Rare Diseases Stocks
Biotechnology and pharmaceutical firms in this sector focus on developing therapies for orphan diseases affecting small patient populations where high unmet medical needs justify premium pricing power and extended regulatory exclusivity.
As of Oct 23, 2026, Rare Diseases has 182 US-listed companies worth $1.5T combined. The median member moved +2.2% over the past week; UTHR led among the 25 largest at +10.2% and CORT trailed at -22.1%.
Companies in Rare Diseases
- AZN – AstraZeneca PLC, $266B
- AMGN – Amgen Inc, $237B
- NVO – Novo Nordisk A/S, $172B
- VRTX – Vertex Pharmaceuticals Inc, $136B
- SNY – Sanofi ADR, $99.7B
- TAK – Takeda Pharmaceutical Co Ltd ADR, $64.7B
- ARGX – argenx NV ADR, $54.9B
- BIIB – Biogen Inc, $37B
- RPRX – Royalty Pharma Plc, $36.8B
- ALNY – Alnylam Pharmaceuticals Inc, $30B
- ROIV – Roivant Sciences Ltd, $27.1B
- UTHR – United Therapeutics Corporation, $25.1B
- INCY – Incyte Corporation, $23.7B
- INSM – Insmed Inc, $20.4B
- ASND – Ascendis Pharma AS, $16B
- NBIX – Neurocrine Biosciences Inc, $14.6B
- JAZZ – Jazz Pharmaceuticals PLC, $13.9B
- BBIO – BridgeBio Pharma Inc, $13.4B
- CORT – Corcept Therapeutics Incorporated, $11.6B
- BMRN – Biomarin Pharmaceutical Inc, $10.8B
- KRYS – Krystal Biotech Inc, $9.6B
- IONS – Ionis Pharmaceuticals Inc, $7.3B
- PRAX – Praxis Precision Medicines Inc, $7.2B
- KNSA – Kiniksa Pharmaceuticals Ltd, $6.3B
- RYTM – Rhythm Pharmaceuticals Inc, $6.3B
- IMVT – Immunovant Inc, $5.9B
- COGT – Cogent Biosciences Inc, $5.5B
- MIRM – Mirum Pharmaceuticals Inc, $5.3B
- PTCT – PTC Therapeutics Inc, $5.3B
- SRRK – Scholar Rock Holding Corp, $5.1B
- TVTX – Travere Therapeutics Inc, $4.9B
- CRSP – Crispr Therapeutics AG, $4.8B
- EWTX – Edgewise Therapeutics Inc, $4.3B
- LEGN – Legend Biotech Corp, $3.7B
- AMLX – Amylyx Pharmaceuticals Inc, $3.6B
- ACAD – ACADIA Pharmaceuticals Inc, $3.5B
- IRON – Disc Medicine Inc., $3B
- DNLI – Denali Therapeutics Inc, $3B
- DYN – Dyne Therapeutics Inc, $2.9B
- VRDN – Viridian Therapeutics Inc, $2.5B
- BEAM – Beam Therapeutics Inc, $2.5B
- MRVI – Maravai Lifesciences Holdings Inc, $2.4B
- VERA – Vera Therapeutics Inc, $2.2B
- AGIO – Agios Pharm, $2.2B
- HRMY – Harmony Biosciences Holdings, $2.1B
- PHVS – Pharvaris BV, $2.1B
- WGS – GeneDx Holdings Corp., $2B
- XERS – Xeris Pharmaceuticals Inc, $2B
- BCRX – BioCryst Pharmaceuticals Inc, $2B
- ZBIO – Zenas BioPharma, Inc. Common Stock, $1.8B
- TRVI – Trevi Therapeutics Inc, $1.8B
- SRPT – Sarepta Therapeutics Inc, $1.8B
- BHVN – Biohaven Ltd., $1.8B
- PVLA – Palvella Therapeutics, Inc, $1.7B
- MESO – Mesoblast Ltd, $1.7B
- COAG – Hemab Therapeutics Holdings, Inc. Common Stock, $1.7B
- ANIP – ANI Pharmaceuticals Inc, $1.7B
- STOK – Stoke Therapeutics Inc, $1.7B
- ETON – Eton Pharmaceuticals Inc, $1.6B
- NTLA – Intellia Therapeutics Inc, $1.6B
- QURE – Uniqure NV, $1.6B
- RARE – Ultragenyx, $1.6B
- MAZE – Maze Therapeutics, Inc. Common Stock, $1.5B
- TYRA – Tyra Biosciences Inc, $1.5B
- INBX – Inhibrx Biosciences, Inc., $1.4B
- TSHA – Taysha Gene Therapies Inc, $1.4B
- KARD – Kardigan, Inc. Common Stock, $1.2B
- OMER – Omeros Corporation, $1.2B
- SVRA – Savara Inc, $1B
- MGTX – MeiraGTx Holdings PLC, $955M
- AVBP – ArriVent BioPharma, Inc. Common Stock, $821M
- IMMX – Immix Biopharma Inc, $803M
- IVA – Inventiva Sa, $770M
- WVE – Wave Life Sciences Ltd, $745M
- SLDB – Solid Biosciences LLC, $737M
- DSGN – Design Therapeutics Inc, $732M
- ANNX – Annexon Inc, $726M
- PRME – Prime Medicine, Inc. Common Stock, $707M
- PHAR – Pharming Group NV, $690M
- AURA – Aura Biosciences Inc, $678M
- ZVRA – Zevra Therapeutics Inc., $676M
- DBVT – DBV Technologies, $663M
- IRWD – Ironwood Pharmaceuticals Inc, $620M
- SLN – Silence Therapeutics PLC, $613M
- FLGT – Fulgent Genetics Inc, $591M
- ETRA – Electra Therapeutics, Inc. Common stock, $549M
- DRUG – Bright Minds Biosciences Inc, $506M
- MNPR – Monopar Therapeutics Inc, $498M
- RGNX – Regenxbio Inc, $473M
- KMDA – Kamada, $466M
- OCS – Oculis Holding AG Ordinary shares, $461M
- EDIT – Editas Medicine Inc, $445M
- PRTA – Prothena Corporation plc, $435M
- NGNE – Neurogene Inc, $431M
- RZLT – Rezolute Inc, $396M
- CAPR – Capricor Therapeutics Inc, $384M
- RLMD – Relmada Therapeutics Inc, $347M
- BNTC – Benitec Biopharma Ltd ADR, $335M
- IRD – Opus Genetics, Inc., $318M
- ARCT – Arcturus Therapeutics Holdings Inc, $308M
- FULC – Fulcrum Therapeutics Inc, $279M
- VNDA – Vanda Pharmaceuticals Inc, $273M
- OCGN – Ocugen Inc, $265M
- CUVL – Clinuvel Pharmaceuticals Limited American Depositary Shares, $262M
- ABEO – Abeona Therapeutics Inc, $252M
- IKT – Inhibikase Therapeutics Inc, $251M
- XFOR – X4 Pharmaceuticals Inc, $243M
- RCKT – Rocket Pharmaceuticals Inc, $239M
- SLGL – Sol Gel Technologies Ltd, $227M
- LRMR – Larimar Therapeutics Inc, $224M
- KROS – Keros Therapeutics Inc, $222M
- LXEO – Lexeo Therapeutics, Inc. Common Stock, $217M
- CAMP – CAMP4 THERAPEUTICS CORPORATION, $215M
- PLX – Protalix Biotherapeutics Inc, $210M
- TRDA – Entrada Therapeutics Inc, $209M
- PRQR – ProQR Therapeutics BV, $195M
- ELDN – Eledon Pharmaceuticals Inc, $190M
- SION – Sionna Therapeutics, Inc. Common Stock, $187M
- AKBA – Akebia Ther, $171M
- ANTX – AN2 Therapeutics Inc, $166M
- PEPG – PepGen Ltd, $166M
- CNTN – Canton Strategic Holdings, Inc., $149M
- CRDL – Cardiol Therapeutics Inc Class A, $146M
- KRRO – Korro Bio, Inc., $141M
- TARA – Protara Therapeutics Inc, $137M
- RNA – Atrium Therapeutics, Inc. Common Stock, $130M
- AVXL – Anavex Life Sciences Corp, $121M
- VYGR – Voyager Therapeutics Inc, $117M
- SPRB – Spruce Biosciences, Inc. Common Stock, $116M
- CPIX – Cumberland Pharmaceuticals Inc, $111M
- AARD – Aardvark Therapeutics, Inc. Common Stock, $104M
- PLYX – Polaryx Therapeutics, Inc. Common Stock, $98.2M
- TNXP – Tonix Pharmaceuticals Holding Corp, $93.1M
- UNCY – Unicycive Therapeutics Inc, $92M
- RLYB – Rallybio Corp, $89.9M
- OSTX – OS Therapies Incorporated, $75.8M
- RFL – Rafael Holdings, Inc., $74.4M
- SPRO – Spero Therapeutics Inc, $71.6M
- GANX – Gain Therapeutics Inc, $71.6M
- FBIO – Fortress Biotech Inc, $67.7M
- ATHE – Alterity Therapeutics Ltd, $66.8M
- MREO – Mereo BioPharma Group PLC ADR, $55.9M
- RNTX – Rein Therapeutics Inc, $54.2M
- INMB – INmune Bio Inc, $53.6M
- ELOX – Eloxx Pharmaceuticals, Inc., $53M
- CLNN – Clene Inc., $47M
- ALDX – Aldeyra The, $42.2M
- GRCE – Grace Therapeutics, Inc., $41M
- MGX – Metagenomi, Inc. Common Stock, $37.8M
- IRLA – IRulya Therapeutics Inc., $35M
- FLNA – Filana Therapeutics, Inc., $34M
- QNCX – Quince Therapeutics, Inc., $32.3M
- QNRX – Quoin Pharmaceuticals Ltd DRC, $21.9M
- JSPR – Jasper Therapeutics Inc, $19M
- ATYR – aTyr Pharma, Inc., $16.8M
- KTTA – Pasithea Therapeutics Corp, $14M
- CLRB – Cellectar Biosciences Inc, $13.1M
- SYBX – Synlogic Inc, $13M
- CNSY – Cerenome, Inc., $12.9M
- PASG – Passage Bio Inc, $11.9M
- BLRX – BioLineRx Ltd, $11.7M
- KPRX – Kiora Pharmaceuticals Inc, $10.9M
- LTRN – Lantern Pharma Inc, $9.6M
- BCLI – Brainstorm Cell Therapeutics Inc, $9.2M
- CUBT – Curative Biotechnology Inc, $8.4M
- CMMB – Chemomab Therapeutics Ltd DRC, $8.4M
- SNGX – Soligenix Inc., $7.4M
- JAGX – Jaguar Animal Health Inc, $6.9M
- NRSN – Neurosense Therapeutics Ltd, $5.8M
- PCSA – Processa Pharmaceuticals Inc, $5.1M
- LGVN – Longeveron LLC, $5.1M
- CVKD – Cadrenal Therapeutics, Inc. Common Stock, $4.1M
- KALA – Kala Pharmaceuticals Inc, $3.7M
- JUNS – Jupiter Neurosciences, Inc. Common Stock, $3.4M
- EGRX – Eagle Pharmaceuticals Inc, $3.3M
- MBIO – Mustang Bio Inc, $3.1M
- XRTX – XORTX Therapeutics Inc, $2.3M
- BDRX – Biodexa Pharmaceticals, $860,974
- PXMD – PaxMedica Inc. Common Stock, $88,385
- LIPO – Lipella Pharmaceuticals Inc. Common Stock, $2,310
- VRPX – Virpax Pharmaceuticals Inc, $248
- FIXX – Homology Medicines Inc