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Gene Therapy a New Paradigm for Healthcare: An Interview With bluebird bio

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The biotechnology company bluebird bio develops gene therapies for severe genetic disorders and cancer.

The company is working to accelerate the delivery of gene therapy to patients because it believes that it provides the chance for individuals with severe genetic diseases to "live fully, regardless of what's written in their DNA". In 2019, bluebird bio's gene therapy Zynteglo™ received conditional marketing authorization in Europe for the treatment of patients with transfusion-dependent β-thalassaemia.

Technology Networks
recently spoke with Martin Butzal, Head of Medical Europe at bluebird bio, to learn more about the exciting developments and key trends in the gene therapy space.

Molly Campbell (MC): What were some of the major highlights for bluebird in 2019?

Martin Butzal (MB):
In June 2019 the European Commission (EC) granted bluebird bio’s gene therapy Zynteglo™ (autologous CD34+ cells encoding βA-T87Q-globin gene) conditional marketing authorization in Europe based on data from completed Phase I/II trials and ongoing Phase III trials. The treatment was approved for a subset of patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

We engaged in early dialogue with European Medicines Agency (EMA) for scientific advice, and the review, under the EMA's Priority Medicines (PRIME) scheme, took just 150 days. In a recent statement, the EMA regulators referenced a new article published in the journal Clinical Pharmacology & Therapeutics that describes these exchanges, using our gene therapy as an exemplary case. 1

In January this year the EMA published its human medicines highlights of 2019, an overview of its key recommendations in 2019 on the authorisation and safety monitoring of medicines for human use. The report identifies bluebird bio’s gene therapy as an important contribution to public health and representative of significant progress in its therapeutic areas.

MC: In bluebird bio's opinion, what key trends can we expect to see in the gene therapy space in 2020?

Although gene therapies are still a nascent technology, they present a new paradigm for healthcare, offering a one-time treatment that can address the underlying genetic cause of certain severe genetic diseases and cancer. We anticipate that the three techniques being studied for gene therapy – gene editing, gene addition and gene-based immunotherapy – will become more distinct and that the entire treatment journey will become more efficient.

For example, our therapy is delivered "ex vivo" using a patient’s own cells. Functional copies of a gene are added to address the underlying genetic cause of the disease – this is known as "gene addition". As part of the process the patient is given chemotherapy to make space in their bone marrow for the modified cells that are to be introduced. This is why we have entered into a research collaboration with Forty Seven on non-chemotherapy conditioning, with the aim of developing an approach that does not need chemotherapy in the future.

MC: In bluebird bio's opinion, what are the key challenges that currently exist when developing and testing gene therapies? How does bluebird hope to overcome such challenges?

A key challenge that we at bluebird have observed relates to the fact that we are breaking new ground. No company has successfully launched a large-scale gene therapy in Europe. In terms of clinical trials, the number of patients receiving cell and gene therapy treatment is relatively small compared to other therapeutic treatments, but that is inevitable. Additionally, gene therapy is expected to have a life-long effect, but long-term safety and efficacy data has yet to be collected.

In terms of production and manufacture, we see huge development efforts around the technology for gene therapy itself. As it is based on viral vector technologies and treatments are highly personalized, it is much more multifaceted than current drugs, and setting up manufacturing procedures that enable large-scale production is a next big step.

Lastly, the process of gaining reimbursement for a new potentially one-time treatment calls for new approaches to assessing value, given that gene therapy is totally different from conventional chronic treatments, administered differently and often supported by significantly less clinical trial data. Their value becomes evident over time and that’s why there is a pressing need for new approaches to patient access, that offer payers an affordable way to fund gene therapies. As gene therapy is a one-time treatment with expected lifelong effect it does not fit with the current funding methods for medicines. If we are going to get gene therapies to patients, health systems must be prepared to work in partnership with industry to remove possible barriers. This means moving towards an approach that’s reflective of the precise nature of advanced therapy medicinal products (ATMPs). We feel that the pharma and biotech industry has a duty to underwrite some of this in the type of funding models that are proposed. We believe that Outcome Based Payments Over Time are one of the ways this can be achieved.


 1. EMA . 2019. Regulators’ advice can make a difference for faster patient access to highly innovative therapies [ONLINE] Available at: https://www.ema.europa.eu/en/news/regulators-advice-can-make-difference-faster-patient-access-highly-innovative-therapies [Accessed December 2019]

Martin Butzal, Head of Medical Europe at bluebirdbio, was speaking to Molly Campbell, Science Writer, Technology Networks.