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, 23^rd 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.

Gene therapy may be the next revolution in healthcare. The opportunity to cure diseases through the modification of the genome was once considered science fiction, but no longer. The United States has seen a few approvals in this area, including the approval of voretigene neparvovec for a rare form of blindness. In addition, CAR-T agents, two of which are available, have demonstrated alternative ways to treat disease by manipulating genes of different cells.

In November 2018, I wrote an article about establishing a supply chain for autologous cell therapies — those formulated using a patient’s own cells. This partner piece highlights considerations that need to be taken into account when developing a commercialization and supply chain strategy for allogeneic cell therapies.

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.

Pharmaceutical companies have three targeted users: physicians, pharmacists, and patients. Various digital platforms are used to create a good customer experience for educating these end users. The core focus is on patients driving patient care, education, and adherence.

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.

Autologous (patient-derived) therapies represent unique challenges to processing. One-patient, single-batch is a radical change from the scale our industry is accustomed to. At this level the filling is more analogous to limited Phase 1 clinical trial materials or even preclinical applications. The logistics and required compliance of manufacturing these personalized therapies is drastically different and presents a combinatorial explosive problem. One aspect of this process where traditional methods fall apart is filling of these therapies.

Today’s clinical trial partner is tomorrow’s post-approval patient. They are the experts who can shape a program over time, be a voice during the regulatory approval process, and provide insight on what other patients hope to find in a therapy.

The difficulty of balancing paying for innovation with budgets has plagued Europe for years, and the emergence of cell and gene therapies has only exacerbated that conundrum. 

Two of the field’s most valuable luminaries representing both academia and industry — Dr. Bruce Levine and Simon Ellison, ISCT Commercialization Committee — discuss determining how CMOs can and should successfully manage cell and gene therapy supply chains.

Being a Greater Philadelphia Area resident affords me the opportunity to meet and collaborate with scientists, doctors, researchers, and other highly skilled professionals knee-deep in the cell and gene therapy sector. From the University of Pennsylvania to CHOP to Spark Therapeutics to Tmunity and many, many more, Philadelphia not only continues to shine as the geographical standout for innovation and continuous forward movement for cell and gene therapies but also as the smart economical choice for SMEs in the field.

Regenerative medicine could be a game-changing approach to meet the needs of insulin-dependent patients. Although the field is still in its infancy, the initial clinical data looks promising. This article takes a closer look at a few innovative companies that are racing to bring the first regenerative medicine for diabetes to the market.

The temperature-sensitivity of cell and gene therapies, their personalized nature, and their circular (“vein-to-vein”) supply chain make maintaining a seamless supply chain critical; however, it is not easily achieved.

One conversation with Michael Blackton, VP QA CMC at Adaptimmune, proves why he is known for being a passionate, results-oriented leader focused on QA, analytical development, and more. Validate for yourself as Blackton answers my questions about process development and quality management. And, he provides a fresh outlook on recruiting senior-level executives.  

The first round of CAR T therapies, now considered “legacy,” is largely being produced using biosafety cabinets and bench-top incubators with multiple open operational steps. The next generation of production processes will reduce or eliminate the open operations and automate cell-based manipulations.

Over the past 20 years, hope has grown around the use of stem cells to enhance regeneration following a heart attack, wherein stem cells would facilitate tissue repair and help restore heart muscle function. However, the legitimacy of cardiac stem cell research is now being called into question.