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Rare disease stocks
We cover 201 companies grouped under the Rare disease theme. The median market value in the group is $292.44M. Across the group, the median move over the past trading day is −0.53%. 53% of the 192 with a 200-day average trade above it. Price change over the past year runs from −74.8% to +218.8% across the middle 80 percent of the group, with a median of +20.1%.
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Table
201 of 268 companies match these filters.Clear filters
| Ticker | Company | Price | 1M return | YTD return | 1Y return |
|---|---|---|---|---|---|
| PTGX | Protagonist Therapeutics, Inc. | $151.50 | +10.71% | +73.78% | +146.62% |
| IMVT | Immunovant, Inc. | $43.89 | +12.83% | +69.39% | +189.13% |
| CRNX | Crinetics Pharmaceuticals, Inc. | $84.84 | +1.17% | +81.55% | +179.08% |
| 4151.T | Kyowa Kirin Co., Ltd. | 2,613.00 JPY | −3.33% | +3.94% | +4.01% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $111.87 | +3.54% | +6.53% | +9.35% |
| SRRK | Scholar Rock Holding Corporation | $60.04 | +21.61% | +40.28% | +74.18% |
| ORKA | Oruka Therapeutics, Inc. | $99.44 | +9.94% | +251.75% | +555.07% |
| LQDA | Liquidia Corporation | $71.36 | −17.81% | +127.26% | +154.86% |
| TVTX | Travere Therapeutics, Inc. | $67.12 | +20.05% | +69.84% | +284.86% |
| DNTH | Dianthus Therapeutics, Inc. | $110.76 | +9.01% | +179.13% | +356.93% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $72.69 | −3.58% | −5.28% | +46.02% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $78.68 | −0.96% | +87.31% | +133.47% |
| MIRM | Mirum Pharmaceuticals, Inc. | $98.51 | −12.22% | +26.18% | +31.96% |
| GRFS | Grifols, S.A. | $7.90 | −1.25% | −14.50% | −19.39% |
| DYN | Dyne Therapeutics, Inc. | $26.36 | +1.78% | +42.49% | +93.26% |
| EWTX | Edgewise Therapeutics, Inc. | $44.38 | +9.99% | +86.86% | +207.13% |
| MANE | Veradermics, Incorporated | $102.80 | −7.84% | +172.32% | +172.32% |
| AMLX | Amylyx Pharmaceuticals, Inc. | $37.08 | +89.67% | +224.69% | +292.38% |
| CPRX | Catalyst Pharmaceuticals Inc | $31.49 | 0.00% | +36.03% | +55.97% |
| CAMX.ST | Camurus AB (publ) | 615.00 SEK | −1.68% | +0.49% | −15.35% |
| CLDX | Celldex Therapeutics, Inc. | $40.57 | +14.02% | +50.20% | +79.51% |
| IRON | Disc Medicine, Inc. | $82.67 | +10.74% | +4.53% | +37.90% |
| ARQT | Arcutis Biotherapeutics, Inc. | $25.14 | −11.54% | −13.31% | +60.64% |
| VRDN | Viridian Therapeutics, Inc. | $24.26 | +24.35% | −20.93% | +30.71% |
| VERA | Vera Therapeutics, Inc. | $36.82 | +10.50% | −24.27% | +67.21% |
| RARE | Ultragenyx Pharmaceutical Inc. | $26.44 | +1.77% | +12.03% | −12.10% |
| PHVS | Pharvaris N.V. | $37.00 | +9.97% | +39.73% | +55.59% |
| TRVI | Trevi Therapeutics, Inc. | $17.92 | −2.77% | +50.71% | +124.00% |
| NKTR | Nektar Therapeutics | $73.77 | +4.24% | +69.90% | +151.95% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $9.83 | +0.92% | +31.07% | +15.38% |
| 009420.KS | Hanall Biopharma Co., Ltd. | 64,000.00 KRW | +53.48% | +51.66% | +112.62% |
| COAG | Hemab Therapeutics Holdings, Inc. | $49.19 | +4.08% | +44.68% | +44.68% |
| MLYS | Mineralys Therapeutics, Inc. | $25.89 | −3.93% | −27.50% | +70.22% |
| PVLA | Palvella Therapeutics, Inc. | $154.33 | +8.04% | +53.90% | +173.39% |
| AUPH | Aurinia Pharmaceuticals Inc. | $16.54 | +10.56% | +7.75% | +36.58% |
| ADMA | ADMA Biologics, Inc. | $9.63 | +9.56% | −46.14% | −44.17% |
| ZBIO | Zenas BioPharma, Inc. | $33.04 | +2.32% | −4.23% | +97.73% |
| STOK | Stoke Therapeutics, Inc. | $33.09 | +12.59% | +7.09% | +71.45% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $33.99 | −5.56% | +25.06% | −13.45% |
| SEPN | Septerna, Inc. | $40.25 | +18.49% | +43.85% | +218.81% |
| NEU.AX | Neuren Pharmaceuticals Limited | 20.59 AUD | +20.20% | +8.88% | +5.54% |
| ETON | Eton Pharmaceuticals, Inc. | $62.62 | +34.20% | +285.12% | +262.38% |
| ANAB | AnaptysBio, Inc. | $58.00 | +11.30% | +93.55% | +326.47% |
| ANIP | ANI Pharmaceuticals, Inc. | $74.56 | −8.00% | −4.93% | −18.85% |
| MAZE | Maze Therapeutics, Inc. | $29.58 | +6.10% | −25.62% | +113.27% |
| AVLN | Avalyn Pharma Inc. | $37.04 | +33.29% | +25.60% | +25.60% |
| FBRX | Forte Biosciences, Inc. | $76.99 | +0.44% | +226.09% | +589.87% |
| PYC.AX | PYC Therapeutics Limited | 2.25 AUD | +38.46% | +35.95% | +74.42% |
| XERS | Xeris Biopharma Holdings, Inc. | $8.68 | +5.34% | +18.10% | +8.64% |
| TRAX | First Tracks Biotherapeutics, Inc. | $44.19 | +5.59% | +132.58% | +132.58% |
| A2RR34.SA | Arrowhead Pharmaceuticals, Inc. | 57.60 BRL | +8.15% | +25.08% | +298.89% |
| TYRA | Tyra Biosciences, Inc. | $26.22 | −15.09% | −2.13% | +103.57% |
| 4516.T | Nippon Shinyaku Co., Ltd. | 3,588.00 JPY | +1.07% | −34.41% | +19.48% |
| KALV | KalVista Pharmaceuticals Inc | $27.00 | 0.00% | +74.64% | +98.68% |
| OMER | Omeros Corporation | $18.22 | +61.52% | +11.23% | +328.71% |
| JBIO | Jade Biosciences, Inc. | $21.33 | +8.27% | +50.64% | +168.30% |
| MLTX | MoonLake Immunotherapeutics | $15.62 | −16.52% | +30.71% | −71.82% |
| SVRA | Savara Inc. | $5.61 | +5.25% | −0.53% | +70.52% |
| AVTX | Avalo Therapeutics, Inc. | $20.55 | +5.28% | +20.74% | +112.95% |
| 006280.KS | GC Biopharma Corp. | 127,100.00 KRW | +12.28% | −19.46% | −2.60% |
| WVE | Wave Life Sciences, Inc. | $5.23 | −11.21% | −67.21% | −48.12% |
| DSGN | Design Therapeutics, Inc. | $15.59 | +31.34% | +71.32% | +194.71% |
| ANNX | Annexon, Inc. | $4.99 | +0.40% | +6.40% | +136.49% |
| DBVT | DBV Technologies S.A. | $14.52 | +7.56% | −23.78% | +53.00% |
| CLYM | Climb Bio Inc | $16.08 | +30.84% | +328.80% | +603.72% |
| DBV.PA | DBV Technologies S.A. | 2.53 EUR | +5.42% | −22.51% | +60.13% |
| PHAR | Pharming Group N.V. | $11.95 | −7.08% | −30.28% | −14.47% |
| SLN | Silence Therapeutics plc | $13.53 | +17.14% | +120.00% | +160.19% |
| GNFT.PA | Genfit S.A. | 14.30 EUR | +5.30% | +172.38% | +310.45% |
| STTK | Shattuck Labs, Inc. | $7.90 | +15.84% | +112.94% | +690.00% |
| ASMB | Assembly Biosciences, Inc. | $35.42 | +38.52% | +4.95% | +36.81% |
| IRWD | Ironwood Pharmaceuticals, Inc. | $4.29 | +12.89% | +0.47% | +240.48% |
| GYRE | Gyre Therapeutics, Inc. | $6.65 | +11.02% | −2.35% | −18.00% |
| AGMB | AgomAb Therapeutics NV | $14.28 | +13.88% | −2.53% | −2.53% |
| TECX | Tectonic Therapeutic, Inc. | $35.97 | +20.70% | +72.02% | +35.43% |
| ZVRA | Zevra Therapeutics, Inc. | $11.74 | +20.66% | +40.77% | +25.43% |
| AQST | Aquestive Therapeutics, Inc. | $5.39 | +43.35% | −16.43% | +40.00% |
| CRMD | CorMedix Inc. | $8.39 | +14.15% | −31.00% | −40.66% |
| VALN | Valneva SE | $6.70 | +32.31% | −25.24% | −23.49% |
| AYJ.DE | Valneva SE | 2.98 EUR | +35.29% | −23.93% | −21.89% |
| ZURA | Zura Bio Limited | $5.99 | +8.12% | +7.93% | +177.31% |
| SPRY | ARS Pharmaceuticals, Inc. | $6.03 | +6.35% | −47.52% | −55.13% |
| 005257.KS | GC Corp. | 16,630.00 KRW | +20.51% | −35.95% | −40.16% |
| EPRX | Eupraxia Pharmaceuticals Inc. | $7.43 | +21.80% | −1.85% | +40.72% |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | 10.28 CAD | +20.23% | −1.72% | +43.78% |
| RZLT | Rezolute, Inc. | $5.04 | +2.44% | +132.79% | −30.00% |
| KMDA | Kamada Ltd. Ordinary Shares | $8.38 | +18.70% | +22.82% | +21.09% |
| QTTB | Q32 Bio Inc. | $16.18 | +2.99% | +370.35% | +840.70% |
| AVIR | Atea Pharmaceuticals, Inc. | $5.51 | +22.17% | +58.33% | +53.06% |
| LRMR | Larimar Therapeutics, Inc. | $4.17 | +8.88% | +20.17% | +6.92% |
| XFOR | X4 Pharmaceuticals, Inc. | $4.17 | +6.11% | +7.20% | −1.42% |
| ENTA | Enanta Pharmaceuticals, Inc. | $14.15 | +12.21% | −1.87% | +65.89% |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | 531.00 JPY | +2.71% | −21.85% | −9.63% |
| UPB | Upstream Bio, Inc. | $7.13 | +11.41% | −73.66% | −57.31% |
| IFRX | InflaRx N.V. | $2.42 | +43.20% | +122.02% | +142.00% |
| IKT | Inhibikase Therapeutics, Inc. | $2.49 | +7.79% | +27.69% | +39.11% |
| CUV.AX | Clinuvel Pharmaceuticals Limited | 9.36 AUD | −2.40% | −26.36% | −24.94% |
| PRQR | ProQR Therapeutics N.V. | $2.35 | +33.52% | +18.09% | +6.33% |
| TRDA | Entrada Therapeutics, Inc. | $7.00 | +20.07% | −30.35% | +23.24% |
| FULC | Fulcrum Therapeutics, Inc. | $3.86 | +3.76% | −64.65% | −41.78% |
| TTRX | Turn Therapeutics Inc. | $9.63 | +25.06% | +105.33% | +37.57% |
| SION | Sionna Therapeutics, Inc. | $6.17 | −86.75% | −84.26% | −75.45% |
| ELDN | Eledon Pharmaceuticals, Inc. | $3.35 | −1.47% | +106.79% | +29.84% |
| IVVD | Invivyd, Inc. | $0.90 | +52.06% | −63.93% | +3.52% |
| CAMP | CAMP4 THERAPEUTICS CORPORATION | $4.20 | +22.81% | −30.00% | +178.15% |
| SLGL | Sol-Gel Technologies Ltd. | $79.43 | −4.38% | +88.45% | +261.87% |
| XUP.DE | Genfit S.A. | 14.08 EUR | −1.81% | +164.41% | +166.82% |
| AKBA | Akebia Therapeutics, Inc. | $0.91 | −28.62% | −41.52% | −69.88% |
| CRDL | Cardiol Therapeutics Inc. | $2.14 | +78.33% | +115.29% | +91.07% |
| CRDL.TO | Cardiol Therapeutics Inc. | 2.97 CAD | +74.71% | +118.38% | +90.38% |
| PEPG | PepGen Inc. | $3.22 | +75.96% | −55.09% | +159.68% |
| RNA | Prosensa Holding B.V. Ordinary Shares | $13.85 | +20.54% | −6.10% | −6.10% |
| SIGA | SIGA Technologies, Inc. | $3.07 | −8.08% | −44.65% | −57.93% |
| KROS | Keros Therapeutics, Inc. | $11.12 | +10.43% | −40.02% | −28.35% |
| ANTX | AN2 Therapeutics, Inc. | $5.53 | +17.66% | +398.20% | +385.09% |
| ARMP | Armata Pharmaceuticals, Inc. | $5.45 | +15.22% | −16.92% | +123.36% |
| MSCL.TO | Satellos Bioscience Inc. | 13.08 CAD | −2.10% | +55.71% | +81.67% |
| PLX | Protalix BioTherapeutics, Inc. | $2.48 | +6.44% | +42.53% | +55.00% |
| MSLE | Satellos Bioscience Inc. | $9.36 | −1.06% | +50.87% | +77.27% |
| SPRB | Spruce Biosciences, Inc. | $61.48 | +47.79% | −32.22% | +721.93% |
| MDWD | MediWound Ltd. | $12.70 | −13.13% | −31.05% | −25.29% |
| EQ | Equillium, Inc. | $2.36 | +1.72% | +54.25% | +23.56% |
| AARD | Aardvark Therapeutics, Inc. | $6.81 | +6.07% | −48.37% | −24.25% |
| UNCY | Unicycive Therapeutics, Inc. | $5.43 | +9.92% | −6.38% | +27.17% |
| CRNO-B.ST | Cereno Scientific AB (publ) | 4.71 SEK | +0.13% | −32.04% | −53.59% |
| MIST | Milestone Pharmaceuticals Inc. | $1.18 | +10.28% | −42.44% | −33.33% |
| 4599.T | StemRIM | 350.00 JPY | +21.53% | +32.08% | +14.75% |
| INO | Inovio Pharmaceuticals, Inc. | $1.30 | +26.21% | −23.08% | −47.15% |
| CPIX | Cumberland Pharmaceuticals Inc. | $8.97 | +69.86% | +125.38% | +163.82% |
| DXB.AX | Dimerix Limited | 0.29 AUD | +20.83% | −47.27% | −34.83% |
| MDP.TO | Medexus Pharmaceuticals Inc. | 5.18 CAD | +7.02% | +67.64% | +66.56% |
| PYPD | PolyPid Ltd. | $5.11 | +36.27% | +17.47% | +49.42% |
| PTHS | Pelthos Therapeutics Inc. | $26.78 | −3.56% | −9.07% | +12.90% |
| ABNX.PA | ABIONYX Pharma SA | 1.97 EUR | +5.79% | −49.58% | −31.34% |
| ILA.AX | Island Pharmaceuticals Limited | 0.45 AUD | +18.42% | +3.45% | +109.30% |
| FBIO | Fortress Biotech, Inc. | $2.81 | −17.11% | −30.27% | +20.09% |
| PLYX | Polaryx Therapeutics, Inc. | $2.09 | +5.03% | −94.03% | −94.03% |
| PAR.AX | Paradigm Biopharmaceuticals Limited | 0.22 AUD | +30.30% | −36.76% | −24.56% |
| RLYB | Rallybio Corporation | $16.88 | +4.49% | +198.44% | +257.63% |
| GOSS | Gossamer Bio, Inc. | $0.17 | −15.17% | −93.96% | −92.93% |
| RCE.AX | Recce Pharmaceuticals Ltd | 0.38 AUD | +2.74% | −43.82% | −10.71% |
| TELO | Telomir Pharmaceuticals, Inc. | $1.17 | −0.85% | −12.69% | −20.95% |
| TENX | Tenax Therapeutics, Inc. | $2.12 | −86.58% | −80.81% | −64.19% |
| STX.L | Shield Therapeutics plc | 4.85 GBP | −15.65% | −50.76% | −37.42% |
| FBIOP | Fortress Biotech, Inc. | $18.40 | −1.92% | +81.10% | +131.74% |
| ALTHX.PA | THX Pharma Société Anonyme | 4.18 EUR | +5.16% | +83.92% | +73.96% |
| SPRO | Spero Therapeutics, Inc. | $1.22 | +2.09% | −48.09% | −38.38% |
| RNTX | Rein Therapeutics Inc. | $0.80 | +2.30% | −36.00% | −27.27% |
| IMA | ImageneBio, Inc. | $5.43 | −5.73% | −19.67% | −60.19% |
| SCYX | SCYNEXIS, Inc. | $5.67 | +56.20% | +787.32% | +579.04% |
| ATYR | aTyr Pharma, Inc. | $0.55 | +11.54% | −27.40% | −89.34% |
| NSRX | Nasus Pharma Ltd. | $4.05 | +33.66% | −46.36% | −49.75% |
| MCRB | Seres Therapeutics, Inc. | $4.76 | −0.83% | −68.58% | −74.79% |
| EDSA | Edesa Biotech, Inc. | $4.94 | −0.60% | +229.33% | +105.83% |
| GRCE | Grace Therapeutics, Inc. | $2.15 | −4.44% | −41.26% | −29.97% |
| MREO | Mereo BioPharma Group plc | $0.28 | −10.03% | −45.49% | −84.20% |
| BOT.AX | Botanix Pharmaceuticals Limited | 0.02 AUD | +5.26% | −85.19% | −86.67% |
| NNVC | NanoViricides, Inc. | $1.28 | −20.50% | +5.79% | −9.86% |
| QNCX | Quince Therapeutics, Inc. | $31.31 | +61.64% | +4.37% | +100.71% |
| IMM.L | ImmuPharma plc | 3.41 GBP | −24.22% | −45.00% | +89.66% |
| 520A.T | J-PHARMACEUTICALS CO LTD | 280.00 JPY | +4.09% | −60.00% | −60.00% |
| ALDVI.PA | Advicenne S.A. | 1.75 EUR | +4.79% | −6.02% | +39.11% |
| 4591.T | Ribomic Inc. | 86.00 JPY | +21.13% | +10.26% | −17.31% |
| BRNS | Barinthus Biotherapeutics plc | $0.71 | +23.93% | −3.89% | −38.04% |
| RVX.TO | Resverlogix Corp. | 0.10 CAD | 0.00% | 0.00% | 0.00% |
| JSPR | Jasper Therapeutics, Inc. | $0.72 | +13.43% | −60.98% | −75.24% |
| TVRD | Tvardi Therapeutics, Inc. | $1.78 | −27.05% | −56.37% | −94.18% |
| 4884.T | Kringle Pharma, Inc. | 440.00 JPY | +42.86% | +12.24% | −11.29% |
| COCP | Cocrystal Pharma, Inc. | $1.12 | +20.43% | +13.13% | −27.74% |
| VYNE | VYNE Therapeutics Inc | $28.56 | +3080.40% | +4824.14% | +8660.74% |
| KTTA | Pasithea Therapeutics Corp. | $0.57 | +26.67% | −52.10% | −24.30% |
| CALC | CalciMedica, Inc. | $0.59 | −18.18% | −91.28% | −79.74% |
| CMMB | Chemomab Therapeutics Ltd. | $2.59 | +81.12% | +44.69% | −25.57% |
| 4881.T | FunPep Company Limited | 51.00 JPY | +2.00% | −37.04% | −55.65% |
| HIND | Vyome Holdings, Inc. | $2.16 | +8.54% | −39.15% | −79.68% |
| ALBPS.PA | Biophytis S.A. | 0.08 EUR | +422.88% | +28.41% | −50.31% |
| AZTR | Azitra, Inc. | $0.17 | +33.00% | −38.03% | −83.21% |
| QNRX | Quoin Pharmaceuticals, Ltd. | $5.18 | +23.33% | −63.21% | −29.81% |
| ANNX.ST | Annexin Pharmaceuticals AB (publ) | 13.60 SEK | +7.09% | −31.66% | −46.03% |
| PHR.WA | Pharmena S.A. | 3.14 PLN | +1.62% | +18.49% | −8.72% |
| IMRN | Immuron Limited | $1.14 | +4.11% | +43.40% | −35.77% |
| IMC.AX | Immuron Limited | 0.04 AUD | +8.82% | +5.71% | −46.38% |
| SNGX | Soligenix, Inc. | $0.38 | +6.67% | −72.96% | −87.20% |
| XCUR | Exicure, Inc. | $1.26 | −27.59% | −77.42% | −77.93% |
| INM | InMed Pharmaceuticals Inc. | $1.43 | −14.37% | +27.68% | −36.44% |
| VBS.AX | Vectus Biosystems Limited | 0.17 AUD | +30.77% | +3.03% | +104.82% |
| PULM | Pulmatrix, Inc. | $1.53 | −9.23% | −32.52% | −68.62% |
| BIOSGN.ST | Biosergen AB (publ) | 2.48 SEK | +262.31% | −93.80% | −94.62% |
| CVKD | Cadrenal Therapeutics, Inc. | $1.55 | −22.89% | −78.85% | −88.87% |
| JAGX | Jaguar Health, Inc. | $0.92 | −4.63% | −97.37% | −98.52% |
| GLMD | Galmed Pharmaceuticals Ltd. | $4.33 | +710.86% | +426.12% | +211.51% |
| GRI | GRI Bio, Inc. | $1.91 | +33.57% | −75.64% | −95.23% |
| XRTX | XORTX Therapeutics Inc. | $2.22 | +8.20% | −22.18% | −43.99% |
| BIT.AX | Biotron Limited | 0.00 AUD | −20.00% | −42.86% | −50.00% |
| SXTP | 60 Degrees Pharmaceuticals, Inc. | $1.08 | −28.00% | −47.67% | −78.23% |
| MTNB | Matinas BioPharma Holdings, Inc. | $0.20 | −32.97% | −68.32% | −90.08% |
| ANR.AX | Anatara Lifesciences Ltd | 0.01 AUD | −10.00% | −35.71% | 0.00% |
| APTA.ST | Aptahem AB (publ) | 0.56 SEK | −15.96% | −51.69% | −67.65% |
| APLI.TO | Appili Therapeutics Inc. | 0.02 CAD | +50.00% | −40.00% | −25.00% |
| VBIO | Valion Bio, Inc. | $0.16 | −45.83% | −89.01% | −94.92% |
| ALNEV.PA | Neovacs S.A. | 0.55 EUR | −31.22% | +6150.00% | −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.