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Rare disease stocks
We cover 173 companies grouped under the Rare disease theme. The median market value in the group is $237.21M. Across the group, the median move over the past trading day is −0.61%. 46% of the 166 with a 200-day average trade above it. Price change over the past year runs from −75.2% to +178.1% across the middle 80 percent of the group, with a median of −2.0%.
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Table
173 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return |
|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | $139.82B | Large cap | +41.64% |
| VRTX | Vertex Pharmaceuticals Incorporated | $138.63B | Large cap | +40.66% |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | $32.28B | Large cap | −10.21% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −47.38% |
| A1LN34.SA | Alnylam Pharmaceuticals, Inc. | $31.78B | Large cap | −49.35% |
| INSM | Insmed Incorporated | $27.16B | Large cap | −7.54% |
| ROIV | Roivant Sciences Ltd. | $26.55B | Large cap | +206.51% |
| JAZZ | Jazz Pharmaceuticals plc | $16.89B | Large cap | +100.35% |
| ASND | Ascendis Pharma A/S | $16.41B | Large cap | +29.66% |
| BBIO | BridgeBio Pharma, Inc. | $15.52B | Large cap | +60.05% |
| CORT | Corcept Therapeutics Incorporated | $12.95B | Large cap | +71.81% |
| 0JAV.L | Insmed Incorporated | $12.89B | Large cap | −7.52% |
| BMRN | BioMarin Pharmaceutical Inc. | $12.70B | Large cap | +11.85% |
| REC.MI | Recordati Industria Chimica e Farmaceutica S.p.A. | $12.57B | Large cap | +1.80% |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $12.52B | Large cap | +308.99% |
| CYTK | Cytokinetics, Incorporated | $11.01B | Large cap | +109.36% |
| IONS | Ionis Pharmaceuticals, Inc. | $10.56B | Large cap | +48.21% |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +197.09% |
| 0JDI.L | Ionis Pharmaceuticals, Inc. | $10.40B | Large cap | +47.26% |
| ABVX.PA | ABIVAX Société Anonyme | $10.21B | Large cap | +48.91% |
| SEPN | Septerna, Inc. | $1.88B | Small cap | +218.81% |
| NEU.AX | Neuren Pharmaceuticals Limited | $1.88B | Small cap | +5.54% |
| ANAB | AnaptysBio, Inc. | $1.73B | Small cap | +326.47% |
| ANIP | ANI Pharmaceuticals, Inc. | $1.71B | Small cap | −18.85% |
| 0L35.L | Sarepta Therapeutics, Inc. | $1.69B | Small cap | −0.06% |
| MAZE | Maze Therapeutics, Inc. | $1.69B | Small cap | +113.27% |
| AVLN | Avalyn Pharma Inc. | $1.64B | Small cap | +25.60% |
| PYC.AX | PYC Therapeutics Limited | $1.60B | Small cap | +74.42% |
| XERS | Xeris Biopharma Holdings, Inc. | $1.58B | Small cap | +8.64% |
| TRAX | First Tracks Biotherapeutics, Inc. | $1.58B | Small cap | +132.58% |
| A2RR34.SA | Arrowhead Pharmaceuticals, Inc. | $1.58B | Small cap | +298.89% |
| TYRA | Tyra Biosciences, Inc. | $1.56B | Small cap | +103.57% |
| 4516.T | Nippon Shinyaku Co., Ltd. | $1.52B | Small cap | +19.48% |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $1.40B | Small cap | −13.39% |
| JBIO | Jade Biosciences, Inc. | $1.39B | Small cap | +168.30% |
| MLTX | MoonLake Immunotherapeutics | $1.32B | Small cap | −71.82% |
| 0HB0.L | Agios Pharmaceuticals, Inc. | $1.19B | Small cap | −13.00% |
| SVRA | Savara Inc. | $1.15B | Small cap | +70.52% |
| AVTX | Avalo Therapeutics, Inc. | $1.10B | Small cap | +112.95% |
| 006280.KS | GC Biopharma Corp. | $1.06B | Small cap | −2.60% |
| WVE | Wave Life Sciences, Inc. | $1.03B | Small cap | −48.12% |
| DSGN | Design Therapeutics, Inc. | $975.65M | Small cap | +194.71% |
| ANNX | Annexon, Inc. | $946.00M | Small cap | +136.49% |
| DBVT | DBV Technologies S.A. | $940.80M | Small cap | +53.00% |
| CLYM | Climb Bio Inc | $923.57M | Small cap | +603.72% |
| DBV.PA | DBV Technologies S.A. | $901.25M | Small cap | +60.13% |
| PHAR | Pharming Group N.V. | $845.80M | Small cap | −14.47% |
| SLN | Silence Therapeutics plc | $840.94M | Small cap | +160.19% |
| GNFT.PA | Genfit S.A. | $830.56M | Small cap | +310.45% |
| STTK | Shattuck Labs, Inc. | $792.87M | Small cap | +690.00% |
| IRWD | Ironwood Pharmaceuticals, Inc. | $708.88M | Small cap | +240.48% |
| GYRE | Gyre Therapeutics, Inc. | $705.12M | Small cap | −18.00% |
| AGMB | AgomAb Therapeutics NV | $703.26M | Small cap | −2.53% |
| TECX | Tectonic Therapeutic, Inc. | $703.19M | Small cap | +35.43% |
| ZVRA | Zevra Therapeutics, Inc. | $697.17M | Small cap | +25.43% |
| CRMD | CorMedix Inc. | $653.95M | Small cap | −40.66% |
| VALN | Valneva SE | $634.84M | Small cap | −23.49% |
| AYJ.DE | Valneva SE | $619.77M | Small cap | −21.89% |
| ZURA | Zura Bio Limited | $574.02M | Small cap | +177.31% |
| SPRY | ARS Pharmaceuticals, Inc. | $568.84M | Small cap | −55.13% |
| 005257.KS | GC Corp. | $547.34M | Small cap | −40.16% |
| EPRX | Eupraxia Pharmaceuticals Inc. | $490.26M | Small cap | +40.72% |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | $487.17M | Small cap | +43.78% |
| RZLT | Rezolute, Inc. | $485.31M | Small cap | −30.00% |
| KMDA | Kamada Ltd. Ordinary Shares | $483.48M | Small cap | +21.09% |
| QTTB | Q32 Bio Inc. | $481.67M | Small cap | +840.70% |
| AVIR | Atea Pharmaceuticals, Inc. | $441.93M | Small cap | +53.06% |
| LRMR | Larimar Therapeutics, Inc. | $432.15M | Small cap | +6.92% |
| XFOR | X4 Pharmaceuticals, Inc. | $412.45M | Small cap | −1.42% |
| ENTA | Enanta Pharmaceuticals, Inc. | $412.12M | Small cap | +65.89% |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | $406.58M | Small cap | −9.63% |
| UPB | Upstream Bio, Inc. | $390.52M | Small cap | −57.31% |
| IFRX | InflaRx N.V. | $356.63M | Small cap | +142.00% |
| IKT | Inhibikase Therapeutics, Inc. | $347.44M | Small cap | +39.11% |
| CUV.AX | Clinuvel Pharmaceuticals Limited | $338.48M | Small cap | −24.94% |
| PRQR | ProQR Therapeutics N.V. | $331.63M | Small cap | +6.33% |
| TRDA | Entrada Therapeutics, Inc. | $296.70M | Small cap | +23.24% |
| FULC | Fulcrum Therapeutics, Inc. | $294.51M | Small cap | −41.78% |
| TTRX | Turn Therapeutics Inc. | $292.44M | Small cap | +37.57% |
| ELDN | Eledon Pharmaceuticals, Inc. | $272.22M | Small cap | +29.84% |
| IVVD | Invivyd, Inc. | $265.78M | Small cap | +3.52% |
| CAMP | CAMP4 THERAPEUTICS CORPORATION | $263.56M | Small cap | +178.15% |
| SLGL | Sol-Gel Technologies Ltd. | $260.05M | Small cap | +261.87% |
| 0QN1.L | Santhera Pharmaceuticals Holding AG | $254.58M | Small cap | +11.86% |
| AKBA | Akebia Therapeutics, Inc. | $251.20M | Small cap | −69.88% |
| 0T6G.L | Q32 Bio Inc. | $246.69M | Small cap | — |
| PEPG | PepGen Inc. | $237.21M | Small cap | +159.68% |
| RNA | Prosensa Holding B.V. Ordinary Shares | $236.91M | Small cap | −6.10% |
| SIGA | SIGA Technologies, Inc. | $220.58M | Small cap | −57.93% |
| KROS | Keros Therapeutics, Inc. | $220.48M | Small cap | −28.35% |
| ANTX | AN2 Therapeutics, Inc. | $208.68M | Small cap | +385.09% |
| ARMP | Armata Pharmaceuticals, Inc. | $202.64M | Small cap | +123.36% |
| MSCL.TO | Satellos Bioscience Inc. | $199.95M | Small cap | +81.67% |
| PLX | Protalix BioTherapeutics, Inc. | $199.82M | Small cap | +55.00% |
| MSLE | Satellos Bioscience Inc. | $198.59M | Small cap | +77.27% |
| 0AB6.L | Hyloris Pharmaceuticals SA | $185.32M | Small cap | −33.23% |
| EQ | Equillium, Inc. | $149.64M | Small cap | +23.56% |
| AARD | Aardvark Therapeutics, Inc. | $149.04M | Small cap | −24.25% |
| UNCY | Unicycive Therapeutics, Inc. | $148.74M | Small cap | +27.17% |
| CRNO-B.ST | Cereno Scientific AB (publ) | $148.23M | Small cap | −53.59% |
| MIST | Milestone Pharmaceuticals Inc. | $146.91M | Small cap | −33.33% |
| 4599.T | StemRIM | $140.21M | Small cap | +14.75% |
| INO | Inovio Pharmaceuticals, Inc. | $134.42M | Small cap | −47.15% |
| DXB.AX | Dimerix Limited | $122.97M | Small cap | −34.83% |
| 0H8T.L | Aclaris Therapeutics, Inc. | $122.82M | Small cap | +237.38% |
| RFL | Rafael Holdings, Inc. | $119.08M | Small cap | +52.67% |
| MDP.TO | Medexus Pharmaceuticals Inc. | $117.29M | Small cap | +66.56% |
| PYPD | PolyPid Ltd. | $103.79M | Small cap | +49.42% |
| PTHS | Pelthos Therapeutics Inc. | $102.53M | Small cap | +12.90% |
| ABNX.PA | ABIONYX Pharma SA | $98.59M | Micro cap | −31.34% |
| 0RAG.L | ABIONYX Pharma SA | $95.77M | Micro cap | −29.10% |
| ILA.AX | Island Pharmaceuticals Limited | $95.70M | Micro cap | +109.30% |
| FBIO | Fortress Biotech, Inc. | $94.38M | Micro cap | +20.09% |
| PLYX | Polaryx Therapeutics, Inc. | $94.21M | Micro cap | −94.03% |
| PAR.AX | Paradigm Biopharmaceuticals Limited | $92.46M | Micro cap | −24.56% |
| RLYB | Rallybio Corporation | $89.64M | Micro cap | +257.63% |
| GOSS | Gossamer Bio, Inc. | $85.01M | Micro cap | −92.93% |
| RCE.AX | Recce Pharmaceuticals Ltd | $82.40M | Micro cap | −10.71% |
| TELO | Telomir Pharmaceuticals, Inc. | $80.47M | Micro cap | −20.95% |
| STX.L | Shield Therapeutics plc | $71.97M | Micro cap | −37.42% |
| FBIOP | Fortress Biotech, Inc. | $71.45M | Micro cap | +131.74% |
| ALTHX.PA | THX Pharma Société Anonyme | $71.22M | Micro cap | +73.96% |
| SPRO | Spero Therapeutics, Inc. | $71.05M | Micro cap | −38.38% |
| RNTX | Rein Therapeutics Inc. | $69.01M | Micro cap | −27.27% |
| IMA | ImageneBio, Inc. | $61.64M | Micro cap | −60.19% |
| SCYX | SCYNEXIS, Inc. | $56.41M | Micro cap | +579.04% |
| ATYR | aTyr Pharma, Inc. | $54.05M | Micro cap | −89.34% |
| NSRX | Nasus Pharma Ltd. | $48.01M | Micro cap | −49.75% |
| MCRB | Seres Therapeutics, Inc. | $47.81M | Micro cap | −74.79% |
| EDSA | Edesa Biotech, Inc. | $47.63M | Micro cap | +105.83% |
| GRCE | Grace Therapeutics, Inc. | $46.28M | Micro cap | −29.97% |
| MREO | Mereo BioPharma Group plc | $44.39M | Micro cap | −84.20% |
| BOT.AX | Botanix Pharmaceuticals Limited | $41.30M | Micro cap | −86.67% |
| NNVC | NanoViricides, Inc. | $32.68M | Micro cap | −9.86% |
| QNCX | Quince Therapeutics, Inc. | $31.84M | Micro cap | +100.71% |
| IMM.L | ImmuPharma plc | $31.75M | Micro cap | +89.66% |
| 520A.T | J-PHARMACEUTICALS CO LTD | $31.71M | Micro cap | −60.00% |
| ALDVI.PA | Advicenne S.A. | $30.93M | Micro cap | +39.11% |
| 4591.T | Ribomic Inc. | $30.52M | Micro cap | −17.31% |
| BRNS | Barinthus Biotherapeutics plc | $28.86M | Micro cap | −38.04% |
| 0RQJ.L | Saniona AB (publ) | $27.98M | Micro cap | −12.20% |
| RVX.TO | Resverlogix Corp. | $24.81M | Micro cap | 0.00% |
| JSPR | Jasper Therapeutics, Inc. | $23.96M | Micro cap | −75.24% |
| TVRD | Tvardi Therapeutics, Inc. | $23.74M | Micro cap | −94.18% |
| 4884.T | Kringle Pharma, Inc. | $22.30M | Micro cap | −11.29% |
| COCP | Cocrystal Pharma, Inc. | $21.57M | Micro cap | −27.74% |
| KTTA | Pasithea Therapeutics Corp. | $19.05M | Micro cap | −24.30% |
| CALC | CalciMedica, Inc. | $18.06M | Micro cap | −79.74% |
| 4881.T | FunPep Company Limited | $15.92M | Micro cap | −55.65% |
| HIND | Vyome Holdings, Inc. | $15.16M | Micro cap | −79.68% |
| ALBPS.PA | Biophytis S.A. | $11.98M | Micro cap | −50.31% |
| AZTR | Azitra, Inc. | $10.48M | Micro cap | −83.21% |
| QNRX | Quoin Pharmaceuticals, Ltd. | $10.40M | Micro cap | −29.81% |
| ANNX.ST | Annexin Pharmaceuticals AB (publ) | $10.27M | Micro cap | −46.03% |
| PHR.WA | Pharmena S.A. | $9.45M | Micro cap | −8.72% |
| IMRN | Immuron Limited | $9.20M | Micro cap | −35.77% |
| IMC.AX | Immuron Limited | $8.58M | Micro cap | −46.38% |
| SNGX | Soligenix, Inc. | $8.36M | Micro cap | −87.20% |
| XCUR | Exicure, Inc. | $8.25M | Micro cap | −77.93% |
| INM | InMed Pharmaceuticals Inc. | $7.64M | Micro cap | −36.44% |
| VBS.AX | Vectus Biosystems Limited | $6.65M | Micro cap | +104.82% |
| PULM | Pulmatrix, Inc. | $5.57M | Micro cap | −68.62% |
| BIOSGN.ST | Biosergen AB (publ) | $5.31M | Micro cap | −94.62% |
| JAGX | Jaguar Health, Inc. | $4.78M | Micro cap | −98.52% |
| GRI | GRI Bio, Inc. | $4.28M | Micro cap | −95.23% |
| XRTX | XORTX Therapeutics Inc. | $4.03M | Micro cap | −43.99% |
| BIT.AX | Biotron Limited | $3.99M | Micro cap | −50.00% |
| SXTP | 60 Degrees Pharmaceuticals, Inc. | $3.80M | Micro cap | −78.23% |
| MTNB | Matinas BioPharma Holdings, Inc. | $2.89M | Micro cap | −90.08% |
| APTA.ST | Aptahem AB (publ) | $1.50M | Micro cap | −67.65% |
| APLI.TO | Appili Therapeutics Inc. | $1.44M | Micro cap | −25.00% |
| VBIO | Valion Bio, Inc. | $647.60K | Micro cap | −94.92% |
| ALNEV.PA | Neovacs S.A. | $22.00 | Micro cap | −11.29% |
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.