October 26, 2018 PAP-Q4-18-CL-003
The viral vector marketplace has moved in two significant directions over the last decade. First, the field has continued to optimize the five main vector systems — AAV, lentivirus, retrovirus, HSV and adenovirus — and to match them to cell and organ targets for efficient gene delivery. As the industry has moved from proof-of-concept first-in-human trials to establishing commercial demand, this has driven development of the manufacturing and analytical platforms and the capacity needed to launch these products commercially.
The approvals in 2017 shifted the focus onto market access and the challenges of bringing new treatments to market that have the potential to be curative with a single administration. It is clear that the need and the impact of a life-changing treatment will have to be carefully assessed by the innovators. Brammer Bio has continued to see high demand for vectors for use in vivo and ex vivo that are supporting potentially curative treatments for serious diseases.
From 2020 to 2022, there is expected to be a ramp-up in approvals leading to around 40 cell and gene therapy approvals by 2022, according to a study by MIT.1 In addition, most companies have several products in their pipelines that will follow an accelerated development path once their first products are approved.
Brammer is investing $200 million in doubling our clinical capacity for products and building over 20 suites for commercial manufacturing of vectors. To support the manufacturing capacity, we are expanding our process development and quality control laboratories. The first phase of commercial-ready capacity was built in 2017 and is operational today, with the remaining build-out being completed by mid-2019. Brammer is already assessing the supply needs beyond even this substantial capacity. By Q3 2019, Brammer will have 700 employees focused solely on providing viral vector products to multiple clients, an increase from its current staff of 500. The company has more than doubled its staff over the past year.
Brammer’s expansion has increased the number of clients that we can serve with early clinical supply. In addition, clients have brought mature products to Brammer to establish commercial manufacturing for them. A number of clients have chosen to lock-in dedicated commercial capacity to ensure that they have control over the scheduling and output of that capacity to meet their evolving launch plans and product pipelines.
Brammer’s vision is to be the leading contract development and manufacturing organization for viral vector supply to cell and gene therapy innovators. We are focused on delivering clinical and commercial vectors to our clients to enable their clinical development, launch and market supply of these products. We are solely focused on meeting their needs and staying ahead of the demand curve.
Brammer has a strong track record from executing over 100 projects over 12 years to supply clinical viral vectors to the industry, many for first-in-human use, and is seen as the technical leader. Over the next two years, we expect that to evolve into Brammer being the leading supplier of both clinical and commercial viral vectors to our clients.
This is still an evolving field despite the tremendous progress that has been made. Many innovators have expertise in product development and clinical design, and they value partnering with Brammer to develop robust, scalable and efficient processes. Due to the speed from first-in-human use to approval in potentially four years or less, they trust Brammer to deliver the launch and commercial quantities to meet the market needs.
For processes that rely on transient transfection, such as AAV and lentiviral vectors, the Pall iCELLis adherent cell culture platform is highly scalable and robust. An area that is making rapid progress is analytical technologies used to characterize the vector products.
Brammer is establishing commercial supply for a potential micro-dystrophin gene therapy product launch and other neuromuscular programs in Sarepta’s pipeline, through partnering with Sarepta on the process development. This leverages all of Brammer’s world-class development and manufacturing capabilities.
There will undoubtedly still be challenges in developing these products and establishing market access. However, we are well beyond establishing proof of concept for these treatments. The regulators have recognized the potential impact to human lives and acted to encourage product development and establishment. In 10 years, these therapies have the potential to have cured many monogenetic diseases and to be useful in tackling more complex genetic diseases, including through the use of gene editing. In 20 years, it is hard to imagine, but gene-based medicines could be considered the standard of care.
The primary challenges facing gene therapy are to confirm the effectiveness of the treatments: to show a sustainable, long-term correction, then to ensure access to patients globally for these therapies and to ensure that efficient, robust supply is in place so that all patients can be treated.
Mark Bamforth is the founder and President & CEO of Arranta Bio, which was established in May 2019. In 2015, Mr. Bamforth founded Brammer Bio, a viral vector CDMO supporting cell and gene therapy companies, which was acquired by Thermo Fisher Scientific in April 2019. In 2010, Mr. Bamforth founded Gallus BioPharmaceuticals, a CDMO supplying biopharmaceuticals. In September 2014, Gallus was sold to DPx Holdings B.V., which later became part of Thermo Fisher Scientific. He holds a bachelor’s degree in chemical engineering from Strathclyde University and an MBA from Henley Management College.