Tools & Techniques

New hepatic stellate cell subset drives liver fibrosis

A team including University of Edinburgh and Gilead researchers has used single-cell sequencing to zero in on a pathogenic subpopulation of hepatic stellate cells that could be targeted to treat liver fibrosis. Reported in a...

Editas shows better gene editing using Cas9 alternative for sickle cell, thalassemia

Editas is using an alternative to Cas9 to develop a differentiated sickle cell and β thalassemia CRISPR gene therapy. Preclinical data presented Monday at ASH suggest that ex vivo gene editing could be more effective...

Using exosomes to switch myeloid cells

Driven by the natural biology of exosomes, Codiak has moved beyond diagnostics and targeted cancer therapies to embrace myeloid switching as a strategy to treat cancer. The biotech presented preclinical data Nov. 18 at the...

Measuring real-time PK/PD by monitoring aberrant chaperone activity

A Memorial Sloan Kettering Cancer Center team developed an imaging probe for aberrant protein networks that could guide more optimal drug dosing and scheduling earlier in development. In cancer, tumor biopsy collection as well as...

Weizmann 10K Project aims for -omics sweet spot

Using structured and unstructured learning, the Weizmann Institute’s Eran Segal wants to mine reams of data over 20 years from 10,000 people who start out healthy, to find the markers that signal disease. His 10K...

Draper’s devices take on cell therapy’s pain points

Having surveyed more than 60 cell therapy developers on what keeps them up at night, The Charles Stark Draper Laboratory Inc. is building a suite of microfluidic and acoustic tools to overcome the inefficiencies that...

Gladstone, Xyphos target universal CARs to latent HIV

Xyphos teamed up with the Gladstone Institutes to direct the company’s universal CAR Ts to cells latently infected with HIV, addressing several of the challenges CAR Ts have faced in the indication. Major barriers to...

NASH: how non-invasive biomarkers promise to displace biopsies

Non-invasive biomarkers in NASH are beginning to show where and how they can upstage liver biopsies, with the first impact appearing in patient recruitment. The biggest value will come when they can serve as regulatory...

Driving safely into solid tumors with CAR exosomes

Therapeutic exosomes derived from CAR T cells could avoid the cytotoxic side effects of the engineered cells and overcome barriers in the tumor microenvironment that limit the cells’ efficacy. The obstacles to deploying CAR T...

Yale takes CRISPR beyond editing, turning cold tumors hot

A Yale team has developed a new use for CRISPR as a cancer vaccine that turns cold tumors hot by boosting neoantigen expression, rather than editing genes. In the realm of cancer, CRISPR’s application to...

Vector rearrangement boosts globin-delivering gene therapies

Through vector genome reorganization, NIH researchers have amplified the manufacturing output and β globin delivery for a lentiviral gene therapy for sickle cell disease and β thalassemia. According to Naoya Uchida, gene therapies delivering β...

Broad takes RNA CRISPR into viral infections

The latest CRISPR study from the Broad Institute takes gene editing into infections caused by RNA viruses, using Cas13 -- which is proving to be one of the most versatile of the CRISPR-Cas enzyme family....

Nobel Prize for cell response to oxygen, a two-sided therapeutic target

Cellular responses to oxygen levels, the focus of this year's Nobel Prize in Physiology or Medicine, are the targets of both inhibiting and activating therapies in clinical development for multiple diseases. William Kaelin, Peter Ratcliffe...

Biotechs propose solutions to impending shortage of AI talent

Competition for AI talent is about to get fierce as companies across biopharma, and every other sector, begin to deploy machine learning in house. Right now, biotechs are aiming to fill their talent needs through...

Locus co-founders open door to new CRISPR systems

As most gene editing companies remain laser-focused on the well-studied CRISPR-Cas9 system marred in IP disputes, a new study from Locus co-founders shows that an entirely different type of CRISPR system can tune gene expression...