A muscular disease affecting 1 in 3,500 male births, making it one of the most common genetic disorders. DMD is a progressive disease, initially impacting pelvic and shoulder muscles before spreading to other areas of the body. The biggest risk with DMD is the potential for life-threatening issues to develop after impacting cardiac and respiratory muscles. Current therapies only address 30% of patients.
Preclinical data shows that OligomicsTx’ treatment improves muscle function, significantly restores dystrophin in skeletal muscles, and prevents cardiac damage with no overt toxicity.
A rare neuromuscular disorder affecting 1 in ~8,000 people that involves weakness and wasting (atrophy), gradually affecting specific areas of the body. The muscles that are mainly affected are in the face (facio), around the shoulder blades (scapulo), and in the upper arms (humeral). Disease progression is typically slower and can have a late or early onset. ~870,000 people worldwide are estimated to be impacted by the disease. There are no approved therapies currently for FSHD.
OligomicsTx has the only ASO+LNP combination in development - a tried-and-true method. Preclinical data shows significant improvement in muscle fusion, reduced DUX4 expression, and improve muscle function.
Comprised of 5 different types, it is characterized by the loss of specific nerve cells in the spinal cord. This causes wasting and weakening of larger voluntary muscles including the back, shoulders, hips, and occasionally those involved with eating, swallowing, and breathing. Disease prevalence is equal across genders, affecting 1 in 10,000 births. Current therapies for SMA are hampered by invasive, potentially risky administrative procedures, poor efficacy outside of motor neurone, and off-target effects.
Preclinical data for OligomicsTx' shows improved SMN levels, muscle function, and no apparent immune response and toxicity.
An ultra-rare disorder, impacting an estimated 1 in 2 million people globally. It causes ligaments, tendons, and skeletal muscles to progressively be replaced by new bone, causing joints to be locked in place as the body hardens into an immobile state over time. Only one approved (small molecule) therapy with limited efficacy.
OligomicsTx is developing the first RNA-based therapy for the disease, targeting the mutated ACVR1 gene only and improving systemic delivery throughout the body. Preclinical data shows 25% improved exercise performance, improved survival, and prevention of extra bone formation.