Beam Therapeutics Inc.

Good morning and welcome to BEAM Therapeutics conference call. At this time, all participants are in a listen-only mode. There will be a question and answer session at the end. Please be advised that this call is being recorded at BEAM's request. I would now like to turn the call over to Holly Manning, Vice President of Investor Relations and External Communications. Please go ahead.

Thank you, operator. Good morning, everyone, and welcome to BEAM's conference call to review updates announced this morning in conjunction with our fourth quarter and year-end 2025 financial results. You can access slides for today's call by going to the investor section of our website, beamtx.com. With me on the call today with prepared remarks are John Evans, our Chief Executive Officer, Dr. Amy Simon, our Chief Medical Officer, Dr. Gopi Shankar, our Chief Scientific Officer, Shravan Amani, our Chief Financial Officer, and Dr. Kiran Musunuru from the University of Pennsylvania. Our President, Dr. Giuseppe Pino-Ciaramella, will join for Q&A. Before we get started, I would like to remind everyone that some of the statements we make on this call will include forward-looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risks, uncertainties, and other factors, including those set forth in the risk factors section of our most recent annual report on Form 10-K and any other filings that we may make with the SEC. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. Except as required by law, BEAM specifically disclaims any obligation to update or revise any forward-looking statements even if our views change. With that, I'll turn the call over to John.

Thanks, Holly, and good morning, everyone. At BEAN, our vision is straightforward but ambitious to provide lifelong cures for patients suffering from serious diseases. We believe base editing has the potential to deliver on that vision through one-time, durable genetic medicines with predictable and reproducible outcomes. Today, we're excited to share several important updates that bring us closer to accomplishing this mission. First, leveraging our platform to bring forward a new and innovative development program to address a serious genetic disease, phenylketonuria, or PKU. And second, further solidifying our balance sheet to support the anticipated commercialization of a potentially transformative one-time base editing therapy for sickle cell disease. BEAM was founded on a simple concept aimed at rewriting broken genes back to normal. Base editing is a next generation form of CRISPR allows us to make precise single-based changes resulting in predictable edits without the need to make double-stranded breaks in dna with consistent gene sequence outcomes conferring potentially lifelong benefit base editing enables predictable reproducible outcomes for patients this scientific foundation underpins everything we do predictability is a theme you'll hear throughout today's discussion we believe it is a powerful driver of progress not just for patients, but across the broader healthcare ecosystem. Predictable outcomes can streamline R&D, reduce development risk, accelerate regulatory pathways, and ultimately improve confidence and deliver value for physicians, patients, and payers alike. Face editing is a highly modular and scalable technology. This means that the core elements of our therapies can be reused again and again. And once they are proven to work a first time, We expect to have a higher probability of technical success as we expand to other genes and other diseases over time. So the power of predictability is built into our business from the start. This is not a one-asset story. It is a repeatable, reproducible model. And as you'll see today, we are now applying that model across a growing pipeline. One of the clearest examples of this platform in action is our liver targeted portfolio. We have built leading lipid nanoparticle, or LNP, capabilities to enable efficient in vivo delivery to the liver that can be leveraged for multiple programs, allowing us to move faster with each successive candidate. We're excited to share today that we're expanding this franchise with an innovative new development program for PKU called Beam 304. Beam 304 exemplifies how we can leverage base editing to directly correct not just one, but multiple disease-causing mutations over time. PKU represents an important strategic expansion of our portfolio and an ideal application of our platform. To start, we have the technology and expertise that positions us well to address this condition. PKU is often caused by a single-point mutation in the fetal alanine hydroxylase, or PAH gene, exactly the type of error base editing is designed to correct. PAH is primarily expressed in hepatocytes, making it highly addressable through LNP delivery, which is an area where we have an industry-leading expertise. There also remains significant unmet need, despite available therapies, in a large population of approximately 20,000 individuals in the U.S., and many more around the world. As Gopi will describe in a moment, our initial focus will be on targeting the two most common mutations found in almost half of patients with PKU. In addition, taking advantage of novel and emerging regulatory pathways, we believe our innovative development approach gives us the potential to address mutations found in a majority of PKU patients over time. Blood phenylalanine, or phe reduction, has been accepted as an endpoint for full approval in both the U.S. and Europe, providing an attractive opportunity for both early clinical proof of concept and an expedited path to market. Taken together, PKU is a compelling opportunity to demonstrate the scalability of our platform and to deliver potentially transformative therapeutic options to patients. With that overview, I'll now turn the call over to Amy to provide additional context on the clinical manifestations of PKU and current standard of care.