The FDA has finalized six draft guidance documents intended to serve as part of a modern, comprehensive framework for how the agency will help advance the field of gene therapy. At the same time, it issued a new draft guidance on determining “sameness” of gene therapy products for the purposes of orphan drug designation/exclusivity. This article highlights key changes made in each of the final guidances and also reviews the new draft guidance.
- Cell & Gene Therapy Deals: Emerging Trends To Watch
- Cell & Gene’s 2020 Outlook: Stem Cell Research
- Reflections On An Unprecedented Decade In The Cell And Gene Therapy Field
- How Cell And Gene Therapy Is Transforming Healthcare
- Cell And Gene Therapy Foresight In 2020: 7 Trends To Watch
- A Harvard Assist For Cell And Gene Bottlenecks
- Straight Talk From BeiGene On Quality, Compliance, And Outsourcing
- The Role Of Technology In Improving The Reputation Of The Life Science Industry
CELL & GENE ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
Large Volume Cryopreservation: A Liquid Nitrogen Free Proof-Of-Concept
This study demonstrates a cell freezing protocol that uses the LN2-free VIA Freeze Quad controlled-rate freezer for the cryopreservation of up to eight cryobags and up to 560 mL total volume.
Guidelines For Phasing Variants Using Bio-Rad Droplet Digital System
We describe a Droplet Digital PCR-based method, Drop-Phase, which allows users to quickly and accurately phase genomic variants. We successfully phased pairs of CFTR variants and show the methodology can measure linked species that are up to 210 kb apart. This method can be used to phase variants discovered through sequencing or to screen unsequenced individuals for a particular haplotype of interest for clinical trials.
Industrializing Autologous Adoptive Immunotherapies: Manufacturing Advances And Challenges
Cell therapy has proven to be a burgeoning field of investigation, evidenced by hundreds of clinical trials being conducted worldwide across a variety of cell types and indications. There is now a more concerted effort among manufacturers to utilize traditional bioprocess principles to close, automate, and control these processes to ensure critical quality attributes (CQA) of the cell product are consistently maintained and manufacturing processes are cost-effective and risk-mitigated. This focused review will look at current solutions across a typical autologous or patient-matched manufacturing workflow and highlight remaining challenges toward industrialization of these processes.
Single-Use Systems For Shipping Frozen Drug Materials – Every Component Counts
Integrated single-use freeze-thaw assemblies with reliable component parts, such as the connectors supplied by CPC, have become the primary implementation choice for achieving successful drug substance transfers between manufacturing facilities.
How To Speed Up Cell And Gene Therapy Treatments For Patients, Reduce The Number Of Manual Operations And Eliminate Operator Errors
This white paper addresses the unique production problems of cell and gene therapy and explains how a manufacturing execution system (MES) can address them. It provides guidance on how to select an MES and a case study of two CAR-T therapy companies scaling up with and without an MES.
Is A Rapid Cooling Step Needed When Freezing Cells For Cryopreservation?
Successful cell cryopreservation requires a controlled cooling rate. Cooling too rapidly or too slowly will lead to a poorer outcome. This study shows comparable results when freezing cells with or without a rapid-cooling nucleation step.
Complete Response: Lymphoma Trial A Success For Five Patients
A trans-Atlantic study to evaluate an antibody for treatment of B-cell non-Hodgkins lymphoma overcame patient recruiting challenges and has already succeeded beyond expectations in the form of five patients declared disease-free a year and counting while still three years from completing patient follow-up.
Transforming Cancer Research: Digital PCR Technology Plays Integral Role At Cancer Research Center
How digital PCR technology is helping scientists in their quest to break through the current limits of nucleic acid detection and quantification.
Purification Of Antibodies With Hydrophobic Anion Exchange Resin
A hydrophobic anion exchange mixed-mode resin has been developed to overcome challenges in antibody purification. This application note describes the purification of acidic and basic antibodies using this resin.
R-Loops And Their Key Role In Cancer Research
Understanding the conflicting nature of R loops and the challenges of balancing them to result in a positive effect.
CELL AND GENE CONTENT COLLECTIONS
The cell and gene therapy sector’s focus on outsourcing is a major area of on-going concern due to the shortage of suitable manufacturing facilities and qualified outsourcing staff. This collection of original articles from Cell & Gene weighs the economic benefits of outsourcing (i.e. productivity, efficiency, time-to-market, and quality gains) as well as best practices for selecting an outsourcing partner.
More Content Collections
CELL & GENE INDUSTRY EVENTS
Clinical Quality Management Systems (QMS): Establishing A Practical & Compliant Program
March 4 - 4, 2020
1pm-2:30pm EST, Online Training
Responding to Clinical Observations: Developing Robust Audit & Inspection CAPAs
March 10 - 10, 2020
Online Training; 1:00 - 2:30 PM EST US
Lunch And Learn Event For Cell And Gene Therapy Leaders!
March 10, 2020
Cleanroom Microbiology – A Foundational Introduction
March 11 - 11, 2020
1pm-2:30pm EST, Online Training
Regulatory GCP Inspections: Preparation, Participation & Follow-up
March 12 - 12, 2020
1pm-2:30pm EDT, Online Training