Rocket Pharmaceuticals Inc at Bank of America Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Cameron Bozdog - BofA Securities - Analyst : Perfect. Well, maybe let's just dive right into it. And to start broadly, could you briefly describe your platform, especially for those -- for those who may not be as familiar in the audience?

Question: Cameron Bozdog - BofA Securities - Analyst : Perfect. And maybe to kind of focus here on what differentiates you from other developers of gene therapies? Is it maybe the indications you're targeting something else from your platform that differentiates you?

Question: Cameron Bozdog - BofA Securities - Analyst : Great. And then a perfect segue into my next question. Could you maybe comment on your manufacturing capabilities and why this has become such a critical piece for gene therapy developers?

Question: Cameron Bozdog - BofA Securities - Analyst : Perfect. You're currently advancing programs, specifically in cardiology and hematology -- no, what makes these specific indications attractive when you're looking at them from a mechanistic standpoint and maybe from a commercial standpoint as well?

Question: Cameron Bozdog - BofA Securities - Analyst : Great. Maybe shifting gears here to your commercial stage. We're coming up on your first PDUFA on June 30. Can you talk about your preclinical efforts? And what elements you're hoping to have in place at this time?

Question: Cameron Bozdog - BofA Securities - Analyst : And then kind of focusing here on what you think investors should expect as far as the initial pace of uptake? Any chance you can start this process immediately. And then more broadly kind of where you expect the biggest near-term hurdles to be?

Question: Cameron Bozdog - BofA Securities - Analyst : Hurdles, challenges?

Question: Cameron Bozdog - BofA Securities - Analyst : And then if you could briefly just touch on your expectations for what the label could look like?

Question: Cameron Bozdog - BofA Securities - Analyst : Perfect. Then moving here to Fanconi anemia, is the BLA still on track for 1H '24? And then what elements are you still up to do?

Question: Cameron Bozdog - BofA Securities - Analyst : Yes. You make a great point there. Do you think this initial launch could be an approval to just get your foot in the door? And how derisking is that first approval for the overall platform?

Question: Cameron Bozdog - BofA Securities - Analyst : Great. Great. Now maybe turning now to Danon, based on your research, what levels of expression of LAMP2 do you think are necessary to be clinically relevant here?

Question: Cameron Bozdog - BofA Securities - Analyst : And then when you're thinking about your pivotal trial design, was there 1 specific element the FDA was focused on and agreeing to it? Or do you think it was more so the totality of data and -- just what's the overall flexibility potentially due to the fact that your rare disease is with limited options?

Question: Cameron Bozdog - BofA Securities - Analyst : Perfect. And can you briefly touch on the enrollment progress here? Are we still on track to see interim data in 2024? And then ultimately, what should investors be looking for an update -- and yes, what would you constitute, I guess, a good readout here?

Question: Cameron Bozdog - BofA Securities - Analyst : Perfect. Well, it looks like we're out of time here. But thank you so much for such a great, exciting conversation and looking forward to Rocket has a story in the next couple of months.