Journal Archive

Commercial Insights

Cell & Gene Therapy Commercial Insight – April 2017

Mark Curtis & Richard Philipson

Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.

GENE THERAPY:

This month sees mixed fortunes for uniQure, with the decision not to renew the marketing authorisation for Glybera, whilst at the same time releasing good news, in the form of European PRIME designation, for its hemophilia B product. Glybera was rejected three times by the European Medicines Agency before finally gaining approval in November 2012, only to see the product used commercially in just a handful of patients. The decision to discontinue the product is no great surprise, but must leave Chiesi Farmaceutici with questions about the long-term viability of its partnership with uniQure. On a more positive note, several companies have released positive clinical data, including early evidence of clinical benefit for Abeona’s cell-based therapy for epidermolysis bullosa, and confirmation of clinical benefit for Biogen’s recently approved, ASO-based treatment Spinraza in spinal muscular atrophy.

CELL THERAPY:

The use of T cells to treat autoimmune disorders is a relatively niche area of study, by comparison to the activity we are seeing in oncology, but is an application that holds promise. Atara Biotherapeutics announced positive interim data from an early study investigating ATA188, an autologous T-cell product targeting Epstein–Barr virus, in patients with primary and secondary progressive multiple sclerosis. The company is pursuing the technology on the premise that B cells and plasma cells expressing viral antigens can spur an autoimmune response and MS pathology. We saw Opexa recently try and fail in this space, albeit with a different approach, but given the limited entry of really game-changing therapeutics for MS patients over the years, a cell-based immunotherapy approach is one worth pursuing. What is most intriguing about the early data presented by Atara is the range of objective clinical outcomes experienced by patients, which include reduction in fatigue and nocturnal episodes, improvements in dexterity and walking ability, and improvements in eye sight.

DOI: 10.18609/cgti.2017.033
Citation: Cell Gene Therapy Insights 2017;3(5),313-327.

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Interview

Product and Process Development: Considerations around Validation and Control

Spotlight Article

Interview

Michelle myers

Latest Advances in Addressing Biopreservation Challenges

 
 

Michelle Myers

Michele Myers is Senior Director of Validation and Lifecycle Management for Cell and Gene Therapies within GSK’s Platform and Technology Sciences Department with responsibility for technology transfer, process validation and product lifecycle management. She played an important role as part of the team responsible for gaining approval of Strimvelis™, an ex vivo gene therapy product for the treatment of ADA-SCID. Before joining the Platform and Technology Sciences team, Michele worked in GSK’s Global Manufacturing and Supply organization where she supported commercial biopharmaceutical production, development of production control strategies and process validation. Michele was previously an associate director at Johnson & Johnson where she worked in support of commercial biopharmaceutical production, managed a group responsible for process development for a cell therapy product and prepared regulatory filings in support of cell therapy programs. Michele is a certified Six Sigma Black Belt and holds a BA in biology from Temple University and a Ph.D. in biochemistry from the Pennsylvania State University.

DOI: 10.18609/cgti.2017.034
Citation: Cell Gene Therapy Insights 2017; 3(5), 379-384.
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Expert Insight

The role of biopreservation in cell and gene therapy bioprocessing

Spotlight Article

Expert Insight

Qasim A Rafiq, Alvin W Nienow, Christopher J Hewitt & Karen Coopman

Latest Advances in Addressing Biopreservation Challenges

Cell and gene-based therapies represent a novel therapeutic modality that has the potential to provide a treatment option for a range of medical conditions. There are, however, numerous processing and manufacturing challenges that must be addressed before such therapies are considered commercially and clinically viable. A significant challenge associated with the manufacture of such therapies is ensuring cell quality and the product’s critical quality attributes are retained throughout the entire bioprocess. Biopreservation is an important aspect of cell and gene-based therapy bioprocessing, which enables the development of cell banks. It increases process flexibility by providing a shelf-life to the product, enables the storage of intermediates and provides breakpoints within the process. In this article, we summarize the advances and challenges associated with biopreservation of cell and gene therapies.

Submitted for review: May 3 2017 Published:June 6 2017
DOI: 10.18609/cgti.2017.037
Citation: Cell Gene Therapy Insights 2017;3(5), 335-344.
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Setting the standards for cryopreservation of stem cells for clinical applications

Spotlight Article

Interview

Latest Advances in Addressing Biopreservation Challenges

Glyn Stacey

GS

Glyn Stacey has a background in microbiology and cancer research and has worked on the development of cell substrates for manufacture of biological medicines for over eighteen years. He is currently at the National Institute for Biological Standards and Control which is a part of the Medicines and Healthcare Products Regulatory Agency. He is Director for the UK Stem Cell Bank (UKSCB). The UKSCB has been a licensed clinical tissue bank since 2004. The work of his group has covered safety and quality issues in cell therapy, cells used for manufacturing purposes, development of novel cell-based assays and the development of reference materials for tissue typing and diagnosis of genetic disorders. This work includes the need for scale up of preservation techniques and long-term storage of DNA and cell lines of various types including human stem cell lines and cells used in bioassays and vaccine production. Glyn has served on numerous steering groups for organizations promoting and funding regenerative medicine. He has also chaired the UK National Clinical hESC Forum and the scientific advisory board for a Public Private Partnership not-for-profit company called Stem Cells for Safer Medicine.

DOI: 10.18609/cgti.2017.036
Citation: Cell Gene Therapy Insights 2017; 3(5), 385-388.
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Biopreservation Best Practices for regenerative medicine GMP manufacturing & focus on optimized biopreservation media

Spotlight Article

Innovator Insight

Brian J Hawkins, Alireza Abazari & Aby J Mathew

Latest Advances in Addressing Biopreservation Challenges

Cellular therapies are cell and tissue products sourced from biological materials that are employed as ‘living drugs.’ Such ‘living drugs’ require specialized biological support, namely biopreservation, to maintain optimal recovery, viability and return to function post-preservation. To achieve successful biopreservation, optimization of a multitude of parameters, including cooling/thawing rates based on the biophysics of specific cell types, the temperature of storage and transportation, as well as biopreservation media are of paramount importance. In particular, the choice of biopreservation media is pivotal for clearing regulatory hurdles and facilitating commercialization. Traditional extracellular-like (isotonic), home-brew cocktails (which may contain serum) may not be compatible with a Good Manufacturing Practices (GMP) clinical manufacturing process. This article will address the challenges associated with maintaining the viable recovery and functionality of ‘living drugs’, discuss the benefits and consequences of low-temperature biopreservation, outline Biopreservation Best Practices, and propose considerations for incorporating Biopreservation Best Practices into a GMP cell therapy product. Integration of Biopreservation Best Practices, beginning with the selection of optimized media, ensures that cells remain viable and functional throughout the cell product lifecycle, thereby optimizing manufacturing and clinical outcomes.

Submitted for review: Apr 10 2017 Published: June 5 2017
DOI: 10.18609/cgti.2017.035
Citation: Cell Gene Therapy Insights 2017;3(5), 345-358.
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Applications and optimization of cryopreservation technologies to cellular therapeutics

Spotlight Article

Expert Insight

Barry Fuller, Jordi Gonzalez-Molina, Eloy Erro, Joana De Mendonca, Sheri Chalmers, Maooz Awan, Aurore Poirier, Clare Selden

Latest Advances in Addressing Biopreservation Challenges

Delivery of cell therapies often requires the ability to hold products in readiness whilst logistical, regulatory and potency considerations are dealt with and recorded. This requires reversibly stopping biological time, a process which is often achieved by cryopreservation. However, cryopreservation itself poses many biological and biophysical challenges to living cells that need to be understood in order to apply the low temperature technologies to their best advantage. This review sets out the history of applied cryopreservation, our current understanding of the various processes involved in storage at cryogenic temperatures, and challenges for robust and reliable uses of cryopreservation within the cell therapy arena.

Submitted for review: Apr 24 2017 Published:June 6 2017
DOI: 10.18609/cgti.2017.038
Citation: Cell Gene Therapy Insights 2017;3(5), 359-378.
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