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Rare disease stocks

We cover 226 companies grouped under the Rare disease theme. The median market value in the group is $432.15M. Across the group, the median move over the past trading day is −0.49%. 51% of the 212 with a 200-day average trade above it. Price change over the past year runs from −69.4% to +184.7% across the middle 80 percent of the group, with a median of +11.9%.

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Table

226 of 268 companies match these filters.Clear filters

TickerCompanyPrice1M returnYTD return1Y return
VX1.DEVertex Pharmaceuticals Incorporated475.00 EUR+10.21%+23.55%+41.64%
VRTXVertex Pharmaceuticals Incorporated$547.29+11.60%+21.05%+40.66%
DUL.DEAlnylam Pharmaceuticals, Inc.204.00 EUR−19.59%−40.19%−10.21%
ALNYAlnylam Pharmaceuticals, Inc.$239.00−17.13%−40.28%−47.38%
A1LN34.SAAlnylam Pharmaceuticals, Inc.62.25 BRL−15.31%−44.14%−49.35%
INSMInsmed Incorporated$124.39+19.46%−29.77%−7.54%
ROIVRoivant Sciences Ltd.$36.75+8.60%+67.58%+206.51%
JAZZJazz Pharmaceuticals plc$249.29−2.97%+43.97%+100.35%
ASNDAscendis Pharma A/S$250.25−1.65%+17.33%+29.66%
BBIOBridgeBio Pharma, Inc.$79.37−7.22%+1.45%+60.05%
CORTCorcept Therapeutics Incorporated$119.80+27.19%+213.61%+71.81%
0JAV.LInsmed Incorporated$125.00+0.43%−28.28%−7.52%
BMRNBioMarin Pharmaceutical Inc.$65.61+5.70%+10.36%+11.85%
REC.MIRecordati Industria Chimica e Farmaceutica S.p.A.52.70 EUR+2.53%+10.68%+1.80%
ARWRArrowhead Pharmaceuticals, Inc.$89.12+3.89%+31.46%+308.99%
CYTKCytokinetics, Incorporated$79.20−0.31%+28.30%+109.36%
IONSIonis Pharmaceuticals, Inc.$63.36+14.58%−20.47%+48.21%
KYMRKymera Therapeutics, Inc.$125.55+18.26%+72.55%+197.09%
0JDI.LIonis Pharmaceuticals, Inc.$63.37+3.44%−19.72%+47.26%
ABVX.PAABIVAX Société Anonyme102.30 EUR−5.63%−11.04%+48.91%
PTGXProtagonist Therapeutics, Inc.$151.50+10.71%+73.78%+146.62%
IMVTImmunovant, Inc.$43.89+12.83%+69.39%+189.13%
4151.TKyowa Kirin Co., Ltd.2,613.00 JPY−3.33%+3.94%+4.01%
RYTMRhythm Pharmaceuticals, Inc.$111.87+3.54%+6.53%+9.35%
SRRKScholar Rock Holding Corporation$60.04+21.61%+40.28%+74.18%
0HI3.LArrowhead Pharmaceuticals, Inc.$89.35+3.26%+32.80%+294.59%
ORKAOruka Therapeutics, Inc.$99.44+9.94%+251.75%+555.07%
LQDALiquidia Corporation$71.36−17.81%+127.26%+154.86%
TVTXTravere Therapeutics, Inc.$67.12+20.05%+69.84%+284.86%
DNTHDianthus Therapeutics, Inc.$110.76+9.01%+179.13%+356.93%
PTCTPTC Therapeutics, Inc. Common Stock$72.69−3.58%−5.28%+46.02%
KNSAKiniksa Pharmaceuticals International, plc$78.68−0.96%+87.31%+133.47%
MIRMMirum Pharmaceuticals, Inc.$98.51−12.22%+26.18%+31.96%
GRFSGrifols, S.A.$7.90−1.25%−14.50%−19.39%
DYNDyne Therapeutics, Inc.$26.36+1.78%+42.49%+93.26%
EWTXEdgewise Therapeutics, Inc.$44.38+9.99%+86.86%+207.13%
MANEVeradermics, Incorporated$102.80−7.84%+172.32%+172.32%
CAMX.STCamurus AB (publ)615.00 SEK−1.68%+0.49%−15.35%
CLDXCelldex Therapeutics, Inc.$40.57+14.02%+50.20%+79.51%
IRONDisc Medicine, Inc.$82.67+10.74%+4.53%+37.90%
ARQTArcutis Biotherapeutics, Inc.$25.14−11.54%−13.31%+60.64%
0RD1.LCamurus AB (publ)609.98 SEK−0.21%−0.53%−15.10%
VRDNViridian Therapeutics, Inc.$24.26+24.35%−20.93%+30.71%
VERAVera Therapeutics, Inc.$36.82+10.50%−24.27%+67.21%
RAREUltragenyx Pharmaceutical Inc.$26.44+1.77%+12.03%−12.10%
PHVSPharvaris N.V.$37.00+9.97%+39.73%+55.59%
TRVITrevi Therapeutics, Inc.$17.92−2.77%+50.71%+124.00%
NKTRNektar Therapeutics$73.77+4.24%+69.90%+151.95%
BCRXBioCryst Pharmaceuticals, Inc.$9.83+0.92%+31.07%+15.38%
009420.KSHanall Biopharma Co., Ltd.64,000.00 KRW+53.48%+51.66%+112.62%
COAGHemab Therapeutics Holdings, Inc.$49.19+4.08%+44.68%+44.68%
MLYSMineralys Therapeutics, Inc.$25.89−3.93%−27.50%+70.22%
PVLAPalvella Therapeutics, Inc.$154.33+8.04%+53.90%+173.39%
AUPHAurinia Pharmaceuticals Inc.$16.54+10.56%+7.75%+36.58%
ADMAADMA Biologics, Inc.$9.63+9.56%−46.14%−44.17%
ZBIOZenas BioPharma, Inc.$33.04+2.32%−4.23%+97.73%
STOKStoke Therapeutics, Inc.$33.09+12.59%+7.09%+71.45%
AGIOAgios Pharmaceuticals, Inc. Common Stock$33.99−5.56%+25.06%−13.45%
SEPNSepterna, Inc.$40.25+18.49%+43.85%+218.81%
NEU.AXNeuren Pharmaceuticals Limited20.59 AUD+20.20%+8.88%+5.54%
ANABAnaptysBio, Inc.$58.00+11.30%+93.55%+326.47%
ANIPANI Pharmaceuticals, Inc.$74.56−8.00%−4.93%−18.85%
0L35.LSarepta Therapeutics, Inc.$18.51+1.60%−13.25%−0.06%
MAZEMaze Therapeutics, Inc.$29.58+6.10%−25.62%+113.27%
AVLNAvalyn Pharma Inc.$37.04+33.29%+25.60%+25.60%
PYC.AXPYC Therapeutics Limited2.25 AUD+38.46%+35.95%+74.42%
XERSXeris Biopharma Holdings, Inc.$8.68+5.34%+18.10%+8.64%
TRAXFirst Tracks Biotherapeutics, Inc.$44.19+5.59%+132.58%+132.58%
A2RR34.SAArrowhead Pharmaceuticals, Inc.57.60 BRL+8.15%+25.08%+298.89%
TYRATyra Biosciences, Inc.$26.22−15.09%−2.13%+103.57%
4516.TNippon Shinyaku Co., Ltd.3,588.00 JPY+1.07%−34.41%+19.48%
0LIF.LUltragenyx Pharmaceutical Inc.$26.19+1.39%+12.52%−13.39%
JBIOJade Biosciences, Inc.$21.33+8.27%+50.64%+168.30%
MLTXMoonLake Immunotherapeutics$15.62−16.52%+30.71%−71.82%
0HB0.LAgios Pharmaceuticals, Inc.$34.05+0.18%+26.06%−13.00%
SVRASavara Inc.$5.61+5.25%−0.53%+70.52%
AVTXAvalo Therapeutics, Inc.$20.55+5.28%+20.74%+112.95%
006280.KSGC Biopharma Corp.127,100.00 KRW+12.28%−19.46%−2.60%
WVEWave Life Sciences, Inc.$5.23−11.21%−67.21%−48.12%
DSGNDesign Therapeutics, Inc.$15.59+31.34%+71.32%+194.71%
ANNXAnnexon, Inc.$4.99+0.40%+6.40%+136.49%
DBVTDBV Technologies S.A.$14.52+7.56%−23.78%+53.00%
CLYMClimb Bio Inc$16.08+30.84%+328.80%+603.72%
DBV.PADBV Technologies S.A.2.53 EUR+5.42%−22.51%+60.13%
PHARPharming Group N.V.$11.95−7.08%−30.28%−14.47%
SLNSilence Therapeutics plc$13.53+17.14%+120.00%+160.19%
GNFT.PAGenfit S.A.14.30 EUR+5.30%+172.38%+310.45%
STTKShattuck Labs, Inc.$7.90+15.84%+112.94%+690.00%
IRWDIronwood Pharmaceuticals, Inc.$4.29+12.89%+0.47%+240.48%
GYREGyre Therapeutics, Inc.$6.65+11.02%−2.35%−18.00%
AGMBAgomAb Therapeutics NV$14.28+13.88%−2.53%−2.53%
TECXTectonic Therapeutic, Inc.$35.97+20.70%+72.02%+35.43%
ZVRAZevra Therapeutics, Inc.$11.74+20.66%+40.77%+25.43%
CRMDCorMedix Inc.$8.39+14.15%−31.00%−40.66%
VALNValneva SE$6.70+32.31%−25.24%−23.49%
AYJ.DEValneva SE2.98 EUR+35.29%−23.93%−21.89%
ZURAZura Bio Limited$5.99+8.12%+7.93%+177.31%
SPRYARS Pharmaceuticals, Inc.$6.03+6.35%−47.52%−55.13%
005257.KSGC Corp.16,630.00 KRW+20.51%−35.95%−40.16%
EPRXEupraxia Pharmaceuticals Inc.$7.43+21.80%−1.85%+40.72%
EPRX.TOEupraxia Pharmaceuticals Inc.10.28 CAD+20.23%−1.72%+43.78%
RZLTRezolute, Inc.$5.04+2.44%+132.79%−30.00%
KMDAKamada Ltd. Ordinary Shares$8.38+18.70%+22.82%+21.09%
QTTBQ32 Bio Inc.$16.18+2.99%+370.35%+840.70%
AVIRAtea Pharmaceuticals, Inc.$5.51+22.17%+58.33%+53.06%
LRMRLarimar Therapeutics, Inc.$4.17+8.88%+20.17%+6.92%
XFORX4 Pharmaceuticals, Inc.$4.17+6.11%+7.20%−1.42%
ENTAEnanta Pharmaceuticals, Inc.$14.15+12.21%−1.87%+65.89%
4552.TJCR Pharmaceuticals Co., Ltd.531.00 JPY+2.71%−21.85%−9.63%
UPBUpstream Bio, Inc.$7.13+11.41%−73.66%−57.31%
IFRXInflaRx N.V.$2.42+43.20%+122.02%+142.00%
IKTInhibikase Therapeutics, Inc.$2.49+7.79%+27.69%+39.11%
CUV.AXClinuvel Pharmaceuticals Limited9.36 AUD−2.40%−26.36%−24.94%
PRQRProQR Therapeutics N.V.$2.35+33.52%+18.09%+6.33%
TRDAEntrada Therapeutics, Inc.$7.00+20.07%−30.35%+23.24%
FULCFulcrum Therapeutics, Inc.$3.86+3.76%−64.65%−41.78%
TTRXTurn Therapeutics Inc.$9.63+25.06%+105.33%+37.57%
ELDNEledon Pharmaceuticals, Inc.$3.35−1.47%+106.79%+29.84%
IVVDInvivyd, Inc.$0.90+52.06%−63.93%+3.52%
CAMPCAMP4 THERAPEUTICS CORPORATION$4.20+22.81%−30.00%+178.15%
SLGLSol-Gel Technologies Ltd.$79.43−4.38%+88.45%+261.87%
0QN1.LSanthera Pharmaceuticals Holding AG15.66 CHF−1.88%+29.00%+11.86%
AKBAAkebia Therapeutics, Inc.$0.91−28.62%−41.52%−69.88%
0T6G.LQ32 Bio Inc.$16.34+7.94%
PEPGPepGen Inc.$3.22+75.96%−55.09%+159.68%
RNAProsensa Holding B.V. Ordinary Shares$13.85+20.54%−6.10%−6.10%
SIGASIGA Technologies, Inc.$3.07−8.08%−44.65%−57.93%
KROSKeros Therapeutics, Inc.$11.12+10.43%−40.02%−28.35%
ANTXAN2 Therapeutics, Inc.$5.53+17.66%+398.20%+385.09%
ARMPArmata Pharmaceuticals, Inc.$5.45+15.22%−16.92%+123.36%
MSCL.TOSatellos Bioscience Inc.13.08 CAD−2.10%+55.71%+81.67%
PLXProtalix BioTherapeutics, Inc.$2.48+6.44%+42.53%+55.00%
MSLESatellos Bioscience Inc.$9.36−1.06%+50.87%+77.27%
0AB6.LHyloris Pharmaceuticals SA4.38 EUR−1.79%−38.83%−33.23%
EQEquillium, Inc.$2.36+1.72%+54.25%+23.56%
AARDAardvark Therapeutics, Inc.$6.81+6.07%−48.37%−24.25%
UNCYUnicycive Therapeutics, Inc.$5.43+9.92%−6.38%+27.17%
CRNO-B.STCereno Scientific AB (publ)4.71 SEK+0.13%−32.04%−53.59%
MISTMilestone Pharmaceuticals Inc.$1.18+10.28%−42.44%−33.33%
4599.TStemRIM350.00 JPY+21.53%+32.08%+14.75%
INOInovio Pharmaceuticals, Inc.$1.30+26.21%−23.08%−47.15%
DXB.AXDimerix Limited0.29 AUD+20.83%−47.27%−34.83%
0H8T.LAclaris Therapeutics, Inc.$6.14−6.69%+105.92%+237.38%
RFLRafael Holdings, Inc.$2.29+9.05%+81.75%+52.67%
MDP.TOMedexus Pharmaceuticals Inc.5.18 CAD+7.02%+67.64%+66.56%
PYPDPolyPid Ltd.$5.11+36.27%+17.47%+49.42%
PTHSPelthos Therapeutics Inc.$26.78−3.56%−9.07%+12.90%
ABNX.PAABIONYX Pharma SA1.97 EUR+5.79%−49.58%−31.34%
0RAG.LABIONYX Pharma SA2.01 EUR+3.40%−48.40%−29.10%
ILA.AXIsland Pharmaceuticals Limited0.45 AUD+18.42%+3.45%+109.30%
FBIOFortress Biotech, Inc.$2.81−17.11%−30.27%+20.09%
PLYXPolaryx Therapeutics, Inc.$2.09+5.03%−94.03%−94.03%
PAR.AXParadigm Biopharmaceuticals Limited0.22 AUD+30.30%−36.76%−24.56%
RLYBRallybio Corporation$16.88+4.49%+198.44%+257.63%
GOSSGossamer Bio, Inc.$0.17−15.17%−93.96%−92.93%
RCE.AXRecce Pharmaceuticals Ltd0.38 AUD+2.74%−43.82%−10.71%
TELOTelomir Pharmaceuticals, Inc.$1.17−0.85%−12.69%−20.95%
STX.LShield Therapeutics plc4.85 GBP−15.65%−50.76%−37.42%
FBIOPFortress Biotech, Inc.$18.40−1.92%+81.10%+131.74%
ALTHX.PATHX Pharma Société Anonyme4.18 EUR+5.16%+83.92%+73.96%
SPROSpero Therapeutics, Inc.$1.22+2.09%−48.09%−38.38%
RNTXRein Therapeutics Inc.$0.80+2.30%−36.00%−27.27%
IMAImageneBio, Inc.$5.43−5.73%−19.67%−60.19%
SCYXSCYNEXIS, Inc.$5.67+56.20%+787.32%+579.04%
ATYRaTyr Pharma, Inc.$0.55+11.54%−27.40%−89.34%
NSRXNasus Pharma Ltd.$4.05+33.66%−46.36%−49.75%
MCRBSeres Therapeutics, Inc.$4.76−0.83%−68.58%−74.79%
EDSAEdesa Biotech, Inc.$4.94−0.60%+229.33%+105.83%
GRCEGrace Therapeutics, Inc.$2.15−4.44%−41.26%−29.97%
MREOMereo BioPharma Group plc$0.28−10.03%−45.49%−84.20%
BOT.AXBotanix Pharmaceuticals Limited0.02 AUD+5.26%−85.19%−86.67%
NNVCNanoViricides, Inc.$1.28−20.50%+5.79%−9.86%
QNCXQuince Therapeutics, Inc.$31.31+61.64%+4.37%+100.71%
IMM.LImmuPharma plc3.41 GBP−24.22%−45.00%+89.66%
520A.TJ-PHARMACEUTICALS CO LTD280.00 JPY+4.09%−60.00%−60.00%
ALDVI.PAAdvicenne S.A.1.75 EUR+4.79%−6.02%+39.11%
4591.TRibomic Inc.86.00 JPY+21.13%+10.26%−17.31%
BRNSBarinthus Biotherapeutics plc$0.71+23.93%−3.89%−38.04%
0RQJ.LSaniona AB (publ)11.80 SEK+0.47%−44.08%−12.20%
RVX.TOResverlogix Corp.0.10 CAD0.00%0.00%0.00%
JSPRJasper Therapeutics, Inc.$0.72+13.43%−60.98%−75.24%
TVRDTvardi Therapeutics, Inc.$1.78−27.05%−56.37%−94.18%
4884.TKringle Pharma, Inc.440.00 JPY+42.86%+12.24%−11.29%
COCPCocrystal Pharma, Inc.$1.12+20.43%+13.13%−27.74%
KTTAPasithea Therapeutics Corp.$0.57+26.67%−52.10%−24.30%
CALCCalciMedica, Inc.$0.59−18.18%−91.28%−79.74%
4881.TFunPep Company Limited51.00 JPY+2.00%−37.04%−55.65%
HINDVyome Holdings, Inc.$2.16+8.54%−39.15%−79.68%
ALBPS.PABiophytis S.A.0.08 EUR+422.88%+28.41%−50.31%
AZTRAzitra, Inc.$0.17+33.00%−38.03%−83.21%
QNRXQuoin Pharmaceuticals, Ltd.$5.18+23.33%−63.21%−29.81%
ANNX.STAnnexin Pharmaceuticals AB (publ)13.60 SEK+7.09%−31.66%−46.03%
PHR.WAPharmena S.A.3.14 PLN+1.62%+18.49%−8.72%
IMRNImmuron Limited$1.14+4.11%+43.40%−35.77%
IMC.AXImmuron Limited0.04 AUD+8.82%+5.71%−46.38%
SNGXSoligenix, Inc.$0.38+6.67%−72.96%−87.20%
XCURExicure, Inc.$1.26−27.59%−77.42%−77.93%
INMInMed Pharmaceuticals Inc.$1.43−14.37%+27.68%−36.44%
VBS.AXVectus Biosystems Limited0.17 AUD+30.77%+3.03%+104.82%
PULMPulmatrix, Inc.$1.53−9.23%−32.52%−68.62%
BIOSGN.STBiosergen AB (publ)2.48 SEK+262.31%−93.80%−94.62%
JAGXJaguar Health, Inc.$0.92−4.63%−97.37%−98.52%
GRIGRI Bio, Inc.$1.91+33.57%−75.64%−95.23%
XRTXXORTX Therapeutics Inc.$2.22+8.20%−22.18%−43.99%
BIT.AXBiotron Limited0.00 AUD−20.00%−42.86%−50.00%
SXTP60 Degrees Pharmaceuticals, Inc.$1.08−28.00%−47.67%−78.23%
MTNBMatinas BioPharma Holdings, Inc.$0.20−32.97%−68.32%−90.08%
APTA.STAptahem AB (publ)0.56 SEK−15.96%−51.69%−67.65%
APLI.TOAppili Therapeutics Inc.0.02 CAD+50.00%−40.00%−25.00%
VBIOValion Bio, Inc.$0.16−45.83%−89.01%−94.92%
ALNEV.PANeovacs S.A.0.55 EUR−31.22%+6150.00%−11.29%
0A43.LInovio Pharmaceuticals, Inc.$1.32+4.35%−20.55%−44.09%
0A4D.LEquillium, Inc.$2.41+10.15%+63.95%+37.32%
0A6I.LSoligenix, Inc.$0.39−4.04%−71.78%−87.16%
0A9G.LMereo BioPharma Group plc$0.28−2.36%−42.56%−84.25%
0QNS.LCuratis Holding AG19.05 CHF0.00%−15.71%−15.71%
0UNL.LNektar Therapeutics$74.20+6.73%+14.04%+14.04%
1AE.DEargenx SE898.60 EUR+15.89%+24.05%+38.37%
2X1.DEABIVAX Société Anonyme100.50 EUR−6.77%−12.30%+33.82%
BIOV.SWBioVersys AG25.20 CHF−6.67%+6.33%−9.03%
BPBS.PABIOPHYTIS BSA0.00 EUR0.00%0.00%0.00%
CESSA.COCessatech A/S9.42 DKK+9.53%−48.38%−48.38%
CURN.SWCuratis Holding AG19.00 CHF−11.21%+45.59%+65.22%
DBV.DEDBV Technologies S.A.2.46 EUR+12.83%−25.17%+64.35%
PHGN.DEPharming Group N.V.0.98 EUR−10.95%−32.74%−21.41%
SANN.SWSanthera Pharmaceuticals Holding AG15.70 CHF+9.79%+29.32%+13.44%

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