BOOST Pharma
BOOST Pharma is a clinical-stage biopharmaceutical company developing BT-101, a first-in-class allogeneic mesenchymal stem cell therapy for children with Osteogenesis Imperfecta (Brittle Bone Disease), a rare genetic disorder with no approved disease-modifying treatments.
- Company typePrivate
- Founded2020
- HeadquartersCopenhagen, Denmark
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
What BOOST Pharma does
BOOST Pharma is a clinical-stage biopharmaceutical company developing BT-101, a first-in-class allogeneic mesenchymal stem cell (MSC) therapy for the treatment of Osteogenesis Imperfecta (OI), a rare genetic disorder also known as Brittle Bone Disease. The company was founded in 2019-2020 based on collaborative research from Karolinska Institutet in Stockholm, with headquarters at the Bio Innovation Institute in Copenhagen, Denmark. BT-101 is built on proprietary "BOOST Cells" that demonstrate higher calcium deposition, higher alkaline phosphatase activity, and high ectopic bone formation in preclinical models. Treatment involves four doses of allogeneic MSCs every fourth month, with the company specifically pursuing early intervention starting at prenatal diagnosis or as early as possible after birth to maximize therapeutic benefit for children with Type III and IV OI.
The company's lead clinical program, the BOOSTB4 Phase I/II multi-centre trial, has produced a strong efficacy signal: over 75% reduction in bone fracture rates at one year, approximately 78% reduction at two years, and over 50% of treated patients with zero fractures in the second year of follow-up. The product has been administered with a clean safety profile, with no adverse reactions or immune responses against donor MSCs. BOOST Pharma has secured Orphan Drug Designation in both the EU (December 2021) and the US (May 2022), as well as FDA Rare Paediatric Disease Designation (October 2022), positioning the program for market exclusivity and eligibility for a Priority Review Voucher upon marketing authorization. Following a successful FDA pre-IND meeting in November 2024, the company is preparing for a pivotal Phase III study in the US and Europe, with manufacturing being transferred to RoslinCT in Edinburgh for GMP production.
As a pre-revenue clinical-stage company, BOOST Pharma's commercial model relies on three planned revenue streams upon regulatory approval: (1) direct cell therapy product sales distributed through specialty pharmacies, hospital pharmacies, and rare pediatric treatment centers; (2) licensing or strategic partnership income from larger pharmaceutical companies for co-development or regional commercialization rights; and (3) potential monetization of the Priority Review Voucher from the Rare Paediatric Disease Designation. The customer base is narrow by design, focused on the estimated 56,000+ OI patients across the US and EU, with the company engaging regulatory authorities, clinical trial sites, and patient advocacy organizations such as OIFE. Leadership has been substantially upgraded in late 2025 and early 2026 with the appointments of CEO Hans Schambye (former Galecto Inc. CEO), Board Chair Elaine Jones (former Pfizer Ventures), and CRO Louise Himmelstrup to support Phase III execution and a future commercialization or partnership transaction.
BOOST Pharma firmographics
Firmographics- Name
- BOOST Pharma
- Legal name
- BOOST Pharma
- Website
- https://boostpharma.com
- Company type
- Private
- Founded year
- 2020
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- BOOST Pharma is a clinical-stage biopharmaceutical company developing BT-101, a first-in-class allogeneic mesenchymal stem cell therapy for children with Osteogenesis Imperfecta (Brittle Bone Disease), a rare genetic disorder with no approved disease-modifying treatments.
- Ownership category
- akta.pro rank
BOOST Pharma industry classification
Industry- Product category
- Rare Disease Biopharmaceuticals
- NAICS
- Biological Product (except Diagnostic) Manufacturing (325414), Pharmaceutical and Medicine Manufacturing (3254), Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)
- akta.pro primary industry
- Stem Cell–Based Regenerative Therapies (HLAAAGAA)
- akta.pro secondary industries
- Stem Cell Therapies (MSC/iPSC/HSC-derived) (HLAAABAE), Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)
Keywords
Where BOOST Pharma is headquartered
LocationHeadquarters
- HQ city
- Copenhagen
- HQ country
- Denmark
- HQ region
- Europe
Offices2 records
Markets served
BOOST Pharma business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Supply Chain, Marketing or Sales
Revenue model
- Cell Therapy Product Sales: Once BT-101 receives regulatory approval, the company plans to commercialize its cell therapy treatment for Osteogenesis Imperfecta through traditional pharmaceutical distribution channels, targeting hospitals and specialized treatment centers treating rare pediatric bone diseases.
- Priority Review Voucher (PRV): The FDA's Rare Paediatric Disease (RPD) Designation allows BOOST Pharma to receive a priority review voucher upon marketing authorization, which can be sold to other pharmaceutical companies - representing a potential additional revenue stream.
- Strategic Partnerships and Licensing: The company may pursue licensing agreements or strategic partnerships with larger pharmaceutical companies for co-development, commercialization rights, or regional partnerships for BT-101 distribution.
Go-to-market motion2 records
Distribution channels2 records
Marketing channels5 records
BOOST Pharma product offering
Product offeringCore offering
BOOST Pharma is a clinical-stage biopharmaceutical company developing BT-101, an allogeneic mesenchymal stem cell (MSC) therapy using proprietary BOOST Cells that migrate to bone, engraft and form new bone tissue. The therapy targets Osteogenesis Imperfecta (Brittle Bone Disease), a rare genetic pediatric disorder, with treatment administered as four doses every fourth month beginning at prenatal diagnosis or as early as possible after birth.
Product overview
BOOST Pharma is a clinical-stage biopharmaceutical company developing a single lead product, BT-101, an off-the-shelf allogeneic mesenchymal stem cell therapy for Osteogenesis Imperfecta (OI). BT-101 is administered through the BOOSTB4 Phase I/II clinical trial and works by migrating to patient bones where cells engraft and initiate bone formation. Treatment can begin at the prenatal stage when OI is first diagnosed, or as early as possible after birth, targeting early intervention for maximum therapeutic benefit.
Differentiator
Problem solved
Functional benefit
Products and services
- BT-101 (BOOST Cells) allogeneic mesenchymal stem cell therapy First-in-class allogeneic mesenchymal stem cell (MSC) therapy with high bone-forming capabilities for the treatment of Osteogenesis Imperfecta (OI / Brittle Bone Disease). Administered as four doses every fourth month, beginning at prenatal diagnosis or as early as possible after birth, with cells migrating to bone, engrafting and forming new bone tissue. Targets pediatric patients (Types III and IV OI) with no currently approved disease-modifying therapy.
Quantifiable outcome
- Over 75% reduction in bone fracture rate observed in Phase I/II trial
- +3 more outcomes
Companies that use BOOST Pharma
Customer profileSegments1 record
Ideal customer profiles2 records
BOOST Pharma technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
BOOST Pharma partnerships and signals
Strategic signalPartnerships
Two partnerships are on record, tiered core.
- RoslinCTcoreStrategic manufacturing partnership between RoslinCT and BOOST Pharma to support Phase III development of BT-101, an allogeneic mesenchymal stem cell therapy for Osteogenesis Imperfecta. BOOST Pharma is transferring its manufacturing process to RoslinCT's Edinburgh facilities for GMP production.
- Karolinska InstitutetcoreBOOST Pharma is based on collaborative research from Karolinska Institutet in Stockholm. Co-founder Dr. Cecilia Götherström is Associate Professor at Karolinska Institutet, and co-founder Professor Magnus Westgren is Senior Professor there. The company was established based on the research teams' work showing that BOOST cell treatment greatly enhanced quality of life for OI patients.
Scale indicators8 records
Recent moves6 records
Expansion highlights5 records
BOOST Pharma competitors and assessment
Company assessmentEmerging players
- Gamida Cell: Gamida Cell develops cell therapies for hematologic malignancies and rare diseases. Similar to BOOST Pharma, they are a clinical-stage biotech with FDA-approved cell therapy products and use advanced cell manufacturing capabilities.
- Capricor Therapeutics: Capricor develops cell-based therapies for Duchenne muscular dystrophy and other rare diseases. Both companies are clinical-stage biotechs developing innovative cell therapies for rare diseases with high unmet need.
Broad incumbents
- BioMarin Pharmaceutical: BioMarin is a major rare disease biopharmaceutical company. They are also developing therapies for OI and represent a broader incumbent with the resources to develop and commercialize rare disease therapies globally.
- Alexion (AstraZeneca Rare Disease): Alexion is a leading rare disease biopharmaceutical company acquired by AstraZeneca. They have extensive experience commercializing high-priced orphan drugs and could be a potential acquirer or partner for BOOST Pharma's BT-101.
- Ultragenyx Pharmaceutical: Ultragenyx is a rare disease biopharmaceutical company with setrusumab in development for Osteogenesis Imperfecta. While a broader rare disease player, they represent direct competition in the OI space with an alternative therapeutic approach.
Direct peers
- Athersys: Athersys develops MultiStem, an allogeneic stem cell therapy platform for inflammatory, neurological, and cardiovascular conditions. Similar to BOOST Pharma, it focuses on off-the-shelf cell therapy products targeting diseases with significant unmet medical need.
- Mesoblast: Mesoblast develops allogeneic mesenchymal stem cell (MSC) therapies for inflammatory and degenerative diseases. Both companies leverage MSC technology platforms with proprietary manufacturing processes, serving as direct competitors in the allogeneic cell therapy space.
- TiGenix (acquired by Takeda): TiGenix developed Cx601, an allogeneic MSC therapy for Crohn's disease that received EMA approval. As a pioneer in commercializing allogeneic MSC therapies, they represent a relevant benchmark for BOOST Pharma's regulatory and commercialization pathway.
- Pluristem Therapeutics: Pluristem develops placenta-derived mesenchymal-like stem cell therapies. Both companies are clinical-stage biotechs focused on allogeneic cell therapies with proprietary manufacturing platforms and similar regulatory pathways.
Others
- RoslinCT: RoslinCT is a contract development and manufacturing organization (CDMO) specializing in cell and gene therapies. They are BOOST Pharma's strategic manufacturing partner for Phase III and commercial supply of BT-101.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks5 records
Key highlights7 records
Customer concentration
BOOST Pharma social profiles
Digital presenceBOOST Pharma compliance and trust
Trust signalCompliance3 records
BOOST Pharma financial estimates
Financial estimateRevenue estimate
Valuation estimate
BOOST Pharma leadership team
Management profileNumber of profiles
Profiles6 records
BOOST Pharma funding detail
Funding detailFunding overview
Funding rounds4 records
Investors4 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
BOOST Pharma M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about BOOST Pharma
What does BOOST Pharma do?
BOOST Pharma is a clinical-stage biopharmaceutical company developing BT-101, an allogeneic mesenchymal stem cell (MSC) therapy using proprietary BOOST Cells that migrate to bone, engraft and form new bone tissue. The therapy targets Osteogenesis Imperfecta (Brittle Bone Disease), a rare genetic pediatric disorder, with treatment administered as four doses every fourth month beginning at prenatal diagnosis or as early as possible after birth.
Is BOOST Pharma a public or private company?
BOOST Pharma is a private company. It is classified as venture growth investor backed and is currently operating.
When was BOOST Pharma founded?
BOOST Pharma was founded in 2020. It employs 1 to 10 people.
Where is BOOST Pharma based?
BOOST Pharma is headquartered in Copenhagen, Denmark, in the Europe region.
How does BOOST Pharma make money?
Three revenue lines are on record. Cell Therapy Product Sales are the primary driver. The others are priority Review Voucher (PRV) and strategic Partnerships and Licensing.
Who are BOOST Pharma's main competitors?
Emerging players on record are Gamida Cell and Capricor Therapeutics. Broad incumbents are BioMarin Pharmaceutical, Alexion (AstraZeneca Rare Disease) and Ultragenyx Pharmaceutical. Direct peers are Athersys, Mesoblast, TiGenix (acquired by Takeda) and Pluristem Therapeutics. RoslinCT is listed as an others.
Does BOOST Pharma have an API?
No public API is recorded for BOOST Pharma.
What industry is BOOST Pharma in?
BOOST Pharma's product category is Rare Disease Biopharmaceuticals. Its primary akta.pro industry code is HLAAAGAA, Stem Cell–Based Regenerative Therapies, with a secondary code of HLAAABAE, Stem Cell Therapies (MSC/iPSC/HSC-derived). Its NAICS code is 325414 and its SIC code is 2836.