FDA just accepted an IND from Voyageur therapeutics for Parkinson’s disease. This trial essentially adds a gene for an enzyme that Aromatic L Amino acid Decarboxylase (AADC) that catalyzes the conversion of levodopa to dopamine. This is added to the putamen (brain) and adds the adeno associated virus that would carry the gene.
It is good to note that there are many companies using AAV as the transfection vehicle that enable gene therapy. A somewhat dated list is in the link below and it is amazing to see how many diseases are being addressed by gene therapy. Some examples are almost obvious – Avexis is delivering the SMN gene to the Spinal Muscular Atrophy patients who have a specific genetic defect.
Hemophilia is another big one and that explains why company like Bioverativ was acquired by Sanofi for their hemophilia assets besides strengthening its Eloctate and Alprolix portfolio to an eventual $10B market.
Either ways, progress from the lab science and journal articles to applied sciences is happening.