Data and Safety Monitoring Board Approves LyGenesis's Phase 2a Clinical Trial to Continue and Dose Escalate in Novel Trial of Organ Regeneration
The next four patients will receive a higher dose of LyGenesis's cell therapy to grow functioning liver tissue in patients with end stage liver disease
PITTSBURGH, March 4, 2025 /PRNewswire/ -- LyGenesis, Inc., a clinical-stage biotechnology company developing cell therapies for organ regeneration, today announced that the Data and Safety Monitoring Board (DSMB) has completed its review of the first cohort of the company's Phase 2a clinical trial for patients with end-stage liver disease and has recommended that the trial continue and dose escalate as previously planned for the next four patients.
The DSMB reviewed the safety, tolerability, and efficacy data from the trial's first cohort of four patients. Based on their review, the DSMB's recommendation to proceed highlights the safety and tolerability of LyGenesis's innovative approach to organ regeneration as they explore the ideal dose of their allogenic cell therapy to take into later stage clinical trials.
Dr. Constance Mobley, Principal Investigator of the Phase 2a clinical trial at Houston Methodist Hospital, and Associate Director of Liver Transplantation at Houston Methodist J.C. Walter Jr. Transplant Center and the Associate Surgical Director of Liver Transplantation in the Department of Surgery in the Houston Methodist Department of Surgery, said "We are very encouraged with the safety and tolerability of the cell therapy based on this initial first cohort of patients, and encouraged by the DSMB's recommendation to proceed. This milestone represents an important step forward in evaluating the potential of allogeneic hepatocyte transplantation for patients with end-stage liver disease. Our team remains committed to advancing groundbreaking approaches that have the potential to address the urgent need for our patients."
LyGenesis's proprietary cell therapy platform uses a patient's lymph nodes as bioreactors to grow functioning ectopic organs, offering a transformative therapeutic strategy for organ regeneration. The Phase 2a clinical trial is designed to evaluate the safety and early efficacy of hepatocyte transplantation in patients with severe liver disease.
Contact Information
Media contacts:
LyGenesis – Jacqueline Jeha, MPH, +1.401.374.0955, [email protected].
Houston Methodist – Amy K. Rose, +1.412.613.1428, [email protected]
About LyGenesis' Liver Cell Therapy Program
LYG-LIV-001 is an allogeneic cell therapy regulated by the FDA as a Section 351 Human Cellular Therapy/Product and as an investigational biologic product. This biologic therapy is created starting from donated otherwise unmatched livers. A complex, multi-step Current Good Manufacturing Practices process carefully isolates and suspends hepatocytes in a solution to ready them for transplantation into that patient's upper abdominal lymph nodes using an endoscopic ultrasound, a minimally invasive approach. The lymph nodes then act an as in vivo bioreactors, helping the hepatocytes to engraft, proliferate, and generate functional ectopic liver tissue. LyGenesis' cell therapies do not involve genetic engineering, which reduces the time, risks, and costs associated with manufacturing and commercializing their therapies.
About LyGenesis' LYG-LIV-02-001 Clinical Trial
This open-label, dose-escalation Phase 2a clinical trial is recruiting 12 patients with end stage liver diease (ESLD). Each patient receiving the cell therapy is closely monitored over 1-year through follow-up visits to examine safety, tolerability, and efficacy of the transplanted allogeneic hepatocytes as they relate to the signs and symptoms of their ESLD. A data safety monitoring board monitors each cohort of patients (N=4) to evaluate near-term safety prior to dose escalation for the next cohort of patients. A full list of the inclusion and exclusion criteria for the trial is available at ClinicalTrials.gov Identifier: NCT04496479.
About End-Stage Liver Disease
Almost 2% of the U.S. population is diagnosed with liver disease and over 50,000 Americans die annually of chronic liver disease1. ESLD is a potentially fatal disease that progresses over years and sometimes decades. Patients with ESLD have a broad constellation of symptoms and disease-related complications that affect their survival and health-related quality of life. Most often, ESLD is the result of cirrhosis, a condition in which scar tissue replaces healthy liver leading to irreversible changes in the anatomy and function of the hepatic tissue until the patients reach different stages of liver failure. Up to half of all ESLD patients who could benefit from a standard liver transplant are medically ineligible for one2, while approximately 12% of patients on the transplant wait list die annually3.
About LyGenesis, Inc.
LyGenesis is a clinical-stage biotechnology company developing a novel cell therapy platform leveraging patients' lymph nodes as bioreactors to grow functioning ectopic organs. LyGenesis' lead allogeneic cell therapy program is currently in a Phase 2a clinical trial for patients with end-stage liver disease (ClinicalTrials.gov Identifier: NCT04496479). LyGenesis' cell therapy platform including therapies in development designed to produce an ectopic thymus (for aging and multiple other potential indications), pancreas (for Type 1 diabetes), and kidney (for renal disease). Privately held, LyGenesis is headquartered in Pittsburgh, Pennsylvania. To learn more, please visit our website at www.LyGenesis.com.
About IIAM
THE INTERNATIONAL INSTITUTE FOR THE ADVANCEMENT OF MEDICINE (IIAM) is a leader for providing non-transplantable organs to the medical research community for combatting and curing disease. IIAM has unprecedented collaborations in the U.S. to source organs and other human tissue for groundbreaking medical research. IIAM can provide over 100 distinct types of non-transplanted human organs and tissues. For more information, please visit www.iiam.org or contact us directly at [email protected]
1 https://www.cdc.gov/nchs/fastats/liver-disease.htm
2 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3672443/pdf/0560e39.pdf
3 https://srtr.transplant.hrsa.gov/ADR/Chapter?name=Liver&year=2019
SOURCE LyGenesis, Inc.
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