This is so amazing. Lentiviral therapies are exploding, although I'm used to seeing them referred to as "Cell Therapies" to distinguish them from AAV gene therapy that doesn't use the patients cells to produce the target viral vector.
The trick now is being able to get a commercial manufacturer to take the risk of developing this into an approved product, knowing that a. It's a one time treatment so your customer base erodes down to only new babies born with the condition after a few years, and b. Insurance companies will fight not to pay since there are existing treatments that will likely be cheaper.
Best of luck to whatever startup decides to take on that challenge.
