Three therapeutic platforms target different muscle diseases.
Platform
Indication
Pipeline
Satori
Satori EEC
R&D
Pre-Clinical
Clinical

Indication: Exstrophy-Epispadias-Complex

Original PhSats for autologous treatment of muscle defects: The first-in-human trial with the lead candidate (Satori-01) will begin in 2024. The phase 1/2a MuST trial targets an orphan disease and aims for conditional market approval.

Satori tba
R&D
Pre-Clinical
Clinical

Indication currently not disclosed

Satuni
Satuni tba
R&D
Pre-Clinical
Clinical

Engineered PHSats for allogeneic use: Induced pluripotent stem cell (iPSC)-derived muscle precursor cells for allogenic applications. Preclinical work is in progress.

Satgeno
Satgeno LGMD
R&D
Pre-Clinical
Clinical

Indication: Limb Girdle Muscular Dystrophies

Gene-edited PHSats: Several mutations associated with muscular dystrophies can be efficiently and precisely repaired with our gene editing tools. A first-in-human bASKet trial is at an advanced stage of planning.

MyoPax therapeutic strategies

Satori: Autologous PHSats therapy  to treat local muscle defects.  From a small muscle specimen, the patient’s own muscle stem cells are isolated and expanded using our innovative PHSats technology. The Satori product is delivered to the hospital in a ready-to-use formulation.

Satuni: Allogeneic therapy with engineered PHSats to treat acute muscle wasting. Human induced pluripotent stem cells (hiPSCs) are engineered so that they are not targeted by the recipient’s immune system. From these, we induce the PHSats phenotype. Satuni products are manufactured in large non-patient-specific batches.

Satgeno: Autologous therapy with genetically corrected PHSats to treat muscular dystrophies. The disease-causing mutation in the isolated cell population is repaired with specific gene editing tools. The repaired population is expanded with our PHSats technology and injected into selected affected muscles to build healthy muscle tissue.

First-in-human trial: The MUST Trial

The first-in-human trial with the lead candidate (Satori-01) targets an orphan disease and current unmet need: A prenatally incompletely formed bladder sphincter muscle leads to lifelong incontinence in Exstrophy-Epispadias patients (EEC). The phase 1/2a trial is aimed to repair the EEC sphincter defect and investigates safety and efficacy.
The sponsor of the trial is the Charité Universitätsmedizin. It is financed by the German Ministry of Education and Research and the Else Kröner Fresenius Foundation.

© Eric Metzler
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