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Rare disease stocks
We cover 149 companies grouped under the Rare disease theme. The median market value in the group is $568.84M. Across the group, the median move over the past trading day is −0.78%. 56% of the 142 with a 200-day average trade above it. Price change over the past year runs from −75.8% to +206.6% across the middle 80 percent of the group, with a median of +29.8%.
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Table
149 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return |
|---|---|---|---|---|
| VRTX | Vertex Pharmaceuticals Incorporated | $138.63B | Large cap | +40.66% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −47.38% |
| 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% |
| BMRN | BioMarin Pharmaceutical Inc. | $12.70B | Large cap | +11.85% |
| 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% |
| PTGX | Protagonist Therapeutics, Inc. | $9.80B | Mid cap | +146.62% |
| IMVT | Immunovant, Inc. | $9.07B | Mid cap | +189.13% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +9.35% |
| SRRK | Scholar Rock Holding Corporation | $7.31B | Mid cap | +74.18% |
| ORKA | Oruka Therapeutics, Inc. | $6.58B | Mid cap | +555.07% |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +154.86% |
| TVTX | Travere Therapeutics, Inc. | $6.24B | Mid cap | +284.86% |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +356.93% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.07B | Mid cap | +46.02% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.05B | Mid cap | +133.47% |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +31.96% |
| GRFS | Grifols, S.A. | $5.38B | Mid cap | −19.39% |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +93.26% |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +207.13% |
| MANE | Veradermics, Incorporated | $4.29B | Mid cap | +172.32% |
| CLDX | Celldex Therapeutics, Inc. | $3.19B | Mid cap | +79.51% |
| IRON | Disc Medicine, Inc. | $3.17B | Mid cap | +37.90% |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +60.64% |
| VRDN | Viridian Therapeutics, Inc. | $2.75B | Mid cap | +30.71% |
| VERA | Vera Therapeutics, Inc. | $2.65B | Mid cap | +67.21% |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −12.10% |
| PHVS | Pharvaris N.V. | $2.60B | Mid cap | +55.59% |
| TRVI | Trevi Therapeutics, Inc. | $2.55B | Mid cap | +124.00% |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +151.95% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +15.38% |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.30B | Mid cap | +44.68% |
| MLYS | Mineralys Therapeutics, Inc. | $2.28B | Mid cap | +70.22% |
| PVLA | Palvella Therapeutics, Inc. | $2.22B | Mid cap | +173.39% |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.20B | Mid cap | +36.58% |
| ADMA | ADMA Biologics, Inc. | $2.16B | Mid cap | −44.17% |
| ZBIO | Zenas BioPharma, Inc. | $2.15B | Mid cap | +97.73% |
| STOK | Stoke Therapeutics, Inc. | $2.14B | Mid cap | +71.45% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $2.03B | Mid cap | −13.45% |
| SEPN | Septerna, Inc. | $1.88B | Small cap | +218.81% |
| ANAB | AnaptysBio, Inc. | $1.73B | Small cap | +326.47% |
| ANIP | ANI Pharmaceuticals, Inc. | $1.71B | Small cap | −18.85% |
| MAZE | Maze Therapeutics, Inc. | $1.69B | Small cap | +113.27% |
| AVLN | Avalyn Pharma Inc. | $1.64B | Small cap | +25.60% |
| XERS | Xeris Biopharma Holdings, Inc. | $1.58B | Small cap | +8.64% |
| TRAX | First Tracks Biotherapeutics, Inc. | $1.58B | Small cap | +132.58% |
| TYRA | Tyra Biosciences, Inc. | $1.56B | Small cap | +103.57% |
| JBIO | Jade Biosciences, Inc. | $1.39B | Small cap | +168.30% |
| MLTX | MoonLake Immunotherapeutics | $1.32B | Small cap | −71.82% |
| SVRA | Savara Inc. | $1.15B | Small cap | +70.52% |
| AVTX | Avalo Therapeutics, Inc. | $1.10B | Small cap | +112.95% |
| 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% |
| PHAR | Pharming Group N.V. | $845.80M | Small cap | −14.47% |
| SLN | Silence Therapeutics plc | $840.94M | Small cap | +160.19% |
| 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% |
| ZURA | Zura Bio Limited | $574.02M | Small cap | +177.31% |
| SPRY | ARS Pharmaceuticals, Inc. | $568.84M | Small cap | −55.13% |
| EPRX | Eupraxia Pharmaceuticals Inc. | $490.26M | Small cap | +40.72% |
| 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% |
| 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% |
| 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% |
| AKBA | Akebia Therapeutics, Inc. | $251.20M | Small cap | −69.88% |
| 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% |
| PLX | Protalix BioTherapeutics, Inc. | $199.82M | Small cap | +55.00% |
| MSLE | Satellos Bioscience Inc. | $198.59M | Small cap | +77.27% |
| 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% |
| MIST | Milestone Pharmaceuticals Inc. | $146.91M | Small cap | −33.33% |
| INO | Inovio Pharmaceuticals, Inc. | $134.42M | Small cap | −47.15% |
| RFL | Rafael Holdings, Inc. | $119.08M | Small cap | +52.67% |
| PYPD | PolyPid Ltd. | $103.79M | Small cap | +49.42% |
| PTHS | Pelthos Therapeutics Inc. | $102.53M | Small cap | +12.90% |
| FBIO | Fortress Biotech, Inc. | $94.38M | Micro cap | +20.09% |
| PLYX | Polaryx Therapeutics, Inc. | $94.21M | Micro cap | −94.03% |
| RLYB | Rallybio Corporation | $89.64M | Micro cap | +257.63% |
| GOSS | Gossamer Bio, Inc. | $85.01M | Micro cap | −92.93% |
| TELO | Telomir Pharmaceuticals, Inc. | $80.47M | Micro cap | −20.95% |
| FBIOP | Fortress Biotech, Inc. | $71.45M | Micro cap | +131.74% |
| 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% |
| NNVC | NanoViricides, Inc. | $32.68M | Micro cap | −9.86% |
| QNCX | Quince Therapeutics, Inc. | $31.84M | Micro cap | +100.71% |
| BRNS | Barinthus Biotherapeutics plc | $28.86M | Micro cap | −38.04% |
| JSPR | Jasper Therapeutics, Inc. | $23.96M | Micro cap | −75.24% |
| TVRD | Tvardi Therapeutics, Inc. | $23.74M | Micro cap | −94.18% |
| 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% |
| HIND | Vyome Holdings, Inc. | $15.16M | Micro cap | −79.68% |
| AZTR | Azitra, Inc. | $10.48M | Micro cap | −83.21% |
| QNRX | Quoin Pharmaceuticals, Ltd. | $10.40M | Micro cap | −29.81% |
| IMRN | Immuron Limited | $9.20M | Micro cap | −35.77% |
| 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% |
| PULM | Pulmatrix, Inc. | $5.57M | Micro cap | −68.62% |
| 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% |
| SXTP | 60 Degrees Pharmaceuticals, Inc. | $3.80M | Micro cap | −78.23% |
| MTNB | Matinas BioPharma Holdings, Inc. | $2.89M | Micro cap | −90.08% |
| VBIO | Valion Bio, Inc. | $647.60K | Micro cap | −94.92% |
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.