← All themes Genomics & Gene Editing

Gene Therapy

Gene therapy delivers a working copy of a gene, or a correction to a faulty one, usually once and for life. Manufacturing the delivery vector is often harder than designing the therapy.

131 stocks

#	Name	Funds holding	Growth	Growth %	Fund value
1	1 Novartis NVS $279B	927	+1	+0.1%	$20.2B
2	2 Charles River Laboratories CRL $14.4B	268	+10	+4%	$3.62B
3	3 DELISTED bluebird bio BLUE	193	+1	+0.5%	$2.53B
4	4 DELISTED CATALENT, INC. CTLT	188	+12	+7%	$3.11B
5	5 Precigen PGEN $2.63B	180	+7	+4%	$3.09B
6	6 Ultragenyx Pharmaceutical RARE $1.47B	172	+10	+6%	$3.99B
7	ISEE 7 DELISTED IVERIC bio, Inc. Common Stock ISEE	152	+8	+6%	$2.47B
8	8 Sarepta Therapeutics SRPT $1.78B	151	+2	+1%	$1.1B
9	9 PTC Therapeutics PTCT $4.98B	141	-16	-10%	$1.18B
10	10 DELISTED SANGAMO THERAPEUTICS INC SGMO	141	0	0%	$448M
11	AGTC 11 DELISTED Applied Genetic Technologies Corporation AGTC	88	+28	+47%	$223M
12	12 Atara Biotherapeutics ATRA $96M	85	-6	-7%	$509M
13	BLCM 13 DELISTED Bellicum Pharmaceuticals, Inc. BLCM	81	-2	-2%	$333M
14	14 DELISTED Adverum Biotechnologies ADVM	76	-16	-17%	$153M
15	SIOX 15 DELISTED Sio Gene Therapies Inc. Common Stock SIOX	70	-1	-1%	$454M
16	16 Lineage Cell Therapeutics LCTX $232M	68	-6	-8%	$134M
17	17 Regenxbio RGNX $454M	66	+18	+38%	$247M
18	18 uniQure QURE $1.45B	64	+2	+3%	$136M
19	19 Rocket Pharmaceuticals RCKT $243M	60	+7	+13%	$218M
20	20 Fate Therapeutics FATE $232M	54	+10	+23%	$62.7M
21	ADAP 21 DELISTED Adaptimmune Therapeutics ADAP	49	+8	+20%	$250M
22	ATHX 22 DELISTED Athersys, Inc. Common Stock ATHX	49	+3	+7%	$7.54M
23	23 Cellectis CLLS $113M	48	+5	+12%	$219M
24	24 Abeona Therapeutics ABEO $249M	45	+2	+5%	$26M
25	25 Lakefront Biotherapeutics American Depositary Shares LKFT $1.93B	44	+6	+16%	$518M