Generation Bio Co is a biotechnology company that focuses on developing genetic medicines. It was founded in 2016 by Atlas Venture and Fidelity Investments and is headquartered in Cambridge, Massachusetts. The company's mission is to create new ways to target and treat genetic diseases by utilizing gene therapy techniques. They use a novel approach called non-viral gene therapy to deliver treatment to the cells. The non-viral approach offers a safer and more efficient way to deliver genetic material than traditional viral gene therapy.
Generation Bio Co went public on June 3, 2021, with an initial public offering (IPO) of 10 million shares of common stock priced at $19 per share, raising a total of $190 million. The company trades on the Nasdaq Global Select Market under the ticker symbol GBIO. The funds raised through the IPO will be used to advance the company's preclinical and clinical development programs, as well as for general corporate purposes.
The company's lead product candidate is a gene therapy for phenylketonuria (PKU), a genetic disorder that causes high levels of phenylalanine in the blood. The candidate, called GB001, is a non-viral gene therapy designed to deliver a functional copy of the phenylalanine hydroxylase (PAH) gene, which is mutated in PKU patients. The therapy is administered by a single injection and has shown promising results in preclinical studies.
In addition to PKU, Generation Bio Co is working on developing therapies for other genetic diseases, including hemophilia A and B, Fabry disease, and Pompe disease. The company's technology platform allows them to develop treatments for a wide range of genetic disorders by customizing the genetic material delivered to cells. The company believes that their non-viral gene therapy approach will revolutionize the field of genetic medicine and help to address unmet medical needs in genetic diseases.