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Search for companies, drugs, and catalysts

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Search for companies, drugs, and catalysts

All Therapeutic Areas
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Rare & Genetic Diseases

59 drugs in pipeline from 10 companies

59

Drugs in Pipeline

10

Companies

8

Upcoming Catalysts

59

Phase 3 Trials

Upcoming Catalysts
Jun '26
VSTMPhase 2

avutometinib and sotorasib Phase 2 Est. Readout

avutometinib and sotorasib153
Jun 2026~
CT.gov
00%
Jun '26
MOLNPhase 2

4D-310 Phase 2 Est. Readout

4D-31018
Jun 2026~
CT.gov
00%
Jun '26
FOLDPhase 3

Cipaglucosidase Alfa Phase 3 Est. Readout

Cipaglucosidase Alfa21
Jun 2026~
CT.gov
00%
Jun '26
RCUSPhase 3
Negative

Zimberelimab Phase 3 Results Expected

Zimberelimab1,040
Jun 2026~
CT.gov
Jun '26
GNPXPhase 2

quaratusugene ozeplasmid Phase 2 Est. Readout

quaratusugene ozeplasmid62
Jun 2026~
CT.gov
00%
Jun '26
SKBBYPhase 2

PD-1 monoclonal antibody Phase 2 Est. Readout

PD-1 monoclonal antibody66
Jun 2026~
CT.gov
00%
Jun '26
TLXPhase 3

68Ga-PSMA-11 Phase 3 Est. Readout

68Ga-PSMA-11204
Jun 2026~
CT.gov
00%
2d
days
SNYPDUFA
Phase 3Approved

FDA PDUFA Date Fitusiran (priority)

Fitusiran
Jun 16
SEC
Pipeline by Phase
Phase 3
59
Related Conditions
rare diseaseorphangeneticgene therapyhereditarymuscular dystrophycystic fibrosissickle cellhemophiliathalassemiagaucherfabrypompehunter syndromephenylketonuriapkusmaspinal muscular atrophy