A recent CDMO press release essentially asserted superiority via proximity: Geography as God of outsourcing. I contacted two biopharma executives familiar with the Boston/Cambridge scene for reactions: Michael Kaufman, Senior Vice President, CMC, Mersana Therapeutics, and 15-year veteran of Merck; and George Avgerinos, Senior Vice President of Biologics Operations, Fortress Biotechnologies, and 22-year veteran of BASF/Abbott/Abbvie.
Global regulatory standards for clinical trials are, simply put, complex. In this case study, learn how one company worked with its partner to overcome the challenge of a protocol based in the US, an investigational medical product (IMP) approved in the EU, and varying QP (qualified person) rules from country to country, to conduct their clinical trial in Isreal.
Emerging cellular therapies aim to manufacture cell populations for clinical indications, such as cancer, autoimmune, and cardiovascular diseases. As hospitals and translational facilities explore implementation of this technology they concurrently evaluate whether or not to manufacture the therapies onsite. Here is a guideline of how and when to get started.
The coming years will be an exciting era in genetic research as we discover new biological mechanisms and functions for noncoding RNAs. Our improved understanding of this class of genes should provide insight into novel treatments for cancers.
Single use technologies are expected to play a big role in the commercialization of cell and gene therapies because of their cost, speed, and sterility benefits.
Freezing cells without liquid nitrogen
Helpful tips such as carefully choosing the most suitable fixative and permeabilization reagents as well as other factors that should be considered when designing intracellular flow experiments.
The availability of commercial multiplex cytokine assays enables end users to interrogate an entire network of cytokines in a single sample.
Challenges of T cell immunotherapies include the costly manufacturing process relying on lengthy and complex open workflows with high manual labor requirements that influence product variability. This application note describes the details of a robust CAR T cell manufacturing workflow that can be adapted for cGMP compliance in commercial production of CAR T cells.
A demonstration of a simple workflow to verify the reliability of the S3e Cell Sorter in sorting single
cells. Using this method, a confirmed sorting accuracies of >94% and 100% for single cells and single beads is achieved, respectively.
Single use connection technologies are expected to play a critical role in commercial manufacturing of cell and gene therapies. Learn about the types of connection technologies and selection considerations.
In Part 1 of our two-part series with Adaptimmune, CEO James Noble, who has announced his retirement since the publication of this article, provided thoughtful insight on the status of combatting solid tumors as well as the ongoing pricing issue affecting the cell and gene therapy sector. Here in Part 2, along with John Lunger, SVP, Manufacturing and Supply Chain, and Mark Dudley, SVP, Product Development at Adaptimmune, we dig deeper into the topics discussed in Part 1 — we shed granular light on how pricing will affect manufacturing; we talk outsourced versus in-house manufacturing and more.
On the heels of our sister print publication, Life Science Leader’s cover story on the whirlwind rise of Orchard Therapeutics, I had the opportunity to talk to company’s Co-Founder and Chief Scientific Officer, Dr. Bobby Gaspar about how the company has developed innovative gene therapies to treat CNS diseases, such as metachromatic leukodystrophy (MLD), tackle ADA-SCID, and more.
Minovia Therapeutics recently announced dosing of the first patient in a Phase I/II clinical trial of the company’s Mitochondrial Augmentation Therapy (MAT) for the treatment of Pearson syndrome. As stem cell transplantations and cellular therapies continue to gain momentum rapidly, this is an example of trial that could pave the way for the development of treatments for all mitochondrial diseases.
Erin Harris, Editor-In-Chief, Cell & Gene conferred with several SMEs in the cell and gene sector to get their take on the challenges for both industry and patient associated with cell and gene therapy prices, and what can be done, realistically, to bring costs down over time.
James Noble, CEO of Adaptimmune, explains where the industry is with attacking solid tumors and sheds light on the ever-present pricing issue.
Cell & Gene is committed to judiciously developing our best-in-class Editorial Advisory Board (EAB). Cell & Gene’s EAB is diverse and interactive, and members hail from both industry and academia. We’re pleased and honored to announce our newest member of Cell & Gene’s Editorial Advisory Board — Vijay Chiruvolu, SVP of Global Process Development, Cell Therapy at Kite Pharma.
Last month, the FDA released a statement by former FDA Commissioner Scott Gottlieb, M.D., and Biologics Center Director Peter Marks, M.D., Ph.D. on FDA’s continued efforts to stop stem cell clinics and manufacturers from marketing unapproved products that put patients at risk. I caught up with Dr. Aaron Levine, Associate Professor, School of Public Policy, Georgia Institute of Technology to get his take on the statement and how dubious players affect the good of the cell and gene sector.
Just recently, I interviewed Dr. Reni Benjamin, Managing Director, Biotechnology Equity Research at Raymond James & Associates about the panel he moderated at ARM’s 2019 Cell & Gene Therapy Investor Day. In the article, Dr. Benjamin explained how investors are thinking about the cell and gene therapy space in 2019.