Orchard Therapeutics plc (NASDAQ:ORTX) Q3 2022 Earnings Conference Call November 14, 2022 8:00 AM ET

Company Participants

Bobby Gaspar - Chief Executive Officer

Braden Parker - Chief Commercial Officer

Frank Thomas - President and COO

Leslie Meltzer - Chief Medical Officer

Conference Call Participants

Pete Stavropoulos - Cantor Fitzgerald

David Hoang - SMBC Group

Operator

Thank you for standing by. My name is Rihanna and I will be your conference operator today. At this time, I would like to welcome everyone to the Orchard Therapeutics Q3 2022 Earnings Call. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. [Operator Instructions]

I will now turn the call over to CEO, Bobby Gaspar. You may begin your conference.

Bobby Gaspar

Thank you. Hello, everyone, and welcome to Orchard Therapeutics Third Quarter 2022 Financial Results and Corporate Webcast. I'm CEO and Co-Founder, Bobby Gaspar. Before we get started, I want to remind everyone that this presentation contains forward-looking statements. Please refer to the slide and our latest SEC filings for more information.

I have three members of the leadership team joining me on today's call. Braden Parker, our Chief Commercial Officer, will highlight recent commercial activities for Libmeldy; Frank Thomas, President and Chief Operating Officer, will review our financial results for the quarter; and Leslie Meltzer, our Chief Medical Officer, will also be joining for Q&A.

As many of you know, Orchard's aim is to leverage the power of gene modified hematopoietic stem cells or HSCs to treat and potentially cure severe genetic diseases. Our focus in this area is twofold. We have a commercial and late-stage clinical pipeline of therapies for neurodegenerative conditions, where the migration of gene modified HSCs into the CNS is key to delivering the gene where it is needed.

Secondly, we are developing HSC gene therapy for specific larger disease indications with genetic susceptibilities, such as the NOD2 form of Crohn's disease, one of our research programs approaching IND-enabling studies next year. With Libmeldy, our HSC gene therapy for metachromatic leukodystrophy or MLD, we have seen how this approach can lead to clinical benefit, regulatory approval, and increasingly sustained commercial success.

Throughout this year, we have made great strides for curing reimbursement for Libmeldy in Europe, establishing qualified treatment centers and treating our first MLD patient with commercial therapy. We are especially encouraged by the continued progress we are making to identify, refer and treat eligible patients and believe disease awareness for MLD is growing among the physicians diagnosing and managing patients.