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.
Cell therapies, in particular, chimeric antigen receptor modified T cells (CAR-T), are showing enormous therapeutic promise, with cancer remission rates over 80 percent.1 This alone is forcing cell therapy manufacturing companies to develop strategies to build and scale up production at levels never contemplated before.
Heart problems have touched many of our lives, or the lives of those we care about. Heart attacks and heart failure are leading killers in the U.S.,1 with limited treatment options available today. Typically, a heart attack is followed by significant heart muscle cell death, leaving the organ in a weakened state and the patient at risk of cardiovascular dysfunction and eventual heart failure. This leads to many of the deaths attributed to cardiovascular disease.
Jiwen Zhang, Executive Director, Regulatory Affairs at Tmunity Therapeutics Incorporated and Cell & Gene Editorial Advisory Board member explains the status of the cell and gene sector’s domestic and international regulatory framework. As President of the International Standards Coordinating Body (SCB), she also provides a synopsis of the most recent Stakeholder meeting.
Unmet medical need (UMN) is not a new concept, but it is an increasingly important one. Regulators and payers are nudging the industry to steer R&D investments toward areas with higher unmet need and less crowded pipelines.
While recent scientific breakthroughs for regenerative cell therapies are bringing great hope to patients and providers in desperate need of treatments and cures, they have created a major question mark for the healthcare system at large. How do you determine payment for something of enormously high value that has literally never existed before?
A quick and nimble approach is often sought after in the cell and gene therapy product space due to the likely one-time or limited patient dosing and/or rare disease conditions limiting the number of available patients, which reduces the number of clinical studies.
With the recent breakthroughs in cell and gene therapy, there is increased emphasis on the design and implementation of different supply chain models to support the movement of materials and drug product across the chain of care. Unlike more traditional supply chains, many of these therapies have unpredictable sources and manufacturing and infusion locations. The most extreme example is loosely referred to as the “vein-to-vein” supply chain — particularly for autologous therapies.
Nicole Gularte is currently battling cancer for the 8th time. The last time she had to do so, it seemed a CAR-T cell therapy trial might be the solution. This article is about her long battle with cancer, the struggle to get into a CAR-T cell trial, and the ups and downs she has faced along the way. Her story is an inspiration to others.
The FDA published a new draft guidance on August 10, 2018 entitled, Expansion Cohorts: Use in First-In-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics. The comment period for the draft guidance closed on October 12, 2018 and approximately 20 comments were submitted to the docket. The guidance provides sponsors with recommendations for designing and conducting first-in-human (FIH) multiple expansion cohort trials within their oncology development programs.
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