Maryland, US headquartered company, Orgenesis, is championing a model that aims to bring down those costs – it works with partner hospitals throughout the commercialization process.
The company’s CGT platform, consisting of a pipeline of licensed cell and gene therapies, scientific expertise, customised processing systems, and an ecosystem of healthcare providers and research institutes, is designed to provide a pathway for groundbreaking autologous therapies to become commercially available on an industrial scale and at prices accessible to large populations.
Orgenesis’ business model is one focused on decentralization, enabling precision medicines to be prepared on-site at hospitals. “In this way, we can really expedite cell and gene therapy development,” said Orgenesis CEO, Vered Caplan.
With operations in the US, Europe, Israel and South Korea, Orgenesis has now created an international network of point of care (POCare) centers to serve patients directly in the hospital setting.
“Beyond the US, we have POCare centers in many countries in Europe such as Greece, the Netherlands, Belgium, Slovenia, Italy and Spain; we also have centers in Israel, in Korea and in India and we will be starting up soon in Dubai,” said the CEO.
The goal is to make gene and cell therapies feasible for large numbers of patients, said Caplan. “We used to work as a contract development and manufacturing organization (CDMO) but we sold that business to Catalent at the beginning of the year.”
The centralized processing and supply chain model only served to create a frustrating working environment, with plenty of constraints, said the Orgenesis lead.
“We realized very quickly that we couldn’t really ramp up to large scale relying on that kind of centralized model, particularly for autologous products, which represent most of the market today. It takes six months to train someone to work in a high-grade cleanroom – there is a lot of work and expense involved in that – and there is a limited number of patients that can be treated in such cleanrooms – the utilization rate is very low - it [centralized processing and supply] is a very inefficient and costly way to supply and to develop medicine – there is so much manual work involved,” she told BioPharma-Reporter.
The company had been working for a number of years, investing a huge amount of effort in developing a range of automation solutions to supplant those manual processes, as well as building its mobile CGT processing labs and units (OMPULs), she said.
“We had been fielding so many requests from hospitals that wanted to collaborate with us, asking us to make or scale up their CAR-T and other therapies. We realized that in order to get this done, we needed to take a decentralized approach and that we needed to provide a solution, not only for one hospital, but for every hospital that wanted these type of therapies; and we saw that such a model brings down the price of the therapy tremendously.”
A hospital gives Orgenesis a license to work on the therapy, on the processing; production of the final product is automated and supplied via an on-site point-of-care processing unit. Orgenesis then sets about democratizing the treatment, making it available to any hospital in its POCare network.
The company says the final customized, automated processing system it has developed, with the integrated specific therapy, solves a variety of processing and cost hurdles. It results in a lower required grade of cleanroom, it simplifies facility management requirements, it enables multi-batch processing per cleanroom, which means reduced technical staffing. Moreover, the localized processing eliminates the many logistical difficulties associated with traditional, centralized manufacturing and transport.
Overall, it is said to provide faster turnaround, increased safety, and improved quality control management on-site.
Uberization of the CGT space
Hospitals really want to supply CGTs, while patients are reading about such treatments and making inquiries of healthcare providers, she added.
“Ours is really a combined licensing and service model.
“We are like Uber. If you have a car, you want to make some extra revenue, you call up Uber and it gives you the network, the technology and all the operating procedures to be a taxi driver. That is very much what we do in terms of hospitals – we give them the ability to be biotech companies, because this is not the standard thing they do, they don’t want to take responsibility for cell and gene therapy – it is too much for them. They want to treat patients, but they want to have that local supply, so we give them the technology and the capabilities to do that. We give them regulatory support for clinical trials, we give them CRO support, we give them a network - so they can function and do what they need to do, which is to undertake research and treat patients.”
Orgenesis intends to leverage its network of regional partners to advance the development and commercialization of its therapeutic pipeline. Towards this end, it said its partners have committed to funding the clinical programs. In turn, the company typically grants its partners geographic rights in exchange for future royalties, and a partnership with Orgenesis to support the supply of the targeted therapies. Through this model, Orgenesis has already signed contracts, which it expect to generate over US$40M in revenue over the next three years, if fully realized.
On the therapeutic front, Orgenesis is focused on several key verticals, including immuno-oncology, anti-viral, and metabolic/auto-immune diseases.
It recently acquired Koligo Therapeutics, with the aim of leveraging Koligo’s 3D-V bioprinting technology across its POCare Platform. That technology, which utilizes 3D bioprinting and vascularization with autologous cells to create biodegradable and shelf-stable three-dimensional cell and tissue implants, is being developed for diabetes and pancreatitis, with longer term applications for neural, liver, and other cell/tissue transplants.
In February this year, Orgenesis announced that it has entered into a collaboration agreement with the John Hopkins University to utilize the POCare platform to develop and supply a variety of CGTs including cell-based immunotherapy technologies.
And the University of California, Davis (UC Davis) joined its POCare network in January. The collaboration will involve the scale up and integration of UC Davis’ lentiviral vector process.
“Today we are very much in validation mode. Most of the therapies in this space, and the ones we have licensed from the hospitals – I think we have about 25 today – are all at different stages of clinical development. Some have been used to treat patients but that has all been done under hospital exception.
“When we adopt a therapy into the network, we run it through the entire R&D, formal clinical and regulatory processes as [our goal] is a harmonized process, to have the same standard [in our closed systems] at our [POCare] centers, whether that is in Germany or Korea,” said the CEO.