BOOST Pharma is pioneering a first-in-class cell therapy aimed at treating Osteogenesis Imperfecta (OI), a rare genetic disorder characterized by fragile bones. The company utilizes mesenchymal stem cells (MSCs), referred to as BOOST Cells, which have demonstrated significant bone-forming capabilities. Preclinical studies in animal models have shown that these cells can enhance calcium deposition and promote ectopic bone formation. Human proof-of-concept studies have indicated that treatment with BOOST Cells leads to a notable reduction in fracture rates and improved growth trajectories in children with OI, showcasing the therapy's potential effectiveness and safety.

The company has achieved several key milestones in its clinical development program, including the successful completion of Phase I/II trials and obtaining orphan drug designations in both the U.S. and Europe. BOOST Pharma's approach involves administering BOOST Cells either prenatally or postnatally, with treatment regimens tailored to individual patient needs. The ongoing clinical trials, such as BOOSTB4 in Europe and BOOST2B in India, are designed to further evaluate the safety and efficacy of this innovative therapy, with plans for a pivotal Phase III trial on the horizon. Through these advancements, BOOST Pharma aims to address a significant unmet medical need for children suffering from OI.

Denmark

BOOST Pharma is based on years of collaborative research from Karolinska Institute in Stockholm with the focus on novel cell therapy treatments for Osteogenesis Imperfecta. The research teams of associate professor Cecilia Götherström and professor Magnus Westgren have shown that that treatment with BOOST Cells greatly enhanced the quality of life for patients suffering from this otherwise extremely debilitating disease.

BOOST Pharma

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