Best Gene Therapy Companies in Cambridge, United States
The 38 best Gene Therapy companies in Cambridge, United States in 2026, ranked by funding and momentum. Funding, investors, founders, valuation, tech stack and live signals. Top: Moderna.
Ranked: Best Gene Therapy companies in Cambridge
| # | Company | Disclosed funding | Last round | Employees | Founder | Tech stack | Patents & IP focus | Recent signals |
|---|---|---|---|---|---|---|---|---|
| 1 | Moderna Developer of mRNA-based therapeutics for a wide range of diseases 82Tech top 11%Funding top 15%Market top 22%Partners top 25% All 12 signals ▾Techtop 11% Fundingtop 15% Markettop 22% Partnerstop 25% Talenttop 25% Newstop 31% Moattop 32% Patentstop 34% Globaltop 38% Innovationtop 39% Growthbottom 49% Fin. healthbottom 48% Tech: 57 technologies across 6/8 capability areas · Cloud: Amazon Cloudfront, Amazon Ses Funding: Raised $2.8B across 20 rounds · 20 rounds over 14 yrs (1.4/yr) rank vs industry peers · Fliar Intelligence Index 82/100 | $2.8B | 19 mo ago | 1,312 | Robert Langer Co-Founder | Apple iCloud MailMicrosoft 365TypeScript+24 | 31 patents mRNA therapeutics, nucleic acid modifications, and gene‑therapy vaccines for metabolic diseases | 1 internship |
| 2 | Laronde Developer of RNA therapeutics | $440M | 60 mo ago | 116 | — | — | — | — |
| 3 | ReNAgade Developer of RNA-based medicines to treat multiple diseases | $300M | 39 mo ago | 136 | — | — | — | — |
| 4 | Generation Bio Developer of gene therapies for rare liver and eye diseases | $236M | 80 mo ago | 155 | Phillip Samayoa Co-Founder and Chief Strategy Officer | DrupalApple iCloud MailPHP+20 | 447 patents Synthetic AAV vectors and polyglycerol lipid nanoparticle gene delivery platforms | — |
| 5 | Ring Therapeutics Developer of gene therapy platform by harnessing the human commensal virome | $230M | 42 mo ago | 117 | Tuyen Ong CEO | WordPressBugHerdApple iCloud Mail+17 | — | — |
| 6 | Beam Therapeutics Developer of gene therapy using CRISPR base editing to treat diseases | $222M | 58 mo ago | 340 340 → 596 (+75%) · sector median +4% | Keith Joung Co-Founder | DrupalApple iCloud MailPHP+23 | 556 patents Base editing gene therapies and lipid nanoparticle delivery for genetic disease treatment | — |
| 7 | Verve Developer of gene-editing therapies for treating coronary artery disease | $216M | 67 mo ago | 255 | J Joung Co-Founder | DrupalPHPAmazon Web Services+21 | 14 patents Privacy‑preserving AI profiling, gesture‑based demographics, and synthetic location generation for mobile apps | — |
| 8 | Editas Medicine Developer of therapeutics for rare genetic diseases | $210M | 32 mo ago | 257 257 → 183 (-29%) · sector median +4% | Cynthia Collins Ex-CEO & President | DrupalPHPBootstrap+25 | 713 patents CRISPR-based gene editing therapies for blood disorders and immune cell modulation | — |
| 9 | Bluebird Bio Developer of gene therapies for severe genetic and rare diseases | $136M | 51 mo ago | 508 508 → 312 (-39%) · sector median +4% | — | DrupalApple iCloud MailPHP+35 | 631 patents Gene and cell therapy platforms for hemoglobin disorders and CAR‑T immunotherapy | — |
| 10 | City Therapeutics Developer of gene therapies for multiple diseases | $135M | 23 mo ago | — | John Maraganore Co-Founder | Cart FunctionalitySquarespace CommerceSquarespace+19 | — | — |
| 11 | Dimension Therapeutics Provider of gene therapy for rare diseases | $120M | 136 mo ago | 53 | — | — | — | — |
| 12 | Alltrna Developer of tRNA medicines for various therapeutic areas | $109M | 37 mo ago | 46 46 → 48 (+4%) · sector median +4% | Theonie Anastassiadis Co-Founder | — | — | — |
| 13 | Voyager Therapeutics Neurogenetic medicine innovator, developing transformative treatments for neurological diseases | $105M | 32 mo ago | 183 183 → 161 (-12%) · sector median +4% | Steven Paul Co-Founder | WordPressApple iCloud MailMySQL+21 | 790 patents Engineered AAV gene‑therapy vectors and delivery systems for neurodegenerative disease treatment | — |
| 14 | Strand Therapeutics Provider of platform to deliver mRNA therapeutics for multi functional treatments 64Growth top 13%Funding top 24%Moat top 31%News top 41% All 7 signals ▾Growthtop 13% Fundingtop 24% Moattop 31% Newstop 41% Talenttop 41% Globalbottom 39% Marketbottom 38% Growth: Headcount 16 → 47 (+194%) across vintages · Fresh capital in last 24 mo Funding: Raised $103M across 6 rounds · 6 rounds over 7 yrs (0.9/yr) rank vs industry peers · Fliar Intelligence Index 64/100 | $103M | 15 mo ago | 47 | Tasuku Kitada Co-founder | — | — | 2 awards |
| 15 | Solid Biosciences Developer of gene therapy platform for Duchenne muscular dystrophy 53Patents top 20%Funding top 24%Innovation top 31%Tech top 33% All 10 signals ▾Patentstop 20% Fundingtop 24% Innovationtop 31% Techtop 33% Moattop 38% Newstop 48% Marketbottom 49% Growthbottom 44% Globalbottom 42% Fin. healthbottom 32% Patents: 60 patents matched (strict assignee match) · 43 filings since 2022 vs 17 in 2017-21 Funding: Raised $92.5M across 7 rounds · 7 rounds over 11 yrs (0.6/yr) rank vs industry peers · Fliar Intelligence Index 53/100 | $92M | 6 mo ago | 101 | — | WordPressApple iCloud MailMySQL+22 | 60 patents AAV gene‑therapy vector production, formulation, and therapeutic constructs for cardiac disease | — |
| 16 | Avrobio Developer of gene therapies to treat genetic diseases | $85M | 103 mo ago | 114 | — | — | 42 patents Enzyme replacement and gene therapies for lysosomal storage and neurodegenerative diseases | — |
| 17 | Intellia Therapeutics Developer of therapeutics based on CRISPR/Cas9 technology for multiple diseases | $85M | 132 mo ago | 526 526 → 495 (-6%) · sector median +4% | Tenzin Yangdon Co-Founder | WordPressMySQLPHP+25 | 871 patents CRISPR genome editing therapeutics for genetic diseases and protein replacement | — |
| 18 | Skylark Bio Developer of therapeutics for ear disorders | $41M | 49 mo ago | — | Casey Maguire Co-Founder | — | — | 1 job opening · 1 partnership |
| 19 | Sarepta Developer of RNA-targeted therapeutics for the treatment of rare and infectious diseases | $40M | 78 mo ago | 820 820 → 1,163 (+42%) · sector median +4% | — | DrupalApple iCloud MailPHP+25 | — | 2 awards |
| 20 | Kernal Biologics Developer of mRNA therapeutics for immuno-oncology using deep learning technology | $35M | 11 mo ago | 18 18 → 22 (+22%) · sector median +4% | Burak Yilmaz Co-Founder | — | 27 patents Targeted lipid nanoparticle delivery systems for oncoselective cancer therapeutics | — |
| 21 | Agilis Biotherapeutics Provider of gene therapy platform for rare monogenic diseases | $21M | 114 mo ago | 14 | — | — | — | — |
| 22 | Onkaido Developer of mRNA-based oncology treatments | $20M | 151 mo ago | 1,190 | Stephen Hoge President & CEO | — | — | — |
| 23 | Alnylam Developer of RNA interference-based therapeutics for multiple disorders | $17M | 72 mo ago | 2,100 2,100 → 2,892 (+38%) · sector median +4% | Paul Schimmel Co-Founder | DrupalApple iCloud MailMariaDB+32 | — | — |
| 24 | Cirrus Therapeutics Developer of therapies designed to extend ocular healthspan and restore vision | $11M | 13 mo ago | — | Andrew Dick Co-Founder | — | — | 1 award |
| 25 | Hunterian Provider of gene-editing platform based on CRISPR delivery technology | $8M | 19 mo ago | 6 | Vinod Ranga Founder & CEO | — | — | — |
| 26 | Factor Bioscience Developer of genomic technologies to treat disease and improve health | $1M | 11 mo ago | 10 | Christopher Rohde Co-Founder & CTO | — | 289 patents Lipid-based nucleic acid delivery vectors for gene and protein expression therapies | — |
| 27 | Regel Therapeutics Developer of targeted gene therapies for genetic diseases | $1M | 35 mo ago | 17 | Navneet Matharu Founder/CSO | — | — | — |
| 28 | gensaic Developer of gene therapies for multiple diseases | $669K | 19 mo ago | 10 | Jonathan Hus Co-Founder | WebflowSlickjQuery+10 | 4 patents Targeted tissue delivery formulations and therapeutic phage-derived nanoparticle platforms | — |
| 29 | Combined Therapeutics Early stage biopharma company | $250K | 105 mo ago | 6 | Romain Micol President and co-founder CEO | — | — | — |
Top 12 Gene Therapy companies in Cambridge
#1
Public · Cambridge, Massachusetts · founded 2014 · 101 employees$92.5Mraised▾
Developer of gene therapy platform for Duchenne muscular dystrophy. The lead candidate SGT-001 is Microdystrophin, a synthetic version of the dystrophin gene which stabilizes the muscle membrane and dystrophin-glycoprotein complex. Also, developed a soft wearable device for mobility and help preserve muscle function.
- Mar 2026Post IPO$240M
- Jan 2024Post IPO$108.9M
- Dec 2020Post IPO$90M
- Oct 2020Post IPO$40M
#2
Public · Cambridge, Massachusetts · founded 2010 · 1312 employees$2.8Braised▾
Developer of mRNA-based therapeutics for a wide range of diseases. The company produces human proteins, antibodies, or vaccines inside patient cells, which are in turn active intracellularly or secreted. The company is also developing vaccines in different disease areas such as infectious diseases, cancer, rare diseases, and COVID-19.
- Jan 2025Grant (prize money)$590M
- Jul 2024Grant (prize money)$176M
- Oct 2020Grant (prize money)$56M
- May 2020Post IPO—
#3
Series A · Cambridge, Massachusetts · founded 2016 · 18 employees$35.4Mraised▾
Developer of mRNA therapeutics for immuno-oncology using deep learning technology. The company is developing platforms using deep learning algorithms and big data to design better mRNA therapeutics. The platform helps in three processes: codon-engineering, smart designing and non-immunogenic selection.
- Oct 2025Grant (prize money)$48M
- Feb 2022Series A$25M
- Jul 2021Seed$10M
- Oct 2018Grant (prize money)—
#4
Series B · Cambridge, Massachusetts · founded 2017 · 47 employees$103.1Mraised▾
Provider of a platform to deliver mRNA therapeutics for multi-functional treatments. The programming platform detects the location and enhances the cell type protein expression in the patient's body to optimize the immunotherapy treatments.
- May 2025Series B$153M
- Nov 2022Series A$45M
- Jun 2021Series A$52.1M
- May 2021Grant (prize money)$399.5K
#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
Seed · Cambridge, Massachusetts · founded 2021$11Mraised▾
Developer of therapies designed to extend ocular healthspan and restore vision. The company focuses on age-related macular degeneration treatments by rejuvenating the eye’s defenses across multiple pathways. Their pioneering research offers a preventative and restorative therapy with the potential to slow or halt disease progression and reverse early signs of vision loss. The company’s therapeutic approach delivers genetic instructions directly into the eye, restoring balance, reducing inflammation, and protecting cells essential for clear vision.
- Aug 2025Seed$11M
#7
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
#8
Seed · Cambridge, Massachusetts · founded 2011 · 10 employees$1.4Mraised▾
Developer of genomic technologies to treat disease and improve health. The company's gene-editing and cell-reprogramming technologies enable the generation of clonal lines of pluripotent stem cells development of regenerative cell therapies. Also, it has developed disease-focused technologies to create treatments for cancer, genetic, infectious, and degenerative diseases.
- Sep 2025Seed$1M
- Sep 2013Grant (prize money)$150K
- Sep 2012Grant (prize money)$79.2K
- Sep 2012Grant (prize money)$150K
#9
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
#10
Public · Cambridge, Massachusetts · founded 2014 · 183 employees$105Mraised▾
Neurogenetic medicine innovator, developing transformative treatments for neurological diseases. This biotechnology company leverages human genetics to modify and cure neurological diseases. The company focuses on genetic medicines for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and other central nervous system diseases. Revolutionizing delivery across the blood-brain barrier is a key focus, utilizing a proprietary AAV capsid discovery platform to generate novel capsids. The company aims to advance its pipeline by achieving preclinical and clinical milestones, establishing proof-of-biology and proof-of-concept. The goal is to transform the treatment of neurological diseases, creating disease-modifying neurogenetic medicines by identifying validated targets and advancing multiple therapeutic modalities. The company is dedicated to making transformative treatments and cures available to those afflicted with neurological diseases.
- Jan 2024Post IPO$20M
- Mar 2023Post IPO$39M
- Jan 2019Post IPO$50M
- Apr 2015Series B$60M
#11
Acquired · Cambridge, Massachusetts · founded 2021 · 136 employees$300Mraised▾
Developer of RNA-based medicines to treat multiple diseases. The company combines novel RNA delivery platforms allowing for coding, editing, and gene insertion to develop new medicines.
- May 2023Series A$300M
#12
Series C · Cambridge, Massachusetts · founded 2018 · 117 employees$230Mraised▾
Developer of gene therapy platform by harnessing the human commensal virome
- Mar 2023Series C$86.5M
- Jul 2021Series B$117M
Funding & investor landscape
Notable founders
Market landscape — 10 sub-themes by coverage
Share of Gene Therapy coverage by sub-theme.
Regulatory-risk over time rising
Funding & growth over time
funding rising · growth rising
How research on Gene Therapy in Cambridge is moving
A read on the academic research around Gene Therapy in Cambridge — which themes are gaining ground, the institutions and researchers driving them, and how the tone is shifting. Based on 44 papers.
- University of Cambridge41
- Wellcome/MRC Cambridge Stem Cell Institute3
- Cambridge School1
- Cambridge University Hospitals NHS Foundation Trust1
- Cambridge–MIT Institute1
- Transfection5
- Transduction (biophysics)5
- Gene delivery4
- Transgene4
- Genetic enhancement3
- Genetic enhancement6
- Viral vector4
- Virology4
- Gene delivery3
- Immune system3
- Genetic enhancement2
- Viral vector2
- CRISPR1
- Genome editing1
- Cas91
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.