LyGenesis adds orphan paediatric indications with large unmet needs to its drug development pipeline with novel approach which first aims to treat paediatric patients with Maple syrup urine disease.

LyGenesis, 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.

Longevity.Technology: Our website often covers research news at a particular end of the age spectrum, but longevity aims should start as early as possible, and access to therapy that can ameliorate life-limiting diseases must be researched regardless of age of intended recipients. Earlier work from LyGenesis has demonstrated functional mini-livers can be grown using the lymph node as a bioreactor and the company hopes to enroll its first patient in its Phase 2a clinical trial for patients with end-stage liver disease who are currently ineligible for standard liver transplantation this year or early next year. Today’s announcement further demonstrates that LyGenesis is continuing to spearhead tissue and organ regeneration technology, and is making exciting progress in the space.

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 metabolise food into energy. Comprising 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. MSUD prevents the body from processing certain amino acids – leucine, isoleucine and valine – which causes a dangerous build-up of substances in the blood and urine. 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 Greg Bailey, 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.”
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