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.
|
$279.56B |
$90.20
+0.19%
|
|
AMGN
Amgen Inc.
UPLIZNA for IgG4-RD places Amgen in Biotech - Rare Diseases.
|
$177.60B |
$331.94
-2.42%
|
|
GILD
Gilead Sciences, Inc.
Liver disease and broader rare-disease opportunities position Biotech - Rare Diseases.
|
$150.41B |
$121.48
-0.93%
|
|
VRTX
Vertex Pharmaceuticals Incorporated
Vertex's research and development targets rare diseases (e.g., SCD, IgA nephropathy, AMKD), supporting a rare-disease biotech focus.
|
$116.78B |
$455.33
-0.44%
|
|
BMY
Bristol-Myers Squibb Company
Reblozyl and related hematology assets position Biotech - Rare Diseases as a relevant niche within BMY's portfolio.
|
$106.15B |
$52.09
+0.27%
|
|
REGN
Regeneron Pharmaceuticals, Inc.
Some pipeline targets (e.g., PNH, gMG) align with rare diseases indications.
|
$76.14B |
$721.57
-0.17%
|
|
ALNY
Alnylam Pharmaceuticals, Inc.
ALNY develops therapies for rare diseases (e.g., porphyria indications and primary hyperoxaluria) via RNAi.
|
$60.05B |
$459.11
-1.34%
|
|
ARGX
argenx SE
The pipeline includes therapies for rare autoimmune diseases, aligning with Biotech - Rare Diseases.
|
$53.89B |
$903.05
-1.41%
|
|
TAK
Takeda Pharmaceutical Company Limited
Takeda has programs targeting rare diseases, exemplified by assets like TAKHZYRO and other rare-disease indications.
|
$44.96B |
$14.21
-0.46%
|
|
INSM
Insmed Incorporated
Insmed emphasizes rare and serious diseases in its pipeline, aligning with Biotech - Rare Diseases.
|
$43.12B |
$204.53
-0.25%
|
|
BIIB
Biogen Inc.
The company targets rare diseases (e.g., ALS with QALSODY) as a core strategic area.
|
$26.58B |
$182.37
+0.21%
|
|
NBIX
Neurocrine Biosciences, Inc.
CRENESSITY targets classic congenital adrenal hyperplasia (CAH), a rare disease indication.
|
$15.42B |
$155.56
+0.87%
|
|
ROIV
Roivant Sciences Ltd.
Roivant's pipeline includes orphan/rare diseases (e.g., DM, SjD, CLE), supporting Biotech - Rare Diseases.
|
$14.32B |
$21.14
-0.17%
|
|
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.
|
$14.08B |
$73.62
-1.52%
|
|
IONS
Ionis Pharmaceuticals, Inc.
Pipeline includes therapies for rare diseases (FCS, HAE, Alexander disease, Angelman), making Rare Diseases a core investable segment.
|
$13.02B |
$81.33
-0.58%
|
|
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.
|
$11.82B |
$205.68
-1.39%
|
|
MRNA
Moderna, Inc.
Rare disease therapeutics in the pipeline indicate Moderna's involvement in developing treatments for low-prevalence genetic disorders.
|
$10.78B |
$27.71
+8.73%
|
|
JAZZ
Jazz Pharmaceuticals plc
Jazz's portfolio includes rare-disease oncology programs and Epidiolex, supporting a Rare Diseases focus.
|
$10.29B |
$170.72
+2.28%
|
|
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.
|
$10.25B |
$53.30
-0.97%
|
|
RNA
Avidity Biosciences, Inc.
Biotech - Rare Diseases: pipeline targets DM1, FSHD, and DMD, aligning with rare disease focus.
|
$9.22B |
$71.73
+0.27%
|
|
ARWR
Arrowhead Pharmaceuticals, Inc.
Pipeline includes therapies for rare genetic/metabolic diseases (e.g., FCS), placing it in Biotech - Rare Diseases.
|
$8.49B |
$61.41
-5.03%
|
|
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'.
|
$7.86B |
$66.40
-0.21%
|
|
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.
|
$6.95B |
$103.92
-1.68%
|
|
KRYS
Krystal Biotech, Inc.
DEB is a rare disease and Krystal Biotech targets a rare-disease patient population with its gene therapy pipeline.
|
$6.78B |
$234.22
+5.52%
|
|
PTCT
PTC Therapeutics, Inc.
PTC Therapeutics operates primarily in rare diseases across multiple programs, a core focus.
|
$6.14B |
$77.50
-0.45%
|
|
PTGX
Protagonist Therapeutics, Inc.
Rare disease focus: Rusfertide for polycythemia vera categorized as a rare hematology disorder.
|
$5.58B |
$89.68
+2.69%
|
|
COGT
Cogent Biosciences, Inc.
Cogent's focus on rare disease indications (Non-AdvSM, AdvSM, KIT-driven diseases) fits Biotech - Rare Diseases.
|
$5.42B |
$38.35
-2.44%
|
|
PRAX
Praxis Precision Medicines, Inc.
Pipeline includes rare-disease CNS DEEs (SCN2A/SCN8A), aligning with Rare Diseases.
|
$5.22B |
$185.00
-2.62%
|
|
CRSP
CRISPR Therapeutics AG
Sickle cell disease and transfusion-dependent beta-thalassemia are rare diseases, aligning with Rare Diseases.
|
$5.17B |
$56.78
-2.41%
|
|
ADMA
ADMA Biologics, Inc.
SG-001 and the plasma-derived product portfolio place ADMA in the biotech/rare-disease immunology space.
|
$4.74B |
$19.99
-0.60%
|
|
ACAD
ACADIA Pharmaceuticals Inc.
Rett syndrome and Prader-Willi syndrome in ACADIA's pipeline place the company in the rare diseases biotech space.
|
$4.63B |
$27.32
+2.90%
|
|
CRNX
Crinetics Pharmaceuticals, Inc.
Pipeline includes rare endocrine diseases (acromegaly, CAH, ACTH-dependent Cushing's) and orphan indications.
|
$4.40B |
$46.66
-1.61%
|
|
BLTE
Belite Bio, Inc
Belite Bio's STGD1 program targets a rare genetic retinal disease, placing it in Biotech - Rare Diseases.
|
$4.37B |
$148.82
-1.62%
|
|
VKTX
Viking Therapeutics, Inc.
VK0214 targets X-ALD, a rare disease, placing Viking in the rare diseases biotech space.
|
$4.33B |
$39.00
+1.11%
|
|
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.
|
$4.33B |
$45.02
-0.41%
|
|
CNTA
Centessa Pharmaceuticals plc
Narcolepsy and related sleep disorders can be considered rare diseases, aligning with 'Biotech - Rare Diseases'.
|
$4.01B |
$29.85
+0.98%
|
|
ACLX
Arcellx, Inc.
gMG trial indicates development for autoimmune/rare disease; aligns with Rare Diseases category.
|
$3.86B |
$67.48
-1.41%
|
|
SWTX
SpringWorks Therapeutics, Inc.
Pipeline and approved products target rare diseases, including desmoid tumors and NF1-associated plexiform neurofibromas.
|
$3.52B |
$46.99
|
|
RARE
Ultragenyx Pharmaceutical Inc.
Ultragenyx is a rare-disease focused biotech company with approved therapies and a pipeline centered on ultra-rare diseases.
|
$3.51B |
$36.93
+1.07%
|
|
MIRM
Mirum Pharmaceuticals, Inc.
The company focuses on rare diseases, including CTX and Fragile X pipeline.
|
$3.44B |
$68.55
-4.17%
|
|
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.
|
$3.24B |
$93.28
-0.54%
|
|
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.
|
$3.12B |
$34.99
-0.95%
|
|
KNSA
Kiniksa Pharmaceuticals, Ltd.
Recurrent pericarditis represents a rare disease area, aligning with the rare diseases biotech theme.
|
$3.09B |
$41.73
+1.81%
|
|
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.
|
$3.05B |
$9.91
-0.40%
|
|
APLS
Apellis Pharmaceuticals, Inc.
The company is advancing a pipeline for C3G/IC-MPGN in nephrology, aligning with Rare Diseases as an investable category.
|
$2.98B |
$24.00
+8.16%
|
|
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.
|
$2.92B |
$19.98
-0.67%
|
|
DYN
Dyne Therapeutics, Inc.
Pipeline targets include DM1, DMD, Pompe disease, and FSHD, aligning with a Rare Diseases biotech focus.
|
$2.89B |
$20.46
-0.90%
|
|
CPRX
Catalyst Pharmaceuticals, Inc.
The company focuses on rare diseases with its portfolio of therapies addressing small patient populations.
|
$2.88B |
$23.40
+1.39%
|
|
VERA
Vera Therapeutics, Inc.
IgA Nephropathy is a rare disease, and Vera's lead program targets this orphan/rare disease indication.
|
$2.87B |
$41.31
-3.05%
|
|
BEAM
Beam Therapeutics Inc.
Pipeline includes rare genetic diseases (SCD, AATD, GSD1a), aligning with Rare Diseases initiatives.
|
$2.74B |
$27.07
-6.38%
|
|
SLNO
Soleno Therapeutics, Inc.
Biotech - Rare Diseases aligns with Soleno's focus on a rare genetic disorder (PWS) and orphan indication.
|
$2.73B |
$51.51
-2.22%
|
|
VRDN
Viridian Therapeutics, Inc.
Company's focus on Thyroid Eye Disease and autoimmune disease pipelines aligns with Biotech - Rare Diseases.
|
$2.61B |
$32.13
-1.00%
|
|
EWTX
Edgewise Therapeutics, Inc.
Sevasemten and EDG-7500 address muscular dystrophy and other muscle diseases, aligning with Rare Diseases biotechnology.
|
$2.49B |
$23.93
+0.93%
|
|
HRMY
Harmony Biosciences Holdings, Inc.
Harmony's WAKIX and pipeline assets target rare neurological diseases, aligning with Biotech - Rare Diseases.
|
$2.29B |
$39.83
+4.13%
|
|
SRPT
Sarepta Therapeutics, Inc.
Focus on rare genetic diseases (DMD, LGMD) aligns with the Rare Diseases biotech subsegment.
|
$2.18B |
$22.27
-2.41%
|
|
MESO
Mesoblast Limited
Focus on rare diseases with pediatric SR-aGvHD and other rare indications in the pipeline.
|
$2.06B |
$17.95
+4.39%
|
|
RXRX
Recursion Pharmaceuticals, Inc.
Pipeline includes rare-disease programs (e.g., FAP) and preclinical assets.
|
$2.04B |
$4.33
-11.99%
|
|
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.
|
$1.83B |
$41.69
-2.15%
|
|
SION
Sionna Therapeutics, Inc.
Sionna Therapeutics develops therapies targeting a rare genetic disease (cystic fibrosis), aligning with Biotech - Rare Diseases.
|
$1.78B |
$40.80
-0.21%
|
|
ANIP
ANI Pharmaceuticals, Inc.
Cortrophin Gel and related Rare Disease assets position ANI in Biotech - Rare Diseases as a core growth engine.
|
$1.77B |
$81.79
-1.15%
|
|
STOK
Stoke Therapeutics, Inc.
Lead programs target rare genetic diseases (Dravet syndrome and ADOA), aligning Stoke with the Biotech - Rare Diseases investable theme.
|
$1.69B |
$30.75
-0.98%
|
|
ZBIO
Zenas BioPharma, Inc.
IgG4-RD is a rare disease indication targeted by the pivotal program.
|
$1.66B |
$39.21
+2.89%
|
|
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.
|
$1.61B |
$27.91
+1.79%
|
|
BCRX
BioCryst Pharmaceuticals, Inc.
Pipeline includes rare-disease programs (Netherton syndrome and DME), a key strategic focus for BioCryst.
|
$1.59B |
$7.59
-3.00%
|
|
PHVS
Pharvaris N.V.
HAE and related angioedema therapies fall under Rare Diseases, a core space Pharvaris competes in.
|
$1.43B |
$26.68
-3.67%
|
|
DNTH
Dianthus Therapeutics, Inc.
Clinical programs target rare autoimmune neuromuscular diseases such as gMG, CIDP, and MMN.
|
$1.42B |
$44.02
-1.55%
|
|
TSHA
Taysha Gene Therapies, Inc.
Rett syndrome is a rare disease, aligning with the company's focus on rare disease gene therapies.
|
$1.38B |
$5.06
|
|
RLAY
Relay Therapeutics, Inc.
The PROS program targets a rare genetic overgrowth condition, placing part of the pipeline in Rare Diseases.
|
$1.32B |
$7.67
-4.13%
|
|
AMLX
Amylyx Pharmaceuticals, Inc.
PBH and other pipeline programs target rare diseases, supporting the 'Biotech - Rare Diseases' theme.
|
$1.27B |
$14.29
-1.38%
|
|
CAPR
Capricor Therapeutics, Inc.
DMD and potential Becker muscular dystrophy indications place Capricor in the rare diseases therapeutic space.
|
$1.23B |
$26.59
+4.69%
|
|
WVE
Wave Life Sciences Ltd.
Clinical programs target rare diseases (DMD, AATD, HD), aligning with the Rare Diseases biotech segment.
|
$1.19B |
$7.50
-1.51%
|
|
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.
|
$1.17B |
$21.03
-7.52%
|
|
TYRA
Tyra Biosciences, Inc.
TYRA's pediatric achondroplasia program places it in the rare diseases category.
|
$1.15B |
$21.61
-2.26%
|
|
PHAR
Pharming Group N.V.
Core rare-disease therapeutic developer focused on RUCONEST and an expanding rare-disease pipeline.
|
$1.14B |
$16.69
-3.80%
|
|
SVRA
Savara Inc.
Company focuses on rare respiratory diseases (aPAP), a high-need orphan condition, aligning with the Rare Diseases biotech theme.
|
$1.12B |
$6.53
+2.43%
|
|
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 |
$101.33
+8.03%
|
|
MBX
MBX Biosciences, Inc. Common Stock
MBX targets rare endocrine/metabolic disorders (e.g., chronic hypoparathyroidism), aligning with Rare Diseases biotech focus.
|
$1.07B |
$31.70
+3.81%
|
|
PGEN
Precigen, Inc.
RRP is a rare disease; company has rare-disease focus across pipeline.
|
$1.07B |
$3.69
-1.73%
|
|
XERS
Xeris Biopharma Holdings, Inc.
Recorlev and Keveyis target rare endocrine/neuromuscular disorders, aligning with Biotech - Rare Diseases.
|
$1.05B |
$6.50
-3.27%
|
|
NTLA
Intellia Therapeutics, Inc.
Targets rare genetic diseases (HAE and ATTR amyloidosis) with disease-modifying therapy.
|
$1.03B |
$9.59
-1.54%
|
|
BHVN
Biohaven Ltd.
IgA nephropathy and spinocerebellar ataxia (rare diseases) in the pipeline reflect a Rare Diseases focus.
|
$1.01B |
$9.64
+0.36%
|
|
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.
|
$946.25M |
$10.43
+7.53%
|
|
TBPH
Theravance Biopharma, Inc.
Ampreloxetine's focus on a rare disease indication aligns with the Biotech - Rare Diseases investable theme.
|
$934.20M |
$18.64
-0.32%
|
|
VIR
Vir Biotechnology, Inc.
HDV/HBV programs position Vir in Rare Diseases through hepatitis delta and hepatitis B therapeutic strategies.
|
$902.96M |
$6.50
-4.13%
|
|
KROS
Keros Therapeutics, Inc.
Duchenne muscular dystrophy (DMD) and associated cytopenias are rare diseases, aligning Keros Therapeutics' focused assets to Rare Diseases.
|
$880.95M |
$21.78
+7.45%
|
|
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.
|
$877.49M |
$48.99
+3.40%
|
|
KALV
KalVista Pharmaceuticals, Inc.
KalVista's focus on therapies for rare diseases (HAE) aligns with the Biotech - Rare Diseases investable theme.
|
$820.74M |
$16.27
-0.79%
|
|
DAWN
Day One Biopharmaceuticals, Inc.
Pediatric cancer and rare disease context; Day One's focus aligns with Rare Diseases.
|
$811.26M |
$8.04
-3.60%
|
|
ESPR
Esperion Therapeutics, Inc.
Pipeline program for Primary Sclerosing Cholangitis, a rare liver disease.
|
$752.05M |
$3.69
-2.51%
|
|
OMER
Omeros Corporation
TA-TMA, PNH, and C3G represent ultra-rare disease programs in Omeros' pipeline.
|
$738.41M |
$10.89
-1.27%
|
|
ATXS
Astria Therapeutics, Inc.
HAE is a rare disease, and Astria's pipeline includes therapies for niche/rare conditions.
|
$729.70M |
$12.93
-1.45%
|
|
MGTX
MeiraGTx Holdings plc
Pipeline includes LCA4 and other rare disease targets, aligning with Rare Diseases as a major focus area.
|
$695.87M |
$8.59
-0.12%
|
|
RGNX
REGENXBIO Inc.
RGX-121 targets a rare disease (MPS II) in a late-stage program.
|
$664.77M |
$13.05
+0.58%
|
|
CRVS
Corvus Pharmaceuticals, Inc.
ALPS is a rare genetic condition pursued in Phase 2, indicating Rare Diseases as a focus area.
|
$656.47M |
$9.01
+2.21%
|
|
VTYX
Ventyx Biosciences, Inc.
Pipeline includes a therapy (VTX2735) for CAPS, a rare disease, aligning with Rare Diseases.
|
$621.81M |
$8.61
+7.62%
|
|
PRTA
Prothena Corporation plc
Coramitug/ATTR amyloidosis represents a rare disease program, aligning with Prothena's rare disease portfolio.
|
$595.90M |
$11.08
-0.72%
|
|
IRWD
Ironwood Pharmaceuticals, Inc.
Apraglutide is a next-generation therapy being developed for a rare disease (SBS-IF), matching the Rare Diseases investable theme.
|
$565.27M |
$3.46
-7.10%
|
|
DSGN
Design Therapeutics, Inc.
Pipeline targets rare genetic diseases (FA, FECD, DM1, HD), fitting Rare Diseases in biotech.
|
$559.23M |
$10.01
+1.93%
|
|
ANNX
Annexon, Inc.
GBS and related autoimmune targets place Annexon in Biotech - Rare Diseases.
|
$506.58M |
$4.64
+3.34%
|
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