Advanced Therapy Medicinal Products (ATMPs) comprise novel cell, tissue and gene therapies and offer the potential of durable remissions for diseases where there is a high unmet clinical need. Once considered a niche area of academic research, ATMPs now represent one of the fastest-growing areas of clinical development. The field has seen a rapid expansion of academic and commercial entities successfully translating ATMP research into the clinic. This is reflected in projection that the global gene and cell therapy market will be worth US $11.96 billion by 2025. However, these treatments are complex to deliver and frequently do not fit naturally into established healthcare systems. In the United Kingdom (UK) there has been a long-standing interest in ATMP research and, in order to meet the ambition to act as an international hub of activity for delivery of ATMPs, a collaborative network of Advanced Therapy Treatment Centres (ATTCs) has been established. This review explores the challenges of delivery in the clinical setting, focussing on one form of ATMP, Adoptive Cell Therapy (ACT). We describe the strategy being implemented in the UK to optimise the roll-out of these exciting new therapies.
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http://dx.doi.org/10.1177/2515135520944355 | DOI Listing |
Clin Cancer Res
January 2025
Bristol-Myers Squibb (United States), Summit, New Jersey, United States.
Purpose: Orvacabtagene autoleucel (orva-cel; JCARH125), a CAR T-cell therapy targeting B-cell maturation antigen (BCMA), was evaluated in relapsed/refractory multiple myeloma (RRMM) patients in the EVOLVE phase 1/2 study (NCT03430011). We applied a modified piecewise model to characterize orva-cel transgene kinetics and assessed the impact of various covariates on its pharmacokinetics (PK).
Experimental Design: The population PK analysis included 159 patients from the EVOLVE study.
Invest Ophthalmol Vis Sci
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Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom.
Purpose: This study aimed to evaluate early-phase safety of subretinal application of AAVanc80.CAG.USH1Ca1 (OT_USH_101) in wild-type (WT) pigs, examining the effects of a vehicle control, low dose, and high dose.
View Article and Find Full Text PDFComput Methods Biomech Biomed Engin
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Department of Gastroenterolgy, The Second Affiliated Hospital of Chengdu Medical College, China National Nuclear Corporation 416 Hospital, Chengdu, China.
The global rise in Crohn's Disease (CD) incidence has intensified diagnostic challenges. This study identified circadian rhythm-related biomarkers for CD using datasets from the GEO database. Differentially expressed genes underwent Weighted Gene Co-Expression Network Analysis, with 49 hub genes intersected from GeneCards data.
View Article and Find Full Text PDFStem Cells Dev
January 2025
Department of Clinical Pharmacy and Pharmacy Practices, Faculty of Pharmacy, University Malaya, Kuala Lumpur, Malaysia.
Hypertension, commonly known as high blood pressure, is a significant health issue that increases the risk of cardiovascular diseases, stroke, and renal failure. This condition broadly encompasses both primary and secondary forms. Despite extensive research, the underlying mechanisms of systemic arterial hypertension-particularly primary hypertension, which has no identifiable cause and is affected by genetic and lifestyle agents-remain complex and not fully understood.
View Article and Find Full Text PDFStem Cell Rev Rep
January 2025
Institute for Cellular and Molecular Medicine, Department of Immunology, SAMRC Extramural Unit for Stem Cell Research and Therapy, University of Pretoria, Pretoria, 0084, South Africa.
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