genedrug

Genedrug refers to therapeutics that rely on genetic material to influence biological pathways in order to treat disease. The term covers gene therapy, which introduces functional genetic information; gene editing, which aims to correct or modify faulty DNA; and RNA-based approaches that regulate gene expression or silence disease-causing transcripts. Genedrugs can be delivered directly to the patient in vivo, or produced outside the body and reintroduced as modified cells (ex vivo).

Modalities include gene replacement therapies that supply a working copy of a defective gene, gene silencing therapies such as antisense oligonucleotides or RNA interference, and genome editing tools (for example CRISPR-Cas systems). Delivery methods range from viral vectors (AAV, lentiviral) to non-viral carriers like lipid nanoparticles, as well as ex vivo cellular therapies in which patient-derived cells are engineered before reinfusion. Genedrugs may aim to provide long-lasting effects from a single treatment or require repeat dosing depending on the mechanism and tissue target.

Applications span rare inherited disorders, certain cancers, and some infectious diseases under investigation. A small number of genedrugs have received regulatory approval in various jurisdictions, including Luxturna for RPE65-associated retinal dystrophy and Zolgensma for spinal muscular atrophy, as well as other approved gene therapies and CAR-T cell products that involve genetic modification of cells. Ongoing research continues to expand targets and improve delivery, specificity, and durability.

Concerns in the field include safety risks such as off-target genome edits and immune responses, manufacturing complexity, cost, and equitable access. Regulatory agencies emphasize long-term follow-up to monitor adverse effects. The field remains rapidly evolving, with potential to transform treatment approaches for many conditions, but practical realization requires careful risk-benefit assessment and robust quality control.