Brock Reeve

Generating iPSCs: translating cell reprogramming science into scalable and robust biomanufacturing strategies.

Induced pluripotent stem cells (iPSCs) have the potential to transform drug discovery and healthcare in the 21(st) century. However, successful commercialization will require standardized manufacturing platforms. Here we highlight the need to define standardized practices for iPSC generation and processing and discuss current challenges to the robust manufacture of iPSC products.

The impact of market volatility on the cell therapy industry.

Stock market volatility in the cell therapy industry has greatly hindered the investment necessary to fund translational therapies. Here, we review the volatility of leading companies and suggest that a distinct industry is maturing to a point at which the volatility should subside, providing a more attractive environment for future growth.

Cell Therapy Companies Make Strong Progress from October 2012 to March 2013 Amid Mixed Stock Market Sentiment

During Q4 2012 and Q1 2013, the cell therapy industry made strong progress in translation and commercialization. Continued development of the companies included in a dedicated stock market index suggests emergence of this industry as a distinct healthcare sector.

Enabling Consistency in Pluripotent Stem Cell-Derived Products for Research and Development and Clinical Applications Through Material Standards

There is a need for physical standards (reference materials) to ensure both reproducibility and consistency in the production of somatic cell types from human pluripotent stem cell (hPSC) sources. We have outlined the need for reference materials (RMs) in relation to the unique properties and concerns surrounding hPSC‐derived products and suggest in‐house approaches to RM generation relevant to basic research, drug screening, and therapeutic applications. hPSCs have an unparalleled potential as a source of somatic cells for drug screening, disease modeling, and therapeutic application. Undefined variation and product variability after differentiation to the lineage or cell type of interest impede efficient translation and can obscure the evaluation of clinical safety and efficacy. Moreover, in the absence of a consistent population, data generated from in vitro studies could be unreliable and irreproducible. Efforts to devise approaches and tools that facilitate improved consistency of hPSC‐derived products, both as development tools and therapeutic products, will aid translation. Standards exist in both written and physical form; however, because many unknown factors persist in the field, premature written standards could inhibit rather than promote innovation and translation. We focused on the derivation of physical standard RMs. We outline the need for RMs and assess the approaches to in‐house RM generation for hPSC‐derived products, a critical tool for the analysis and control of product variation that can be applied by researchers and developers. We then explore potential routes for the generation of RMs, including both cellular and noncellular materials and novel methods that might provide valuable tools to measure and account for variation. Multiparametric techniques to identify “signatures” for therapeutically relevant cell types, such as neurons and cardiomyocytes that can be derived from hPSCs, would be of significant utility, although physical RMs will be required for clinical purposes.

The global intellectual property landscape of induced pluripotent stem cell technologies

Will freedom to research and innovate be restricted as the induced pluripotent stem cell field advances toward the clinic, or are concerns premature within a rapidly changing ecosystem?

The Global Cell Therapy Industry Continues to Rise during the Second and Third Quarters of 2012

During Q2-Q3 2012, the cell therapy industry benefited from a number of positive external influences including advantageous changes to future FDA regulation, but stock market activity was highly mixed. The FDA approved two more products and an appreciable number of public-company-sponsored clinical trials are progressing through phases 1-3.

Quantitative assessment of barriers to the clinical development and adoption of cellular therapies: A pilot study.

There has been a large increase in basic science activity in cell therapy and a growing portfolio of cell therapy trials. However, the number of industry products available for widespread clinical use does not match this magnitude of activity. We hypothesize that the paucity of engagement with the clinical community is a key contributor to the lack of commercially successful cell therapy products. To investigate this, we launched a pilot study to survey clinicians from five specialities and to determine what they believe to be the most significant barriers to cellular therapy clinical development and adoption. Our study shows that the main concerns among this group are cost-effectiveness, efficacy, reimbursement, and regulation. Addressing these concerns can best be achieved by ensuring that future clinical trials are conducted to adequately answer the questions of both regulators and the broader clinical community.

Promising Growth and Investment in the Cell Therapy Industry during the First Quarter of 2012

In the first quarter of 2012, publicly traded companies in the cell-based therapy industry continued to show promising overall growth. Highlights included

Global strategic partnerships in regenerative medicine

85 million in new capital investment and steady clinical trial progress.

The implementation of novel collaborative structures for the identification and resolution of barriers to pluripotent stem cell translation.

The approach to research and development in biomedical science is changing. Increasingly, academia and industry seek to collaborate, and share resources and expertise, by establishing partnerships. Here, we explore the co-development partnership landscape in the field of regenerative medicine, focusing on agreements involving one or more private entities. A majority of the largest biopharmaceutical companies have announced strategic partnerships with a specific regenerative medicine focus, signifying the growth and widening appeal of this emerging sector.