← 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	1,272	+50	+4%	$14.3B
2	2 Charles River Laboratories CRL $14.4B	634	+13	+2%	$12.3B
3	3 CRISPR Therapeutics CRSP $4.86B	470	+65	+16%	$3.48B
4	4 DELISTED CATALENT, INC. CTLT	424	-10	-2%	$8.9B
5	5 Sarepta Therapeutics SRPT $1.78B	396	+1	+0.3%	$7.92B
6	6 Intellia Therapeutics NTLA $1.6B	293	+8	+3%	$2.6B
7	7 BridgeBio Pharma BBIO $12.7B	247	+27	+12%	$6.69B
8	8 Ultragenyx Pharmaceutical RARE $1.47B	237	+17	+8%	$3.85B
9	9 Vericel Corp VCEL $2.06B	227	+14	+7%	$1.85B
10	10 Legend Biotech LEGN $3.7B	206	+6	+3%	$5.16B
11	11 Krystal Biotech KRYS $9.79B	194	+6	+3%	$3.03B
12	12 Rocket Pharmaceuticals RCKT $243M	194	+19	+11%	$2.67B
13	13 DELISTED bluebird bio BLUE	187	+19	+11%	$180M
14	14 PTC Therapeutics PTCT $4.98B	173	-7	-4%	$2.34B
15	15 Regenxbio RGNX $454M	171	+12	+8%	$684M
16	16 Allogene Therapeutics ALLO $449M	170	+16	+10%	$392M
17	17 Fate Therapeutics FATE $232M	159	-11	-6%	$357M
18	18 DELISTED Arcellx ACLX	156	+20	+15%	$2.59B
19	19 Caribou Biosciences CRBU $49.3M	144	+18	+14%	$396M
20	20 uniQure QURE $1.45B	144	-15	-9%	$256M
21	21 DELISTED BioLife Solutions BLFS	140	-13	-8%	$738M
22	22 Precigen PGEN $2.63B	137	-1	-0.7%	$83.9M
23	23 Sana Biotechnology SANA $698M	132	-4	-3%	$508M
24	24 DELISTED SANGAMO THERAPEUTICS INC SGMO	130	-4	-3%	$47.2M
25	25 Cabaletta Bio CABA $275M	128	+31	+32%	$1.1B