The Biozentrum spin-off develops an innovative gene therapy to treat congenital muscular dystrophy.
Cross-section through treated LAMA2 MD muscle with individual muscle fibers surrounded by laminin (green) and regenerated small muscle fibers (red).
Congenital muscular dystrophy is a rare hereditary disease which leads to muscle weakness and loss already at young age. The more than thirty known forms of this neuromuscular disease differ in the type of genetic defect and in the severity of disease progression. SEAL Therapeutics focuses on LAMA2-related muscular dystrophy (LAMA2 MD), a severe form of muscular dystrophy caused by the absence of laminin-alpha 2 in the muscle fibers. There is currently no specific treatment for patients with LAMA2 MD.
Innovative SEAL Technology
This innovative gene therapy approach developed by Prof. Markus Rüegg and his team and with international collaborators overcomes the functional defects causing LAMA2 MD. The approach of SEAL Therapeutics involves delivering specifically designed linker proteins that compensate for the missing laminin-alpha 2 in the muscle fibers (see SEAL technology). Available data demonstrate that the simultaneous expression of two types of specifically designed linker proteins functionally corrects the underlying cause of laminin-alpha 2 deficiency, leads to sustained improvement in muscle histology, increased muscle mass and strength, improved body weight, and results in a remarkable increase in life span compared to untreated animals (see literature).
Improving the lives of patients
SEAL Therapeutics was founded to make this innovative gene therapy available to LAMA2 MD patients and to improve their quality of life. The research team continues to develop their treatment approach and aims to team-up with partners in the pharmaceutical industry for clinical development with LAMA2 MD patients (see collaborations).
Contact details: SEAL Therapeutics AG