Pipeline | Prime Medicine

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style="background-image:url('https://primemedicine.com/wp-content/uploads/2022/07/banner-science.png');"> <div class="container-fluid"> <div class="row"> <div class="col-sm-12 content"> <h1>Pipeline</h1> </div> </div> </div> </div> <div class="content-wrapper"> <div class="section " id="our-pipeline"> <div class="container-wrapper section-padding bg-white"> <div class="container-fluid"> <div class="row"> <div class="col-md-12 content content-layer"> <h2>Our Pipeline</h2> <p class="headline">Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: hematology, immunology & oncology, liver and lung. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing’s broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. For more information, please visit <a href="http://www.primemedicine.com">www.primemedicine.com</a>.</p> </div> </div> </div> </div> </div> <div class="section" id="pipeline"> <div class="container-wrapper"> <div class="container-fluid"> <div class="row"> <div class="col-md-12 content content-layer"> <h2>prime medicine pipeline</h2> </div> </div> </div> <div class="container-fluid extra-wide"> <div id="pipeline-table"> <div class="row visible-xs" style="display:none !important;"> <div class="col-md-12 content content-layer"> <div id="pipeline-mobile"> <div class="pt-pc-mobile" id="pc-1"> <div class="pt-mobile-app"> <div class="pt-app-mobile"> <a href="" class="pt-pc-mobile-header"> <div class="mobile-cell-wrapper"><div class="mobile-cell"></div></div> <h3>Hematology, Immunology & Oncology: </h3> </a> <div class="cr-content-mobile"> <h5>COMMERCIALIZATION RIGHTS:</h5> </div> <div class="pc-mobile"> <div class="candidate-title candidate-info"> <p><strong></strong><br /> <span></span></p> <div class="pt-bar-container-mobile"> <div class="pt-bar" style="width:88%;"><span></span> </div> </div> </div> </div> <div class="pc-mobile"> <div class="candidate-title candidate-info"> <p><strong></strong><br /> <span></span></p> <div class="pt-bar-container-mobile"> <div class="pt-bar" style="width:13%;"><span></span> </div> </div> </div> </div> <div class="pc-mobile"> <div class="candidate-title candidate-info"> <p><strong></strong><br /> <span></span></p> <img class="ct-logo" src="https://primemedicine.com/wp-content/uploads/2024/09/Bristol-Myers_Squibb_logo.svg_.png" alt="" /> <div class="pt-bar-container-mobile"> <div class="pt-bar" style="width:40%;"><span></span> </div> </div> </div> </div> </div> </div> </div> <div class="pt-pc-mobile" id="pc-2"> <div class="pt-mobile-app"> <div class="pt-app-mobile"> <a href="" class="pt-pc-mobile-header"> <div class="mobile-cell-wrapper"><div class="mobile-cell"></div></div> <h3>Liver: </h3> </a> <div class="cr-content-mobile"> <h5>COMMERCIALIZATION RIGHTS:</h5> </div> <div class="pc-mobile"> <div class="candidate-title candidate-info"> <p><strong></strong><br /> <span></span></p> <div class="pt-bar-container-mobile"> <div class="pt-bar" style="width:40%;"><span></span> </div> </div> </div> </div> <div class="pc-mobile"> <div class="candidate-title candidate-info"> <p><strong></strong><br /> <span></span></p> <div class="pt-bar-container-mobile"> <div class="pt-bar" style="width:40%;"><span></span> </div> </div> </div> </div> </div> </div> </div> <div class="pt-pc-mobile" id="pc-3"> <div class="pt-mobile-app"> <div class="pt-app-mobile"> <a href="" class="pt-pc-mobile-header"> <div class="mobile-cell-wrapper"><div class="mobile-cell"></div></div> <h3>Lung: </h3> </a> <div class="cr-content-mobile"> <h5>COMMERCIALIZATION RIGHTS:</h5> </div> <div class="pc-mobile"> <div class="candidate-title candidate-info"> <p><strong></strong><br /> <span></span></p> <img class="ct-logo" src="https://primemedicine.com/wp-content/uploads/2024/02/cystic-fibrosis-logo.png" alt="" /> <div class="pt-bar-container-mobile"> <div class="pt-bar" style="width:13%;"><span></span> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="row"> <div class="col-md-12 content content-layer"> <div id="pipeline"> <div class="pt-row"> <div class="pt-header" id="pt-header"> <div class="pt-column th1 p11"> <div class="pt-l2-column p100 pt-cell p1"> <h5>Modular <br />Platform</h5> </div> </div> <div class="pt-column th2 p89"> <div class="pt-l2-column p28 pt-cell p2"> <h5>Indication</h5> </div> <div class="pt-l2-column p12 pt-cell p3"> <h5>Delivery</h5> </div> <div class="pt-l2-column p15 pt-cell p4"> <h5>Discovery</h5> </div> <div class="pt-l2-column p15 pt-cell p5"> <h5>Lead <br />Optimization</h5> </div> <div class="pt-l2-column p15 pt-cell p6"> <h5>IND-<br />Enabling</h5> </div> <div class="pt-l2-column p15 pt-cell p7"> <h5>Phase 1/2</h5> </div> </div> </div> </div> <div class="inner-pipeline"> <div class="pt-cat-table" id="pt-1">  <div class="pt-row target-row" style="border-color:#f10c7c;"> <div class="pt-column p11 pt-cell p1" style="border-color:#f10c7c;"> <h6 style="color:#f10c7c;">Hematology, Immunology & Oncology</h6> </div> <div class="pt-column p89"> <div class="pc-row" style="border-color:#f10c7c;"> <div class="pt-l2-column p28 indication-title pt-cell p2"> <h5 style="color:#f10c7c;">p47<sup>phox</sup> Chronic Granulomatous Disease (CGD)</h5> </div> <div class="pt-l2-column p12 pt-cell p3 delivery-title"> <h6><em>ex vivo</em></h6> </div> <div class="pt-l2-column p15 pt-cell p4"> </div> <div class="pt-l2-column p15 pt-cell p5"> </div> <div class="pt-l2-column p15 pt-cell p6"> </div> <div class="pt-l2-column p15 pt-cell p7"> </div> <div class="pt-bar-container"> <div class="pt-bar" style="background-color:#f10c7c;width:88%;"> </div> </div> </div> <div class="pc-row" style="border-color:#f10c7c;"> <div class="pt-l2-column p28 indication-title pt-cell p2"> <h5 style="color:#f10c7c;">X-linked CGD (with PASSIGE™)</h5> </div> <div class="pt-l2-column p12 pt-cell p3 delivery-title"> <h6><em>ex vivo</em></h6> </div> <div class="pt-l2-column p15 pt-cell p4"> </div> <div class="pt-l2-column p15 pt-cell p5"> </div> <div class="pt-l2-column p15 pt-cell p6"> </div> <div class="pt-l2-column p15 pt-cell p7"> </div> <div class="pt-bar-container"> <div class="pt-bar" style="background-color:#f10c7c;width:13%;"> </div> </div> </div> <div class="pc-row" style="border-color:#f10c7c;"> <div class="pt-l2-column p28 indication-title pt-cell p2"> <h5 style="color:#f10c7c;"><em>Ex vivo</em> CAR-T<sup>1</sup> (with PASSIGE™)</h5> </div> <div class="pt-l2-column p12 pt-cell p3 delivery-title"> <h6><em>ex vivo</em></h6> </div> <div class="pt-l2-column p15 pt-cell p4"> </div> <div class="pt-l2-column p15 pt-cell p5"> </div> <div class="pt-l2-column p15 pt-cell p6"> </div> <div class="pt-l2-column p15 pt-cell p7"> </div> <div class="pt-bar-container"> <div class="pt-bar" style="background-color:#f10c7c;width:40%;"> </div> <img class="ct-logo" src="https://primemedicine.com/wp-content/uploads/2024/09/Bristol-Myers_Squibb_logo.svg_.png" alt="" /> </div> </div> </div> </div> </div> <div class="pt-cat-table" id="pt-2">  <div class="pt-row target-row" style="border-color:#f4a30d;"> <div class="pt-column p11 pt-cell p1" style="border-color:#f4a30d;"> <h6 style="color:#f4a30d;">Liver</h6> </div> <div class="pt-column p89"> <div class="pc-row" style="border-color:#f4a30d;"> <div class="pt-l2-column p28 indication-title pt-cell p2"> <h5 style="color:#f4a30d;">Wilson’s Disease</h5> </div> <div class="pt-l2-column p12 pt-cell p3 delivery-title"> <h6>LNP</h6> </div> <div class="pt-l2-column p15 pt-cell p4"> </div> <div class="pt-l2-column p15 pt-cell p5"> </div> <div class="pt-l2-column p15 pt-cell p6"> </div> <div class="pt-l2-column p15 pt-cell p7"> </div> <div class="pt-bar-container"> <div class="pt-bar" style="background-color:#f4a30d;width:40%;"> </div> </div> </div> <div class="pc-row" style="border-color:#f4a30d;"> <div class="pt-l2-column p28 indication-title pt-cell p2"> <h5 style="color:#f4a30d;">Undisclosed</h5> </div> <div class="pt-l2-column p12 pt-cell p3 delivery-title"> <h6>LNP</h6> </div> <div class="pt-l2-column p15 pt-cell p4"> </div> <div class="pt-l2-column p15 pt-cell p5"> </div> <div class="pt-l2-column p15 pt-cell p6"> </div> <div class="pt-l2-column p15 pt-cell p7"> </div> <div class="pt-bar-container"> <div class="pt-bar" style="background-color:#f4a30d;width:40%;"> </div> </div> </div> </div> </div> </div> <div class="pt-cat-table" id="pt-3">  <div class="pt-row target-row" style="border-color:#00c5ed;"> <div class="pt-column p11 pt-cell p1" style="border-color:#00c5ed;"> <h6 style="color:#00c5ed;">Lung</h6> </div> <div class="pt-column p89"> <div class="pc-row" style="border-color:#00c5ed;"> <div class="pt-l2-column p28 indication-title pt-cell p2"> <h5 style="color:#00c5ed;">Cystic Fibrosis<sup>2</sup></h5> </div> <div class="pt-l2-column p12 pt-cell p3 delivery-title"> <h6>LNP/AAV</h6> </div> <div class="pt-l2-column p15 pt-cell p4"> </div> <div class="pt-l2-column p15 pt-cell p5"> </div> <div class="pt-l2-column p15 pt-cell p6"> </div> <div class="pt-l2-column p15 pt-cell p7"> </div> <div class="pt-bar-container"> <div class="pt-bar" style="background-color:#00c5ed;width:13%;"> </div> <img class="ct-logo" src="https://primemedicine.com/wp-content/uploads/2024/02/cystic-fibrosis-logo.png" alt="" /> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="row content-margin"> <div class="col-md-12 content content-layer"> <p><span style="color: #381f9c;">Prime Medicine is identifying opportunities to advance its other programs, including neurological diseases, cell therapy, ocular diseases and hearing loss, in partnership or through internal efforts in the future.</span></p> <p style="color: #999999; font-size: 17px; line-height: 22px;"> <sup>1</sup> In September 2024, Prime entered into a strategic license and broad collaboration agreement with Bristol Myers Squibb to develop and commercialize ex vivo T-cell Prime Editor products.<br /> <sup>2</sup> In January 2024, entered into agreement with CF Foundation for up to $15 million to support development of Prime Editors for Cystic Fibrosis. </p> </div> </div> </div> </div> </div> </div> <div class="section " id="clinical-programs"> <div class="container-wrapper section-padding bg-white"> <div class="container-fluid"> <div class="row"> <div class="col-md-12 content content-layer"> <h2>Clinical Programs</h2> <h4>Prime-0101 Study: Chronic Granulomatous Disease</h4> <p>Prime Medicine is currently enrolling patients in Prime-0101, a Phase 1/2 clinical trial evaluating the Prime Editing program PM359 for the treatment of p47<sup>phox</sup> Chronic Granulomatous Disease (CGD). People with p47<sup>phox</sup> CGD inherit a NCF1 gene mutation from each parent, which causes the body to produce abnormal white blood cells that lead to inflammation and increased susceptibility to infections. Prime-0101 will evaluate the safety and efficacy of investigational therapy PM359 in reducing or preventing infections and inflammatory conditions in people living with p47<sup>phox</sup> CGD. Learn more about CGD <a href="/patients-caregivers/#about-our-programs">here</a>.</p> <p>Learn more about Prime-0101 <a href="/patients-caregivers/#current-clinical-trials">here</a>, visit <a href="https://clinicaltrials.gov/study/NCT06559176" target="_blank" rel="noopener">clinicaltrials.gov</a> or email us at <a href="mailto:cgdtrial@primemedicine.com">cgdtrial@primemedicine.com</a> for more information.</p> <h4>About PM359</h4> <p>PM359, Prime Medicine’s first product candidate within its hematology, immunology & oncology area of focus, is an investigational therapy designed to treat the p47<sup>phox</sup> variant of CGD. PM359 is comprised of autologous hematopoietic stem cells (HSCs) modified ex vivo (that is, outside the body) using an all RNA-based Prime Editor that has been designed to correct a high percentage of cells containing the disease-causing mutation. The Prime Editor is delivered into the HSCs by electroporation, which is a clinically and commercially validated delivery technology. PM359 has received rare pediatric drug designation and orphan drug designation from the U.S. Food and Drug Administration. </p> </div> </div> </div> </div> </div> </div> </div> <footer class="dark"> <div class="container"> <div class="row"> <div class="col-md-12 content"> <a href="https://primemedicine.com"><h1 class="text-center">prime_</h1></a> </div> </div> <div class="row"> <div class="col-md-12"> <ul id="menu-footer-menu" class="nav navbar-nav"><li id="menu-item-211" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-211"><a href="https://primemedicine.com/about/"><span class="nav-label">About<span class="underscore">_</span></span></a></li> <li id="menu-item-214" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-214"><a href="https://primemedicine.com/science/"><span class="nav-label">Science<span class="underscore">_</span></span></a></li> <li id="menu-item-482" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-482"><a href="https://investors.primemedicine.com"><span class="nav-label">Investors<span class="underscore">_</span></span></a></li> <li id="menu-item-212" class="menu-item menu-item-type-post_type menu-item-object-page menu-item-212"><a href="https://primemedicine.com/careers/"><span class="nav-label">Careers<span class="underscore">_</span></span></a></li> <li id="menu-item-215" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-215"><a target="_blank" href="https://www.linkedin.com/company/prime-medicine/"><span class="nav-label"><i class="fab fa-linkedin-in"></i><span class="underscore">_</span></span></a></li> <li id="menu-item-571" class="menu-item menu-item-type-custom menu-item-object-custom menu-item-571"><a target="_blank" href="https://twitter.com/PrimeMedicine"><span class="nav-label"><i class="fab fa-twitter"></i><span class="underscore">_</span></span></a></li> </ul> </div> </div> <div class="row"> <div class="col-md-12 content"> <p class="text-center"><a href="mailto:info@primemedicine.com">Contact Us</a> | <a href="/privacy-policy">Privacy Policy</a> | <a href="/terms-of-use">Terms of Use</a></p> </div> </div> <div class="row"> <div class="col-md-12 content"> <p class="text-center">© 2021-2023 Prime Medicine, Inc. 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