Modulation of RNA holds the promise for opening up entirely new concepts for pharmaceutical intervention. Providing the precision tools to modulate this wealth of new RNA targets is our mission. Our development programs focus on severe neuromuscular diseases.
Alpha Anomeric was founded in February 2018 to develop and commercialize new live-saving drugs for severe neuromuscular diseases based on the proprietary alpha anomeric bicyclo DNA platform. Our lead drug development program focuses on Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disorder, which is characterized by progressive muscle degeneration and death in early adulthood.
The Alpha Anomeric platform relies on the use of alpha-bicyclo-DNA, a new DNA analog with unprecedented pharmacological properties. The incorporation of an alpha anomeric bicyclic sugar into the oligonucleotide backbone and a new linkage geometry (7’-5’ instead of 5’-3’) allowed the design and synthesis of new DNA scaffolds with very high biostability, low toxicity and high exon-skipping activity.