OligomicsTx is developing world-class RNA-targeting therapies for rare neuromuscular disorders with significant unmet needs. Our current pipeline targets fibrodysplasia ossificans progressiva (FOP), facioscapulohumeral muscular dystrophy (FSHD), duchenne muscular dystrophy (DMD), and spinal muscular atrophy (SMA). It’s estimated more than 2,000,000 people worldwide have these rare genetic disorders. The name OligomicsTx comes from the molecules at the heart of our treatment development: Antisense Oligonucleotides or ‘ASOs’. OligomicsTx’s next generation ASOs have already demonstrated significant improvements in animal models and offer the potential to help a spectrum of rare diseases. While there are currently several ASOs on the market, many of them lack the delivery mechanisms to properly affect target tissues. The results are less potent treatments that collect in major organs, causing potential toxicity risks. By combining cutting-edge ASOs with groundbreaking delivery mechanisms to access vital regions in the body, we’re creating significant improvements and unlocking new treatment potential for thousands of patients. Our research is led by the work of our founding scientists, which has been published in industry-leading journals and led to the development of FDA-approved drug viltolarsen for DMD. PARTNERS/INVESTORS We are actively seeking partnerships with pharmaceutical companies and other industry connections to help get our treatments into the hands of patients. Get in touch with our team to learn more.
OligomicsTx is developing world-class RNA-targeting therapies for rare neuromuscular disorders with significant unmet needs. Our current pipeline targets fibrodysplasia ossificans progressiva (FOP), facioscapulohumeral muscular dystrophy (FSHD), duchenne muscular dystrophy (DMD), and spinal muscular atrophy (SMA). It’s estimated more than 2,000,000 people worldwide have these rare genetic disorders. The name OligomicsTx comes from the molecules at the heart of our treatment development: Antisense Oligonucleotides or ‘ASOs’. OligomicsTx’s next generation ASOs have already demonstrated significant improvements in animal models and offer the potential to help a spectrum of rare diseases. While there are currently several ASOs on the market, many of them lack the delivery mechanisms to properly affect target tissues. The results are less potent treatments that collect in major organs, causing potential toxicity risks. By combining cutting-edge ASOs with groundbreaking delivery mechanisms to access vital regions in the body, we’re creating significant improvements and unlocking new treatment potential for thousands of patients. Our research is led by the work of our founding scientists, which has been published in industry-leading journals and led to the development of FDA-approved drug viltolarsen for DMD. PARTNERS/INVESTORS We are actively seeking partnerships with pharmaceutical companies and other industry connections to help get our treatments into the hands of patients. Get in touch with our team to learn more.