Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

After axing a wide swath of federal research projects and grants, a recent slew of awards offers a glimpse into the kinds of ...

University College London's National Amyloidosis Center leads a multinational team reporting that a single infusion of an in ...

CRISPR Therapeutics’ stock is surging as in-vivo therapies show promise. Learn about CRSP stock's risks, long-term upside, ...

Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) highlight a potential best-in-class profile--The AATD ...

Company is Developing EN-374 as a One-Time Treatment for X-Linked Chronic Granulomatous Disease Ensoma, a genomic medicines company advancing the future of medicine through one-time, in vivo therapies ...

DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc. (DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies, ...

The illustration shows how high trafficking of blood stem cells in the circulation early post-natally allows lentiviral-vector mediated in vivo gene transfer. “After birth, blood stem cells need to ...

Robust efficacy data: ≥90% LDL-C reduction in non-human primates achieved within 48 hours of a single dose of EDIT-401; ≥90% LDL-C reduction in mice with high baseline LDL-C and reduced LDLR function ...

Gene therapy using lentiviral vectors (LVs) has proven effective in ex vivo applications, where a patient’s stem cells are extracted, genetically modified, and then reinfused to achieve therapeutic ...