Periodic Reporting for period 1 - MUSCLEGUY (A novel muscle disorders 3D in vitro system for drug screening and validation.)
Berichtszeitraum: 2016-10-01 bis 2018-03-31
The innovation: As part of the ERC Consolidator project PhONICs, my group has generated a novel 3D in vitro model to study skeletal muscle disorders using human muscle cells. This model is the first to generate highly matured myofibers from precursor cells. Our novel 3D in vitro model recapitulates the most important physiological function of skeletal muscles (contraction). We had previously demonstrated that this system can be used as a model for human diseases by using primary mouse muscle cells, specifically for centronuclear myopathy (CNM) and myotubular myopathy. Our 3D model opens up new possibilities for personalised medicine and thus targeted therapeutic approaches.
The proposition: The objective of the MUSCLEGUY project was to develop a commercial product based on a unique in vitro muscle system that reflects in vivo muscle architecture. Other in vitro muscle systems do not mature sufficiently to mimic fully differentiated muscle cells. These lack key structural components that limit their use for muscle drug development. Using our in vitro technology, we envisioned to develop a high-throughput screening system for muscle disorders, a muscle cell culture kit or a platform.
The approach: The MUSCLEGUY project was tackled on a scientific and business front. Scientifically, the in vitro system required several enhancements such as increased robustness, transition to human cell source and contractility measurements. Commercially, a market research assessment, an IP strategy, and a competitive analysis were established.
The outcome: The work plan of `MUSCLEGUY´ consisted of four interlinked activities: technical proof of concept (A1), commercial proof of concept (A2), development of a patent portfolio and IP strategy (A3) and business development (A4).
(A1) Technical proof of concept: Due to technical challenges (part of the technical proof of concept), the maturation of human iPSCs into fully formed myofibers is not currently possible. This limits the applicability of our in vitro system for their use as a platform for high-throughput screening system for muscle disorders. Thus, we currently face challenges in demonstrating the technical proof of concept. Efforts are now being done to better differentiate hIPSCs into muscle cells using alternative methods.
(A2) Commercial proof of concept: We have performed an elaborate market analysis (see Annex I-III). Based on desk research (Annex I and II) and interviews with Key Opinion Leaders (Annex II), we have identified a clear unmet need for an in vitro system in the field of muscle disorders and we have evaluated two possible commercial products: maturation medium and mature muscle cells.
Future plans: Based on the new results from the performed experiments and the positive outcomes from the market analysis, we will further proceed on improving the maturation, scalability and reproducibility of our in vitro (human) muscle system.
The proposition: The objective of the MUSCLEGUY project was to develop a commercial product based on a unique in vitro muscle system that reflects in vivo muscle architecture. Other in vitro muscle systems do not mature sufficiently to mimic fully differentiated muscle cells. These lack key structural components that limit their use for muscle drug development. Using our in vitro technology, we envisioned to develop a high-throughput screening system for muscle disorders, a muscle cell culture kit or a platform.
The approach: The MUSCLEGUY project was tackled on a scientific and business front. Scientifically, the in vitro system required several enhancements such as increased robustness, transition to human cell source and contractility measurements. Commercially, a market research assessment, an IP strategy, and a competitive analysis were established.
The outcome: The work plan of `MUSCLEGUY´ consisted of four interlinked activities: technical proof of concept (A1), commercial proof of concept (A2), development of a patent portfolio and IP strategy (A3) and business development (A4).
(A1) Technical proof of concept: Due to technical challenges (part of the technical proof of concept), the maturation of human iPSCs into fully formed myofibers is not currently possible. This limits the applicability of our in vitro system for their use as a platform for high-throughput screening system for muscle disorders. Thus, we currently face challenges in demonstrating the technical proof of concept. Efforts are now being done to better differentiate hIPSCs into muscle cells using alternative methods.
(A2) Commercial proof of concept: We have performed an elaborate market analysis (see Annex I-III). Based on desk research (Annex I and II) and interviews with Key Opinion Leaders (Annex II), we have identified a clear unmet need for an in vitro system in the field of muscle disorders and we have evaluated two possible commercial products: maturation medium and mature muscle cells.
Future plans: Based on the new results from the performed experiments and the positive outcomes from the market analysis, we will further proceed on improving the maturation, scalability and reproducibility of our in vitro (human) muscle system.