← 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	1,028	+41	+4%	$22.5B
2	2 Charles River Laboratories CRL $14.4B	366	+18	+5%	$6.27B
3	3 Sarepta Therapeutics SRPT $1.78B	345	+27	+8%	$9.89B
4	4 DELISTED bluebird bio BLUE	288	-19	-6%	$8.48B
5	5 DELISTED CATALENT, INC. CTLT	277	+15	+6%	$6.49B
6	6 DELISTED SANGAMO THERAPEUTICS INC SGMO	217	+7	+3%	$1.1B
7	7 Regenxbio RGNX $454M	203	+26	+15%	$2.16B
8	8 PTC Therapeutics PTCT $4.98B	180	+14	+8%	$2.2B
9	9 CRISPR Therapeutics CRSP $4.86B	179	+5	+3%	$1.01B
10	10 Ultragenyx Pharmaceutical RARE $1.47B	177	+19	+12%	$3.8B
11	11 Precigen PGEN $2.64B	170	+4	+2%	$2.08B
12	12 Intellia Therapeutics NTLA $1.6B	157	+1	+0.6%	$833M
13	13 Vericel Corp VCEL $2.06B	132	+3	+2%	$489M
14	14 Voyager Therapeutics VYGR $124M	119	-6	-5%	$547M
15	15 Fate Therapeutics FATE $232M	116	+13	+13%	$893M
16	16 Atara Biotherapeutics ATRA $96M	115	-9	-7%	$1.92B
17	17 Lineage Cell Therapeutics LCTX $232M	106	-1	-0.9%	$130M
18	18 Abeona Therapeutics ABEO $249M	104	+12	+13%	$444M
19	19 DELISTED Adverum Biotechnologies ADVM	101	+1	+1%	$269M
20	BLCM 20 DELISTED Bellicum Pharmaceuticals, Inc. BLCM	100	+4	+4%	$150M
21	ATHX 21 DELISTED Athersys, Inc. Common Stock ATHX	99	-5	-5%	$58.2M
22	22 uniQure QURE $1.45B	99	+2	+2%	$822M
23	ADAP 23 DELISTED Adaptimmune Therapeutics ADAP	93	+7	+8%	$888M
24	24 Lakefront Biotherapeutics American Depositary Shares LKFT $1.93B	92	+13	+16%	$1.06B
25	25 Rocket Pharmaceuticals RCKT $243M	84	+8	+11%	$906M