NEWTON, Mass. — Satellite Biosciences Inc. said the U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to SB-101, its lead liver therapy candidate for the treatment of urea cycle disorders.
Satellite Bio is developing off-the-shelf liver therapies intended to restore liver function in patients with severe liver diseases. SB-101 is being developed as a first-in-class off-the-shelf liver therapy for infants with severe early-onset urea cycle disorders. The company plans to begin a Phase 1/2 clinical trial of the therapy in 2026.
Urea cycle disorders are serious, life-threatening conditions that can cause neurocognitive deficits, behavioral impairment, encephalopathy, coma, seizures, organ failure and death. The disorders primarily affect people from birth to age 18. Satellite Bio said current standards of care remain inadequate, with mortality rates exceeding 25%, cognitive impairment affecting about half of patients and no curative treatment available in the first weeks of life.
“This designation marks an important milestone for Satellite Bio and reinforces the potential of our approach to restore essential liver function for patients with severe liver diseases,” said Tom Lowery, PhD, President and Chief Executive Officer of Satellite Bio. “UCDs are some of the most devastating diseases affecting infants with no curative treatments available in the first weeks of life. We look forward to advancing SB-101 in clinical trials this year and expanding treatment options for individuals with UCDs.”
Satellite Bio said SB-101 is intended to help restore metabolic liver function and protect the developing brain. The therapy is designed to deliver healthy liver cells into the body to replace lost liver function and restore essential biological processes.
The company’s platform uses a proprietary cell expansion and manufacturing process designed for large-scale production. Satellite Bio said SB-101 is manufactured in advance, cryopreserved and ready when needed, with the goal of improving availability, reliability and cost compared with conventional cell therapies.
The liver performs more than 500 vital functions, including detoxifying harmful substances, processing nutrients and producing essential blood proteins. Severe liver diseases occur when hepatocytes, the liver’s functional cells, can no longer perform those functions.
Satellite Bio’s pipeline also includes additional drug candidates designed to improve liver function in other pediatric liver diseases and in adults with chronic liver disease.
Rare Pediatric Disease designation is granted to drug candidates intended to treat serious or life-threatening diseases that primarily affect children from birth to age 18. If a qualifying marketing application is approved, the sponsor may be eligible for a Priority Review Voucher, which can be used for priority review of a future application or sold or transferred to another sponsor.
