Early intervention for children born with brittle bone disease
BT-101 is a Phase III-ready investigational therapy with disease-modifying potential in severe pediatric Osteogenesis Imperfecta
ORPHAN DRUG DESIGNATION
ODD in EU & US
RARE PEDIATRIC DISEASE DESIGNATION
FDA RPDD
SCIENTIFIC ORIGIN
Karolinska Institutet
The case for BT-101
High unmet medical need and a Phase III-ready program for severe pediatric Osteogenesis Imperfecta (OI)
01
The disease burden
Severe OI begins early
In severe pediatric OI, defective bone formation leads to fragile bones, with fractures and skeletal deformities occurring from the earliest stages of development.
02
The treatment gap
No disease-modifying treatment
Current care can help manage fractures, pain and other manifestations of OI, but no approved disease-modifying treatment is currently available.
03
The BT-101 response
Built for early intervention
BT-101 is designed to target defective bone formation before fracture burden accumulates and skeletal deformities progress.
As an off-the-shelf product, BT-101 is manufactured in advance rather than individually for each patient, supporting timely availability and scalable supply.
BT-101 also requires no conditioning or other pretreatment, allowing treatment without added burden for children or complexity for clinical teams.
Explore BT-101 ›
Clinical & Regulatory Status
A Phase III-ready program
BT-101 is in clinical development for severe pediatric OI. The Phase I/II BOOSTB4 study evaluated repeated administration and provided clinical experience to support the planned pivotal Phase III study.
BOOST Pharma is now focused on the clinical, regulatory and operational activities required to enable initiation of the planned pivotal study.
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Regulatory recognition
BT-101 has received key regulatory designations in Europe and the United States, reflecting the serious unmet need in severe pediatric OI.
Scientific origin
Karolinska Institutet
Investor backing
Industrifonden
KDventures
Sound Bioventures
BioInnovation Institute
BOOSTB4 study support
EU Horizon 2020
Swedish Research Council
Latest Updates
EVENTS
June 22, 2026
BOOST Pharma presents BT-101 at RESI San Diego 2026
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EVENTS
May 19, 2026
BOOST Pharma presents at the 3rd ATMP Mini-Symposium in Lund
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PRESS RELEASE
March 19, 2026
Elaine Jones Appointed as Board Chair of BOOST Pharma
Read more ›
View all News & Events ›
Partnering and Investment
BOOST Pharma welcomes partnering and investment conversations to accelerate development of BT-101.
Contact ›
Explore Next
Science
Biological and scientific rationale
BT-101
Program concept and treatment approach
Clinical
Development progress and next steps
Company
Team, board, and scientific advisors