Periodic Reporting for period 1 - CancerQR (Next generation cancer therapies targeting cancer networks)
Período documentado: 2019-01-01 hasta 2019-06-30
A persistent challenge in cancer therapeutics is the failure to reach long-term therapeutic effects due to tumor heterogeneity, severe side effects, and drug resistance. At the same time, strides in biological data acquisition, increase in computational power, and progress in machine learning and artificial intelligence enable an unprecedented insight into tumor biology and disease progression in individual patients. However, this knowledge translates at best into personalized adjustments to the patients’ treatment regimen, with the physicians choosing among available options that are not ideal and are not themselves personalized. Pattern BioSciences’ suite of platform technologies allows for the first time harnessing the detailed knowledge of tumor biology and patient heterogeneity to develop therapies that are tailored precisely to specific tumor types.
Clearly, better ways to treat cancer are urgently needed. In addition, the convergence of a number of other developments generates a unique opportunity at this moment of time. The exponential grows in biological data collection and data processing capabilities, mentioned above, improve our understanding of disease mechanism. Strides in advanced therapies including gene and cell therapy have, for the first time, led to a number of approved therapies, growth in accompanying manufacturing infrastructure, and regulatory acceptance. Yet these exciting advances are not yet fully exploited in a coherent fashion due to the disconnect between the data, the therapeutic leads, and the clinical practice. Approaches developed by Pattern BioSciences are key to enable broad exploitation of the already-built infrastructures, and therefore increase the return on the already-made investments by private sector and governments.
The objectives of the Action were a feasibility study to assess the parameters and boundary conditions for continuing commercialization of the platform technologies, engaging potential Pharma and Biotech partners on one hand, and technology and clinical partners on the other. We sought detailed feedback on our technical approach and business strategy, as well as IP management. The conclusion of the action was a broad validation of the business strategy and the technology approach, as well as fine-tuning of various aspects on both the business and the technical side, to enable robust growth of the platform and the clinical pipeline.
Clearly, better ways to treat cancer are urgently needed. In addition, the convergence of a number of other developments generates a unique opportunity at this moment of time. The exponential grows in biological data collection and data processing capabilities, mentioned above, improve our understanding of disease mechanism. Strides in advanced therapies including gene and cell therapy have, for the first time, led to a number of approved therapies, growth in accompanying manufacturing infrastructure, and regulatory acceptance. Yet these exciting advances are not yet fully exploited in a coherent fashion due to the disconnect between the data, the therapeutic leads, and the clinical practice. Approaches developed by Pattern BioSciences are key to enable broad exploitation of the already-built infrastructures, and therefore increase the return on the already-made investments by private sector and governments.
The objectives of the Action were a feasibility study to assess the parameters and boundary conditions for continuing commercialization of the platform technologies, engaging potential Pharma and Biotech partners on one hand, and technology and clinical partners on the other. We sought detailed feedback on our technical approach and business strategy, as well as IP management. The conclusion of the action was a broad validation of the business strategy and the technology approach, as well as fine-tuning of various aspects on both the business and the technical side, to enable robust growth of the platform and the clinical pipeline.
The work in this project involved feasibility study and it covered multiple aspects of this effort. In particular, we focused on industrial partner search and market study; building relationships with clinical partners; connecting with service providers along the value chain, and exploring possibilities for mutually-beneficial partnerships in the areas of platform development and consolidation; intellectual property strategy and freedom to operate analysis; and initial interactions with regulators to define our non-clinical study plans.
The main results of the action were as follows:
(1) potential pharma and biotech partners were engaged to discuss the technology platform and its utilization for cancer as well as non-cancer indications. As a result of these discussions, further technical milestones were defined in both classes of indications, and those milestones were incorporated in our development plans
(2) clinical partners, in particular oncology departments, were approached and presented with an overview of our approach. The approach was assessed as promising and groundbreaking, and follow up interactions and collaborations ensued. The solid link to the clinical oncologists early in the development process will ensure the clinical relevance of the pipeline.
(3) Regulators were engaged in early scientific discussion, and extensive feedback was provided as a result. On this basis, detailed non-clinical development plans were formulated. Importantly, the regulators appreciated the potential of the tailored cancer gene therapies
(4) Technology partners and service providers were engaged in discussions and in-depth exchanges; as a result, Pattern BioSciences mapped the partners it would need to scale up its technology platform and accelerate the pace of preclinical development
(5) The IP strategy was worked out and the freedom to operate analysis was undertaken. No major risks were identified.
The results as a whole impacted the business, technology and development strategy of Pattern BioSciences by aligning it with the market need, external constraints, and positioning it against the competition.
The main results of the action were as follows:
(1) potential pharma and biotech partners were engaged to discuss the technology platform and its utilization for cancer as well as non-cancer indications. As a result of these discussions, further technical milestones were defined in both classes of indications, and those milestones were incorporated in our development plans
(2) clinical partners, in particular oncology departments, were approached and presented with an overview of our approach. The approach was assessed as promising and groundbreaking, and follow up interactions and collaborations ensued. The solid link to the clinical oncologists early in the development process will ensure the clinical relevance of the pipeline.
(3) Regulators were engaged in early scientific discussion, and extensive feedback was provided as a result. On this basis, detailed non-clinical development plans were formulated. Importantly, the regulators appreciated the potential of the tailored cancer gene therapies
(4) Technology partners and service providers were engaged in discussions and in-depth exchanges; as a result, Pattern BioSciences mapped the partners it would need to scale up its technology platform and accelerate the pace of preclinical development
(5) The IP strategy was worked out and the freedom to operate analysis was undertaken. No major risks were identified.
The results as a whole impacted the business, technology and development strategy of Pattern BioSciences by aligning it with the market need, external constraints, and positioning it against the competition.
The current project was focused on the feasibility study, and thus the results are mainly in the domain of business strategy, improved understanding of the market, regulatory environment, and the IP situation and future strategy. The main conclusion of the study, namely the broad validation of the initial premises behind the company strategy and their refinement to reflect the insight gained during the study, will enable Pattern BioSciences to continue to develop as a cutting edge biotechnology start up that uses molecular profiling data to tailor rationally-engineered therapeutic candidates for cancer and other diseases amenable for gene therapy approaches. Thus, the immediate socio-economic impact of the Project is the continuation of Pattern BioSciences effort to establish itself as a leading participant in the next wave of advanced therapies integrating big data, machine learning, synthetic biology and sophisticated, predictive in vitro and in vivo preclinical models for successful translation to human patients. In the context of European biotechnology industry, success of Pattern BioSciences will be instrumental in strengthening the European position in the advanced therapy space.