Summary The Human Pluripotent Stem Cell Registry established a database of clinical
studies using human pluripotent stem cells (PSCs) as starting material for cell therapies …

Lysosomal storage disorders are a group of progressive multisystemic hereditary diseases
with a combined incidence of 1: 4,800. Here we review the clinical and molecular …

This paper presents the results of a qualitative study based on semi-structured interviews of
10 expert patient advocates on several different issues around Advanced Therapy Medicinal …

Background aims The advanced therapy product tisagenlecleucel is a CD19-directed
genetically modified autologous T-cell immunotherapy that has brought hope for children …

ABSTRACT Introduction Artificial Intelligence (AI) is becoming increasingly utilized in the
medical device industry as it can address unmet demands in clinical sites and provide more …

Manufacturing of a tissue-engineered product (TEP) is an essential part of the road from “lab
to patient,” with its own set of challenges which are different from those covered in other …

Introduction The marketing authorization of Advanced Therapy Medicinal Products (ATMPs)
in Brazil is recent. The features of these therapies impose specialized regulatory action and …

Gene therapies (GTs) have recently emerged as revolutionary personalized therapeutic
options. Despite their promising potential, challenges such as uncertainty regarding long …

Background aims Advanced therapy medicinal products (ATMPs) have reached the forefront
of biotechnological innovation, partly due to public funders' efforts in the early stages of …

Advanced Therapies are a class of innovative complex biological products used for
therapeutic purposes, encompassing cell therapy, tissue engineering, and gene therapy …