Radar · Gene Therapy in Massachusetts
Best Gene Therapy Companies in Massachusetts, United States
The 76 best Gene Therapy companies in Massachusetts, United States in 2026, ranked by funding and momentum. Funding, investors, founders, valuation, tech stack and live signals. Top: Moderna.
By Riya SenguptaFinancials
76companies tracked
$7.7Bdisclosed funding
0unicorns
58have raised
Ranked: Best Gene Therapy companies in Massachusetts
| # | 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 | 17 mo ago | 1,312 | Robert Langer Co-Founder | Apple iCloud MailMicrosoft 365TypeScript+24 | 31 patents mRNA therapeutics, nucleic‑acid modifications, and gene‑therapy vectors for metabolic diseases | 1 internship |
| 2 | Laronde Developer of RNA therapeutics | $440M | 57 mo ago | 116 | — | — | — | — |
| 3 | ReNAgade Developer of RNA-based medicines to treat multiple diseases | $300M | 37 mo ago | 136 | — | — | — | — |
| 4 | Generation Bio Developer of gene therapies for rare liver and eye diseases | $236M | 77 mo ago | 155 | Phillip Samayoa Co-Founder and Chief Strategy Officer | DrupalApple iCloud MailPHP+20 | 447 patents Non‑viral gene delivery platforms: synthetic AAV vectors, polyglycerol lipid nanoparticles, cell‑free DNA synthesis | — |
| 5 | Ring Therapeutics Developer of gene therapy platform by harnessing the human commensal virome | $230M | 39 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 | 56 mo ago | 340 340 → 596 (+75%) · sector median +26% | Keith Joung Co-Founder | DrupalApple iCloud MailPHP+23 | 556 patents Base editing gene therapies delivered via ionizable lipid nanoparticles for metabolic and infectious diseases | 1 internship |
| 7 | Verve Developer of gene-editing therapies for treating coronary artery disease | $216M | 65 mo ago | 255 | J Joung Co-Founder | DrupalPHPAmazon Web Services+21 | 14 patents Privacy‑preserving gesture‑based demographic profiling and synthetic location generation for mobile ads | — |
| 8 | Editas Medicine Developer of therapeutics for rare genetic diseases | $210M | 29 mo ago | 257 257 → 183 (-29%) · sector median +26% | Cynthia Collins Ex-CEO & President | DrupalPHPBootstrap+25 | 713 patents CRISPR-based gene editing therapeutics for blood disorders and immune cell engineering | — |
| 9 | Skyhawk Therapeutics Developer of RNA-modifying drug candidates for the treatment of multiple disorders | $181M | 57 mo ago | 86 | — | WixSentryBootstrap+14 | 229 patents RNA splicing modulators for treating neurodegenerative diseases | 1 launch |
| 10 | Akouos Developer of gene therapies to restore and preserve hearing capabilities in genetically defined populations | $163M | 75 mo ago | 92 | Manny Simons Founder & CEO | — | 279 patents Gene‑therapy and inner‑ear drug delivery for non‑age‑related sensorineural hearing loss. | — |
| 11 | Translate Bio Developer of RNA therapeutics to activate therapeutic protein expression | $138M | 72 mo ago | 209 | — | — | — | — |
| 12 | Bluebird Bio Developer of gene therapies for severe genetic and rare diseases | $136M | 48 mo ago | 508 508 → 312 (-39%) · sector median +26% | — | DrupalApple iCloud MailPHP+35 | 631 patents Gene and cell therapy vectors, genome editing, and CAR‑T for blood disorders | — |
| 13 | City Therapeutics Developer of gene therapies for multiple diseases | $135M | 20 mo ago | — | John Maraganore Co-Founder | Cart FunctionalitySquarespace CommerceSquarespace+19 | — | — |
| 14 | Stoke Therapeutics Developer of antisense oligonucleotide drugs for treating inherited diseases by targeting RNA splicing | $131M | 92 mo ago | 117 117 → 224 (+91%) · sector median +26% | Adrian Krainer Co-Founder & Director | DrupalApple iCloud MailPHP+27 | 275 patents Antisense oligonucleotide therapeutics targeting OPA1 and RNA decay pathways | 2 awards |
| 15 | Homology Medicines Developer of gene therapies for rare genetic diseases | $127M | 67 mo ago | 221 | Frank Mathias CEO | — | 162 patents Engineering AAV gene therapy vectors and capsids for liver‑detargeted PAH restoration | — |
| 16 | OncXerna Developer of therapeutics to treat cancer | $126M | 51 mo ago | 18 | — | — | — | — |
| 17 | Dimension Therapeutics Provider of gene therapy for rare diseases | $120M | 134 mo ago | 53 | — | — | — | — |
| 18 | Comanche Developer of RNA therapeutics to treat preeclampsia | $111M | 29 mo ago | 17 | Mike Young Co-Founder & President | WordPressMySQLPHP+23 | — | — |
| 19 | Atalanta Therapeutics Developer of RNAi therapeutics to treat neurodegenerative diseases | $110M | 16 mo ago | 62 | Neal Aronin Co-Founder | WordPressApple iCloud MailMicrosoft 365+29 | — | — |
| 20 | Alltrna Developer of tRNA medicines for various therapeutic areas | $109M | 34 mo ago | 46 46 → 48 (+4%) · sector median +26% | Theonie Anastassiadis Co-Founder | — | — | — |
| 21 | Dicerna Pharmaceuticals Developer of drugs by using RNA interference technology platform | $106M | 90 mo ago | 337 | — | — | 992 patents RNAi therapeutics targeting disease-related genes for metabolic and infectious disorders | — |
| 22 | Voyager Therapeutics Neurogenetic medicine innovator, developing transformative treatments for neurological diseases | $105M | 29 mo ago | 183 183 → 161 (-12%) · sector median +26% | Steven Paul Co-Founder | WordPressApple iCloud MailMySQL+21 | 790 patents Engineered AAV gene therapy vectors for neurodegenerative and metabolic disorder treatment | — |
| 23 | 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 | 13 mo ago | 47 | Tasuku Kitada Co-founder | — | — | 2 awards |
| 24 | 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 | 3 mo ago | 101 | — | WordPressApple iCloud MailMySQL+22 | 60 patents AAV gene‑therapy vector production, formulation, and cardiomyopathy therapeutic constructs | — |
| 25 | Ascidian Therapeutics Developer of RNA therapeutics to treat genetic diseases | $90M | 31 mo ago | 37 37 → 48 (+30%) · sector median +26% | Michael Ehlers Founder & CEO | — | — | 1 job opening · 4 partnerships |
| 26 | Avrobio Developer of gene therapies to treat genetic diseases | $85M | 100 mo ago | 114 | — | — | 42 patents Enzyme replacement and gene therapies for lysosomal storage and neurodegenerative diseases | — |
| 27 | Intellia Therapeutics Developer of therapeutics based on CRISPR/Cas9 technology for multiple diseases | $85M | 129 mo ago | 526 526 → 495 (-6%) · sector median +26% | Tenzin Yangdon Co-Founder | WordPressMySQLPHP+25 | 871 patents CRISPR genome editing therapeutics for genetic diseases and protein replacement | — |
| 28 | Stylus Medicine Developer of gene-based therapeutics for multiple diseases | $85M | 13 mo ago | 21 | — | — | — | — |
| 29 | WAVE Life Sciences RNA medicines to transform human health, treating rare and common disorders | $84M | 130 mo ago | 233 233 → 381 (+64%) · sector median +26% | Paul Bolno President and CEO | WordPressApple iCloud MailAddsearch+22 | 128 patents Therapeutic oligonucleotides for RNA interference, exon skipping, and gene modulation. | 1 launch · 1 partnership |
| 30 | INTERGALACTIC THERAPEUTICS Developer of gene therapies for the treatment of multiple diseases | $75M | 56 mo ago | 34 | — | — | — | — |
| 31 | Alcyone Developer of gene therapies to treat neurological conditions | $56M | 15 mo ago | 32 | PJ Anand Founder | WordPressMySQLPHP+12 | — | — |
| 32 | NextRNA Developer of RNA-targeted therapeutics for multiple areas | $56M | 51 mo ago | 32 | Leon Lamas Co-Founder | — | — | — |
| 33 | Phio Pharmaceuticals Developer of RNAi-based therapeutics for cancer immunology | $55M | 36 mo ago | 12 12 → 16 (+33%) · sector median +26% | — | WordPressMySQLPHP+18 | — | — |
| 34 | LogicBio Developer of viral vectors for the treatment of genetic and infectious diseases | $49M | 83 mo ago | 62 | Adi Barzel Founder | — | — | — |
| 35 | 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 | 12 12 → 11 (-8%) · sector median +26% | Mark Pykett CEO | WordPressMySQLPHP+20 | 4 patents Therapeutic agents targeting myelin repair and neurodegenerative disease treatment | — |
| 36 | Skylark Bio Developer of therapeutics for ear disorders | $41M | 46 mo ago | — | Casey Maguire Co-Founder | — | — | 1 job opening · 1 partnership |
| 37 | Sarepta Developer of RNA-targeted therapeutics for the treatment of rare and infectious diseases | $40M | 75 mo ago | 820 820 → 1,163 (+42%) · sector median +26% | — | DrupalApple iCloud MailPHP+25 | — | 1 internship · 2 awards |
| 38 | Carbon Biosciences Venture capital firm focused on noval gene therapy | $38M | 48 mo ago | 21 | John Engelhardt Co-Founder | — | — | — |
| 39 | Kernal Biologics Developer of mRNA therapeutics for immuno-oncology using deep learning technology | $35M | 8 mo ago | 18 18 → 22 (+22%) · sector median +26% | Burak Yilmaz Co-Founder | — | 27 patents Targeted lipid-based delivery systems for oncoselective cancer therapeutics | — |
| 40 | Aldena Therapeutics Developer of gene therapies for dermatological conditions | $30M | 37 mo ago | — | Thibaud Portal CEO | — | — | — |
| 41 | hC Bioscience Developer of RNA-based therapeutics to treat cancer and rare diseases | $24M | 54 mo ago | 31 | Leslie Williams Co-Founder & CEO | — | 102 patents Patents concentrated in health & consumer products | — |
| 42 | Agilis Biotherapeutics Provider of gene therapy platform for rare monogenic diseases | $21M | 111 mo ago | 14 | — | — | — | — |
| 43 | Alnylam Developer of RNA interference-based therapeutics for multiple disorders | $17M | 70 mo ago | 2,100 2,100 → 2,892 (+38%) · sector median +26% | Paul Schimmel Co-Founder | DrupalApple iCloud MailMariaDB+32 | — | — |
| 44 | IMEL Developer of cell-based therapies to treat mitochondrial disorders | $15M | 55 mo ago | 5 | Silvia Noiman Founder & CEO | — | — | — |
| 45 | Cirrus Therapeutics Developer of therapies designed to extend ocular healthspan and restore vision | $11M | 10 mo ago | — | Andrew Dick Co-Founder | — | — | 1 award |
| 46 | Hunterian Provider of gene-editing platform based on CRISPR delivery technology | $8M | 16 mo ago | 6 | Vinod Ranga Founder & CEO | — | — | — |
| 47 | Remedium Bio Developer of gene therapies for a broad range of diseases 7Funding top 37%Moat top 48%Global bottom 36%Tech bottom 22% All 5 signals ▾Fundingtop 37% Moattop 48% Globalbottom 36% Techbottom 22% Marketbottom 20% Funding: Raised $5.6M across 5 rounds · 5 rounds over 5 yrs (1.0/yr) Moat: Editorial + unicorn rating 4/10 · 10 curated direct competitors tracked rank vs industry peers · Fliar Intelligence Index 7/100 | $6M | 1 mo ago | 5 | — | Google MapsBootstrapNginx+6 | — | — |
| 48 | RNatives Developer of RNA-based treatments for cardiovascular diseases | $2M | 12 mo ago | 5 | — | — | 2 patents Synthetic microRNA mimic design and production for gene regulation therapies | — |
| 49 | Factor Bioscience Developer of genomic technologies to treat disease and improve health | $1M | 9 mo ago | 10 | Christopher Rohde Co-Founder & CTO | — | 289 patents Patents concentrated in chemistry & materials | — |
| 50 | gensaic Developer of gene therapies for multiple diseases | $669K | 16 mo ago | 10 | Jonathan Hus Co-Founder | WebflowSlickjQuery+10 | 4 patents Targeted tissue-specific delivery systems and phage-derived nanocarrier therapeutics | — |
Top 12 Gene Therapy companies in Massachusetts
#1
Public · Cambridge, Massachusetts · founded 2014 · 101 employees$92.5Mraised▾
Public · Cambridge, Massachusetts · founded 2014 · 101 employees
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.
Fliar Intelligence Index™ 53how this company ranks against its industry peers
Patents
top 20%Funding
top 24%Innovation
top 31%Tech
top 33%Moat
top 38%News
top 48%Patents: 60 patents matched (strict assignee match) · 43 filings since 2022 vs 17 in 2017-21 · Latest publication 2026
Funding: Raised $92.5M across 7 rounds · 7 rounds over 11 yrs (0.6/yr) · Last round Post IPO (Mar 2026)
Innovation: 60 patents (top 27% of patent-holders) · 43 filings since 2022 vs 17 in 2017-21
Funding rounds
- Mar 2026Post IPO$240M
- Jan 2024Post IPO$108.9M
- Dec 2020Post IPO$90M
- Oct 2020Post IPO$40M
Investors
RA Capital Management, RTW Investments, Foresite Capital, Perceptive Advisors, Janus Henderson Investors, Biogen
Tech stack
Acquia Cloud PlatformAcquia Cloud Site FactoryAdobe AnalyticsAdobe Experience Platform Identity ServiceAdobe Experience Platform LaunchAmazon Web Services
#2
Public · Cambridge, Massachusetts · founded 2010 · 1312 employees$2.8Braised▾
Public · Cambridge, Massachusetts · founded 2010 · 1312 employees
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.
Fliar Intelligence Index™ 82how this company ranks against its industry peers
Tech
top 11%Funding
top 15%Market
top 22%Partners
top 25%Talent
top 25%News
top 31%Tech: 57 technologies across 6/8 capability areas · Cloud: Amazon Cloudfront, Amazon Ses · Devops: Envoy, Nginx
Funding: Raised $2.8B across 20 rounds · 20 rounds over 14 yrs (1.4/yr) · Last round Grant (prize money) (Jan 2025)
Market: 1,312 employees (top 7% of Sustainability Tech) · Revenue ~$1.9B · Top-5,000,000 web property
Audience (LinkedIn): 791k LinkedIn followers — 34.9% of this cohort's audience
Funding rounds
- Jan 2025Grant (prize money)$590M
- Jul 2024Grant (prize money)$176M
- Oct 2020Grant (prize money)$56M
- May 2020Post IPO—
Founders & team
Investors
ADIA, BB Biotech, Julius Baer, Sequoia Capital, Fidelity Investments, Viking Global Investors
Tech stack
AccessibeAmazon CloudfrontAmazon SesAmazon Web ServicesApolloApple Icloud Mail
#3
Series C · Wakefield, Massachusetts · founded 2021 · 12 employees$44.1Mraised▾
Series C · Wakefield, Massachusetts · founded 2021 · 12 employees
Developer of gene therapies for the treatment of neurological diseases. The company's lead program is a novel investigational gene therapy treatment for Canavan disease. It is developing a vector, called rAAV-Olig001-ASPA to deliver a therapeutic ASPA gene directly to the oligodendrocyte cells that are responsible for producing myelin to restore the ability of oligodendrocytes to make the ASPA enzyme on their own.
Fliar Intelligence Index™ 35how this company ranks against its industry peers
Funding
top 31%Growth
top 35%Moat
top 45%Patents
top 49%Talent
bottom 49%Innovation
bottom 47%Funding: Raised $44.1M across 3 rounds · 3 rounds over 2 yrs (1.5/yr) · Last round Series C (Dec 2025)
Growth: Fresh capital in last 24 mo
Moat: Editorial + unicorn rating 4/10 · IP moat: 4 patents
Audience (LinkedIn): 2k LinkedIn followers
Funding rounds
- Dec 2025Series C$30.5M
- Mar 2025Conventional Debt$7.5M
- May 2024Conventional Debt$6M
Founders & team
Investors
Panacea Venture
Tech stack
AddtoanyAddtoany Share ButtonsAnimate.CssBootstrapCdnjsCloudflare
#4
Series A · Needham, Massachusetts · founded 2020 · 5 employees$5.6Mraised▾
Series A · Needham, Massachusetts · founded 2020 · 5 employees
Developer of gene therapies for a broad range of diseases. The company is developing a pipeline of a broad range of therapies for immune and age-related conditions which includes RMD 1101 for rheumatology, RMD 1202 for endocrinology, RMD 1501 for neurology, and RMD 1201 for Type-1 diabetes.
Fliar Intelligence Index™ 7how this company ranks against its industry peers
Funding
top 37%Moat
top 48%Global
bottom 36%Tech
bottom 22%Market
bottom 20%Funding: Raised $5.6M across 5 rounds · 5 rounds over 5 yrs (1.0/yr) · Last round Series A (May 2026)
Moat: Editorial + unicorn rating 4/10 · 10 curated direct competitors tracked
Global: Investors from 2 countries beyond United States
Audience (LinkedIn): 3k LinkedIn followers
Hiring (LinkedIn): 7 hiring posts in 12 mo (public) · momentum -2 · hiring: Research Associate, Scientist/Senior Scientist, Molecular Biology
Activity (LinkedIn): 1 partnerships (incl. Lilly) announced in 12 mo
Funding rounds
- May 2026Series A$1.5M
- Sep 2024Seed—
- Aug 2023Grant (prize money)$275K
- Aug 2022Seed$2.3M
Investors
LifeSpan Vision Ventures, Sherwood Ventures, Longevitytech.fund, Primo Medical Group, Angel Star Ventures, APIS Health Angels
#5
Series A · Cambridge, Massachusetts · founded 2016 · 18 employees$35.4Mraised▾
Series A · Cambridge, Massachusetts · founded 2016 · 18 employees
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.
LinkedIn Pulse recent public activity · Jul 2026
Audience: 5k LinkedIn followers
Activity: 2 partnerships (incl. NVIDIA) announced in 12 mo
Funding rounds
- Oct 2025Grant (prize money)$48M
- Feb 2022Series A$25M
- Jul 2021Seed$10M
- Oct 2018Grant (prize money)—
Founders & team
Investors
Hummingbird Ventures, MassChallenge, Amgen, HBM Genomics, Civilization Ventures, Arpa H
Tech stack
Windows ServerGsapIisCloudflareUnpkgModernizr
#6
Series B · Cambridge, Massachusetts · founded 2017 · 47 employees$103.1Mraised▾
Series B · Cambridge, Massachusetts · founded 2017 · 47 employees
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.
Fliar Intelligence Index™ 64how this company ranks against its industry peers
Growth
top 13%Funding
top 24%Moat
top 31%News
top 41%Talent
top 41%Global
bottom 39%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) · Last round Series B (May 2025)
Moat: Editorial + unicorn rating 8/10 · Soonicorn · 10 curated direct competitors tracked
Audience (LinkedIn): 27k LinkedIn followers
Activity (LinkedIn): 2 awards announced in 12 mo
Funding rounds
- May 2025Series B$153M
- Nov 2022Series A$45M
- Jun 2021Series A$52.1M
- May 2021Grant (prize money)$399.5K
Founders & team
Investors
Playground Global, Alexandria, ANRI, Redmile Group, BeiGene, Camford Capital
#7
Series A · Boston, Massachusetts · founded 2022 · 21 employees$85Mraised▾
Series A · Boston, Massachusetts · founded 2022 · 21 employees
Developer of gene-based therapeutics for multiple diseases. It offers genome engineering and chromatin regulation technology for developing delivery methods for enzymes and drugs to provide targeted gene editing in-vivo.
LinkedIn Pulse recent public activity · Jul 2026
Audience: 6k LinkedIn followers
Activity: 2 product launches · 2 awards announced in 12 mo
Funding rounds
- May 2025Series A$85M
Investors
RA Capital Management, Khosla Ventures, Chugai Venture Fund, Lilly, Johnson & Johnson Innovation – JJDC, Brainstorm Ventures
#8
Series B · Boston, Massachusetts · founded 2019 · 62 employees$110Mraised▾
Series B · Boston, Massachusetts · founded 2019 · 62 employees
Developer of RNAi therapeutics to treat neurodegenerative diseases. The company's proprietary branched siRNA enables RNA interference to be deployed as a therapeutic throughout the brain and spinal cord. This platform helps in the treatment of Huntington’s disease, Parkinson’s disease, Alzheimer’s disease, and other neurodegenerative diseases.
LinkedIn Pulse recent public activity · Jul 2026
Audience: 9k LinkedIn followers
Funding rounds
- Jan 2025Series B$97M
- Jan 2021Series A$110M
Founders & team
Investors
F-Prime Capital, Biogen, Genentech, EQT, RiverVest Venture Partners, abrdn
Tech stack
AosApple Icloud MailBootstrapCloudflareCloudflare Bot ManagementCookiebot
#9
Acquired · Lowell, Massachusetts · founded 2010 · 32 employees$56.2Mraised▾
Acquired · Lowell, Massachusetts · founded 2010 · 32 employees
Developer of gene therapies to treat neurological conditions. The company's lead candidates include ACTX-101 a gene therapy for Rett syndrome using X-reactivation technology and ACTX-401 for spinal muscular atrophy with respiratory distress type 1. Also, has a transformational CNS delivery technology platform.
LinkedIn Pulse recent public activity · Jul 2026
Audience: 4k LinkedIn followers
Funding rounds
- Mar 2025Grant (prize money)$1.3M
- Nov 2023Series C$19M
- Jun 2021Series C$37.1M
- Mar 2012Series A$1.8M
Founders & team
Investors
NASA, RTW Investments
#10
Series A · Cambridge, Massachusetts · founded 2006$135Mraised▾
Series A · Cambridge, Massachusetts · founded 2006
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.
LinkedIn Pulse recent public activity · Jul 2026
Audience: 12k LinkedIn followers
Activity: 1 awards announced in 12 mo
Funding rounds
- Oct 2024Series A$135M
Founders & team
Investors
Arch Venture Partners, Fidelity Investments, Invus, Slate Path Capital, Rock Springs Capital, Regeneron Ventures
Tech stack
AngularApple Icloud MailCart FunctionalityCloudflareCore-JsDocusign
#11
Seed · Cambridge, Massachusetts · founded 2021$11Mraised▾
Seed · Cambridge, Massachusetts · founded 2021
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.
LinkedIn Pulse recent public activity · Jul 2026
Audience: 2k LinkedIn followers
Activity: 1 awards announced in 12 mo
Funding rounds
- Aug 2025Seed$11M
Founders & team
Investors
Clavystbio, Polaris Partners, SEEDS Capital
#12
Series A · Cambridge, Massachusetts · founded 2004 · 6 employees$8.1Mraised▾
Series A · Cambridge, Massachusetts · founded 2004 · 6 employees
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.
Funding rounds
- Feb 2025Grant (prize money)$350K
- Jan 2025Grant (prize money)$320K
- Sep 2023Grant (prize money)$350K
- Aug 2022Grant (prize money)$320K
Founders & team
Investors
HHS
Funding & investor landscape
$7.7Btotal disclosed funding
57companies funded
191disclosed rounds
1rounds last 90 days
Most active investors
Atlas Venture7 rounds
Notable founders
Robert Langer
Co-Founder · Moderna
Kenneth Chien
Co-Founder · Moderna
Mark Pykett
CEO · Myrtelle
Burak Yilmaz
Co-Founder · Kernal Biologics
Catherine Moroski Erkul
Co-Founder · Kernal Biologics
Tasuku Kitada
Co-founder · Strand Therapeutics
Neal Aronin
Co-Founder · Atalanta Therapeutics
Craig Mello
Co-Founder · Atalanta Therapeutics
PJ Anand
Founder · Alcyone
John Maraganore
Co-Founder · City Therapeutics
Robert Nelsen
Co-Founder & Managing Director · City Therapeutics
Andrew Dick
Co-Founder · Cirrus Therapeutics
Prism market signals · United States · country-scoped Prism corpus, 11,961,869 docs
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
RESEARCH IN MASSACHUSETTS · Health & Bio
How research on Gene Therapy in Massachusetts is moving
A read on the academic research around Gene Therapy in Massachusetts — which themes are gaining ground, the institutions and researchers driving them, and how the tone is shifting. Based on 335 papers.
335 papers · stance +0.58 · 48% breakthrough · 11% concern
top institutions
- University of Massachusetts Chan Medical School116
- Massachusetts Institute of Technology96
- Massachusetts General Hospital78
- University of Massachusetts Amherst34
- Massachusetts Eye and Ear Infirmary28
leading researchers
Guangping Gao · Daniel G. Anderson · Dan Wang · Terence R. Flotte · Róbert Langer · Qin Su
prism intelligence · tone & topics
98% positive · 1% critical tone
PharmaceuticalsBiotechnologyMedical ResearchHealthHealthcareClinical Trials
▲
Breakthroughs148 · 44%
▲ 37%→52% share
▲1.4× share momentum8 median citations45% since 2021mostly methods-led
Breakthroughs are rising (37%→52% of field), led by Genetic enhancement.
rising sub-themes (early→recent)
- Genetic enhancement16→24
- Gene delivery20→26
- Capsid3→8
- Virology3→7
- In vivo5→9
Newly emergingVirus 0→6 · Progenitor cell 0→3 · Nanocarriers 0→3
Landmark A Versatile Viral System for Expression and Depletion of Proteins in Mammalian Cells · 1,029 cites
◆
Advancements125 · 37%
▼ 45%→28% share
▼0.6× share momentum24 median citations29% since 2021mostly empirical
Advancements are cooling (45%→28% of field), led by Cell biology.
rising sub-themes (early→recent)
- Cell biology2→6
- Nucleic acid2→3
- Central nervous system2→3
Newly emergingImmunogenicity 0→3
Landmark Promises and Pitfalls of Intracellular Delivery of Proteins · 315 cites
▼
Concerns34 · 10%
▲ 8%→12% share
▲1.5× share momentum23 median citations47% since 2021mostly reviews
Concerns are rising (8%→12% of field), led by Genetic enhancement.
rising sub-themes (early→recent)
- Genetic enhancement4→9
- Adeno-associated virus1→5
- Vector (molecular biology)1→5
- Immune system1→3
- Viral vector3→4
Newly emergingClinical trial 0→3
Landmark Adeno-associated virus as a delivery vector for gene therapy of human diseases · 601 cites
Common questions
How many Gene Therapy companies are there in Massachusetts, United States?
Radar tracks 76 indexable Gene Therapy companies headquartered in Massachusetts, United States that meet our profile-completeness bar.
What is the total funding raised by Gene Therapy companies in Massachusetts, United States?
The Gene Therapy companies in Massachusetts, United States on this list have raised a combined $7.7B in disclosed equity funding across 57 funded companies.
Which is the highest-funded Gene Therapy company in Massachusetts, United States?
Moderna is the highest-funded Gene Therapy company in Massachusetts, United States, with $2.8B in disclosed equity funding.
How many Gene Therapy companies in Massachusetts, United States are funded?
57 of the 76 Gene Therapy companies in Massachusetts, United States on this list have disclosed at least one equity funding round.
How many Gene Therapy unicorns are there in Massachusetts, United States?
None of the Gene Therapy companies in Massachusetts, United States on this list currently has a disclosed valuation of $1B or more.
Who are the top investors in Massachusetts, United States Gene Therapy companies?
The most active investors in Massachusetts, United States Gene Therapy companies on this list include HHS, Fidelity Investments, RA Capital Management, Arch Venture Partners, Atlas Venture.
Riya SenguptaFinancials
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.