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Rare disease stocks
We cover 132 companies grouped under the Rare disease theme. The median market value in the group is $702.72M. Across the group, the median move over the past trading day is +1.55%. 61% of the 126 with a 200-day average trade above it. Price change over the past year runs from −39.8% to +268.9% across the middle 80 percent of the group, with a median of +41.7%.
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Table
132 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return |
|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | $140.77B | Large cap | +40.14% |
| VRTX34.SA | Vertex Pharmaceuticals Incorporated | $140.14B | Large cap | −83.17% |
| VRTX | Vertex Pharmaceuticals Incorporated | $140.04B | Large cap | +42.14% |
| 0QZU.L | Vertex Pharmaceuticals Incorporated | $112.52B | Large cap | +42.39% |
| ARGX | argenx SE | $63.78B | Large cap | +50.52% |
| A1RG34.SA | argenx SE | $63.17B | Large cap | +44.49% |
| 0HD2.L | Alnylam Pharmaceuticals, Inc. | $35.05B | Large cap | −46.85% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −45.88% |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | $31.87B | Large cap | −11.75% |
| A1LN34.SA | Alnylam Pharmaceuticals, Inc. | $31.78B | Large cap | −49.89% |
| INSM | Insmed Incorporated | $27.07B | Large cap | −3.45% |
| ROIV | Roivant Sciences Ltd. | $26.26B | Large cap | +213.32% |
| JAZZ | Jazz Pharmaceuticals plc | $16.89B | Large cap | +108.87% |
| ASND | Ascendis Pharma A/S | $16.59B | Large cap | +32.89% |
| BBIO | BridgeBio Pharma, Inc. | $15.85B | Large cap | +70.01% |
| CORT | Corcept Therapeutics Incorporated | $13.36B | Large cap | +79.56% |
| 0JAV.L | Insmed Incorporated | $12.89B | Large cap | −6.25% |
| BMRN | BioMarin Pharmaceutical Inc. | $12.80B | Large cap | +13.03% |
| REC.MI | Recordati Industria Chimica e Farmaceutica S.p.A. | $12.60B | Large cap | +2.09% |
| B1MR34.SA | BioMarin Pharmaceutical Inc. | $12.54B | Large cap | +6.36% |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $12.52B | Large cap | +329.16% |
| CYTK | Cytokinetics, Incorporated | $10.77B | Large cap | +109.29% |
| IONS | Ionis Pharmaceuticals, Inc. | $10.56B | Large cap | +52.01% |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +202.82% |
| ABVX.PA | ABIVAX Société Anonyme | $10.42B | Large cap | +42.59% |
| 0JDI.L | Ionis Pharmaceuticals, Inc. | $10.40B | Large cap | +49.07% |
| PTGX | Protagonist Therapeutics, Inc. | $10.01B | Large cap | +164.75% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $1.95B | Small cap | −9.02% |
| SEPN | Septerna, Inc. | $1.93B | Small cap | +245.32% |
| ETON | Eton Pharmaceuticals, Inc. | $1.80B | Small cap | +268.93% |
| ANIP | ANI Pharmaceuticals, Inc. | $1.74B | Small cap | −16.24% |
| ANAB | AnaptysBio, Inc. | $1.73B | Small cap | +335.60% |
| AVLN | Avalyn Pharma Inc. | $1.69B | Small cap | +29.54% |
| 0L35.L | Sarepta Therapeutics, Inc. | $1.69B | Small cap | +4.63% |
| MAZE | Maze Therapeutics, Inc. | $1.69B | Small cap | +128.55% |
| FBRX | Forte Biosciences, Inc. | $1.63B | Small cap | +580.25% |
| TRAX | First Tracks Biotherapeutics, Inc. | $1.61B | Small cap | +137.42% |
| XERS | Xeris Biopharma Holdings, Inc. | $1.59B | Small cap | +12.48% |
| TYRA | Tyra Biosciences, Inc. | $1.59B | Small cap | +122.42% |
| 4516.T | Nippon Shinyaku Co., Ltd. | $1.53B | Small cap | +18.84% |
| A2RR34.SA | Arrowhead Pharmaceuticals, Inc. | $1.51B | Small cap | +301.52% |
| PYC.AX | PYC Therapeutics Limited | $1.48B | Small cap | +63.71% |
| KALV | KalVista Pharmaceuticals Inc | $1.44B | Small cap | +107.85% |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $1.40B | Small cap | −11.63% |
| OMER | Omeros Corporation | $1.40B | Small cap | +343.22% |
| JBIO | Jade Biosciences, Inc. | $1.39B | Small cap | +176.46% |
| MLTX | MoonLake Immunotherapeutics | $1.32B | Small cap | −71.52% |
| 0HB0.L | Agios Pharmaceuticals, Inc. | $1.19B | Small cap | −14.25% |
| SVRA | Savara Inc. | $1.15B | Small cap | +78.34% |
| 006280.KS | GC Biopharma Corp. | $1.06B | Small cap | −1.88% |
| AVTX | Avalo Therapeutics, Inc. | $1.06B | Small cap | +110.00% |
| WVE | Wave Life Sciences, Inc. | $1.03B | Small cap | −47.18% |
| CLYM | Climb Bio Inc | $1.02B | Small cap | +619.35% |
| DSGN | Design Therapeutics, Inc. | $981.28M | Small cap | +201.54% |
| ANNX | Annexon, Inc. | $976.33M | Small cap | +139.53% |
| DBVT | DBV Technologies S.A. | $940.80M | Small cap | +52.91% |
| DBV.PA | DBV Technologies S.A. | $928.30M | Small cap | +54.11% |
| SLN | Silence Therapeutics plc | $849.65M | Small cap | +170.16% |
| GNFT.PA | Genfit S.A. | $836.50M | Small cap | +293.99% |
| PHAR | Pharming Group N.V. | $833.06M | Small cap | −19.75% |
| STTK | Shattuck Labs, Inc. | $792.87M | Small cap | +710.87% |
| 0QNA.L | Basilea Pharmaceutica AG | $762.45M | Small cap | +38.97% |
| TECX | Tectonic Therapeutic, Inc. | $737.21M | Small cap | +53.48% |
| IRWD | Ironwood Pharmaceuticals, Inc. | $725.40M | Small cap | +245.67% |
| ASMB | Assembly Biosciences, Inc. | $718.32M | Small cap | +32.72% |
| ZVRA | Zevra Therapeutics, Inc. | $714.98M | Small cap | +28.63% |
| AGMB | AgomAb Therapeutics NV | $690.46M | Small cap | −4.30% |
| GYRE | Gyre Therapeutics, Inc. | $682.86M | Small cap | −18.54% |
| CRMD | CorMedix Inc. | $667.20M | Small cap | −37.65% |
| AYJ.DE | Valneva SE | $659.69M | Small cap | −25.10% |
| VALN | Valneva SE | $642.90M | Small cap | −28.10% |
| ZURA | Zura Bio Limited | $605.64M | Small cap | +208.29% |
| AQST | Aquestive Therapeutics, Inc. | $602.46M | Small cap | +31.61% |
| 0KBU.L | Omeros Corporation | $575.28M | Small cap | +341.05% |
| SPRY | ARS Pharmaceuticals, Inc. | $568.84M | Small cap | −57.66% |
| 005257.KS | GC Corp. | $553.81M | Small cap | −41.91% |
| KMDA | Kamada Ltd. Ordinary Shares | $497.90M | Small cap | +26.12% |
| EPRX | Eupraxia Pharmaceuticals Inc. | $490.26M | Small cap | +41.87% |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | $487.17M | Small cap | +41.67% |
| QTTB | Q32 Bio Inc. | $485.84M | Small cap | +728.43% |
| RZLT | Rezolute, Inc. | $482.42M | Small cap | −28.43% |
| AVIR | Atea Pharmaceuticals, Inc. | $441.93M | Small cap | +48.36% |
| LRMR | Larimar Therapeutics, Inc. | $432.15M | Small cap | +11.83% |
| XFOR | X4 Pharmaceuticals, Inc. | $412.45M | Small cap | +4.79% |
| ENTA | Enanta Pharmaceuticals, Inc. | $410.67M | Small cap | +62.59% |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | $397.97M | Small cap | −7.40% |
| UPB | Upstream Bio, Inc. | $393.26M | Small cap | −57.08% |
| CUV.AX | Clinuvel Pharmaceuticals Limited | $381.21M | Small cap | −21.84% |
| IKT | Inhibikase Therapeutics, Inc. | $348.84M | Small cap | +42.05% |
| PRQR | ProQR Therapeutics N.V. | $335.86M | Small cap | +5.78% |
| IFRX | InflaRx N.V. | $302.10M | Small cap | +167.10% |
| TRDA | Entrada Therapeutics, Inc. | $296.70M | Small cap | +35.35% |
| TTRX | Turn Therapeutics Inc. | $295.48M | Small cap | +39.00% |
| FULC | Fulcrum Therapeutics, Inc. | $294.51M | Small cap | −39.78% |
| SION | Sionna Therapeutics, Inc. | $282.58M | Small cap | −74.02% |
| ELDN | Eledon Pharmaceuticals, Inc. | $281.98M | Small cap | +35.02% |
| CAMP | CAMP4 THERAPEUTICS CORPORATION | $279.25M | Small cap | +185.26% |
| IVVD | Invivyd, Inc. | $277.81M | Small cap | +67.62% |
| 0OB3.L | Valneva SE | $270.41M | Small cap | −24.84% |
| AKBA | Akebia Therapeutics, Inc. | $265.58M | Small cap | −68.37% |
| SLGL | Sol-Gel Technologies Ltd. | $265.20M | Small cap | +289.14% |
| XUP.DE | Genfit S.A. | $258.22M | Small cap | +166.82% |
| 0QN1.L | Santhera Pharmaceuticals Holding AG | $254.58M | Small cap | +12.30% |
| 0T6G.L | Q32 Bio Inc. | $246.69M | Small cap | — |
| RNA | Prosensa Holding B.V. Ordinary Shares | $239.48M | Small cap | −5.08% |
| PEPG | PepGen Inc. | $237.21M | Small cap | +161.45% |
| CRDL | Cardiol Therapeutics Inc. | $233.65M | Small cap | +75.44% |
| CRDL.TO | Cardiol Therapeutics Inc. | $233.07M | Small cap | +72.50% |
| KROS | Keros Therapeutics, Inc. | $213.74M | Small cap | −20.87% |
| ANTX | AN2 Therapeutics, Inc. | $213.59M | Small cap | +403.11% |
| SIGA | SIGA Technologies, Inc. | $211.24M | Small cap | −60.50% |
| ARMP | Armata Pharmaceuticals, Inc. | $206.73M | Small cap | +113.85% |
| MSLE | Satellos Bioscience Inc. | $200.92M | Small cap | +82.17% |
| MSCL.TO | Satellos Bioscience Inc. | $199.37M | Small cap | +80.56% |
| PLX | Protalix BioTherapeutics, Inc. | $193.37M | Small cap | +50.62% |
| 0AB6.L | Hyloris Pharmaceuticals SA | $185.32M | Small cap | −32.01% |
| SPRB | Spruce Biosciences, Inc. | $182.21M | Small cap | +697.48% |
| MDWD | MediWound Ltd. | $167.45M | Small cap | −24.81% |
| AARD | Aardvark Therapeutics, Inc. | $158.67M | Small cap | −19.80% |
| CRNO-B.ST | Cereno Scientific AB (publ) | $155.36M | Small cap | −53.95% |
| UNCY | Unicycive Therapeutics, Inc. | $146.28M | Small cap | +27.51% |
| MIST | Milestone Pharmaceuticals Inc. | $144.42M | Small cap | −31.36% |
| 4599.T | StemRIM | $140.21M | Small cap | +15.91% |
| EQ | Equillium, Inc. | $139.50M | Small cap | +14.74% |
| DXB.AX | Dimerix Limited | $131.21M | Small cap | −35.79% |
| CPIX | Cumberland Pharmaceuticals Inc. | $130.35M | Small cap | +166.87% |
| INO | Inovio Pharmaceuticals, Inc. | $129.25M | Small cap | −33.85% |
| 0H8T.L | Aclaris Therapeutics, Inc. | $122.82M | Small cap | +265.21% |
| MDP.TO | Medexus Pharmaceuticals Inc. | $116.99M | Small cap | +80.70% |
| RFL | Rafael Holdings, Inc. | $113.88M | Small cap | +52.38% |
| PTHS | Pelthos Therapeutics Inc. | $104.13M | Small cap | +20.41% |
| PYPD | PolyPid Ltd. | $101.25M | Small cap | +47.83% |
Covers ~22,000 companies across the US, Europe and Asia-Pacific (ex mainland China, Hong Kong and India), refreshed nightly. Every listed stock opens as a full analysis for $9.90.