NEWTON, Mass. — Satellite Biosciences, Inc. (Satellite Bio), a biotechnology company developing off-the-shelf liver therapies designed to restore liver function in patients with severe liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SB-101 for the treatment of urea cycle disorders (UCDs).
UCDs are a class of devastating conditions in which serious or life-threatening manifestations, such as neurocognitive deficits, behavioral impairment, encephalopathy, coma, seizures, organ failure, and mortality, primarily affect individuals aged from birth to 18 years. The current standard of care for UCDs remains inadequate, with mortality rates exceeding 25%, cognitive impairment affecting half of the 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’s lead program, SB-101, is a first-in-class off-the-shelf liver therapy being developed as a treatment for infants with severe early onset UCD. The company plans to initiate a Phase 1/2 clinical trial of SB-101 in 2026. Satellite Bio’s pipeline also includes additional drug candidates designed to improve liver function in other pediatric liver diseases and adult patients with chronic liver disease.
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 these critical activities.
Satellite Bio is developing off-the-shelf therapies that deliver healthy liver cells into the body to replace lost liver function and restore the vital processes patients need to survive. Satellite Bio’s therapies are enabled by a proprietary cell expansion and manufacturing process designed for large-scale production. Manufactured in advance, cryopreserved, and ready when needed, SB-101 is intended to offer the availability, reliability, and cost profile superior to conventional cell therapies. This platform supports multiple therapeutic strategies by delivering healthy liver cells to receptive sites in the body, where they can engraft and restore function. Just as prior breakthrough biologic therapies reshaped treatment across cancer and autoimmune diseases, Satellite Bio aims to harness living cells as medicines to address severe liver disease at its source.
RPD designation is granted to drug candidates intended to treat serious or life-threatening diseases that primarily affect children from birth to age 18. Upon approval of a qualifying marketing application, the sponsor may be eligible for a Priority Review Voucher, which can be used to obtain priority review for a future application or sold or transferred to another sponsor.
About Satellite Bio
Satellite Bio is a biotechnology company developing off-the-shelf liver therapies designed to address dysfunctional liver cells, the root cause of hepatic insufficiency, with the goal of restoring failing cellular function and improve liver performance in patients with severe life-threatening liver diseases. By combining deep biological understanding with a proprietary, scalable manufacturing and two complementary therapeutic strategies, the company is positioned to redefine how hepatic insufficiency is treated. For more information, visit www.satellite.bio.
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