First gene therapy pulled from market

A dichotomy: While efforts to advance new gene therapies to the clinic are progressing at a staggering pace, one drug that had passed such hurdles is now going the opposite direction. Dutch biotechnology company uniQure announced last week it was removing its gene therapy — going by the commercial name Glybera (alipogene tiparvovec) — from the market, by not

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