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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	905	+28	+3%	$18.2B
2	2 Charles River Laboratories CRL $14.4B	297	+13	+5%	$3.52B
3	3 DELISTED CATALENT, INC. CTLT	206	+4	+2%	$3.45B
4	4 DELISTED bluebird bio BLUE	199	-2	-1%	$2.88B
5	5 Sarepta Therapeutics SRPT $1.78B	178	-10	-5%	$1.08B
6	6 Ultragenyx Pharmaceutical RARE $1.47B	175	+1	+0.6%	$2.93B
7	7 Precigen PGEN $2.64B	171	+2	+1%	$2.42B
8	ISEE 8 DELISTED IVERIC bio, Inc. Common Stock ISEE	137	-55	-29%	$113M
9	9 DELISTED SANGAMO THERAPEUTICS INC SGMO	126	+1	+0.8%	$127M
10	10 PTC Therapeutics PTCT $4.98B	123	+7	+6%	$313M
11	SIOX 11 DELISTED Sio Gene Therapies Inc. Common Stock SIOX	101	+6	+6%	$1.25B
12	12 Lineage Cell Therapeutics LCTX $232M	92	+12	+15%	$146M
13	AGTC 13 DELISTED Applied Genetic Technologies Corporation AGTC	90	-5	-5%	$108M
14	14 Atara Biotherapeutics ATRA $96M	86	+10	+13%	$330M
15	15 DELISTED Adverum Biotechnologies ADVM	83	+7	+9%	$43.8M
16	16 Rocket Pharmaceuticals RCKT $243M	82	+4	+5%	$109M
17	BLCM 17 DELISTED Bellicum Pharmaceuticals, Inc. BLCM	80	+10	+14%	$266M
18	ATHX 18 DELISTED Athersys, Inc. Common Stock ATHX	79	+6	+8%	$23.1M
19	19 Regenxbio RGNX $454M	78	+7	+10%	$339M
20	20 Intellia Therapeutics NTLA $1.6B	70	+5	+8%	$241M
21	21 Voyager Therapeutics VYGR $124M	56	+3	+6%	$140M
22	22 Abeona Therapeutics ABEO $249M	54	+15	+38%	$72M
23	23 Lakefront Biotherapeutics American Depositary Shares LKFT $1.93B	52	+4	+8%	$642M
24	24 Fortress Biotech FBIO $63.4M	51	0	0%	$18.1M
25	25 uniQure QURE $1.45B	51	+2	+4%	$42.7M