Rocket Pharmaceuticals, Inc. (RCKT) Presents at TD Cowen 46th Annual Health Care Conference Transcript

Unknown Analyst

Thanks, everyone, for being here for TD Cowen’s 46th Annual Healthcare Conference. Our next session we have here is with Rocket Pharma. And from Rocket, we have Gaurav Shah, the CEO. Thanks so much for being here. It’s really a privilege to have you. I guess let’s go ahead and get started on your overview, the history of some highlights before we get into Q&A. So go ahead.

Gaurav Shah
CEO & Director

Great. Thanks. [indiscernible] Cowen as well. [indiscernible] always back here in Boston. Rocket Pharma, we are a late gene therapy company. We are linearly integrated everything from discovery to development, internal [indiscernible] AAV commercialization. Our focus is rare cardiac diseases using the AAV platform. We also have a hematology and immunology platform using LV ex vivo, more on that in a bit. At the end of last year, we had $189 million, and that will be sufficient to cover cash into Q2 ’27.

And I thought I’d use this time to go through what I think are the most important milestones for the year, and that will be a way to talk about each program one by one. First and foremost, we’re very excited that we have a potential approval with the PDUFA date of March 28, 2026, for KRESLADI, which is our LAD-1 ex vivo lenti program, something that we’ve been working on. You asked about history since near day one of Rocket more than 9 years ago.

LAD-1 is a primary immunodeficiency affects children whose neutrophils cannot extravasate to fight sites of infection and most boys and girls pass away in their single-digit years. And we are