CatalystAlert

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Search for companies, drugs, and catalysts

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Search for companies, drugs, and catalysts

All Therapeutic Areas
🧬

Rare & Genetic Diseases

44 drugs in pipeline from 10 companies

44

Drugs in Pipeline

10

Companies

9

Upcoming Catalysts

44

Phase 3 Trials

Upcoming Catalysts
Oct '26
MRTXPhase 2

Adagrasib Phase 2 Est. Readout

Adagrasib
Oct 2026~
CT.gov
00%
Oct '26
VRTXPhase 3

VX-121/TEZ/D-IVA Phase 3 Est. Readout

VX-121/TEZ/D-IVA
Oct 2026~
CT.gov
00%
Oct '26
SNYPhase 2

venglustat (GZ402671) Phase 2 Est. Readout

Venglustat (GZ402671)
Oct 2026~
CT.gov
00%
Oct '26
SRPTPhase 3

Eteplirsen Phase 3 Est. Readout (MIS51ON)

Eteplirsen
Oct 2026~
CT.gov
00%
Oct '26
TAKPhase 2

TAK-788 Phase 2 Est. Readout

TAK-788
Oct 2026~
CT.gov
00%
Oct '26
RCUSPhase 2

Zimberelimab Phase 2 Est. Readout

Zimberelimab
Oct 2026~
CT.gov
00%
Oct '26
RYTMPhase 2

Setmelanotide Phase 2 Est. Readout

Setmelanotide
Oct 2026~
CT.gov
00%
4d
AGIOPDUFA
Phase 3

FDA PDUFA Date mitapivat (sickle cell disease)

Mitapivat
Nov 1
Source
00%
Nov '26
SRRKPhase 3

Apitegromab Phase 3 Est. Readout (ONYX)

Apitegromab
Nov 2026~
CT.gov
00%
Pipeline by Phase
Phase 3
44
Related Conditions
rare diseaseorphangeneticgene therapyhereditarymuscular dystrophycystic fibrosissickle cellhemophiliathalassemiagaucherfabrypompehunter syndromephenylketonuriapkusmaspinal muscular atrophy