Rocket Pharmaceuticals Inc Present Updated Data from Phase 1 Danon Disease Trial for RP-A501 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022 Transcript

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

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Congrats on the data. So the expression numbers that you've reported, they look in line with or a little higher than the adult cohorts at the same time. What does this imply to you in terms of benefit of earlier treatment?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Got it. That's helpful. And then just 1 more, if I can. How are you thinking about the trend in the LAMP2B grade for the adult patients who were treated early on? It looks like it's decreased and some at the later follow-up. But the trend in vector copy number is less clear. How do you interpret these results? And what does that mean for your expectations for durability?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : I'll also add my congrats. Really great progress here. A few things to expand on from my side. Just on the adult BNP data, I mean, obviously, you highlighted this has been the best lab biomarker. We touched upon it a little bit already, but we've seen sustained lowering in 2 patients, but 2 patients saw the level rise back up above the baseline. And then obviously, when there is [once] or no improvement in the NYSA classification. Could you just -- yes -- what are your thoughts around these trends? Second, just sort of more of a housekeeping question. On your vector copy number data, it looks like some of the numbers have changed versus your 2021 update. Just wondering what the reason is for that? Is the -- have you changed your quantification method, or is it due to your assay centralization? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 30, 2022 / 12:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc Present Updated Data from Phase 1 Danon Disease Trial for RP-A501 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. And on the (inaudible) copy number disparity.