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Nucleic Acid (13 companies)

Ticker Company Therapeutic areas Programs Lead stage Programs
AMLX Amylyx Pharmaceuticals MetabolicNeurology Rare 1 Phase 3 5
BEAM Beam Therapeutics HematologyMetabolic+1 Rare 3 Phase 2 6
DYN Dyne Therapeutics MetabolicNeurology Rare 9 NDA Submitted 10
ELTX Elicio Therapeutics Oncology 3 Phase 2 6
GERN Geron Oncology 3 Approved 3
IONS Ionis Pharmaceuticals CardiovascularHematology+5 Rare 28 Approved 29
PEPG PepGen Neurology Rare 1 Phase 2 1
PHIO Phio Pharmaceuticals Oncology 4 Phase 1 4
PRQR ProQR Therapeutics MetabolicNeurology Rare 7 Phase 1 7
SRPT Sarepta Therapeutics NeurologyRespiratory Rare 8 Approved 10
RARE Ultragenyx Pharmaceutical CardiovascularGI/Hepatology+2 Rare 1 Approved 12
VNDA Vanda Pharmaceuticals CardiovascularDermatology+6 Rare 3 Approved 27
WVE Wave Life Sciences MetabolicNeurology+1 Rare 5 Phase 2 5