Gazing Long into a Clinical and Social Abyss? Treating Hypertrophic Scarring and Keloids.

This commentary discusses the unmet clinical and social needs associated with hypertrophic scars and keloids. The authors critically appraise these issues within the context of contemporary clinical standards of care and social mores catalyzed by the COVID-19 pandemic.

Potential lifetime quality of life benefits of choroideremia gene therapy: projections from a clinically informed decision model.

Background: The first gene therapy for an inherited retinal dystrophy recently received market approval in the United States; multiple other gene therapies are in the clinical pipeline. Thus far, gene therapy has commanded prices in the range of $500,000 to over $1,000,000 for the one-time doses and have been indicated for highly orphan diseases where there is no other viable treatment option. To be adopted by healthcare systems, gene therapy will need to show clinical benefit in line with its increased costs.

The Reporting Items for Patent Landscapes statement.

The reporting quality of patent landscapes is inadequate. The Reporting Items for Patent Landscapes (RIPL) checklist can improve reporting quality.

Biotechnology Governance 2.0: A Proposal for Minimum Standards in Biotechnology Corporate Governance.

Based on experience and the existing limited research literature in biotechnology corporate governance, the authors propose potential attributes of minimum corporate governance standards for biotechnology companies, as a basis for further quantitative and qualitative research. The authors assert that the recent proliferation of biotechnology start-up organizations is substantively changing inter- and intraorganizational cultures throughout the health care sector via a "." Therefore, governance decisions and actions-both positive and negative-that are instituted in start-up biotech companies may set new norms for other start-up biotech companies, the larger (bio)pharmaceutical companies by which they are acquired and the range of health care subsector actors that interact with biotechnology companies.

Bioprocess decision support tool for scalable manufacture of extracellular vesicles.

Newly recognized as natural nanocarriers that deliver biological information between cells, extracellular vesicles (EVs), including exosomes and microvesicles, provide unprecedented therapeutic opportunities. Large-scale and cost-effective manufacturing is imperative for EV products to meet commercial and clinical demands; successful translation requires careful decisions that minimize financial and technological risks. Here, we develop a decision support tool (DST) that computes the most cost-effective technologies for manufacturing EVs at different scales, by examining the costs of goods associated with using published protocols.

Bioprocessing automation in cell therapy manufacturing: Outcomes of special interest group automation workshop.

Phacilitate held a Special Interest Group workshop event in Edinburgh, UK, in May 2017. The event brought together leading stakeholders in the cell therapy bioprocessing field to identify present and future challenges and propose potential solutions to automation in cell therapy bioprocessing. Here, we review and summarize discussions from the event.

The European Falsified Medicines Directive in Poland: background, implementation and potential recommendations for pharmacists.

By February 2019, the Polish pharmaceutical industry, community and hospital pharmacies, wholesalers and parallel traders must all comply with the EU-wide Falsified Medicines Directive (FMD) legislation (2011/62/EU), to ensure that no medicinal product is dispensed to a patient without proper tracking and authentication. Here we describe how Poland is complying with the new EU regulations, the actions that have been taken to incorporate the FMD into Polish Pharmaceutical Law and whether or not these actions are sufficient. We found that Poland is only partially compliant with the FMD and further actions need to be undertaken to fully meet the Delegated Act (DA) requirements.

Regulatory Considerations for Gene Therapy Products in the US, EU, and Japan.

Developers of gene therapy products (GTPs) must adhere to additional regulation beyond that of traditional small-molecule therapeutics, due to the unique mechanism-of-action of GTPs and the subsequent novel risks arisen. We have provided herein a summary of the regulatory structure under which GTPs fall in the United States, the European Union, and Japan, and a comprehensive overview of the regulatory guidance applicable to the developer of GTP. Understanding the regulatory requirements for seeking GTP market approval in these major jurisdictions is crucial for an effective and expedient path to market.

Adipose-Derived Stem Cells in Aesthetic Surgery: A Mixed Methods Evaluation of the Current Clinical Trial, Intellectual Property, and Regulatory Landscape.

Background: Adipose tissue, which can be readily harvested via a number of liposuction techniques, offers an easily accessible and abundant source of adipose-derived stem cells (ASCs). Consequently, ASCs have become an increasingly popular reconstructive option and a novel means of aesthetic soft tissue augmentation.

Objectives: This paper examines recent advances in the aesthetic surgery field, extending beyond traditional review formats to incorporate a comprehensive analysis of current clinical trials, adoption status, and the commercialization pathway.

Cell assisted lipotransfer in breast augmentation and reconstruction: A systematic review of safety, efficacy, use of patient reported outcomes and study quality.

Background: Cell assisted lipotransfer serves as a novel technique for both breast reconstruction and breast augmentation. This systematic review assesses the efficacy, safety and use of patient reported outcome measures in studies involving cell assisted lipotransfer. We also carry out an objective assessment of study quality focussing on recruitment, follow-up and provide an up-to-date clinical trial landscaping analysis.

Corrigendum: A Quantitative Assessment of Factors Affecting the Technological Development and Adoption of Companion Diagnostics.

[This corrects the article on p. 357 in vol. 6, PMID: 26858745.

A Quantitative Assessment of Factors Affecting the Technological Development and Adoption of Companion Diagnostics.

Rapid innovation in (epi)genetics and biomarker sciences is driving a new drug development and product development pathway, with the personalized medicine era dominated by biologic therapeutics and companion diagnostics. Companion diagnostics (CDx) are tests and assays that detect biomarkers and specific mutations to elucidate disease pathways, stratify patient populations, and target drug therapies. CDx can substantially influence the development and regulatory approval for certain high-risk biologics.

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.

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.

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.

Global strategic partnerships in regenerative medicine.

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.

CASMI TSCC launch event, Paris, France, July 2013: an assessment of the key barriers to the commercialization and clinical adoption of pluripotent stem cell therapies.

The high incidence of unmet medical needs in combination with the rising burden of chronic diseases, linked to an increasingly aging population, necessitates new approaches to therapeutic intervention. One potential class of health care innovation that may offer an alternative approach to addressing current shortfalls is stem cell therapies. The CASMI Translational Stem Cell Consortium (CTSCC) was formed to elucidate the key hurdles to the commercialization and clinical adoption of stem cell technologies, with a particular focus on pluripotent stem cell (PSC) technologies.

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

Increased global connectivity has catalyzed technological development in almost all industries, in part through the facilitation of novel collaborative structures. Notably, open innovation and crowd-sourcing-of expertise and/or funding-has tremendous potential to increase the efficiency with which biomedical ecosystems interact to deliver safe, efficacious and affordable therapies to patients. Consequently, such practices offer tremendous potential in advancing development of cellular therapies.