Newton, Mass. — Satellite Biosciences, Inc. has received U.S. Food and Drug Administration clearance for its Investigational New Drug application for SB-101, an off-the-shelf liver cell therapy being developed to treat urea cycle disorders.
The clearance allows the company to begin clinical testing and marks Satellite Bio’s transition into a clinical-stage biotechnology company.
Satellite Bio plans to initiate a Phase 1/2 trial in late 2026 evaluating SB-101 in infants with severe early-onset urea cycle disorders. The study will assess the therapy’s safety, tolerability, pharmacodynamic effects and preliminary efficacy.
SB-101 is designed to deliver healthy liver cells that can replace lost metabolic function, including the liver’s ability to remove toxic ammonia from the body. The therapy is manufactured in advance, cryopreserved and intended to be available for urgent use.
“We are thrilled to transition to a clinical-stage company with this cleared IND,” said Tom Lowery, Ph.D., President and Chief Executive Officer of Satellite Bio.
“While Satellite Bio is initially focused on UCDs, our platform’s ability to deliver healthy hepatocytes to address a broad range of liver diseases means the clinical entry of SB-101 is the first of a much broader pipeline,” Lowery said.
Urea cycle disorders are rare inherited conditions in which the liver cannot properly convert ammonia into urea for elimination. The resulting ammonia buildup can cause seizures, encephalopathy, irreversible brain injury, coma, organ failure and death.
Satellite Bio said mortality rates exceed 25 percent and cognitive impairment affects about half of surviving patients. Curative treatment is not available during the first weeks of life, when infants with severe forms of the disease may need urgent intervention.
The company said SB-101 is intended to restore essential liver function and help protect the developing brain while patients await longer-term treatment options.
“We believe SB-101 has the potential to be a transformative treatment for individuals with early-onset UCDs as well as other severe liver diseases,” Lowery said.
The FDA previously granted SB-101 Rare Pediatric Disease designation.
Satellite Bio’s platform uses a proprietary cell expansion and manufacturing process designed to produce healthy hepatocytes at commercial scale. The company aims to deliver the cells to sites in the body where they can engraft and restore missing liver functions.
The company said the platform could eventually support therapies for a broader range of severe liver diseases.
