Global manufacturing of CAR-T therapies: Looking at challenges based on Novartis’ CTL019

Novartis has been advancing CTL019, an investigational chimeric antigen receptor T cell (CAR T) therapy for relapsed/refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) through clinical trials. Novartis shared insights from the development of the therapy in an opinion manuscript, as a global perspective on the considerations and challenges of

From nature to health: Waste and plants become biofunctional materials for tissue engineering

We have written, on multiple occasions in the past, that the cost of biopharmaceuticals, and therapies based on them, is, in large part, dependent on the choice of raw materials used in their development and manufacture. Cost-effective scaleability and manufacturability are important considerations in this regard. Here, we highlight two recent studies that have investigated how natural materials

New studies look at better materials and cold chain management strategies for improved cryopreservation

As new regenerative medicines advance through the clinical pipeline, cryopreservation – most simply described as storage of biological material at sub-freezing temperatures – becomes of increasing importance. While preservation of organs for transplantation involves maintaining organs in an “alive” state in the short window prior to transplantation at 2-8C, cryopreservation requires a different set of

FDA Introduces ‘Regenerative Medicine Advanced Therapy’ Drug Designation

At the end of 2016, the United States Congress passed the 21st Century Cures Act. Addressing some of the provisions included in the Act, the FDA followed through, on January 17, 2017, by introducing a new designation process for regenerative advanced therapies, called Regenerative Medicine Advanced Therapies (RMAT). On a fundamental level, this new designation provides an

New injectable hydrogels deliver drugs and growth factors for wound healing, ischemia and arthritis therapy

Injectable hydrogels – three-dimensional networks of polymers engineered to be stable in liquid form at room temperature – are attractive for their utility in soft tissue repair. One of their main features is the ability to enhance the delivery and uptake of active drugs and biomolecules. Their utility as carriers of bioactive molecules has, in recent

Microfluidic Platforms aim to Accelerate CAR-T cell therapy manufacturing

Researchers at MIT’s Charles Stark Draper Laboratory Draper Laboratory – a not-for-profit research and development company focused on development of technical solutions for health, energy, national security and space – have recently announced the development of microfluidic platforms to accelerate the manufacturing of CAR-T therapies. The current focus is on two platforms: one for enrichment

Moving Cell and Gene Therapies Forward – Updates from the EMA

On 16 December 2016, the European Medicines Agency (EMA), together with the European Biopharmaceutical Enterprises (EBE) held the fifth annual “Optimizing the development of ATMPs to meet patient needs” regulatory conference on the optimization of the development of advanced therapy medicinal products (ATMPs) to meet patient needs in London, UK. The conference featured a group of international pharmaceutical

In vivo delivery of genes and drugs via nanoparticles can now be screened quickly via DNA “barcodes,” new study shows

The science of developing nanoparticles as delivery tools for various biologically active agents – genes and drugs – has come a long way. Material science advances have allowed for the construction of nanoparticles with different physicochemical properties, which has, in turn, resulted in remarkable advances, particularly in the area of nucleic acid delivery for the