Tessa Romero
JPMorgan Chase & Co, Research Division
Welcome, everyone, to the JPMorgan Healthcare Conference. My name is Tessa Romero, and I’m one of the senior biotech analysts here at JPMorgan. Our next presenting company is Rocket Pharmaceuticals. And presenting on behalf of the company, we have CEO, Gaurav Shah. Gaurav, over to you.
Gaurav Shah
CEO & Director
Thank you for having us. Welcome to 2026. Thanks, Tessa, for hosting us and JPMorgan as well. Standard disclaimer, forward-looking statement disclaimer here. So Rocket is a late-stage biotech company. We have a pipeline of gene therapies that are designed to correct the root cause of complex and rare genetic diseases. We have 2 platforms, AAV and lentiviral and we do everything from discovery to manufacturing, including in-house manufacturing for AAV.
And we think that the secret sauce to success in gene therapy is an asset selection. And we’re focused on assets that target medical conditions that are not only rare, but are devastating or fatal. Often in childhood and that have a direct mechanism of action that can be targeted and focused on the protein that is responsible for the disease and have a reasonable market size to create a business opportunity that can expand over time.
As of September 30, 2025, Rocket reported $222.8 million in cash, cash equivalents and restricted cash. We expect that these resources, excluding any sale of a PRV that could be granted on the KRESLADI approval that’s slated for March 28, 2026, the cash that we have now will be sufficient to fund operations into the second quarter of 2027. And while we don’t expect
