March 2019 Volume 15, Issue 3

Volume 15, Issue 3 | March 2019
March 2019
In this Issue
Discovery

ALS hope at last?
Small-molecule targets may prevent ALS
Copper conflict
Staphyloccocus genes offer potential pathways for overcoming antibacterial resistance
GWAS and new gene answers
Genomic exploration by Chinese researchers uncovers new information regarding genetic schizophrenia risk
CDD gains grants
CDD awarded two SBIR grants on digital representation of chemical mixtures, deep learning strategy for drug discovery
Stem cells and screening
Ncardia seeks to bring human disease biology earlier into drug discovery and developmentPreclinical

Alternative to animal testing
‘Human-on-a-chip’ models evaluate systemic toxicity
Paving the way
Shingles treatment the first of several herpesvirus drug candidates to enter IND-enabling studies
The data are in on CB 2679d-GT
Catalyst Biosciences announces preclinical data on gene therapy candidate for hemophilia B
Natural killer cells promise life
GT Biopharma encouraged by clinical potential of triple-threat TriKE cells in cancer
Orion reports positive results for OB-002O
Investigational therapeutic for Colorectal Cancer shows promise in preclinical studyClinical Trials

Immunotherapy before surgery?
Trial indicates that the process shows benefit for gliobastoma patients
Mixed results
Companies face both trial roadblocks and progress as they tackle DMD
Nuzyra fights pneumonia
Drug shown safe and effective in adults with pneumonia and skin infections
Combating rejection
Kamada’s Phase 2 proof-of-concept trial shows prevention of lung transplant rejection for one year
Positive data for TRC105
TRACON announces results from ongoing Phase 1b/2 trial in hepatocellular carcinomaSpecial Reports

Special Report on Immunology & Autoimmunity: Turn of the coin
Treading the edge between autoimmunity and oncologyCommentary

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
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 marketFeature

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
Cancer on the mind
Glioblastoma remains a difficult cancer to treat, but the brain malignancy is seeing increasing attention in R&D
Focus Feature: Artificial Intelligence 2.0
Artificial intelligence cannot solve every problem, but it has the potential to break up many bottlenecksDiagnostics

‘Changing the face of genetic diagnostics’
Study shows Bionano’s Saphyr system accurately detects genetic disorder FSHD
Prediction precision
New tool said to offer objective prostate cancer assessment
Taking it to the nose to fight lung cancer
Veracyte announces strategic collaboration with Johnson & Johnson for development of nasal swab test
From mice to man
Elevated hormone flags liver problems related to methylmalonic acidemiaResearch & Development

Dual approach to pancreatic cancer
Roswell Park researchers publish two studies on progress against the disease
New options with nitrogen
Rice University researchers describe a novel chemical synthesis approach
From single-cell to many cells
Researchers use RNA sequencing to investigate spermatogonial stem cells and tackle infertility
Harvard develops kidney organoid
Method for growing kidney organoids under flow enhances their vascularization and maturation
A decline for R&D
Research & development returns fall to lowest level in nine yearsContract Services

UK’s Pathios signs CRO Sygnature
Deal focused on development of therapies in autoimmune disease and immuno-oncology
Under the tongue rather than getting the needle
WuXi STA and BioLingus sign technology and marketing collaboration for sublingual delivery
Six.02 Bioservices launches
Company intends to be a ‘new model’ for providing bioservices in protein-based R&DBusiness & Government Policy

Targets acquired
A look at a few recent merger and acquisition deals in the pharma world
On the cutting edge
A roundup of instrumentation, software and other tools and technology news
Novartis exercises option to license AKCEA-APO(a)-LRx
Phase 3 planning and initiation activities underway; Akcea earns $150-million license fee
Commentary: FDA draft guidance on in-vitro DDI studies
How will it impact your drug discovery program?
Patent Docs: When is an invention “ready for patenting”?
Federal Circuit panel disputes question in Barry v. MedtronicEditor's Focus

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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