Upstream bioprocessing at large and commercial scales is projected to be the fastest growing segment of the single-use equipment market as products now in development using SUS move up to commercial manufacturing. In addition, as cellular and gene therapies emerge, we will likely see SUS technologies created and adapted explicitly for these personalized applications.
James Noble, CEO of Adaptimmune, explains where the industry is with attacking solid tumors and sheds light on the ever-present pricing issue.
In recent years, cell and gene therapies have been generating highly promising results in clinical studies, advancing them toward the market. However, the small number of products launched to date have not been proven commercial successes, with a number of advanced therapies being withdrawn from the EU market and limited sales of CAR-T therapies in the U.S.
We’ve been thrust from ruminating around the worth of a generic “human life” towards contemplating starker terms of specific two-year-old children. This thanks to Novartis’ gene-therapy Zolgensma, priced at $2.125 million. However, is this our hoped-for future of gene and cell therapies? The irony of life-saving drugs that financially cancel their celebration at the point of FDA approval?
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.
Cell and gene therapy offers extended-term relief from disease states but comes at high cost with a complex reimbursement model. What proportion of intervention costs should be levied up front, and what proportion can be phased into the future (when the patient benefits accrue and are proven)? Separating the payment timeline from the treatment schedule is often referred to as outcome-based costing.
It is certainly no surprise that risk management continues to hold the spotlight as a hot topic within the biotechnology/pharmaceutical industry. With the increased focus by regulatory authorities on an organization’s ability to identify, mitigate, and control risks, the industry remains in a state of growth, developing and evolving practices to ensure proper alignment with industry best practices and regulators’ expectations. Many organizations are finding it difficult to establish and embed risk management practices, as doing so requires a paradigm shift from a traditional risk-averse industry culture.
Innovation in gene therapy brings the potential for transforming patient care and obviating the need for chronic therapy through single-dose cures. Despite the potential long-term benefits of this new therapeutic modality, gene therapy companies face a number of underappreciated challenges.
A Catalent customer wanted to run a clinical trial in Israel and was arranging to ship their clinical trial product from a non-Catalent facility in Europe to a Catalent-managed and audited depot in Israel. With the protocol based in the US and the investigational medicinal product (IMP) approved in the EU, the customer incorrectly assumed and, without checking further requirements, that Israel had the same QP (qualified person) approval process as the EU. This case study outlines the steps that Catalent took to ensure the QP release process could quickly advance in Israel while simultaneously updating the warning labels to meet the QP requirements.
Abeona Therapeutics is developing investigational gene and cell therapies for people living with serious and rare diseases. Last year, Abeona held a ribbon-cutting ceremony to celebrate the groundbreaking of a manufacturing facility that is one year later producing gene therapy material for their clinical and pre-clinical programs. As is the norm with gene therapy, Abeona uses a virus as vehicles to deliver functional copies of defective genes.
Over the past four decades, and especially in recent years, progress in the development of cell and gene therapies has reached unprecedented levels. In gene therapy alone, between 1989 and 2015 there were more than 2,330 clinical research programs targeting almost 50 different indications.1,2 As more cell and gene therapies reach commercial stage, industry insiders expect research in the sector to continue to expand in the coming years. For patients, clinicians, and health systems, new cell and gene therapies bring the promise of historic and transformative advances in the treatment of many serious diseases.
Because the parameters for ex vivo cell and gene therapy manufacturing differ considerably from chemical drug production, life sciences companies are likely to encounter challenges along key stages of the product life cycle.
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.
The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) has published technology roadmaps addressing needs and gaps in three key product areas: gene therapy, antibody-dug conjugates, and vaccines. The roadmaps were developed with the collaborative input of industry, academic, and government experts. This article highlights technology opportunities for gene therapy manufacturing.
In recent months, the U.S. Food and Drug Administration has increased its compliance focus on the regenerative medicine industry, vowing to crack down on illicit stem cell clinics and others advertising the untested use of human cell and tissue-based products (HCT/P), many of which are used in orthopedics.
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.
Whether because of unfamiliarity or lack of resources, master service or supply agreement (MSA) execution can be time consuming and difficult.
In the first part of this two-part article, common cryogenic shipping methods for gene therapy products were presented, and pros and cons of each method were discussed. In this part, major refrigerated transport methods of gene therapy intermediates will be addressed.
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.
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.
What a week. As you know, The Washington Post broke the news of Scott Gottlieb’s resignation as FDA Commissioner — the news, while not entirely unexpected, shot disappointment and concern through the entire healthcare industry. Nancy Bradish Myers, JD, president of Catalyst Healthcare Consulting penned an article for Cell & Gene that explains the silver lining of Gottlieb’s exit.
In Part 2 of my conversation with Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics, he addresses the unique demands of a gene therapy supply chain.
The ability to interrogate protein phosphorylation events and aberrant phosphorylation levels is critical to our understanding of various biological and pathological processes, particularly in the research of diseases, like cancer. However, detection of phosphorylated proteins in western blots can be challenging. Following are ten tips to ensure robust data generation and cleaner blots.
Which parameters really matter for cell therapy process development? G. John Morris, CEO Asymptote, part of GE Healthcare Life Sciences discusses the evidence in this blog post.
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.
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.
