In cell therapy trials, the ability to track a patient’s cells is essential. When you have over 100 patients, and the cells will require multiple shipments, using a spreadsheet is not possible. “For the safety of our patient, accurate tracking is a must,” says Gerald Garrett, VP of clinical operations for MarkerTherapeutics. “We need to know exactly where the product is at any step of the way.”
NeuBase is attempting to create a treatment for two rare diseases: Huntington’s disease and myotonic dystrophy. The company will face several challenges along the way, with the first being patient recruitment. Will young patients even want to know if they have the disease?
The decades-long focus on more “personalized” medicines has paved the way for the recent and anticipated advances in gene and cell therapies and progress in many other areas. The first generation of gene and cell therapies has the potential to transform the way we treat some diseases and can bring new hope to patients with many rare and serious conditions.
Since research into gene therapy first began more than 40 years ago, the field has expanded dramatically. For patients suffering from these rare diseases, however, progress is slow. It’s often difficult to get a correct diagnosis, let alone accurate information about potential treatment for a rare disease. These patients must rely collectively on scientists, physicians, pharmaceutical and biotechnology companies, and rare disease advocates to learn about these disorders.
Novel technologies to address areas left unaddressed by Kymriah and Yescarta have started streaming into the research arena. This article focuses on the barriers to widespread commercial adoption of the currently available CAR T cell therapies, and opportunities for developers of next-generation treatments.
Regenerative medicine holds promise, though evidence to back claims about current therapies is underwhelming. The FDA's announcement of the Tissue Reference Group Rapid Inquiry Program returns our attention to the administration's regenerative medicine policy framework.
Understanding the impact and requirements for a clearly defined supply chain strategy and implementing this into the complex environment of cell and gene therapies is not a simple task. It requires dedication and participation from all the stakeholders across the development-to-delivery life cycle. All the details need to be defined, simulation models developed, and risk assessed — and remediation plans put in place.
For a patient with a debilitating disease, such as pulmonary fibrosis (PF), moments of hope can be few and far between. Many of these patients search desperately for new therapies to improve their quality of life, or possibly cure their condition, sometimes outside of the approved treatments prescribed by their healthcare providers.
Are the methods used for developing the current generation of biopharmaceuticals, monoclonal antibodies, hormone replacements, etc., applicable to the next generation of Cellular and gene therapies?
The Institute for Clinical and Economic Review (ICER) recently conducted a study of value assessment methods considered for “cures” or “potential cures” and solicited input from international HTA bodies. The research will form the basis of a value framework that will be applied in the assessment of potential cures and other treatments that qualify as what ICER refers to as “single or short-term transformative therapies.”
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