7.1.1 Advanced therapies, in general, are complex products. The Wall Street Journal reports that, on average, 557 clinical holds were issued each year from 2012 through 2017, increasing to 664 from 2017 to 2021. In 2022 (through mid-December), 747 holds had been placed by the FDA.[156]
Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.
bluebird bio: elivaldogene autotemcel
7.2.1 In 2021, a hold was placed on bluebird bio’s trial for the gene-modified cell therapy elivaldogene autotemcel (eli-cel, Lenti-D) for cerebral adrenoleukodystrophy (CALD) after a study participant developed a bone marrow disorder that can lead to leukemia, likely due to Lenti-D LVV insertion. The hold was ultimately removed, and the drug received FDA approval under the brand name Skysonain September 2022.
Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.
7.3.1 The science of cell and gene therapies has outpaced regulatory development, leading to a regulatory framework that can, in some cases, impede effective translation from R&D to the clinic and commercialization.[181]
Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.
7.4.1 In addition to the need for more communication between sponsors and regulatory authorities, there is a general recognition that standardization of global regulations covering cell and gene therapies is needed. Regulations in different regions have some similarities but also many differences, even when it comes to core GMP requirements, which create real challenges for cell and gene therapy developers and manufacturers, particularly when the requirements conflict.[194]
Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.Nice Insight provides customers with the latest trends in the market, hard-to-find pricing, and analysis of important scientific innovation and challenges. Our research reports help innovators understand the price they'll pay for a contractor and what challenges they should anticipate, and allow CDMOs to evaluate their position in the market.