BT-101
Built for early intervention and practical use
in severe pediatric OI
BT-101 in brief
BT-101 is BOOST Pharma’s lead investigational therapy for children with severe Osteogenesis Imperfecta (OI), also known as brittle bone disease.
It is based on selected mesenchymal stem cells (MSCs) and uses an allogeneic product model, meaning the cells originate from a donor cell source rather than being produced from each patient’s own cells.
BT-101 is being developed as an off-the-shelf therapy for early intervention during skeletal development.
Indication
Severe pediatric Osteogenesis Imperfecta (OI)
Modality
Allogeneic cell therapy
Development
Phase III-ready
How BT-101 is intended to work
BT-101 combines selected MSCs with high osteogenic potential and early intervention during skeletal development.
Their capacity to form healthy bone is intended to help compensate for defective bone formation in OI and limit its downstream consequences.
Together, the functional properties of the cells and the timing of intervention underpin BT-101’s disease-modifying potential in severe OI.
Designed for practical pediatric use
Off-the-shelf availability
BT-101 is being developed as a cryopreserved, off-the-shelf product that can be manufactured in advance, supporting timely availability when needed.
Standardized and scalable
BT-101 is not manufactured from each patient’s own cells, allowing production to be standardized rather than patient-specific.
No conditioning required
BT-101 requires no patient matching, conditioning or immunosuppression, allowing treatment without added burden for children or complexity for clinical teams.
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Science
Biological and scientific rationale
Clinical
Development progress and next steps
Company
Team, board, and scientific advisors