Biotech - Rare Diseases
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All Stocks (207)
| Company | Market Cap | Price |
|---|---|---|
|
RCKT
Rocket Pharmaceuticals, Inc.
Pipeline targets rare genetic diseases (LAD-I, Fanconi anemia, Danon disease, PKP2-ACM, PKD), aligning with a rare diseases focus.
|
$521.63M |
$4.66
-3.42%
|
|
ANNX
Annexon, Inc.
GBS and related autoimmune targets place Annexon in Biotech - Rare Diseases.
|
$515.37M |
$4.67
-6.87%
|
|
TECX
Tectonic Therapeutic, Inc.
TX2100 development for Hereditary Hemorrhagic Telangiectasia (HHT) aligns TECX with Rare Diseases biotech initiatives.
|
$513.01M |
$27.11
-1.09%
|
|
VNDA
Vanda Pharmaceuticals Inc.
VCA-894A and VGT-1849A ASO programs indicate a Rare Diseases focus.
|
$484.59M |
$8.10
-1.22%
|
|
SLDB
Solid Biosciences Inc.
The programs target rare diseases (DMD, Friedreich's ataxia, CPVT), aligning with Biotech - Rare Diseases.
|
$478.12M |
$5.71
-6.92%
|
|
TRDA
Entrada Therapeutics, Inc.
DMD and DM1 programs indicate a focus on rare diseases within the biotech portfolio.
|
$477.74M |
$12.15
-2.76%
|
|
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.
|
$470.11M |
$17.66
+0.71%
|
|
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%
|
|
LRMR
Larimar Therapeutics, Inc.
Company focuses on Friedreich's ataxia, a rare disease, aligning with Biotech - Rare Diseases.
|
$466.47M |
$5.47
+0.46%
|
|
RGNX
REGENXBIO Inc.
RGX-121 targets a rare disease (MPS II) in a late-stage program.
|
$461.70M |
$8.41
-7.93%
|
|
FULC
Fulcrum Therapeutics, Inc.
Company's focus on sickle cell disease and Diamond-Blackfan anemia positions it in rare genetic diseases.
|
$451.89M |
$8.04
-3.71%
|
|
AVXL
Anavex Life Sciences Corp.
Rett syndrome is a rare disease in the pipeline, supporting Rare Diseases biotech tag.
|
$446.74M |
$5.00
-0.10%
|
|
AUTL
Autolus Therapeutics plc
Lupus nephritis represents a rare autoimmune indication in the pipeline.
|
$433.81M |
$1.58
-2.76%
|
|
FDMT
4D Molecular Therapeutics, Inc.
FDMT's CF program (4D-710) targets a rare disease indication, aligning with the Rare Diseases Biotech theme.
|
$417.05M |
$8.45
-5.38%
|
|
PRTC
PureTech Health plc
IPF program situates PureTech within rare-disease indications.
|
$409.41M |
$17.65
+2.32%
|
|
DMAC
DiaMedica Therapeutics Inc.
Preeclampsia indication places the program within biotech initiatives targeting rare or high-need diseases.
|
$401.62M |
$7.78
+0.13%
|
|
LXEO
Lexeo Therapeutics, Inc. Common Stock
Lead programs target Friedreich ataxia and PKP2 cardiomyopathy, both rare diseases.
|
$390.97M |
$6.89
-4.77%
|
|
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
|
|
BNTC
Benitec Biopharma Inc.
BB-301 targets a rare genetic disease (OPMD), aligning with Rare Diseases in biotech.
|
$369.44M |
$10.90
-0.09%
|
|
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%
|
|
NGNE
Neurogene Inc.
Rett syndrome is a rare neurological disease target; company focuses on rare diseases.
|
$341.24M |
$23.65
-1.09%
|
|
ALDX
Aldeyra Therapeutics, Inc.
ADX2191 targets rare retinal diseases; pipeline emphasis on rare diseases.
|
$312.55M |
$4.99
-4.04%
|
|
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.
|
$304.15M |
$3.24
-1.22%
|
|
AARD
Aardvark Therapeutics, Inc. Common Stock
Aardvark focuses on Prader-Willi Syndrome and related rare diseases, supported by Orphan Drug Designation.
|
$272.95M |
$12.70
+0.95%
|
|
SLN
Silence Therapeutics plc
Company’s focus on rare disease programs (e.g., Divesiran for PV) fits the rare diseases biotech theme.
|
$272.74M |
$5.19
-10.21%
|
|
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
|
|
RNA
Avidity Biosciences, Inc.
Biotech - Rare Diseases: pipeline targets DM1, FSHD, and DMD, aligning with rare disease focus.
|
$256.62M |
$16.39
-0.88%
|
|
IMMX
Immix Biopharma, Inc.
Relates to a rare orphan disease (AL Amyloidosis), aligning with Rare Diseases.
|
$246.82M |
$8.53
-0.35%
|
|
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.
|
$246.26M |
$89.64
+1.40%
|
|
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%
|
|
AVTX
Avalo Therapeutics, Inc.
The company references HS as an indication and discusses a rare-disease pipeline emphasis, fitting Biotech - Rare Diseases.
|
$241.48M |
$18.38
+0.11%
|
|
TARA
Protara Therapeutics, Inc.
Lymphatic malformations and rare pediatric indications place the company in Rare Diseases.
|
$239.21M |
$6.03
-2.74%
|
|
ALEC
Alector, Inc.
FTD-GRN is a rare disease indication, aligning with Rare Diseases Biotech tag.
|
$234.68M |
$2.08
-3.26%
|
|
VYGR
Voyager Therapeutics, Inc.
Friedreich’s ataxia and Gaucher disease programs via partnerships indicate exposure to Rare Diseases.
|
$229.09M |
$3.92
-4.96%
|
|
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%
|
|
PEPG
PepGen Inc.
DM1 is a rare genetic disease, placing PepGen in Biotech - Rare Diseases as a core focus area.
|
$221.73M |
$6.75
+5.14%
|
|
IRD
Opus Genetics, Inc.
Rare disease focus: inherited retinal diseases are rare conditions, aligning with IRD’s target indications.
|
$206.68M |
$3.65
+5.80%
|
|
RNAC
Cartesian Therapeutics, Inc.
Pipeline targets or includes rare autoimmune/related indications, aligning with Rare Diseases.
|
$204.64M |
$7.87
|
|
TLSA
Tiziana Life Sciences Ltd
MSA, na-SPMS, and ALS represent rare/neurodegenerative indications targeted by TLSA, aligning with Rare Diseases.
|
$185.78M |
$1.45
+1.05%
|
|
NTHI
Neonc Technologies Holdings, Inc.
Rare Pediatric Disease designation aligns with pediatric brain tumor program (NEO100 pediatric).
|
$184.50M |
$9.68
+0.52%
|
|
PRQR
ProQR Therapeutics N.V.
AX-2402 targets Rett syndrome, placing ProQR in the Rare Diseases biotech focus.
|
$183.11M |
$1.68
-1.18%
|
|
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%
|
|
ARCT
Arcturus Therapeutics Holdings Inc.
CF and OTC deficiency are rare diseases; pipeline targets rare disease indications.
|
$169.72M |
$6.21
+1.22%
|
|
THTX
Theratechnologies Inc.
Partnerships targeting rare diseases (donidalorsen, olezarsen) position Theratechnologies in the rare-diseases space.
|
$155.87M |
$3.39
|
|
RLMD
Relmada Therapeutics, Inc.
Prader-Willi Syndrome is a rare-disease indication being pursued for Sepranolone.
|
$155.00M |
$4.67
-3.51%
|
|
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%
|
|
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%
|
|
IKT
Inhibikase Therapeutics, Inc.
PAH is a rare disease indication and the company is pursuing therapy development in this orphan space.
|
$145.31M |
$1.92
-1.79%
|
|
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%
|
|
ATRA
Atara Biotherapeutics, Inc.
Ebvallo targets EBV-related diseases which are rare.
|
$123.46M |
$17.70
+0.71%
|
|
TNXP
Tonix Pharmaceuticals Holding Corp.
TNX-2900 targets Prader-Willi syndrome, representing a rare-disease program within Tonix's portfolio.
|
$122.55M |
$14.01
+0.21%
|
|
SGMO
Sangamo Therapeutics, Inc.
Fabry disease program ST-920 targets a rare disease with accelerated approval pathway.
|
$119.24M |
$0.39
-0.56%
|
|
KRRO
Korro Bio, Inc.
AATD and rare metabolic disease programs position KRRO within rare disease biotech indications.
|
$113.91M |
$12.18
+0.66%
|
|
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%
|
|
TNYA
Tenaya Therapeutics, Inc.
TN-201/TN-401 target rare genetic cardiomyopathies, aligning with the rare diseases biotech focus.
|
$94.75M |
$0.61
+4.35%
|
|
ATYR
aTyr Pharma, Inc.
Sarcoidosis and SSc-ILD are rare diseases, and the company’s lead programs target these ILD indications.
|
$94.06M |
$0.96
-3.19%
|
|
FBIO
Fortress Biotech, Inc.
Asset focus on rare disease programs (e.g., copper histidinate for Menkes disease) fits Biotech - Rare Diseases.
|
$92.24M |
$3.08
-0.48%
|
|
COYA
Coya Therapeutics, Inc.
ALS and Frontotemporal Dementia programs position COYA within rare neurodegenerative diseases.
|
$89.65M |
$5.36
-0.09%
|
|
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%
|
|
CRDL
Cardiol Therapeutics Inc.
Recurrent pericarditis is presented as a rare-disease indication with Phase III development.
|
$84.33M |
$1.02
|
|
CAMP
CAMP4 Therapeutics Corporation
Pipeline targets rare genetic disorders (UCD, SYNGAP-related disorders) indicating focus on rare diseases.
|
$82.26M |
$4.00
-1.96%
|
|
PLRX
Pliant Therapeutics, Inc.
PLN-101325 targets muscular dystrophies, placing the company in the rare diseases biotech space.
|
$79.19M |
$1.28
-0.78%
|
|
RFL
Rafael Holdings, Inc.
NPC1 rare-disease program (Trappsol Cyclo) places Rafael Holdings in the Biotech - Rare Diseases category.
|
$75.07M |
$1.46
+0.34%
|
|
CLNN
Clene Inc.
ALS/MS pipeline involves rare-disease CNS targets, aligning with a rare diseases biotech focus.
|
$72.13M |
$7.22
|
|
MREO
Mereo BioPharma Group plc
Mereo's core focus is developing therapeutics for rare diseases, fitting the Biotech - Rare Diseases category.
|
$65.88M |
$0.41
-1.55%
|
|
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%
|
|
ATHE
Alterity Therapeutics Limited
MSA and other neurodegenerative diseases fall under Rare Diseases, a notable focus in Alterity's pipeline.
|
$62.07M |
$3.71
+9.12%
|
|
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%
|
|
DTIL
Precision BioSciences, Inc.
PBGENE-DMD targets Duchenne muscular dystrophy, a rare genetic disease, aligning with the company's rare-disease pipeline program.
|
$56.22M |
$4.77
|
|
CPIX
Cumberland Pharmaceuticals Inc.
DMD cardiomyopathy orphan/rare pediatric disease designations place the pipeline in the rare diseases biotech space.
|
$54.90M |
$3.67
|
|
RLYB
Rallybio Corporation
Core focus on rare diseases with lead and preclinical assets addressing complement dysregulation and iron overload.
|
$54.87M |
$10.66
+1.52%
|
|
KZR
Kezar Life Sciences, Inc.
Autoimmune hepatitis is a rare disease area, aligning with the Rare Diseases investable theme.
|
$53.53M |
$7.27
-0.55%
|
|
TELO
Telomir Pharmaceuticals, Inc. Common Stock
Preclinical work includes models for Werner Syndrome and Progeria, placing the company in the rare diseases biotech space.
|
$41.00M |
$1.25
-4.20%
|
|
XFOR
X4 Pharmaceuticals, Inc.
Company develops therapies targeting rare immune disorders (WHIM syndrome, CN), fitting Biotech - Rare Diseases.
|
$39.93M |
$3.49
-0.29%
|
|
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%
|
|
CRVO
CervoMed Inc.
DLB/FTD indications and FDA orphan designation place the program within Rare Diseases biotech.
|
$37.20M |
$4.03
+0.25%
|
|
ANTX
AN2 Therapeutics, Inc.
Chagas disease program places the company in the rare diseases biotech space.
|
$36.62M |
$1.33
-0.75%
|
|
INMB
INmune Bio, Inc.
CORDStrom MSC therapy for RDEB places INmune Bio in the Rare Diseases biotech arena.
|
$35.09M |
$1.31
-0.76%
|
|
PASG
Passage Bio, Inc.
FTD-GRN and related neurodegenerative indications are rare diseases targeted by Passage Bio's pipeline.
|
$26.83M |
$8.24
-2.37%
|
|
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.
|
$24.43M |
$0.61
+1.83%
|
|
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.
|
$23.58M |
$6.87
+22.24%
|
|
CVKD
Cadrenal Therapeutics, Inc. Common Stock
The program receives orphan drug designations for niche indications, aligning with the Rare Diseases tag.
|
$16.99M |
$8.31
+0.18%
|
|
JUNS
Jupiter Neurosciences, Inc.
Biotech - Rare Diseases: Pipeline includes therapies for Friedreich's Ataxia, MELAS, and other rare conditions.
|
$15.78M |
$0.44
-2.27%
|
|
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.
|
$13.32M |
$2.81
|
|
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.
|
$12.23M |
$1.03
-2.83%
|
|
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%
|
|
QNRX
Quoin Pharmaceuticals, Ltd.
Lead program targets a rare disease (Netherton Syndrome) and is developed by a biotech focused on rare diseases.
|
$8.75M |
$8.72
+2.59%
|
|
LGVN
Longeveron Inc.
HLHS and pediatric DCM programs place the company in Biotech - Rare Diseases.
|
$7.96M |
$0.54
+2.21%
|
|
AIMD
Ainos, Inc.
VELDONA human therapeutic programs target rare diseases, aligning with Biotech - Rare Diseases.
|
$7.59M |
$1.69
+3.68%
|
|
KPRX
Kiora Pharmaceuticals, Inc.
Kiora's lead programs target rare retinal diseases with orphan drug designations, aligning with the Rare Diseases biotech investment theme.
|
$7.00M |
$2.02
-1.23%
|
|
XAIR
Beyond Air, Inc.
BA-102 in NeuroNOS has Orphan Drug Designation, aligning with rare-disease biotech development.
|
$6.94M |
$0.85
-1.96%
|
|
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%
|
|
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.
|
$5.64M |
$3.01
+7.50%
|
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