Biotech - Rare Diseases
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All Stocks (207)
| Company | Market Cap | Price |
|---|---|---|
|
AZN
AstraZeneca PLC
Alexion-derived rare disease products expand AZN's rare disease franchise.
|
$289.29B |
$93.37
+0.14%
|
|
AMGN
Amgen Inc.
UPLIZNA for IgG4-RD places Amgen in Biotech - Rare Diseases.
|
$185.50B |
$344.55
+1.01%
|
|
GILD
Gilead Sciences, Inc.
Liver disease and broader rare-disease opportunities position Biotech - Rare Diseases.
|
$158.22B |
$127.53
+0.32%
|
|
VRTX
Vertex Pharmaceuticals Incorporated
Vertex's research and development targets rare diseases (e.g., SCD, IgA nephropathy, AMKD), supporting a rare-disease biotech focus.
|
$110.80B |
$432.20
+0.55%
|
|
BMY
Bristol-Myers Squibb Company
Reblozyl and related hematology assets position Biotech - Rare Diseases as a relevant niche within BMY's portfolio.
|
$100.25B |
$49.25
+0.41%
|
|
REGN
Regeneron Pharmaceuticals, Inc.
Some pipeline targets (e.g., PNH, gMG) align with rare diseases indications.
|
$83.16B |
$784.81
-0.32%
|
|
ALNY
Alnylam Pharmaceuticals, Inc.
ALNY develops therapies for rare diseases (e.g., porphyria indications and primary hyperoxaluria) via RNAi.
|
$58.35B |
$445.07
+3.55%
|
|
ARGX
argenx SE
The pipeline includes therapies for rare autoimmune diseases, aligning with Biotech - Rare Diseases.
|
$54.87B |
$917.91
-0.00%
|
|
TAK
Takeda Pharmaceutical Company Limited
Takeda has programs targeting rare diseases, exemplified by assets like TAKHZYRO and other rare-disease indications.
|
$46.03B |
$14.52
+0.10%
|
|
INSM
Insmed Incorporated
Insmed emphasizes rare and serious diseases in its pipeline, aligning with Biotech - Rare Diseases.
|
$43.23B |
$204.49
-2.17%
|
|
BIIB
Biogen Inc.
The company targets rare diseases (e.g., ALS with QALSODY) as a core strategic area.
|
$26.68B |
$181.93
-0.02%
|
|
NBIX
Neurocrine Biosciences, Inc.
CRENESSITY targets classic congenital adrenal hyperplasia (CAH), a rare disease indication.
|
$15.03B |
$151.56
+5.60%
|
|
ROIV
Roivant Sciences Ltd.
Roivant's pipeline includes orphan/rare diseases (e.g., DM, SjD, CLE), supporting Biotech - Rare Diseases.
|
$14.25B |
$20.86
+0.02%
|
|
BBIO
BridgeBio Pharma, Inc.
BridgeBio's pipeline targets multiple rare genetic diseases (ATTR-CM, ADH1, achondroplasia, LGMD2I, Canavan), a core focus area for rare-disease biotech.
|
$13.71B |
$71.71
+1.53%
|
|
IONS
Ionis Pharmaceuticals, Inc.
Pipeline includes therapies for rare diseases (FCS, HAE, Alexander disease, Angelman), making Rare Diseases a core investable segment.
|
$13.18B |
$82.70
+2.19%
|
|
ASND
Ascendis Pharma A/S
Ascendis Pharma focuses on rare-disease endocrinology therapies (YORVIPATH, SKYTROFA, TransCon CNP) within biotech, fitting the Biotech - Rare Diseases category.
|
$12.08B |
$210.41
+1.82%
|
|
JAZZ
Jazz Pharmaceuticals plc
Jazz's portfolio includes rare-disease oncology programs and Epidiolex, supporting a Rare Diseases focus.
|
$10.73B |
$176.95
-1.72%
|
|
BMRN
BioMarin Pharmaceutical Inc.
BioMarin's core commercial therapies (VOXZOGO for skeletal disorders and enzyme replacement therapies like PALYNZIQ, VIMIZIM, NAGLAZYME) are all in the rare diseases space, fitting the Biotech - Rare Diseases investable theme.
|
$10.66B |
$55.48
-1.44%
|
|
MRNA
Moderna, Inc.
Rare disease therapeutics in the pipeline indicate Moderna's involvement in developing treatments for low-prevalence genetic disorders.
|
$9.73B |
$25.00
+1.03%
|
|
RNA
Avidity Biosciences, Inc.
Biotech - Rare Diseases: pipeline targets DM1, FSHD, and DMD, aligning with rare disease focus.
|
$9.21B |
$71.58
+0.03%
|
|
BPMC
Blueprint Medicines Corporation
Pipeline focuses on rare mast cell disorders, aligning with Biotech - Rare Diseases.
|
$8.27B |
$129.46
|
|
CYTK
Cytokinetics, Incorporated
CK-089 represents a neuromuscular/kindred disease program (muscular dystrophies), aligning with 'Biotech - Rare Diseases'.
|
$8.15B |
$68.16
+0.90%
|
|
ARWR
Arrowhead Pharmaceuticals, Inc.
Pipeline includes therapies for rare genetic/metabolic diseases (e.g., FCS), placing it in Biotech - Rare Diseases.
|
$7.98B |
$57.72
+23.36%
|
|
RYTM
Rhythm Pharmaceuticals, Inc.
Rhythm's core business is developing and commercializing therapies for ultra-rare diseases (e.g., setmelanotide for MC4R pathway deficiencies, BBS), a hallmark of rare-disease biotech.
|
$7.13B |
$107.45
-1.20%
|
|
PTCT
PTC Therapeutics, Inc.
PTC Therapeutics operates primarily in rare diseases across multiple programs, a core focus.
|
$6.85B |
$86.24
+3.99%
|
|
KRYS
Krystal Biotech, Inc.
DEB is a rare disease and Krystal Biotech targets a rare-disease patient population with its gene therapy pipeline.
|
$6.33B |
$218.97
+0.92%
|
|
COGT
Cogent Biosciences, Inc.
Cogent's focus on rare disease indications (Non-AdvSM, AdvSM, KIT-driven diseases) fits Biotech - Rare Diseases.
|
$5.64B |
$40.38
+1.66%
|
|
PTGX
Protagonist Therapeutics, Inc.
Rare disease focus: Rusfertide for polycythemia vera categorized as a rare hematology disorder.
|
$5.60B |
$90.08
+0.25%
|
|
CRSP
CRISPR Therapeutics AG
Sickle cell disease and transfusion-dependent beta-thalassemia are rare diseases, aligning with Rare Diseases.
|
$4.85B |
$53.30
-0.51%
|
|
ADMA
ADMA Biologics, Inc.
SG-001 and the plasma-derived product portfolio place ADMA in the biotech/rare-disease immunology space.
|
$4.57B |
$19.14
-0.34%
|
|
CRNX
Crinetics Pharmaceuticals, Inc.
Pipeline includes rare endocrine diseases (acromegaly, CAH, ACTH-dependent Cushing's) and orphan indications.
|
$4.33B |
$46.05
+3.00%
|
|
ACAD
ACADIA Pharmaceuticals Inc.
Rett syndrome and Prader-Willi syndrome in ACADIA's pipeline place the company in the rare diseases biotech space.
|
$4.19B |
$24.82
+1.78%
|
|
SRRK
Scholar Rock Holding Corporation
SMA is a rare disease; Scholar Rock's lead therapy targets a rare disease indication with a broader rare-disease pipeline.
|
$4.15B |
$43.14
+4.03%
|
|
VKTX
Viking Therapeutics, Inc.
VK0214 targets X-ALD, a rare disease, placing Viking in the rare diseases biotech space.
|
$4.08B |
$36.30
+2.37%
|
|
ACLX
Arcellx, Inc.
gMG trial indicates development for autoimmune/rare disease; aligns with Rare Diseases category.
|
$4.06B |
$73.24
+0.33%
|
|
PRAX
Praxis Precision Medicines, Inc.
Pipeline includes rare-disease CNS DEEs (SCN2A/SCN8A), aligning with Rare Diseases.
|
$3.97B |
$188.85
+2.50%
|
|
CNTA
Centessa Pharmaceuticals plc
Narcolepsy and related sleep disorders can be considered rare diseases, aligning with 'Biotech - Rare Diseases'.
|
$3.89B |
$28.97
+0.03%
|
|
BLTE
Belite Bio, Inc
Belite Bio's STGD1 program targets a rare genetic retinal disease, placing it in Biotech - Rare Diseases.
|
$3.78B |
$128.21
+0.32%
|
|
MIRM
Mirum Pharmaceuticals, Inc.
The company focuses on rare diseases, including CTX and Fragile X pipeline.
|
$3.67B |
$73.17
+1.96%
|
|
SWTX
SpringWorks Therapeutics, Inc.
Pipeline and approved products target rare diseases, including desmoid tumors and NF1-associated plexiform neurofibromas.
|
$3.52B |
$46.99
|
|
IRON
Disc Medicine, Inc.
Disc Medicine concentrates on rare hematologic diseases (e.g., EPP, Diamond-Blackfan anemia) by targeting iron and heme pathways, with a pipeline centered on rare-disease indications.
|
$3.26B |
$93.73
+1.30%
|
|
RARE
Ultragenyx Pharmaceutical Inc.
Ultragenyx is a rare-disease focused biotech company with approved therapies and a pipeline centered on ultra-rare diseases.
|
$3.25B |
$33.75
+0.67%
|
|
TVTX
Travere Therapeutics, Inc.
Travere Therapeutics concentrates on rare kidney diseases and rare metabolic diseases (e.g., FSGS, HCU), aligning with the Biotech - Rare Diseases investable theme.
|
$3.23B |
$36.27
+0.72%
|
|
KNSA
Kiniksa Pharmaceuticals, Ltd.
Recurrent pericarditis represents a rare disease area, aligning with the rare diseases biotech theme.
|
$3.15B |
$42.53
-0.42%
|
|
FOLD
Amicus Therapeutics, Inc.
Amicus Therapeutics’s marketed products address rare genetic diseases (Fabry and Pompe) and its pipeline includes a rare-kidney disease program (DMX-200 for FSGS), placing it in Biotech - Rare Diseases.
|
$3.09B |
$10.02
+1.47%
|
|
DYN
Dyne Therapeutics, Inc.
Pipeline targets include DM1, DMD, Pompe disease, and FSHD, aligning with a Rare Diseases biotech focus.
|
$3.01B |
$21.17
+1.78%
|
|
CPRX
Catalyst Pharmaceuticals, Inc.
The company focuses on rare diseases with its portfolio of therapies addressing small patient populations.
|
$2.87B |
$23.43
-0.68%
|
|
DNLI
Denali Therapeutics Inc.
Denali's lead programs target lysosomal storage diseases (Hunter syndrome and Sanfilippo Type A), placing it in the Rare Diseases biotech space.
|
$2.84B |
$19.40
+1.41%
|
|
APLS
Apellis Pharmaceuticals, Inc.
The company is advancing a pipeline for C3G/IC-MPGN in nephrology, aligning with Rare Diseases as an investable category.
|
$2.73B |
$21.57
+2.74%
|
|
EWTX
Edgewise Therapeutics, Inc.
Sevasemten and EDG-7500 address muscular dystrophy and other muscle diseases, aligning with Rare Diseases biotechnology.
|
$2.69B |
$25.51
+6.03%
|
|
SLNO
Soleno Therapeutics, Inc.
Biotech - Rare Diseases aligns with Soleno's focus on a rare genetic disorder (PWS) and orphan indication.
|
$2.63B |
$49.59
-0.36%
|
|
VRDN
Viridian Therapeutics, Inc.
Company's focus on Thyroid Eye Disease and autoimmune disease pipelines aligns with Biotech - Rare Diseases.
|
$2.59B |
$31.73
+1.02%
|
|
BEAM
Beam Therapeutics Inc.
Pipeline includes rare genetic diseases (SCD, AATD, GSD1a), aligning with Rare Diseases initiatives.
|
$2.53B |
$25.06
+1.50%
|
|
VERA
Vera Therapeutics, Inc.
IgA Nephropathy is a rare disease, and Vera's lead program targets this orphan/rare disease indication.
|
$2.13B |
$33.34
+13.30%
|
|
SRPT
Sarepta Therapeutics, Inc.
Focus on rare genetic diseases (DMD, LGMD) aligns with the Rare Diseases biotech subsegment.
|
$2.05B |
$20.97
+8.37%
|
|
HRMY
Harmony Biosciences Holdings, Inc.
Harmony's WAKIX and pipeline assets target rare neurological diseases, aligning with Biotech - Rare Diseases.
|
$2.05B |
$35.59
+0.71%
|
|
MESO
Mesoblast Limited
Focus on rare diseases with pediatric SR-aGvHD and other rare indications in the pipeline.
|
$2.00B |
$17.36
+8.09%
|
|
SION
Sionna Therapeutics, Inc.
Sionna Therapeutics develops therapies targeting a rare genetic disease (cystic fibrosis), aligning with Biotech - Rare Diseases.
|
$1.93B |
$43.83
-1.02%
|
|
RXRX
Recursion Pharmaceuticals, Inc.
Pipeline includes rare-disease programs (e.g., FAP) and preclinical assets.
|
$1.91B |
$4.41
+0.80%
|
|
ANIP
ANI Pharmaceuticals, Inc.
Cortrophin Gel and related Rare Disease assets position ANI in Biotech - Rare Diseases as a core growth engine.
|
$1.83B |
$84.37
+1.38%
|
|
STOK
Stoke Therapeutics, Inc.
Lead programs target rare genetic diseases (Dravet syndrome and ADOA), aligning Stoke with the Biotech - Rare Diseases investable theme.
|
$1.71B |
$31.21
+4.66%
|
|
ZBIO
Zenas BioPharma, Inc.
IgG4-RD is a rare disease indication targeted by the pivotal program.
|
$1.71B |
$40.38
+1.30%
|
|
AGIO
Agios Pharmaceuticals, Inc.
Agios's strategic pivot to rare diseases centers on PK activation therapies and a pipeline addressing rare hematologic conditions, aligning with Biotech - Rare Diseases.
|
$1.69B |
$29.04
+2.16%
|
|
MAZE
Maze Therapeutics, Inc.
Maze's program portfolio includes ALS and kidney disease programs, reflecting a focus on rare diseases within its CVRM/hep area.
|
$1.67B |
$37.98
+3.50%
|
|
QURE
uniQure N.V.
Huntington's disease and other rare diseases targeted by uniQure's gene therapies position the company in the rare diseases biotech space.
|
$1.52B |
$27.60
-1.29%
|
|
PHVS
Pharvaris N.V.
HAE and related angioedema therapies fall under Rare Diseases, a core space Pharvaris competes in.
|
$1.50B |
$27.80
+9.23%
|
|
BCRX
BioCryst Pharmaceuticals, Inc.
Pipeline includes rare-disease programs (Netherton syndrome and DME), a key strategic focus for BioCryst.
|
$1.49B |
$7.12
+3.94%
|
|
DNTH
Dianthus Therapeutics, Inc.
Clinical programs target rare autoimmune neuromuscular diseases such as gMG, CIDP, and MMN.
|
$1.42B |
$44.02
-0.15%
|
|
RLAY
Relay Therapeutics, Inc.
The PROS program targets a rare genetic overgrowth condition, placing part of the pipeline in Rare Diseases.
|
$1.38B |
$8.03
-0.31%
|
|
AMLX
Amylyx Pharmaceuticals, Inc.
PBH and other pipeline programs target rare diseases, supporting the 'Biotech - Rare Diseases' theme.
|
$1.34B |
$15.01
+0.84%
|
|
TSHA
Taysha Gene Therapies, Inc.
Rett syndrome is a rare disease, aligning with the company's focus on rare disease gene therapies.
|
$1.33B |
$4.87
+6.10%
|
|
WVE
Wave Life Sciences Ltd.
Clinical programs target rare diseases (DMD, AATD, HD), aligning with the Rare Diseases biotech segment.
|
$1.22B |
$7.69
+3.50%
|
|
PHAR
Pharming Group N.V.
Core rare-disease therapeutic developer focused on RUCONEST and an expanding rare-disease pipeline.
|
$1.20B |
$17.46
+2.36%
|
|
TYRA
Tyra Biosciences, Inc.
TYRA's pediatric achondroplasia program places it in the rare diseases category.
|
$1.17B |
$22.01
+0.50%
|
|
XERS
Xeris Biopharma Holdings, Inc.
Recorlev and Keveyis target rare endocrine/neuromuscular disorders, aligning with Biotech - Rare Diseases.
|
$1.16B |
$7.18
+1.20%
|
|
MBX
MBX Biosciences, Inc. Common Stock
MBX targets rare endocrine/metabolic disorders (e.g., chronic hypoparathyroidism), aligning with Rare Diseases biotech focus.
|
$1.14B |
$33.86
+7.15%
|
|
PGEN
Precigen, Inc.
RRP is a rare disease; company has rare-disease focus across pipeline.
|
$1.11B |
$3.75
+2.18%
|
|
PVLA
Palvella Therapeutics, Inc.
Palvella Therapeutics concentrates on rare genetic skin diseases and markets a proprietary topical rapamycin platform (QTORIN), indicating a core focus on rare disease biotech development.
|
$1.11B |
$99.68
+5.07%
|
|
TBPH
Theravance Biopharma, Inc.
Ampreloxetine's focus on a rare disease indication aligns with the Biotech - Rare Diseases investable theme.
|
$1.01B |
$19.97
+5.44%
|
|
BHVN
Biohaven Ltd.
IgA nephropathy and spinocerebellar ataxia (rare diseases) in the pipeline reflect a Rare Diseases focus.
|
$1.00B |
$9.51
+1.06%
|
|
DAWN
Day One Biopharmaceuticals, Inc.
Pediatric cancer and rare disease context; Day One's focus aligns with Rare Diseases.
|
$963.88M |
$9.42
-0.11%
|
|
SVRA
Savara Inc.
Company focuses on rare respiratory diseases (aPAP), a high-need orphan condition, aligning with the Rare Diseases biotech theme.
|
$926.41M |
$5.37
+4.27%
|
|
NTLA
Intellia Therapeutics, Inc.
Targets rare genetic diseases (HAE and ATTR amyloidosis) with disease-modifying therapy.
|
$914.60M |
$8.53
-0.23%
|
|
RIGL
Rigel Pharmaceuticals, Inc.
R289 targets lower-risk myelodysplastic syndrome (MDS), aligning with Biotech - Rare Diseases due to orphan/fast-track potential and niche unmet needs.
|
$914.09M |
$51.05
+3.01%
|
|
VIR
Vir Biotechnology, Inc.
HDV/HBV programs position Vir in Rare Diseases through hepatitis delta and hepatitis B therapeutic strategies.
|
$887.68M |
$6.41
-0.47%
|
|
RZLT
Rezolute, Inc.
HI is a rare disease; the therapy targets rare pediatric congenital HI and tumor HI indications, aligning with Rare Diseases biotech focus.
|
$868.16M |
$9.54
+0.16%
|
|
ESPR
Esperion Therapeutics, Inc.
Pipeline program for Primary Sclerosing Cholangitis, a rare liver disease.
|
$796.41M |
$3.96
+7.18%
|
|
VTYX
Ventyx Biosciences, Inc.
Pipeline includes a therapy (VTX2735) for CAPS, a rare disease, aligning with Rare Diseases.
|
$712.37M |
$10.00
-4.40%
|
|
ATXS
Astria Therapeutics, Inc.
HAE is a rare disease, and Astria's pipeline includes therapies for niche/rare conditions.
|
$711.08M |
$12.62
+2.27%
|
|
KROS
Keros Therapeutics, Inc.
Duchenne muscular dystrophy (DMD) and associated cytopenias are rare diseases, aligning Keros Therapeutics' focused assets to Rare Diseases.
|
$710.77M |
$17.48
+0.95%
|
|
KALV
KalVista Pharmaceuticals, Inc.
KalVista's focus on therapies for rare diseases (HAE) aligns with the Biotech - Rare Diseases investable theme.
|
$704.35M |
$14.11
+6.25%
|
|
CRVS
Corvus Pharmaceuticals, Inc.
ALPS is a rare genetic condition pursued in Phase 2, indicating Rare Diseases as a focus area.
|
$675.84M |
$9.06
+2.20%
|
|
RGNX
REGENXBIO Inc.
RGX-121 targets a rare disease (MPS II) in a late-stage program.
|
$669.82M |
$13.26
-1.45%
|
|
MGTX
MeiraGTx Holdings plc
Pipeline includes LCA4 and other rare disease targets, aligning with Rare Diseases as a major focus area.
|
$659.67M |
$8.21
+8.25%
|
|
OMER
Omeros Corporation
TA-TMA, PNH, and C3G represent ultra-rare disease programs in Omeros' pipeline.
|
$655.38M |
$9.63
+1.90%
|
|
IRWD
Ironwood Pharmaceuticals, Inc.
Apraglutide is a next-generation therapy being developed for a rare disease (SBS-IF), matching the Rare Diseases investable theme.
|
$627.00M |
$3.87
+23.48%
|
|
FULC
Fulcrum Therapeutics, Inc.
Company's focus on sickle cell disease and Diamond-Blackfan anemia positions it in rare genetic diseases.
|
$612.32M |
$11.32
-1.91%
|
|
PRTA
Prothena Corporation plc
Coramitug/ATTR amyloidosis represents a rare disease program, aligning with Prothena's rare disease portfolio.
|
$561.45M |
$10.43
-0.67%
|
|
MNPR
Monopar Therapeutics Inc.
ALXN1840 (Wilson disease) in-licensing expands Monopar's rare disease pipeline, aligning with Biotech - Rare Diseases.
|
$539.01M |
$87.19
-1.45%
|
|
DSGN
Design Therapeutics, Inc.
Pipeline targets rare genetic diseases (FA, FECD, DM1, HD), fitting Rare Diseases in biotech.
|
$533.60M |
$9.40
-0.11%
|
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