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Rare disease stocks
We cover 102 companies grouped under the Rare disease theme. The median market value in the group is $1.00B. Across the group, the median move over the past trading day is −0.69%. 65% of the 98 with a 200-day average trade above it. Price change over the past year runs from −33.3% to +194.2% across the middle 80 percent of the group, with a median of +45.4%.
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Table
102 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return | Next report |
|---|---|---|---|---|---|
| PTGX | Protagonist Therapeutics, Inc. | $9.80B | Mid cap | +146.62% | Nov 5, 2026 |
| IMVT | Immunovant, Inc. | $9.07B | Mid cap | +189.13% | Nov 9, 2026 |
| 4151.T | Kyowa Kirin Co., Ltd. | $8.51B | Mid cap | +4.01% | Nov 2, 2026 |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +9.35% | Nov 3, 2026 |
| SRRK | Scholar Rock Holding Corporation | $7.31B | Mid cap | +74.18% | Nov 12, 2026 |
| ORKA | Oruka Therapeutics, Inc. | $6.58B | Mid cap | +555.07% | Nov 11, 2026 |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +154.86% | Nov 3, 2026 |
| TVTX | Travere Therapeutics, Inc. | $6.24B | Mid cap | +284.86% | Oct 29, 2026 |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +356.93% | Nov 5, 2026 |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.07B | Mid cap | +46.02% | Nov 5, 2026 |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.05B | Mid cap | +133.47% | Oct 27, 2026 |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +31.96% | Nov 4, 2026 |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +93.26% | Nov 5, 2026 |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +207.13% | Nov 5, 2026 |
| MANE | Veradermics, Incorporated | $4.29B | Mid cap | +172.32% | Nov 12, 2026 |
| CAMX.ST | Camurus AB (publ) | $3.79B | Mid cap | −15.35% | Nov 5, 2026 |
| CLDX | Celldex Therapeutics, Inc. | $3.19B | Mid cap | +79.51% | Nov 9, 2026 |
| IRON | Disc Medicine, Inc. | $3.17B | Mid cap | +37.90% | Nov 5, 2026 |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +60.64% | Oct 27, 2026 |
| VRDN | Viridian Therapeutics, Inc. | $2.75B | Mid cap | +30.71% | Nov 5, 2026 |
| VERA | Vera Therapeutics, Inc. | $2.65B | Mid cap | +67.21% | Nov 5, 2026 |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −12.10% | Nov 3, 2026 |
| PHVS | Pharvaris N.V. | $2.60B | Mid cap | +55.59% | Nov 12, 2026 |
| TRVI | Trevi Therapeutics, Inc. | $2.55B | Mid cap | +124.00% | Nov 12, 2026 |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +151.95% | Nov 12, 2026 |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +15.38% | Nov 2, 2026 |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.30B | Mid cap | +44.68% | Nov 16, 2026 |
| MLYS | Mineralys Therapeutics, Inc. | $2.28B | Mid cap | +70.22% | Nov 10, 2026 |
| PVLA | Palvella Therapeutics, Inc. | $2.22B | Mid cap | +173.39% | Nov 11, 2026 |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.20B | Mid cap | +36.58% | Nov 9, 2026 |
| ADMA | ADMA Biologics, Inc. | $2.16B | Mid cap | −44.17% | Nov 4, 2026 |
| ZBIO | Zenas BioPharma, Inc. | $2.15B | Mid cap | +97.73% | Nov 12, 2026 |
| STOK | Stoke Therapeutics, Inc. | $2.14B | Mid cap | +71.45% | Nov 3, 2026 |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $2.03B | Mid cap | −13.45% | Oct 29, 2026 |
| SEPN | Septerna, Inc. | $1.88B | Small cap | +218.81% | Nov 9, 2026 |
| ANIP | ANI Pharmaceuticals, Inc. | $1.71B | Small cap | −18.85% | Nov 6, 2026 |
| 0L35.L | Sarepta Therapeutics, Inc. | $1.69B | Small cap | −0.06% | Nov 4, 2026 |
| MAZE | Maze Therapeutics, Inc. | $1.69B | Small cap | +113.27% | Nov 5, 2026 |
| AVLN | Avalyn Pharma Inc. | $1.64B | Small cap | +25.60% | Nov 16, 2026 |
| XERS | Xeris Biopharma Holdings, Inc. | $1.58B | Small cap | +8.64% | Nov 5, 2026 |
| TRAX | First Tracks Biotherapeutics, Inc. | $1.58B | Small cap | +132.58% | Nov 5, 2026 |
| TYRA | Tyra Biosciences, Inc. | $1.56B | Small cap | +103.57% | Nov 4, 2026 |
| 4516.T | Nippon Shinyaku Co., Ltd. | $1.52B | Small cap | +19.48% | Nov 13, 2026 |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $1.40B | Small cap | −13.39% | Nov 3, 2026 |
| JBIO | Jade Biosciences, Inc. | $1.39B | Small cap | +168.30% | Nov 12, 2026 |
| MLTX | MoonLake Immunotherapeutics | $1.32B | Small cap | −71.82% | Nov 5, 2026 |
| 0HB0.L | Agios Pharmaceuticals, Inc. | $1.19B | Small cap | −13.00% | Oct 29, 2026 |
| SVRA | Savara Inc. | $1.15B | Small cap | +70.52% | Nov 11, 2026 |
| AVTX | Avalo Therapeutics, Inc. | $1.10B | Small cap | +112.95% | Nov 12, 2026 |
| 006280.KS | GC Biopharma Corp. | $1.06B | Small cap | −2.60% | Nov 2, 2026 |
| WVE | Wave Life Sciences, Inc. | $1.03B | Small cap | −48.12% | Nov 9, 2026 |
| DSGN | Design Therapeutics, Inc. | $975.65M | Small cap | +194.71% | Nov 5, 2026 |
| ANNX | Annexon, Inc. | $946.00M | Small cap | +136.49% | Nov 9, 2026 |
| DBVT | DBV Technologies S.A. | $940.80M | Small cap | +53.00% | Oct 28, 2026 |
| DBV.PA | DBV Technologies S.A. | $901.25M | Small cap | +60.13% | Oct 28, 2026 |
| SLN | Silence Therapeutics plc | $840.94M | Small cap | +160.19% | Nov 5, 2026 |
| GNFT.PA | Genfit S.A. | $830.56M | Small cap | +310.45% | Sep 29, 2026 |
| STTK | Shattuck Labs, Inc. | $792.87M | Small cap | +690.00% | Nov 12, 2026 |
| IRWD | Ironwood Pharmaceuticals, Inc. | $708.88M | Small cap | +240.48% | Nov 9, 2026 |
| GYRE | Gyre Therapeutics, Inc. | $705.12M | Small cap | −18.00% | Nov 5, 2026 |
| TECX | Tectonic Therapeutic, Inc. | $703.19M | Small cap | +35.43% | Nov 5, 2026 |
| ZVRA | Zevra Therapeutics, Inc. | $697.17M | Small cap | +25.43% | Nov 4, 2026 |
| CRMD | CorMedix Inc. | $653.95M | Small cap | −40.66% | Nov 16, 2026 |
| ZURA | Zura Bio Limited | $574.02M | Small cap | +177.31% | Nov 12, 2026 |
| SPRY | ARS Pharmaceuticals, Inc. | $568.84M | Small cap | −55.13% | Nov 10, 2026 |
| EPRX | Eupraxia Pharmaceuticals Inc. | $490.26M | Small cap | +40.72% | Nov 3, 2026 |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | $487.17M | Small cap | +43.78% | Nov 3, 2026 |
| RZLT | Rezolute, Inc. | $485.31M | Small cap | −30.00% | Sep 16, 2026 |
| KMDA | Kamada Ltd. Ordinary Shares | $483.48M | Small cap | +21.09% | Nov 9, 2026 |
| AVIR | Atea Pharmaceuticals, Inc. | $441.93M | Small cap | +53.06% | Nov 11, 2026 |
| LRMR | Larimar Therapeutics, Inc. | $432.15M | Small cap | +6.92% | Nov 5, 2026 |
| XFOR | X4 Pharmaceuticals, Inc. | $412.45M | Small cap | −1.42% | Nov 5, 2026 |
| ENTA | Enanta Pharmaceuticals, Inc. | $412.12M | Small cap | +65.89% | Nov 16, 2026 |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | $406.58M | Small cap | −9.63% | Oct 29, 2026 |
| UPB | Upstream Bio, Inc. | $390.52M | Small cap | −57.31% | Nov 4, 2026 |
| IFRX | InflaRx N.V. | $356.63M | Small cap | +142.00% | Nov 9, 2026 |
| IKT | Inhibikase Therapeutics, Inc. | $347.44M | Small cap | +39.11% | Nov 13, 2026 |
| CUV.AX | Clinuvel Pharmaceuticals Limited | $338.48M | Small cap | −24.94% | Aug 27, 2026 |
| PRQR | ProQR Therapeutics N.V. | $331.63M | Small cap | +6.33% | Nov 5, 2026 |
| TRDA | Entrada Therapeutics, Inc. | $296.70M | Small cap | +23.24% | Nov 5, 2026 |
| FULC | Fulcrum Therapeutics, Inc. | $294.51M | Small cap | −41.78% | Oct 29, 2026 |
| TTRX | Turn Therapeutics Inc. | $292.44M | Small cap | +37.57% | Nov 12, 2026 |
| ELDN | Eledon Pharmaceuticals, Inc. | $272.22M | Small cap | +29.84% | Nov 13, 2026 |
| IVVD | Invivyd, Inc. | $265.78M | Small cap | +3.52% | Nov 5, 2026 |
| SLGL | Sol-Gel Technologies Ltd. | $260.05M | Small cap | +261.87% | Nov 19, 2026 |
| AKBA | Akebia Therapeutics, Inc. | $251.20M | Small cap | −69.88% | Nov 9, 2026 |
| PEPG | PepGen Inc. | $237.21M | Small cap | +159.68% | Nov 11, 2026 |
| SIGA | SIGA Technologies, Inc. | $220.58M | Small cap | −57.93% | Nov 5, 2026 |
| KROS | Keros Therapeutics, Inc. | $220.48M | Small cap | −28.35% | Nov 4, 2026 |
| ARMP | Armata Pharmaceuticals, Inc. | $202.64M | Small cap | +123.36% | Nov 11, 2026 |
| MSCL.TO | Satellos Bioscience Inc. | $199.95M | Small cap | +81.67% | Nov 12, 2026 |
| PLX | Protalix BioTherapeutics, Inc. | $199.82M | Small cap | +55.00% | Nov 12, 2026 |
| MSLE | Satellos Bioscience Inc. | $198.59M | Small cap | +77.27% | Nov 12, 2026 |
| 0AB6.L | Hyloris Pharmaceuticals SA | $185.32M | Small cap | −33.23% | Sep 24, 2026 |
| AARD | Aardvark Therapeutics, Inc. | $149.04M | Small cap | −24.25% | Nov 12, 2026 |
| MIST | Milestone Pharmaceuticals Inc. | $146.91M | Small cap | −33.33% | Nov 12, 2026 |
| INO | Inovio Pharmaceuticals, Inc. | $134.42M | Small cap | −47.15% | Nov 10, 2026 |
| 0H8T.L | Aclaris Therapeutics, Inc. | $122.82M | Small cap | +237.38% | Nov 5, 2026 |
| RFL | Rafael Holdings, Inc. | $119.08M | Small cap | +52.67% | Oct 28, 2026 |
| MDP.TO | Medexus Pharmaceuticals Inc. | $117.29M | Small cap | +66.56% | Nov 11, 2026 |
| PYPD | PolyPid Ltd. | $103.79M | Small cap | +49.42% | Nov 11, 2026 |
| PTHS | Pelthos Therapeutics Inc. | $102.53M | Small cap | +12.90% | Nov 12, 2026 |
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.