Product Candidates

Ophthotech is developing three new molecular entities targeting the key molecular events contributing to the pathology of age-related macular degeneration (AMD). These include:

  • E10030, an anti-platelet-derived growth factor (PDGF) pegylated aptamer
  • ARC1905, a pegylated aptamer targeting the C5 component of the complement cascade
  • Volociximab, a monoclonal antibody targeting the transmembrane receptor, α5β1 integrin.

Each drug product is currently in a clinical trial for AMD.

 

Aptamer Technology

Aptamer therapeutics have many attractive attributes. Like monoclonal antibodies, they have similar molecular recognition properties, high binding affinity and specificity to their targets [1]. Aptamers are single-stranded oligonucleotides that fold in specific well-defined, three-dimensional shapes, which allow them to interact with a folded, three-dimensional protein target, like a key in a lock. Unlike monoclonal antibodies, aptamers have not been observed to be immunogenic; because nucleic acids are not typically recognized by the human immune system as foreign agents, aptamers do not generally trigger an antibody response. The three-dimensional structure of aptamers creates a large surface area of interaction between an aptamer and its target protein, making aptamers ideally suited for blocking protein-protein interactions. Furthermore, since aptamers interact with proteins found on the surface of and outside cells, aptamers do not have to cross the cell membrane, which may make it easier to deliver an effective quantity of aptamer to the target. Aptamers are chemically stable and can be designed to resist metabolic degradation in the human body. Using standard, defined chemical modifications and conjugations, aptamers can be specifically engineered to have an optimal half-life, or the ability to maintain effective concentration. Aptamers are chemically synthesized allowing for rapid, scalable and reproducible production at lower costs.

References

1. Mayer G, Andreas J. Aptamers in Research and Drug Development. BioDrugs: Volume 18(6)2004. pp315-359.