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
: Congrats on both of these recent data sets. The first question I had was related to the ophthalmology portfolio. So we haven't seen as many dual
AAV vector strategies yet in gene therapy. I know it's something that others have talked about for some time. So maybe the first part of the question
is just understanding what you think is differentiated about your construct that led to the successful findings at this preclinical stage. And then the
latter half of that question is just, frankly, looking at the dual AAV vector landscape, what are the expectations on how you would expect this to
translate into human studies. And I guess the key things to look out for, given the difference between the models.
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: The second part of the question was just essentially on how you think it's going to translate into human studies. And I guess, what are the key
things to consider the risks of course, going from the different models outside of what you normally expect.
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MAY 24, 2023 / 12:30PM, ABEO.OQ - Q1 2023 Abeona Therapeutics Inc Earnings Call and Portfolio Update
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: And then for the autosomal dominant optic atrophy study that you presented on, you have the bullet here that visual acuity assessments demonstrate
function recovery. I think with a lot of the AAV ophthalmology gene therapy trials, the goal essentially is to show slowing down of disease progression
rather than reversal. So I guess I'm just kind of interested in the context of that comment. And maybe how you think about this clinically and
understand a lot of it is going to have to do with age of intervention. And these different diseases progress at different rates, but what the underlying
goal you're essentially looking at will be.
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: Great. And then last question for me related to ARDA. ISID 2 key new findings, we're just understanding the effects at an earlier point of valuation
and then also around some of these other secondary end points. So since you presented these data, I wanted to hear as you have all these discussions
with thought leaders, how important these 2 components are. And I know in the past, we've talked about looking at understanding pain reductions
correlated with wound healing, but now you have a number of other data points to support these notions.
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