Biotech Values

[ghmm]

GT:

On retreatment...
There are a number of efforts I've discovered in researching GT companies. Some have given more details than others and I am sure not all will succeed. My general thinking is some will be successful. Here are some specific ones just going by memory:
1-QURE (I've posted prior about a couple papers they've published)
2-BOLD is the first company I've heard that will try something in the clinic. One of the two Pompe trials they will start will be an intramuscular injection trial (investigator led) where they will try retreatment. They will use a combination of immunosuppressants not just steroids.
3-BMRN though vague have said they aren't giving up on those with preexisting antibodies to their Hemophilia GT. Though no specific were disclosed.
4-??? Don't remember which company has said they are developing vectors that may be able to get around the antibody issues.

On RGNX IP/Licensing.
Not surprising at all since they own the IP for AAV6-10 via U of PA. GT is still relatively early so anything being tested in man (using AAV) is either using one of these vectors or one of the earlier which aren't under RNGX IP. What are the companies going to do just wait to bring products to market? What RGNX hasn't made clear is they say they have different relationships with each licensee perhaps they have somewhere terms go through the products being only GT therapy for indication (for example if they Orphan exclusivity goes beyond the patents).

There is one particularly nice talk where CEO's of various GT companies talk about the industry. They (I recall RGNX & VYGR) acknowledge the industry is in its infancy and things like durability, level of expression, vectors, etc. have a long way to go. Its a little old 2015 but I found it a nice panel.
Here is the link: