The U.S. Food and Drug Administration (FDA) has made significant strides in gene therapy approvals, with several groundbreaking treatments now available for ultra-rare disorders. PTC Therapeutics has achieved FDA approval for Kebilidi, a first-of-its-kind brain-delivered gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. This marks a notable advancement as it is the first gene therapy authorized for direct administration into the brain. Additionally, ClearPoint Neuro has received FDA De Novo Marketing Authorization for its SmartFlow Cannula, designed to facilitate direct delivery of gene therapy to the brain. These approvals highlight the potential of gene therapies to address debilitating enzyme deficiencies and provide new treatment options for patients with rare genetic disorders.