A new study has presented an innovative electromagnetic field (EMF)-responsive gene switch that enables fully reversible, safe, and precise control over gene expression. “Gene switches” hold significant potential for understanding gene expression and for therapeutic applications, particularly for the non-invasive treatment or management of genetic disorders. … Read More
University of Queensland (UQ) scientists will accelerate research into cures for life-threatening genetic diseases from a Brisbane-based node of the new Medical Research Council Centre of Research Excellence for Mitochondrial Genome Therapeutics. Associate Professor Seth Cheetham from UQ’s Australian Institute for Bioengineering and Nanotechnology and Chief Investigator of… Read More
Broken String Biosciences, a biotechnology company enabling gene editing teams to accelerate development of safer, more effective genetic medicines, has launched BaseMap ABE. Built on the company’s INDUCE-seq technology, BaseMap ABE extends the platform’s capabilities to support adenine base editing (ABE) applications. Base editing is rapidly gaining momentum as a… Read More
The US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for Beam Therapeutics’ BEAM-304 for the treatment of phenylketonuria (PKU). PKU is a rare, inherited metabolic disorder that results in toxic accumulation of phenylalanine (Phe), leading to serious neurologic and neurocognitive impairments… Read More
Broken String Biosciences has appointed Bruce Eaton as Chairman of the Board. The appointment from the biotechnology company allows it to drive the global commercialisation of its INDUCE-seq platform, which it says can provide rapid, unbiased, genome-wide measurement of on- and off-target DNA breaks directly in customers’ own… Read More
An engineered CRISPR-Cas12l nuclease could spark renewed interest in the CRISPR approach, Caszyme says. The gene editing company published research in The CRISPR Journal detailing the performance characteristics of Asp2Cas12l M82, denoted as M82. The publication suggests M82 could be a compelling new tool for… Read More
A gene editing technology has shown promising early results in treating patients with Hepatitis B. Participants in the ELIMINATE-B trial were dosed in PBGENE-HBV, developed by gene editing company Precision BioSciences, at multiple ascending dose levels. Results show PBGENE-HBV, which comprises an ARCUS-encoding mRNA encapsulated in a lipid nanoparticle… Read More
The US Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to TSRA-196, Tessera Therapeutics’ lead in vivo gene editing programme. TSRA-196 is being jointly developed with Regeneron for the treatment of adults with alpha-1 antitrypsin deficiency (AATD) who are homozygous… Read More
David Vallerand, Director of Scientific Engagement for Crown Bioscience, examines the technologies advancing cell and gene therapy drug discovery. … Read More
Manufacturer Omega Bio-tek has presented a new workflow offering a high-throughput, automation-friendly platform at SLAS2026. The workflow, developed in collaboration with Dynamic Devices, integrates the M1272 Mag-Bind Endo-free Plasmid Midi Kit. It is designed to meet the growing demand for scalable, endotoxin-free plasmid purification, which the company says is suitable for sensitive downstream applications including transfection, viral… Read More
Eli Lilly and Seamless Therapeutics are teaming up to develop and commercialise programmable recombinase-based treatments targeting hearing loss indications, using Seamless’ proprietary recombinase platform. The company’s technology performs large, precise DNA insertions in any target gene sequence, and operates independent of the cell’s natural DNA repair… Read More
What does the coming year hold for advanced therapies? Diana Turner asks industry leaders for their predictions for 2026. 2025 was a mixed year for cell and gene therapies (CGT) with some incredible breakthroughs, but also a withdrawal of many companies from their cell therapy programmes. Read More
The US Food and Drug Administration (FDA) has cleared GenEditBio to initiate Phase I/II CLARITY trial activities for its lead in vivo genome-editing programme GEB-101 for TGFBI corneal dystrophy. The Phase 1/2 CLARITY trial will collect initial data on the safety, tolerability and efficacy of… Read More
Diana Turner counts down the biggest DDW news stories of 2025. 10. First use of CAR-T therapy to treat ulcerative colitis In October, a team of physicians at the German Center for Immunotherapy at Erlangen University Hospital succeeded for the first time in treating a… Read More
Duchenne UK has awarded £500,000 ($653,000) to biotech company MyoGene Bio to support the development of an innovative gene editing therapy for Duchenne muscular dystrophy (DMD). The funding is part of Duchenne UK’s Therapeutic Call, a competitive initiative designed to support innovative research with the potential to deliver new treatments… Read More
Intellia Therapeutics announced it has temporarily paused its MAGNITUDE and MAGNITUDE-2 Phase III clinical trials of nex-z for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively. The action follows a report of Grade 4 liver transaminases and increased total bilirubin in a patient who… Read More
