Friday, June 28, 2019 Daily Archives

Immunotherapy: Taking Aim at Solid Cancers

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As cell and gene therapies arrive on the market, all eyes have focused on autologous chimeric antigen receptor (CAR) T – cell therapies. At the 2019 Phacilitate Leaders World and Stem Cell Summit in Miami, FL, delegates looked at where the biopharmaceutical industry is going in the cellular immunotherapy space. Whether for off-the-shelf CAR T-cell products, personalized cancer vaccines, or modified natural killer (NK) cells derived from human induced pluripotent stem cells (iPSCs) — cell and gene therapy development is…

June 28, 2019

Supply Chain Solutions for Cell and Gene Therapy Companies

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Stakeholders across the supply chain stress that quality of starting material will be key to the success of cell and gene therapies. This is a topic that has created issues in the past, is puzzling the industry presently, and is likely to cause more problems going forward. This topic was front and center at the 2019 Phacilitate Leaders World and World Stem Cell Summit in Miami FL, with presentations focusing on supply chain solutions to address these complex challenges; cell…

June 28, 2019

Automation in Cell and Gene Therapy Development

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The US approvals of chimeric antigen receptor (CAR) T-Cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) and gene therapy Luxturna (voretigene neparvovec) in 2017 heralded a “new frontier of medicine.†But with great innovation comes great costs and criticism (such as the Kymriah’s US$475,000 price tag). Many companies argue that these one-off therapies represent good value for patients and payers compared with traditional treatments, however, no matter your perspective, the COGs picture for cell and gene therapies isn’t good and…

June 28, 2019

Manufacturing of Cell and Gene Therapies

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The peak of demand for curative cell and gene therapies will be unlike that of traditional drugs and thus could cause forecasting and overcapacity issues going forward. Predicting the future is always difficult, and poor decisions can be costly and highly damaging for a company. At the 2019 Phacilitate Leaders World and Stem Cell Summit in Miami, FL, several presentations and conversations focused on the forecasting dilemma and how manufacturing needs innovation, just to name two. This eBook details these…

June 28, 2019

Stem cell clinic compliance slower than expected, says FDA

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The US FDA continues its clampdown on unapproved stem cell therapies but says the pace of progress in making clinics comply is slower than expected. In November 2017, the US Food and Drug Administration (FDA) laid down a comprehensive regenerative medicine framework aimed at spurring innovation and access to potentially transformative products, while ensuring safety and efficacy for such products. This included warnings against providers of unapproved and unproven stem cell therapies, and a period of a risk-based approach to…

June 28, 2019

ambr alert: Sartorius vessel specialized for cell & gene therapies

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Sartorius has launched an ambr 250 vessel for cell and gene therapy applications it says taps into industry’s transition from adherent to suspension cell culture formats. Sartorius Stedim Biotech (SSB) has added to its range of ambr 250 modular benchtop bioreactor systems with a single-use vessel designed to accelerate the process development of cell and gene therapy applications, before scaling-up into single-use bioreactors and bags. Existing ambr mammalian vessels are often used for process development of monoclonal antibodies (MAbs) in…

June 28, 2019