The past decade has witnessed extraordinary progress in developing and approving new therapies. Notably, the first gene therapies for an inherited retinopathy and a CNS disorder (spinal muscular atrophy; SMA) have emerged, offering significant benefits. SMA has also proven amenable to both drug and antisense oligonucleotide therapies targeting RNA splicing. Amyloid-targeting antibodies for Alzheimer’s disease have shown modest disease-modifying efficacy. Furthermore, early-stage trials suggest potential for antisense oligonucleotides in some forms of ALS and epidural stimulation for spinal cord injury. The future holds promise with advancements in gene therapy for other inherited disorders, the potential of gene editing for neurological conditions, and ongoing stem cell therapy trials. Bioengineering and neuromodulation are already used, with further progress in spinal cord injury treatments and device development. Overcoming the “valley of death” in funding and maintaining long-term investment are crucial for translating these discoveries into approved therapies, as evidenced by the decades-long journeys of monoclonal antibodies and gene therapy.