06.23.22 -- How to Use RWE for Access to CGTs

The success of using viral vectors to deliver a molecular payload into a cell or replace defective genes with functional ones is an inflection point in the future of modern medicine.

Examine how RMM differs from traditional microbiologic methods that rely on lengthy incubation periods and understand why there is a shift favoring RMM for pharmaceutical manufacturing.

A comprehensive gap analysis examines documented procedures, SOPs, and QMS processes, but also includes discussions with key personnel and scrutinizes outputs derived from those SOPs and processes. Learn about the “report card” — an independent key deliverable that helps the organization identify, prioritize, and remediate potentially problematic areas.

Frost & Sullivan's Virtual Think Tank brought together leading minds to discuss key challenges and other insights related to reducing the cost of goods (COGs) for gene therapy.

Packaging is critical as biotechnology products become more valuable and require colder storage.

Explore the latest strategies and technologies to develop a comprehensive, optimal approach to assay development and product characterization.

Look at the elements involved in building a robust packaging strategy and the key technical considerations in packaging design and operational planning for products of various sizes.

We demonstrate that multiple AAV samples with different serotypes or different genome sizes can be run simultaneously on the multi-capillary system to accelerate the execution of sensitive AAV genome integrity analysis.

Learn more about platform technologies that are being developed and implemented to support large-scale production of viral vectors.

Understand how to manage impurities in drug development with swift isolation, synthesis, and analysis. A client discovered an impurity during ongoing stability studies that exceeded ICH guidelines.

Like the ancient purple bacteria and archaebacteria, multiple scientific, medical, and biotech disciplines might need to engage in a little bit of “symbiosis.” The benefits of kickstarting this endeavor will surely be energizing, not merely for regenerative medicine but, most of all, for patients in need.

Krabbe disease is a devastating neurodegenerative disease inherited in an autosomal recessive manner. Learn more about this disease and FBX-101, which utilizes adeno-associated viral (AAV) gene therapy.

Cell and gene therapies (CGT) are bringing new hope to patients experiencing rare and serious diseases. And they are disrupting the biopharma market in new and challenging ways.

Explore how the 505(b)(2) regulatory pathway can help drug developers gain approval for new drugs in a fraction of the time and cost required by traditional paths.

In this paper, delve into how eConsent is changing the informed consent process for clinical trial sponsors and participants.

In this article, we review how particulate removal through filtration effects column life and how to handle difficult to filter samples.