The startup develops a stem cell therapy utilizing novel mesenchymal stem cells with enhanced osteogenic potential for the treatment of osteogenesis imperfecta. This therapy enables targeted bone formation by allowing the stem cells to migrate and engraft in the patient's bone, facilitating early intervention and improving long-term patient outcomes.
Funding
$550K raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
KFounders
Product
Problem
Osteogenesis Imperfecta (OI), or brittle bone disease, results in fragile bones, frequent fractures, deformities, pain, and disability, with limited palliative therapies available and no approved treatments addressing the underlying genetic cause. Current treatments like physiotherapy, surgery, and bisphosphonates do not reduce fracture frequency, leaving over 56,000 individuals in the US and EU with severe disabilities.
Solution
BOOST Pharma is developing BT-101, a first-in-class cell therapy using mesenchymal stem cells (MSCs) with enhanced bone-forming capabilities to treat OI by addressing the underlying collagen deficiency. BT-101 cells migrate to the patient's bones, engraft, and initiate bone formation, leading to higher calcium deposition and increased bone strength. Clinical data from the BOOSTB4 Phase I/II trial demonstrated a significant reduction in fracture rates in children with severe OI, with treated children following their own growth curve and showing increased lengthwise growth. The therapy is designed for administration prenatally or shortly after birth to maximize benefits, with postnatal infusions every four months during childhood and adolescence.
Target Audience
The primary target audience includes children diagnosed with Osteogenesis Imperfecta (OI) Type III/IV and their families, as well as clinicians specializing in rare bone diseases and fetal therapies.
Features
- Novel mesenchymal stem cell (MSC) therapy with high bone-forming capabilities (BOOST Cells/BT-101)
- Prenatal and postnatal administration options for early intervention
- Demonstrated reduction in fracture rates in Phase I/II clinical trials
- Improved bone quality through increased calcium deposition and alkaline phosphatase activity
- Allogeneic MSCs considered safe with no adverse reactions or immune responses observed
- Orphan Drug Designation in both the EU and USA
- Rare Pediatric Disease (RPD) Designation by the FDA