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.
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.
Evaluating how much a process step contributes to viral clearance is an essential part of process validation in order to be sure you are manufacturing safe drugs. Learn more about how HiScale 10/40, packed with MabSelect PrismA, is a reliable choice for the capture step in a virus clearance study.
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.
As the second wave of CAR-T therapies begins coming to market in the next five years, their manufacturers will need to anticipate and prepare to address many of the same commercial challenges as their predecessors, as well as several additional ones.
Temperature-controlled packages used for the transport of gene therapy products use a combination of thermal insulators or thermoregulators to decrease the heat transfer rate from outside of the shipper to the inside. In this two-part article, we will explore common methods of temperature-controlled transportation and delve into their pros and cons. In Part 1, we focus on passive cryogenic options.
Today, March 21st, is ARM’s 7th Annual Cell & Gene Investor Day. The New York City-based one-day event provides institutional, strategic, and venture investors with unique insight into financing opportunities for cell and gene therapy-based treatment and tools.
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.
It was the announcement many have been dreading — but expecting — for several months now. Dr. Scott Gottlieb, 23rd Commissioner of the FDA, announced his decision to step down after 663 days (22 months) on the job. The news is sure to raise concerns for many, from investors to parents of teens tempted to vape. But I think the biggest surprise is how equally sad thought-leaders within the agency will be to say goodbye.
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.
Parenteral Drug Association (PDA) recently released Technical Report No. 81, “Cell-Based Therapy Control Strategy,” written by PDA’s Cell and Gene Therapy Task Force. Michael Blackton, Global Head of Quality at adaptimmune and PDA’s Co-Chair of the Cell and Gene Therapy Interest Group co-authored the report. Blackton’s also a member of the Biopharmaceutical Advisory Board and International Board of Directors. Here, he breaks down Technical Report No. 81 and explains the report’s biggest takeaways.
I caught up with Ryan Bartock, Head of Supply Chain and Network Strategy at Spark Therapeutics, to talk through some of the gene therapy sector’s heftiest supply chain challenges. In Part 1 of a 2 part series, hear what advice he has for pharma, bio executives, and CMOs to successfully manage and even streamline the supply chain.
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.
Tube welding versus using aseptic connectors—as with any debate, there are strong opinions on both sides. Following are some factors to consider that may tip the scale in favor of aseptic connectors.
Three considerations to give you confidence when selecting fluid handling connectors for your bio application.
Overcoming the challenges of manufacturing and administering a complex product to any patient is critical for delivering cell therapies. Two industry experts detail strategies to meet supply chain challenges.
Key Biologics is harnessing new freezing technology to help deliver the future of medicine. Digitally enabled cryogenic cold chain technology can now deliver the raw materials needed to manufacture cellular therapies.
