All Therapeutic Areas
🧬
Rare & Genetic Diseases
53 drugs in pipeline from 10 companies
53
Drugs in Pipeline
10
Companies
0
Upcoming Catalysts
53
Phase 3 Trials
Upcoming Catalysts
No upcoming catalysts found for Rare & Genetic Diseases
Pipeline by Phase
Phase 3
53
Active Companies
GILD
2 drugsGilead Sciences, Inc.
SKBBY
2 drugsSichuan Kelun-Biotech Biopharmaceutical Co., Ltd./ADR
AZNCF
6 drugsASTRAZENECA PLC
CELG-RI
1 drugsBRISTOL MYERS SQUIBB CO
MRK
5 drugsMERCK
NVSEF
1 drugsNOVARTIS AG
SNYNF
2 drugsSanofi
AZN
4 drugsAstraZeneca PLC
TAK
1 drugsTAKEDA PHARMACEUTICAL CO LTD
ABT
1 drugsABBOTT LABORATORIES
Related Conditions
rare diseaseorphangeneticgene therapyhereditarymuscular dystrophycystic fibrosissickle cellhemophiliathalassemiagaucherfabrypompehunter syndromephenylketonuriapkusmaspinal muscular atrophy