Recent advances in gene and cell therapies include FDA approvals and promising trial results. PTC Therapeutics obtained FDA approval for Sephience, an oral therapy for phenylketonuria, expanding access across age groups. A novel gene therapy in newborn primates demonstrated sustained protection against HIV-1, leveraging early-life immune tolerance. Calidi Biotherapeutics received FDA Fast Track for CLD-201, a stem-cell loaded viral therapy in soft tissue sarcoma. These efforts signify progress in treating conditions with significant unmet medical needs through targeted genetic and cellular interventions.