Cambridge, Mass., January 4, 2018 — Atlas Venture, a leading biotech venture capital firm, today announced the launch of Generation Bio, a company developing the first genetic medicines that can be titrated to effect and re-dosed for a lifetime of benefit. Atlas founded the company in 2016 with a $25 million Series A.
Generation Bio is developing a breakthrough class of genetic medicines based on its proprietary GeneWave technology, which delivers high levels of durable gene expression and can be re-dosed to titrate and sustain effect.
Rare disease leader Geoffrey McDonough is President and Chief Executive Officer of Generation Bio. Dr. McDonough was Chief Executive Officer of Swedish Orphan Biovitrum (Sobi), and previously served as Genzyme’s President EMEA, and as General Manager of the Lysosomal Storage Disease franchise at Genzyme.
“Generation Bio is pioneering this transformative approach to enable a new generation of people to live free of the disease they were born with,” Dr. McDonough said. “Our unique capsid-free technology enables the development of genetic medicines that can be titrated and maintained to optimally impact each patient’s disease over a lifetime. In addition, it avoids the immunogenicity associated with viral vector-based gene therapies that limits the number of patients who can be treated and prevents re-dosing.”
The company’s core technology was discovered by Generation Bio scientific founder and Head of Discovery, Robert Kotin, Ph.D. As a senior investigator at the National Institutes of Health, Dr. Kotin discovered a novel modality for non-viral gene transfer, known as closed-ended DNA, or ceDNA. This eukaryotic DNA has a unique ability to translocate from the cytoplasm of the cell to the nucleus without the use of a viral capsid. Once in the nucleus, ceDNA forms stable, non-integrating episomes that result in high levels of long-term gene expression. The technology can also accommodate much larger genes and regulatory elements, allowing for a greater number of diseases to be addressed.
Generation Bio has built on this foundation to create the GeneWave platform, its first therapeutic application for ceDNA. GeneWave uses a state-of-the-art lipid nanoparticle to intravenously deliver ceDNA to the liver, where it may be used to address diseases of the liver itself or to transform the liver into a living “biofactory” for expressing systemic proteins to treat a wide variety of genetic diseases.
Since its founding in 2016, Generation Bio has demonstrated durable, dose-dependent expression and the ability to increase expression in the liver with re-dosing in vivo with the GeneWave platform. The company is initially focused on developing medicines for rare genetic diseases, including inherited metabolic disorders and lysosomal storage diseases, as well as emerging treatment areas where large numbers of patients cannot be addressed by current gene therapy modalities. Beyond the liver, the company has also demonstrated durable in vivo expression in muscle, the retina, and the central nervous system.
“With a bold vision for transforming treatment paradigms across a range of genetic diseases, Generation Bio is leading the charge to fulfill gene therapy’s potential to treat patients for a lifetime,” said Jason Rhodes, a partner at Atlas Venture and a founder and Chairman of Generation Bio’s board of directors. “GeneWave represents an opportunity to accomplish what no other gene therapy has yet done – to enable genetic medicines with truly drug-like properties and scale. Generation Bio is working to rapidly move this powerful new class of medicines into clinical development.”
In addition to Dr. McDonough and Dr. Kotin, Generation Bio’s leadership team includes Mark Angelino, Chief Operating Officer and a company founder; Doug Kerr, Head of Preclinical Research and Clinical Development; Matt Stanton, Head of Platform Research; and Glenn Goddard, Chief Financial Officer.
The company’s board and scientific advisors are a team of experienced life science innovators with extensive expertise in rare disease research and drug development.