Biotech - Rare Diseases
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Jan 06, 2026
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Biogen Secures China NMPA Acceptance for Subcutaneous LEQEMBI Autoinjector
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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.
|
$282.78B |
$94.78
+3.90%
|
|
AMGN
Amgen Inc.
UPLIZNA for IgG4-RD places Amgen in Biotech - Rare Diseases.
|
$172.66B |
$329.68
+2.79%
|
|
GILD
Gilead Sciences, Inc.
Liver disease and broader rare-disease opportunities position Biotech - Rare Diseases.
|
$146.79B |
$121.58
+2.77%
|
|
VRTX
Vertex Pharmaceuticals Incorporated
Vertex's research and development targets rare diseases (e.g., SCD, IgA nephropathy, AMKD), supporting a rare-disease biotech focus.
|
$115.07B |
$466.52
+3.95%
|
|
BMY
Bristol-Myers Squibb Company
Reblozyl and related hematology assets position Biotech - Rare Diseases as a relevant niche within BMY's portfolio.
|
$108.00B |
$54.65
+2.99%
|
|
REGN
Regeneron Pharmaceuticals, Inc.
Some pipeline targets (e.g., PNH, gMG) align with rare diseases indications.
|
$80.89B |
$772.67
+1.24%
|
|
ALNY
Alnylam Pharmaceuticals, Inc.
ALNY develops therapies for rare diseases (e.g., porphyria indications and primary hyperoxaluria) via RNAi.
|
$52.15B |
$408.34
+2.64%
|
|
TAK
Takeda Pharmaceutical Company Limited
Takeda has programs targeting rare diseases, exemplified by assets like TAKHZYRO and other rare-disease indications.
|
$49.74B |
$15.80
+0.73%
|
|
ARGX
argenx SE
The pipeline includes therapies for rare autoimmune diseases, aligning with Biotech - Rare Diseases.
|
$47.89B |
$801.07
+0.02%
|
|
INSM
Insmed Incorporated
Insmed emphasizes rare and serious diseases in its pipeline, aligning with Biotech - Rare Diseases.
|
$37.03B |
$174.04
-0.66%
|
|
BIIB
Biogen Inc.
The company targets rare diseases (e.g., ALS with QALSODY) as a core strategic area.
|
$25.60B |
$182.07
+4.26%
|
|
ROIV
Roivant Sciences Ltd.
Roivant's pipeline includes orphan/rare diseases (e.g., DM, SjD, CLE), supporting Biotech - Rare Diseases.
|
$14.55B |
$21.46
+0.77%
|
|
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.
|
$14.04B |
$72.83
-0.80%
|
|
NBIX
Neurocrine Biosciences, Inc.
CRENESSITY targets classic congenital adrenal hyperplasia (CAH), a rare disease indication.
|
$13.49B |
$136.72
+0.54%
|
|
IONS
Ionis Pharmaceuticals, Inc.
Pipeline includes therapies for rare diseases (FCS, HAE, Alexander disease, Angelman), making Rare Diseases a core investable segment.
|
$12.52B |
$81.17
+3.36%
|
|
MRNA
Moderna, Inc.
Rare disease therapeutics in the pipeline indicate Moderna's involvement in developing treatments for low-prevalence genetic disorders.
|
$12.52B |
$35.54
+10.48%
|
|
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.
|
$12.26B |
$209.17
-1.87%
|
|
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.
|
$11.30B |
$59.99
+1.90%
|
|
JAZZ
Jazz Pharmaceuticals plc
Jazz's portfolio includes rare-disease oncology programs and Epidiolex, supporting a Rare Diseases focus.
|
$10.58B |
$166.29
-4.64%
|
|
RNA
Avidity Biosciences, Inc.
Biotech - Rare Diseases: pipeline targets DM1, FSHD, and DMD, aligning with rare disease focus.
|
$9.27B |
$72.20
+0.18%
|
|
ARWR
Arrowhead Pharmaceuticals, Inc.
Pipeline includes therapies for rare genetic/metabolic diseases (e.g., FCS), placing it in Biotech - Rare Diseases.
|
$8.83B |
$71.16
+11.40%
|
|
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'.
|
$7.33B |
$64.28
+4.94%
|
|
KRYS
Krystal Biotech, Inc.
DEB is a rare disease and Krystal Biotech targets a rare-disease patient population with its gene therapy pipeline.
|
$7.08B |
$248.60
+1.59%
|
|
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.
|
$6.77B |
$98.98
-2.83%
|
|
PTCT
PTC Therapeutics, Inc.
PTC Therapeutics operates primarily in rare diseases across multiple programs, a core focus.
|
$6.10B |
$77.20
+0.57%
|
|
PRAX
Praxis Precision Medicines, Inc.
Pipeline includes rare-disease CNS DEEs (SCN2A/SCN8A), aligning with Rare Diseases.
|
$5.83B |
$270.86
-2.19%
|
|
PTGX
Protagonist Therapeutics, Inc.
Rare disease focus: Rusfertide for polycythemia vera categorized as a rare hematology disorder.
|
$5.24B |
$82.11
-2.46%
|
|
CRSP
CRISPR Therapeutics AG
Sickle cell disease and transfusion-dependent beta-thalassemia are rare diseases, aligning with Rare Diseases.
|
$5.02B |
$58.25
+5.62%
|
|
COGT
Cogent Biosciences, Inc.
Cogent's focus on rare disease indications (Non-AdvSM, AdvSM, KIT-driven diseases) fits Biotech - Rare Diseases.
|
$4.79B |
$34.51
+0.72%
|
|
BLTE
Belite Bio, Inc
Belite Bio's STGD1 program targets a rare genetic retinal disease, placing it in Biotech - Rare Diseases.
|
$4.65B |
$159.78
+0.23%
|
|
CRNX
Crinetics Pharmaceuticals, Inc.
Pipeline includes rare endocrine diseases (acromegaly, CAH, ACTH-dependent Cushing's) and orphan indications.
|
$4.54B |
$46.29
-3.92%
|
|
ACAD
ACADIA Pharmaceuticals Inc.
Rett syndrome and Prader-Willi syndrome in ACADIA's pipeline place the company in the rare diseases biotech space.
|
$4.41B |
$26.75
+2.29%
|
|
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.
|
$4.39B |
$14.27
+0.11%
|
|
ADMA
ADMA Biologics, Inc.
SG-001 and the plasma-derived product portfolio place ADMA in the biotech/rare-disease immunology space.
|
$4.30B |
$18.27
+1.30%
|
|
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.
|
$4.11B |
$41.23
-3.58%
|
|
MIRM
Mirum Pharmaceuticals, Inc.
The company focuses on rare diseases, including CTX and Fragile X pipeline.
|
$3.80B |
$75.44
-0.17%
|
|
VKTX
Viking Therapeutics, Inc.
VK0214 targets X-ALD, a rare disease, placing Viking in the rare diseases biotech space.
|
$3.61B |
$32.10
-0.11%
|
|
SWTX
SpringWorks Therapeutics, Inc.
Pipeline and approved products target rare diseases, including desmoid tumors and NF1-associated plexiform neurofibromas.
|
$3.52B |
$46.99
|
|
ACLX
Arcellx, Inc.
gMG trial indicates development for autoimmune/rare disease; aligns with Rare Diseases category.
|
$3.43B |
$62.83
+1.62%
|
|
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.
|
$3.40B |
$35.88
-5.86%
|
|
APLS
Apellis Pharmaceuticals, Inc.
The company is advancing a pipeline for C3G/IC-MPGN in nephrology, aligning with Rare Diseases as an investable category.
|
$3.14B |
$23.76
-4.46%
|
|
CNTA
Centessa Pharmaceuticals plc
Narcolepsy and related sleep disorders can be considered rare diseases, aligning with 'Biotech - Rare Diseases'.
|
$3.09B |
$22.89
-0.56%
|
|
KNSA
Kiniksa Pharmaceuticals, Ltd.
Recurrent pericarditis represents a rare disease area, aligning with the rare diseases biotech theme.
|
$3.04B |
$42.84
+4.34%
|
|
VERA
Vera Therapeutics, Inc.
IgA Nephropathy is a rare disease, and Vera's lead program targets this orphan/rare disease indication.
|
$3.00B |
$46.07
-2.12%
|
|
CPRX
Catalyst Pharmaceuticals, Inc.
The company focuses on rare diseases with its portfolio of therapies addressing small patient populations.
|
$2.79B |
$22.39
-1.80%
|
|
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.
|
$2.73B |
$76.42
-2.81%
|
|
BEAM
Beam Therapeutics Inc.
Pipeline includes rare genetic diseases (SCD, AATD, GSD1a), aligning with Rare Diseases initiatives.
|
$2.72B |
$28.43
+5.92%
|
|
DYN
Dyne Therapeutics, Inc.
Pipeline targets include DM1, DMD, Pompe disease, and FSHD, aligning with a Rare Diseases biotech focus.
|
$2.59B |
$18.09
-0.74%
|
|
EWTX
Edgewise Therapeutics, Inc.
Sevasemten and EDG-7500 address muscular dystrophy and other muscle diseases, aligning with Rare Diseases biotechnology.
|
$2.48B |
$23.09
-2.06%
|
|
VRDN
Viridian Therapeutics, Inc.
Company's focus on Thyroid Eye Disease and autoimmune disease pipelines aligns with Biotech - Rare Diseases.
|
$2.47B |
$30.47
+0.89%
|
|
SLNO
Soleno Therapeutics, Inc.
Biotech - Rare Diseases aligns with Soleno's focus on a rare genetic disorder (PWS) and orphan indication.
|
$2.40B |
$45.44
+0.49%
|
|
WVE
Wave Life Sciences Ltd.
Clinical programs target rare diseases (DMD, AATD, HD), aligning with the Rare Diseases biotech segment.
|
$2.39B |
$15.46
+3.07%
|
|
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.
|
$2.34B |
$17.04
+6.37%
|
|
RARE
Ultragenyx Pharmaceutical Inc.
Ultragenyx is a rare-disease focused biotech company with approved therapies and a pipeline centered on ultra-rare diseases.
|
$2.23B |
$22.52
-2.55%
|
|
HRMY
Harmony Biosciences Holdings, Inc.
Harmony's WAKIX and pipeline assets target rare neurological diseases, aligning with Biotech - Rare Diseases.
|
$2.11B |
$36.65
-0.27%
|
|
MESO
Mesoblast Limited
Focus on rare diseases with pediatric SR-aGvHD and other rare indications in the pipeline.
|
$2.11B |
$18.58
+1.59%
|
|
SRPT
Sarepta Therapeutics, Inc.
Focus on rare genetic diseases (DMD, LGMD) aligns with the Rare Diseases biotech subsegment.
|
$2.06B |
$21.80
+3.46%
|
|
RXRX
Recursion Pharmaceuticals, Inc.
Pipeline includes rare-disease programs (e.g., FAP) and preclinical assets.
|
$1.90B |
$4.59
+5.03%
|
|
SION
Sionna Therapeutics, Inc.
Sionna Therapeutics develops therapies targeting a rare genetic disease (cystic fibrosis), aligning with Biotech - Rare Diseases.
|
$1.80B |
$37.84
-7.37%
|
|
STOK
Stoke Therapeutics, Inc.
Lead programs target rare genetic diseases (Dravet syndrome and ADOA), aligning Stoke with the Biotech - Rare Diseases investable theme.
|
$1.78B |
$34.55
+6.14%
|
|
ANIP
ANI Pharmaceuticals, Inc.
Cortrophin Gel and related Rare Disease assets position ANI in Biotech - Rare Diseases as a core growth engine.
|
$1.71B |
$76.98
-2.14%
|
|
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.
|
$1.69B |
$37.87
-1.97%
|
|
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.
|
$1.59B |
$27.30
-0.53%
|
|
BCRX
BioCryst Pharmaceuticals, Inc.
Pipeline includes rare-disease programs (Netherton syndrome and DME), a key strategic focus for BioCryst.
|
$1.56B |
$7.50
+1.28%
|
|
TYRA
Tyra Biosciences, Inc.
TYRA's pediatric achondroplasia program places it in the rare diseases category.
|
$1.40B |
$26.11
-0.70%
|
|
RLAY
Relay Therapeutics, Inc.
The PROS program targets a rare genetic overgrowth condition, placing part of the pipeline in Rare Diseases.
|
$1.36B |
$8.12
+3.37%
|
|
PHVS
Pharvaris N.V.
HAE and related angioedema therapies fall under Rare Diseases, a core space Pharvaris competes in.
|
$1.35B |
$24.04
-3.40%
|
|
TSHA
Taysha Gene Therapies, Inc.
Rett syndrome is a rare disease, aligning with the company's focus on rare disease gene therapies.
|
$1.33B |
$5.24
+7.16%
|
|
PGEN
Precigen, Inc.
RRP is a rare disease; company has rare-disease focus across pipeline.
|
$1.25B |
$4.50
+7.78%
|
|
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.
|
$1.24B |
$22.52
-0.49%
|
|
DNTH
Dianthus Therapeutics, Inc.
Clinical programs target rare autoimmune neuromuscular diseases such as gMG, CIDP, and MMN.
|
$1.19B |
$36.28
-1.65%
|
|
XERS
Xeris Biopharma Holdings, Inc.
Recorlev and Keveyis target rare endocrine/neuromuscular disorders, aligning with Biotech - Rare Diseases.
|
$1.14B |
$6.97
-1.62%
|
|
PHAR
Pharming Group N.V.
Core rare-disease therapeutic developer focused on RUCONEST and an expanding rare-disease pipeline.
|
$1.14B |
$16.96
+0.89%
|
|
CAPR
Capricor Therapeutics, Inc.
DMD and potential Becker muscular dystrophy indications place Capricor in the rare diseases therapeutic space.
|
$1.13B |
$26.19
+5.56%
|
|
BHVN
Biohaven Ltd.
IgA nephropathy and spinocerebellar ataxia (rare diseases) in the pipeline reflect a Rare Diseases focus.
|
$1.05B |
$9.65
-2.77%
|
|
OMER
Omeros Corporation
TA-TMA, PNH, and C3G represent ultra-rare disease programs in Omeros' pipeline.
|
$1.05B |
$13.78
-10.55%
|
|
AMLX
Amylyx Pharmaceuticals, Inc.
PBH and other pipeline programs target rare diseases, supporting the 'Biotech - Rare Diseases' theme.
|
$1.02B |
$11.28
-0.97%
|
|
NTLA
Intellia Therapeutics, Inc.
Targets rare genetic diseases (HAE and ATTR amyloidosis) with disease-modifying therapy.
|
$1.01B |
$9.64
+2.88%
|
|
SVRA
Savara Inc.
Company focuses on rare respiratory diseases (aPAP), a high-need orphan condition, aligning with the Rare Diseases biotech theme.
|
$1.00B |
$5.58
-3.79%
|
|
MBX
MBX Biosciences, Inc. Common Stock
MBX targets rare endocrine/metabolic disorders (e.g., chronic hypoparathyroidism), aligning with Rare Diseases biotech focus.
|
$984.64M |
$29.00
-1.06%
|
|
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.
|
$978.12M |
$89.10
+0.75%
|
|
TBPH
Theravance Biopharma, Inc.
Ampreloxetine's focus on a rare disease indication aligns with the Biotech - Rare Diseases investable theme.
|
$927.66M |
$18.11
-1.71%
|
|
DAWN
Day One Biopharmaceuticals, Inc.
Pediatric cancer and rare disease context; Day One's focus aligns with Rare Diseases.
|
$875.79M |
$8.64
+1.11%
|
|
VIR
Vir Biotechnology, Inc.
HDV/HBV programs position Vir in Rare Diseases through hepatitis delta and hepatitis B therapeutic strategies.
|
$804.33M |
$5.97
+3.20%
|
|
KALV
KalVista Pharmaceuticals, Inc.
KalVista's focus on therapies for rare diseases (HAE) aligns with the Biotech - Rare Diseases investable theme.
|
$782.28M |
$15.69
+0.19%
|
|
ESPR
Esperion Therapeutics, Inc.
Pipeline program for Primary Sclerosing Cholangitis, a rare liver disease.
|
$768.18M |
$3.72
-2.36%
|
|
IRWD
Ironwood Pharmaceuticals, Inc.
Apraglutide is a next-generation therapy being developed for a rare disease (SBS-IF), matching the Rare Diseases investable theme.
|
$745.57M |
$4.30
-6.21%
|
|
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.
|
$738.84M |
$42.67
+3.59%
|
|
KROS
Keros Therapeutics, Inc.
Duchenne muscular dystrophy (DMD) and associated cytopenias are rare diseases, aligning Keros Therapeutics' focused assets to Rare Diseases.
|
$727.83M |
$17.64
-1.59%
|
|
ATXS
Astria Therapeutics, Inc.
HAE is a rare disease, and Astria's pipeline includes therapies for niche/rare conditions.
|
$723.50M |
$12.90
+0.66%
|
|
ZBIO
Zenas BioPharma, Inc.
IgG4-RD is a rare disease indication targeted by the pivotal program.
|
$699.45M |
$15.02
-9.60%
|
|
RGNX
REGENXBIO Inc.
RGX-121 targets a rare disease (MPS II) in a late-stage program.
|
$676.89M |
$14.05
+4.85%
|
|
MGTX
MeiraGTx Holdings plc
Pipeline includes LCA4 and other rare disease targets, aligning with Rare Diseases as a major focus area.
|
$607.38M |
$7.40
-1.99%
|
|
FULC
Fulcrum Therapeutics, Inc.
Company's focus on sickle cell disease and Diamond-Blackfan anemia positions it in rare genetic diseases.
|
$586.35M |
$9.86
-9.09%
|
|
DRUG
Bright Minds Biosciences Inc.
Lead indications (Absence Epilepsy and DEE) are rare neurological disorders.
|
$566.84M |
$91.70
+14.61%
|
|
VTYX
Ventyx Biosciences, Inc.
Pipeline includes a therapy (VTX2735) for CAPS, a rare disease, aligning with Rare Diseases.
|
$557.63M |
$7.38
-5.69%
|
|
ANNX
Annexon, Inc.
GBS and related autoimmune targets place Annexon in Biotech - Rare Diseases.
|
$515.37M |
$4.67
-6.87%
|
|
LXEO
Lexeo Therapeutics, Inc. Common Stock
Lead programs target Friedreich ataxia and PKP2 cardiomyopathy, both rare diseases.
|
$509.77M |
$9.45
+0.05%
|
|
CRVS
Corvus Pharmaceuticals, Inc.
ALPS is a rare genetic condition pursued in Phase 2, indicating Rare Diseases as a focus area.
|
$505.95M |
$6.63
-2.36%
|
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