Best Rare Diseases Companies in Cambridge, United States
The 46 best Rare Diseases companies in Cambridge, United States in 2026, ranked by funding and momentum. Funding, investors, founders, valuation, tech stack and live signals. Top: Generation Bio.
Ranked: Best Rare Diseases companies in Cambridge
| # | Company | Disclosed funding | Last round | Employees | Founder | Patents & IP focus | Recent signals |
|---|---|---|---|---|---|---|---|
| 1 | Generation Bio Developer of gene therapies for rare liver and eye diseases | $236M | 79 mo ago | 155 | Phillip Samayoa Co-Founder and Chief Strategy Officer | 447 patents Synthetic AAV vectors and polyglycerol lipid nanoparticle gene delivery platforms | — |
| 2 | Cyclerion Developer of therapies for treating sickle cell disease and diabetic nephropathy | $217M | 62 mo ago | 32 | — | — | — |
| 3 | Editas Medicine Developer of therapeutics for rare genetic diseases | $210M | 31 mo ago | 257 257 → 183 (-29%) · sector median +40% | Cynthia Collins Ex-CEO & President | 713 patents CRISPR-based gene editing therapies for blood disorders and immune cell modulation | — |
| 4 | Goldfinch Developer of precision therapies for the treatment of genetic kidney disease | $157M | 73 mo ago | 68 | — | 12 patents Multispectral/hyperspectral imaging systems for meat quality and composition analysis | — |
| 5 | 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 | 5 mo ago | 38 | — | — | — |
| 6 | 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 | 12 mo ago | 38 38 → 87 (+129%) · sector median +40% | Kasper Roet Founder & CEO | 69 patents Antisense oligonucleotide therapeutics targeting UNC13A for neurological disease treatment | — |
| 7 | Bluebird Bio Developer of gene therapies for severe genetic and rare diseases | $136M | 50 mo ago | 508 508 → 312 (-39%) · sector median +40% | — | 631 patents Gene and cell therapy vectors, genome editing, and CAR‑T for blood disorders | — |
| 8 | City Therapeutics Developer of gene therapies for multiple diseases | $135M | 22 mo ago | — | John Maraganore Co-Founder | — | — |
| 9 | 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 | 7 mo ago | — | Alexey Lugovskoy CEO | — | — |
| 10 | Agios Pharmaceuticals Developer of therapeutics in the field of cancer metabolism and rare genetic disorders of metabolism | $120M | 106 mo ago | 383 383 → 643 (+68%) · sector median +40% | — | 1,395 patents Metabolic enzyme modulators for hematologic and metabolic disease therapy | — |
| 11 | Dimension Therapeutics Provider of gene therapy for rare diseases | $120M | 136 mo ago | 53 | — | — | — |
| 12 | Biopure Mold remediation, water damage restoration, and infectious disease control services | $119M | 153 mo ago | — | Zafiris Zafirelis CEO | 40 patents Developing and stabilizing hemoglobin-based blood substitutes and red cell purification technologies. | — |
| 13 | Inozyme Pharma Developer of treatments for rare and debilitating metabolic diseases | $116M | 88 mo ago | 59 | Axel Bolte Co-Founder & CEO | — | — |
| 14 | Fulcrum Therapeutics Developing small molecules for control mechanisms and genes regulation for the treatment of rare diseases | $115M | 74 mo ago | 104 104 → 65 (-38%) · sector median +40% | Robert Gould CEO | — | — |
| 15 | Sigilon Developer of therapeutics to treat chronic diseases | $104M | 77 mo ago | 66 | Omid Veiseh Co-Founder | — | — |
| 16 | Zafgen Health and wellness resource providing information on products and health topics | $101M | 148 mo ago | 7 | — | 364 patents Design and synthesis of MetAP-2 inhibitory small‑molecule pharmaceuticals for metabolic disease | — |
| 17 | Apic Bio Developer of gene therapies for rare diseases | $97M | 53 mo ago | 30 | Christian Mueller Co-Founder and Board Member | — | — |
| 18 | Imara Small molecules to treat rare blood diseases such as sickle cell disease | $94M | 89 mo ago | 41 | Rahul Ballal CEO | 76 patents Pharmaceutical PDE9 inhibitor compounds, crystalline forms, and related formulation methods. | — |
| 19 | Cydan Online platform providing Togel Hongkong results, data, and live draws | $91M | 106 mo ago | 7 | — | — | — |
| 20 | AIRNA Developer of gene therapies for rare and common diseases | $90M | 16 mo ago | — | Jin Li Co-Founder | 6 patents Modified antisense oligonucleotides for targeted RNA editing and delivery. | — |
| 21 | Avrobio Developer of gene therapies to treat genetic diseases | $85M | 102 mo ago | 114 | — | 42 patents Enzyme replacement and gene therapies for lysosomal storage and neurodegenerative diseases | — |
| 22 | Tisento Therapeutics Developer of therapeutics for the treatment of rare genetic diseases | $81M | 36 mo ago | — | Peter Hecht CEO | — | — |
| 23 | Ra Pharmaceuticals Developing complement system inhibitors (peptides) for rare diseases. | $77M | 133 mo ago | 92 | — | 437 patents Small‑molecule modulators of CXCR4/CXCR2, Btk, IL‑6 and complement pathways | — |
| 24 | Cogent Biosciences Developer of precision therapy for genetically defined diseases | $77M | 30 mo ago | 164 164 → 294 (+79%) · sector median +40% | Charles Wilson Founder & CEO | 40 patents Small-molecule kinase inhibitors targeting PI3K, ERBB2, and JAK2 for cancer therapy | — |
| 25 | Mitobridge Small molecule drugs for mitochondrial dysfunction | $45M | 154 mo ago | 28 | Kazumi Shiosaki Founder & CEO | 290 patents Small‑molecule therapeutics for metabolic (PPAR) and DNA repair (PARP) targets. | — |
| 26 | Gemini Therapeutics Developer of precision therapeutics for dry AMD and rare genetic diseases | $45M | 106 mo ago | 45 | — | — | — |
| 27 | Sarepta Developer of RNA-targeted therapeutics for the treatment of rare and infectious diseases | $40M | 77 mo ago | 820 820 → 1,163 (+42%) · sector median +40% | — | — | 2 awards |
| 28 | Edimer Pharma Therapeutics for rare dermatology diseases | $40M | 156 mo ago | 5 | Neil Kirby CEO | — | — |
| 29 | Sanofi Genzyme Developer of antibody drug conjugates for the treatment of multiple diseases | $37M | — | 10,000 | David Meeker CEO & President | — | — |
| 30 | Synlogic Therapeutics Developer of drugs using engineered microbes | $34M | 86 mo ago | 85 | James Collins Co-Founder | — | — |
| 31 | Comet Therapeutics Developer of enzyme substitution therapy for neurological diseases | $28M | 86 mo ago | 4 | Enej Kuscer Founder | — | — |
| 32 | Agilis Biotherapeutics Provider of gene therapy platform for rare monogenic diseases | $21M | 113 mo ago | 14 | — | — | — |
| 33 | Alnylam Developer of RNA interference-based therapeutics for multiple disorders | $17M | 72 mo ago | 2,100 2,100 → 2,892 (+38%) · sector median +40% | Paul Schimmel Co-Founder | — | — |
| 34 | Hunterian Provider of gene-editing platform based on CRISPR delivery technology | $8M | 18 mo ago | 6 | Vinod Ranga Founder & CEO | — | — |
| 35 | Censa Pharmaceuticals Developing drugs for Tetrahydrobiopterin deficiency and other rare CNS disorders | $3M | 105 mo ago | 2 | Jonathan Reis CEO & Managing Director | — | — |
Top 12 Rare Diseases companies in Cambridge
#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 · 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
#3
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
#4
Series B · Cambridge, Massachusetts · founded 2021$90Mraised▾
Developer of gene therapies for rare and common diseases. It offers RNA editing therapeutics based on a platform RESTORE+ to treat the inherited genetic disease alpha-1 antitrypsin deficiency (AATD).
- Apr 2025Series B$155M
- Jul 2024Series A$60M
- Sep 2023Series A$30M
#5
Series A · Cambridge, Massachusetts · founded 2006$135Mraised▾
Developer of gene therapies for multiple diseases. It offers RNAi therapeutics based on next-generation engineering of small interfering RNAs for patients across multiple disease indications.
- Oct 2024Series A$135M
#6
Series A · Cambridge, Massachusetts · founded 2004 · 6 employees$8.1Mraised▾
Provider of gene-editing platform based on CRISPR delivery technology. The company offers proprietary CRISPR delivery technology through a single adeno-associated virus (AAV) to cure genetic diseases by changing DNA mutations. They use a two-for-one genetic control element to overcome the problem of delivering CRISPR through AAV. Mutations in skeletal and cardiac muscle, lungs, brain, and other tissues can be diagnosed using CRISPR technology.
- Feb 2025Grant (prize money)$350K
- Jan 2025Grant (prize money)$320K
- Sep 2023Grant (prize money)$350K
- Aug 2022Grant (prize money)$320K
#7
Public · Cambridge, Massachusetts · founded 2013 · 257 employees$210Mraised▾
Developer of therapeutics for rare genetic diseases. It has developed a novel class of genome editing therapeutics using CRISPR/Cas genome editing technology. It is utilized for the treatment of rare genetic eye diseases including Leber congenital amaurosis, and usher syndrome. It is also used for the treatment of beta-thalassemia,& Duchenne muscular dystrophy, sickle cell anemia, cystic fibrosis, alpha 1-antitrypsin deficiency, and many more.
- Dec 2023Post IPO$2M
- Aug 2015Series B$120M
- Jul 2015Grant (prize money)$220.4K
- May 2015PE$47M
#8
Public · Cambridge, Massachusetts · founded 2014 · 164 employees$77Mraised▾
Cogent Biosciences is a developer of precision therapy for treating diseases like cancer, rare diseases, autoimmune conditions & others. The company is developing solutions to treat genetically driven diseases & its lead therapeutic product PLX9486 is a precision kinase inhibitor designed to selectively and potently inhibit the KIT D816V mutation. It is also developing PLX9486 to treat gastrointestinal stromal tumors (GIST) which is dependent on oncogenic KIT signaling.
- Feb 2024Post IPO—
- Jul 2020Post IPO$104.4M
- Mar 2020Post IPO$25M
- Mar 2017Venture Debt$15M
#9
Series A · Cambridge, Massachusetts · founded 2023$81Mraised▾
Developer of therapeutics for the treatment of rare genetic diseases. The company's product line include zagociguat a brain-penetrant sGC stimulator, which is advancing in MELAS and other genetic mitochondrial diseases.
- Jul 2023Series A$81M
#10
Acquired · Cambridge, Massachusetts · founded 1992 · 508 employees$135.7Mraised▾
Developer of gene therapies for severe genetic and rare diseases. The company's products include Lenti-D for Childhood Cerebral Adrenoleukodystrophy (ALD) and LentiGlobin for rare hemoglobinopathies. The company's other products for multiple myeloma and HPV-associated cancer are in the preclinical stage. The company is also using homing endonuclease and megaTAL gene-editing technologies in a variety of potential applications and disease areas, including hematology and oncology.
- Jun 2022Post IPO—
- Sep 2021Post IPO$75M
- Oct 2012Grant (prize money)$9.3M
- Jul 2012Series D$60M
#11
Public · Cambridge, Massachusetts · founded 2018 · 32 employees$217.3Mraised▾
Cyclerion is involved in developing therapies for treating sickle cell disease (SCD) and diabetic nephropathy. They are focussed on the nitric oxide-cyclic guanosine monophosphate (cGMP) signaling pathway. Their lead candidate, Olinciguat, which is a vascular guanylate cyclase agonist. The candidate is in phase II clinical trial. They have granted Orphan Drug Designation for SCD by the U.S. Food and Drug Administration.
- Jun 2021Post IPO$18M
- Aug 2020Grant (prize money)—
- Aug 2020Post IPO$24.2M
- Apr 2019Post IPO$175M
#12
Deadpooled · Cambridge, Massachusetts · founded 2017 · 30 employees$96.8Mraised▾
Developer of gene therapies for rare diseases. The company is working on treatments for Alpha-1 Antitrypsin Deficiency (Alpha-1, or AATD) and genetic Amyotrophic Lateral Sclerosis (ALS) and has developed an AAV delivered Dual Function vector(df-AAV), known as APB-101. Their df-AAV platform allows treatment of other diseases with complex mechanisms where the mutant gene product must be reduced and the normal gene product must be augmented.
- Mar 2022Series B$4.5M
- Nov 2020Series B$42.3M
- Jan 2019Series A$40M
- 2018Seed$10M
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.