Orchard Therapeutics (NASDAQ:ORTX) Q2 2020 Earnings Conference Call - Final Transcript

Aug 06, 2020 • 08:00 am ET

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Orchard Therapeutics (NASDAQ:ORTX) Q2 2020 Earnings Conference Call - Final Transcript

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Operator
Operator

Good day, ladies and gentlemen, and thank you for standing by. Welcome to the Orchard Therapeutics Second Quarter 2020 Earnings Conference Call. [Operator Instructions]

At this time, I would like to turn the conference over to Ms. Renee Leck, Director of Investor Relations. Please begin, ma'am.

Executive
Renee Leck

Thanks, operator. Good morning everyone and welcome to Orchard's second quarter 2020 investor call. Our latest corporate slide deck with information supporting today's call can be found on the Investors section of our website, orchardtx.com.

Before we get started, I'd like to remind everyone that statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, including those set forth in our quarterly Form 10-Q filed with the SEC and any other filings that we make. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

And with that, I'll turn the call over to our CEO, Bobby Gaspar.

Executive
Bobby Gaspar

Hello, everyone, and welcome. We're happy to join you today to discuss the progress we've made over the last few months executing Orchard's new strategic vision. You remember that our vision is comprised of four pillars that we feel position Orchard for long-term growth and sustainable value creation. These are: first, prioritizing our portfolio investments and placing emphasis on our neurometabolic programs. Second, establishing a focus commercial model. Third, investing in next generation manufacturing innovations and fourth, creating operational efficiencies.

Today, our Chief Operating Officer, Frank Thomas and I will focus on developments within two of those pillars, including our momentum and progress in neurometabolic disorders and the advancement of our manufacturing strategy. Finally, we've done a thorough analysis of the potential value of OTL-200, and I'll close with some important insights on how that assessment has informed our thinking around price as we approach a potential approval and launch. Later in the year, you'll also hear more about our work in larger indications, including genetic subsets of Frontotemporal Dementia or FTD and Crohn's disease at an investor event we plan to host in the fourth quarter.

Starting with neurometabolic diseases. We have seen that HSC gene therapy has the potential to dramatically improve the lives of patients who are often children by potentially curing them of their disease. We believe this is due to the unique properties of HSCs to naturally migrate into the CNS and deliver therapeutic genes and proteins to the brain which is why we are placing special emphasis on these diseases in our portfolio. Our pipeline stands multiple indications and developmental stages in this field, including a registrational program that would be the first approved therapy for a devastating condition known as metachromatic leukodystrophy or MLD. Two promising proof-of-concept programs in MPS-I and MPS-IIIA