The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: The first one that I have is for both of these lead programs, could you talk a bit more about the types of partnerships you're open to looking at, at
this point? Specifically, how are you thinking about different geographies as well as the upfront payment and other financial structures to support
some of the next initiatives at the company, including a focus on ophthalmology? And then also considering the rare pediatric disease designation
and some of the monetary deals that we've seen recently around priority review voucher sales.
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: No, please, not at all.
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: Yes. So that one was really just based off of the different types of deals you're potentially looking to explore for these lead programs considering
capital needs to really support that next initiative at the company, which may include a focus on ophthalmology. So, like, are you looking for
partnerships with like a good capital upfront to really support moving that pipeline along?
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: Okay. Appreciate that. And for MPS IIIA, could you help us understand the 60-month age time point for follow-up based off of the agency feedback?
Is this based on the cognitive age equivalent, natural history data where you see that slope start to -- the downward slope, excuse me, start to
become more apparent at 60 months of age. And then despite the fact that you're looking for a partnership here, is this still a program that you
anticipate having some further durability data from at future conferences?
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: Okay. And if I may sneak in one last question here. I understand MPS IIIB is earlier in development, but is there any particular reason for discontinuing
this program versus perhaps looking at partnering both as a package deal given some of the synergies in MPS IIIA and the programs here?
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