Drug intelligence / Profile preview

LAM 111

Development stage
Preclinical
Lead developer
Sarcomatrix Therapeutics
Modality
Recombinant Proteins and Enzymes
Administration
Intramuscular
01

Overview

LAM 111 (Laminin-111) is a recombinant protein therapy being developed by Sarcomatrix Therapeutics for the treatment of LAMA2-related congenital muscular dystrophy (LAMA2-RD), also known as Merosin-deficient congenital muscular dystrophy type 1A (MDC1A). LAMA2-RD is a severe muscle-wasting disease caused by mutations in the LAMA2 gene, which leads to a deficiency in the laminin-alpha2 chain and the subsequent absence of laminin-211 in the skeletal muscle basement membrane. LAM 111 is a naturally occurring embryonic isoform of laminin (composed of alpha1, beta1, and gamma1 chains) that can functionally substitute for the missing laminin-211. By restoring the structural integrity of the basement membrane and its linkage to the muscle cell cytoskeleton via alpha-dystroglycan and integrin alpha7beta1, LAM 111 aims to stabilize the muscle cell membrane, prevent muscle fiber degeneration, and improve muscle function. The drug is currently in IND-enabling studies.

Other names
Laminin-111Laminin111Laminin 111Laminin-1Laminin1Laminin 1
02

Targets

α7β1 (Integrin α7β1)DAG1 (Dystroglycan 1)

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