Systemic mastocytosis and KIT-driven hematologic disease
Blueprint Medicines Corp. is active in advanced systemic mastocytosis, indolent systemic mastocytosis, and related KIT-altered blood cancers, with research also extending to SM-AHN and other mast cell disorders.
- Advanced systemic mastocytosis
- Indolent systemic mastocytosis
- KIT-altered hematologic malignancies
- SM-AHN
Its clinical portfolio in this area includes therapies aimed at controlling mast cell disease activity and addressing symptom burden in both adult and pediatric settings.
Allergic and inflammatory skin disease
The sponsor also funds research in chronic inducible urticaria and chronic spontaneous urticaria, focusing on conditions marked by persistent itch, wheals, and recurrent flare patterns.
- Chronic inducible urticaria
- Chronic spontaneous urticaria
- Urticaria-related symptom control
This area reflects a therapeutic interest in non-oncologic immune-mediated disease with emphasis on symptom relief and long-term disease management.
Advanced solid tumors and targeted oncology
Blueprint Medicines Corp. is involved in studies across advanced solid tumors, including cancers with EGFR exon 20 insertions, CCNE1 amplification, and tumors dependent on KIT or PDGFRA signaling.
- Advanced solid tumors
- EGFR exon 20 insertion cancers
- CCNE1 amplification
- KIT– or PDGFRA-dependent tumors
The oncology program includes interest in both monotherapy and combination-based treatment strategies for genetically defined malignancies.
Breast cancer, ovarian cancer, endometrial cancer, and gastric cancer
The company’s cancer research also spans HR-positive breast cancer, HER2-negative breast cancer, ovarian cancer, endometrial cancer, and gastric cancer, with emphasis on difficult-to-treat solid tumors.
- HR-positive breast cancer
- HER2-negative breast cancer
- Ovarian cancer
- Endometrial cancer
- Gastric cancer
These studies reflect a broad interest in tumor biology across hormonally driven and gynecologic malignancies as well as gastrointestinal cancer.
Pediatric oncology
Blueprint Medicines Corp. also supports research in pediatric solid tumors, particularly cancers linked to KIT or PDGFRA signaling.
- Pediatric solid tumors
- KIT-dependent cancers
- PDGFRA-dependent cancers
This area extends the sponsor’s targeted oncology focus into younger patient populations with molecularly defined disease.
