Written by Alex Hoehn and Lena Clerici
Our A.P Biology was honored with visiting three biology based laboratories. Sharp Edge Labs was one of our stops, and our favorite stop of the day. Sharp Edge Labs is a small company who specializes in discovering drugs to treat genetic disorders of protein trafficking. Being able to meet such a compelling and intelligent scientist such as Dr. Scott Sneddon only increased our interest in the field of biology and ensured that this is the field that we want to pursue. The way he spoke about very complicated subjects was very interesting and we could not help but listen to every word he said and actually understand. Sharp Edge labs is a breakthrough company and we are sure that we will be hearing about them in the future. Even our AP Biology teacher, Mrs. Amy Murray, walked away with the statement, “that actually changes how (even what!) I teach.”
Sharp Edge Labs has launched a patient-driven program with the purpose discovering drugs that prevent or reduce the effects of faulty protein trafficking due to monogenic diseases. These monogenic diseases encompass disorders such as Cystic Fibrosis, Muscular Dystrophy, and Gaucher’s disease. The root cause of these conditions is a due to a defect in one gene caused by improper protein trafficking. Other companies used gene replacement therapy to replace the defective enzyme, permitting proper function. However, Sharp Edge Labs is taking a new route in the journey to accomplish the same goal. Rather than using gene replacement therapy, they are using the “small-molecule” approach. The size of the molecule causes no hindrance when traveling towards the target.
These molecules are targeted towards deformed proteins with the purpose of reconstructing the damaged proteins. Reconstructing the damaged proteins allows proper protein trafficking to resume. Protein trafficking is a “lock and key” task. Each signal chemical has a 3D specific counterpart receptor. If this protein receptor is damaged, the protein does not receive the signal; therefore, the protein does not perform its necessary function, sometimes leading to the aggregation of proteins. This break in the chain of commands results in diseases such as Gaucher’s as well as ALS.
Today, in Sharp Edge Labs patient program, when a patient enters the trial, a sample of the patient’s cells are used to determine which compounds should be properly used to restore protein trafficking. This approach provides more effective treatments earlier than if the patient was given the compound upon beginning the trial. Sharp Edge Labs currently is running three different trials; Cystic Fibrosis: CFTR Trafficking, Lysosomal Storage Disorders: Trafficking Assays, and Trafficking Defects in Parkinson’s Disease.
We were able to experience and learn about a drug discovery program that has the potential to revolutionize the medical treatment industry. Contrary to the what we thought as we began the day, these profound discoveries are not only made by companies with large research and development departments. Rather, small companies such as Sharp Edge Labs have the capability to make these types of discoveries.
If someone is interested in studying medicine/biology, we would highly recommend visiting Sharp Edge Labs located in Pittsburgh, Pa. It helped us to realize that even the most devastating diseases can be prevented and our future as biologists can save many lives in the process. For such a small company, their attention to detail and pride in what they are doing is inspiring to us and to many others. They are a very diverse team who wants people of different mindsets to come up with various ideas on how to treat many fatal diseases. They truly want to help people in need, and that quality is attractive to many people. Overall, the trip to Sharp Edge Labs was our favorite of our three trips and gave us an insightful view of how the medical industry is evolving.