This comprehensive guide explores the essential tools of quantitative PCR (qPCR) and digital droplet PCR (ddPCR) in the biopharmaceutical industry, offering invaluable insights into their applications, strengths, and limitations. Download now to stay ahead of the curve in pharmaceutical research and development...
The guidance provides recommendations regarding information that should be provided in an investigational new drug (IND) application to assess the safety and quality of the investigational GE product, including information on product design, product manufacturing and testing, nonclinical safety...
In this draft guidance, the FDA emphasizes that potency assays and their corresponding acceptance criteria should be designed to make meaningful contributions to potency assurance by reducing risks to product potency. They provide illustrative examples of approaches to potency assay development that are...
The mRNA COVID-19 vaccines approved and authorized today are supported by the FDA’s evaluation of manufacturing data to support the change to the 2023-2024 formula and non-clinical immune response data on the updated formulations including the XBB.1.5 component...
Specifically, the guidance recommends conducting analytical comparability studies to provide scientific evidence of the impact that manufacturing changes may have on the safety, potency, and purity of human cellular and gene therapy products...
The goal of the hypoimmune platform is to overcome the immunologic rejection of allogeneic cells, which if true for SC291 may result in longer CAR T cell persistence and a higher rate of durable complete responses for patients with B-cell lymphomas or leukemias. The hypoimmune platform includes disruption of major...
In the study, published in Nature Biomedical Engineering, researchers from MD Anderson Cancer Center use EV-encapsulated mRNA to initiate and sustain collagen production for several months in the cells of photoaged skin in laboratory models. It is the first therapy to demonstrate this ability and represents a proof-of-concept for deploying the EV mRNA therapy...
ASGCT held its fifth annual liaison meeting with FDA CBER’s Office of Tissues and Advanced Therapies (OTAT) on Nov. 14, 2022. A group of Society leaders and members gave two presentations to FDA on significant topics in the field, followed by a presentation from FDA. Dr. Keith Wonnacott, ASGCT’s Regulatory Affairs Committee Chair, chaired the meeting and moderated discussion...
On March 15th, 2022 the FDA is announcing the availability of two draft guidance documents: “Human Gene Therapy Products Incorporating Human Genome Editing,” and “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products.” The draft guidance, “Human Gene Therapy Products Incorporating Human Genome Editing,” is intended to provide recommendations to sponsors [...]
