MyoPax's EIC Accelerator Repairs Muscle Stem Cells Gene by Gene

Limb-girdle muscular dystrophies are a group of more than 30 inherited disorders, and none of them has an approved therapy that stops the muscle wasting. Patients get physiotherapy and symptomatic care. MyoPax, a 2022 spin-off of the Muscle Research Unit of Charité and the Max Delbrück Center in Berlin, wants to repair the cause: take a patient's own muscle stem cells, correct the mutation with CRISPR tools delivered as mRNA rather than by a virus, expand the cells and inject them into the affected muscles so they build healthy tissue.

The EIC Accelerator selected the project, Satgeno, at its October 2024 cut-off for blended finance, and CORDIS records a grant of €2,499,999 from June 2025 to November 2027 to run a first-in-human trial, DELI550, against a frequent founder mutation. If it works, the platform is meant to extend to other mutations affecting more than 400,000 people in the EU and US. As of October 2026 the trial has not been applied for.

Thirty years of muscle research behind one company

The technology is Simone Spuler's. She is a professor of myology who heads Charité's outpatient clinic for muscle disorders and the Muscle Research Unit at Charité and the Max Delbrück Center, and she is named as the inventor of MyoPax's proprietary method for isolating and growing muscle stem cells, which the company calls PHSats, for primary human satellite cell-derived muscle stem cells. CEO Verena Schöwel-Wolf is a physician trained in myology with an MBA; Diane Seimetz, who co-founded Biopharma Excellence, sits on the board. All three founders are women.

MyoPax joined the BioInnovation Institute in Copenhagen when it launched in 2022 and took a €1.3 million convertible loan from it, as the Max Delbrück Center reported. In November 2024 it became the first tenant of Bayer's Co.Lab incubator in Berlin, and in June 2026 the EU health commissioner, Olivér Várhelyi, visited it there, according to the company's news page.

Three platforms, three stages

The pipeline has three programmes. Satori injects a patient's own unedited muscle stem cells to repair local muscle defects; its indication is no longer disclosed, and it sits in preclinical development. Satuni engineers hypo-immune cells from induced pluripotent stem cells for off-the-shelf use against acute muscle wasting, and is in research. Satgeno, the EIC-funded programme, adds gene correction for muscular dystrophies.

The first product of that first platform has US designations: in July 2023 the FDA granted Satori-01 orphan drug and rare pediatric disease designation for exstrophy-epispadias complex, a congenital condition. A Charité-sponsored phase 1/2a trial of muscle stem cells in that indication is registered as NCT04729582 and was listed as not yet recruiting at its last update. Nature Medicine named the work among the clinical trials it expected to shape medicine in 2023.

Satgeno and the DELI550 trial

Satgeno-01 corrects a single mutation, CAPN3 c.550delA, with Cas9 delivered as mRNA into the patient's own muscle stem cells. The planned DELI550 trial is a first-in-human phase 1/2a study in patients with limb-girdle muscular dystrophy type R1/2A who carry one or two copies of that variant. The company says close to 70,000 patients worldwide have CAPN3 mutations, that disease onset is usually early in the second decade of life, and that patients typically lose the ability to walk independently within 15 years.

The trial website is explicit about where the programme stands: "we are in the preclinical phase for the preparation of the clinical trial", with the next steps being pharmaceutical-grade GMP manufacturing of the gene-edited cells and then the trial application to regulators. Its own timeline puts technology transfer to a contract manufacturer, the application through the EU clinical trials system, approval and then the trial itself across 2026 to 2028, beyond the end of the EIC project in November 2027. No recruitment is taking place. CORDIS shows no periodic report yet.

1. Gene-edited muscle stem cellsPreclinicalEditing toolkit described; no clinical batch.
2. GMP manufacturingNext stepNamed as still to be proven on the trial site.
3. Trial application to regulatorsNot filedNo recruitment; application still ahead.
4. First-in-human resultsToo earlyCompany timeline runs to 2028.

Grants, a loan and a first cheque

MyoPax has announced no priced venture round. Its disclosed money is the BioInnovation Institute loan, non-dilutive public grants, and the EIC award, which the company described in February 2025 as €2.5 million of grant with an option of further EIC Fund equity. In October 2026 it became the first company funded through IBB Ventures' new B# Plus instrument, which the announcement says will help prepare the first therapeutic candidate for clinical development; the amount was not disclosed. Investment director Ute Mercker said IBB had been in contact with the team "even before the company was founded". The Charité-sponsored Satori trial is financed by the German research ministry and ForTra of the Else Kröner-Fresenius Foundation. MyoPax's German accounts were not obtained for this article.

European cell therapy at the slowest stage

MyoPax is the kind of company the EIC says it exists for: a university spin-off with a patented platform, a rare disease with no therapy, and a step, GMP manufacturing and a first trial, that is too expensive for grants from research budgets and too early for most investors. It is also a reminder of the timescale. The grant runs to November 2027; the company's own plan has the trial finishing after that.