Below you can find an interactive table where the WP specific objectives and deliverables are shown. Please see image below for explanation.
| Work Package | Objectives | Timeline |
|---|---|---|
| WP1 | - Develop quasi-vivo multilineage human skeletal muscle for disease modelling. - Bioengineer next-generation scalable neuromuscular models - Identify and validate disease phenotypes in quasi-vivo neuromuscular models. | Months 1-24 |
| WP2 | - Explore rational design and directed evolution strategies to engineer next generation AAV vectors. - Explore transcriptional targeting for tissue specificity. | Months 1-24 |
| WP3 | - Develop gene and base editing approaches for genetic diseases like DMD, L-CMD and UCMD - Use novel sequencing tools analyse on-target and genome wide off-target alterations. | Months 1-30 |
| WP4 | - Validation of therapeutic approaches and next generation viral vectors in the quasi-vivo disease specific bioengineered muscles - Identify optimal delivery of validated viral vectors into engineered muscles. - Assess amelioration of in vitro phenotypic readouts upon administration of candidate vectors | Months 18-36 |
| WP5 | - Assess fidelity and response to genetic therapies of muscle-on-chip models compared to pre-clinical animal models for DMD, L-CMD; UCMD and XLCNM. - Assess vector toxicity and efficacy under a humanized setup by implanting engineered muscles in immunodeficient mice. | Months 18-42 |
| WP6 | - Preclinical immunology and genotoxicity of newly developed vectors | Months 18-42 |
| WP7 | - Develop scalable GMP-compatible processes for novel vectors and manufacture preclinically validated viral vectors. - Qualify organ-on-chip devices and achieve readiness for commercialisation. - Evaluate the safety and biodistribution of clinical candidates in NHPs | Months 36-48 |
| WP8 | - Develop and implement communication and dissemination plan and tools (project website, educational activities, Stakeholder engagement and clustering activities) - Generate the Data Management Plan and dataset collection in the DDF Data Repository - Design pilot clinical trials | Months 1-48 |
| WP9 | - Define, monitor, and manage IP and IPR strategy. - Commence exploitation activities for future market launch. - Organise a workshop for potential commercial and other interested stakeholders. - Commercialisation of exploitable methodology and tools - Monitor and advise on all ethical issues. | Months 1-48 |
| WP10 | - To provide effective administration, management, and governance of MAGIC - To incorporate duties covering project monitoring, reporting, financial and contractual administration - To assure proper communication within the consortium - To ensure that gender and ethical matters are met. | Months 1-48 |
| WP11 | - To develop neuromuscular disease models of DMD, XLCNM and L-CMD using Myofiber-on-chip and Myochip platform. - To carry out disease phenotyping using their novel disease platforms. - To model vector delivery using novel Myochip platforms. | Months 12-48 |