovery program

Diagnostics

IBM’s Watson takes on cancer

IBM’s Watson takes on cancer

More than a dozen cancer institutes set to join computing giant to accelerate personalized medicine

Best in blood

Best in blood

Amarantus’ blood-based version of MSPrecise could become a gold standard

Cruising along with companion diagnostics

Cruising along with companion diagnostics

Myriad expands two CDx partnerships and presents Prolaris data

Business & Government Policy

Kidney capabilities

Kidney capabilities

Pfizer gets minority interest with option to acquire AM-Pharma

A $250-million foundation

A $250-million foundation

FMI and Roche complete strategic transaction to advance molecular information and precision medicine in oncology

On the cutting edge

On the cutting edge

A roundup of instrumentation, software and other tools and technology news

Sweet on Sobi?

Sweet on Sobi?

Not-so-mystery bidder Pfizer expresses interest in acquiring Swedish rare-disease company

Rallying around rare diseases

Rallying around rare diseases

Alexion bets billions on Synageva and its drug candidate Kanuma

Nabbing a leukemia portfolio for $900M

Nabbing a leukemia portfolio for $900M

Baxter BioScience seeks to expand global oncology footprint with acquisition of Oncaspar

Q&A

Q&A: A ‘Trojan Horse’ to fight cancer

Q&A: A ‘Trojan Horse’ to fight cancer

Smuggling in an army to help the immune system unwittingly destroy tumors

Special Reports

Cancer Special Report: Body, heal thyself

Cancer Special Report: Body, heal thyself

To attack cancer, researchers are tapping evolution’s weapon of choice: the immune system

Editor's Focus

We need to ‘C’ the big picture

We need to ‘C’ the big picture

What drives this editor nuts faster than almost anything else? Conspiracy theories about pharma and biotech, particularly the ones that insist that companies are sitting on the cure for cancer so that they can get ever richer on non-curative therapies for the disease.

Commentary

The Internet of medical things

The Internet of medical things

It isn't that mobile health devices and other, similar technologies aren't a potential boon for things like clinical trial data handling. It's just that we aren't quite at the point where we can realize their true worth or even use them properly in most cases.

Commentary: Encouraging advances now underway in cancer research

Commentary: Encouraging advances now underway in cancer research

The field of cancer genomics is rapidly advancing both in knowledge and in relevance to the treatment of cancer, as detailed genetic profiling of human cancers has now become possible. Mass spec imaging is helping to take that farther.
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