Proteins play important and diverse roles in every aspect of our biology and therefore can impact our health or lead to disease when mutations in our genes are translated to those encoded proteins. Because AMD and linked diseases develop slowly over the course of many years, even minor changes to certain proteins that result in only a modest decrease in the function of the protein, over time, can lead to the onset of disease.
One strategy in treating disease with an underlying genetic association is to first identify patients with a specific protein variant that is dysfunctional, and thus a potential driver of disease, and compensate for that dysfunction by administering a functional version of the protein. When the defect leading to disease is matched with a treatment that directly addresses that lesion it is sometimes referred to as a precision or personalized medicine approach. When we produce proteins for use as treatments in these circumstances the proteins are produced externally and referred to as recombinant proteins. The technology used to produce these proteins is well-established and many steps and precautions are taken that result in a safe and effective drug that can be given to humans.
Though the technology behind the production of recombinant proteins is well-established, the individual characteristics of the protein being produced can present significant challenges. The first step in recombinant protein production involves engineering a cell line to produce the desired protein. Once that is achieved, the cells are expanded so that sufficient protein is made. After that process is complete, the desired protein is selectively isolated resulting in a very pure preparation that is suitable for use in humans.
While many recombinant versions of proteins are large and difficult to produce, Gemini has made significant advances in addressing the numerous technical challenges of the recombinant proteins relevant to treatment of AMD and linked diseases. We are in the process of submitting the documentation necessary to enable clinical use and are investigating the potential for both recombinant proteins and gene therapy to work together as part of our multimodal approach.