In the ascendant world of mRNA medicines, circular RNA shows promise as an alternative way for making proteins inside human cells.

The biotech sector must devote more resources to cybersecurity — especially those companies that are manufacturers of essential medical products.

Recent moves of note in and around the biotech and pharma industries.

Companies are deploying siRNA and antisense oligonucleotides to tackle dangerously high cholesterol driven by genetics, betting that a wider population will benefit.

Recent patents relating to checkpoint inhibitors and their use in treating cancers.

Companies pursue souped-up IL-2 candidates, undeterred by Nektar’s setback.

The digitalization of R&D can potentially transform the life sciences, but it cannot succeed without experts collaborating to develop a vision of what a better working environment can look like.

After over a decade of experience with immune checkpoint inhibitors in oncology, more effort needs to be spent unraveling why some patients respond — and why the majority do not — and integrating knowledge about biomarkers into patient selection in trials.

We developed two quantitative PCR-based assays to detect SARS-CoV-2-specific T cell immunity: qTACT and dqTACT. The assays quantify CXCL10 mRNA, after incubation of whole blood with viral peptides, as a proxy of an antigen-specific T cell response, and will allow population-level monitoring of cellular immunity to SARS-CoV-2.

Recent patents relating to CRISPR methods and compositions for gene editing and therapeutic use.

The first herpesvirus replacement gene therapy for epidermolysis bullosa, a blistering skin condition, approaches the clinic.

Recent moves of note in and around the biotech and pharma industries.

A new era of cancer immunotherapy beckons as γδ T cell trials enter final stage.

Even if Amarin prevails in its patent infringement lawsuit, its key patents on Vascepa are likely to be ruled invalid in ongoing litigation, opening the way for generic formulations.

An intensive program supplements student education and collaboration while narrowing the disconnect between rising healthcare needs and trainees with the technical expertise to tackle pandemic-related challenges.

A new study describes a microscope combining three-photon excitation and adaptive optics capable of high-resolution in vivo imaging of fine neuronal structures in the mouse cortex through the intact skull. The authors demonstrate the use of this platform to guide precise laser-mediated microsurgery and for accurate and sensitive functional calcium imaging.

Red light penetrates deep into mammalian tissues and has low phototoxicity, but few optogenetic tools that use red light have been developed. Here we present MagRed, a red light–activatable photoswitch that consists of a red light–absorbing bacterial phytochrome incorporating a mammalian endogenous chromophore, biliverdin and a photo-state-specific binder that we developed using Affibody library selection

Single-cell RNA sequencing studies have suggested that total mRNA content correlates with tumor phenotypes. Technical and analytical challenges, however, have so far impeded at-scale pan-cancer examination of total mRNA content. Here we present a method to quantify tumor-specific total mRNA expression (TmS) from bulk sequencing data, taking into account tumor transcript proportion, purity and ploidy

Fast, high-throughput methods for measuring the level and duration of protective immune responses to SARS-CoV-2 are needed to anticipate the risk of breakthrough infections. Here we report the development of two quantitative PCR assays for SARS-CoV-2-specific T cell activation. The assays are rapid, internally normalized and probe-based: qTACT requires RNA extraction and dqTACT avoids sample preparation

High-resolution optical imaging deep in tissues is challenging because of optical aberrations and scattering of light caused by the complex structure of living matter. Here we present an adaptive optics three-photon microscope based on analog lock-in phase detection for focus sensing and shaping (ALPHA-FSS). ALPHA-FSS accurately measures and effectively compensates for both aberrations and scattering

Reprogramming of antibody responses in mice is achieved via adeno-associated virus delivery of SaCas9, single guide RNA (sgRNA) and a repair template targeting immunoglobulin genes.

Transplantation of B cells engineered ex vivo to secrete broadly neutralizing antibodies (bNAbs) has shown efficacy in disease models. However, clinical translation of this approach would require specialized medical centers, technically demanding protocols and major histocompatibility complex compatibility of donor cells and recipients. Here we report in vivo B cell engineering using two adeno-associated

RNA interference (RNAi) therapeutics can silence disease-causing gene transcripts, but extrahepatic delivery has been challenging. Conjugating short interfering RNAs (siRNAs) to a lipophilic alkyl chain enabled safe delivery and long-term mRNA silencing in the brain, eye and lung in animal models, thereby opening new applications for RNAi therapeutics.

Interest is growing in genome-editing tools that can insert large chunks of DNA into the genome — and avoid the double-strand breaks associated with CRISPR–Cas9 genotoxicity.

Existing technologies can map the intricate spatial distribution of biomolecules and cell types within tissues, but not in specimens that remain alive and intact. This study introduces scission-accelerated fluorophore exchange (SAFE) bioorthogonal imaging tools that enable living cells and tissues to be deeply and serially profiled, revealing their biological dynamics across both space and time.

Our multidisciplinary team used novel perfusion technology to successfully keep an injured liver graft discarded by all other centers alive for several days, allowing proper graft evaluation and repair. The graft was successfully transplanted into a sick patient, who recovered quickly and enjoyed a normal life at one-year follow up.