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Novel human neurodegenerative disease specific 3D cell-models with applications in drug development and toxicity testing

Periodic Reporting for period 1 - BTech (Novel human neurodegenerative disease specific 3D cell-models with applications in drug development and toxicity testing)

Reporting period: 2018-02-01 to 2018-05-31

The field of neurodegenerative diseases is characterized by low success rates of new drugs that begin human testing and reach the marketplace (8% in comparison to 15% for drugs overall). This is due to the inadequacy of the current in vitro (2D cell culture) and in vivo (rodent) models that fail to capture the complexity of the human brain.
BTech has developed a new 3D in vitro Brain-on-a-Chip technology that can offer medium-throughput screening of drugs during pre-clinical testing that more accurately reflects the effects of drugs on humans than conventional 2D cell cultures and mouse models. The technology, which will be provided as a service by BTech, is based on microfluidics where cells are cultured in three dimensional hydrogel environments mimicking the conditions in vivo.

The company also offers ready-made midbrain organoids as products for customers to self-investigate the effect of drugs/toxins or for disease modelling. Midbrain organoids, or so called “mini-brains”, are 3D structures having different cell types spatial organised to recapitulate the human midbrain, which is the region affected in Parkinson’s disease. These 3D brain technologies mimic human neurodegenerative diseases more adequately than other standard methods and that reduce the need for animal testing.
We not only want to use these advanced models to provide customers with novel tools for their drug development campaigns, but also use the knowledge and experience gained during these services to develop drug candidates. These candidates are developed in a targeted manner based on literature investigations, licensing from academic partners, or own large scale screens. Particularly, our wide academic network allows us to stay current in the latest development in the field and to rapidly identify new potential candidate compounds.
First achievements –Two customers that test the Brain-on-a-Chip service, reproducible organoid generation
With regard to the service, BTech has achieved its first proof-of-concept (TRL 5) with an acknowledged compound. In addition, the BTech team was partnering with a big pharma company for a pilot project. From this project, the company has generated revenues of 60k€ and a follow-up project with a volume of 138k€ is currently discussed.
Concerning the mini-brains, BTech is able to generate these midbrain organoids with a good throughput in a reproducible manner. A first offer from a worldwide active distributer for cell culture models has been received.

Milestones – Quality, customization, and growth
The main targets on the service level, are to standardise the service delivery (establish robust and high-quality processes) and to advance the service by including additional parameters in the compound analysis (increased customisation). Furthermore, additional proof-of-concept with more tested compounds had been be achieved within the performance for two studies for the Juntendo University of Tokyo and the University of Miami.

On the product level, the main goals are to standardize the organoid generation protocol and to integrate automation for increasing the throughput (optimize a fast and reliable procedure for organoid generation), to develop an optimal freezing technique that will allow shipping intact and viable 3D structures and to demonstrate the usability of organoids for toxicity testing. Furthermore, increasing the complexity of the mode, e.g. by including vascularization and immune system activity, are aspired for the future.
Eventually, the goal of BTech as a company is to move to own lab facilities to be able to acquire new customers (scale-up and grow).
The main targets on the service level, had been achieved since we've been able to standardise the service delivery (establish robust and high-quality processes) and to advance the service by including additional parameters in the compound analysis (increased customisation). Furthermore, additional proof-of-concept with more tested compounds had been achieved.
On the product level, the main goals are to standardize the organoid generation protocol and to integrate automation for increasing the throughput (optimize a fast and reliable procedure for organoid generation), to develop an optimal freezing technique that will allow shipping intact and viable 3D structures and to demonstrate the usability of organoids for toxicity testing had been achieved as well. Furthermore we had been able to increasing the complexity of the mode, e.g. by starting the inclusion of vascularization and immune system activity, which are are aspired in the near future.
Finally, the goal of BTech as a company is to move to own lab facilities to be able to acquire new customers (scale-up and grow) had been achieved.
MINIBRAIN