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Search for companies, drugs, and catalysts

All Therapeutic Areas
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Rare & Genetic Diseases

50 drugs in pipeline from 10 companies

50

Drugs in Pipeline

10

Companies

5

Upcoming Catalysts

50

Phase 3 Trials

Upcoming Catalysts
Sep '26
EWTXPhase 2
Positive

Sevasemten 10 mg Phase 2 Est. Readout

Sevasemten 10 mg
Sep 2026~
CT.gov
Sep '26
BAYRYPhase 2
Positive

BAY2599023 (DTX201) Phase 2 Est. Readout

BAY2599023 (DTX201)
Sep 2026~
CT.gov
2w
NUVLPDUFA
Phase 3Positive

FDA PDUFA Date NUV-1034 (standard)

Neladalkib (NVL-655)
Sep 26
SEC
Sep '26
MKKGYPhase 2

M1774 Phase 2 Est. Readout

M1774
Sep 2026~
CT.gov
00%
3w
RVMDRegulatory
Phase 3Positive

Regulatory Decision - daraxonrasib

daraxonrasib
Sep 30
Source
Pipeline by Phase
Phase 3
50
Related Conditions
rare diseaseorphangeneticgene therapyhereditarymuscular dystrophycystic fibrosissickle cellhemophiliathalassemiagaucherfabrypompehunter syndromephenylketonuriapkusmaspinal muscular atrophy