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Gene Therapy (19 companies)

Ticker	Company	Therapeutic areas	Programs	Lead stage	Programs
FDMT	4D Molecular Therapeutics	OphthalmologyRespiratory Rare	5	Phase 3	5
BEAM	Beam Therapeutics	HematologyMetabolic+1 Rare	2	Phase 1	7
CGON	CG Oncology	Oncology	1	NDA Submitted	1
GUTS	Fractyl Health	Metabolic	2	Phase 3	3
IBRX	ImmunityBio	Infectious DiseaseOncology	3	NDA Submitted	16
NTLA	Intellia Therapeutics	CardiovascularImmunology Rare	5	NDA Submitted	5
LXEO	Lexeo Therapeutics	CardiovascularNeurology Rare	8	Phase 1	8
NGNE	Neurogene	Neurology Rare	1	Phase 3	1
OCGN	Ocugen	Infectious DiseaseOphthalmology Rare	8	Phase 3	13
IRD	Opus Genetics	Ophthalmology Rare	8	Approved	12
REPL	Replimune Group	Oncology	5	NDA Submitted	5
RCKT	Rocket Pharmaceuticals	CardiovascularImmunology Rare	4	Approved	4
SANA	Sana Biotechnology	MetabolicOncology	2	Phase 1	5
SRPT	Sarepta Therapeutics	Neurology Rare	5	Approved	10
SLDB	Solid Biosciences	CardiovascularNeurology Rare	6	Phase 3	6
TSHA	Taysha Gene Therapies	Neurology Rare	4	Phase 3	4
RARE	Ultragenyx Pharmaceutical	CardiovascularGI/Hepatology+2 Rare	4	Approved	12
QURE	uniQure	HematologyMetabolic+1 Rare	6	Approved	6
VYGR	Voyager Therapeutics	MetabolicNeurology Rare	4	Phase 1	7