Gene therapy encompasses a broad set of approaches, including in vivo and ex vivo viral and non-viral delivery of corrective genetic material, that are transforming treatment of rare and common diseases. Several gene therapies have achieved regulatory approval for haemophilia, spinal muscular atrophy, and certain inherited retinal dystrophies. Manufacturing scalability, long-term durability of effect, and pricing frameworks remain the defining challenges of the field.
Next-Gen CRISPR Improves Embryo Editing Accuracy, Stoking Ethical Debate
Advanced CRISPR tools enhance precision in human embryo gene editing, reducing off-target effects but intensifying ethical concerns over germline modification.
Prime Medicine Wins Gene-Editing Dispute Against Beam Therapeutics
Prime Medicine has secured a victory in its intellectual property dispute with Beam Therapeutics over foundational gene-editing technology.
ARPA-H Funds $160M Gene Editing Effort for Custom Therapies
ARPA-H launched a $160 million initiative to develop custom gene editing drugs, aiming to create rapid, on-demand therapies for a range of diseases.
Exosomes Emerge as Cell-Free Option in Regenerative Medicine
Exosomes, nanoscale vesicles, are gaining attention in regenerative medicine as a cell-free therapeutic alternative, offering potential benefits over traditional cell therapies.