Project Team

UCL, has extensive experience in managing EU funded projects under FP4, FP5, FP6, FP7, Horizon 2020 and Horizon Europe and will be supporting the coordinating institution (INSERM) with the successful delivery of the MAGIC project through its European Research and Innovation Office, a unit within Research and Innovation Services. UCL is currently involved in more than 1400 EU-funded projects, of which more than 720 are funded through Horizon 2020 and 145 have been awarded in the frame of the Horizon Europe programme.  UCL was the top performing university in the first year of Horizon 2020 and 7th institution overall in EU collaborative research (#3 in the UK).

Based on a research strategy that is oriented around a series of ambitious “Grand Challenges”, including ‘Human wellbeing’, UCL nurtures thriving and engaged communities of world-leading academics across the entire research and innovation spectrum, from arts and humanities to the basic and applied sciences and healthcare. Complementing its leading research and innovation portfolio, UCL is internationally recognised as a centre of excellence for science communication.

Role

Prof Tedesco’s group at both University College London and The Francis Crick Institute lead the MAGIC consortium. They are involved in multiple work packages, with key input on developing advanced disease modelling platforms for the target diseases of the project. As pioneers of in-vitro modelling of muscular dystrophies using human induced pluripotent stem cells (iPSCs) and bioengineered muscles, they have expertise in developing isogenic, multilineage 3D muscles using primary and iPSC-derived cells. They will team with other partners in the consortium to develop stimulable, human muscle-on-chip devices for multiple disorders. Thereafter, they will investigate novel therapeutic strategies on the 3D bioengineered disease muscles using innovative viral vectors, CRISPR/Cas9 genome editing tools and therapeutic transgenes.

Team

The Tedesco laboratory studies skeletal muscle regeneration, focusing on the development of novel therapies for incurable neuromuscular disorders of childhood. They work pioneered the use of cutting-edge technologies such as human induced pluripotent stem (iPS) cells, artificial chromosomes and tissue engineering for advanced disease modelling and gene/cell therapies of muscle diseases. Current projects investigate iPS cell-derived myogenesis for complex neuromuscular disease and therapy modelling, as well as the use of small molecules to improve myogenic cell delivery. The overall goal of the Tedesco laboratory is the translation of the aforementioned regenerative strategies into novel therapies to improve outcomes for children with neuromuscular disorders.

INSERM Unit 951 is hosted by Genethon, a non-profit organization created in 1990 by the French association AFM-Telethon. Dedicated to the fight against rare diseases, Genethon has developed unique expertise in the design and development of gene therapy products, from laboratory research to clinical trials. Its work has resulted in several treatments at the clinical trial stage or already on the market that are changing the lives of patients and their families. With its 200 scientists and professionals, Genethon pursues its mission: to develop treatments that change the lives of patients suffering from rare genetic diseases and to eradicate these pathologies.

Role

Our group will contribute to the Magic grant at multiple levels:

• By developing novel genome editing strategies to treat Duchenne muscular dystrophy and optimizing editing tools for in vivo
• By identifying and creating novel tissue specific promoters for efficient and specific skeletal muscle and heart tissue expression.
• By studying the adaptive immune response against the new viral vectors and transgenes, its impact on treatment efficacy as well as by establishing appropriate immunomodulation.

Team

The team is composed by three researchers:

Prof Mario Amendola
Director
Therapeutic Genome Editing Group

Dr Giuseppe Ronzitti
Director
Immunology and Liver Diseases Group

Dr David-Alexandre Gross
INSERM Senior Researcher
Immunology and Liver Diseases Group

 

UPEC offers personalised support for all those who want to succeed, through initial training courses, accreditation of prior learning and continuing education, as well as apprenticeships and initiatives to promote entrepreneurship.

With its 32 laboratories and 1,650 teaching and research staff, UPEC has a very large pool of experts who talk about their research, publish and offer their analyses.

Role

Prof Fred Relaix will lead WP5, focusing on in vitro preclinical assessment. He is also participating in WP3. He is a leader in muscle stem cells and developmental biology, combining basic research in myogenesis and adult muscle regeneration with preclinical modelling and development of innovative therapies. He leads a large academic research team with experts in biological muscle research, medical doctors, and veterinary experts. His team includes also a renowned large animal facility and has close links with clinical teams at the Mondor Hospital.

Team

Our team is part of the neuroscience department of our research center, the Mondor Biomedical Research Institute (14 independent teams, around 600 researchers) It is the largest team on-site (team biology of Neuromuscular System), gathering >110 persons working both on fundamental and translation research in skeletal muscle stem cells, muscle biology, pathophysiological evaluations, and therapies of neuromuscular disorders. Our research team capitalises on a unique structure that combines a strong background in fundamental muscle and stem cell biology with a continuum of expertise in translational research, preclinical modelling and clinical research. Our research project therefore combines fundamental research, muscle pathology, animal modelling, cohort studies and therapeutic trials. Our internationally recognised expertise in these areas is combined with our privileged access to original preclinical animal models as well as human tissues and cell cultures.

 

The 10 faculty within the Division of Molecular Cardiovascular Biology performs basic heart and skeletal muscle research. The faculty have published ground-breaking research and routinely collaborate with our clinical and translationally minded faculty to pioneer new treatments for disease and clinical trials. A deep collaboration among the brightest biomedical science minds and the most talented pediatric clinicians ensures that The Cincinnati Children’s Research Foundation continues to be one of the most significant sources of scientific discovery and innovation worldwide.

Role

The Millay laboratory will optimize lentiviruses pseudotyped with the skeletal muscle-specific membrane fusogens Myomaker and Myomerger for gene delivery in vivo. By hijacking the fusion machinery this approach will provide the first muscle-specific LV for systemic delivery. These lentiviruses will be applied to 3D, bioengineered muscles and a rat model of Duchenne muscular dystrophy.

Team

Research in the Millay laboratory aims to identify the factors and biochemical mechanisms that govern muscle stem cell fusion and then ultimately translate that information to augment pathological conditions such as muscular dystrophy. We discovered the muscle-specific proteins (Myomaker and Myomerger) that are necessary and sufficient for myoblast fusion. We have a wide-range of projects in the lab, funded by NIH and industry sources, that encompass basic and translational biology related to the muscle fusogens and the consequences of fusion, namely formation of a multinucleated syncytium that impacts muscle development, adaptations, and disease. Specifically related to MAGIC, Sajedah Hindi (postdoctoral fellow in the lab) designed a strategy to engineer Myomaker and Myomerger on the surface of lentiviruses and showed that these are efficacious in mice. Sajedah and Molly Duszynski (Research Assistant) will continue to work on the lentiviruses to achieve the goals of MAGIC.

From research that underpinned discovery of the structure of DNA, to developing life-changing therapies and making maths education available to underrepresented groups, we continue to have a transformational impact on society. Fourteen people from King’s and its associated institutions have been awarded the Nobel Prize. King’s has more than 33,000 students from over 190 countries and £273m annual research income, being 6th in the world for research ‘power’ (2021 Research Excellence Framework) and ranked 35th in the world (Times Higher Education World Rankings 2023).

Role

The Zammit group will use accurate humanised muscle models for muscular dystrophies to assess safety and efficacy of novel gene therapies based on adeno-associated and lentiviral vectors specific to muscle cells, targeting diseases including Duchenne muscular dystrophy (DMD), X-linked centronuclear myopathy (XLCNM), and LMNA- and COL6-related congenital muscular dystrophies (CMDs).

The Serio lab will develop advanced bioengineered platforms to establish tissue engineering pipelines with different functionalities for both analysis and stimulation of skeletal muscle constructs in vitro. These novel engineered approaches will be fundamental to test the proposed viral vector gene therapy strategies for musculoskeletal disorders.

Team

The Zammit group investigates stem cell function in health and disease. The main theme of the Zammit group currently include examining the transcriptional and signalling control of satellite cell activation and cell fate choice, and studying pathomechanisms and potential therapies for muscular dystrophies including Fascioscapulohumeral muscular dystrophy and Emery-Dreifuss muscular dystrophy, and muscle-related cancers such as rhabdomyosarcoma.

The Serio Lab is a interdisciplinary research group with expertise in bioengineering, tissue modelling approaches, 3D organoids, biophotonics and biological engineering. Projects in the lab are focused on creating novel in vitro platforms to model different aspects of the neural and skeletal system in health and disease.

Maynooth University provides a high-quality educational experience to over 15,000 students on a campus with 18^th century roots and 21^st century dynamism. It is now acknowledged to be one of the leading young universities in the world, and in 2022 ranked # 1 in Ireland in the latest Times Higher Education (THE) Best Young University Rankings. Maynooth University is home to the Kathleen Lonsdale Institute for Human Health Research.

Role

Maynooth University and the Kathleen Lonsdale Institute for Human Health Research has a strong reputation for Immunology research. Maynooth University’s Professor Karen English has extensive expertise in investigating the immune response to advanced therapeutic medicinal products. Prof English will lead WP6 – Preclinical immunology and genotoxicity assessment of newly developed vectors and platforms.

Team

Prof English leads the Cellular Immunology group at Maynooth University consisting of four PhD students and three postdocs and one research assistant. Within the magic consortium, one postdoc and one research assistant will be involved in the project. The Group are actively working to understand how the immune system recognises and responds to advanced therapeutic medicinal products (ATMPs) such as cell and gene therapy. The group are also investigating ways to reduce the immune response to ATMPs or to induce tolerance.

Bi/ond Solutions engineers microfluidic chips (Organ-on-Chips) that recreate human physiology and pathology, with the aim of developing new drugs that better represent patients’ genetic diversity. Our unique platform includes electrophysiology functionality, enabling stimulation and monitoring of muscle contractions. This technology is crucial for patients with rare genetic diseases, as it facilitates the development of tailored treatments. By studying organs on our platform, researchers gain insights into disease mechanisms and test the effects of drugs on human-derived tissues.

Role

Bi/ond will empower the consortium partners by providing its expertise in developing intricate and dynamic skeletal muscle models combined with extensive knowledge in large-scale microfabrication, microfluidics and sensors integration.

Bi/ond will collaborate with the consortium to tailor its technological platform for diseased skeletal muscle models and pharmacokinetics/pharmacodynamics (Pk/Pd) assays, providing support through their protocols and engineering experience.

“We aim at revolutionizing muscular dystrophy treatment through the MAGIC Project, harnessing the power of Bi/ond’s innovative technology. By uniting with esteemed researchers and partners worldwide, we will do our best to bring hope and transformation to the lives of patients who cannot wait any longer.“
— Cinzia Silvestri, CEO of Bi/ond.

Team

The main people involved in the MAGIC project will be Mitchell Han – Senior Scientist, Cinzia Silvestri – CEO and founder, and Nikolas Gaio – CTO and founder.

Our team’s mission is to revolutionize medicine by embracing individual uniqueness.

With our diverse, multidisciplinary team of 12 professionals from 6 nationalities, we combine deep engineering expertise, biological knowledge, and business acumen. Together, we are turning personalized medicine into a reality, using our collective skills to make a lasting impact.

Our founders have over 24 years of combined experience in applying electronics to biology, with two patents to their name. We take pride in counting amongst our customers two top European hospitals and several prestigious academic Centers of Excellence.

VIVEbiotech owns the worldwide licensed “lentisoma” technology (WO 2015/078999 A1), a non-integrative episomal stable lentiviral vector.

Currently, VIVEbiotech is working on +40 international projects (for +20 different worldwide companies) who employ lentiviral vectors in different applications (e.g. immuno-oncology, rare diseases…) both ex-vivo and in-vivo.

VIVEbiotech’s track record of +100 batches in a continuously optimized and specific lentivirus production platform adds value to the MAGIC consortium and is a guarantee for high-quality muscle-specific lentiviral vector production.

Role

VIVEbiotech as a GMP CDMO fully specialized in lentiviral vectors will contribute to the development and manufacture of muscle-specific lentiviral vectors.

VIVEbiotech will be involved in early-stage development of the lentivector technology to ensure that the main steps of the process are designed in a way that can be later translated into GMP manufacturing. VIVEbiotech will later on produce the optimized lentiviral vector candidate at a large scale following a GMP-like process with the aim of providing the lentiviral vectors for preclinical studies in NHPs.

Linked to the high innovative specific features of the needed vector, VIVEbiotech will also focus on the optimization of production and purification conditions prior to manufacturing in reactors to ensure that the final lentiviral vector product process has a high titer and is pure. Thus, VIVEbiotech will contribute to establish a very cost-effective, scalable and regulatory compliant process for muscle-specific lentiviral vector manufacture.

Team

In the development of this project, 4 VIVEbiotech groups will be mainly involved:

• The Customer Development Services branch of the Technological Innovation Department will carry out the early optimization of the lentiviral vector to ensure that it can subsequently be manufactured under GMP conditions.
• The Process Development team belonging to the Operations Department will finetune the process focusing on the purification process aiming to obtain the purest possible lentivector.
• The Manufacturing team that is also part of the Operations Department will be the one taking the optimized conditions and bring the process to large scale to manufacture the engineering batches for the pre-clinical trial in NHPs.
• The Project Management department will coordinate the tasks within VIVEbiotech as well as maintain active and fluid communication with the rest of the consortium.

ReiThera has extensive expertise in developing scalable processes for viral-vector manufacturing and a consolidated experience in GMP production of Adeno-Associated Vector (AAVx), Lentivirus, Adeno Viral vector (AdV), Modified Vaccinia Ankara and Herpes Simplex Vector.

The company’s core manufacturing capacity is based in a state-of-the-art facility, which includes stirred-tank bioreactors at scales of 50L, 200L, 1000L, and 2000L, as well as fixed-bed bioreactors for cell growth in adherence. The GMP facility also comprises a filling suite and quality control laboratories.

ReiThera’s headquarters, R&D laboratories, and GMP facilities are located in Rome, Italy.

Role

ReiThera will devote its internal Process Development (PD) laboratory to set up in small scale both Upstream Process (USP) and Downstream Process (DSP) conditions for GMP-compatible purification of an AAV candidate, derived from one of the rational design approaches pursued in Work Package 2 for highly specific retargeting of AAV to cell types expressing specific receptors. Specifically, ReiThera will concentrate on AAV vector candidate(s) retargeted through Spy-Tag/Spy-Catcher coupling technology.

Following the initial PD study, ReiThera will generate the preclinical AAV batch needed for the safety and biodistribution study in NHP; the material will be carefully characterized in terms of physical and infectious titer, and impurities.

Team

The team that ReiThera will devote to MAGIC activities has an extensive experience in developing technologies and processes supporting the production and purification of a large number of viruses, including AAV, which are offered with varying degree of purity depending on the need. The team is composed of PhD-level molecular and cellular biologists and biochemists, which have already successfully generated AAV material used in clinical trials and is able to operate on different scale bioreactors, AKTA chromatographic system, Clarification/Tangential Flow Filtration devices and to design and setup tailored analytical methods suitable to qualification/validation.

 DINAMIQS provides a comprehensive and integrated range of services:

• AAV engineering
• Process development
• AAV manufacturing
• AAV vector characterization and quality control

Our facilities are located in Schlieren-Zurich Bio-Technopark, Switzerland: A fully operational state-of-the-art R&D facility dedicated to process development, manufacturing up to 50L scale and analytical testing. Our GMP manufacturing facility including two 500L USP-DSP suites with Fill & Finish capacity will open in Q1 2025.

Our manufacturing approaches provide high quality vectors. From a very early stage, we use single-use materials and fully GMP compatible process de-risking and accelerating time to the clinic.

Role

DINAMIQS will be an associated partner in 5 work packages of the project.

DINAMIQS will coordinate the work of our 3 SME partners in WP7 to get readiness of technologies for clinical application and/or industrialization/commercialization.

As part of WP2 (Developing novel tissue-specific vectors for neuromuscular genetic therapies). DINAMIQS will perform quality testing of novel capsid AAV vectors that will then be evaluated in vivo, and will generate high titer stocks in bioreactors when needed.

As part of WP7 (Bioprocessing and advanced pre-clinical translation), DINAMIQS will perform process development activities at bioreactor scale to deliver a GMP-compatible process, and manufacture 2 AAV batches providing the material for pre-clinical studies.

Team

Our team is led by Dr. Eduard Ayuso, one of the world’s leading experts in viral vector manufacturing and quality control. Dr. Ayuso has extensive experience in multiple EU-funded networks.

Our team brings decades of  process development, adaptation of processes to large-scale bioreactor systems and manufacturing expertise on a variety of AAV serotypes, including novel engineered capsids, to customize  programs and fulfill in vitro and in vivo needs.

MAGIC will benefit from a designated project manager and a cross-functional team of technical and scientific experts with defined roles to effectively meet the objectives of the different work packages in which we are involved.

We share expert advice and support so people can live well; fund groundbreaking research to better understand the different conditions and to lead to new treatments; work with the NHS towards universal access to specialist health; and campaign for people’s rights, better understanding, accessibility, and access to treatments.

We support people with muscle weakening and wasting conditions through every stage of their life. From the point of diagnosis to living the best life possible.

Role

Together with our fellow MAGIC charities, Muscular Dystrophy UK will disseminate the work of MAGIC to people living with neuromuscular conditions, their families and the public. Importantly, we will make sure that our communities and the public can find out about the goals, progress and outcomes of the research. We will use our website, social media, newsletters, webinars, press releases and other channels to share information in plain and simple language. MDUK is delighted to be a part of the MAGIC consortium because innovation and partnership underpins all that we do so that research is given every chance to succeed.

Team

Dr Andrea Gubas is Muscular Dystrophy UK’s Research Communications Officer. She has been with the charity since spring 2022. She works to present the outcomes of research with the community through various channels, shares stories about and from MDUK’s researchers, and provides updates on clinical studies relevant to people living with neuromuscular conditions.

Dr Kate Adcock is MDUK’s Director of Research and Innovation. She has been with the charity since summer 2018. Part of her role is to oversee MDUK’s investment in high-quality research to further our understanding of conditions, develop effective treatments, help people to live well and inform better care. This is because every day Muscular Dystrophy UK is working towards a future with effective treatments and ultimately cures for all muscle-wasting and associated neuromuscular conditions.

PP’s main goals are to support the families; to promote and fund scientific research; to develop a cooperative network. Over time, the association has grown and has equipped itself with an organizational structure and a staff. Parents and, nowadays, patients also play a central role at the decision-making level.

PP organizes every year an International Conference on Duchenne and Becker, that started off in 1999 as a meeting for Italian parents and became in time an international reference event, involving around 700 people every year from over 30 countries.

PP leads the Italian DMD/BMD Patient Registry.

Role

PP is involved in Work Package 8 (WP8) – Dissemination, communication and knowledge management. More specifically, PP’s role in the project is the dissemination of MAGIC findings to the public and patient communities in collaboration with other patient advocacy groups. All partners, led by DDF, will reach out to European and global charities and other target audiences to communicate the goals and progress of MAGIC through their websites, annual conferences, leaflets, presentations, social media, newsletters and press releases. Our main activity will be presenting MAGIC at our International Conference on Duchenne and Becker muscular dystrophy, held every year in February in Rome, Italy.

Team

Parent Project’s scientific office manages all the activities related to supporting research and disseminating scientific information to patients, families and the outside world. At the same time and in the same field, the area curates the Italian DMD/BMD Patient Registry, collecting demographic, genetic and clinical information from patients. As part of the Treat-NMD network, the registry contributes to the coordination and the harmonization of neuromuscular disease research.

The communication office’s activities are related to promoting the external visibility of the association through traditional and digital media and to all the communication within the association. The area, in synergy with the scientific and the psychosocial offices, contributes to disseminate information on the pathology, including the therapeutic strategies and the clinical management and to promote the projects where the association is involved.

By collecting and analyzing this data, the Duchenne Data Foundation facilitates collaboration among researchers, clinicians, and industry partners to accelerate the development of innovative treatments and therapies for DMD. Through their efforts, the organization plays a pivotal role in enhancing understanding, advancing research, and ultimately improving the lives of those affected by dystrophinopathies.

Role

Within the MAGIC consortium, Duchenne Data Foundation (DDF) leads WP8, dedicated to dissemination, communication, and knowledge management. Together with the support of all partners, DDF communicates project goals, progress, and results through various channels, including a project website, promotional material, international conferences, press releases, and scientific publications. Additionally, DDF coordinates data management, including secure storage, curation, and exchange of MAGIC data among partners. They ensure publication of new data and implement FAIR principles for improved accessibility. The DDF facilitates statistical analysis by providing access to the MAGIC data in a suitable format.

Team

The DDF MAGIC team consists of Dr George Paliouras (DDF Chairman), Dr Paraskevi Sakellariou (Research Coordinator) and Sergiu Siminiuc (Technical Officer) who focus on data management and knowledge exploitation. Subsequently, Suzie-Ann Bakker (Communications Coordinator) is liaising with partners to ensure proper project communication and dissemination.

Cure CMD is also the managing partner of the Congenital Muscle Disease International Registry (CMDIR; https://www.cmdir.org/) that collects patient-reported, health records-curated data from individuals affected with congenital-onset muscle conditions. Registrants originate from 72 countries and represent 35 muscle disease subtypes caused by mutations in one of 135 genes.

Role

Cure CMD is involved in Work Package 8 (WP8): “Dissemination, Communication and Knowledge Management” in collaboration with other patient advocacy groups. More specifically, Cure CMD’s role in the project is the dissemination of MAGIC findings to the CMD community stakeholders, with special focus on the affected individuals, their families, and caregivers. Cure CMD will reach out to the global community to communicate the goals and progress of MAGIC through its website, conferences, leaflets, presentations, social media, newsletters and press releases.

Team

Dr. Gustavo Dziewczapolski is Cure CMD’s Scientific Director. He stays abreast of the latest research developments. Part of his role is to manage Cure CMD’s investment in high-quality research to further our understanding of conditions, develop effective treatments, and identify synergies between labs and industry working in similar areas.

Rachel Alvarez is Cure CMD’s Executive Director. She was diagnosed at birth with an unspecified neuromuscular condition, finally confirmed in 2009 as congenital muscular dystrophy, subtype: Col6-RD. Rachel works for and on behalf of families living with congenital muscular dystrophy, to not only support their current needs, but to help ensure treatments in the foreseeable future for this group of ultra-rare conditions.

Founded in 2023, GIMM is a unique research institute in the fields of life and biomedical sciences. The translational potential of GIMM discoveries is streamlined through the Lisbon Academic Medical Centre (CAML), a privileged strategic partnership with the University of Lisbon Medical School and North Lisbon University Hospital Center, the largest hospital in Portugal. This consortium aims at innovative medical education and facilitates the translation of research findings from the bench to the bedside.

Role

The Gomes group and GIMM will exert a significant impact on MAGIC by introducing a novel scale to the project, adding a cellular perspective to the muscle-on-chip technologies it aims to develop. This will provide unparalleled temporal and spatial resolution, greatly enhancing MAGIC ability to characterize disease-specific muscle-on-chip models. Gomes will also expand MAGIC by establishing two additional disease models of centronuclear myopathies, that will broaden the impact on the development of genetic therapies with benefit to clinical care and society. This ambitious project will greatly improve muscle-on-chip disease models, expanding our knowledge and abilities in this critical area of research.

Team

The Gomes group is dedicated to study skeletal myofiber formation and maintenance, which requires the connection between the nucleus and the cytoskeleton and precise nuclear positioning. The lab uses different molecular and cellular approaches in combination with time-lapse imaging analysis to address these questions. Due to their interdisciplinary expertise in bioengineering, Gomes group established the first in vitro model of differentiated myofibers with peripheral nuclei and transverse triads, which led to the identification of the first molecular pathway that control nuclear positioning in myofibers and is disrupted in centronuclear myopathies.