Biotech - Rare Diseases
•215 stocks
•
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Market Cap Distribution
Price Performance Heatmap
5Y Price (Market Cap Weighted)
All Stocks (215)
%
| Company | Market Cap | Price |
|---|---|---|
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The Rare Diseases portfolio (e.g., Ultomiris, Strensiq, Koselugo) anchors AstraZeneca’s orphan/rare disease strategy.
|
$262.04B |
$84.53
-1.28%
|
|
Fabhalta and related programs target Rare Diseases, a major disease category.
|
$252.00B |
$130.00
|
|
UPLIZNA (IgG4-RD, gMG) places Amgen in Biotech - Rare Diseases.
|
$156.00B |
$290.13
-2.04%
|
|
Pfizer has products addressing rare diseases (e.g., tafamidis family for ATTR amyloidosis).
|
$137.19B |
$24.09
-0.15%
|
|
Sanofi pursues therapies in Rare Diseases, a substantive segment of its pipeline and portfolio.
|
$120.19B |
$98.46
|
|
CASGEVY targets sickle cell disease and transfusion-dependent beta-thalassemia, aligning with rare-disease therapeutics.
|
$104.72B |
$407.79
-1.34%
|
|
Reblozyl targets rare-disease anemia indications, aligning with the Rare Diseases category.
|
$89.46B |
$43.96
-1.83%
|
|
Biotech - Rare Diseases captures programs targeting rare conditions (e.g., PNH, gMG) mentioned in the pipeline.
|
$60.96B |
$564.63
-0.82%
|
|
ATTR amyloidosis and broader rare-disease indications (ATTR-CM and PN) position Alnylam within Biotech - Rare Diseases.
|
$60.04B |
$460.46
+0.04%
|
|
Biotech - Rare Diseases tag captures ARGx's focus on indications like gMG and CIDP which are rare/neuroimmunologic.
|
$47.65B |
$796.92
-0.76%
|
|
IgA nephropathy and other rare-disease opportunities align with Takeda's rare diseases focus.
|
$43.58B |
$27.33
-2.62%
|
|
TAKHZYRO targets hereditary angioedema, aligning with Biotech - Rare Diseases.
|
$43.47B |
$13.71
-1.01%
|
|
Insmed's INS1201 program targets Duchenne muscular dystrophy, placing it in the Rare Diseases biotech space.
|
$29.57B |
$162.33
-1.08%
|
|
Biogen's late-stage portfolio includes rare-disease programs (e.g., QALSODY for SOD1-ALS) and other rare-disease assets.
|
$21.48B |
$146.57
-2.29%
|
|
PAH/ILD-related therapies align with Rare Diseases-focused biotechnology categories, given the niche, high-need patient populations.
|
$19.85B |
$440.00
-1.27%
|
|
Ascendis is cornerstone in rare-disease therapies with marketed products YORVIPATH and SKYTROFA and a pipeline (TransCon CNP) targeting rare conditions, matching the Biotech - Rare Diseases theme.
|
$12.06B |
$209.55
-2.16%
|
|
Ionis has multiple late-stage and marketed assets across rare diseases (e.g., FCS, HAE, Alexander disease, Angelman), forming a core rare-disease portfolio.
|
$11.14B |
$69.97
-0.04%
|
|
The pipeline includes rare autoimmune diseases (e.g., dermatomyositis, Sjögren's, CLE), aligning with Rare Diseases.
|
$10.90B |
$16.04
-0.50%
|
|
BridgeBio's pipeline targets rare genetic diseases (e.g., ADH1, achondroplasia, LGMD2I, Canavan disease) with high unmet need.
|
$10.54B |
$55.49
+1.20%
|
|
Moderna is advancing rare-disease programs (e.g., PA mRNA-3927 and MMA mRNA-3705), identifying the company as active in rare-disease therapeutics.
|
$10.38B |
$26.83
-3.42%
|
|
BioMarin focuses on rare genetic disorders and enzyme therapies, aligning with the rare-disease biotech investment theme.
|
$10.03B |
$52.32
-3.40%
|
|
Systemic mastocytosis is a rare disease, and the company centers on therapies for this rare-disease indication.
|
$8.27B |
$129.46
|
|
Dordaviprone and other activities align with rare diseases focus.
|
$7.99B |
$129.59
-0.16%
|
|
The hypercortisolism focus aligns with rare diseases/endocrine disorder therapeutics within the biotech landscape.
|
$7.84B |
$73.96
-15.51%
|
|
CK-089 represents a rare neuromuscular disease program, aligning with Biotech - Rare Diseases.
|
$7.17B |
$60.04
-0.38%
|
|
Rhythm's lead therapy IMCIVREE targets rare neuroendocrine diseases (MC4R pathway deficiencies) and the company is expanding into hypothalamic obesity, a rare disease indication.
|
$6.41B |
$100.82
+1.04%
|
|
The company targets rare/life-threatening conditions with plasma-derived therapies, aligning with the Rare Diseases biotech theme.
|
$6.41B |
$9.32
-1.58%
|
|
Hemoglobinopathies and Type 1 Diabetes gene-edited therapies align with rare disease-focused programs.
|
$6.31B |
$73.07
-5.51%
|
|
Rusfertide targets a rare hematologic disorder (polycythemia vera) and the pipeline includes additional rare-disease/complex metabolic candidates, fitting Biotech - Rare Diseases.
|
$5.39B |
$87.00
+29.10%
|
|
PTC Therapeutics focuses on rare genetic disorders across its portfolio, including Sephience, Translarna/Emflaza, vatiquinone, and Huntington's program, aligning with Rare Diseases.
|
$5.25B |
$66.23
+0.23%
|
|
DEB and other pipeline programs target rare diseases, aligning with the Rare Diseases category.
|
$5.24B |
$181.47
-4.43%
|
|
Pipeline targets rare neuromuscular diseases (DM1, FSHD, DMD), a rare disease focus.
|
$5.18B |
$42.95
+3.74%
|
|
Familial chylomicronemia syndrome (rare genetic disease) fits the rare diseases franchise.
|
$4.89B |
$35.41
-1.64%
|
|
Pipeline targets rare endocrine diseases and endocrine-related tumors, aligning with Rare Diseases within Biotech.
|
$4.08B |
$42.32
-2.74%
|
|
VK0214 targets X-ALD, a rare disease, supporting the Biotech - Rare Diseases category.
|
$3.77B |
$33.56
-6.13%
|
|
SMA is a rare disease and Scholar Rock's lead program apitegromab targets this indication.
|
$3.75B |
$39.46
-3.94%
|
|
Mirum's core business is focused on rare diseases with approved medicines and a pipeline targeting orphan conditions (CTX, PFIC/ALGS, PSC/PBC).
|
$3.64B |
$73.57
-0.65%
|
|
ACADIA is focused on neuro-rare diseases (e.g., Rett syndrome, Prader-Willi–related programs) and expands via pipeline in rare CNS conditions.
|
$3.52B |
$21.05
-0.94%
|
|
Biotech - Rare Diseases: The desmoid tumor and NF1-PN programs target rare genetic/rare diseases with high unmet need.
|
$3.52B |
$46.99
|
|
Biotech - Rare Diseases: company focusing on rare disease diagnostics and data assets.
|
$3.47B |
$121.72
-4.53%
|
|
Company focuses on therapies for rare diseases (PWS as a primary indication).
|
$3.27B |
$62.72
-1.74%
|
|
Nav1.1 program for Dravet Syndrome aligns with rare disease drug development for pediatric epilepsy.
|
$3.04B |
$39.64
-4.34%
|
|
The lead and pipeline programs target Huntington's disease and other rare disorders, placing the company in the rare diseases therapeutic space.
|
$3.01B |
$54.50
-2.45%
|
|
Focused on rare/ultra-rare genetic diseases with multiple approved therapies and a pipeline targeting these indications.
|
$2.97B |
$31.45
-1.13%
|
|
Pipeline includes rare-disease nephrology indications (C3G/IC-MPGN) and a rare-disease therapy (PNH), supporting a Rare Diseases focus.
|
$2.86B |
$22.76
-1.85%
|
|
Rare diseases focus: recurrent pericarditis is a rare condition and the company emphasizes addressing this niche.
|
$2.76B |
$37.89
-0.58%
|
|
Beam targets rare and genetic diseases (e.g., SCD, AATD, GSD1a), aligning with the Rare Diseases biotech investable theme.
|
$2.65B |
$26.32
-7.58%
|
|
Core business focus on rare-disease therapies (Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease) sold/commercialized directly.
|
$2.56B |
$8.30
|
|
Disc Medicine focuses on rare hematologic diseases (e.g., EPP and Diamond-Blackfan Anemia) with pipeline targeting iron/heme pathways, fitting the Rare Diseases theme.
|
$2.42B |
$69.90
-0.87%
|
|
Company focus is on rare diseases (PK deficiency, thalassemia, sickle cell, etc.), a core rare-disease biotech strategy.
|
$2.35B |
$40.61
-4.47%
|
|
Catalyst's core business is developing and commercializing rare-disease therapies (FIRDAPSE, AGAMREE, FYCOMPA), aligning with Biotech - Rare Diseases.
|
$2.33B |
$19.13
-2.60%
|
|
Belite Bio's pipeline targets rare diseases (STGD1/GA) and retinal degenerations.
|
$2.31B |
$79.28
+1.03%
|
|
Denali focuses on rare lysosomal storage diseases (e.g., Hunter syndrome, Sanfilippo Type A) with DNL310 and DNL126, fitting the Biotech - Rare Diseases theme.
|
$2.23B |
$15.37
-5.12%
|
|
Travere's lead product FILSPARI (sparsentan) targets rare kidney diseases (IgA nephropathy) and is the core commercial/late-stage product, classed under Rare Diseases.
|
$2.21B |
$24.85
+2.10%
|
|
Focus on rare-disease therapeutics (DMD, LGMD, DM1, FSHD, etc.) aligns Sarepta with the Rare Diseases investment theme.
|
$2.17B |
$22.11
-4.49%
|
|
Rare disease focus: pipeline targets pediatric SR-aGvHD, inflammatory colitis, and HLHS-related indications, aligning with rare/orphan disease therapeutics.
|
$2.11B |
$18.32
-4.03%
|
|
Pipeline includes rare-disease programs, indicating a focus on Rare Diseases as an investable theme.
|
$2.02B |
$4.81
-3.99%
|
|
Biotech - Rare Diseases aligns with Cortrophin Gel and ophthalmology assets (ILUVIEN, YUTIQ) as ANI's primary growth engine.
|
$1.99B |
$90.17
-1.51%
|
|
PAH/PH-ILD represent rare diseases, aligning Liquidia with the Rare Diseases biotech focus.
|
$1.98B |
$23.19
-5.19%
|
|
Non-Advanced Systemic Mastocytosis and related KIT-driven indications are rare diseases, aligning with the Rare Diseases tag.
|
$1.85B |
$16.27
|
|
TED is a rare autoimmune condition; the company is pursuing therapies for rare diseases.
|
$1.71B |
$20.99
-3.94%
|
|
Lead indications (HAE and ATTR) are rare genetic diseases, aligning with Biotech - Rare Diseases.
|
$1.69B |
$16.31
-1.33%
|
|
IgA nephropathy is a rare disease; Vera's lead product targets this rare-disease indication.
|
$1.67B |
$26.14
-0.04%
|
|
STOKE is targeting rare genetic diseases (haploinsufficiency) with lead programs for Dravet syndrome and ADOA, a core Rare Diseases focus.
|
$1.65B |
$30.19
-2.46%
|
|
Covers rare/disease-focused programs (e.g., spin for SCA) within the pipeline.
|
$1.61B |
$15.80
-9.97%
|
|
Focuses on genetically driven neuromuscular diseases, a hallmark of rare-disease therapeutic development.
|
$1.60B |
$14.05
-0.57%
|
|
Programs target rare muscle/heart diseases (Duchenne/Becker muscular dystrophy and hypertrophic cardiomyopathy), fitting the Biotech - Rare Diseases category.
|
$1.59B |
$15.11
-5.92%
|
|
ZYN002 and EPX-100/EPX-200 address rare CNS disorders (Fragile X syndrome and other DEEs), placing Harmony in the Biotech - Rare Diseases category.
|
$1.52B |
$26.54
-1.19%
|
|
The company concentrates on rare-disease therapies with a pipeline that includes BCX17725 for Netherton syndrome and avoralstat for DME, indicating a strategic focus on rare diseases.
|
$1.51B |
$7.22
-0.48%
|
|
Cystic fibrosis is a rare genetic disease; the company’s CF-focused therapies place it in the Rare Diseases biotech space.
|
$1.32B |
$29.93
-0.28%
|
|
PPP and related endocrine disorders (e.g., certain rare indications) align with the Rare Diseases investable theme, which can apply to Xeris via its niche, targeted therapies.
|
$1.28B |
$7.99
-2.44%
|
|
PBH and Wolfram PSP represent rare diseases addressed by Amylyx's pipeline, aligning with the Biotech - Rare Diseases theme.
|
$1.27B |
$14.26
-1.11%
|
|
Lead programs target rare disease indications (e.g., APOL1 kidney disease), aligning Maze with Biotech - Rare Diseases.
|
$1.20B |
$27.43
-4.62%
|
|
Pharvaris focuses on rare diseases (HAE and AAE-C1INH), aligning with the Rare Diseases biotech/therapeutics category.
|
$1.19B |
$21.98
-3.21%
|
|
WVE targets rare diseases (e.g., Huntington's disease, DMD, AATD) with specialized RNA therapies, aligning with the Rare Diseases biotech theme.
|
$1.17B |
$7.60
-8.21%
|
|
The PROS (PIK3CA-related overgrowth spectrum) program situates Relay in rare genetic diseases, fitting Biotech - Rare Diseases.
|
$1.11B |
$6.50
-5.80%
|
|
Lead product is a monoclonal antibody targeting active C1s to inhibit the classical complement pathway for rare autoimmune and inflammatory diseases.
|
$1.11B |
$34.46
-1.06%
|
|
PTH1R/hypoparathyroidism and mast cell disease programs place Septerna in Rare Diseases within biotech.
|
$1.03B |
$23.07
+5.20%
|
|
Targeting Rett syndrome, a rare genetic CNS disease, making Rare Diseases a core focus.
|
$1.01B |
$4.81
+1.91%
|
|
The company is focused on rare-disease therapies, including RUCONEST, Joenja, and KL1333, aligning with the Rare Diseases biotech theme.
|
$968.01M |
$14.27
-3.58%
|
|
IgG4-RD is the lead indication, classifying under rare diseases
|
$963.44M |
$23.03
+5.84%
|
|
RRP is a rare disease; Precigen's lead asset targets a rare-disease indication.
|
$959.34M |
$3.25
-3.85%
|
|
Praxis targets rare genetic CNS epilepsies (e.g., SCN2A-DEE, SCN8A-DEE) and other rare diseases within its pipeline.
|
$859.98M |
$47.67
-1.37%
|
|
Rett syndrome and other rare CNS disorders in the pipeline align with Biotech - Rare Diseases.
|
$791.40M |
$9.27
-1.80%
|
|
Palvella Therapeutics focuses on rare genetic skin diseases (microcystic LMs and cutaneous VMs) with its QTORIN topical rapamycin platform, which aligns with the Biotech - Rare Diseases investable theme.
|
$772.79M |
$69.90
+1.29%
|
|
HDV program represents a rare-disease hepatitis-focused therapeutic strategy with accelerated development potential.
|
$758.93M |
$5.49
+5.98%
|
|
Targets rare diseases (Wilson's Disease, Alpha-1 Antitrypsin Deficiency) in its liver-franchise programs.
|
$745.75M |
$5.68
-9.55%
|
|
Company pipeline targets pediatric cancer with rare-disease indications (pLGG), aligning with Rare Diseases in biotech.
|
$733.86M |
$7.24
-3.34%
|
|
Rezolute is focused on ultra-rare diseases, with its lead asset ersodetug targeting hyperinsulinism disorders.
|
$707.24M |
$8.27
-6.02%
|
|
Rare disease focus (MSA-related nOH) places ampreloxetine within the rare diseases biotech/drug development theme.
|
$699.52M |
$13.99
-3.12%
|
|
Gyre Therapeutics is a fibrosis-focused biotech developing and commercializing anti-fibrotic therapies (ETUARY pirfenidone derivative, Hydronidone/F351, Nintedanib, Avatrombopag) and pursuing global liver-fibrosis indications, aligning with the Biotech - Rare Diseases theme.
|
$694.74M |
$7.41
-2.50%
|
|
TYRA is pursuing therapies for ACH, a genetically defined rare disease driven by FGFR3 mutations.
|
$689.72M |
$12.97
+3.10%
|
|
Duchenne muscular dystrophy (DMD) and myelodysplastic syndromes (MDS) are rare diseases, and Keros' assets target these indications via TGF-β pathway modulation.
|
$637.66M |
$15.70
-2.73%
|
|
Rare disease focus: LCA4 program constitutes a rare disease asset in the pipeline.
|
$630.06M |
$7.84
-11.11%
|
|
Zevra Therapeutics is vertically focused on developing and commercializing therapies for rare diseases (NPC, UCDs, VEDS, IH/narcolepsy), directly aligning with Biotech - Rare Diseases.
|
$602.02M |
$11.01
+0.18%
|
|
Savara's focus is on rare respiratory diseases (e.g., autoimmune pulmonary alveolar proteinosis) and orphan lung conditions.
|
$582.46M |
$3.37
-1.75%
|
|
HAE is a rare genetic disease and KalVista is developing a therapy, aligning with the 'Biotech - Rare Diseases' investable theme.
|
$574.47M |
$11.50
-2.54%
|
|
Biotech - Rare Diseases: ALPS is a rare genetic disease targeted by the company's Phase 2 program.
|
$569.88M |
$7.39
+11.30%
|
|
RGX-121 targets MPS II (Hunter syndrome), a rare disease, aligning with Biotech - Rare Diseases.
|
$547.75M |
$10.92
-9.83%
|
|
ATTR amyloidosis program coramitug (depleter) with Novo Nordisk represents a rare-disease pipeline asset.
|
$540.96M |
$10.05
-2.80%
|
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