Bringing you the latest cutting-edge research and commentary in bioscience.

Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Issue Vol 1 Issue 1

Welcome to Cell and Gene Therapy Insights

Chris Mason & Elisa Manzotti


Foreword


In the 12 months since the inception of Cell & Gene Therapy Insights, barely a week has gone by without some mention of a scientific advancement or investment in the space. Whilst the immunotherapy clinical data points to a transformative step-change in oncology treatment, the field of cell and gene therapy comprises an incredibly diverse range of therapeutic modalities and products, many of which remain at an early stage of development.

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A US Cell Therapy Regulatory Toolkit

Spotlight Article

Navigating the Regulatory Landscape

Emily J Culme-Seymour, James Lawford Davies, Julian Hitchcock, Julian Mason, Melissa K Carpenter & Chris Mason Navigating the Global Regulatory Landscape The successful translation of cell and gene therapies into the clinic requires careful navigation of the regulatory pathways during product development and commercialization. The US Cell Therapy Regulatory Toolkit has been developed to inform […]

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Editorial

Cell and gene therapies: improving and extending quality of life for seniors

Editorial

Chris Mason, Emily J Culme-Seymour & Geoff MacKay

Navigating the Regulatory Landscape


Editorial


Emily-C-SeymourChris-Mason

Cell and gene therapy represents a new paradigm in human healthcare with the ability to resolve unmet medical needs by directly targeting the underlying cause of disease. One of the major social drivers in the 21st century is to address the medical needs of the aging population. Today in the USA, UK, and Japan, people over the age of 65 make up 14%, 18% and 26% of the population, respectively [1]. However, over the coming decades these proportions will dramatically rise due to a combination of longer life spans and aging baby boomers. With increasing age comes increasing health challenges. Biological aging reduces the ability of organs, tissues and cells to respond effectively to everyday wear-and-tear, trauma, disease and cancer. “Once-and-done” cell and gene therapies with their potential to transform patients’ lives (and, in some cases, even provide a cure) are a major step-change from the traditional pharmaceutical “a-pill-a-day-for-life” approach, which at best only manages disease and related symptoms. So what do cell and gene therapies potentially offer seniors to enable a sustained quality of life? This editorial highlights cell and gene therapies that specifically target medical conditions associated with aging. Below are examples of significant causes of morbidity and mortality in seniors where cell and/or gene therapies are either already available in the clinic or in the clinical trials’ phase of development.

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Exploiting RNA activation for therapeutic applications

Editorial

Paul J Mintz, Vikash Reebye, Pål Sætrom, John J Rossi & Nagy A Habib

Navigating the Regulatory Landscape

Editorial

The next frontier in RNA biology is the advancement of RNA therapeutics and cellular reprogramming for unmet clinical needs. Since the discovery of post-transcriptional RNA interference (RNAi) by Fire and Mello, the RNA field has made tremendous progress, and small double-stranded RNAs (dsRNAs) play a pivotal role in our understanding of gene regulation. These small RNA molecules are known as non-coding RNAs (ncRNA) because they do not translate into biological proteins. The best classical examples and highly studied ncRNAs are microRNAs (miRNA) and small interfering RNAs (siRNA). They are essential in gene regulation through their ability to suppress target genes. Over the years, many other types of ncRNA have been identified such as long non-coding RNAs (lnc-RNA), Piwi-interacting RNA (piRNA), enhancer RNAs (eRNA), and extracellular RNAs (exRNA). This editorial will not cover these ncRNAs but only mention them in passing since they are already the subject of excellent published review articles. Rather, we will focus on another class of lesser known ncRNA called small/short activation RNAs (saRNA), or antigene RNAs (agRNA), due to their unique properties and controversial status. As the name suggests, these are short stretches of dsRNA molecules similar to siRNAs but with a unique ability to turn on genes rather than suppress them.

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Commercial Insights

Commercial Insight – September 2015

Commercial Insights

Alan Boyd, Mark Curtis & Rahul Sarugaser

Navigating the Regulatory Landscape


COMMERCIAL INSIGHT


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.

Cell therapy:

Competition in the development of chimeric antigen receptor (CAR) and T-cell receptor (TCR) products is aggressive, and has been a driver in M&A transactions in the space for the last 12 months as cell-based immunotherapy companies vie for auxiliary assets to enhance core platforms and capabilities. This past month, Aduro Biotech joined the ranks of Juno Therapeutics and Kite Pharma by acquiring Netherlands-based BioNovion for €29 million, a private company with a portfolio of antibody candidates and a discovery platform for development of novel CARs. In March of this year Kite Pharma purchased T Cell Factory, also located in the Netherlands, which gave the company a platform for discovery of novel TCRs. A few months later Juno followed suit with the purchase of Boston-based X-BODY, a company with a platform for the discovery of fully human antibodies. Whereas, historically, companies may have struck up research collaborations to access these platforms, large IPOs and private placements have endowed immunotherapy companies with the cash to bring expertise in-house. This is a trend we are also seeing on the manufacturing front, where Juno, Kite, and Adaptimmune are in the process of building-out GMP cell manufacturing capabilities

Gene therapy:

This month’s news stories have been all about fund raising and IPOs but it is noticeable that this is all focused on the USA, with even companies based outside of the USA turning to the USA for investment. The USA has, for a long time, been the place to go to raise money for life science companies but why are we not seeing more activity on the other side of the Atlantic? There have been a few IPOs over the past year in Europe but nothing like the numbers we have seen in the USA nor raising the same amounts of monies that have been raised there. It is difficult to know why – is it that Europeans are more risk averse or is it related to overall European economy? A question I think that should be addressed.

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Commercial Insight – August 2015

Commercial Insights

Alan Boyd, Mark Curtis & Rahul Sarugaser

Navigating the Regulatory Landscape


COMMERCIAL INSIGHT


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:

From the past months’ news stories it is clear that gene therapy companies and their products are generating a lot of interest, particularly in the investment arena. The IPO window has been open for some time now for companies developing advanced medicines and for the time being it looks like this is going to continue to happen. Big Pharma are also very interested in the sector now as evidenced by the deals being driven by them; this is a very different picture from as recent as 5 years ago. Finally, it is good that the sector is now having to consider the price of gene-based therapies. In my view this is a nice problem to have as a few years ago a discussion about price was way down the agenda, at least publically. Having open discussions about price has only come about due to the fact that gene-based products are getting closer to market and hopefully becoming available to patients – so not such a bad issue to be facing for many reasons.

Cell therapy:

The biotech industry is currently enjoying tech-like valuations for novel paradigms at early stages of development, including cell-based immunotherapy, ex vivo gene therapy, and antibodies targeting cancer stem cells (Stemcentrx, a private pre-clinical company with a $3 billion valuation). Either the innovation is real, or we are in the largest biotech bubble in history. Given the curative potential of these new paradigms and the robustness of platforms in industry, we would argue the former.

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Clinical Trial Insights
Regulatory Insight

Japan’s regulatory framework: seeking to provide impetus to the commercialization of regenerative medicine products

Spotlight Article

Regulatory Insight

Shintaro Sengoku, Mitsuya Sakurai, Yoshimi Yashiro

Navigating the Regulatory Landscape


REGULATORY INSIGHT


The time is right for innovation in the fields of regenerative medicine and cell therapy in Japan. Beginning with the enactment of the Promotion Act on Regenerative Medicines in 2013, the regulatory framework has undergone sweeping reforms, including the establishment of the Act on the Safety of Regenerative Medicines (or RMS Act) and the Pharmaceutical and Medical Device Act (or PMD Act, formerly the Pharmaceutical Affairs Act) in November 2014. In this article, industry trends and the social impact of regenerative medicine and cell therapies will be discussed in the context of these recent legal and regulatory framework changes.

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Japan’s regulatory gamble and what it means for the Industry

Spotlight Article

Regulatory Insight

Lee Buckler & Colin Lee Novick

Navigating the Regulatory Landscape


REGULATORY INSIGHT


The Follower Takes the Lead:  How Japan’s trifecta of economic policy, regulatory innovation, and an aging demographic may disrupt a global industry and the very future of medicine.

On November 20th 2014, the Japanese legislature passed the new Act on the Safety of Regenerative Medicine (ASRM) and the revised Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act (PMD Act) [1]. It is hard to make that sentence sound exciting, but what many outsiders have yet to fully appreciate is the tectonic shift in Japanese pharmaceutical regulatory policy this represents. Put another way, these two pieces of legislation lay the foundation for Japan to become a world leader in regenerative medicine and cellular therapy. The most interesting question for many is “why?” Why has a country synonymous with “risk aversion” and “following” taken such a drastic step away from the norm and more importantly, what does this mean for commercial players involved in the regenerative medicine/cellular therapy industries? To answer this, it is important to understand what the Japanese regenerative medicine and cellular therapy regulatory environment looked like prior to these laws and the dynamics that pushed Japan to change the status quo.

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Regulatory viewpoints on the development of advanced stem cell–based medicinal products in light of the first EU-approved stem cell product

Spotlight Article

Regulatory Insight

Egbert Flory, Paolo Gasparini, Veronika Jekerle, Tiina Palomäki, Patrick Celis, Tomáš Boráň, James W McBlane, John Joseph Borg, Jan Kyselovic, Metoda Lipnik–Stangelj, Toivo Maimets, Margarida Menezes-Ferreira, Guido Pante, Stefanie Prilla, Una Riekstina, Christian K Schneider, Asterios Tsiftsoglou and Paula Salmikangas

Navigating the Regulatory Landscape


REGULATORY INSIGHT


Recent years have seen a dramatic increase in research activity and investment into therapeutic applications of stem-cell-based medicinal products. In April 2015, the first stem cell product, ex vivo autologous corneal epithelial cells including stem cells (Holoclar®, Holostem Advanced Therapies), was granted a Marketing Authorization in the European Union (EU). This product is based on limbal stem cells and is intended for the treatment of limbal stem cell deficiency caused by ocular burns. Whilst only one stem cell product has been approved, there are many others being studied for various indications. Approximately 166 clinical trials are testing the use of adult stem cells in regeneration of lost or damaged tissue and in hematological or solid-organ malignancies in the EU. Embryonic stem cells and induced pluripotent stem cells are mainly being explored in nonclinical studies, although early-stage clinical studies with these pluripotent cells have also been reported. Unfortunately, there are also reports of patients travelling outside the EU for unregulated treatments with stem cells. In order to meet patient’s demands for authorized, safe and effective treatments and to foster innovation in this dynamic area, in 2011 the Committee for Advanced Therapies of the European Medicines Agency developed and published regulatory guidance with contributions from European and international experts. It has become clear that a multidisciplinary point of view has to be applied to stem-cell-based products in order to regulate them adequately, thus spanning the bridge from quality criteria and impact of starting materials, animal models, biodistribution and niche, to safety issues including tumorigenicity, and clinical aspects. In this article, we report on the regulatory and scientific framework for stem-cell-based products in Europe and how this can be successfully applied in the development and evaluation of novel stem-cell-based products.

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Conference Insight

ISCT 2015 Annual Meeting

Conference Insight

Fabio D'Agostino

Navigating the Regulatory Landscape


Conference Insight


vegas_isct1

Over 1200 delegates from around the world gathered at Caesars Palace, Las Vegas for the 21st ISCT Annual Meeting in May 2015. From eminent academic professors to Wall Street analysts, the field came together to discuss, share, debate and network across the three days. The notable heterogeneity of the audience reflected the need for people from different backgrounds to work together to deliver this new class of medicinal products in what is often referred to as the pre-competitive era of the cell therapy industry.

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Review

Advances and challenges of successful cell therapies for liver disease

Review

Reenam S Khan & Philip N Newsome

Navigating the Regulatory Landscape


Review


End-stage liver disease is a common cause of morbidity and mortality. Currently, the gold standard treatment is orthotopic liver transplant; however, the mismatch between organ demand and supply, and the risks associated with organ transplantation has led to a search for alternative therapies. Cellular therapies could potentially present such an alternative to transplant or bridge to transplant. The earliest work has involved transplantation of allogeneic hepatocytes, with modest benefits shown. Hepatic progenitor cells have also been used in experimental settings, but cellular engraftment is challenging. Use of embryonic stem cells and pluripotent stem cells to form hepatocyte-like cells has also been investigated. Various bone marrow cells have shown therapeutic potential, likely via paracrine effects on liver repair and regeneration. This review summarizes the key advances in cellular therapies for liver disease, and discusses the challenges that need to be overcome before these therapies can be translated into clinical practice.

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Expert Insight

FDA resources for cell and gene therapy product development

Spotlight Article

Expert Insight

Scott R Burger

Navigating the Regulatory Landscape


In the USA, the FDA Office of Cellular, Tissue, and Gene Therapies (OCTGT) is principally responsible for regulation of cell and gene therapy products. Based in FDA’s Center for Biologics Evaluation and Research, OCTGT’s perspective on regulating cell and gene therapy products reflects the growth and development of the field, as well as FDA experience reviewing these complex biologics. OCTGT guidance documents and the webinar series OCTGT Learn are invaluable resources for cell and gene therapy product development.

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