Alnylam launches era of RNAi drugs

Alnylam launches era of RNAi drugs

On August 10, the US Food and Drug Administration approved the first RNA interference (RNAi) therapeutic, a treatment for polyneuropathy caused by transthyretin (TTR) amyloidosis from Alnylam Therapeutics. The go-ahead for Onpattro (patisiran) sees the RNAi field clear an approval hurdle considered unlikely as recently as six years ago, when pharma exited the RNAi field en masse. The US approval, with Europe expected to follow by early September, is “a major milestone,” says Anastasia Khvorova, an RNAi researcher at the University of Massachusetts in Worcester. Onpattro has an excellent safety record, but there are lingering concerns about potential long-term toxicity from newer, more potent RNAi therapeutics. And the field as a whole still faces investor skepticism in the wake of a decade of clinical trial failures.  Read more

A new approach for DNA synthesis

A new approach for DNA synthesis

Ordering synthetic oligos or genes online is now commonplace and an essential resource to scientists across disciplines. But the phosphoramidite chemistry currently used to synthesize DNA is limited to direct synthesis of about 200 nucleotides, with longer stretches requiring assembly. The capacity to synthesize long stretches of DNA is important for a variety of applications, including DNA storage, DNA origami, and to synthesize DNA containing regions with repeats, which are difficult to put together. In a paper published recently in Nature Biotechnology, Jay Keasling and colleagues report a promising new approach to DNA synthesis. Using a terminal deoxynucleotidyl transferase (TdT) conjugated to a single deoxyribonucleoside triphosphate (dNTP), they tether the primer to TdT after extending it by one nucleotide.  Read more

Hunting connections between cell types and cytokines

Hunting connections between cell types and cytokines

Cytokines are small proteins that mediate signalling among immune and non-immune cells, and they trigger a range of cellular activity, such as proliferation, activation and killing. Over many decades, immunologists have described countless associations between cell types and the cytokines they produce or sense, but many of these findings, although published, are difficult to access. Associations may have been discovered in a particular disease context or cell type, or uncovered as part of a larger study and thus not corroborated or expanded. Work from Shai Shen-Orr and colleagues, published in Nature Biotechnology, aims to unearth these connections and provide a useful resource for enabling new discoveries.  Read more

Will the EU deregulate gene-edited plants?

Will the EU deregulate gene-edited plants?

At the beginning of the year, the advocate general of the Court of Justice of the European Union (CJEU) issued an opinion that plants created using new plant breeding techniques, including gene-editing platforms like CRISPR, TALENs and the like, are eligible for the so-called mutagenesis exemption. This exemption relates to rules the European Union uses to regulate the release and marketing of genetically modified organisms (GMOs), which are outlined in Directive (2001/18/EC), originally drafted in 2001. The exemption covers any plants considered ‘safe’ or produced using techniques that have a history of safety, including plants derived from traditional mutagenesis (hence the mutagenesis exemption).  Read more

Rumen microbial genomics resource

Rumen microbial genomics resource

The Hungate1000 project, named after one of the great microbiologists, Robert E. Hungate (pictured), was launched with the aim of producing a reference set of rumen microbial genome sequences. When this project began there was only a handful of rumen reference microbial genomes available. The first output of the Hungate1000 project, comprising 410 high-quality genome sequences, is reported online today in Nature Biotechnology. Seshadri et al. highlight discovery of degradative enzymes, biosynthetic gene clusters and Crispr sequences. These reference genomes will enable robust interpretation of rumen metagenomes, which should result in a better understanding of rumen functions. Genome-enabled research into feed conversion efficiency, methanogenesis and cellulose degradation will, in turn, assist development of strategies to balance food production with efforts to minimize greenhouse gas emissions. Finally, access to cultivated Hungate Collection strains will provide vital tools for studying carbon flow in the rumen, breakdown of lignocelluloses and methane formation.  Read more

The Developing World Needs GMOs

The Developing World Needs GMOs

The need to feed growing populations in developing countries, especially countries in Africa, must be met by increasing the yields of crops. Also, climate-change related problem such as drought continue to worsen hunger problem and humanitarian crisis in the continent. Genetically modified organisms (GMOs) could greatly help with these issues, yet resistance persists in Europe and Africa both.  Read more

Linking the scientific and patent literatures

Linking the scientific and patent literatures

The scientific literature and patent literature have for a long time been viewed as two different worlds, with publications in the latter one measure of a researcher’s translational activity. But a much larger cadre of researchers influence inventions beyond those who are named as inventors on patents. Within patent filings there is often an extensive list of citations to the non-patent literature, including peer-reviewed papers, monographs, meetings and more. In a Patent article, Jefferson Osmat and her colleagues have created a tool to mine an open database termed the Lens containing filings from the US Patent and Trademark Office, The European Patent Office, the World Intellectual Property Organization Patent Cooperation Treaty applications and IP Australia for the non-patent literature. This enables an assessment of individual and institutional contributions to the global patent literature.  Read more

Gottlieb on pricing, competition and new therapeutic modalities

While freelancing for Nature Biotechnology, I recently talked to US Food and Drug Administration (Rockville, MD) Commissioner Scott Gottlieb. The conversation ranged from pricing, to market competition to new therapeutic modalities like gene therapy coming down the pipeline. A more extended extract of our discussion is also available in the News Feature.  Read more

Why Every Life Science CEO Needs a Leadership Coach

Why Every Life Science CEO Needs a Leadership Coach

The biggest graduation for an academic researcher comes without diplomas or Latin superlatives, caps or gowns. It’s the leap from academia to business, the shepherding of our beloved ideas and inventions out of the known world of the lab and into the strange land of the marketplace.  Read more

A new tool to study the immune system

A new tool to study the immune system

Beyond fighting infection, the immune system has important roles in many systems in the body. To study the involvement of T cells and B cells, researchers often sequence their T cell and B cell receptors (TCRs and BCRs), which provide insights into their clonal diversity. However, even more useful would be to gather T cell and B cell receptor information together with the transcriptomic profile of their tissue sample of origin. Now, in a paper published in Nature Biotechnology, Dmitriy Chudakov and colleagues report a software tool that enables extracting TCR and BCR sequences from bulk RNA-seq data sets. Because RNA-seq data is already available for thousands of tumor samples, this method will allow revisiting those data sets to extract important information.  Read more