One‑off drugs for single patients

Clinicians and small teams are designing molecular therapies (for example antisense oligonucleotides or tailored gene treatments) intended to treat a single patient’s rare genetic variant, rather than developing mass‑market drugs. These interventions are technically feasible, funded by foundations or families, and move through accelerated, case‑by‑case regulatory paths. — This trend forces policy choices about approval standards, liability, public funding, access inequality, and how regulators scale safety oversight for bespoke biological treatments.