LyGenesis Adds Inborn Errors of Metabolism, Orphan Pediatric Indications ... to its Drug Development Pipeline

LyGenesis, Inc., a clinical-stage biotechnology company developing cell therapies for patients with end stage liver disease, Type 1 diabetes, end stage renal disease, and aging, announced today that it has achieved positive in vitro results of a novel combination drug-biologic product for patients with inborn errors of metabolism, adding these orphan pediatric indications to its drug development pipeline.

Dr. Paulo Fontes, LyGenesis's Co-Founder and Chief Medical Officer, noted: "While our lead therapy is focused on patients with end stage liver disease, we have also been developing a novel hydrogel with growth factors and a proprietary patent-pending surgical catheter to engraft our cell therapy under the liver capsule of patients, enabling a large mass of cells to be engrafted and nurtured by the patient's liver. Our early in vitro work supports this approach, and we are now rapidly moving into preclinical studies so that we can advance this to the clinic for our pediatric patients born with catastrophic and often fatal inborn errors of metabolism."

Inborn errors of metabolism are genetic disorders in which the body cannot properly metabolize food into energy. Comprised of over two dozen separate genetic defects, inborn errors of metabolism can produce profound brain damage and death if not identified and treated soon after birth. The lead indication for this new combination product will focus on Maple syrup urine disease (MSUD), a rare disorder, affecting 1 in 185,000 newborns. Untreated, it leads to brain injury in days and death occurs in weeks or months. Full liver transplant cures MSUD, but it is associated with both surgical risk, lifetime immunosuppression and a risk of complications post-transplant.

Dr. C. Andrew Bonham, an Associate Professor of Surgery and Abdominal Transplantation at Stanford University, commented, "New therapeutic approaches for the treatment of inborn errors of metabolism are desperately needed. Currently children suffering from these diseases risk catastrophic neurologic complications. The only cure is with a liver transplant, a rather draconian procedure to correct an enzymatic defect. LyGenesis combines cell therapies with a deep understanding of anatomy and how structures within the body – whether lymph nodes or the liver capsule – can be used to create a niche for cells to expand and thrive to correct the metabolic defect and help patients. I look forward to helping accelerate this new therapy into the clinic so that we can help patients born with MSUD and other metabolic defects as quickly as possible."

Dr. Gregory Bailey, MD, CEO and Director of Juvenescence Limited, added, "Juvenescence is delighted that LyGenesis has reached another important milestone expanding its regenerative cellular therapies platform following a successful path to the clinic with its lead program for liver regeneration, which should start a phase 2a clinical trial shortly."


LyGenesis is a clinical-stage biotechnology company whose cell therapies use patients' lymph nodes as bioreactors to regrow functioning ectopic organs. LyGenesis's lead allogeneic cell therapy program is currently in a Phase 2a clinical trial for patients with end stage liver disease. LyGenesis's drug development pipeline includes cell therapies that can produce an ectopic thymus (for aging and multiple other potential indications), pancreas (for Type 1 diabetes), and kidney (for end stage renal disease). LyGenesis's latest addition to its drug development pipeline is focused on orphan pediatric indications involving inborn errors of metabolism. Privately held with investment support from Juvenescence, Longevity Vision Fund, and other investors, LyGenesis is headquartered in Pittsburgh, Pennsylvania. To learn more, please visit

View the Press Release here.