May 2018 Volume 14, Issue 5

Volume 14, Issue 5 | May 2018

May 2018

In this Issue

Feature

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ISSCR 2018 show preview: Heading Down Under to bring up matters about stem cells

International Society for Stem Cell Research’s 2018 annual meeting to be hosted in Australia
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ASCO 2018 show preview: Deep dish--ASCO brings cancer researchers to Chicago

Annual clinical cancer research meeting dives deep into discovery
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Special Focus on CRISPR: Regarding lipids and CRISPR

On one side, the gene-editing technology tackles lipids; on the other, it uses them for transportation; plus additional news of CRISPR research and technology and a bonus guest commentary

Special Reports

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Special Report on Epigenetics: More than a number

Epigenetics shows that not all aging is created equal

Discovery

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Gems in the ‘junk’

Noncoding mutations found to be a major mechanism in cancer
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TSRI makes headway in neurology

Two new discoveries have been made regarding Parkinson’s disease and depression
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A new arrow in the treatment quiver

Boosting adenosine levels with a new stem cell population could help to treat epilepsy
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C4XD signs license deal with Indivior for addiction program

Company receives $10 million up front, with potential milestones totaling $284 million plus royalties
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BioAscent establishes integrated drug discovery services offering

New CSO hired to lead company’s extended capabilities in medicinal chemistry and in-vitro biosciences

Clinical Trials

Berubicin blasts glioblastoma

The anthracycline drug is the only one of its class engineered to attack tumor cells at the site
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A setback, but not for long

Serlopitant misses primary and secondary efficacy endpoints, but Menlo continues with development program
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Positive results for novel antiviral in HBV

Assembly Biosciences to launch Phase 2a studies in summer 2018
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Seladelpar impresses at International Liver Congress

CymaBay’s PPARδ agonist reduces alkaline phosphatase levels in PBC patients
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Getting you where you need to go

ClinOne announces integration with Lyft and Uber for free patient transportation to study sites

Research & Development

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The chip craze continues

Research teams worldwide are working to harness the potential of organ-on-a-chip technology
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Clathrin collaboration

Microscopy shows how cells engulf nutrients and viruses
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Roswell Park data may link ovarian and testicular cancer

Men with testicular cancer more likely to have female relatives with ovarian cancer
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TSRI arm gets $34M from NIH

Scripps Translational Science Institute receives NIH Clinical and Translational Science Award
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Agilent and BioTek join forces

Companies create an integrated metabolic analysis and imaging platform

Editor's Focus

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Editor’s focus: Navigating the gray areas

Gray literature isn't the same as peer-reviewed literature, but it doesn't need to be; however, we need to keep use of it in perspective and in balance

Commentary

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Out of order: What lies beneath

Randomized, double-blinded, placebo-controlled clinical trials are the "gold standard" but let's remember that even gold can be tarnished and not all that glitters is truly gold
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Guest commentary: Implications of cGMP for CRISPR/Cas9 cellular therapy

CRISPR/Cas9 offers the hope of a cure for various maladies, including genetic diseases and cancers, but current Good Manufacturing Practices still matter in handling this therapeutic avenue

Contract Services

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Frontage acquires Concord Biosciences

Deal will add preclinical, toxicology and agrochemical services to Frontage’s existing product development portfolio
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Boosting and complementing its DMPK

International CRO gobbles up drug transporter and drug-drug interaction specialist company
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Helsinn wins across the board

Company takes first in all core categories at 2018 CMO Leadership Awards

Business & Government Policy

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Predictive genomic medicine

NIH project aims to determine phenotype from genotype, match researchers to genes and variants of interest
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Novartis takes aim at SMA market with AveXis deal

Company is acquiring AveXis and its spinal muscular atrophy drug candidate for $8.7 billion
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NDA Group releases data comparing FDA and EMA

Information released ahead of annual DIA Europe meeting in Basel
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On the cutting edge

A roundup of instrumentation, software and other tools and technology news
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Making progress: A roundup of recent pharma/biotech milestones

News from AiCuris, Organovo, Catalyst Biosciences and Zosano Pharma
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Patent Docs: Federal Circuit suggests way out of subject matter eligibility morass

A tortured path on applying patent eligibility decisions finally offers some scant hope for better interpretation and understanding

Preclinical

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Repair and regeneration

Cell therapy study offers hope for Duchenne muscular dystrophy
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Rhythm licenses RM-853 from Takeda

Oral GOAT inhibitor complements and expands efforts in Prader-Willi syndrome
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‘Tremendous potential’ against pancreatic cancer

Combination of immune agonists may fight one of the fastest-moving and deadliest of cancers
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THR-149 shows efficacy vs. DME

ThromboGenics announces publication of preclinical data in The Journal of Medicinal Chemistry

Diagnostics

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Sample-to-insight pathogen testing improves

QIAGEN acquires molecular diagnostics company STAT-Dx and its multiplex technologies
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A 'FIGHT' for life in gastric cancer

PGDx to develop companion diagnostic for Five Prime’s bemarituzumab
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Illumina, Loxo and companions

Deal expands Illumina’s oncology offerings for with a CDx version of TruSight Tumor 170 for solid tumors
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Multiple studies show potential of T2’s diagnostics

Data indicate improved detection of sepsis-causing pathogens over blood culture-based diagnostics

Q&A

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Q&A: A new option for oral anticoagulants

A chat with Espero BioPharma’s CEO reveals shortcomings of current standard of care and efforts to advance a better candidate
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Cell therapy's next frontiers

As cell therapy evolves rapidly, what advancements are on the horizon? 
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Reversing rejection: gene therapy in modern transplantation

Emerging gene editing approaches pave the way for safer, more successful transplants.
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Identifying druggable therapeutic targets

Effective target identification is central to drug discovery, but finding the right drug target is not as simple as it may seem in theory. 
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