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All Therapeutic Areas

🧬

Rare & Genetic Diseases

73 drugs in pipeline from 10 companies

73

Drugs in Pipeline

10

Companies

7

Upcoming Catalysts

53

Phase 3 Trials

Upcoming Catalysts

Dec 8

VRTX⭐Phase 3Phase 3

CTX001 Phase 3 Results Expected

CTX001 • Beta-Thalassemia

Dec 2025~

RYTM⭐Phase 3Phase 2

Setmelanotide Phase 3 Results Expected

Setmelanotide • Genetic Obesity

Jan 2026~

TNGXPhase 2Phase 2

TNG260 Phase 2 Results Expected

TNG260 • Non Small Cell Lung Cancer

Jan 31

CGEMPhase 2Phase 2

CLN-081 Phase 2 Results Expected

CLN-081 • Non Small Cell Lung Cancer

Mar 2026~

MRUSPhase 2Phase 2

MCLA-129 Phase 2 Results Expected

MCLA-129 • Non-Small Cell Lung Cancer Metastatic

Apr 30

VRTX⭐Phase 3Phase 3

ELX/TEZ/IVA Phase 3 Results Expected

ELX/TEZ/IVA • Cystic Fibrosis

Apr 30

VRTX⭐Phase 3Phase 3

IVA Phase 3 Results Expected

IVA • Cystic Fibrosis

View all catalysts

Pipeline by Phase

Phase 3

53

Phase 2

20

Active Companies

ArriVent BioPharma, Inc.

Astellas Pharma Inc.

Novo Nordisk A/S

Sanofi

Astria Therapeutics, Inc.

Johnson & Johnson

Avidity Biosciences, Inc.

Idorsia Ltd

Grace Therapeutics, Inc.

Mirati Therapeutics, Inc.

Related Conditions

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