Driven by our mission to profoundly improve the lives of people affected by serious diseases, our AOC platform is expanding the reach of RNA therapeutics to diseases currently not served by traditional RNA targeting approaches. Our AOC platform is built from years of in-house engineering that integrates oligonucleotide therapeutics, modulation of RNA processes, antibody engineering and conjugation, and drug delivery techniques. Today, we continue to follow the data as we build our pipeline focused on skeletal muscle and as we look to expand into other cells and tissues. Our vision is to utilize the agility of the AOC platform to look at multiple disease areas where we can deliver treatments to people living with both rare and common diseases.

We followed the data to engineer each component of our AOCs:

AOC
Components
Data-Driven Component
Characteristics
Our Engineering Impact

Monoclonal

Approved mAbs offer:

  • Well-established safety profiles
  • High specificity and affinity
  • Long half-lives
  • Designed through engineering to be effector function null
  • Epitope selection designed for optimal activity

Linker

  • Known linker
  • Applicable to multiple oligo modalities
  • Enhanced for durability
  • Engineered sites of conjugation
  • Optimized ratio of oligonucleotides to antibodies

siRNA

Approved siRNA drugs have shown:

  • Attractive safety profiles – no known thrombocytopenia, liver or renal toxicity
  • Potency in the nanomolar range
  • Sustained activity in the cytoplasm and nucleus
  • Engineered to withstand lysosomal enzymes
  • Selected and modified to diminish off-target effects

The flexibility of our AOC platform allows us to deploy various types of oligonucleotides, including small interfering RNAs (siRNAs) and phosphorodiamidate morpholino oligomers (PMOs), each of which modify RNA function in different ways. This allows us to use oligonucleotides that are tailored to modulate a specific disease process. Mechanisms of these oligonucleotides can range from reducing the expression of a disease-related RNA with siRNAs to correction of aberrant processing of RNAs with splice-modifying oligonucleotides.

OUR AOCS OFFER DISTINCT ADVANTAGES

We believe that the product candidates derived from our AOC platform will have the potential to impact a diverse set of diseases with the following advantages:

Expanding Scope of
Diseases Beyond Liver

Targeting new tissue and cell types, including muscle, immune cells and others

Selecting Most Potent
Oligonucleotide Type

Archiving ED50s at the nanomolar concentration

Enabling
Infrequent Dosing

Maximizing durability with sustained single dose RNA reductions in NHP beyond 12 weeks

Readily Reproducible
and Scalable

Utilizing the same monoclonal antibody across multiple programs

Beginning with our muscle disease franchise, our platform tackles the underlying genetic cause of disease—from here our deep pipeline is set to advance and expand into new cells and tissues.

We also have development efforts focused on immune cells, cardiac tissue and other cell types. Some of these programs are being worked on through collaborations while others are from our internal discovery efforts. We look forward to pursuing all of these programs as we build an integrated and diverse company in service of our patients.