This article explores how ADOA (Autosomal Dominant Optic Atrophy) occurs, and why it has become a promising target for new approaches to treatment.
- The “Renaissance” Approach To Cell & Gene Therapy Manufacturing
- How To Leverage RWE To Facilitate Access To Cell & Gene Therapies
- How To Test Master Cell Banks As A Starting Material For Gene Therapies
- Natural Killer Cells: Promise For Cancer Immunotherapy
- Weathering The Biotech Storm: Accelerating Manufacturing, Controlling Cost, And Managing Risk With A Strategic CDMO Relationship
- ARW’s C&G (+RNA!) Manufacturing Must-Reads (Dracula Edition!)
- Addressing The “Achilles Heel” Of Gene Therapy Manufacturing
- Making An Unapprovable Drug Product Approvable: Lessons Learned
CELL & GENE ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
Amplification-Free Analysis Of Lentiviral Vector Genome Integrity
We review an amplification-free workflow has been developed for the genome integrity analysis of lentiviral vectors (LV) using capillary electrophoresis with laser induced fluorescence detection (CE-LIF).
Upstream Platform For Continuous Lentiviral Vector Production
Here, we demonstrate a high-yield LV production process, using animal-derived component-free media and reagents, where multiple or continuous vector harvests could be obtained from a single batch.
Patient-Centric Approach And FDA Expertise Lead To Orphan Approval
An international company developing an orphan drug product needed a partner experienced in FDA interactions to help it navigate the U.S. regulatory process from beginning formulation to NDA approval. Find out how this was done in the available case study.
A Platform Standard For Viral Vector Manufacturing And Commercialization
Learn more about platform technologies that are being developed and implemented to support large-scale production of viral vectors.
Superior Biopharmaceutical Manufacturing Facilities Prioritize Cleaning & Disinfection During Design
Thorough consideration of cleaning/disinfection during facility design eases maintenance across the facility lifecycle, optimizes workspace utility, and contributes to efficient facility operation.
First Clinical Trial Using Stem Cell Therapy To Treat Septic Shock
An Ottawa Hospital Research Institute’s Cell Manufacturing Facility successfully conducted a Phase I clinical trial examining the safety and efficacy of allogeneic bone marrow-derived MSCs as a treatment for patients experiencing septic shock. Before initiating clinical Phase II studies, the protocol for generating large quantities of MSCs had to be established to reduce end-product variability, minimize production costs, and to ensure sufficient yield to treat multiple patients. Maximizing the yield of viable cells proved challenging in a limited footprint.
Choose The Right FDA Program To Expedite Your Rare Oncology Trial
With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Faced with limited treatment options, researchers, clinicians, and patients may be seeking approaches to accelerate the development and approval of novel therapies. In this blog post, we review the regulatory programs available to expedite treatments for rare disorders and serious diseases.
Successfully Achieve High Numbers Of Peptide And Protein Identifications
Collecting quality MS/MS at high acquisition rates is key to achieving high numbers of peptide and protein identifications. This study investigates the impact of large sensitivity gain on identification rates.
Fundamentals Of Size Exclusion Chromatography
SEC, also known as gel filtration, can be used for preparative and analytical studies of your protein. But how do you know which parameters give you the best resolution of your protein mixture?
Bioprocessing And Scaleup Of HEK293 Cells For AAV Production
MilliporeSigma has developed the VirusExpress®️ AAV Production Platform as a scalable upstream AAV production solution for viral gene therapies.
CELL AND GENE LIVE
The CGT market is expected to be worth $5.5 Billion by 2026. Yet, the sector struggles with recruiting and retaining enough experienced candidates needed to bring life-saving treatments to market. Why is that, and what can you do now to overcome the talent crunch? Check out our latest Cell & Gene Live - now available On-Demand.Cell & Gene Live On-Demand
CELL AND GENE CONTENT COLLECTIONS
While both allogeneic and autologous therapies use similar technologies common to the growth of cells, the scale is different. In this e-book from Cell & Gene, we've curated insightful editorial that concentrates on the advancements of both allogeneic and autologous therapies and how to manufacture them at scale.More Content Collections