← 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	1,342	+29	+2%	$12.7B
2	2 Charles River Laboratories CRL $14.4B	638	+9	+1%	$9.78B
3	3 Sarepta Therapeutics SRPT $1.78B	465	+7	+2%	$10.7B
4	4 CRISPR Therapeutics CRSP $4.86B	406	-10	-2%	$2.54B
5	5 Ultragenyx Pharmaceutical RARE $1.47B	313	+12	+4%	$3.68B
6	6 Intellia Therapeutics NTLA $1.6B	296	+12	+4%	$1.16B
7	7 Vericel Corp VCEL $2.06B	279	+11	+4%	$2.87B
8	8 Krystal Biotech KRYS $9.79B	264	-19	-7%	$4.35B
9	9 BridgeBio Pharma BBIO $12.7B	263	+10	+4%	$4.85B
10	10 PTC Therapeutics PTCT $4.98B	240	+37	+18%	$3.51B
11	11 Rocket Pharmaceuticals RCKT $243M	225	+34	+18%	$1.38B
12	12 DELISTED Arcellx ACLX	211	+25	+13%	$4.54B
13	13 Legend Biotech LEGN $3.7B	211	+3	+1%	$3.23B
14	14 DELISTED BioLife Solutions BLFS	180	-15	-8%	$1.21B
15	15 Regenxbio RGNX $454M	177	-9	-5%	$325M
16	16 Allogene Therapeutics ALLO $449M	174	+18	+12%	$402M
17	17 Sana Biotechnology SANA $698M	164	+9	+6%	$258M
18	18 Fate Therapeutics FATE $232M	159	-5	-3%	$166M
19	19 4D Molecular Therapeutics FDMT $674M	145	+5	+4%	$262M
20	20 uniQure QURE $1.45B	138	+7	+5%	$736M
21	21 Caribou Biosciences CRBU $49.3M	133	+4	+3%	$93.1M
22	22 Precigen PGEN $2.63B	133	+6	+5%	$84.8M
23	23 Prime Medicine PRME $568M	132	+16	+14%	$188M
24	24 Voyager Therapeutics VYGR $124M	132	+4	+3%	$196M
25	25 Taysha Gene Therapies TSHA $1.14B	131	+12	+10%	$290M