Journal Archive

Commercial Insights

Cell & Gene Therapy Commercial Insight – July 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:

The race to get the first product approved in hemophilia continues apace, as both Shire and uniQure release positive news this month, albeit in different types of the condition. It’s a crowded space – competitors include Biomarin, Dimension Therapeutics, Sangamo Biosciences and Spark Therapeutics. Biomarin leads the field in hemophilia A with a product already in Phase 1/2, so Shire has some catching up to do following the announcement of its IND submission for SHP654. In hemophilia B, uniQure presented clinical data showing that patients may be eligible for treatment with AMY-060 even when they have pre-existing neutralising antibodies; however, the news is somewhat tarnished by the announcement that Chiesi has withdrawn from an agreement to co-develop the treatment, citing “changes in strategic priorities”.

CELL THERAPY:

The cell and gene therapy industry is inching closer to approvals in major markets for the world’s first CART therapies. Both Novartis and Kite have BLAs submitted in the USA for pediatric acute lymphoblastic leukemia (ALL) and relapsed or refractory non-Hodgkin lymphoma (NHL), respectively. In July, Kite Pharma became the first company to submit a Marketing Authorization Application to the EMA for a CART product (axicabtagene ciloleucel). While the first indications being targeted by Novartis and Kite are different, there will be overlap in patient populations in the future. It will be interesting to see how the company’s position their products as approvals are granted following the first set of indications and what the dynamics of market uptake will look like. The latter will be driven by reimbursement, though prospects are strong in both cases.

DOI: 10.18609/cgti.2017.055
Citation: Cell Gene Therapy Insights 2017; 3, 509-520.

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Expert Insight

Advances in the development of bioprocesses for vaccines and viral vectors for gene therapy

Spotlight Article

Interview

Overcoming Downstream Bottlenecks in Cell & Gene Therapy Manufacturing

Manuel Carrondo

GS

Manuel Carrondo has been a Professor of Chemical and Biochemical Engineering at the University Nova de Lisboa for over 20 years. His key areas of research, from which over 230 papers have been published, include animal cell technology and its application for human and animal health; therapeutic and diagnostic recombinant proteins, including fusion proteins; virus like particles and pseudo viruses as vaccination agents and viruses as deliverables for gene therapy. Carrondo’s expertise has been tapped by advisory board roles at a number of companies and institutes, most recently the Fraunhofer Institute for Biomedical Engineering, iNOVA4Health Portugal and the OECD working party on Biotechnology, Nanotechnology and Converging Technologies. Earlier in his career he founded the Portuguese Academy of Engineering as well as iBET – Institute for Experimental and Technological Biology of which he is currently vice president. He has served as visiting professor at Carnegie- Mellon University and MIT, among others. Manuel Carrondo graduated in Chemical Engineering from Universidade do Porto in Portugal, followed by an MSc and PhD at Imperial College London in Environmental Engineering, and finally a Habilitation in Biochemical Engineering at his present university.

DOI: 10.18609/cgti.2017.053
Citation: Cell Gene Therapy Insights 2017; 3(6), 483-487.
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Bioprocess engineering strategies for autologous human MSC-based therapies: one size does not fit all

Spotlight Article

Expert Insight

Ioannis Papantoniou, Toon Lambrechts & Jean-Marie Aerts

Overcoming downstream bottlenecks in cell and gene therapy manufacturing

Autologous cell therapies are currently being evaluated in multiple clinical trials and are becoming a reality in advanced healthcare services. Compared to allogeneic cell therapies, where one batch of cells can be used to treat multiple patients and which allows a business model that is more closely related to the traditional biologics, autologous or patient-specific cell-based therapies present a whole new set of challenges. While these new challenges can originate from ethical issues (e.g., concerns about the patient in cases of batch failure) or from safety concerns (e.g., cross-contamination between patients), this article provides an overview of the technical side of cell-therapy manufacturing that is subject to donor-related variability. Although several studies have managed to produce batches of cells with a scale that satisfies therapeutic needs, there are still a number of challenges that need to be tackled. Unlike traditional manufacturing processes where the input material is relatively constant over time, personalization aspects inherent to the autologous reality will expose manufacturing to significant variability and production risks. The authors argue that for autologous cell production, where every patient-specific production batch can be considered as an unknown process, a combination of automated production processes and robust process monitoring & control capabilities can provide quantitative process understanding. At a second stage, provided large data becomes available through (on-line) data-based process analytics, minimized risk and cost-effective cell production for clinical use will become a reality.

Submitted for review: May 19 2017 Published: Aug 4 2017
DOI: 10.18609/cgti.2017.040
Citation: Cell Gene Therapy Insights 2017;3(6), 469-482.
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The importance of fill & finish in the commercialization of your cell & gene therapy

Spotlight Article

Innovator insight

Jean-Sébastien Parisse

Jean Sebastian Parisse

Overcoming Downstream Bioprocessing Bottlenecks

 
 

Jean-Sébastien is Head of Sales at Aseptic Technologies, a company he joined in 2007. His role is to manage and direct global sales efforts of Aseptic Technologies; accelerating growth and creating tighter connections between customer requirements and innovation, along with increased service levels, with a special attention to advanced therapy medicinal products since 2009. Jean-Sébastien is also a member of the Process and Product committee of the International Society for Cell Therapy (ISCT).

DOI: 10.18609/cgti.2017.043
Citation: Cell Gene Therapy Insights 2017; 3(6), 405-408.
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Advances and challenges in umbilical cord blood and tissue bioprocessing: procurement and storage

Spotlight Article

Expert Insight

Lindsey Parker, Shaun Mansfield, Kate Sneddon, Ben Charles and Qasim A. Rafiq

Overcoming downstream bottlenecks in cell and gene therapy manufacturing

Umbilical cord tissue and blood is banked to complement the rapidly advancing field of tissue engineering and regenerative medicine for both autologous and allogeneic therapeutic applications. Whilst many problems concerning the use of the hematopoietic and multipotential mesenchymal stromal cells contained therein may be addressed through the future development of GMP-compliant manufacturing strategies, collection and bioprocessing of these tissues can be optimised in the present to maximise clinical outcomes. In this review, we describe current procurement, processing and storage approaches for umbilical cord blood and tissue; current challenges and how these may be met to augment translation and use of therapeutics harnessing their derivatives.

Submitted for review: May 18 2017 Published: Aug 4 2017
DOI: 10.18609/cgti.2017.042
Citation: Cell Gene Therapy Insights 2017;3(6), 489-508.
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Challenges and advances in scale-up of label-free downstream processing for allogeneic cell therapies

Spotlight Article

Expert Insight

Fernanda Masri, Marieke A Hoeve, Paul A De Sousa & Nicholas A Willoughby

Overcoming downstream bottlenecks in cell & gene therapy manufacturing

Recent advances in stem cell research and regenerative medicine are leading towards the realistic commercial prospect of more complex cell-based therapeutic products, offering the potential to revolutionize aspects of healthcare system. To date however, there are no truly ‘large-scale’ cell therapy products available. To achieve successful commercial production, many factors come into play. To name a few; economics, robustness, reproducibility and, what this review is concerned about: scalability. With cell therapies, a change in the processing environment may lead to a product change, which ultimately may be the difference between a successful batch (meeting product specifications) or a failed one [1]. To minimize process changes throughout the scales, processing steps must be carefully selected from an early stage. A particular challenge faced is that current ‘gold standard’ techniques for cell separation are not generally compatible with large scale processes. Dead-end batch centrifugation is a clear example of a process step that is heavily manual, difficult to automate while maintaining sterility, and limited in scalability [1]. The scope of this article is to explore and evaluate current and potential future techniques for cell separation at large scale only.

Submitted for review: May 2 2017 Published: Aug 4 2017
DOI: 10.18609/cgti.2017.041
Citation: Cell Gene Therapy Insights 2017;3(6), 447-467.
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Cell therapy post-production technologies: a select review of key innovations in the field

Expert Insight

Maria L Thompson, Brian Schryver & Rolf O Ehrhardt

The cell therapy industry continues to gain momentum as an increasing number of treatments move forward from clinical trials to commercialization. Recent changes in regulatory language and procedures specifically recognize the potential of cell-based therapeutics. The expected and observed boost in manufacturing of cellular therapies must be accompanied by innovations in the post-production processing of these products to accommodate the higher production volume and increased variety of cellular therapies. Novel post-production technologies facilitate and accelerate product delivery, allowing manufactured live cell therapies to reach patients more reliably and efficiently. This article reviews a select number of the latest innovations projected to drive successful commercialization of cell therapy products.

Submitted for review: Apr 26 2017 Published: July 19 2017
DOI: 10.18609/cgti.2017.049
Citation: Cell Gene Therapy Insights 2017; 433-441.
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