‘What convinced investors, and what convinced our alliance partners were the clinical result we had in chronic lymphatic leukaemia, I think that’s important. I think with animal models, they are very helpful in a sense, in just the bare bones test system, but to prove efficacy, in the end is to run clinical trials.’ Jan Joseph Melenhorst, Director of Product Development & Correlative Sciences Laboratory from the University of Pennsylvania, discusses the necessary steps that must be taken for their cell therapy to become a viable treatment option.
Within his department at the university, Melenhorst examines the post infusion specimens of cells to better understand the potency and toxicity of the cells they infuse into patients. To enhance the potency of cells, additional gene editing tools can be included to modify the genome of the T-Cells and further enhance the expression of the targeting gene. Melenhorst speaks of his own experience with the CRISPR-Cas9 gene editing tool, but also draws upon the use of TALENs, stressing its importance to ‘the future of autologous and allogeneic cell therapies’. It is the autologous therapies Melenhorst believes are the current future of CAR-T therapy. Continue reading “Proving the efficacy of cell therapy treatments to convince investors”
‘Start working on raw materials early on’ – for Dieter Hauwaerts, Vice President of Operations at Celyad, the way to combat one of the biggest challenges in cell therapy manufacture is clear.
Limited availability of raw materials is a major issue for large scale manufacture of new therapies, but planning well in advance can help mitigate it: ‘In process development, take into account the quantity and quality of raw materials you will need in a clinical stage or commercial manufacturing process and build good relationships with suppliers.’
However, even with the necessary raw materials, Hauwaerts still sees issues with ‘the way we can manufacture products in a reliable and consistent way’, though he hopes that in 2017 automation will begin to negate this.
Watch the full exclusive interview with Hauwaerts – filmed at Cell Therapy Manufacturing & Gene Therapy Congress 2016 – above or here.
An Interview with Denis Bedoret of MaSTherCell
We recently sat down with Denis Bedoret, Chief BD Officier at MaSTherCell in Amsterdam at the Cell Therapy Manufacturing & Gene Therapy Congress conference to discuss the critical issues and opportunities present in the cell & gene therapy industry today.
Continue reading “Affordable Breakthrough Therapies”
By Manuel J. T. Carrondo, Prof. Chem & Biochem Eng., FCT/UNL & Vice-president, iBET
Since the early nineties iBET has been involved in production and purification of viruses for gene therapy. Early on, enveloped retroviruses and non-enveloped adenoviruses where the targets; from late nineties onward lentivirus and baculovirus were added to the portfolio of enveloped viruses and AAV to the non enveloped viruses.
Although originally meant for monogenetic diseases, now some are also produced for cancer treatment (ex. oncolytic adenoviruses made in A547 cells, so replicative) or as reagents for cell therapies (also known as ex-vivo gene therapies).
Having developed scaled down tests and analyticals (including surface plasmom resonance, dynamic light scattering) coupled with its chemical engineering model competencies, iBET has designed membrane or media materials for which our key partners MERCK Millipore, SARTORIUS, GE HealthCare have developed the prototypes tested on our biologies and equipments. In this way, improved DSP processes have been created increasing viral yields and infective to total particle ratios or yields and viability for cell therapies.
Continue reading “Novel Strategies for Gene and Cell Therapies”
David Brindley, Senior Research Fellow at University of Oxford Department of Pediatrics comes from a very interesting background that covers both academia and industry. He joined us during Biotech Week Boston to discuss the relationship between academia and industry, what he identifies as the key regulatory challenges in both the US & Europe, and then finally he explores the recent growth of biotech clusters around the globe.
By design, there are a number of initiatives around that globe to create biotech clusters. “The two greatest biotech clusters in the world are undoubtedly the 128 corridor around Boston and Silicon Valley on the West Coast” Brindley says. However, he does mention that these clusters were development completely by accident, and not by design. Moving forward, as the growing of biotech cluster initiatives progress, these regional hubs need to focus/specialize on a very deep niche. If you are looking to set up a new cluster, Brindley says, “you should worry less about the platform technology it will develop, is it monoclonal or is it small molecule. And [instead], focus more on ‘what is the bioprocessing problem that we are going to address here? Is this going to be a center of manufacturing?’ because I think even saying we are going to be a center of drug development is too broad.” This train of thought shows that collaboration is necessary for large-scale success of the industry.
Continue reading “The Recent Growth of Biotech Clusters Around the Globe”
KNect365 is pleased to introduce a special Digital Week program, comprised of a week-long series of free webinars with live Q&A – connecting cell and gene therapy leaders throughout the year from the comfort of your desk.
Cell Therapy Manufacturing & Gene Therapy Digital Week connects cell and gene therapy leaders to drive manufacturing and commercialisation through direct access to innovative discovery, product development, and regulatory know-how.
Register now to watch free educational sessions presented by leading industry experts, get answers to your toughest questions, network with colleagues and partners, and download useful resources.
Continue reading “Cell Therapy Manufacturing and Gene Therapy Digital Week”
This article was originally published on www.CellCultureDish.com
By Brandy Sargent, Editor, Cell Culture Dish
Flexibility in cell culture manufacturing via a reduction in process duration can be a key strategy for maximizing facility utilization and facilitating the production of multiple therapeutics from a facility. A key bottleneck is the seed train, which can add weeks to the timeline of the production culture. Seeding production bioreactors with a direct, cryopreserved CHO cell inoculum could possibly eliminate the need for a lengthy, continuous seed train and provide other numerous benefits.
At this year’s Boston Biotech Week, two feasibility studies were presented that showed the use of a Frozen Seed Bag (FSB) for direct cell inoculum into a production bioreactor. Shahid Rameez of KBI Biopharma and Nikhil Ramsubramaniam of Merck presented separate studies demonstrating the use of frozen inoculum in fed-batch production bioreactors (KBI) or perfusion bioreactors (Merck).
KBI described a current state-of-the-art production process consisting of scale up from spinner flasks, through a bag bioreactor into stirred tank bioreactors – a 5-week process (see Figure 1). According to the study results presented, implementation of a direct frozen inoculum approach could potentially reduce a 5-week timeline to as little as 1.5 – 2 weeks. Such an improvement could vastly increase the productivity of current cell culture manufacturing facilities.
Continue reading “Direct Inoculum of Bioreactors with CHO Cells from Frozen Seed Bags to Eliminate Continual Seed Trains and Improve Facility Utilization”