March 2019 Volume 15, Issue 3

Volume 15, Issue 3 | March 2019

March 2019

In this Issue

Discovery

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ALS hope at last?

Small-molecule targets may prevent ALS
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Copper conflict

Staphyloccocus genes offer potential pathways for overcoming antibacterial resistance
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GWAS and new gene answers

Genomic exploration by Chinese researchers uncovers new information regarding genetic schizophrenia risk
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CDD gains grants

CDD awarded two SBIR grants on digital representation of chemical mixtures, deep learning strategy for drug discovery
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Stem cells and screening

Ncardia seeks to bring human disease biology earlier into drug discovery and development

Preclinical

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Alternative to animal testing

‘Human-on-a-chip’ models evaluate systemic toxicity
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Paving the way

Shingles treatment the first of several herpesvirus drug candidates to enter IND-enabling studies
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The data are in on CB 2679d-GT

Catalyst Biosciences announces preclinical data on gene therapy candidate for hemophilia B
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Natural killer cells promise life

GT Biopharma encouraged by clinical potential of triple-threat TriKE cells in cancer
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Orion reports positive results for OB-002O

Investigational therapeutic for Colorectal Cancer shows promise in preclinical study

Clinical Trials

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Immunotherapy before surgery?

Trial indicates that the process shows benefit for gliobastoma patients
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Mixed results

Companies face both trial roadblocks and progress as they tackle DMD
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Nuzyra fights pneumonia

Drug shown safe and effective in adults with pneumonia and skin infections
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Combating rejection

Kamada’s Phase 2 proof-of-concept trial shows prevention of lung transplant rejection for one year
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Positive data for TRC105

TRACON announces results from ongoing Phase 1b/2 trial in hepatocellular carcinoma

Special Reports

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Special Report on Immunology & Autoimmunity: Turn of the coin

Treading the edge between autoimmunity and oncology

Commentary

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Out of order: Keep talking

Active discussion regarding diseases and clinical trial progress is important not just for keeping hope alive and boosting awareness, it's also essential in reminding patients and their families that there are others out there who share their struggles
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Guest Commentary: A tale of safety and consistency

Drs. Ulrike Herbrand, Christopher Sucato and Alvaro Jorge Amor of Charles River Laboratories discuss the challenges of bringing antibody-drug conjugates to market

Feature

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AACR 2019 Show Preview: Integrative cancer science meets individualized care

Over 20,000 attendees will converge on Atlanta this year for the 110th AACR Annual Meeting
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Cancer on the mind

Glioblastoma remains a difficult cancer to treat, but the brain malignancy is seeing increasing attention in R&D
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Focus Feature: Artificial Intelligence 2.0

Artificial intelligence cannot solve every problem, but it has the potential to break up many bottlenecks

Diagnostics

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‘Changing the face of genetic diagnostics’

Study shows Bionano’s Saphyr system accurately detects genetic disorder FSHD
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Prediction precision

New tool said to offer objective prostate cancer assessment
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Taking it to the nose to fight lung cancer

Veracyte announces strategic collaboration with Johnson & Johnson for development of nasal swab test
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From mice to man

Elevated hormone flags liver problems related to methylmalonic acidemia

Research & Development

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Dual approach to pancreatic cancer

Roswell Park researchers publish two studies on progress against the disease
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New options with nitrogen

Rice University researchers describe a novel chemical synthesis approach
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From single-cell to many cells

Researchers use RNA sequencing to investigate spermatogonial stem cells and tackle infertility
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Harvard develops kidney organoid

Method for growing kidney organoids under flow enhances their vascularization and maturation
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A decline for R&D

Research & development returns fall to lowest level in nine years

Contract Services

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UK’s Pathios signs CRO Sygnature

Deal focused on development of therapies in autoimmune disease and immuno-oncology
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Under the tongue rather than getting the needle

WuXi STA and BioLingus sign technology and marketing collaboration for sublingual delivery
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Six.02 Bioservices launches

Company intends to be a ‘new model’ for providing bioservices in protein-based R&D

Business & Government Policy

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Targets acquired

A look at a few recent merger and acquisition deals in the pharma world
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On the cutting edge

A roundup of instrumentation, software and other tools and technology news
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Novartis exercises option to license AKCEA-APO(a)-LRx

Phase 3 planning and initiation activities underway; Akcea earns $150-million license fee
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Commentary: FDA draft guidance on in-vitro DDI studies

How will it impact your drug discovery program?
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Patent Docs: When is an invention “ready for patenting”?

Federal Circuit panel disputes question in Barry v. Medtronic

Editor's Focus

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Editor’s focus: Filling the voids

Looking at some of the progress in areas we had been hoping to see more from in the past, including schizophrenia, antibiotics and artificial intelligence
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