首页> Therapeutic innovation & regulatory science> Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases-by DahShu Innovative Design Scientific Working Group (IDSWG).

Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases-by DahShu Innovative Design Scientific Working Group (IDSWG).

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第一作者： Chenkun,Wang

第一单位： Vertex Pharmaceuticals, Inc., 50 Northern Avenue, Boston, MA, 02210, USA. Chenkun_Wang@vrtx.com.

作者： Chenkun,Wang ^[1] ; Junrui,Di ^[2] ; Mercedeh,Ghadessi ^[3] ; Rui,Tang ^[4] ; Caroline,Mulatya ^[5] ; Daoyuan,Shi ^[6] ; Tu,Xu ^[7] ; Wenquan,Wang ^[8] ; Chaoqun,Mei ^[9] ; Susan,Wang ^[10] ; Bryan,McComb ^[11] ; Robert A,Beckman ^[12] ; Gianna,McMillan ^[13]

作者单位： Vertex Pharmaceuticals, Inc., 50 Northern Avenue, Boston, MA, 02210, USA. Chenkun_Wang@vrtx.com. ^[1] Dexcom Inc., San Diego, CA, 02139, USA. ^[2] Bayer U.S. LLC, Pharmaceuticals, 100 Bayer Boulevard, Whippany, NJ, 07981, USA. ^[3] Astellas Pharma Inc, 441 Morgan Ave, Cambridge, MA, 02139, USA. ^[4] REGENXBIO Inc., 9804 Medical Center Dr, Rockville, MD, 20850, USA. ^[5] BlueRock Therapeutics, 238 Main St, Cambridge, MA, 02142, USA. ^[6] Novo Nordisk, 75 Hayden Ave, Lexington, MA, 02421, USA. ^[7] Pfizer Inc., Collegeville, PA, 19426, USA. ^[8] Bristol Myers Squibb, Madison, NJ, 07940, USA. ^[9] Boehringer Ingelheim Pharmaceuticals Inc., 900 Ridgebury Road, Ridgefield, CT, 06877, USA. ^[10] Pfizer Inc., 66 Hudson Blvd East, New York, NY, 10001, USA. ^[11] Lombardi Comprehensive Cancer Center and Innovation Center for Biomedical Informatics, Georgetown University Medical Center, Washington, DC, 20007, USA. ^[12] Bioethics Institute at Loyola Marymount University, 1 LMU Drive, Los Angeles, CA, 90045, USA. ^[13]

关键词 Cell and gene therapy Clinical development Regulatory requirements and guidelines Safety monitoring

医学主题词少见病(Rare Diseases);人类(Humans);临床试验(主题)(Clinical Trials as Topic);美国(United States);美国食品和药物管理局(United States Food and Drug Administration)

DOI 10.1007/s43441-025-00853-4

PMID 40779197

发布时间 2025-11-02

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准确一般字义偏差语意偏离译文作用小提交