Clinical portfolio strategy for regenerative muscle stem cell therapies

Project Information

MyoPax

Grant agreement ID: 101114323

DOI

10.3030/101114323

Project closed

EC signature date 26 May 2023

Start date 1 June 2023

End date 31 May 2024

Funded under

• European innovation ecosystems

Total cost

No data

EU contribution

€ 75 000,00

Coordinated by

MYOPAX GMBH
Germany

Periodic Reporting for period 1 - MyoPax (Clinical portfolio strategy for regenerative muscle stem cell therapies)

Reporting period: 2023-06-01 to 2024-05-31

Summary of the context and overall objectives of the project

Myopax's mission is to transform muscle diseases through the development of cell and gene therapies for patients with high unmet medical needs by leveraging its proprietary muscle stem cell platform.

Need:
Without functioning muscle, you cannot smile, hold a cup, nor breath. Muscle weakness leads to loss of personal autonomy and premature death. Muscle disorders affect >20 million EU people and are largely untreatable. Muscle atrophy is a consequence in common diseases (e.g. trauma, critical illness), in ageing, or in rare diseases caused by gene mutations.

Technological Innovation:
MyoPax is a spin-off from the Charité Universitätsmedizin and the Max-Delbrueck-Center of the Helmholtz Association in Berlin, Germany. We are pioneering proprietary enabling technologies to source and manufacture highly regenerative muscle stem cell products with long-term treatment effect. Beyond the replacement utilization of these cells to regenerate and replenish muscle, mRNA-delivered gene correction is applied precisely and efficiently.

Therapeutic Platform:
These innovative capabilities are being deployed into clinical applications for targeted cell and gene therapies to treat the different muscle disorders (muscle defects, genetic muscular dystrophies, secondary muscle wasting). The start of the first-in-human studies is imminent.

Impact of Women TechEU:
With support from Women TechEU, MyoPax advanced its R&D and business strategy.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

Market Analysis: Sharpening of competitive analyses, market entry strategies and pricing
Regulatory Strategy: Shaping international regulatory strategy.
Clinical Perspective: Interviewed key opinion leaders (KOLs), network-expansion.
Intellectual Property (IP): IP strategy.
Production: Screened contract manufacturers

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

With support from Women TechEU, MyoPax advanced its clinical and business strategy. This includes developing an international regulatory and IP strategy, conducting detailed market analysis, and expanding its network with key opinion leaders (KOLs). Further public and private funding will leverage the MyoPax technological potential and expand its clinical portfolio to restore muscle strength to those in need.