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: Geulah Livshits - Chardan Capital Markets LLC - Analyst
: Great, thanks for that. So, I guess to drill down on some of the points that you've made during this presentation with regard to the data that has
been presented from IGNITE DMD study thus far, can you elaborate on the significance of -- for example, the pulmonary data that has been presented
today and the patient-reported outcomes. And why it's important to look at these points relative to some of the other programs or, I should say,
metrics that have been evaluated in the space before?
Question: Geulah Livshits - Chardan Capital Markets LLC - Analyst
: Got it. And so, you mentioned also that the data supported additional dosing. Can you give a little bit of color on how you view the totality of the
data that has been presented and also remind us what the key milestones and potential catalysts are for the program going forward?
Question: Geulah Livshits - Chardan Capital Markets LLC - Analyst
: Got it. And you shared a little bit about the capsid discovery work and the engineering (inaudible) that you've done. Any additional color that you
can provide on where the key differences from your perspective are between 001 and 003 in terms of how you envision, let's say, the clinical path
evolves. And then also, a side note on the 003 capsid use there, can you comment on the manufacturability of that capsid? Because that's another
area that we've seen a lot of discussion with regards to the different vectors that have been used.
Question: Geulah Livshits - Chardan Capital Markets LLC - Analyst
: Okay, got it. And I also like to touch on safety a little bit. Obviously, it's been a major topic of discussion within the gene therapy space. The FDA
recently had a panel on the topic and we've also seen some safety signals coming from another DMD gene therapy program. So, can you give some
comments on how you are thinking about safety within the gene therapy space currently and approaches that can be taken to address some of
the challenges, and any takeaways that you had from the recent FDA panel on this?
Question: Geulah Livshits - Chardan Capital Markets LLC - Analyst
: Got it. So, we do have a question from the audience that might be one that you've gotten recently as well. Regarding the current IGNITE DMD
study, can you comment on how you are thinking about the number of patients that might need to be dosed to move forward to Phase III?
Question: Geulah Livshits - Chardan Capital Markets LLC - Analyst
: Got it. That makes sense. And then in the last couple of minutes, from a high-level perspective, what do you think investors, or the market is currently
missing from the Solid story or more generally, the broader story of AAV gene therapy for DMD?
Question: Geulah Livshits - Chardan Capital Markets LLC - Analyst
: All right. Great. So that concludes the session, and thank you both for participating.
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