Monogenic diseases often have a spectrum of genetic mutations and factors. Symptoms, timelines, and treatments rarely follow the same script. With rare and ultra-rare disorders, each patient is unique, and therapies may have to be tailored to the individual. Curi Bio is accelerating the path to investigational new drugs, making personalized medicine a reality and developing potency/release assays featuring human clinically-translatable disease models.
Curi Bio has licensed and/or developed advanced human tissue models for a number of monogenic disorders, including Duchenne Muscular Dystrophy (DMD) (see our case study below). Additional models include:
Myotonic Dystrophy Type 1 (DM1)
Facioscapulohumeral Muscular Dystrophy (FSHD)
X-linked Myotubular Myopathy (XLMTM)
Hypertrophic & Dilated Cardiomyopathies (HCM/DCM)
and More
Contact our team to discover how Curi Bio can help accelerate your disease research and therapeutic discoveries by providing access to our pre-made disease model library, tissue models, and tissue-specific biosystems. Curi Bio is actively pursuing additional partnerships to create new disease models.
Case Study
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact1. A number of mutations within the dystrophin gene can result in DMD or a less severe disorder known as Becker Muscular dystrophy. The earliest clinical symptom of DMD is primarily skeletal muscle weakness, followed by progressive loss of cardiac and diaphragm function.
1MDA.org
Skeletal EMTs bearing a dystrophin-null phenotype (a model of DMD) exhibited significant reductions in contractile twitch and tetanic force and kinetics compared with healthy (wild type) isogenic controls. DMD tissues also display greater fatigue following repetitive contractions by electrical stimulation, similar to in vivo models of the disease and clinical manifestation.
Histological analysis of tissue cross sections showed distribution of myosin positive myotubes throughout the EMT with a pronounced dystrophin ring stained at the cell membrane in healthy tissues. DMD tissues, as expected, completely lacked dystrophin rings surrounding myosin positive myotubes. These data demonstrate the ability of Mantarray to uniquely stratify healthy and diseased phenotypes to recapitulate native developmental architecture and muscle function.
Further disease models include, but are not limited to:
Myotonic Dystrophy Type 1 (DM1)
Facioscapulohumeral Muscular Dystrophy (FSH, FSHD)
X-linked Myotubular Myopathy (XLMTM)
Hypertrophic & Dilated Cardiomyopathies (HCM/DCM)
and More
Contact our team to discover how Curi Bio can help accelerate your disease research and therapeutic discoveries by providing access to our off-the-shelf disease model library, tissue models, and tissue-specific biosystems. Curi Bio is actively pursuing additional partnerships to create new disease models.
