About GeneraBio

GeneraBio has a clear mission…

Closing the gap between treatments and cures and the patients who need them.

We pursue our goals by working with with institutions, researchers, other companies, governmental and regulatory agencies, insurers, foundations and most importantly, the patients we are here to serve. We are hear to help you be heard.

Advances in understanding the human genome, and the role of genetics in disease, have created a unique situation. Hundreds of diseases have now been linked to specific genetic defects. Multiple genetic disorders often manifest similar observable symptoms. Because of this diseases have been historically grouped based on these symptoms. With the specific identification of multiple genetic defects that cause similar symptoms in individuals, scientists now know that what were once thought to be single disorders are actually caused by many different specific genetic defects. One example is retinitis pigmentosa, which is now known to be caused by over 60 different genetic defects, each being a unique disease. Over 200 genetic defects have been linked to specific retinal degenerative disorders, and that number continues to grow. Scientists now believe that virtually all disease is linked to a genetic cause. Many of these diseases are very rare, and for most there are currently no treatment options. Because these disorders are typically progressive, even advanced patients will benefit from some types of treatment. We are also closely following research toward treatments that could restore lost vision for some.

This creates our opportunity to develop an efficient process whereby even small patient populations can receive approved gene therapy, drug, or other treatments specific to their genetic disorder. This requires creating and executing an efficient pathway through the regulatory process, that is still developing for cellular and gene therapy products, a special category created by the United States FDA within their Center for Biologics Evaluation and Research. There are also pharmacological agents that are in human trials for other genetic disorders that could benefit many patients from multiple disease groups, including retinal degenerative disorders. We pursue opportunities to facilitate the preclinical and regulatory approval process for promising technologies. Innovative study design using today’s knowledge, development of expeditious outcome measures, and efficiently driving the process with the involvement of the patient communities will make treatments available sooner, rather than later.