About our RNA Modulation Technology
Amylon’s drug development platform is based on our innovative RNA Modulation Technology. RNA Modulation Technology uses antisense oligonucleotides (ASOs) to alter the splicing of pre-mRNA to counteract the effects of disease-causing mutations. By preventing toxic protein formation at the RNA level, we address the problem at the source without permanently altering the genetic make-up of the cell. Over the past 20 years, ASOs have been pursued as a novel therapeutic modality to target a variety of diseases. These collective efforts have recently led to clinical successes as witnessed by the first approved ASO therapies. Building on this innovative approach, we have the ambition to apply our RNA Modulation Technology to prevent the onset and progression of serious diseases for which there are currently no therapies available.
Molecular mechanisms of RNA Modulation Technology
Antisense oligonucleotides (ASOs) can be designed to specifically bind to any segment of a target messenger RNA (mRNA). Amylon focuses on ASOs that alter the splicing of pre-mRNA and lead to a variety of potential effects on the mature mRNA and protein expression. These effects can include exon skipping, exon inclusion, open reading frame truncation, changes to transcript localization, modification of translation efficiency, and reduced mRNA stability. Depending on the type of mutation causing the disease, the mutation may be removed from the mRNA, or its effects cancelled out. The ASO thus provides a ‘molecular detour’ to restore the disease gene towards its normal function. ASOs that modulate splicing can function as a therapy for a broad range of genetic orphan diseases of the central nervous system (CNS). Our ambition is to advance the great opportunity of this technology by building a platform company with multiple therapeutic candidates in its pipeline.
Amylon has partnered with Alcyone Lifesciences to combine Amylon’s next generation of ASOs with Alcyone’s revolutionary delivery technology. Delivery of therapeutic ASOs to the brain presents several challenges. To address a major barrier to delivery, Alcyone has developed a superior technology for delivering ASOs by an intrathecal injector and catheter system into the CNS. The technology takes advantage of cerebrospinal fluid dynamics to deliver therapeutics to anatomical areas in the CNS that are difficult to reach. This platform reliably increases the effective delivery of ASOs throughout the brain, and at the same time greatly increases convenience to the patient. “This collaboration is based on foundational values that Alcyone and Amylon share”, said PJ Anand, CEO of Alcyone Lifesciences. “To develop life changing therapies based on the highest quality of science and focus on patients.”