Best Rare Diseases Companies in United States
The 340 best Rare Diseases companies in United States in 2026, ranked by funding and momentum. Funding, investors, founders, valuation, tech stack and live signals. Top: BioCryst.
Ranking the best Rare Diseases companies across the United States by disclosed equity funding and recent momentum, this directory tracks 340 companies carrying $18.9B in total disclosed capital. That's real weight. The global rare disease market sits at $218.3B as of 2024, forecast to reach $414.6B by 2034 at a 6.7% CAGR, driven by genomic medicine investment and CRISPR and AAV gene therapy advances. As shown in 2019 research, the regulatory and commercial logic for rare disease drug development has been reshaping biotech pipelines for years. So the funding you see here isn't speculative noise. It reflects durable structural demand.
The sector financials tell you exactly where the money lands. Across 16 validated filers, median revenue is $20M, but the combined pool reaches $3.9B, and the concentration is steep. Bioverativ pulled $1.1B. Horizon Therapeutics filed $832M. BioMarin Pharmaceutical reported $766M, up 3%, while Amicus Therapeutics hit $634M with 20% YoY growth. Net income spreads even wider: PTC Therapeutics posted $683M, 21 times the sector median of $32M. I find that multiple clarifying rather than surprising, given how pricing power accumulates in orphan indications. Among our directory's top-funded names, Angitia ($156M) and Casma Therapeutics ($154M) represent the venture end of this spectrum, backed by investors including RA Capital Management, Orbimed, and Deerfield. The median revenue growth of negative 3% signals compression for smaller filers, but the outsized earners aren't feeling it.
- not explicitly mentioned
Ranked: Best Rare Diseases companies in United States
| # | Company | Disclosed funding | Last round | Stage | Employees | Founder | Market signals (1y) | Patents & IP focus | Recent signals |
|---|---|---|---|---|---|---|---|---|---|
| 1 | BioCryst Developer of small molecule drugs for orphan & autoimmune diseases and antivirals | $867M | 54 mo ago | Public | 536 536 → 615 (+15%) · sector median +18% | Jon Stonehouse CEO | 14 signals ▼67% Earnings · M&A · Regulatory | — | 1 internship |
| 2 | BridgeBio Developer of therapeutics for the treatment of genetic diseases | $434M | 25 mo ago | Acquired | 383 | Neil Kumar Co-Founder & CEO | 8 signals ▼75% Earnings · Funding · M&A | 32 patents Targeted small‑molecule cancer combos (KRAS, EGFR, SHP2) and metabolic kidney disease therapies | 1 award |
| 3 | PTC Therapeutics Developer of small molecule drugs for the treatment of rare diseases | $248M | 37 mo ago | Public | 995 995 → 1,159 (+16%) · sector median +18% | — | 18 signals ▼56% Regulatory · FDA approval · Earnings | 1,951 patents Small-molecule therapeutics for neurodegenerative and metabolic genetic disorders | — |
| 4 | Autobahn Therapeutics Provider of therapeutics for CNS disorders by the regenerative medicine | $228M | 23 mo ago | Series C | 23 | Kevin Finney Chairman and CEO | — | — | — |
| 5 | MBX Biosciences Developer of drugs for the treatment of rare endocrine diseases | $216M | 22 mo ago | Public | 11 | Kent Hawryluk President & CEO | 7 signals Earnings · Announcements · Leadership | — | 1 partnership |
| 6 | Editas Medicine Developer of therapeutics for rare genetic diseases | $210M | 29 mo ago | Public | 257 257 → 183 (-29%) · sector median +18% | Cynthia Collins Ex-CEO & President | 3 signals ▼83% Earnings · Leadership | 713 patents CRISPR-based gene editing therapeutics for blood disorders and immune cell engineering | — |
| 7 | Attralus Developer of drugs for the treatment of Amyloid diseases | $197M | 28 mo ago | Series B | 23 | Jonathan Wall Co-Founder | — | 50 patents Engineered Fc‑fusion and antibody‑peptide biologics for amyloid disease therapy | — |
| 8 | Rallybio Developer of antibodies & protein therapies for rare disorders | $182M | 26 mo ago | Public | 43 43 → 23 (-47%) · sector median +18% | Stephen Uden Co-Founder | 5 signals ▼38% M&A · Funding · Earnings | — | — |
| 9 | Aro Biotherapeutics Developer of tissue-targeted genetic medicines for rare diseases | $177M | 30 mo ago | Series B | 47 | Susan Dillon President & CEO | — | 91 patents Targeted siRNA delivery via FN3 domains binding CD71 for enzyme replacement therapy | — |
| 10 | Angitia Developer of drugs for muscle, bone, and joint diseases 29Funding top 16%Talent top 38%Moat top 47%Market bottom 40% All 7 signals ▾Fundingtop 16% Talenttop 38% Moattop 47% Marketbottom 40% Globalbottom 39% Innovationbottom 23% Patentsbottom 20% Funding: Raised $156M across 5 rounds · 5 rounds over 6 yrs (0.8/yr) Talent: 2 named leaders on record · 2 C-suite/founder profiles curated rank vs industry peers · Fliar Intelligence Index 29/100 | $156M | 4 mo ago | Series D | — | Muyu Li Co-Founder & COO | — | 4 patents Anti‑sclerostin biologics for osteoporosis and bone‑mass therapies | — |
| 11 | Casma Therapeutics Developer of therapies for treating rare genetic disorders 40Funding top 20%Product top 30%Innovation top 38%Moat top 48% All 9 signals ▾Fundingtop 20% Producttop 30% Innovationtop 38% Moattop 48% Marketbottom 48% Growthbottom 41% Globalbottom 37% Newsbottom 22% Techbottom 21% Funding: Raised $154M across 4 rounds · 4 rounds over 8 yrs (0.5/yr) Product: 3 distinct product lines · Company stage: Series C rank vs industry peers · Fliar Intelligence Index 40/100 | $154M | 3 mo ago | Series C | 38 | — | — | — | — |
| 12 | QurAlis Developing drugs & CSF cartridge device for treating amyotrophic lateral sclerosis (ALS) 64Funding top 20%Patents top 23%Innovation top 25%Growth top 26% All 10 signals ▾Fundingtop 20% Patentstop 23% Innovationtop 25% Growthtop 26% Moattop 32% Talenttop 41% Techbottom 43% Globalbottom 42% Marketbottom 36% Newsbottom 22% Funding: Raised $144M across 7 rounds · 7 rounds over 8 yrs (0.9/yr) Patents: 69 patents matched (strict assignee match) · 62 filings since 2022 vs 7 in 2017-21 rank vs industry peers · Fliar Intelligence Index 64/100 | $144M | 9 mo ago | Series B | 38 38 → 87 (+129%) · sector median +18% | Kasper Roet Founder & CEO | — | 69 patents Antisense oligonucleotide therapeutics targeting UNC13A for neurological disease treatment | — |
| 13 | avidity biosciences Avidity Biosciences is a biotechnology company that develops oligonucleotide-based therapies for its patients. | $143M | 27 mo ago | Public | 186 253 → 443 (+75%) · sector median +18% | — | 8 signals ▼43% M&A · Earnings · Announcements | 369 patents Antibody‑oligonucleotide conjugates for treating muscular dystrophy and related muscle disorders | — |
| 14 | City Therapeutics Developer of gene therapies for multiple diseases | $135M | 20 mo ago | Series A | — | John Maraganore Co-Founder | — | — | — |
| 15 | Diagonal Therapeutics Developer of antibody therapeutics for the treatment of rare diseases 40Funding top 19%Moat top 42%Talent bottom 49%Market bottom 40% All 5 signals ▾Fundingtop 19% Moattop 42% Talentbottom 49% Marketbottom 40% Globalbottom 38% Funding: Raised $128M across 2 rounds · 2 rounds over 2 yrs (1.0/yr) Moat: Editorial + unicorn rating 5/10 · Soonicorn rank vs industry peers · Fliar Intelligence Index 40/100 | $128M | 5 mo ago | Series B | — | Alexey Lugovskoy CEO | — | — | — |
| 16 | Mirum Pharmaceuticals Developer of small molecule based therapeutics to treat rare diseases 82Funding top 17%Patents top 18%Innovation top 23%Product top 28% All 12 signals ▾Fundingtop 17% Patentstop 18% Innovationtop 23% Producttop 28% Growthtop 30% Fin. healthtop 31% Markettop 33% Moattop 35% Newstop 36% Techtop 45% Talentbottom 49% Globalbottom 47% Funding: Raised $120M across 3 rounds · 3 rounds over 8 yrs (0.4/yr) Patents: 135 patents matched (strict assignee match) · 115 filings since 2022 vs 20 in 2017-21 rank vs industry peers · Fliar Intelligence Index 82/100 | $120M | 4 mo ago | Public | 196 196 → 465 (+137%) · sector median +18% | Christopher Peetz CEO | 24 signals Announcements · Earnings · M&A | 135 patents IBAT inhibitor drugs for treating cholestasis and pediatric liver disease | — |
| 17 | Kala Pharmaceuticals Developer of ophthalmic treatments based on its mucosal penetrating product platform 45Funding top 22%Product top 24%Talent top 33%News top 34% All 12 signals ▾Fundingtop 22% Producttop 24% Talenttop 33% Newstop 34% Moattop 35% Patentstop 37% Innovationtop 41% Techbottom 49% Marketbottom 42% Growthbottom 42% Globalbottom 40% Fin. healthbottom 36% Funding: Raised $115M across 14 rounds · 14 rounds over 17 yrs (0.8/yr) Product: 2 distinct product lines · Company stage: Public rank vs industry peers · Fliar Intelligence Index 45/100 | $115M | 3 mo ago | Public | 34 34 → 80 (+135%) · sector median +18% | Justin Hanes Co-Founder | 10 signals ▲25% Funding · Earnings · Leadership | 267 patents Mucosal and ocular drug delivery via nanoparticle and polymer-based pharmaceutical formulations | — |
| 18 | Neomorph Developer of therapeutic solutions for the treatment of multiple diseases based on protein degradation… 53Funding top 23%Moat top 28%Talent top 28%Patents top 39% All 10 signals ▾Fundingtop 23% Moattop 28% Talenttop 28% Patentstop 39% Innovationtop 44% Partnerstop 45% Marketbottom 48% Techbottom 42% Globalbottom 33% Newsbottom 20% Funding: Raised $109M across 2 rounds · 2 rounds over 6 yrs (0.3/yr) Moat: Editorial + unicorn rating 8/10 · IP moat: 25 patents rank vs industry peers · Fliar Intelligence Index 53/100 | $109M | 2 mo ago | Series B | 54 | Philip Chamberlain Co-Founder & CEO | — | 25 patents Novel substituted piperidine-imide heterocycles for pharmaceutical active ingredient development | 5 job openings |
| 19 | X4 Pharmaceuticals Developer of small molecule drugs for the treatment of cancer | $98M | 10 mo ago | Public | 70 70 → 79 (+13%) · sector median +18% | — | 3 signals ▼79% Earnings · Layoffs | 437 patents Immunomodulatory small‑molecule drugs targeting CXCR4/CXCR2, BTK, IL‑6 and complement pathways. | — |
| 20 | AIRNA Developer of gene therapies for rare and common diseases | $90M | 14 mo ago | Series B | — | Jin Li Co-Founder | — | 6 patents RNA editing using chemically modified antisense oligonucleotides and targeted delivery | 1 partnership |
| 21 | Aeovian Pharmaceuticals Developer of therpeutics for treating rare diseases | $87M | 7 mo ago | Series B | 10 10 → 13 (+30%) · sector median +18% | Stelios Tzannis Founder & COO | — | — | — |
| 22 | Rocket Pharma Developer of gene-modified cell therapies for the treatment of rare diseases 60Funding top 24%Tech top 32%Market top 42%News top 46% All 7 signals ▾Fundingtop 24% Techtop 32% Markettop 42% Newstop 46% Moattop 48% Growthtop 50% Globalbottom 32% Funding: Raised $83.2M across 6 rounds · 6 rounds over 10 yrs (0.6/yr) Tech: 54 technologies across 5/8 capability areas · Cloud: Akamai, Amazon Web Services rank vs industry peers · Fliar Intelligence Index 60/100 | $83M | 17 mo ago | Public | 268 268 → 222 (-17%) · sector median +18% | — | 7 signals ▼56% Earnings · Layoffs · FDA approval | — | 1 internship |
| 23 | Mendra Developer of AI-enabled therapeutics for rare diseases with high unmet need | $82M | 4 mo ago | Series A | — | Joshua Grass Co-Founder & CEO | — | — | — |
| 24 | Glycomine Provider of replacement therapies for rare genetic diseases | $80M | 14 mo ago | Series C | 20 | — | — | 73 patents Patents concentrated in health & consumer products | — |
| 25 | Cogent Biosciences Developer of precision therapy for genetically defined diseases | $77M | 28 mo ago | Public | 164 164 → 294 (+79%) · sector median +18% | Charles Wilson Founder & CEO | 13 signals ▲62% Earnings · Funding · Announcements | 40 patents Small-molecule kinase inhibitors for oncology (PI3K, HER2/ERBB2, JAK2 V617F) | — |
| 26 | Trefoil Therapeutics Developer protein engineering drugs to treat corneal endothelial diseases | $73M | 13 mo ago | Series A | 10 | Michael Blaber Co-Founder | — | — | — |
| 27 | Avalo Therapeutics Developer of therapeutics for rare and immunological diseases | $71M | 19 mo ago | Public | 45 | — | 4 signals ▼69% Earnings · Leadership | — | — |
| 28 | Epicrispr Bio Developer of therapeutics for multiple disease 35Funding top 23%Talent top 38%Moat top 42%Tech bottom 43% All 6 signals ▾Fundingtop 23% Talenttop 38% Moattop 42% Techbottom 43% Globalbottom 37% Marketbottom 32% Funding: Raised $68.0M across 1 rounds · 1 rounds over 1 yrs (1.0/yr) Talent: 2 named leaders on record · 2 C-suite/founder profiles curated rank vs industry peers · Fliar Intelligence Index 35/100 | $68M | 14 mo ago | Series B | — | Lei Qi Founder | — | — | — |
| 29 | Palvella Therapeutics Developer of therapeutics to treat rare diseases | $64M | 18 mo ago | Series D | 21 | Wes Kaupinen Founder & CEO | 16 signals ▲220% Earnings · Announcements · Regulatory | — | 1 job opening |
| 30 | ReAlta Life Sciences Developer of therapeutic drugs for acute inflammatory and rare diseases | $57M | 2 mo ago | Series C | 19 19 → 25 (+32%) · sector median +18% | Neel Krishna Co-Founder | 5 signals ▲400% Leadership | — | — |
| 31 | Actio Biosciences Developer of genetics-led therapies targeting shared biology between rare, common diseases | $55M | 12 mo ago | Series B | 21 | David Goldstein Co-Founder & CEO | — | — | 1 job opening |
| 32 | American Gene Developer of lentivirus therapies for the treatment of cancer and other chronic human diseases 55Funding top 24%Patents top 31%Moat top 35%Talent top 35% All 11 signals ▾Fundingtop 24% Patentstop 31% Moattop 35% Talenttop 35% Innovationtop 37% Growthtop 40% Producttop 40% Techtop 46% Marketbottom 36% Globalbottom 36% Newsbottom 22% Funding: Raised $52.8M across 10 rounds · 10 rounds over 13 yrs (0.8/yr) Patents: 228 patents matched (strict assignee match) · 90 filings since 2022 vs 138 in 2017-21 rank vs industry peers · Fliar Intelligence Index 55/100 | $53M | 7 mo ago | Series B | 31 | Jeff Galvin Chief Executive Officer | — | 228 patents Non-integrating viral vectors for gene delivery and HIV immunotherapy, plus gamma‑delta T‑cell activation | — |
| 33 | Spruce Developer of therapies for rare endocrine disorders | $50M | 8 mo ago | Public | 29 | Javier Szwarcberg CEO | — | 5 patents Patents concentrated in electrical & electronics | — |
| 34 | Celdara Medical Developer of therapeutics for the treatment of multiple diseases 21Funding top 30%Talent bottom 49%Tech bottom 38%Global bottom 32% All 6 signals ▾Fundingtop 30% Talentbottom 49% Techbottom 38% Globalbottom 32% Marketbottom 28% Newsbottom 22% Funding: Raised $46.5M across 37 rounds · 37 rounds over 17 yrs (2.2/yr) Talent: 1 named leader on record · 1 C-suite/founder profile curated rank vs industry peers · Fliar Intelligence Index 21/100 | $46M | 12 mo ago | Funding Raised | 16 16 → 19 (+19%) · sector median +18% | Jake Reder CEO | — | — | — |
| 35 | Immusoft Developer of programmed B cell therapies for protein delivery | $44M | 24 mo ago | Series B | 20 | Matthew Scholz Founder | — | 66 patents Engineered B cells as living factories for therapeutic protein delivery and disease treatment | — |
| 36 | Myrtelle Developer of gene therapies for the treatment of neurological diseases 35Funding top 31%Growth top 35%Moat top 45%Patents top 49% All 9 signals ▾Fundingtop 31% Growthtop 35% Moattop 45% Patentstop 49% Talentbottom 49% Innovationbottom 47% Techbottom 41% Globalbottom 37% Marketbottom 22% Funding: Raised $44.1M across 3 rounds · 3 rounds over 2 yrs (1.5/yr) Growth: Fresh capital in last 24 mo rank vs industry peers · Fliar Intelligence Index 35/100 | $44M | 6 mo ago | Series C | 12 12 → 11 (-8%) · sector median +18% | Mark Pykett CEO | — | 4 patents Therapeutic biotech targeting myelin repair and neurodegenerative disease treatment | — |
| 37 | Artelo Biosciences Modifying lipid-signaling pathways to improve cancer treatments 53Funding top 17%Growth top 20%News top 23%Product top 24% All 9 signals ▾Fundingtop 17% Growthtop 20% Newstop 23% Producttop 24% Innovationtop 43% Talenttop 46% Techbottom 46% Globalbottom 38% Marketbottom 24% Funding: Raised $39.9M across 13 rounds · 13 rounds over 8 yrs (1.6/yr) Growth: Web rank 50,000,000 → 10,000,000 YoY · Fresh capital in last 24 mo rank vs industry peers · Fliar Intelligence Index 53/100 | $40M | 2 mo ago | Public | 7 | Greg Gorgas CEO & President | 8 signals ▲700% Leadership · Board · Funding | — | 1 partnership |
| 38 | RenBio Developer of antibody-based therapeutic solutions for the treatment of cancer & inflammatory diseases | $29M | 21 mo ago | Series A | 10 10 → 8 (-20%) · sector median +18% | David Ho Co-Founder | — | 11 patents Gene therapy delivery devices and plasmid expression vectors for therapeutic proteins/peptides | — |
| 39 | Athos Therapeutics Developer of AI-based therapeutics for treating autoimmune diseases 35AI top 35%Funding top 36%Talent bottom 49%Moat bottom 45% All 6 signals ▾AItop 35% Fundingtop 36% Talentbottom 49% Moatbottom 45% Marketbottom 32% Globalbottom 25% AI: Product described with: ai-based, machine learning Funding: Raised $28.0M across 3 rounds · 3 rounds over 5 yrs (0.6/yr) rank vs industry peers · Fliar Intelligence Index 35/100 | $28M | 2 mo ago | Series A | 18 | Dimitrios Iliopoulos Co-founder | — | — | — |
| 40 | Oisín Biotechnologies Developer of genetically targeted intervention therapy for age-related diseases | $25M | 18 mo ago | Series A | 18 | Gary Hudson Co-Founder & Chairman | — | — | — |
| 41 | GEn1E Developer of therapeutics for the treatment of inflammatory and rare diseases | $17M | 7 mo ago | Series A | 19 | Ritu Lal Co-Founder & CEO | — | — | — |
| 42 | Expression Therapeutics Provider of gene therapy technology for the treatment of hemophilia A | $13M | 16 mo ago | Funding Raised | 21 | — | — | — | — |
| 43 | Modulight Biotherapeutics Developer of an optogenetic platform targeting pathological neural activity to restore balance | $12M | 7 mo ago | Seed | — | Ofer Yizhar Co-Founder & CSO | — | — | 1 award |
| 44 | Bond Biosciences Therapeutics for ion-related diseases using non-absorbed oral treatments 10Funding top 36%Talent top 38%Global bottom 29%Market bottom 11% All 4 signals ▾Fundingtop 36% Talenttop 38% Globalbottom 29% Marketbottom 11% Funding: Raised $11.8M · 6 rounds over 6 yrs (1.0/yr) Talent: 2 named leaders on record · 2 C-suite/founder profiles curated rank vs industry peers · Fliar Intelligence Index 10/100 | $12M | 2 mo ago | Series B | 4 | Cory Berkland Co-Founder & CEO | — | — | — |
| 45 | NexEos Bio Developer of an imaging agent to detect eosinophil-mediated diseases | $10M | 4 mo ago | Series A | — | Steve Tullman Co-Founder & CEO | — | — | — |
| 46 | Hunterian Provider of gene-editing platform based on CRISPR delivery technology | $8M | 16 mo ago | Series A | 6 | Vinod Ranga Founder & CEO | — | — | — |
| 47 | Elixirgen Therapeutics Developer of RNA therapeutics to treat multiple diseases 9Funding top 37%Talent bottom 49%Global bottom 37%Market bottom 21% All 5 signals ▾Fundingtop 37% Talentbottom 49% Globalbottom 37% Marketbottom 21% Techbottom 18% Funding: Raised $7.3M across 11 rounds · 11 rounds over 14 yrs (0.8/yr) Talent: 1 named leader on record · 1 C-suite/founder profile curated rank vs industry peers · Fliar Intelligence Index 9/100 | $7M | 7 mo ago | Series A | 11 11 → 13 (+18%) · sector median +18% | Minoru Ko Co-Founder | — | — | — |
| 48 | Rarity PBC Developer of gene therapies for rare genetic diseases | $5M | 7 mo ago | Seed | — | Donald Kohn Co-Founder | — | — | — |
| 49 | AcuraStem Developer of therapeutics for neurodegenerative diseases | $780K | 1 mo ago | Seed | 14 | Justin Ichida Co-Founder | — | 70 patents Antisense oligonucleotide therapeutics targeting SYF2, PIKfyve, UNC13A gene expression. | 1 expansion |
| 50 | Vogenx Developer of therapeutics for the treatment of serious metabolic and gastrointestinal diseases | $750K | 6 mo ago | Funding Raised | — | James Green Chief Executive Officer | — | 9 patents Patents concentrated in health & consumer products | — |
Top 12 Rare Diseases companies in United States
#1
Series C · Cambridge, Massachusetts · founded 2017 · 38 employees$154.5Mraised▾
Casma Therapeutics is developing therapies for treating rare genetic disorders. They are focused on autophagy of rare genetic diseases which helps in restoring cellular balance and reversing the course of the disease. The technology enables initiation, the formation of the autophagosome, fusion with lysosomes, and lysosomal biogenesis.
- Mar 2026Grant (prize money)$7.6M
- Nov 2022Series C$46M
- Sep 2020Series B$50M
- May 2018Series A$58.5M
#2
Series B · San Diego, California · founded 2003 · 54 employees$109Mraised▾
Developer of therapeutic solutions for the treatment of multiple diseases based on protein degradation technology. The company has developed a protein degradation entity that mainly focused on molecular glue degraders that destroy proteins linked to disease. These degraders can be classified as heterobifunctional that solve critical problems.
- Apr 2026Series B$100M
- Dec 2020Series A$109M
#3
Public · Waltham, Massachusetts · founded 2009 · 34 employees$115.5Mraised▾
Developer of ophthalmic treatments based on its mucosal penetrating product platform. It offers therapies for severe ophthalmic diseases. The company’s MPP drug delivery technology platform allows therapeutic agents to pass through the mucus layer(tears) of the ocular surface, facilitating penetration into deeper tissues of the eye, including the aqueous humor and retina. Its solutions are used in treating severe ophthalmic diseases, including ocular inflammation and wet age-related macular degeneration. Its drug pipeline includes KPI-121, an ophthalmic MPP suspension in development for the treatment of post-surgical inflammation , dry eye , meibomian gland disease. Also developing MPP based topical drugs for AMD (orphan indication). It has applications in the treatment of both anterior and posterior chamber eye diseases.
- Mar 2026Post IPO$1.8M
- Jan 2026Post IPO$2.3M
- Dec 2024Post IPO$10.8M
- Jun 2024Post IPO$12.5M
#4
Series D · Woodland Hills, California · founded 2018$156Mraised▾
Developer of drugs for muscle, bone, and joint diseases. The company's products include AGAO1 for bone fusion, AGA02 for rare diseases, AGA03 for regeneration, AGA04 for osteoporosis, AGA05 for healing, and others. Also, developed many drugs for multiple disease treatments such as nonunion of fractures, osteoarthritis, rare diseases of skeletal muscle, and mineral metabolism diseases caused by diseases or disorders of endocrine organs.
- Feb 2026Series D$130M
- Dec 2024Series C$120M
- Jan 2024Series B$40M
- Oct 2023Series B$46M
#5
Series C · Norfolk, Virginia · founded 2018 · 19 employees$56.5Mraised▾
Developer of therapeutic drugs for acute inflammatory and rare diseases. The company's EPICC peptides are based on research into the human astrovirus, HAstV-1, which causes a non-inflammatory, self-limiting gastroenteritis unique among viruses by inhibiting components of the innate immune system. The company’s pipeline is led by RLS-0071, for the treatment of acute exacerbations of chronic obstructive pulmonary disease and acute graft-versus-host disease.
- Apr 2026Series C$40M
- Dec 2024Series B$25M
- Feb 2023Series B$29.9M
- Apr 2021Series A$20M
#6
Public · Menlo Park, California · founded 2018 · 196 employees$120Mraised▾
Developer of small molecule based therapeutics to treat rare diseases. The company's lead product candidate Maralixibat inhibits a carrier protein called apical sodium-dependent bile acid transporter for reducing systemic levels of bile acids. It is used for the treatment of both Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis. The other product of the company Volixibat is used for the treatment of adult cholestasis.
- Jan 2026Post IPO$68.5M
- Dec 2020Post IPO$10M
- Nov 2018Series A$120M
#7
Public · San Diego, California · founded 2011 · 7 employees$39.9Mraised▾
Modifying lipid-signaling pathways to improve cancer treatments. This clinical-stage pharmaceutical manufacturing company focuses on oncology and endocannabinoid modulation. They develop treatments for conditions like cancer anorexia-cachexia syndrome, utilizing CBD, FABP5 inhibitors, and synthetic cannabinoids. Their pipeline includes ART27.13, ART26.12, and ART12.11. They aim to restore vitality and daily function in patients. The company is involved in medical innovation and cancer research, striving to improve the lives of those facing limited options. They are exploring novel non-opioid treatments for persistent pain and addressing stress-induced depression and anxiety symptoms.
- Mar 2026Post IPO$11M
- Jan 2026Post IPO$500K
- Oct 2025Post IPO$3.7M
- Aug 2025Post IPO$9.5M
#8
Series B · Cambridge, Massachusetts · founded 2022$128Mraised▾
Developer of antibody therapeutics for the treatment of rare diseases. The company's DIAGONAL platform combines proprietary computational and experimental techniques to develop agonist antibody therapies for hereditary hemorrhagic telangiectasia and pulmonary arterial hypertension.
- Jan 2026Series B$125M
- Apr 2024Series A$128M
#9
Series A · Torrance, California · founded 2019 · 18 employees$28Mraised▾
Developer of AI-based therapeutics for treating autoimmune diseases. The company has developed an innovative computational process based on machine learning algorithms that identify novel and safe drug/gene targets for individual patients. The product pipeline includes ATH-63, ATH-105, and ATH-120 for Ulcerative Colitis & Crohn’s Disease.
- Apr 2026Series A$10.3M
- Jan 2024Series A$7.6M
- Nov 2021Series A$20.4M
#10
Series A · San Francisco, California · founded 2026$82Mraised▾
Developer of AI-enabled therapeutics for rare diseases with high unmet need. The company focuses on accelerating clinical development and commercialization of breakthrough medicines. It leverages artificial intelligence to address diseases where there are significant unmet medical needs.
- Jan 2026Series A$82M
#11
Series B · San Francisco, California · founded 2012 · 10 employees$87.4Mraised▾
Developer of therpeutics for treating rare diseases. The company focused on developing selective small molecule inhibitors targeting the mTORC1 pathway for rare genetic and age-related diseases. The company's product line include AV078, is a CNS-penetrant selective mTORC1 inhibitor for TSC-related refractory epilepsy.
- Nov 2025Series B$55M
- Dec 2023Series B$50.4M
- Oct 2019Series A$37M
- Jun 2019Grant (prize money)$472K
#12
Series B · Cambridge, Massachusetts · founded 2016 · 38 employees$143.5Mraised▾
QurAlis is engaged in discovering and developing new therapies for amyotrophic lateral sclerosis (ALS), which is a common form of motor neuron disease. The company has developed Optopatch technology which helps in stimulating the neurons and notes their electrical behavior. Their proprietary method clears toxic proteins and transcranial magnetic stimulation helps in diagnosing ALS by identifying biomarkers such as protein in the blood. In addition, other therapeutic targets like nerve threshold tracking, proteins in the cerebral spinal fluid helps to predict ALS. The company has a preclinical-stage program for ALS drugs targeting the Kv7.2/7.3 channel & CSF cartridge device to remove DPR proteins.
- Aug 2025Series B$20M
- Mar 2023Series B$88M
- Sep 2022Grant (prize money)$300K
- Jul 2020Grant (prize money)$107.4K
Funding & investor landscape
Notable founders
Market landscape — 10 sub-themes by coverage
Share of Rare Diseases coverage by sub-theme.
Regulatory-risk over time rising
Funding & growth over time
funding rising · growth rising
Common questions
Riya reads income statements for fun. She covers the real money in a sector — revenue, growth, who actually earns — and never met a vague claim she didn't want a number for.