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Gene Therapy (17 companies)

Ticker	Company	Therapeutic areas	Programs	Lead stage	Programs
BEAM	Beam Therapeutics Inc.	HematologyMetabolicRespiratory Rare	2	Phase 1	8
CGON	CG Oncology, Inc.	Oncology	1	NDA Submitted	1
GUTS	FRACTYL HEALTH, INC.	Metabolic	2	Phase 3	3
IBRX	ImmunityBio, Inc.	Infectious DiseaseOncology	3	Approved	17
NTLA	Intellia Therapeutics, Inc.	CardiovascularImmunology Rare	5	NDA Submitted	5
LXEO	Lexeo Therapeutics, Inc.	CardiovascularNeurology Rare	10	Phase 1	10
OCGN	Ocugen, Inc.	Infectious DiseaseOphthalmology Rare	8	Phase 3	13
REPL	Replimune Group, Inc.	Oncology	4	Phase 3	4
RCKT	ROCKET PHARMACEUTICALS, INC.	CardiovascularHematologyImmunology Rare	4	Approved	4
SANA	Sana Biotechnology, Inc.	MetabolicOncology	2	Phase 1	5
SRPT	Sarepta Therapeutics, Inc.	Neurology Rare	5	Approved	10
SLDB	Solid Biosciences Inc.	CardiovascularNeurology Rare	6	Phase 3	6
TSHA	Taysha Gene Therapies, Inc.	Neurology Rare	3	Phase 3	3
RARE	Ultragenyx Pharmaceutical Inc.	CardiovascularGI/HepatologyMetabolicNeurology Rare	4	Approved	12
QURE	uniQure N.V.	HematologyMetabolicNeurology Rare	6	Approved	6
VOR	Vor Biopharma Inc.	ImmunologyNeurologyOncology Rare	0	Phase 3	4
VYGR	Voyager Therapeutics, Inc.	MetabolicNeurology Rare	4	Phase 1	7