Biotech - Rare Diseases
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All Stocks (207)
| Company | Market Cap | Price |
|---|---|---|
|
DSGN
Design Therapeutics, Inc.
Pipeline targets rare genetic diseases (FA, FECD, DM1, HD), fitting Rare Diseases in biotech.
|
$537.02M |
$9.14
-3.08%
|
|
LXEO
Lexeo Therapeutics, Inc. Common Stock
Lead programs target Friedreich ataxia and PKP2 cardiomyopathy, both rare diseases.
|
$527.59M |
$9.86
+0.87%
|
|
FDMT
4D Molecular Therapeutics, Inc.
FDMT's CF program (4D-710) targets a rare disease indication, aligning with the Rare Diseases Biotech theme.
|
$506.25M |
$10.96
+1.15%
|
|
DMAC
DiaMedica Therapeutics Inc.
Preeclampsia indication places the program within biotech initiatives targeting rare or high-need diseases.
|
$485.36M |
$8.61
-8.36%
|
|
ZVRA
Zevra Therapeutics, Inc.
Zevra focuses on rare-disease therapeutics (NPC, UCD, VEDS) and related pipeline assets, core to rare disease biotech.
|
$467.61M |
$8.15
-2.16%
|
|
MNPR
Monopar Therapeutics Inc.
ALXN1840 (Wilson disease) in-licensing expands Monopar's rare disease pipeline, aligning with Biotech - Rare Diseases.
|
$460.31M |
$76.04
+1.92%
|
|
SLDB
Solid Biosciences Inc.
The programs target rare diseases (DMD, Friedreich's ataxia, CPVT), aligning with Biotech - Rare Diseases.
|
$457.09M |
$5.76
-1.87%
|
|
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.
|
$447.85M |
$15.89
-4.82%
|
|
PRTC
PureTech Health plc
IPF program situates PureTech within rare-disease indications.
|
$436.16M |
$16.78
-0.77%
|
|
AUTL
Autolus Therapeutics plc
Lupus nephritis represents a rare autoimmune indication in the pipeline.
|
$428.49M |
$1.64
+1.55%
|
|
TRDA
Entrada Therapeutics, Inc.
DMD and DM1 programs indicate a focus on rare diseases within the biotech portfolio.
|
$421.45M |
$11.13
+0.45%
|
|
KMDA
Kamada Ltd.
Inhaled Alpha-1 Antitrypsin therapy for AATD places Kamada in rare-disease biotech therapeutics.
|
$403.51M |
$7.23
+2.99%
|
|
OCGN
Ocugen, Inc.
RP, Stargardt disease, and geographic atrophy are rare ocular diseases, aligning with the Rare Diseases tag.
|
$397.53M |
$1.41
+3.31%
|
|
KYTX
Kyverna Therapeutics, Inc.
SPS, MG, and LN are autoimmune conditions with niche/rare-disease focus within Kyverna's pipeline.
|
$379.70M |
$10.73
+22.21%
|
|
DRUG
Bright Minds Biosciences Inc.
Lead indications (Absence Epilepsy and DEE) are rare neurological disorders.
|
$375.81M |
$85.13
+1.12%
|
|
VIGL
Vigil Neuroscience, Inc.
ALSP is a rare disease with a microglial dysfunction mechanism, placing Vigil in Biotech - Rare Diseases.
|
$375.71M |
$8.05
|
|
TECX
Tectonic Therapeutic, Inc.
TX2100 development for Hereditary Hemorrhagic Telangiectasia (HHT) aligns TECX with Rare Diseases biotech initiatives.
|
$369.75M |
$18.45
-6.63%
|
|
VNDA
Vanda Pharmaceuticals Inc.
VCA-894A and VGT-1849A ASO programs indicate a Rare Diseases focus.
|
$368.13M |
$6.52
+4.65%
|
|
RCKT
Rocket Pharmaceuticals, Inc.
Pipeline targets rare genetic diseases (LAD-I, Fanconi anemia, Danon disease, PKP2-ACM, PKD), aligning with a rare diseases focus.
|
$365.79M |
$3.46
+2.21%
|
|
LRMR
Larimar Therapeutics, Inc.
Company focuses on Friedreich's ataxia, a rare disease, aligning with Biotech - Rare Diseases.
|
$346.64M |
$3.94
-2.59%
|
|
AVXL
Anavex Life Sciences Corp.
Rett syndrome is a rare disease in the pipeline, supporting Rare Diseases biotech tag.
|
$345.29M |
$4.02
|
|
MREO
Mereo BioPharma Group plc
Mereo's core focus is developing therapeutics for rare diseases, fitting the Biotech - Rare Diseases category.
|
$345.24M |
$2.21
+1.61%
|
|
BNTC
Benitec Biopharma Inc.
BB-301 targets a rare genetic disease (OPMD), aligning with Rare Diseases in biotech.
|
$314.48M |
$12.22
+2.00%
|
|
AARD
Aardvark Therapeutics, Inc. Common Stock
Aardvark focuses on Prader-Willi Syndrome and related rare diseases, supported by Orphan Drug Designation.
|
$313.09M |
$14.76
+2.25%
|
|
NGNE
Neurogene Inc.
Rett syndrome is a rare neurological disease target; company focuses on rare diseases.
|
$298.57M |
$20.60
-1.53%
|
|
SLN
Silence Therapeutics plc
Company’s focus on rare disease programs (e.g., Divesiran for PV) fits the rare diseases biotech theme.
|
$298.10M |
$6.61
+4.59%
|
|
ABEO
Abeona Therapeutics Inc.
ABEO's RDEB program and pipeline target rare genetic diseases (rare pediatric/rare skin disorder space).
|
$274.85M |
$5.07
-5.50%
|
|
ALDX
Aldeyra Therapeutics, Inc.
ADX2191 targets rare retinal diseases; pipeline emphasis on rare diseases.
|
$271.93M |
$4.19
-7.71%
|
|
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
|
|
AVTX
Avalo Therapeutics, Inc.
The company references HS as an indication and discusses a rare-disease pipeline emphasis, fitting Biotech - Rare Diseases.
|
$244.63M |
$18.09
-2.72%
|
|
VYGR
Voyager Therapeutics, Inc.
Friedreich’s ataxia and Gaucher disease programs via partnerships indicate exposure to Rare Diseases.
|
$243.51M |
$4.26
-2.85%
|
|
GNFT
Genfit S.A.
VS-01 and other programs in rare diseases position GENFIT within the Biotech - Rare Diseases space.
|
$242.05M |
$4.90
+1.03%
|
|
EDIT
Editas Medicine, Inc.
Strategic focus on rare/orphan diseases aligns with the targeted in vivo gene-editing programs.
|
$227.50M |
$2.46
-2.57%
|
|
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%
|
|
RNAC
Cartesian Therapeutics, Inc.
Pipeline targets or includes rare autoimmune/related indications, aligning with Rare Diseases.
|
$221.54M |
$9.06
+6.40%
|
|
TARA
Protara Therapeutics, Inc.
Lymphatic malformations and rare pediatric indications place the company in Rare Diseases.
|
$219.92M |
$5.51
-3.33%
|
|
IMMX
Immix Biopharma, Inc.
Relates to a rare orphan disease (AL Amyloidosis), aligning with Rare Diseases.
|
$203.86M |
$6.19
-12.45%
|
|
ARCT
Arcturus Therapeutics Holdings Inc.
CF and OTC deficiency are rare diseases; pipeline targets rare disease indications.
|
$202.57M |
$7.37
-1.27%
|
|
PRQR
ProQR Therapeutics N.V.
AX-2402 targets Rett syndrome, placing ProQR in the Rare Diseases biotech focus.
|
$196.30M |
$2.46
+5.36%
|
|
NTHI
Neonc Technologies Holdings, Inc.
Rare Pediatric Disease designation aligns with pediatric brain tumor program (NEO100 pediatric).
|
$195.61M |
$10.09
+7.68%
|
|
PEPG
PepGen Inc.
DM1 is a rare genetic disease, placing PepGen in Biotech - Rare Diseases as a core focus area.
|
$185.65M |
$5.47
-3.36%
|
|
TLSA
Tiziana Life Sciences Ltd
MSA, na-SPMS, and ALS represent rare/neurodegenerative indications targeted by TLSA, aligning with Rare Diseases.
|
$183.50M |
$1.39
-22.19%
|
|
TNXP
Tonix Pharmaceuticals Holding Corp.
TNX-2900 targets Prader-Willi syndrome, representing a rare-disease program within Tonix's portfolio.
|
$169.01M |
$18.98
-1.56%
|
|
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.
|
$163.46M |
$1.62
-10.00%
|
|
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%
|
|
ALEC
Alector, Inc.
FTD-GRN is a rare disease indication, aligning with Rare Diseases Biotech tag.
|
$150.81M |
$1.46
-2.35%
|
|
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%
|
|
IRD
Opus Genetics, Inc.
Rare disease focus: inherited retinal diseases are rare conditions, aligning with IRD’s target indications.
|
$141.38M |
$2.19
-7.20%
|
|
TNYA
Tenaya Therapeutics, Inc.
TN-201/TN-401 target rare genetic cardiomyopathies, aligning with the rare diseases biotech focus.
|
$138.53M |
$0.80
-5.87%
|
|
SGMO
Sangamo Therapeutics, Inc.
Fabry disease program ST-920 targets a rare disease with accelerated approval pathway.
|
$135.77M |
$0.45
-0.58%
|
|
RLMD
Relmada Therapeutics, Inc.
Prader-Willi Syndrome is a rare-disease indication being pursued for Sepranolone.
|
$134.76M |
$4.34
+7.02%
|
|
CAMP
CAMP4 Therapeutics Corporation
Pipeline targets rare genetic disorders (UCD, SYNGAP-related disorders) indicating focus on rare diseases.
|
$130.85M |
$6.72
+3.54%
|
|
ATRA
Atara Biotherapeutics, Inc.
Ebvallo targets EBV-related diseases which are rare.
|
$126.38M |
$17.95
-0.25%
|
|
IKT
Inhibikase Therapeutics, Inc.
PAH is a rare disease indication and the company is pursuing therapy development in this orphan space.
|
$114.76M |
$1.63
+5.84%
|
|
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.
|
$110.03M |
$5.91
-5.14%
|
|
INO
Inovio Pharmaceuticals, Inc.
RRP is a rare disease targeted by INO-3107, placing the company in the Rare Diseases segment.
|
$109.47M |
$2.27
+9.95%
|
|
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.
|
$108.67M |
$41.13
+5.43%
|
|
COYA
Coya Therapeutics, Inc.
ALS and Frontotemporal Dementia programs position COYA within rare neurodegenerative diseases.
|
$103.36M |
$5.98
-3.24%
|
|
FBIO
Fortress Biotech, Inc.
Asset focus on rare disease programs (e.g., copper histidinate for Menkes disease) fits Biotech - Rare Diseases.
|
$87.18M |
$3.35
+14.16%
|
|
CRVO
CervoMed Inc.
DLB/FTD indications and FDA orphan designation place the program within Rare Diseases biotech.
|
$78.19M |
$8.45
-0.06%
|
|
KRRO
Korro Bio, Inc.
AATD and rare metabolic disease programs position KRRO within rare disease biotech indications.
|
$77.29M |
$8.01
-2.67%
|
|
ATYR
aTyr Pharma, Inc.
Sarcoidosis and SSc-ILD are rare diseases, and the company’s lead programs target these ILD indications.
|
$75.99M |
$0.73
-5.88%
|
|
CRDL
Cardiol Therapeutics Inc.
Recurrent pericarditis is presented as a rare-disease indication with Phase III development.
|
$75.81M |
$1.03
-4.17%
|
|
RFL
Rafael Holdings, Inc.
NPC1 rare-disease program (Trappsol Cyclo) places Rafael Holdings in the Biotech - Rare Diseases category.
|
$75.59M |
$1.26
-13.36%
|
|
PLRX
Pliant Therapeutics, Inc.
PLN-101325 targets muscular dystrophies, placing the company in the rare diseases biotech space.
|
$75.51M |
$1.26
+2.85%
|
|
CLNN
Clene Inc.
ALS/MS pipeline involves rare-disease CNS targets, aligning with a rare diseases biotech focus.
|
$72.13M |
$7.22
|
|
MGX
Metagenomi, Inc. Common Stock
Hemophilia A is a rare disease; MGX's lead program targets rare disease indications through gene editing.
|
$61.93M |
$1.62
-1.52%
|
|
DTIL
Precision BioSciences, Inc.
PBGENE-DMD targets Duchenne muscular dystrophy, a rare genetic disease, aligning with the company's rare-disease pipeline program.
|
$58.82M |
$4.93
-1.10%
|
|
INMB
INmune Bio, Inc.
CORDStrom MSC therapy for RDEB places INmune Bio in the Rare Diseases biotech arena.
|
$53.44M |
$1.90
-5.47%
|
|
TELO
Telomir Pharmaceuticals, Inc. Common Stock
Preclinical work includes models for Werner Syndrome and Progeria, placing the company in the rare diseases biotech space.
|
$46.81M |
$1.44
-1.03%
|
|
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.
|
$45.77M |
$3.27
-1.06%
|
|
KZR
Kezar Life Sciences, Inc.
Autoimmune hepatitis is a rare disease area, aligning with the Rare Diseases investable theme.
|
$45.70M |
$6.30
+0.96%
|
|
XFOR
X4 Pharmaceuticals, Inc.
Company develops therapies targeting rare immune disorders (WHIM syndrome, CN), fitting Biotech - Rare Diseases.
|
$44.61M |
$3.88
-0.77%
|
|
JUNS
Jupiter Neurosciences, Inc.
Biotech - Rare Diseases: Pipeline includes therapies for Friedreich's Ataxia, MELAS, and other rare conditions.
|
$39.01M |
$1.09
-2.68%
|
|
CPIX
Cumberland Pharmaceuticals Inc.
DMD cardiomyopathy orphan/rare pediatric disease designations place the pipeline in the rare diseases biotech space.
|
$37.85M |
$3.01
+18.97%
|
|
RNTX
Rein Therapeutics Inc.
IPF/related fibrotic orphan indications classify Rein's lead assets as addressing a rare disease space.
|
$36.12M |
$1.45
-6.45%
|
|
ATHE
Alterity Therapeutics Limited
MSA and other neurodegenerative diseases fall under Rare Diseases, a notable focus in Alterity's pipeline.
|
$35.01M |
$3.11
-5.47%
|
|
FGEN
FibroGen, Inc.
Roxadustat for anemia in lower-risk MDS positions the program within Biotech - Rare Diseases as a potential orphan/disease-focused asset.
|
$33.89M |
$9.88
+17.84%
|
|
ANTX
AN2 Therapeutics, Inc.
Chagas disease program places the company in the rare diseases biotech space.
|
$32.79M |
$1.15
-4.17%
|
|
PASG
Passage Bio, Inc.
FTD-GRN and related neurodegenerative indications are rare diseases targeted by Passage Bio's pipeline.
|
$31.14M |
$10.20
+4.13%
|
|
RLYB
Rallybio Corporation
Core focus on rare diseases with lead and preclinical assets addressing complement dysregulation and iron overload.
|
$30.59M |
$0.77
+4.71%
|
|
BRNS
Barinthus Biotherapeutics plc
Celias disease represents a rare autoimmune indication, aligning with Rare Diseases biotechnology investment theme.
|
$27.66M |
$0.73
+7.46%
|
|
CVKD
Cadrenal Therapeutics, Inc. Common Stock
The program receives orphan drug designations for niche indications, aligning with the Rare Diseases tag.
|
$22.52M |
$11.93
+8.45%
|
|
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.
|
$19.16M |
$1.64
-1.20%
|
|
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.
|
$16.89M |
$0.11
-4.69%
|
|
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.
|
$15.26M |
$3.92
+1.16%
|
|
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%
|
|
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.
|
$13.59M |
$0.15
-46.22%
|
|
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%
|
|
AIMD
Ainos, Inc.
VELDONA human therapeutic programs target rare diseases, aligning with Biotech - Rare Diseases.
|
$10.57M |
$2.16
-4.85%
|
|
LGVN
Longeveron Inc.
HLHS and pediatric DCM programs place the company in Biotech - Rare Diseases.
|
$9.24M |
$0.58
-5.47%
|
|
ICU
SeaStar Medical Holding Corporation
Pediatric/rare-disease indications for QUELIMMUNE position the company within Rare Diseases.
|
$8.97M |
$0.30
-6.38%
|
|
KTTA
Pasithea Therapeutics Corp.
There is potential expansion into inflammatory diseases and rare conditions (e.g., NF1), suggesting relevance to rare-disease indications.
|
$8.41M |
$1.24
+9.29%
|
|
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.
|
$7.94M |
$3.24
-2.11%
|
|
KPRX
Kiora Pharmaceuticals, Inc.
Kiora's lead programs target rare retinal diseases with orphan drug designations, aligning with the Rare Diseases biotech investment theme.
|
$7.33M |
$2.08
-2.34%
|
|
SNGX
Soligenix, Inc.
BD (Behçet's Disease) and other rare-disease programs classify Soligenix under Biotech - Rare Diseases.
|
$7.07M |
$1.59
-3.33%
|
|
CELZ
Creative Medical Technology Holdings, Inc.
FDA Orphan Drug Designation for brittle Type 1 Diabetes indicates a rare-disease focus.
|
$5.55M |
$2.15
+0.23%
|
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