Revolutionizing treatment: Tandem Bio taps into placental cell power to combat rare metabolic disorders

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Annually, the United States counts about four million newborns, each accompanied by a placenta, a temporary organ ripe with special stem cells. Tandem Biotherapeutics Inc. (TBI) and its visionary co-founders — CEO David Paolella, CSO Christineh Sarkissian, and Head of Technology Roberto Gramignoli — are at the helm of exploiting these cells for medical treatments.

The placenta, previously disregarded, is now a key asset for TBI’s team. Their cutting-edge research focuses on generating stem cells that show promise in maturing into or aiding various cell types within the body. Their initial targets are inborn errors of metabolism (IEMs) detected through newborn screening, with early evidence pointing to potential treatments for three disorders.

David Paolella is personally connected to the cause, with two of his children affected by Phenylketonuria (PKU). A known figure in the PKU community for developing Glytactin — a first-of-its-kind medical food designed for PKU management — Paolella is deeply invested in TBI’s success. He describes the stark reality for newborns with IEMs, where common foods can be harmful, necessitating a critical need for alternative therapies.

While PKU can be managed with a complex diet and emerging drugs, there’s a pressing need for more effective treatments. Dr. Sarkissian, whose early research became the PKU drug Palynziq (BMRN), highlights the severe limitations associated with current treatment modalities including liver transplants, the current last-resort treatment for many IEMs.

Dr. Gramignoli’s extensive experience with hepatocytes and translational stem cell research is central to TBI’s innovative approach. His work with placental cells has laid the groundwork for TBI’s technology, now poised for dialogue with the FDA.TBI’s placental cell therapy represents a potentially groundbreaking approach, offering hope for safer and better treatments. As the company advances toward clinical trials, its primary goal is to redefine the management of hereditary disorders through the promise of placental cells. TBI, with its combination of leadership and expertise, is eager to deliver on this promise.