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  • Perspective
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Enhancing pediatric access to cell and gene therapies

Nature Medicine volume 30, pages 1836–1846 (2024)Cite this article

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Abstract

Increasing numbers of cell and gene therapies (CGTs) are emerging to treat and cure pediatric diseases. However, small market sizes limit the potential return on investment within the traditional biopharmaceutical drug development model, leading to a market failure. In this Perspective, we discuss major factors contributing to this failure, including high manufacturing costs, regulatory challenges, and licensing practices that do not incorporate pediatric development milestones, as well as potential solutions. We propose the creation of a new entity, the Pediatric Advanced Medicines Biotech, to lead late-stage development and commercialize pediatric CGTs outside the traditional biopharmaceutical model in the United States—where organized efforts to solve this problem have been lacking. The Pediatric Advanced Medicines Biotech would partner with the academic ecosystem, manufacture products in academic good manufacturing practice facilities and work closely with regulatory bodies, to ferry CGTs across the drug development ‘valley of death’ and, ultimately, increase access to lifesaving treatments for children in need.

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Fig. 1: Licensing models for pediatric CGTs.
Fig. 2: Life cycle of a pediatric CGT within the PAMB.

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Acknowledgements

On 6 June 2023, approximately 80 CGT investigators representing more than 20 academic medical centers from the United States, Europe and Israel, alongside patient advocates, public officials representing the US FDA, the Advanced Research Projects Agency for Health and the California Institute for Regenerative Medicine and representatives from the private sector and non-profit foundations met in Washington, DC, for a think tank focused on the market failures we are witnessing for CGTs for pediatric diseases. The views discussed here germinated at this meeting54.

Author information

Authors and Affiliations

  1. Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA

    Crystal L. Mackall, Casey Carr & Elena Sotillo

  2. Department of Pediatrics, Division of Pediatric Hematology, Oncology, Stem Cell Transplant and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA

    Crystal L. Mackall

  3. Department of Medicine, Division of Bone Marrow Transplant and Cell Therapy, Stanford University School of Medicine, Stanford, CA, USA

    Crystal L. Mackall

  4. Center for Cancer and Immunology Research and Department of Pediatrics, Children’s National Hospital and The George Washington University, Washington, DC, USA

    Catherine M. Bollard

  5. KidsVCancer, Washington, DC, USA

    Nancy Goodman

  6. St. Jude Children’s Research Hospital, Memphis, TN, USA

    Rebecca Gardner & Julie Park

  7. Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital and Texas Children’s Hospital, Houston, TX, USA

    Rayne Rouce

  8. Exthymic, San Diego, CA, USA

    Rich Stoner

  9. Innovative Genomics Institute, University of California at Berkeley, Berkeley, CA, USA

    Fyodor D. Urnov

  10. Children’s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA

    Alan S. Wayne

  11. Departments of Microbiology, Immunology & Molecular Genetics; Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA

    Donald B. Kohn

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Corresponding author

Correspondence to Crystal L. Mackall.

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Competing interests

C.L.M. holds multiple patents in the arena of CAR T cell therapeutics; receives royalties from CARGO through the US National Institutes of Health and Stanford; holds equity in CARGO Therapeutics, Link Cell Therapies, Ensoma and GBM NewCo, which are developing CAR-based therapies; consults for CARGO, Link, Immatics, Ensoma and GBM NewCo; and receives research funding from Lyell Immunopharma and Tune Therapeutics. C.M.B. has filed patents in the arena of cell therapies, including CAR-modified cell therapies; is a scientific cofounder of Mana Therapeutics and Catamaran Bio; is on the Board of Directors of Cabaletta Bio; and holds stock in Repertoire Immune Medicine and Neximmune, all of which are developing cell therapies (including CAR-based therapies) for cancer or immune-mediated disorders. In addition, C.M.B. serves on the drug safety monitoring board for SOBI and on the scientific advisory board of Minovia TX. C.M.B has also served as president of FACT since 2021. N.G. is a cofounder and holds equity in CARGO Therapeutics. R.A.G. has patents related to CAR therapy and receives royalty payments related to patents from Juno Therapeutics. R.H.R. has received consulting fees from Pfizer and honoraria from Novartis. R.M.S. holds multiple patents in the area of CRISPR bioinformatics and biomanufacturing technologies; is the founder of Exthymic, a cell manufacturing instrument company; sits on the board of Indee Labs; and holds equity in Exthymic, Resilience (a Contract Development and Manufacturing Organization) and Synthego (RNA reagent manufacturer). F.D.U. is a scientific cofounder of Tune Therapeutics; holds equity in, and is a compensated advisor to, Tune Therapeutics and Cimeio Therapeutics; is a paid advisor to Ionis Pharmaceuticals; is a paid consultant to Vertex Pharmaceuticals on the exa-cel program; and receives salary support from the Danaher Corporation. A.S.W. receives institutional research funding (Children’s Hospital Los Angeles) from Kite, A Gilead Company. In addition, A.S.W. has served on advisory boards for Wugen, Kite and Institut de Recherches Internationales Servier, all for cell therapy product development. D.B.K. is an inventor for the University of California Regents for a lentiviral vector to treat ADA-SCID.

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Mackall, C.L., Bollard, C.M., Goodman, N. et al. Enhancing pediatric access to cell and gene therapies. Nat Med 30, 1836–1846 (2024). https://doi.org/10.1038/s41591-024-03035-1

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