{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T22:24:05Z","timestamp":1788560645371,"version":"build-2803163510"},"reference-count":24,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2025,12,5]],"date-time":"2025-12-05T00:00:00Z","timestamp":1764892800000},"content-version":"vor","delay-in-days":4,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clinical Translational Sci"],"published-print":{"date-parts":[[2025,12]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Cell and gene therapies (CGTs) are transforming medicine by offering potential cures for diseases previously considered untreatable. Despite rapid advancements, challenges remain in optimizing efficacy and safety and ensuring patient accessibility and preference due to high costs and clinical uncertainties, particularly for rare diseases and one\u2010time administration. The American Society of Clinical Pharmacology &amp; Therapeutics (ASCPT) held a CGT satellite conference in 2025, titled \u201cCell and Gene Therapy: Transforming Treatment Paradigms for Patient\u2010Centric Care.\u201d This manuscript summarizes the conference, covering gene therapies and T\u2010cell immunotherapies from scientific, clinical, and patient\u2010centered perspectives. Key topics on gene therapy included \u201cplatformization\u201d to streamline development, lessons from adeno\u2010associated virus\u2010based gene therapies for hemophilia from patient and clinical perspectives, clinical pharmacology, and model\u2010informed drug development (MIDD) considerations. The conference also highlighted T\u2010cell immunotherapies including chimeric antigen receptor T therapy (CAR T), focusing on factors affecting cellular kinetics, efficacy, and safety, as well as emerging allogeneic CAR T for autoimmune diseases and MIDD strategies to optimize therapy design and clinical outcomes.<\/jats:p>","DOI":"10.1111\/cts.70430","type":"journal-article","created":{"date-parts":[[2025,12,5]],"date-time":"2025-12-05T08:55:17Z","timestamp":1764924917000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Cell and Gene Therapy: Transforming Treatment Paradigms for Patient\u2010Centric Care"],"prefix":"10.1111","volume":"18","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6659-1585","authenticated-orcid":false,"given":"Sojeong","family":"Yi","sequence":"first","affiliation":[{"name":"Alnylam Pharmaceuticals, Inc  Cambridge Massachusetts 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USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0684-0150","authenticated-orcid":false,"given":"Michael Z.","family":"Liao","sequence":"additional","affiliation":[{"name":"Third Arc Bio, Inc  Lower Gwynedd Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,12,5]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2023.11.001"},{"key":"e_1_2_10_3_1","unstructured":"US Food and Drug Administration Approved Cellular and Gene Therapy Products updated August 15 2025 accessed September 9 2025 https:\/\/www.fda.gov\/vaccines\u2010blood\u2010biologics\/cellular\u2010gene\u2010therapy\u2010products\/approved\u2010cellular\u2010and\u2010gene\u2010therapy\u2010products."},{"key":"e_1_2_10_4_1","unstructured":"American Society of Gene & Cell Therapy and Citeline Gene Cell + RNA Therapy Landscape Report \u2013 Q1 2025 accessed 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