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Rare disease stocks
We cover 218 companies grouped under the Rare disease theme. The median market value in the group is $462.18M. Across the group, the median move over the past trading day is +1.25%. 53% of the 204 with a 200-day average trade above it. Price change over the past year runs from −71.9% to +198.0% across the middle 80 percent of the group, with a median of +15.6%.
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Table
218 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% |
| VRTX | Vertex Pharmaceuticals Incorporated | $140.04B | Large cap | +42.14% |
| ARGX | argenx SE | $63.78B | Large cap | +50.52% |
| 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% |
| 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% |
| 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% |
| IMVT | Immunovant, Inc. | $8.95B | Mid cap | +192.14% |
| 4151.T | Kyowa Kirin Co., Ltd. | $8.51B | Mid cap | +3.09% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +14.26% |
| SRRK | Scholar Rock Holding Corporation | $7.09B | Mid cap | +74.77% |
| 0HI3.L | Arrowhead Pharmaceuticals, Inc. | $6.98B | Mid cap | +317.86% |
| ORKA | Oruka Therapeutics, Inc. | $6.74B | Mid cap | +601.72% |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +168.53% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.17B | Mid cap | +142.74% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.13B | Mid cap | +49.03% |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +378.90% |
| TVTX | Travere Therapeutics, Inc. | $6.06B | Mid cap | +288.91% |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +36.58% |
| GRFS | Grifols, S.A. | $5.44B | Mid cap | −17.62% |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +102.62% |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +209.16% |
| MANE | Veradermics, Incorporated | $4.11B | Mid cap | +171.13% |
| CAMX.ST | Camurus AB (publ) | $3.87B | Mid cap | −14.52% |
| CLDX | Celldex Therapeutics, Inc. | $3.24B | Mid cap | +87.96% |
| IRON | Disc Medicine, Inc. | $3.16B | Mid cap | +38.71% |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +58.44% |
| 0RD1.L | Camurus AB (publ) | $2.84B | Mid cap | −13.50% |
| VRDN | Viridian Therapeutics, Inc. | $2.74B | Mid cap | +30.21% |
| PHVS | Pharvaris N.V. | $2.62B | Mid cap | +57.76% |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −9.60% |
| TRVI | Trevi Therapeutics, Inc. | $2.56B | Mid cap | +146.57% |
| VERA | Vera Therapeutics, Inc. | $2.54B | Mid cap | +65.32% |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +176.20% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +17.16% |
| MLYS | Mineralys Therapeutics, Inc. | $2.32B | Mid cap | +78.70% |
| 009420.KS | Hanall Biopharma Co., Ltd. | $2.32B | Mid cap | +108.57% |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.32B | Mid cap | +46.03% |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.23B | Mid cap | +38.03% |
| ZBIO | Zenas BioPharma, Inc. | $2.20B | Mid cap | +103.93% |
| STOK | Stoke Therapeutics, Inc. | $2.16B | Mid cap | +77.32% |
| ADMA | ADMA Biologics, Inc. | $2.14B | Mid cap | −42.86% |
| PVLA | Palvella Therapeutics, Inc. | $2.14B | Mid cap | +195.71% |
| NEU.AX | Neuren Pharmaceuticals Limited | $2.08B | Mid cap | +18.47% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $1.95B | Small cap | −9.02% |
| SEPN | Septerna, Inc. | $1.93B | Small cap | +245.32% |
| 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% |
| 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% |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $1.40B | Small cap | −11.63% |
| 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% |
| 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% |
| TECX | Tectonic Therapeutic, Inc. | $737.21M | Small cap | +53.48% |
| IRWD | Ironwood Pharmaceuticals, Inc. | $725.40M | Small cap | +245.67% |
| 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% |
| ZURA | Zura Bio Limited | $605.64M | Small cap | +208.29% |
| SPRY | ARS Pharmaceuticals, Inc. | $568.84M | Small cap | −57.66% |
| 005257.KS | GC Corp. | $553.81M | Small cap | −41.91% |
| 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% |
| 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% |
| 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% |
| AKBA | Akebia Therapeutics, Inc. | $265.58M | Small cap | −68.37% |
| SLGL | Sol-Gel Technologies Ltd. | $265.20M | Small cap | +289.14% |
| 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% |
| 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% |
| 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% |
| 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% |
| GOSS | Gossamer Bio, Inc. | $99.97M | Micro cap | −91.33% |
| ILA.AX | Island Pharmaceuticals Limited | $99.59M | Micro cap | +104.35% |
| ABNX.PA | ABIONYX Pharma SA | $97.56M | Micro cap | −16.20% |
| FBIO | Fortress Biotech, Inc. | $94.38M | Micro cap | +23.58% |
| PLYX | Polaryx Therapeutics, Inc. | $94.21M | Micro cap | −94.66% |
| RLYB | Rallybio Corporation | $89.67M | Micro cap | +262.65% |
| TELO | Telomir Pharmaceuticals, Inc. | $81.15M | Micro cap | −19.18% |
| RCE.AX | Recce Pharmaceuticals Ltd | $81.01M | Micro cap | −12.79% |
| FBIOP | Fortress Biotech, Inc. | $71.45M | Micro cap | +141.55% |
| STX.L | Shield Therapeutics plc | $70.81M | Micro cap | −41.98% |
| SPRO | Spero Therapeutics, Inc. | $70.76M | Micro cap | −33.51% |
| ALTHX.PA | THX Pharma Société Anonyme | $70.40M | Micro cap | +89.35% |
| RNTX | Rein Therapeutics Inc. | $69.01M | Micro cap | −32.20% |
| IMA | ImageneBio, Inc. | $63.80M | Micro cap | −60.78% |
| ATYR | aTyr Pharma, Inc. | $55.91M | Micro cap | −88.95% |
| SCYX | SCYNEXIS, Inc. | $51.64M | Micro cap | +514.20% |
| MCRB | Seres Therapeutics, Inc. | $46.81M | Micro cap | −74.00% |
| GRCE | Grace Therapeutics, Inc. | $46.28M | Micro cap | −32.31% |
| MREO | Mereo BioPharma Group plc | $44.97M | Micro cap | −83.82% |
| EDSA | Edesa Biotech, Inc. | $42.90M | Micro cap | +83.88% |
| BOT.AX | Botanix Pharmaceuticals Limited | $39.22M | Micro cap | −87.10% |
| QNCX | Quince Therapeutics, Inc. | $34.05M | Micro cap | +95.76% |
| NNVC | NanoViricides, Inc. | $32.93M | Micro cap | −12.84% |
| 520A.T | J-PHARMACEUTICALS CO LTD | $31.08M | Micro cap | −60.71% |
| ALDVI.PA | Advicenne S.A. | $29.99M | Micro cap | +41.44% |
| 4591.T | Ribomic Inc. | $29.85M | Micro cap | −22.94% |
| IMM.L | ImmuPharma plc | $29.03M | Micro cap | +83.56% |
| 0RQJ.L | Saniona AB (publ) | $27.98M | Micro cap | −13.86% |
| BRNS | Barinthus Biotherapeutics plc | $26.96M | Micro cap | −44.77% |
| JSPR | Jasper Therapeutics, Inc. | $23.96M | Micro cap | −75.67% |
| TVRD | Tvardi Therapeutics, Inc. | $23.74M | Micro cap | −93.77% |
| RVX.TO | Resverlogix Corp. | $23.62M | Micro cap | +5.00% |
| COCP | Cocrystal Pharma, Inc. | $21.77M | Micro cap | −26.62% |
| KTTA | Pasithea Therapeutics Corp. | $18.71M | Micro cap | −21.90% |
| CALC | CalciMedica, Inc. | $17.10M | Micro cap | −78.92% |
| 4881.T | FunPep Company Limited | $15.92M | Micro cap | −56.14% |
| HIND | Vyome Holdings, Inc. | $15.65M | Micro cap | −80.23% |
| ALBPS.PA | Biophytis S.A. | $12.86M | Micro cap | −53.47% |
| ANNX.ST | Annexin Pharmaceuticals AB (publ) | $10.31M | Micro cap | −49.23% |
| QNRX | Quoin Pharmaceuticals, Ltd. | $10.18M | Micro cap | −31.02% |
| AZTR | Azitra, Inc. | $9.55M | Micro cap | −78.97% |
| PHR.WA | Pharmena S.A. | $9.49M | Micro cap | −7.51% |
| IMRN | Immuron Limited | $9.20M | Micro cap | −36.84% |
| SNGX | Soligenix, Inc. | $8.81M | Micro cap | −87.06% |
| IMC.AX | Immuron Limited | $8.33M | Micro cap | −46.38% |
| XCUR | Exicure, Inc. | $7.79M | Micro cap | −79.90% |
| INM | InMed Pharmaceuticals Inc. | $7.69M | Micro cap | −35.43% |
| VBS.AX | Vectus Biosystems Limited | $6.65M | Micro cap | +87.50% |
| PULM | Pulmatrix, Inc. | $5.84M | Micro cap | −67.81% |
| BIOSGN.ST | Biosergen AB (publ) | $5.62M | Micro cap | −95.33% |
| JAGX | Jaguar Health, Inc. | $4.78M | Micro cap | −98.52% |
| GRI | GRI Bio, Inc. | $4.28M | Micro cap | −95.14% |
| XRTX | XORTX Therapeutics Inc. | $4.11M | Micro cap | −44.74% |
| BIT.AX | Biotron Limited | $3.99M | Micro cap | −33.33% |
| SXTP | 60 Degrees Pharmaceuticals, Inc. | $3.97M | Micro cap | −78.10% |
| MTNB | Matinas BioPharma Holdings, Inc. | $2.90M | Micro cap | −90.82% |
| APTA.ST | Aptahem AB (publ) | $1.53M | Micro cap | −72.50% |
| APLI.TO | Appili Therapeutics Inc. | $1.44M | Micro cap | −25.00% |
| VBIO | Valion Bio, Inc. | $660.05K | Micro cap | −95.00% |
| ALNEV.PA | Neovacs S.A. | $29.00 | Micro cap | −11.54% |
| 0A43.L | Inovio Pharmaceuticals, Inc. | — | — | −45.78% |
| 0A4D.L | Equillium, Inc. | — | — | +24.24% |
| 0A6I.L | Soligenix, Inc. | — | — | −86.94% |
| 0A9G.L | Mereo BioPharma Group plc | — | — | −83.87% |
| 0L49.L | SCYNEXIS, Inc. | — | — | +543.07% |
| 0QNS.L | CURATIS AG CURATIS AG AG ORD SH | — | — | −15.04% |
| 0UNL.L | NEKTAR THERAPEUTICS NEKTAR THER | — | — | +14.54% |
| 1AE.DE | argenx SE | — | — | +35.39% |
| 2X1.DE | ABIVAX Société Anonyme | — | — | +35.69% |
| BIOV.SW | BioVersys AG | — | — | −8.49% |
| BPBS.PA | BIOPHYTIS BSA | — | — | 0.00% |
| CESSA.CO | Cessatech A/S | — | — | −46.63% |
| CURN.SW | Curatis Holding AG | — | — | +73.80% |
| DBV.DE | DBV Technologies S.A. | — | — | +61.15% |
| PHGN.DE | Pharming Group N.V. | — | — | −21.41% |
| SANN.SW | Santhera Pharmaceuticals Holding AG | — | — | +15.63% |
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.