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Biotech - Rare Diseases

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207 stocks
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All Stocks (207)

Company Market Cap Price
ANNX Annexon, Inc.
GBS and related autoimmune targets place Annexon in Biotech - Rare Diseases.
 $515.37M
$4.67
-6.87%
PRTA Prothena Corporation plc
Coramitug/ATTR amyloidosis represents a rare disease program, aligning with Prothena's rare disease portfolio.
 $503.31M
$9.36
+1.03%
OCGN Ocugen, Inc.
RP, Stargardt disease, and geographic atrophy are rare ocular diseases, aligning with the Rare Diseases tag.
 $494.00M
$1.69
+4.32%
KMDA Kamada Ltd.
Inhaled Alpha-1 Antitrypsin therapy for AATD places Kamada in rare-disease biotech therapeutics.
 $469.82M
$8.18
-0.49%
ZVRA Zevra Therapeutics, Inc.
Zevra focuses on rare-disease therapeutics (NPC, UCD, VEDS) and related pipeline assets, core to rare disease biotech.
 $466.48M
$8.48
+2.05%
PRTC PureTech Health plc
IPF program situates PureTech within rare-disease indications.
 $459.99M
$19.23
+0.29%
VNDA Vanda Pharmaceuticals Inc.
VCA-894A and VGT-1849A ASO programs indicate a Rare Diseases focus.
 $441.40M
$7.47
+0.34%
AVXL Anavex Life Sciences Corp.
Rett syndrome is a rare disease in the pipeline, supporting Rare Diseases biotech tag.
 $430.33M
$5.01
+3.40%
SLDB Solid Biosciences Inc.
The programs target rare diseases (DMD, Friedreich's ataxia, CPVT), aligning with Biotech - Rare Diseases.
 $425.94M
$5.49
-3.00%
TRDA Entrada Therapeutics, Inc.
DMD and DM1 programs indicate a focus on rare diseases within the biotech portfolio.
 $418.41M
$11.00
+1.66%
ETON Eton Pharmaceuticals, Inc.
ETON's strategic focus on ultra-rare diseases and a portfolio of therapies for rare conditions aligns with the Biotech - Rare Diseases investable theme.
 $406.02M
$15.15
-1.69%
LXEO Lexeo Therapeutics, Inc. Common Stock
Lead programs target Friedreich ataxia and PKP2 cardiomyopathy, both rare diseases.
 $399.07M
$7.39
-1.47%
RCKT Rocket Pharmaceuticals, Inc.
Pipeline targets rare genetic diseases (LAD-I, Fanconi anemia, Danon disease, PKP2-ACM, PKD), aligning with a rare diseases focus.
 $392.77M
$3.65
-1.75%
DMAC DiaMedica Therapeutics Inc.
Preeclampsia indication places the program within biotech initiatives targeting rare or high-need diseases.
 $391.54M
$7.61
-10.95%
MNPR Monopar Therapeutics Inc.
ALXN1840 (Wilson disease) in-licensing expands Monopar's rare disease pipeline, aligning with Biotech - Rare Diseases.
 $380.87M
$56.59
-8.33%
VIGL Vigil Neuroscience, Inc.
ALSP is a rare disease with a microglial dysfunction mechanism, placing Vigil in Biotech - Rare Diseases.
 $375.71M
$8.05
AUTL Autolus Therapeutics plc
Lupus nephritis represents a rare autoimmune indication in the pipeline.
 $367.28M
$1.37
-4.86%
FDMT 4D Molecular Therapeutics, Inc.
FDMT's CF program (4D-710) targets a rare disease indication, aligning with the Rare Diseases Biotech theme.
 $352.60M
$7.53
-1.12%
TECX Tectonic Therapeutic, Inc.
TX2100 development for Hereditary Hemorrhagic Telangiectasia (HHT) aligns TECX with Rare Diseases biotech initiatives.
 $351.79M
$18.86
-3.36%
KYTX Kyverna Therapeutics, Inc.
SPS, MG, and LN are autoimmune conditions with niche/rare-disease focus within Kyverna's pipeline.
 $351.59M
$7.80
-4.06%
AARD Aardvark Therapeutics, Inc. Common Stock
Aardvark focuses on Prader-Willi Syndrome and related rare diseases, supported by Orphan Drug Designation.
 $333.48M
$15.36
+0.03%
BNTC Benitec Biopharma Inc.
BB-301 targets a rare genetic disease (OPMD), aligning with Rare Diseases in biotech.
 $306.61M
$11.69
-0.17%
ALDX Aldeyra Therapeutics, Inc.
ADX2191 targets rare retinal diseases; pipeline emphasis on rare diseases.
 $277.32M
$4.61
+4.54%
LRMR Larimar Therapeutics, Inc.
Company focuses on Friedreich's ataxia, a rare disease, aligning with Biotech - Rare Diseases.
 $267.90M
$3.12
-4.14%
ABEO Abeona Therapeutics Inc.
ABEO's RDEB program and pipeline target rare genetic diseases (rare pediatric/rare skin disorder space).
 $260.49M
$5.05
-0.49%
INZY Inozyme Pharma, Inc.
Inozyme focuses on ENPP1 Deficiency, a rare disease, with INZ-701 as a lead therapy, making it a dedicated rare-disease biopharma company.
 $256.96M
$4.00
NGNE Neurogene Inc.
Rett syndrome is a rare neurological disease target; company focuses on rare diseases.
 $250.47M
$17.59
-2.60%
RZLT Rezolute, Inc.
HI is a rare disease; the therapy targets rare pediatric congenital HI and tumor HI indications, aligning with Rare Diseases biotech focus.
 $247.92M
$2.73
-3.02%
GNFT Genfit S.A.
VS-01 and other programs in rare diseases position GENFIT within the Biotech - Rare Diseases space.
 $242.48M
$4.90
+1.03%
SLN Silence Therapeutics plc
Company’s focus on rare disease programs (e.g., Divesiran for PV) fits the rare diseases biotech theme.
 $237.82M
$5.04
-9.35%
QNCX Quince Therapeutics, Inc.
Lead asset targets Ataxia-Telangiectasia, a rare disease, aligning Quince with the Rare Diseases biotech investment theme.
 $226.67M
$4.28
-2.73%
VYGR Voyager Therapeutics, Inc.
Friedreich’s ataxia and Gaucher disease programs via partnerships indicate exposure to Rare Diseases.
 $208.56M
$3.77
-1.44%
AVTX Avalo Therapeutics, Inc.
The company references HS as an indication and discusses a rare-disease pipeline emphasis, fitting Biotech - Rare Diseases.
 $198.07M
$15.03
-3.90%
SLGL Sol-Gel Technologies Ltd.
SGT-610 and SGT-210 target rare dermatologic diseases (Gorlin syndrome, Darier disease), aligning with the Rare Diseases investable theme.
 $193.61M
$68.59
+0.41%
TARA Protara Therapeutics, Inc.
Lymphatic malformations and rare pediatric indications place the company in Rare Diseases.
 $193.30M
$5.01
-0.50%
RNAC Cartesian Therapeutics, Inc.
Pipeline targets or includes rare autoimmune/related indications, aligning with Rare Diseases.
 $192.94M
$7.41
+0.68%
ALEC Alector, Inc.
FTD-GRN is a rare disease indication, aligning with Rare Diseases Biotech tag.
 $186.23M
$1.84
+1.10%
EDIT Editas Medicine, Inc.
Strategic focus on rare/orphan diseases aligns with the targeted in vivo gene-editing programs.
 $178.94M
$2.04
+2.76%
TLSA Tiziana Life Sciences Ltd
MSA, na-SPMS, and ALS represent rare/neurodegenerative indications targeted by TLSA, aligning with Rare Diseases.
 $177.91M
$1.68
+15.52%
PRQR ProQR Therapeutics N.V.
AX-2402 targets Rett syndrome, placing ProQR in the Rare Diseases biotech focus.
 $177.72M
$1.67
+5.38%
PEPG PepGen Inc.
DM1 is a rare genetic disease, placing PepGen in Biotech - Rare Diseases as a core focus area.
 $170.56M
$5.21
+4.51%
ARCT Arcturus Therapeutics Holdings Inc.
CF and OTC deficiency are rare diseases; pipeline targets rare disease indications.
 $169.72M
$6.21
+1.22%
NTHI Neonc Technologies Holdings, Inc.
Rare Pediatric Disease designation aligns with pediatric brain tumor program (NEO100 pediatric).
 $167.26M
$8.82
+1.38%
THTX Theratechnologies Inc.
Partnerships targeting rare diseases (donidalorsen, olezarsen) position Theratechnologies in the rare-diseases space.
 $155.87M
$3.39
SAVA Cassava Sciences, Inc.
Tuberous Sclerosis Complex (TSC)-related epilepsy is a rare disease area, aligning with Rare Diseases biotech.
 $154.10M
$3.23
+1.10%
TNXP Tonix Pharmaceuticals Holding Corp.
TNX-2900 targets Prader-Willi syndrome, representing a rare-disease program within Tonix's portfolio.
 $150.25M
$17.13
+7.74%
GANX Gain Therapeutics, Inc.
GT-02287 and alpha-1 antitrypsin deficiency program indicate a focus on rare diseases within biotech.
 $148.83M
$4.01
-3.02%
PLX Protalix BioTherapeutics, Inc.
Company focus and pipeline are centered on rare diseases, including Gaucher and Fabry disease, with plans in rare renal indications.
 $143.52M
$1.82
+1.39%
IMMX Immix Biopharma, Inc.
Relates to a rare orphan disease (AL Amyloidosis), aligning with Rare Diseases.
 $135.52M
$4.72
+0.21%
IRD Opus Genetics, Inc.
Rare disease focus: inherited retinal diseases are rare conditions, aligning with IRD’s target indications.
 $125.81M
$2.11
+1.44%
IKT Inhibikase Therapeutics, Inc.
PAH is a rare disease indication and the company is pursuing therapy development in this orphan space.
 $124.44M
$1.68
+1.52%
RLMD Relmada Therapeutics, Inc.
Prader-Willi Syndrome is a rare-disease indication being pursued for Sepranolone.
 $124.14M
$3.75
-1.45%
ATRA Atara Biotherapeutics, Inc.
Ebvallo targets EBV-related diseases which are rare.
 $123.46M
$17.70
+0.71%
SGMO Sangamo Therapeutics, Inc.
Fabry disease program ST-920 targets a rare disease with accelerated approval pathway.
 $121.11M
$0.39
-8.44%
TNYA Tenaya Therapeutics, Inc.
TN-201/TN-401 target rare genetic cardiomyopathies, aligning with the rare diseases biotech focus.
 $118.89M
$0.72
-2.17%
CAMP CAMP4 Therapeutics Corporation
Pipeline targets rare genetic disorders (UCD, SYNGAP-related disorders) indicating focus on rare diseases.
 $113.31M
$5.66
-2.08%
FBIO Fortress Biotech, Inc.
Asset focus on rare disease programs (e.g., copper histidinate for Menkes disease) fits Biotech - Rare Diseases.
 $110.69M
$3.73
+7.04%
UNCY Unicycive Therapeutics, Inc.
Pipeline focuses on kidney disease therapies, a disease-focused biotech area that aligns with rare/low-prevalence or specialized renal indications.
 $106.15M
$6.21
+3.41%
MREO Mereo BioPharma Group plc
Mereo's core focus is developing therapeutics for rare diseases, fitting the Biotech - Rare Diseases category.
 $101.82M
$0.64
-4.64%
COYA Coya Therapeutics, Inc.
ALS and Frontotemporal Dementia programs position COYA within rare neurodegenerative diseases.
 $89.65M
$5.36
-0.09%
CRDL Cardiol Therapeutics Inc.
Recurrent pericarditis is presented as a rare-disease indication with Phase III development.
 $87.64M
$1.06
+10.59%
INO Inovio Pharmaceuticals, Inc.
RRP is a rare disease targeted by INO-3107, placing the company in the Rare Diseases segment.
 $84.50M
$1.57
-0.94%
KRRO Korro Bio, Inc.
AATD and rare metabolic disease programs position KRRO within rare disease biotech indications.
 $82.45M
$8.78
-0.23%
PLRX Pliant Therapeutics, Inc.
PLN-101325 targets muscular dystrophies, placing the company in the rare diseases biotech space.
 $79.19M
$1.29
+1.17%
CLNN Clene Inc.
ALS/MS pipeline involves rare-disease CNS targets, aligning with a rare diseases biotech focus.
 $72.13M
$7.22
CPIX Cumberland Pharmaceuticals Inc.
DMD cardiomyopathy orphan/rare pediatric disease designations place the pipeline in the rare diseases biotech space.
 $69.56M
$4.68
-1.68%
ATYR aTyr Pharma, Inc.
Sarcoidosis and SSc-ILD are rare diseases, and the company’s lead programs target these ILD indications.
 $69.43M
$0.71
+0.03%
CRVO CervoMed Inc.
DLB/FTD indications and FDA orphan designation place the program within Rare Diseases biotech.
 $67.36M
$7.30
+3.18%
MGX Metagenomi, Inc. Common Stock
Hemophilia A is a rare disease; MGX's lead program targets rare disease indications through gene editing.
 $65.31M
$1.78
+2.30%
RFL Rafael Holdings, Inc.
NPC1 rare-disease program (Trappsol Cyclo) places Rafael Holdings in the Biotech - Rare Diseases category.
 $64.20M
$1.24
GRCE Grace Therapeutics, Inc.
Grace Therapeutics' assets target rare diseases and all pipeline candidates have FDA Orphan Drug Designation, aligning with the 'Biotech - Rare Diseases' theme.
 $50.47M
$3.67
+0.69%
DTIL Precision BioSciences, Inc.
PBGENE-DMD targets Duchenne muscular dystrophy, a rare genetic disease, aligning with the company's rare-disease pipeline program.
 $47.74M
$4.08
+4.49%
INMB INmune Bio, Inc.
CORDStrom MSC therapy for RDEB places INmune Bio in the Rare Diseases biotech arena.
 $45.99M
$1.74
+6.75%
KZR Kezar Life Sciences, Inc.
Autoimmune hepatitis is a rare disease area, aligning with the Rare Diseases investable theme.
 $45.40M
$6.17
-0.48%
TELO Telomir Pharmaceuticals, Inc. Common Stock
Preclinical work includes models for Werner Syndrome and Progeria, placing the company in the rare diseases biotech space.
 $44.87M
$1.40
+1.82%
XFOR X4 Pharmaceuticals, Inc.
Company develops therapies targeting rare immune disorders (WHIM syndrome, CN), fitting Biotech - Rare Diseases.
 $42.21M
$3.69
+1.93%
PASG Passage Bio, Inc.
FTD-GRN and related neurodegenerative indications are rare diseases targeted by Passage Bio's pipeline.
 $40.91M
$12.72
-10.92%
FGEN FibroGen, Inc.
Roxadustat for anemia in lower-risk MDS positions the program within Biotech - Rare Diseases as a potential orphan/disease-focused asset.
 $39.28M
$9.53
+5.60%
ATHE Alterity Therapeutics Limited
MSA and other neurodegenerative diseases fall under Rare Diseases, a notable focus in Alterity's pipeline.
 $36.71M
$3.46
+4.53%
ANTX AN2 Therapeutics, Inc.
Chagas disease program places the company in the rare diseases biotech space.
 $32.79M
$1.22
-2.80%
JUNS Jupiter Neurosciences, Inc.
Biotech - Rare Diseases: Pipeline includes therapies for Friedreich's Ataxia, MELAS, and other rare conditions.
 $29.91M
$0.87
+0.74%
BRNS Barinthus Biotherapeutics plc
Celias disease represents a rare autoimmune indication, aligning with Rare Diseases biotechnology investment theme.
 $29.52M
$0.72
-0.68%
RNTX Rein Therapeutics Inc.
IPF/related fibrotic orphan indications classify Rein's lead assets as addressing a rare disease space.
 $28.20M
$1.23
+6.03%
RLYB Rallybio Corporation
Core focus on rare diseases with lead and preclinical assets addressing complement dysregulation and iron overload.
 $27.45M
$0.65
-4.05%
ATHA Athira Pharma, Inc.
ALS is a rare neurodegenerative disease and Athira's focus on HGF/MET small molecules for ALS aligns with the 'Biotech - Rare Diseases' investable theme.
 $24.06M
$6.87
+8.45%
CVKD Cadrenal Therapeutics, Inc. Common Stock
The program receives orphan drug designations for niche indications, aligning with the Rare Diseases tag.
 $16.11M
$8.11
-1.10%
APLT Applied Therapeutics, Inc.
Applied Therapeutics is a biotech focused on rare diseases, with lead programs in rare metabolic/neurologic conditions (e.g., govorestat for CMT-SORD) and out-licensing efforts in rare disease assets.
 $14.44M
$0.09
-7.36%
TPST Tempest Therapeutics, Inc.
TPST-1495 is being developed for Familial Adenomatous Polyposis (rare disease) with regulatory/academic support.
 $13.81M
$3.15
-0.16%
BLRX BioLineRx Ltd.
The company plans to in-license early clinical-stage assets in oncology and rare diseases, signaling a pipeline expansion into rare diseases.
 $13.65M
$2.91
+1.04%
LIPO Lipella Pharmaceuticals Inc.
LP-410 has FDA Orphan Drug Designation and targets rare mucosal/immune-mediated diseases like GVHD.
 $12.20M
$0.31
SYBX Synlogic, Inc.
PKU is a rare metabolic disease and Synlogic's lead programs target rare diseases via a synthetic biology platform.
 $12.17M
$1.05
+0.96%
GDTC CytoMed Therapeutics Limited
Diversification into auto-immune diseases via cord blood-derived NK cells; autoimmune disease therapies fall under Rare Diseases category in some classifications.
 $11.00M
$0.94
+1.08%
QNRX Quoin Pharmaceuticals, Ltd.
Lead program targets a rare disease (Netherton Syndrome) and is developed by a biotech focused on rare diseases.
 $10.57M
$10.82
+1.55%
XAIR Beyond Air, Inc.
BA-102 in NeuroNOS has Orphan Drug Designation, aligning with rare-disease biotech development.
 $8.88M
$1.72
-0.29%
AIMD Ainos, Inc.
VELDONA human therapeutic programs target rare diseases, aligning with Biotech - Rare Diseases.
 $8.85M
$1.96
+1.55%
LGVN Longeveron Inc.
HLHS and pediatric DCM programs place the company in Biotech - Rare Diseases.
 $8.38M
$0.56
-1.22%
KPRX Kiora Pharmaceuticals, Inc.
Kiora's lead programs target rare retinal diseases with orphan drug designations, aligning with the Rare Diseases biotech investment theme.
 $7.42M
$2.19
-0.23%
PCSA Processa Pharmaceuticals, Inc.
The re-evaluation of PCS499 for rare primary glomerular diseases (PGDs) and PCS12852 for gastroparesis broadens Processa's portfolio into Rare Diseases biotech.
 $7.13M
$3.57
-3.77%
ICU SeaStar Medical Holding Corporation
Pediatric/rare-disease indications for QUELIMMUNE position the company within Rare Diseases.
 $7.06M
$2.55
-0.78%
KTTA Pasithea Therapeutics Corp.
There is potential expansion into inflammatory diseases and rare conditions (e.g., NF1), suggesting relevance to rare-disease indications.
 $6.89M
$0.92
+3.34%
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