Biotech firms are engineering smarter, safer gene delivery systems to enhance the safety and efficacy of genetic medicines.

Feb. 7, 2025 — A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD). Patients with ...

The new bid surpasses an offer SK Capital and Carlyle Group made last month for the gene therapy developer, which Bluebird’s board still supports.

It’s called gene therapy. Gene therapy seeks out the root cause of disease by targeting a single gene within a person’s cells. “As techniques advance, treatment becomes more and more ...

In the complex process of producing a gene therapy, bioprocessors depend on assays to monitor the attributes of the product at various stages. Scientists at Thermo Fisher Scientific focus on ...

Gene delivery is a process ... such as genetic research or gene therapy. Gene delivery methods can be mechanical (e.g. microinjection, electroporation or biolistics), chemical (e.g. lipid or ...

Multidisciplinary coordination is crucial for effective gene therapy administration in Duchenne muscular dystrophy, focusing on institutional readiness and patient-centered care. Guidelines ...

A new study in Human Gene Therapy describes a machine learning (ML) model that can be used as a surrogate for laborious in vitro experiments. This in silico approach aims to increase the fitness ...

In another sign of the challenges facing gene therapy developers, Pfizer has abandoned its haemophilia B treatment Beqvez in all world markets, saying weak demand made the business non-viable.

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

Gene therapy involves using genetic material ... Gene therapies can be delivered using viral vectors, such as adeno-associated viruses (AAV) and lentiviruses, or non-viral methods, including ...