LyGenesis treats first patient in first-of-its-kind liver regeneration trial

Phase 2a trial targets end-stage liver disease with regenerative cell therapy transplanted into patients’ lymph nodes.

Cell therapy company LyGenesis today revealed that the first patient has received treatment in the Phase 2a clinical trial of its regenerative cell therapy targeting end-stage liver disease (ESLD). The therapy, known as LYG-LIV-001, treats ESLD via an allogeneic regenerative cell therapy transplanted into patients’ lymph nodes.

ESLD affects nearly 2% of the US population, with more than 50,000 Americans succumbing to chronic liver disease annually. Often stemming from cirrhosis, ESLD poses significant health risks, and many patients are ineligible for traditional liver transplants.

The Phase 2a clinical trial, an open-label, dose-escalation study, is currently recruiting 12 patients diagnosed with ESLD. Each participant will undergo close monitoring for one year to assess the safety, tolerability, and efficacy of the transplanted hepatocytes in addressing the symptoms of ESLD.

“In a medical first, we have now dosed our first patient in a clinical trial using their own lymph nodes as living bioreactors to regenerate an ectopic organ,” said Dr Michael Hufford, CEO of LyGenesis. “This therapy will potentially be a remarkable regenerative medicine milestone by helping patients with ESLD grow new functional ectopic livers in their own body. If our study is successful and we obtain FDA approval, our allogenic cell therapy could enable one donated liver to treat many dozens of ESLD patients, which could help to tilt the current organ supply-demand imbalance in favor of patients.”

Pittsburgh-based LyGenesis uses patients’ lymph nodes as bioreactors to foster the growth of fully functional ectopic organs. The approach aims to harness the body’s natural processes, with the lymph nodes serving as in vivo bioreactors, facilitating the engraftment, proliferation, and generation of functional ectopic liver tissue.

A first-of-its-kind treatment, LYG-LIV-001 is regulated by the FDA as a Section 351 Human Cellular Therapy/Product and an investigational biologic product. It involves a meticulous process starting from donated unmatched livers, where hepatocytes are isolated, suspended, and then transplanted into the patient’s upper abdominal lymph nodes using minimally invasive techniques.

“LyGenesis’ cell therapy platform represents a truly remarkable potential commercial opportunity and may be transformative for chronic liver failure patients who do not have access to a donor liver,” said Justin Briggs from Prime Movers Lab, an investor in LyGenesis. “Their use of an endoscopic ultrasound as a low risk and low-cost route of cell therapy administration is another way this pioneering technology could provide patients with access to life-saving therapies and address complex medical challenges by upending transplant medicine.”

Beyond liver disease, Lygenesis, which secured $19 million in funding last year, is also leveraging its platform to address conditions such as aging, Type 1 diabetes, and renal disease.

“This clinical trial heralds more than an important milestone in the study of bench-to-bedside translational medicine, it is a prime example of LyGenesis’s ongoing investigational efforts to translate cutting-edge innovative scientific discoveries, such as using the lymph nodes as a bioreactor to grow functioning ectopic organs, into clinical reality for patients suffering from life-threatening illnesses,” said Dr Richard Marshall of Juvenescence, also an investor in LyGenesis.

Photograph courtesy of LyGenesis