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Rare disease stocks
We cover 98 companies grouped under the Rare disease theme. The median market value in the group is $484.80M. Across the group, the median move over the past trading day is +1.67%. 59% of the 94 with a 200-day average trade above it. Price change over the past year runs from −58.5% to +219.4% across the middle 80 percent of the group, with a median of +37.2%.
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Table
98 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return | Next report |
|---|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | $140.77B | Large cap | +40.14% | Nov 2, 2026 |
| VRTX | Vertex Pharmaceuticals Incorporated | $140.04B | Large cap | +42.14% | Nov 2, 2026 |
| 0QZU.L | Vertex Pharmaceuticals Incorporated | $112.52B | Large cap | +42.39% | Nov 2, 2026 |
| ARGX | argenx SE | $63.78B | Large cap | +50.52% | Oct 22, 2026 |
| 0HD2.L | Alnylam Pharmaceuticals, Inc. | $35.05B | Large cap | −46.85% | Oct 29, 2026 |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −45.88% | Oct 29, 2026 |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | $31.87B | Large cap | −11.75% | Oct 29, 2026 |
| INSM | Insmed Incorporated | $27.07B | Large cap | −3.45% | Oct 29, 2026 |
| BBIO | BridgeBio Pharma, Inc. | $15.85B | Large cap | +70.01% | Oct 28, 2026 |
| 0JAV.L | Insmed Incorporated | $12.89B | Large cap | −6.25% | Oct 29, 2026 |
| BMRN | BioMarin Pharmaceutical Inc. | $12.80B | Large cap | +13.03% | Oct 26, 2026 |
| CYTK | Cytokinetics, Incorporated | $10.77B | Large cap | +109.29% | Nov 5, 2026 |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +202.82% | Nov 3, 2026 |
| ABVX.PA | ABIVAX Société Anonyme | $10.42B | Large cap | +42.59% | Sep 21, 2026 |
| PTGX | Protagonist Therapeutics, Inc. | $10.01B | Large cap | +164.75% | Nov 5, 2026 |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $1.95B | Small cap | −9.02% | Oct 29, 2026 |
| SEPN | Septerna, Inc. | $1.93B | Small cap | +245.32% | Nov 9, 2026 |
| ETON | Eton Pharmaceuticals, Inc. | $1.80B | Small cap | +268.93% | Nov 12, 2026 |
| ANIP | ANI Pharmaceuticals, Inc. | $1.74B | Small cap | −16.24% | Nov 6, 2026 |
| AVLN | Avalyn Pharma Inc. | $1.69B | Small cap | +29.54% | Nov 16, 2026 |
| 0L35.L | Sarepta Therapeutics, Inc. | $1.69B | Small cap | +4.63% | Nov 4, 2026 |
| MAZE | Maze Therapeutics, Inc. | $1.69B | Small cap | +128.55% | Nov 5, 2026 |
| FBRX | Forte Biosciences, Inc. | $1.63B | Small cap | +580.25% | Nov 9, 2026 |
| TRAX | First Tracks Biotherapeutics, Inc. | $1.61B | Small cap | +137.42% | Nov 5, 2026 |
| XERS | Xeris Biopharma Holdings, Inc. | $1.59B | Small cap | +12.48% | Nov 5, 2026 |
| TYRA | Tyra Biosciences, Inc. | $1.59B | Small cap | +122.42% | Nov 4, 2026 |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $1.40B | Small cap | −11.63% | Nov 3, 2026 |
| OMER | Omeros Corporation | $1.40B | Small cap | +343.22% | Nov 12, 2026 |
| JBIO | Jade Biosciences, Inc. | $1.39B | Small cap | +176.46% | Nov 12, 2026 |
| MLTX | MoonLake Immunotherapeutics | $1.32B | Small cap | −71.52% | Nov 5, 2026 |
| 0HB0.L | Agios Pharmaceuticals, Inc. | $1.19B | Small cap | −14.25% | Oct 29, 2026 |
| SVRA | Savara Inc. | $1.15B | Small cap | +78.34% | Nov 11, 2026 |
| ANNX | Annexon, Inc. | $976.33M | Small cap | +139.53% | Nov 9, 2026 |
| DBVT | DBV Technologies S.A. | $940.80M | Small cap | +52.91% | Oct 28, 2026 |
| DBV.PA | DBV Technologies S.A. | $928.30M | Small cap | +54.11% | Oct 28, 2026 |
| SLN | Silence Therapeutics plc | $849.65M | Small cap | +170.16% | Nov 5, 2026 |
| GNFT.PA | Genfit S.A. | $836.50M | Small cap | +293.99% | Sep 29, 2026 |
| STTK | Shattuck Labs, Inc. | $792.87M | Small cap | +710.87% | Nov 12, 2026 |
| TECX | Tectonic Therapeutic, Inc. | $737.21M | Small cap | +53.48% | Nov 5, 2026 |
| ZVRA | Zevra Therapeutics, Inc. | $714.98M | Small cap | +28.63% | Nov 4, 2026 |
| GYRE | Gyre Therapeutics, Inc. | $682.86M | Small cap | −18.54% | Nov 5, 2026 |
| CRMD | CorMedix Inc. | $667.20M | Small cap | −37.65% | Nov 16, 2026 |
| ZURA | Zura Bio Limited | $605.64M | Small cap | +208.29% | Nov 12, 2026 |
| AQST | Aquestive Therapeutics, Inc. | $602.46M | Small cap | +31.61% | Nov 4, 2026 |
| 0KBU.L | Omeros Corporation | $575.28M | Small cap | +341.05% | Nov 12, 2026 |
| SPRY | ARS Pharmaceuticals, Inc. | $568.84M | Small cap | −57.66% | Nov 10, 2026 |
| KMDA | Kamada Ltd. Ordinary Shares | $497.90M | Small cap | +26.12% | Nov 9, 2026 |
| EPRX | Eupraxia Pharmaceuticals Inc. | $490.26M | Small cap | +41.87% | Nov 3, 2026 |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | $487.17M | Small cap | +41.67% | Nov 3, 2026 |
| RZLT | Rezolute, Inc. | $482.42M | Small cap | −28.43% | Sep 16, 2026 |
| AVIR | Atea Pharmaceuticals, Inc. | $441.93M | Small cap | +48.36% | Nov 11, 2026 |
| LRMR | Larimar Therapeutics, Inc. | $432.15M | Small cap | +11.83% | Nov 5, 2026 |
| XFOR | X4 Pharmaceuticals, Inc. | $412.45M | Small cap | +4.79% | Nov 5, 2026 |
| ENTA | Enanta Pharmaceuticals, Inc. | $410.67M | Small cap | +62.59% | Nov 16, 2026 |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | $397.97M | Small cap | −7.40% | Oct 29, 2026 |
| UPB | Upstream Bio, Inc. | $393.26M | Small cap | −57.08% | Nov 4, 2026 |
| CUV.AX | Clinuvel Pharmaceuticals Limited | $381.21M | Small cap | −21.84% | Aug 27, 2026 |
| IKT | Inhibikase Therapeutics, Inc. | $348.84M | Small cap | +42.05% | Nov 13, 2026 |
| PRQR | ProQR Therapeutics N.V. | $335.86M | Small cap | +5.78% | Nov 5, 2026 |
| TRDA | Entrada Therapeutics, Inc. | $296.70M | Small cap | +35.35% | Nov 5, 2026 |
| TTRX | Turn Therapeutics Inc. | $295.48M | Small cap | +39.00% | Nov 12, 2026 |
| FULC | Fulcrum Therapeutics, Inc. | $294.51M | Small cap | −39.78% | Oct 29, 2026 |
| SION | Sionna Therapeutics, Inc. | $282.58M | Small cap | −74.02% | Nov 5, 2026 |
| ELDN | Eledon Pharmaceuticals, Inc. | $281.98M | Small cap | +35.02% | Nov 13, 2026 |
| IVVD | Invivyd, Inc. | $277.81M | Small cap | +67.62% | Nov 5, 2026 |
| SLGL | Sol-Gel Technologies Ltd. | $265.20M | Small cap | +289.14% | Nov 19, 2026 |
| XUP.DE | Genfit S.A. | $258.22M | Small cap | +166.82% | Sep 29, 2026 |
| CRDL | Cardiol Therapeutics Inc. | $233.65M | Small cap | +75.44% | Nov 12, 2026 |
| CRDL.TO | Cardiol Therapeutics Inc. | $233.07M | Small cap | +72.50% | Nov 12, 2026 |
| SIGA | SIGA Technologies, Inc. | $211.24M | Small cap | −60.50% | Nov 5, 2026 |
| ARMP | Armata Pharmaceuticals, Inc. | $206.73M | Small cap | +113.85% | Nov 11, 2026 |
| MSLE | Satellos Bioscience Inc. | $200.92M | Small cap | +82.17% | Nov 12, 2026 |
| MSCL.TO | Satellos Bioscience Inc. | $199.37M | Small cap | +80.56% | Nov 12, 2026 |
| PLX | Protalix BioTherapeutics, Inc. | $193.37M | Small cap | +50.62% | Nov 12, 2026 |
| 0AB6.L | Hyloris Pharmaceuticals SA | $185.32M | Small cap | −32.01% | Sep 24, 2026 |
| AARD | Aardvark Therapeutics, Inc. | $158.67M | Small cap | −19.80% | Nov 12, 2026 |
| MIST | Milestone Pharmaceuticals Inc. | $144.42M | Small cap | −31.36% | Nov 12, 2026 |
| CPIX | Cumberland Pharmaceuticals Inc. | $130.35M | Small cap | +166.87% | Nov 3, 2026 |
| INO | Inovio Pharmaceuticals, Inc. | $129.25M | Small cap | −33.85% | Nov 10, 2026 |
| 0H8T.L | Aclaris Therapeutics, Inc. | $122.82M | Small cap | +265.21% | Nov 5, 2026 |
| MDP.TO | Medexus Pharmaceuticals Inc. | $116.99M | Small cap | +80.70% | Nov 11, 2026 |
| RFL | Rafael Holdings, Inc. | $113.88M | Small cap | +52.38% | Oct 28, 2026 |
| PTHS | Pelthos Therapeutics Inc. | $104.13M | Small cap | +20.41% | Nov 12, 2026 |
| PYPD | PolyPid Ltd. | $101.25M | Small cap | +47.83% | Nov 11, 2026 |
| GOSS | Gossamer Bio, Inc. | $99.97M | Micro cap | −91.33% | Nov 4, 2026 |
| SPRO | Spero Therapeutics, Inc. | $70.76M | Micro cap | −33.51% | Nov 12, 2026 |
| ATYR | aTyr Pharma, Inc. | $55.91M | Micro cap | −88.95% | Nov 5, 2026 |
| SCYX | SCYNEXIS, Inc. | $51.64M | Micro cap | +514.20% | Nov 4, 2026 |
| MCRB | Seres Therapeutics, Inc. | $46.81M | Micro cap | −74.00% | Nov 4, 2026 |
| MREO | Mereo BioPharma Group plc | $44.97M | Micro cap | −83.82% | Nov 9, 2026 |
| QNCX | Quince Therapeutics, Inc. | $34.05M | Micro cap | +95.76% | Nov 12, 2026 |
| JSPR | Jasper Therapeutics, Inc. | $23.96M | Micro cap | −75.67% | Nov 10, 2026 |
| TVRD | Tvardi Therapeutics, Inc. | $23.74M | Micro cap | −93.77% | Nov 12, 2026 |
| RVX.TO | Resverlogix Corp. | $23.62M | Micro cap | +5.00% | Nov 12, 2026 |
| INM | InMed Pharmaceuticals Inc. | $7.69M | Micro cap | −35.43% | Sep 23, 2026 |
| JAGX | Jaguar Health, Inc. | $4.78M | Micro cap | −98.52% | Nov 12, 2026 |
| XRTX | XORTX Therapeutics Inc. | $4.11M | Micro cap | −44.74% | Nov 12, 2026 |
| APLI.TO | Appili Therapeutics Inc. | $1.44M | Micro cap | −25.00% | 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.