Biotech - Rare Diseases
•215 stocks
•
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All Stocks (215)
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| Company | Market Cap | Price |
|---|---|---|
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ALXN1840 asset for Wilson disease places the company in the rare diseases domain.
|
$537.04M |
$87.82
-14.34%
|
|
Eton is focused on ultra-rare disease therapies (ALKINDI SPRINKLE, INCRELEX, GALZIN, and pipeline ET-400/ET-600/ET-700/ET-800), aligning with the Biotech - Rare Diseases investable theme.
|
$534.47M |
$19.93
+0.66%
|
|
PAH/PH-ILD involve rare diseases; seralutinib targets disease modification in a subset of rare pulmonary vascular diseases.
|
$518.25M |
$2.28
-10.59%
|
|
RP and Stargardt are rare ocular diseases, aligning with Rare Diseases.
|
$516.90M |
$1.79
+1.41%
|
|
CF and OTC deficiency are rare diseases, aligning with the Rare Diseases tag.
|
$513.94M |
$18.95
-5.11%
|
|
Pipeline includes a program for Primary Sclerosing Cholangitis (PSC), placing future efforts in the Rare Diseases space.
|
$501.44M |
$2.53
+2.85%
|
|
The company targets genetically defined rare diseases (e.g., sickle cell disease and Diamond-Blackfan anemia), aligning with the Rare Diseases biotech theme.
|
$485.27M |
$8.99
-7.37%
|
|
HAE is a rare disease and Astria's navenibart targets plasma kallikrein, contributing to a dual-asset pipeline focused on rare immunologic conditions.
|
$468.97M |
$8.31
-4.04%
|
|
Pipeline includes therapies for Friedreich's ataxia, CPVT, and DMD—indicating a focus on rare diseases.
|
$461.99M |
$5.96
-7.31%
|
|
Rett syndrome is a rare genetic neurological disease, and Neurogene's lead program targets Rett syndrome, placing it in the Rare Diseases biotech space.
|
$430.86M |
$30.21
-4.28%
|
|
4D-710 addresses cystic fibrosis, a rare genetic disease, aligning FDMT with the Rare Diseases biotech segment.
|
$412.29M |
$8.90
-7.29%
|
|
Biotech - Rare Diseases: KRRO-110 targets Alpha-1 Antitrypsin Deficiency, a rare genetic condition.
|
$403.51M |
$42.97
-8.18%
|
|
The company focuses on rare-disease therapies (e.g., AAT deficiency and CMV-related products) with InnovAATe pipeline targeting AATD.
|
$390.86M |
$6.80
-2.72%
|
|
BB-301 targets OPMD, highlighting a focus on rare disease therapeutics.
|
$381.96M |
$14.92
+5.89%
|
|
ALSP is a rare neurodegenerative disease, placing Vigil in the Biotech - Rare Diseases space.
|
$375.71M |
$8.05
|
|
Development for Prader-Willi Syndrome hyperphagia places the company in the rare diseases niche, aligning with the 'Biotech - Rare Diseases' investable theme.
|
$352.78M |
$16.26
-1.99%
|
|
Pipeline targets rare genetic diseases (LAD-I, Fanconi Anemia, Danon disease, PK deficiency, BAG3-DCM, etc.).
|
$351.23M |
$3.27
+0.46%
|
|
Company targets inherited nucleotide repeat expansion diseases (FA, FECD, DM1, HD), a class of rare genetic diseases, which fits the Biotech - Rare Diseases investment theme.
|
$346.86M |
$6.11
-4.08%
|
|
The company targets rare genetic heart diseases, aligning with Biotech - Rare Diseases.
|
$341.60M |
$2.10
-5.19%
|
|
Lead products target rare/low-prevalence autoimmune and neuroinflammatory diseases (GBS, GA).
|
$340.11M |
$3.10
-4.91%
|
|
ADX2191 targets rare retinal diseases (PVRL, RP), placing the company in the rare diseases biotech space.
|
$330.02M |
$5.51
-4.67%
|
|
DMD and Becker muscular dystrophy are rare genetic diseases, aligning Capricor with the Rare Diseases biotech focus through its deramiocel program.
|
$324.07M |
$7.09
-6.96%
|
|
HETLIOZ for Non-24-Hour Sleep-Wake Disorder and SMS, plus rare-disease pipeline assets (e.g., ASOs) position the company in Rare Diseases.
|
$312.94M |
$5.31
-3.80%
|
|
Direct focus on developing therapies for rare genetic diseases (OI, AATD-LD) classifies Mereo under Biotech - Rare Diseases.
|
$311.64M |
$1.96
-6.67%
|
|
The company’s pivot targets rare/orphan diseases with in vivo gene-editing technologies, fitting the Rare Diseases investable theme.
|
$311.41M |
$3.72
-11.85%
|
|
FTD-GRN is a rare disease target and latozinemab/AL001 aims to elevate progranulin, a core disease-modifying strategy for a rare CNS disorder.
|
$301.98M |
$3.02
-5.48%
|
|
TX2100 is being developed for hereditary hemorrhagic telangiectasia, a rare disease.
|
$299.13M |
$16.02
-6.10%
|
|
Stiff Person Syndrome and related autoimmune indications are rare diseases, aligning with rare disease biotech focus.
|
$286.54M |
$6.63
-5.69%
|
|
DEE and Absence Epilepsy are rare neurological disorders, aligning the company with Biotech - Rare Diseases.
|
$282.52M |
$63.29
+2.58%
|
|
Larimar focuses on a rare-disease target (Friedreich's ataxia) and develops niche therapeutic solutions, fitting Biotech - Rare Diseases.
|
$279.16M |
$4.36
-3.75%
|
|
Pipeline includes rare-disease targets (Friedreich's ataxia, Gaucher-related GBA1, SOD1 ALS components), indicating focus on rare diseases within biotech.
|
$276.68M |
$5.00
-3.29%
|
|
The company focuses on rare disease indications (RDEB) via gene/cell therapies.
|
$273.18M |
$5.34
-1.11%
|
|
Inozyme's lead program INZ-701 targets ENPP1 Deficiency, a rare genetic disorder, placing the company squarely in Biotech - Rare Diseases.
|
$256.96M |
$4.00
|
|
Apraglutide targets a rare-disease indication (SBS-IF) and is part of Ironwood's strategic pivot into Rare Diseases.
|
$256.48M |
$1.58
-15.24%
|
|
Zaltenibart and related programs target rare diseases (e.g., PNH, C3G).
|
$245.50M |
$4.19
-8.71%
|
|
Systemic sclerosis (and related autoimmune indications in the pipeline) intersects with Rare Diseases, reflecting the potential rare-disease focus of its development program.
|
$243.17M |
$3.94
+0.13%
|
|
Pipeline includes indications for rare autoimmune diseases (e.g., MG), aligning with Rare Diseases focus.
|
$241.89M |
$9.32
-8.09%
|
|
Divesiran (PV) and Zerlasiran target rare diseases, placing Silence in the Rare Diseases biotech space.
|
$235.84M |
$5.00
-7.41%
|
|
Duchenne muscular dystrophy and other rare diseases are core focus for therapies.
|
$231.14M |
$6.09
-10.44%
|
|
Company focuses its proprietary siRNA therapies on rare diseases and orphan conditions (PV is rare) with high unmet needs.
|
$226.72M |
$1.60
|
|
Lead indications are rare genetic diseases (Friedreich ataxia cardiomyopathy, PKP2 cardiomyopathy).
|
$219.76M |
$6.62
-1.05%
|
|
NEO100-3.00 and pediatric brain tumor programs carry Rare Diseases/rare pediatric indications.
|
$219.38M |
$11.53
-1.11%
|
|
The company focuses on rare liver diseases (PBC, PSC) and rare-disease indications, fitting the Rare Diseases biotech theme.
|
$212.11M |
$4.25
-2.29%
|
|
Rare diseases focus given instances like MSA and na-SPMS with limited options; positions in rare neurodegenerative indications.
|
$210.30M |
$2.04
-5.56%
|
|
AX-2402 targets Rett syndrome, a rare disease, aligning ProQR with the Rare Diseases biotech space.
|
$203.04M |
$2.41
-3.98%
|
|
IV Choline Chloride targets rare-disease/rare-patient scenarios in parenteral support, aligning with Rare Diseases as a core focus.
|
$201.40M |
$5.22
-8.42%
|
|
Protalix develops recombinant protein therapies for rare diseases (Elelyso and Elfabrio) and has a pipeline focused on rare renal indications.
|
$188.17M |
$2.36
+5.36%
|
|
Fabry disease program and prion/other rare-disease targets place the company in the Biotech - Rare Diseases category.
|
$156.76M |
$0.67
-10.35%
|
|
Ionis partnership (donidalorsen and olezarsen) targets rare diseases in Canada with meaningful profitability potential.
|
$155.87M |
$3.39
+0.89%
|
|
TNX-2900 targets Prader-Willi syndrome, placing Tonix in the rare diseases biotech space.
|
$155.21M |
$21.19
-4.46%
|
|
PepGen focuses on a rare disease (DM1), indicating involvement in the rare diseases biotech segment.
|
$154.28M |
$4.71
-3.78%
|
|
The lead asset targets hidradenitis suppurativa, a rare/low-prevalence disease, aligning with the Rare Diseases category.
|
$145.09M |
$13.40
+3.47%
|
|
Tuberous Sclerosis Complex (a rare genetic disorder)–related epilepsy places Cassava in the Rare Diseases biotech space.
|
$144.44M |
$2.90
-2.84%
|
|
Direct focus on rare disease therapies under the Biotech - Rare Diseases category (govorestat and related ARI programs).
|
$133.12M |
$0.94
-9.59%
|
|
EB612 targets hypoparathyroidism, an orphan/rare disease, aligning with Biotech - Rare Diseases.
|
$120.45M |
$2.65
+6.00%
|
|
PAH is a rare/orphan disease, aligning with the Rare Diseases tag.
|
$120.43M |
$1.62
|
|
Inherited retinal diseases are a rare-disease focus, aligning with Biotech - Rare Diseases.
|
$116.94M |
$1.96
-2.97%
|
|
SGT-610 targets Gorlin Syndrome, a rare genetic disease, aligning with the Biotech - Rare Diseases theme.
|
$108.62M |
$38.99
-2.48%
|
|
MGX's lead programs include therapies for rare diseases (e.g., hemophilia A) via gene editing.
|
$106.91M |
$2.86
-8.04%
|
|
ALS/FTD programs position COYA within the Rare Diseases biotech niche.
|
$101.52M |
$6.07
-3.04%
|
|
ILD indications like sarcoidosis and SSc-ILD are rare diseases, and ATYR's lead program targets this niche.
|
$94.34M |
$1.06
-0.93%
|
|
INO-3107 targets recurrent respiratory papillomatosis (RRP), a rare disease, placing INOVIO in the rare diseases therapeutic segment.
|
$94.25M |
$2.57
-1.91%
|
|
EBV+ PTLD represents a rare disease indication, aligning with the Rare Diseases tag.
|
$93.18M |
$15.09
-3.45%
|
|
PLRX's PLN-101325 targets muscular dystrophies, aligning with Biotech - Rare Diseases.
|
$91.47M |
$1.49
-2.61%
|
|
Lead programs target rare neurodegenerative diseases (e.g., MSA), aligning with the Rare Diseases biotech theme.
|
$85.62M |
$0.01
|
|
CUTX-101 (Cyprium) targets a rare disease (Menkes) within Fortress's asset portfolio.
|
$80.13M |
$2.71
-3.56%
|
|
Quince Therapeutics is focused on a rare pediatric neurodegenerative disease (Ataxia-Telangiectasia) with its lead asset and platform, fitting the Rare Diseases biotech theme.
|
$75.02M |
$1.65
+0.61%
|
|
The recurrent pericarditis/rare-disease focus (aligned with Orphan Drug designation) supports the Biotech - Rare Diseases theme.
|
$73.00M |
$1.04
-4.59%
|
|
Expansion into rare neurodegenerative indications (DLB without AD co-pathology, FTD) supports a 'Rare Diseases' investable theme.
|
$70.93M |
$8.15
-5.12%
|
|
Lead program targets relapsed/refractory AL amyloidosis, a rare/orphan disease.
|
$69.69M |
$2.50
-7.41%
|
|
PBGENE-DMD targets Duchenne muscular dystrophy, placing the company in the rare diseases gene-therapy space.
|
$62.87M |
$5.67
+4.23%
|
|
ALS is a rare disease, making the ALS focus align with the 'Biotech - Rare Diseases' investable theme.
|
$62.78M |
$1.28
+3.23%
|
|
Pipeline targets rare genetic diseases (e.g., Urea Cycle Disorders and SYNGAP1-related disorders).
|
$61.09M |
$3.03
+1.00%
|
|
Alpha-1 antitrypsin deficiency program places the company in Rare Diseases biotech efforts.
|
$56.75M |
$2.23
+16.15%
|
|
Pipeline includes potential expansion into rare/neurodegenerative diseases (e.g., Huntington's chorea) via the POZ platform.
|
$52.93M |
$5.31
-0.38%
|
|
The company’s pipeline targets rare diseases (e.g., DMD cardiomyopathy) and has Orphan Drug/Rare Pediatric Disease designations, fitting the 'Biotech - Rare Diseases' theme.
|
$51.62M |
$3.45
-2.27%
|
|
Pursuit of therapies for rare genetic/neurodegenerative diseases (e.g., Friedreich's Ataxia) aligns with the Biotech - Rare Diseases theme.
|
$50.65M |
$1.53
-3.77%
|
|
Telomir's work includes rare-disease models (Werner syndrome, Progeria), aligning with rare-disease biotech.
|
$49.11M |
$1.65
-7.82%
|
|
CORDStrom-based MSC therapy for RDEB, a rare disease, fits Biotech - Rare Diseases.
|
$48.97M |
$2.08
-1.18%
|
|
MSA is a rare neurodegenerative disease, supporting the Rare Diseases biotech tag.
|
$45.65M |
$4.29
-1.38%
|
|
Alopecia areata is a autoimmune/rare-disease area; bempikibart aligns with Rare Diseases focus as a disease-targeted biologic.
|
$43.55M |
$3.57
-0.56%
|
|
Roxadustat's use in lower-risk MDS aligns FibroGen with rare-disease focused therapies.
|
$42.64M |
$10.55
-3.74%
|
|
Grace Therapeutics focuses on rare diseases with orphan designation across its pipeline (GTx-104, GTx-102, GTx-101), aligning with the Biotech - Rare Diseases investable theme.
|
$41.35M |
$2.99
-1.01%
|
|
Chagas disease is a rare infectious disease; AN2-502998 targets chronic Chagas disease (Phase 1 expected H2 2025).
|
$39.23M |
$1.30
+2.36%
|
|
TPST-1495 is being developed for a rare disease (FAP), aligning with Rare Diseases.
|
$38.30M |
$10.40
-2.07%
|
|
Pursuing plant-based therapeutics for rare diseases such as Adrenoleukodystrophy (ALD).
|
$35.75M |
$2.02
|
|
Rafael is focused on Trappsol Cyclo for Niemann-Pick Disease Type C1, a rare disease, making Rare Diseases a core investable theme.
|
$33.38M |
$1.34
-1.47%
|
|
Autoimmune hepatitis is a rare autoimmune disease, which aligns Kezar with Biotech - Rare Diseases as a focused indication.
|
$30.83M |
$4.22
-5.17%
|
|
Lead assets (LTI-3.00, LTI-1.00) target Idiopathic Pulmonary Fibrosis and related rare pulmonary/fibrosis conditions with peptide-based therapeutics, placing Rein Therapeutics in the Biotech - Rare Diseases space.
|
$28.80M |
$1.30
-4.41%
|
|
Orphan Drug designation and niche, rare-indication focus place Cadrenal in Rare Diseases biotech context.
|
$27.59M |
$14.03
+0.14%
|
|
FTD-GRN is a rare neurodegenerative disease target, aligning Passage Bio with the Biotech - Rare Diseases investable theme.
|
$26.32M |
$8.47
-5.57%
|
|
Rallybio centers on rare-disease therapies (RLYB116 and RLYB332) and aligns its development strategy around treating rare conditions.
|
$21.26M |
$0.51
-7.11%
|
|
HT-KIT targets KIT-driven cancers and has a rare cancer focus (GIST, systemic mastocytosis), aligning with Biotech - Rare Diseases.
|
$20.87M |
$1.58
-5.39%
|
|
The lead program targeted a rare metabolic disease (PKU), aligning with the Rare Diseases investable theme.
|
$19.51M |
$1.67
-1.30%
|
|
The company centers on therapies for rare immune-related diseases (WHIM syndrome and CN), aligning with the Rare Diseases biotech focus.
|
$17.89M |
$3.09
-6.36%
|
|
ABPA and other respiratory conditions targeted by Pulmatrix's assets align with rare-disease/low-incidence biotech therapeutics.
|
$17.42M |
$4.77
+1.27%
|
|
PMN's ALS and MSA programs position the company within Rare Diseases (low-prevalence/orphan diseases).
|
$16.28M |
$0.50
+4.64%
|
|
VELDONA low-dose interferon platform targets rare autoimmune/infectious diseases (biotech category Rare Diseases).
|
$15.66M |
$3.77
-4.80%
|
|
ALS is a rare neurodegenerative disease, placing Athira within the Biotech - Rare Diseases investment theme.
|
$14.84M |
$3.80
-3.06%
|
|
Pipeline focuses on rare diseases (e.g., HLHS and pediatric DCM), aligning with Rare Diseases in biotech.
|
$12.20M |
$0.81
-7.66%
|
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