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Biotech - Rare Diseases

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207 stocks
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All Stocks (207)

Company Market Cap Price
TECX Tectonic Therapeutic, Inc.
TX2100 development for Hereditary Hemorrhagic Telangiectasia (HHT) aligns TECX with Rare Diseases biotech initiatives.
 $552.32M
$29.46
-0.74%
RLMD Relmada Therapeutics, Inc.
Prader-Willi Syndrome is a rare-disease indication being pursued for Sepranolone.
 $548.54M
$7.48
+6.86%
TRDA Entrada Therapeutics, Inc.
DMD and DM1 programs indicate a focus on rare diseases within the biotech portfolio.
 $534.69M
$13.98
+3.10%
ANNX Annexon, Inc.
GBS and related autoimmune targets place Annexon in Biotech - Rare Diseases.
 $515.37M
$4.67
-6.87%
LXEO Lexeo Therapeutics, Inc. Common Stock
Lead programs target Friedreich ataxia and PKP2 cardiomyopathy, both rare diseases.
 $504.34M
$6.90
+4.86%
KMDA Kamada Ltd.
Inhaled Alpha-1 Antitrypsin therapy for AATD places Kamada in rare-disease biotech therapeutics.
 $488.22M
$8.48
+2.91%
ESPR Esperion Therapeutics, Inc.
Pipeline program for Primary Sclerosing Cholangitis, a rare liver disease.
 $487.69M
$2.04
-0.73%
RGNX REGENXBIO Inc.
RGX-121 targets a rare disease (MPS II) in a late-stage program.
 $480.41M
$9.49
+3.26%
BNTC Benitec Biopharma Inc.
BB-301 targets a rare genetic disease (OPMD), aligning with Rare Diseases in biotech.
 $476.78M
$13.99
+3.94%
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%
FULC Fulcrum Therapeutics, Inc.
Company's focus on sickle cell disease and Diamond-Blackfan anemia positions it in rare genetic diseases.
 $448.64M
$8.29
+3.82%
PRTC PureTech Health plc
IPF program situates PureTech within rare-disease indications.
 $438.14M
$18.09
-0.71%
KYTX Kyverna Therapeutics, Inc.
SPS, MG, and LN are autoimmune conditions with niche/rare-disease focus within Kyverna's pipeline.
 $434.90M
$9.92
+4.09%
NGNE Neurogene Inc.
Rett syndrome is a rare neurological disease target; company focuses on rare diseases.
 $431.39M
$27.85
+8.66%
VNDA Vanda Pharmaceuticals Inc.
VCA-894A and VGT-1849A ASO programs indicate a Rare Diseases focus.
 $429.63M
$7.26
+1.47%
AUTL Autolus Therapeutics plc
Lupus nephritis represents a rare autoimmune indication in the pipeline.
 $425.83M
$1.60
-0.62%
RCKT Rocket Pharmaceuticals, Inc.
Pipeline targets rare genetic diseases (LAD-I, Fanconi anemia, Danon disease, PKP2-ACM, PKD), aligning with a rare diseases focus.
 $423.15M
$3.92
+0.38%
LRMR Larimar Therapeutics, Inc.
Company focuses on Friedreich's ataxia, a rare disease, aligning with Biotech - Rare Diseases.
 $410.83M
$4.81
+1.91%
IRD Opus Genetics, Inc.
Rare disease focus: inherited retinal diseases are rare conditions, aligning with IRD’s target indications.
 $396.54M
$5.77
+0.35%
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
XFOR X4 Pharmaceuticals, Inc.
Company develops therapies targeting rare immune disorders (WHIM syndrome, CN), fitting Biotech - Rare Diseases.
 $375.10M
$4.27
+3.14%
DMAC DiaMedica Therapeutics Inc.
Preeclampsia indication places the program within biotech initiatives targeting rare or high-need diseases.
 $356.73M
$6.86
+3.55%
KROS Keros Therapeutics, Inc.
Duchenne muscular dystrophy (DMD) and associated cytopenias are rare diseases, aligning Keros Therapeutics' focused assets to Rare Diseases.
 $355.54M
$11.68
-0.34%
SLN Silence Therapeutics plc
Company’s focus on rare disease programs (e.g., Divesiran for PV) fits the rare diseases biotech theme.
 $355.32M
$7.53
-0.92%
IMMX Immix Biopharma, Inc.
Relates to a rare orphan disease (AL Amyloidosis), aligning with Rare Diseases.
 $347.53M
$10.35
+3.87%
EDIT Editas Medicine, Inc.
Strategic focus on rare/orphan diseases aligns with the targeted in vivo gene-editing programs.
 $340.69M
$3.49
+4.18%
AVXL Anavex Life Sciences Corp.
Rett syndrome is a rare disease in the pipeline, supporting Rare Diseases biotech tag.
 $325.23M
$3.63
+6.91%
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.
 $320.84M
$3.45
-2.54%
ABEO Abeona Therapeutics Inc.
ABEO's RDEB program and pipeline target rare genetic diseases (rare pediatric/rare skin disorder space).
 $298.05M
$5.52
+2.60%
ALEC Alector, Inc.
FTD-GRN is a rare disease indication, aligning with Rare Diseases Biotech tag.
 $280.52M
$2.57
+0.78%
AVTX Avalo Therapeutics, Inc.
The company references HS as an indication and discusses a rare-disease pipeline emphasis, fitting Biotech - Rare Diseases.
 $261.67M
$14.43
+0.03%
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
ARCT Arcturus Therapeutics Holdings Inc.
CF and OTC deficiency are rare diseases; pipeline targets rare disease indications.
 $251.45M
$8.87
+0.34%
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%
VYGR Voyager Therapeutics, Inc.
Friedreich’s ataxia and Gaucher disease programs via partnerships indicate exposure to Rare Diseases.
 $229.63M
$4.13
+4.29%
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%
RNA Atrium Therapeutics, Inc.
Biotech - Rare Diseases: pipeline targets DM1, FSHD, and DMD, aligning with rare disease focus.
 $223.57M
$14.40
+2.64%
PRQR ProQR Therapeutics N.V.
AX-2402 targets Rett syndrome, placing ProQR in the Rare Diseases biotech focus.
 $214.34M
$2.00
+2.31%
CAMP CAMP4 Therapeutics Corporation
Pipeline targets rare genetic disorders (UCD, SYNGAP-related disorders) indicating focus on rare diseases.
 $210.50M
$4.50
-0.11%
TARA Protara Therapeutics, Inc.
Lymphatic malformations and rare pediatric indications place the company in Rare Diseases.
 $209.91M
$5.44
+6.04%
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.
 $205.79M
$74.82
-0.66%
PLX Protalix BioTherapeutics, Inc.
Company focus and pipeline are centered on rare diseases, including Gaucher and Fabry disease, with plans in rare renal indications.
 $186.58M
$2.31
+0.43%
RNAC Cartesian Therapeutics, Inc.
Pipeline targets or includes rare autoimmune/related indications, aligning with Rare Diseases.
 $183.07M
$7.03
+10.19%
TLSA Tiziana Life Sciences Ltd
MSA, na-SPMS, and ALS represent rare/neurodegenerative indications targeted by TLSA, aligning with Rare Diseases.
 $169.24M
$1.25
-7.09%
TNXP Tonix Pharmaceuticals Holding Corp.
TNX-2900 targets Prader-Willi syndrome, representing a rare-disease program within Tonix's portfolio.
 $164.64M
$13.96
-1.17%
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%
IKT Inhibikase Therapeutics, Inc.
PAH is a rare disease indication and the company is pursuing therapy development in this orphan space.
 $137.57M
$1.81
+6.76%
TNYA Tenaya Therapeutics, Inc.
TN-201/TN-401 target rare genetic cardiomyopathies, aligning with the rare diseases biotech focus.
 $130.22M
$0.78
+4.87%
PEPG PepGen Inc.
DM1 is a rare genetic disease, placing PepGen in Biotech - Rare Diseases as a core focus area.
 $116.87M
$1.72
+2.69%
KRRO Korro Bio, Inc.
AATD and rare metabolic disease programs position KRRO within rare disease biotech indications.
 $113.91M
$12.18
+0.66%
AARD Aardvark Therapeutics, Inc. Common Stock
Aardvark focuses on Prader-Willi Syndrome and related rare diseases, supported by Orphan Drug Designation.
 $113.87M
$5.24
+5.01%
ALDX Aldeyra Therapeutics, Inc.
ADX2191 targets rare retinal diseases; pipeline emphasis on rare diseases.
 $108.29M
$1.81
+0.56%
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%
DTIL Precision BioSciences, Inc.
PBGENE-DMD targets Duchenne muscular dystrophy, a rare genetic disease, aligning with the company's rare-disease pipeline program.
 $104.46M
$7.89
+4.85%
NTHI Neonc Technologies Holdings, Inc.
Rare Pediatric Disease designation aligns with pediatric brain tumor program (NEO100 pediatric).
 $103.99M
$5.29
+5.38%
CRDL Cardiol Therapeutics Inc.
Recurrent pericarditis is presented as a rare-disease indication with Phase III development.
 $103.34M
$1.24
+11.71%
COYA Coya Therapeutics, Inc.
ALS and Frontotemporal Dementia programs position COYA within rare neurodegenerative diseases.
 $100.02M
$4.79
+6.92%
ANTX AN2 Therapeutics, Inc.
Chagas disease program places the company in the rare diseases biotech space.
 $98.92M
$3.61
+14.97%
SGMO Sangamo Therapeutics, Inc.
Fabry disease program ST-920 targets a rare disease with accelerated approval pathway.
 $88.16M
$0.25
-7.96%
ATYR aTyr Pharma, Inc.
Sarcoidosis and SSc-ILD are rare diseases, and the company’s lead programs target these ILD indications.
 $83.62M
$0.86
+3.01%
PLRX Pliant Therapeutics, Inc.
PLN-101325 targets muscular dystrophies, placing the company in the rare diseases biotech space.
 $81.73M
$1.34
+3.46%
ATHE Alterity Therapeutics Limited
MSA and other neurodegenerative diseases fall under Rare Diseases, a notable focus in Alterity's pipeline.
 $81.60M
$4.34
+9.32%
GANX Gain Therapeutics, Inc.
GT-02287 and alpha-1 antitrypsin deficiency program indicate a focus on rare diseases within biotech.
 $76.89M
$2.14
+4.39%
FBIO Fortress Biotech, Inc.
Asset focus on rare disease programs (e.g., copper histidinate for Menkes disease) fits Biotech - Rare Diseases.
 $73.25M
$2.35
+0.21%
RFL Rafael Holdings, Inc.
NPC1 rare-disease program (Trappsol Cyclo) places Rafael Holdings in the Biotech - Rare Diseases category.
 $65.22M
$1.28
+4.92%
CLNN Clene Inc.
ALS/MS pipeline involves rare-disease CNS targets, aligning with a rare diseases biotech focus.
 $65.21M
$6.40
+0.79%
INO Inovio Pharmaceuticals, Inc.
RRP is a rare disease targeted by INO-3107, placing the company in the Rare Diseases segment.
 $63.75M
$1.20
+3.02%
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.
 $60.61M
$4.29
+4.13%
MGX Metagenomi, Inc. Common Stock
Hemophilia A is a rare disease; MGX's lead program targets rare disease indications through gene editing.
 $55.57M
$1.48
+2.78%
KZR Kezar Life Sciences, Inc.
Autoimmune hepatitis is a rare disease area, aligning with the Rare Diseases investable theme.
 $53.68M
$7.33
-0.47%
MREO Mereo BioPharma Group plc
Mereo's core focus is developing therapeutics for rare diseases, fitting the Biotech - Rare Diseases category.
 $53.56M
$0.33
+2.64%
TELO Telomir Pharmaceuticals, Inc. Common Stock
Preclinical work includes models for Werner Syndrome and Progeria, placing the company in the rare diseases biotech space.
 $49.85M
$1.46
+8.96%
CPIX Cumberland Pharmaceuticals Inc.
DMD cardiomyopathy orphan/rare pediatric disease designations place the pipeline in the rare diseases biotech space.
 $48.31M
$3.25
+5.18%
RLYB Rallybio Corporation
Core focus on rare diseases with lead and preclinical assets addressing complement dysregulation and iron overload.
 $43.41M
$8.20
-1.62%
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%
INMB INmune Bio, Inc.
CORDStrom MSC therapy for RDEB places INmune Bio in the Rare Diseases biotech arena.
 $38.55M
$1.45
+8.21%
ATRA Atara Biotherapeutics, Inc.
Ebvallo targets EBV-related diseases which are rare.
 $38.00M
$5.32
+1.53%
PASG Passage Bio, Inc.
FTD-GRN and related neurodegenerative indications are rare diseases targeted by Passage Bio's pipeline.
 $37.10M
$12.02
+2.91%
CRVO CervoMed Inc.
DLB/FTD indications and FDA orphan designation place the program within Rare Diseases biotech.
 $36.83M
$4.00
+0.25%
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.
 $34.94M
$6.87
-16.52%
RNTX Rein Therapeutics Inc.
IPF/related fibrotic orphan indications classify Rein's lead assets as addressing a rare disease space.
 $26.57M
$1.16
-3.33%
BRNS Barinthus Biotherapeutics plc
Celias disease represents a rare autoimmune indication, aligning with Rare Diseases biotechnology investment theme.
 $24.09M
$0.62
+4.91%
LGVN Longeveron Inc.
HLHS and pediatric DCM programs place the company in Biotech - Rare Diseases.
 $23.47M
$1.09
-0.45%
JUNS Jupiter Neurosciences, Inc.
Biotech - Rare Diseases: Pipeline includes therapies for Friedreich's Ataxia, MELAS, and other rare conditions.
 $15.05M
$0.43
+7.70%
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.
 $14.83M
$0.11
+4.37%
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.
 $12.79M
$2.43
+1.67%
SNGX Soligenix, Inc.
BD (Behçet's Disease) and other rare-disease programs classify Soligenix under Biotech - Rare Diseases.
 $12.61M
$1.25
+6.41%
LIPO Lipella Pharmaceuticals Inc.
LP-410 has FDA Orphan Drug Designation and targets rare mucosal/immune-mediated diseases like GVHD.
 $12.20M
$0.31
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.
 $11.66M
$1.00
-1.96%
CVKD Cadrenal Therapeutics, Inc. Common Stock
The program receives orphan drug designations for niche indications, aligning with the Rare Diseases tag.
 $10.84M
$5.50
+13.40%
QNRX Quoin Pharmaceuticals, Ltd.
Lead program targets a rare disease (Netherton Syndrome) and is developed by a biotech focused on rare diseases.
 $9.48M
$6.42
-2.43%
KPRX Kiora Pharmaceuticals, Inc.
Kiora's lead programs target rare retinal diseases with orphan drug designations, aligning with the Rare Diseases biotech investment theme.
 $9.27M
$2.55
+2.41%
AIMD Ainos, Inc.
VELDONA human therapeutic programs target rare diseases, aligning with Biotech - Rare Diseases.
 $7.59M
$1.69
+3.68%
SYBX Synlogic, Inc.
PKU is a rare metabolic disease and Synlogic's lead programs target rare diseases via a synthetic biology platform.
 $7.42M
$1.05
+65.48%
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.
 $7.02M
$3.11
-2.20%
ICU SeaStar Medical Holding Corporation
Pediatric/rare-disease indications for QUELIMMUNE position the company within Rare Diseases.
 $6.64M
$2.36
-0.84%
KTTA Pasithea Therapeutics Corp.
There is potential expansion into inflammatory diseases and rare conditions (e.g., NF1), suggesting relevance to rare-disease indications.
 $6.56M
$0.85
+1.50%
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