Treatment using cell-based therapies involves significant investment and, therefore, can be expected to have concomitantly significant costs to patients and their insurers. Accordingly, patent exclusivity will be important to defray high development and regulatory compliance costs.

	The nature of such therapies, and recent patent law trends regarding natural products and methods relating to the practice of medicine, suggest these therapies may not be given the type of robust patent protection conventionally available for small-molecule drugs. The author discusses the impact of patent considerations for cell-based therapies.

Development Strategies for Emerging Therapies


	Vol. 30
	September 2017
	Pages: 19–22

	When referring to this article, please cite it as K. Noonan, " Patenting Prospects for Cell-Based Therapies," 

BioPharm International Development Strategies for Emerging Therapies eBook

Articles

Features

Cell-based therapies are moving medical treatments forward, but intellectual property uncertainties may delay progress.

Patenting Prospects for Cell-Based Therapies

Hundreds of clinical trials are currently underway testing the merits of cell therapies, as evidenced by the clinicaltrials.gov database and highlighted by the Alliance for Regenerative Medicine. A number of trials and commercially marketed products use adult, multipotent cells such as human mesenchymal stem/stromal cells (hMSCs). Large-scale industrialized production of these cells is necessary to continue their advancement through human clinical trials and commercialization as therapeutics. However, achieving this level of production while meeting rigorous quality standards will depend on further progress in the areas of cell culture and scale-up, characterization, enrichment, and purification to deliver a consistent and reproducible supply of cells. 


	Vol. 30
	September 2017
	Pages: 23–26

	When referring to this article, please cite it as A. Schnitzler et al., "Harvest of Human Mesenchymal Stem Cells from Microcarriers," 

Traditional planar culture formats are being superceded by microcarriers for large-scale cell therapy manufacturing.

Harvest of Human Mesenchymal Stem Cells from Microcarriers

On Nov. 2, 2012, UniQure’s Glybera, an AAV-1 based drug, was approved in Europe for use in adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD). Since then, interest in gene therapies has skyrocketed, with a fourfold increase in the number of gene therapies being developed between 2012 and 2015. With the power to overcome genetic disorders, these therapies could prove revolutionary to the field of medicine.

	The technology aims to treat diseases by delivering genetic material encoding a protein with a therapeutic effect into a patient’s cells. In most gene-therapy studies, a carrier molecule or a vector must be used to deliver the therapeutic gene to the target cells. There are a number of molecular vectors and techniques available for use; however, most frequently this is achieved with viruses. 


	Vol. 30
	September 2017
	Pages: 27–30

	When referring to this article, please cite it as O. Terova et al., "Affinity Chromatography Accelerates Viral
	Vector Purification for Gene Therapies," 

A case study demonstrates that affinity chromatography can offer efficiency and scalability for gene therapy manufacturing using viral vectors.

Affinity Chromatography Accelerates Viral Vector Purification for Gene Therapies

As anticipation builds about accelerated clinical adoption of advanced cell therapies, more attention is turning to the underlying manufacturing strategies that will allow these therapies to benefit patients worldwide. While cell therapies hold great promise clinically, high manufacturing costs relative to reimbursement present a formidable roadblock to commercialization. Therefore, the need for cost effectiveness, process efficiency, and product consistency is driving efforts for automation in numerous cell therapy fields and particularly for T-cell immunotherapies. 


	Vol. 30
	September 2017
	Pages: 10–18

	When referring to this article, please cite it as J. Morrissey, N. Trainor, and Y. Shi, "End-to-End Cell Therapy Automation: An Immunotherapy Case Study," 

Consider automation early in the rollout of clinical translation and scale up of clinical-trial protocols.

End-to-End Cell Therapy Automation: An Immunotherapy Case Study

3M Drug Delivery Systems and Panacea Pharmaceuticals are collaborating to deliver Panacea’s investigational cancer vaccine, PAN- 301-1, directly to the dermis via the 3M hollow microstructured transdermal system (hMTS).

	PAN-301-1 vaccine is a biologic that cannot be administered orally, or via a transdermal patch; therefore, it was thought that intradermal delivery to the skin could potentially be the optimal route of administration. The hMTS offers high-volume, reproducible, direct delivery capability to the highly-vascularized dermis.

	When referring to this article, please cite it as S. Fuller et al., "Optimizing a Unique Cancer Vaccine for Intradermal Delivery, " 

Using a hollow microstructured transdermal system to deliver vaccine directly to the dermis. 

Optimizing a Unique Cancer Vaccine for Intradermal Delivery

Microfluidics is fast becoming an essential tool for encapsulating nucleic acids. Microfluidics allows fine control over process parameters, enabling optimization of nanoparticle characteristics and nucleic acid encapsulation. The continuous, inherently scalable nature of the microfluidic process allows formulations to be optimized at low volumes, before being scaled-up using multiple microfluidic mixers in parallel. This review examines how microfluidics has been used in the formulation, preclinical, and clinical development of gene-delivery nanoparticles.

	When referring to this article, please cite it as S. Ip, D. Reichert, I. Captain, and R. J. Taylor, "Accelerating Genetic Medicine R&D Using Microfluidics," 

This review examines how microfluidics has been used in the formulation, preclinical, and clinical development of gene-delivery nanoparticles.

Accelerating Genetic Medicine R&D Using Microfluidics

As precision medicines garner increasing attention, drug makers have intensified their efforts to bring emerging therapies such as cell and gene therapies to market. This article gives an overview of cell/gene therapies in the making and explores the challenges biomanufacturers face in developing and manufacturing such inherently complex molecules. Unlike traditional small-molecule drugs and conventional antibody-based biologics, the roadblocks to developing and manufacturing cell and gene therapies can get complicated because of their molecular complexity and the fact that their mechanisms of action are not fully understood.

	When referring to this article, please cite it as F. Mirasol, "Overcoming Hurdles in Emerging Therapy Development, " 

The development of emerging therapies poses unique manufacturing and formulation challenges for drug developers as candidates like cell and gene therapies advance through the pipeline.