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Cell and Gene Therapy Innovations | Children's Hospital of Philadelphia
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href="/inpatient-care-locations" class="tertiary-nav__menu-link tertiary-nav__menu-link--link tertiary-nav__menu-link--level-1" data-drupal-link-system-path="node/122201"> <span class="tertiary-nav__menu-link-inner tertiary-nav__menu-link-inner--level-2"> Inpatient Care for Kids </span> </a> </li> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-1"> <a href="/community-affiliates" class="tertiary-nav__menu-link tertiary-nav__menu-link--link tertiary-nav__menu-link--level-1" data-drupal-link-system-path="node/122202"> <span class="tertiary-nav__menu-link-inner tertiary-nav__menu-link-inner--level-2"> Community Hospital Affiliates </span> </a> </li> </ul> </li> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-0 tertiary-nav__menu-item--has-children"> <button class='tertiary-nav__button-toggle no-link' data-drupal-selector='tertiary-nav-submenu-toggle-button' aria-expanded='false'> <div class="button-title"> Patients & Families </div> <span class='icon--menu-toggle'> <chop-icon name='chevron-down' library='system' size='1.5rem'></chop-icon> </span> </button> <ul class="menu tertiary-nav__menu tertiary-nav__menu--level-1"> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-1"> <a href="/patients-and-families" class="tertiary-nav__menu-link tertiary-nav__menu-link--link tertiary-nav__menu-link--level-1" data-drupal-link-system-path="node/100006"> <span class="tertiary-nav__menu-link-inner tertiary-nav__menu-link-inner--level-2"> Resources for families </span> </a> </li> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-1"> <a href="/caring-for-your-child" class="tertiary-nav__menu-link tertiary-nav__menu-link--link tertiary-nav__menu-link--level-1" data-drupal-link-system-path="caring-for-your-child"> <span class="tertiary-nav__menu-link-inner tertiary-nav__menu-link-inner--level-2"> Caring for your child </span> </a> </li> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-1"> <a href="/patients-and-visitors/obtaining-medical-records" class="tertiary-nav__menu-link tertiary-nav__menu-link--link tertiary-nav__menu-link--level-1" data-drupal-link-system-path="node/115284"> <span class="tertiary-nav__menu-link-inner tertiary-nav__menu-link-inner--level-2"> Request medical records </span> </a> </li> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-1"> <a href="/research-studies" class="tertiary-nav__menu-link tertiary-nav__menu-link--link tertiary-nav__menu-link--level-1" data-drupal-link-system-path="node/122311"> <span class="tertiary-nav__menu-link-inner tertiary-nav__menu-link-inner--level-2"> Find research studies </span> </a> </li> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-1 tertiary-nav__menu-item--has-children"> <button class='tertiary-nav__button-toggle no-link' data-drupal-selector='tertiary-nav-submenu-toggle-button' aria-expanded='false'> <div class="button-title"> Navigating your visit </div> <span class='icon--menu-toggle'> <chop-icon name='chevron-down' library='system' size='1.5rem'></chop-icon> </span> </button> <ul class="menu tertiary-nav__menu tertiary-nav__menu--level-2"> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-2"> <a href="/philadelphia-campus/your-childs-inpatient-visit" class="tertiary-nav__menu-link tertiary-nav__menu-link--link tertiary-nav__menu-link--level-2"> <span class="tertiary-nav__menu-link-inner tertiary-nav__menu-link-inner--level-3"> Inpatient stays in Philadelphia </span> </a> </li> <li class="tertiary-nav__menu-item tertiary-nav__menu-item--link tertiary-nav__menu-item--level-2"> <a href="/king-prussia-campus/your-childs-inpatient-visit" class="tertiary-nav__menu-link 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<div>The future of improving children’s health depends on making breakthroughs at the microscopic level: by treating diseases with cell and gene therapy. </div> </div> <div class="render-appointment-button"></div> </chop-image-hero> <chop-grid class="layout one-col one-col--dynamic" background-color="transparent" content-max-width="medium" gap-size="small" y-padding="0" x-padding="true"> <chop-column class="layout__region layout__region--main gap-y-4 md:gap-y-16" vertical-align="top" horizontal-align="left" col-span-default="12" col-span-md="12" row-span="1"> </chop-column> </chop-grid> <chop-grid class="layout full-width full-width--dynamic" background-color="transparent" content-max-with="medium" gap-size="small" y-padding="small" x-padding="true" > <chop-column class="layout__region layout__region-- gap-y-4 md:gap-y-16" vertical-align="top" horizontal-align="left" col-span-default="12" col-span-md="12" row-span="1" > <div><chop-styled-text><p>Across Children’s Hospital of Philadelphia (CHOP) — in multiple departments and laboratories — we are committed to discovering the next life-changing treatments. As pioneers of the first FDA-approved gene and cell therapies for children, we bring solutions from our labs to the bedside quickly and safely — so children with severe illnesses can reach their full potential. </p></chop-styled-text></div> <div> <chop-column class="gap-y-4 md:gap-y-12" vertical-align="top" horizontal-align="left" col-span-default="12" col-span-md="9" hide-first-jumpnav-jump > <div class="paragraph paragraph--type--three-stats paragraph--view-mode--default w-full"> <chop-container container-width="wide"> <chop-card-grid equal-height border max-cards="3" border-gap="4.375rem" class="px-4" > <div class="paragraph paragraph--type--stats paragraph--view-mode--default w-full"> <chop-stat stat-color="dark_blue"> <div slot="stat"> <div>80+</div> </div> <div slot="stat-label"> <div>faculty</div> </div> <div slot="description"> <div><chop-styled-text><p>More than 80 faculty engaged in research </p></chop-styled-text></div> </div> </chop-stat> </div> <div class="paragraph paragraph--type--stats paragraph--view-mode--default w-full"> <chop-stat stat-color="green"> <div slot="stat"> <div>20+</div> </div> <div slot="stat-label"> <div>programs </div> </div> <div slot="description"> <div><chop-styled-text><p>More than 20 unique clinical and pre-clinical programs</p></chop-styled-text></div> </div> </chop-stat> </div> <div class="paragraph paragraph--type--stats paragraph--view-mode--default w-full"> <chop-stat stat-color="dark_pink"> <div slot="stat"> <div>45+</div> </div> <div slot="stat-label"> <div>clinical trials</div> </div> <div slot="description"> <div><chop-styled-text><p>More than 45 active clinical trials </p></chop-styled-text></div> </div> </chop-stat> </div> </chop-card-grid> </chop-container> </div> <div class="paragraph paragraph--type--card-circle-image paragraph--view-mode--default w-full"> <chop-container container-width="full" background-color="lightest_blue" content-align="" > <chop-small-circle-feature theme-color="lightest_blue" content-align="" > <div slot="image"> </div> <div slot="heading"> <chop-heading level-size="lg"> <h3 class="chop-headline headline-lg">FDA approves two gene therapies for sickle cell disease </h3> </chop-heading> </div> <div slot="description"> <chop-styled-text><p>The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). </p></chop-styled-text> </div> <div slot="cta-link"> <chop-cta-link link-variant="primary" icon-name="chevron-right"> <a href="/news/fda-approves-two-gene-therapies-sickle-cell-disease">About the breakthroughs</a> </chop-cta-link> </div> </chop-small-circle-feature> </chop-container> </div> <div class="paragraph paragraph--type--card-callout paragraph--view-mode--default w-full"> <chop-callout-card theme-color="beige" icon-name="microphone" content-align="12" > <div slot="heading"> <chop-heading level-size="lg"> <h3>FDA-approved cell & gene therapies </h3> </chop-heading> </div> <div slot="description"> <chop-styled-text><p>There are currently several FDA-approved cell and gene therapies available for use in children in the United States. Two of them were developed by experts at Children's Hospital of Philadelphia. </p></chop-styled-text> </div> <div slot="links"> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.pfizer.com/products/product-detail/beqvez" target="_blank">BEQVEZ ™</a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.casgevy.com/" target="_blank">CASGEVY™ </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.elevidys.com/" target="_blank">ELEVIDYS </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/treatments/gene-therapy-hemophilia-b">HEMGENIX® </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/treatments/car-t-cell-therapy-immunotherapy-b-cell-acute-lymphoblastic-leukemia">KYMRIAH® </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://lenmeldy.com/" target="_blank">LENMELDY™ </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/treatments/gene-therapy-inherited-retinal-dystrophy-luxturna">LUXTURNA® </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.lyfgenia.com/" target="_blank">LYFGENIA™ </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.roctavian.com/" target="_blank">ROCTAVIAN™ </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.skysona.com/" target="_blank">SKYSONA® </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.vyjuvek.com/" target="_blank">VYJUVEK™ </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/treatments/gene-therapy-spinal-muscular-atrophy-sma">ZOLGENSMA® </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/treatments/gene-therapy-transfusion-dependent-beta-thalassemia">ZYNTEGLO™ </a> </chop-cta-link> </div> </chop-callout-card> </div> <div class="paragraph paragraph--type--expandable-two-column-list paragraph--view-mode--default w-full"> <chop-expandible-link-list variant="default"> <chop-heading level-size="lg" slot="heading"> <h2> <chop-styled-text>Diseases of focus</chop-styled-text> </h2> </chop-heading> <div slot="description"><chop-styled-text><p>Diseases and disorders with known and potential targets for cell and gene therapies are the focus of research in numerous departments and divisions across CHOP. Conditions with active research, clinical trials and treatments include: </p></chop-styled-text></div> <div slot="list"> <chop-styled-text> <ul> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/cellular-therapy-and-transplant-section" aria-label="Cancer" > Cancer </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/gene-therapy-neurological-disorders" aria-label="Neurological disorders " > Neurological disorders </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/gene-therapy-hematological-disorders" aria-label="Hematological disorders " > Hematological disorders </a> </chop-cta-link> </li> <li> Ophthalmological disorders </li> <li> Immunological/Inflammatory disorders </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/genetherapy4inheritedmetabolicdisorders" aria-label="Metabolic disorders " > Metabolic disorders </a> </chop-cta-link> </li> <li> Lysosomal storage disorders </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/treatments/gene-therapy-genetic-hearing-loss" aria-label="Auditory disorders " > Auditory disorders </a> </chop-cta-link> </li> </ul> </chop-styled-text> </div> <span slot="opened-button-text">View all</span> <span slot="closed-button-text">View all</span> </chop-expandible-link-list> </div> <div class="paragraph paragraph--type--card-content-width paragraph--view-mode--default w-full"> <chop-content-width-card image-position="left" icon-name="campaign" color="light_blue" class= > <div slot="eyebrow"><chop-heading level-size="eyebrow"><span> <chop-styled-text>Success Stories</chop-styled-text> </chop-heading></div> <div slot="heading"> <chop-heading level-size="lg"> <h3><a href="/centers-programs/cell-and-gene-therapy-innovations/patient-stories"> <chop-styled-text>Gene therapy for Duchenne Muscular Dystrophy: Cash's story</chop-styled-text> </a></h3> </chop-heading> </div> <div slot="description"> <chop-styled-text><p>Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.</p></chop-styled-text> </div> <div slot="cta-link"> <chop-cta-link link-variant="link" icon-name="chevron-right" is-reversed> <a href="/centers-programs/cell-and-gene-therapy-innovations/patient-stories" alt="View more success stories">View more success stories</a> </chop-cta-link> </div> <div slot="image" part="image"> <div class="media media--type-image media--view-mode-content-width-card"> <picture> <source srcset="/sites/default/files/styles/square_100_480x480_2x/public/gene-therapy-duchenne-muscular-dystrophy-cash-s-story-16x9.webp?itok=eH7G6UP0 480w, /sites/default/files/styles/square_100_640x640_1x/public/gene-therapy-duchenne-muscular-dystrophy-cash-s-story-16x9.webp?itok=oBa8dXUf 640w, /sites/default/files/styles/square_100_720x720_2x/public/gene-therapy-duchenne-muscular-dystrophy-cash-s-story-16x9.webp?itok=z1VflSoT 720w, 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/sites/default/files/styles/landscape_133_4x3_4000x3000_2x/public/gene-therapy-duchenne-muscular-dystrophy-cash-s-story-16x9.jpg?itok=p26jXq-M 4000w" type="image/jpeg" sizes="100vw" width="960" height="720"/> <img alt="" src="/sites/default/files/styles/landscape_133_4x3_160x120_1x/public/gene-therapy-duchenne-muscular-dystrophy-cash-s-story-16x9.jpg?itok=UW6n5TWA" width="160" height="120" loading="lazy"/> </picture> </div> </div> </chop-content-width-card> </div> <div class="paragraph paragraph--type--expandable-two-column-list paragraph--view-mode--default w-full"> <chop-expandible-link-list variant="default"> <chop-heading level-size="lg" slot="heading"> <h2> <chop-styled-text>Clinical programs offering cell & gene therapies</chop-styled-text> </h2> </chop-heading> <div slot="description"><chop-styled-text><p>Children facing serious illness for which cell and gene therapies are available are cared for in the CHOP program that can best treat their specific illness. These include: </p></chop-styled-text></div> <div slot="list"> <chop-styled-text> <ul> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/cellular-therapy-and-transplant-section" aria-label="Cellular Therapy and Transplant Section " > Cellular Therapy and Transplant Section </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/cancer-immunotherapy-program" aria-label="Cancer Immunotherapy Program " > Cancer Immunotherapy Program </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/sickle-cell-and-red-cell-disorders-curative-therapy-center-cured" aria-label="Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED) " > Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED) </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/news/novel-therapeutics-bleeding-disorders-not-bleeding-program" aria-label="Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program " > Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/division-ophthalmology" aria-label="Division of Ophthalmology " > Division of Ophthalmology </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/division-neurology" aria-label="Division of Neurology " > Division of Neurology </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/metabolic-disease-program" aria-label="Metabolic Disease Program " > Metabolic Disease Program </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/division-hematology" aria-label="Division of Hematology " > Division of Hematology </a> </chop-cta-link> </li> <li> <chop-cta-link link-variant="link" full-width="" icon-name="arrow-right" link-size="large"> <a href="/centers-programs/division-otolaryngology-ear-nose-and-throat" aria-label="Division of Otolaryngology " > Division of Otolaryngology </a> </chop-cta-link> </li> </ul> </chop-styled-text> </div> <span slot="opened-button-text">View all</span> <span slot="closed-button-text">View all</span> </chop-expandible-link-list> </div> <div class="paragraph paragraph--type--card-callout paragraph--view-mode--default w-full"> <chop-callout-card theme-color="beige" icon-name="conversation" content-align="12" > <div slot="heading"> <chop-heading level-size="lg"> <h3>Cell & gene therapy supportive programs </h3> </chop-heading> </div> <div slot="description"> <chop-styled-text><p>The path from an idea in the lab to a clinical trial and finally to an FDA-approved therapy winds through these support programs. It’s with the expertise of these programs that discoveries are ushered through the complicated but critical steps toward safe and effective treatments. </p></chop-styled-text> </div> <div slot="links"> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/centers-programs/clinical-vivo-gene-therapy">Clinical In Vivo Gene Therapy </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.research.chop.edu/cell-and-gene-therapy-collaborative" target="_blank">Cell & Gene Therapy Collaborative </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/centers-programs/pathology-and-laboratory-medicine/cell-based-therapy-laboratory">Cell Based Therapy Laboratory </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" no-hover="" full-width > <a href="/centers-programs/cell-and-gene-therapy-informatics-team">Cell & Gene Therapy Informatics </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.research.chop.edu/ccmt" target="_blank">Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.research.chop.edu/clinical-vector-core" target="_blank">Clinical Vector Core </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.research.chop.edu/research-vector-core" target="_blank">Research Vector Core </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.research.chop.edu/human-pluripotent-stem-cell-core" target="_blank">Human Pluripotent Stem Cell Core </a> </chop-cta-link> <chop-cta-link icon-name="external" link-variant="link" no-hover="" full-width > <a href="https://www.research.chop.edu/comparative-medicine-services-core" target="_blank">Comparative Medicine Services Core </a> </chop-cta-link> <chop-cta-link icon-name="arrow-right" link-variant="link" 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