The questions for “make vs. buy" raised by Bristol-Myers Squibb’s Christopher Sinko, SVP and Head of Product Development, are news because of this: Sinko reaffirms for us they remain cemented within the otherwise changing dynamics impacting drug development and manufacturing outsourcing decisions.
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.
With our team of packaging experts and extensive cold chain capabilities, this case study discusses the creation a custom packaging and distribution solution to meet this client’s unique needs. Through our partnership, the product was successfully packaged in sufficient quantity and on time for the Phase I trial.
Polysorbate-80 (PS-80) is a surfactant commonly used as an excipient to improve API stability. However, degradation of PS-80 can lead to decreased drug potency or stability, and there is inconsistency in the quality and composition of PS-80 provided by different vendors.
Current state-of-the-art continuous manufacturing technologies are being developed and implemented to manufacture a wide variety of products including monoclonal antibodies, recombinant proteins, and other biological modalities. Though upstream fed batch and perfusion bioreactors unit processes are relatively mature, downstream process unit operations are less mature. In this case study, Catalent compared the productivity of purifications running in batch versus continuous mode.
Studying biology at a single-cell level can provide crucial insights into how individual cells contribute to human biology and disease. Here we demonstrate that the resolution of single-cell RNA sequencing (RNA-Seq) can be greatly enhanced by adding a cell sorting step prior to RNA-Seq analysis.
Cryopreservation of small sample volumes is common in many scientific applications. However, there is increasing demand for large-volume cryopreservation, especially in cell and gene therapies, where treatments require large cell numbers. This study demonstrates an optimized cooling protocol for cryopreserving large-volume cryovials in a VIA Freeze Quad controlled-rate freezer, avoiding the need for liquid nitrogen.
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.
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.
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.
The logistics of decoupling drug substance manufacturing from final drug product formulation is now possible using integrated single-use freeze-thaw assemblies which incorporate reliable components.
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.
The ASGCT 22nd Annual Meeting will begin on the morning of Monday, April 29, 2019 and continue through 12:15 PM on Thursday, May 2, 2019. Exhibits will be open Monday, April 29 through Wednesday, May 1.
ARM’s 2019 Cell & Gene Therapy Investor Day featured expert-led panels to help the audience wrap their minds around the investment outlook for the cell and gene therapy space. One such panel, aptly titled, “The Investment Outlook for the Cell and Gene Space,” was comprised of investors from DEFTA Partners, Roivant Sciences, Aisling Capital, and Aquilo Capital Management. Dr. Reni Benjamin, Managing Director, Biotechnology Equity Research at Raymond James & Associates moderated the panel.
ARM’s 2019 Cell and Gene Investor Day took place on March 21st in New York, NY. Now in its seventh year, Investor Day provides insight into financing opportunities for cell and gene therapy-based treatment and tools. I caught up with ARM’s CEO and Cell & Gene Editorial Advisory Board member, Janet Lambert, after the event to discuss top takeaways, near-term financing opportunities, and more.
Partnering with a CDMO that marries innovation with technical, regulatory, and manufacturing experience can be cell therapy and gene therapy companies’ best opportunity for scalability and yet their biggest hurdle. From process development through commercial supply and all the steps in between, cell and gene therapy companies expect CDMOs to meet an understandably high bar.