A range of factors — including small patient populations, complex manufacturing processes, and lack of specialized expertise — are positioned to both drive up costs and require new options for stakeholder engagement and risk sharing along the development pathway. New approaches in development are needed to support the next generation of novel drugs on the horizon.
Single use technologies are expected to play a big role in the commercialization of cell and gene therapies because of their cost, speed, and sterility benefits. In some ways, will cell and gene therapy manufacturing be similar to biopharmaceutical manufacturing? What are the key differences and gaps in learning?
This article highlights the diverse interests and range of opinions about pricing innovative and often high-cost therapies, but also demonstrates that most players in the sector see an urgent need for new pricing models to accommodate cell and gene therapy products.
Part 2 of this two-part article reviews lessons from Spark Therapeutics’ pivotal program for Luxturna, a gene therapy approved for the treatment of patients with retinal dystrophy associated with confirmed biallelic mutation in the RPE65 gene, and summarize key considerations for the clinical development and commercialization of gene therapies.
Aside from the clinical opportunity that gene therapy products present; they do introduce tremendous complexity into both the manufacturing and commercial processes. Remember these three points before developing your gene therapy plan.
With several first-in-class gene therapies now approved, distinct new challenges are now coming into focus. This article provides a high-level overview of the gene therapy pipeline for non-oncology rare diseases and discuss key issues impacting clinical development.
This article posits that the orphan market is actually a collection of seven distinct business model archetypes that are driven by key disease, patient, and market characteristics. By disaggregating and characterizing these business models and their key success factors/risks, readers may be able to focus their corporate strategies to optimize the value and impact of their orphan therapies.
In this series on "Life Science Leadership In Action," we discuss PolarityTE, which focuses on regenerating lost tissues in their original complex forms.
The story of how Oxford BioMedica won a key contract from Novartis to provide the commercial and clinical supply of lentiviral vectors used to produce the Big Pharma's CAR-T cell therapy Kymriah.
The Orphan Drug Act of 1983 (ODA) has been wildly successful in its primary goal of spurring innovation that has led to lifesaving therapies. Unfortunately, the design of the ODA and several of the incentives the act provides have led to a host of unintended consequences.