Science
The scientific rationale for early intervention in severe pediatric Osteogenesis Imperfecta
OI biology and lifelong burden
Osteogenesis Imperfecta (OI), also known as brittle bone disease, is a rare genetic connective tissue disorder. Many cases involve mutations affecting type I collagen, a key structural protein in the bone matrix. This disrupts normal bone formation, resulting in fragile bone and abnormal skeletal development.
In severe pediatric OI, this can lead to fractures, skeletal deformities and pain from the earliest stages of life, sometimes even before birth. These consequences can continue throughout life, affecting growth, mobility, independence and daily function.
The need for a new treatment approach
Current treatment gap
Current care can help manage fractures, pain and other manifestations of OI, but there is still no approved disease-modifying treatment.
Early intervention opportunity
Intervening during early skeletal development offers the potential to influence disease progression before fracture burden accumulates and deformities progress.
The BT-101 approach
BT-101 is based on selected mesenchymal stem cells (MSCs) with high osteogenic potential and the ability to produce normal type I collagen.
By introducing healthy MSCs early in skeletal development, the approach is designed to compensate for defective bone formation, with the aim of limiting its downstream consequences.
Together, these cellular properties and the timing of intervention provide the biological rationale for BT-101’s disease-modifying potential in severe OI.
Scientific origins and evidence base
BOOST’s program builds on long-standing academic research in regenerative medicine, bone biology and Osteogenesis Imperfecta, with scientific origins at Karolinska Institutet in Sweden.
Ongoing academic collaboration and translational research continue as BOOST prepares for the planned pivotal Phase III study.
Selected publications
An exploratory open-label multicentre Phase I/II trial…
Sagar et al. • BMJ Open (2024)
Stem Cell Therapy as a Treatment for Osteogenesis Imperfecta
Götherström C, Walther-Jallow L. • Current Osteoporosis Reports (2020)
Prenatal stem cell therapy for inherited diseases: Past, present, and future treatment strategies
Ekblad-Nordberg Å, Walther-Jallow L, Westgren M, Götherström C. •
Stem Cells Translational Medicine (2020)
Fetal mesenchymal stem-cell engraftment in bone after in utero transplantation in a patient with severe Osteogenesis Imperfecta
Le Blanc et al. • Transplantation (2005)
Explore Next
BT-101
Program concept and treatment approach
Clinical
Development progress and next steps
Company
Team, board, and scientific advisors