Cell therapy that can reduce fractures in a rare bone disease
BOOST Pharma is developing BT-101, a first-in-class cell therapy for children with osteogenesis imperfecta, a congenital disease that causes very fragile bones.
Industry pharma
Target year 2029
Ownership 14%
BOOST Pharma is developing a cell therapy for osteogenesis imperfecta (OI), a rare genetic disease that leads to fragile bones, recurrent fractures and reduced mobility. The treatment is based on mesenchymal stem cells with the ability to contribute to bone formation and can be administered early in life.
The Phase 1/2 clinical study BOOSTB4 has shown a favourable safety profile and a substantial reduction in the number of fractures. The company is now preparing BT-101 for registration-enabling Phase 3 development.
In brief:
- Nearly 78% reduction in fractures compared with before treatment
- Over 50% of patients were fracture-free during the second year after the last dose
- Phase 3 preparations are ongoing