RE:RE:RE:Denali/Sirion deal to get AAVs across the BBB With respect to ultrasound, aside from the problems created by physically opening the BBB, it's hard to make a case for a business model with this method of drug delivery. Application of the method requires multiple medical personnel and technologists, and an operating room with complicated and expensive technologies. I don't see room within the complete procedure package for IP-generated revenue from anything other than the equipment.
xB3 is capable of delivering drugs, by infusion or injection, with essentially uniform distribution throughout the CNS. For most targets, this uniform distribution would be either desired or acceptable. However, there are some target/treatment combinations where the treatment must be delivered only to the target because the treatment may be very toxic if delivered throughout the CNS. Ultrasound may have great value in these situations where delivery of the treatment must be very localized within a small section of the CNS. This may be the only type of indication where ultrasound could do something that xB3 could not. I can't even name such a target/treatment example and I don't think it would have any noticeable effect on xB3's potential commercial value.
With respect to Denali's deal with Sirion to get AAVs (
a gene therapy delivery method) across the BBB, let's face it, Denali needs to do something in that space. The clock is ticking for their F-Star technology. We can get 4 to 6 times as much drug per dose into the CNS compared to that technology.
With respect to AAVs and the gene therapies they deliver, we have already discussed them here. It doesn't make any difference who owns them.
Advanced
gene therapy programs currently target only diseases that are
monogenetic, caused by a single gene. Changing a single gene is complicated enough. Changing multiple genes can have immensely complicated side effects. The two links in this paragraph offer some insight into the field.
We're on the right path. xB3 and its payloads have the potential to generate tens of billions in annual revenue. There is no compelling case to be made that gene therapies, AAVs or ultrasound methods can prevent xB3 from becoming something of great value.
jdstox