Rocket Pharmaceuticals Inc at Chardan Genetic Medicines & Cell Therapy Manufacturing Summit (Virtual) Transcript

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

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : So Kinnari, thank you for joining us. To start, could you please discuss Rocket's manufacturing capabilities and strategic approach across your ex vivo and in vivo modalities?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Yeah. There you go. CDMOs have visibly amassed considerable expertise in cell and gene therapy manufacturing over the recent years. But there have also been some instances of setbacks of partner sites among a number of programs. So how does rocket minimize the risk of issues at your partner sites?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : And then I guess for both modalities, can you expand a little bit on how you decide what elements to develop internally versus what can be manufactured or outsourced?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Great. So as you mentioned, Rocket is nearing regulatory submissions for two ex vivo lenti programs. So can you walk us through a little bit about what the manufacturing process for this entails? And then we've got a couple of follow-up questions on that.

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : So building on that last point, some autologous antiviral gene therapies have achieved regulatory approvals. But then there's also been peers that have faced delays, specifically with the CMC questions. So what are you focusing on to try to ensure that things go smoothly for your programs? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 25, 2023 / 1:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Chardan Genetic Medicines & Cell Therapy Manufacturing Summit (Virtual)

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : So on that front, in terms of what you've learned from your interactions with regulators, can you talk a little bit about what they're looking for with respect to process and product characterization at a commercial level?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Got it. And so in terms of commercial supply, are you and your partners positioned to supply the commercial market? And as a follow-up, do you anticipate that further scaling might be needed for subsequent programs such as PKD, which does have a larger population? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 25, 2023 / 1:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Chardan Genetic Medicines & Cell Therapy Manufacturing Summit (Virtual)

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Great. So let's turn to AAV. You mentioned Rocket is positioned to start a pivotal trial in Danon soon, and you'll be using product that's manufactured at your facility. So can you talk a little bit more about that and how that, and how the in-house product compares to the product that was used in the Phase 1 trial?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : What changes have enabled the improvements that you talked about, with respect to yield and purity?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : And so what have you learned regarding the key analytics that you need to look out for to ensure that product quality and purity and consistency?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Got it. And so career discussion with regulators, again, echoing similar topics that we just discussed for the lenti programs, can you talk about what they're looking for with respect to the AAV gene therapy, CMC, at the pivotal or commercial stage and as opposed to at the earlier stages?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : So you mentioned that you'll be able to take some of the learnings and some of the work that you've done and apply to the PKP2 program. That program does use a different capsid. So to what extent did you need to tailor a new process for that program versus using something similar to Danon, and more broadly, in terms of efficiencies to the program to share equipment and other components?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Got it. So in terms of scale, is your current capacity at your facility expected to support both commercial products? And in terms of expansion, what is the capabilities to expand scale further?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Absolutely, great. So just stepping back from your specific pipeline, what do you see as the biggest challenges in cell and gene therapy space relating to CMC?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : And sorry, just the last minute or so, are there any innovations that you're particularly excited about on the CMC front?

Question: Geulah Livshits - Chardan Capital Markets, LLC. - Analyst : Great. So on that note, I think we've reached the end of our session. So I'd like to thank you again, Kinnari, for the great discussion and the overview.