← 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	1,405	+19	+1%	$17.2B
2	2 Charles River Laboratories CRL $14.4B	556	+37	+7%	$7.92B
3	3 CRISPR Therapeutics CRSP $4.86B	441	+7	+2%	$4.68B
4	4 BridgeBio Pharma BBIO $12.7B	392	+33	+9%	$9.62B
5	5 Sarepta Therapeutics SRPT $1.78B	320	-43	-12%	$1.67B
6	6 Ultragenyx Pharmaceutical RARE $1.47B	303	-7	-2%	$2.87B
7	7 PTC Therapeutics PTCT $4.98B	294	+10	+4%	$5.07B
8	8 Krystal Biotech KRYS $9.79B	284	+9	+3%	$4.94B
9	9 Intellia Therapeutics NTLA $1.6B	284	+2	+0.7%	$1.8B
10	10 Vericel Corp VCEL $2.06B	249	-23	-8%	$1.67B
11	11 uniQure QURE $1.45B	219	+76	+53%	$3.23B
12	12 DELISTED Arcellx ACLX	209	+7	+3%	$5.18B
13	13 Legend Biotech LEGN $3.7B	200	-11	-5%	$2.73B
14	14 DELISTED BioLife Solutions BLFS	192	-2	-1%	$1.23B
15	15 Sana Biotechnology SANA $698M	178	+23	+15%	$582M
16	16 Rocket Pharmaceuticals RCKT $243M	173	-23	-12%	$315M
17	17 Allogene Therapeutics ALLO $449M	170	+12	+8%	$201M
18	18 Precigen PGEN $2.64B	167	+20	+14%	$366M
19	19 Prime Medicine PRME $568M	167	+46	+38%	$512M
20	20 Regenxbio RGNX $454M	166	+7	+4%	$399M
21	21 Taysha Gene Therapies TSHA $1.14B	155	+5	+3%	$866M
22	22 Fate Therapeutics FATE $232M	143	+4	+3%	$102M
23	23 4D Molecular Therapeutics FDMT $674M	134	+14	+12%	$346M
24	24 Solid Biosciences SLDB $586M	127	+13	+11%	$502M
25	25 MeiraGTx Holdings MGTX $904M	123	+11	+10%	$528M