Best Gene Therapy Companies in United States
The 295 best Gene Therapy companies in United States in 2026, ranked by funding and momentum. Funding, investors, founders, valuation, tech stack and live signals. Top: Moderna.
$15.9 billion in disclosed funding across 295 tracked Gene Therapy companies in the United States tells you this sector isn't running on promise alone. Beacon Therapeutics leads our directory at $291M raised, with Precision BioSciences at $136M and Senti Bio at $158M rounding out the top tier. Backers like RA Capital Management, Deerfield, and Fidelity Investments aren't writing small checks here. The [global gene therapy market sits at roughly $9.4B today, forecast to hit $28.2B by 2030 at a 22.8% CAGR](https://www.businessupturn.com/brand-post/global-gene-therapy-market-to-surge-to-usd-36-55-billion-by-2032-marketsandmarkets/), and the capital concentration in U.S. companies reflects exactly that trajectory.
The revenue picture is where it gets interesting. Across six validated filers, the combined pool reaches $725M, with a median of $29M and median growth of 153% year-over-year. Moderna pulled $389M, 13x that sector median, while Arrowhead Pharmaceuticals posted $264M on a staggering 10,461% YoY gain. So what's driving the science underneath all this capital? Recent coverage points to meaningful clinical progress, from early Huntington's disease results to combination approaches targeting brain cancer. I'd argue the earned-revenue story here is the one worth watching, not just the funding headlines.
- Global Gene Therapy Market to Surge to USD 36.55 Billion by 2032
- Gene Therapy Market To Grow at a Stayed CAGR with Huge Profits by 2029
Ranked: Best Gene Therapy companies in United States
| # | Company | Disclosed funding | Last round | Employees | Founder | Tech stack | Market signals (1y) | 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 | 47 signals Earnings · Contracts · Leadership | 31 patents Patents concentrated in health & consumer products | 1 internship |
| 2 | Kriya Therapeutics Developer of therapeutic solutions for the treatment of diabetes 53Funding top 16%News top 24%Moat top 31%Growth top 43% All 8 signals ▾Fundingtop 16% Newstop 24% Moattop 31% Growthtop 43% Markettop 44% Talenttop 50% Globalbottom 37% Techbottom 32% Funding: Raised $618M across 4 rounds · 4 rounds over 7 yrs (0.6/yr) News: 5 BI events in 12 mo (of 16 tracked, 3 types) · 3 high-value events (IPO/M&A/funding/contracts) in 12 mo rank vs industry peers · Fliar Intelligence Index 53/100 | $618M | 10 mo ago | 159 | Shankar Ramaswamy Co-Founder & CEO | WordPress Site EditorWordPressMySQL+12 | 5 signals Funding · Capital raise · Leadership | — | — |
| 3 | ReCode Therapeutics Developer of precision medicines for treating lung related diseases 67Funding top 17%Talent top 28%Moat top 31%Market top 42% All 7 signals ▾Fundingtop 17% Talenttop 28% Moattop 31% Markettop 42% Newstop 45% Techtop 48% Globalbottom 39% Funding: Raised $342M across 7 rounds · 7 rounds over 7 yrs (1.0/yr) Talent: 3 named leaders on record · 3 C-suite/founder profiles curated rank vs industry peers · Fliar Intelligence Index 67/100 | $342M | 8 mo ago | 105 | Daniel Siegwart Co-Founder | WordPressApple iCloud MailMicrosoft 365+28 | — | — | — |
| 4 | ReNAgade Developer of RNA-based medicines to treat multiple diseases | $300M | 36 mo ago | 136 | — | — | — | — | — |
| 5 | Beacon Therapeutics Developer of therapeutics for retinal disorders | $291M | 5 mo ago | 76 | Lance Baldo Chief Executive Officer | WordPressMySQLRedis+16 | — | — | — |
| 6 | Arrowhead Pharmaceuticals Developer of therapeutics to treat multiple diseases | $290M | 12 mo ago | 427 427 → 809 (+89%) · sector median -8% | Christopher Anzalone CEO & President | DrupalApple iCloud MailMicrosoft 365+25 | 20 signals ▲67% Earnings · Funding · Contracts | 1,202 patents RNA interference therapeutics targeting disease genes and targeted delivery systems | — |
| 7 | Sirnaomics Developer of RNA based therapeutics for the treatment of cancer and fibrosis disease 80Patents top 14%Funding top 15%Innovation top 17%Growth top 28% All 9 signals ▾Patentstop 14% Fundingtop 15% Innovationtop 17% Growthtop 28% Moattop 35% Markettop 46% Talentbottom 49% Globalbottom 48% Newsbottom 21% Patents: 277 patents matched (strict assignee match) · 191 filings since 2022 vs 63 in 2017-21 Funding: Raised $267M across 14 rounds · 14 rounds over 19 yrs (0.7/yr) rank vs industry peers · Fliar Intelligence Index 80/100 | $267M | 9 mo ago | 88 | Patrick Lu President and CEO | — | — | 277 patents siRNA therapeutics and nanoparticle delivery systems for cancer and viral diseases | 1 launch |
| 8 | Tenaya Therapeutics Developer of regenerative and gene therapeutics for heart failure | $248M | 16 mo ago | 141 141 → 93 (-34%) · sector median -8% | Benoit Bruneau Co-Founder | WordPressDrupalMySQL+24 | — | 207 patents Engineered AAV capsids and CRISPR guides for cardiac gene‑therapy targeting PLN mutations | — |
| 9 | Editas Medicine Developer of therapeutics for rare genetic diseases | $210M | 29 mo ago | 257 257 → 183 (-29%) · sector median -8% | Cynthia Collins Ex-CEO & President | DrupalPHPBootstrap+25 | 3 signals ▼83% Earnings · Leadership | 713 patents CRISPR-based gene editing therapeutics for blood disorders and immune cell engineering | — |
| 10 | Senti Bio Developer of cell and gene therapies for patients living with incurable diseases 45Product top 14%Funding top 21%Talent top 22%Tech top 32% All 11 signals ▾Producttop 14% Fundingtop 21% Talenttop 22% Techtop 32% Innovationtop 34% Newsbottom 43% Moatbottom 43% Growthbottom 39% Globalbottom 39% Marketbottom 32% Fin. healthbottom 20% Product: 4 distinct product lines · Company stage: Public Funding: Raised $158M across 8 rounds · 8 rounds over 8 yrs (1.0/yr) rank vs industry peers · Fliar Intelligence Index 45/100 | $158M | 1 mo ago | 122 | Jim Collins Co-Founder | DrupalPHPSalesforce+24 | 3 signals ▼75% Earnings · Announcements | — | — |
| 11 | Precision BioSciences Developer of gene editing therapies to treat rare genetic diseases 57Product top 19%Funding top 21%Moat top 32%Patents top 36% All 11 signals ▾Producttop 19% Fundingtop 21% Moattop 32% Patentstop 36% Newstop 38% Markettop 39% Techtop 41% Innovationtop 42% Growthbottom 47% Globalbottom 42% Fin. healthbottom 39% Product: 3 distinct product lines · Company stage: Public Funding: Raised $136M across 9 rounds · 9 rounds over 16 yrs (0.6/yr) rank vs industry peers · Fliar Intelligence Index 57/100 | $136M | 3 mo ago | 236 | — | DrupalApple iCloud MailMicrosoft 365+23 | 3 signals ▲50% Regulatory · Earnings · Capital raise | 68 patents Therapeutic genome editing using engineered meganucleases and engineered T‑cell therapies | 1 launch |
| 12 | City Therapeutics Developer of gene therapies for multiple diseases | $135M | 20 mo ago | — | John Maraganore Co-Founder | Cart FunctionalitySquarespace CommerceSquarespace+19 | — | — | — |
| 13 | Kate Therapeutics Developer of AAV-based gene therapies to treat genetically defined muscle disease | $124M | 25 mo ago | 46 | — | — | — | — | — |
| 14 | Comanche Developer of RNA therapeutics to treat preeclampsia | $111M | 29 mo ago | 17 | Mike Young Co-Founder & President | WordPressMySQLPHP+23 | — | — | — |
| 15 | Atalanta Therapeutics Developer of RNAi therapeutics to treat neurodegenerative diseases | $110M | 16 mo ago | 62 | Neal Aronin Co-Founder | WordPressApple iCloud MailMicrosoft 365+29 | — | — | — |
| 16 | Addition Therapeutics Developer of RNA-mediated genetic medicines 42Funding top 22%Talent top 38%Moat top 42%Market bottom 40% All 5 signals ▾Fundingtop 22% Talenttop 38% Moattop 42% Marketbottom 40% Globalbottom 36% Funding: Raised $106M 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 42/100 | $106M | 6 mo ago | — | Kathleen Collins Founder | — | — | — | — |
| 17 | Ray Therapeutics Developer of therapeutics for degenerative retinal diseases 48Funding top 19%Growth top 35%Tech top 40%Moat top 42% All 7 signals ▾Fundingtop 19% Growthtop 35% Techtop 40% Moattop 42% Talentbottom 49% Globalbottom 39% Marketbottom 28% Funding: Raised $106M across 6 rounds · 6 rounds over 4 yrs (1.5/yr) Growth: Fresh capital in last 24 mo rank vs industry peers · Fliar Intelligence Index 48/100 | $106M | 1 mo ago | 10 | Paul Bresge Co-Founder & CEO | WordPressMySQLMariaDB+21 | — | — | — |
| 18 | Voyager Therapeutics Neurogenetic medicine innovator, developing transformative treatments for neurological diseases | $105M | 29 mo ago | 183 183 → 161 (-12%) · sector median -8% | Steven Paul Co-Founder | WordPressApple iCloud MailMySQL+21 | 4 signals ▼64% Earnings | 790 patents Engineered AAV gene therapy vectors for neurodegenerative and metabolic disorder treatment | — |
| 19 | 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 | 12 mo ago | 47 | Tasuku Kitada Co-founder | — | — | — | 2 awards |
| 20 | 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 | 4 signals ▼33% Earnings · Funding · Leadership | 60 patents AAV vector manufacturing and formulation for cardiac gene therapies | — |
| 21 | Atsena Therapeutics Developer of gene therapies for the treatment of ocular diseases | $88M | 9 mo ago | 23 23 → 49 (+113%) · sector median -8% | Shannon Boye Founder & Director | WordPressMySQLPHP+18 | — | — | 1 partnership |
| 22 | Stylus Medicine Developer of gene-based therapeutics for multiple diseases | $85M | 13 mo ago | 21 | — | — | — | — | — |
| 23 | 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 | 268 268 → 222 (-17%) · sector median -8% | — | WordPressApple iCloud MailMySQL+34 | 7 signals ▼56% Earnings · Layoffs · FDA approval | — | 1 internship |
| 24 | Exicure developer of gene silencing & immunomodulatory drugs using spherical nucleic acid (SNA) technology | $72M | 19 mo ago | 13 13 → 7 (-46%) · sector median -8% | — | Q4MediaElement.jsMicrosoft ASP.NET+12 | — | 41 patents Liposomal spherical nucleic acid delivery systems for immunotherapy and gene‑silencing | — |
| 25 | 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 | — | Lei Qi Founder | WordPressMySQLPHP+21 | — | — | — |
| 26 | Locus Biosciences Developer of therapeutics for infectious disease and inflammatory indications | $67M | 28 mo ago | 74 74 → 61 (-18%) · sector median -8% | Paul Garofolo Chief Executive Officer | WordPress Block EditorWordPressMySQL+24 | — | 60 patents Engineered CRISPR‑enhanced bacteriophage therapeutics targeting antibiotic‑resistant bacterial infections | — |
| 27 | Adverum Biotechnologies Developer of gene therapy based therapies for wet age-related macular degeneration & ophthalmologic disorders | $61M | 28 mo ago | 123 123 → 138 (+12%) · sector median -8% | — | WordPressApple iCloud MailMicrosoft 365+21 | 5 signals ▼17% M&A | 319 patents Gene‑therapy AAV vectors for treating ocular neovascular and inflammatory eye diseases | — |
| 28 | Alcyone Developer of gene therapies to treat neurological conditions | $56M | 15 mo ago | 32 | PJ Anand Founder | WordPressMySQLPHP+12 | — | — | — |
| 29 | Latus Developer of gene therapy medicines for neurological diseases 35Funding top 26%Moat top 31%Talent bottom 49%Market bottom 40% All 5 signals ▾Fundingtop 26% Moattop 31% Talentbottom 49% Marketbottom 40% Globalbottom 36% Funding: Raised $54.0M across 2 rounds · 2 rounds over 2 yrs (1.0/yr) Moat: Editorial + unicorn rating 8/10 · Soonicorn rank vs industry peers · Fliar Intelligence Index 35/100 | $54M | 1 mo ago | — | P Ghoroghchian CEO | — | — | — | — |
| 30 | 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 | 31 | Jeff Galvin Chief Executive Officer | WordPressMySQLPHP+34 | — | 228 patents Non-integrating viral vectors for gene delivery and HIV immunotherapy, plus gamma‑delta T‑cell activation | — |
| 31 | 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 -8% | Mark Pykett CEO | WordPressMySQLPHP+20 | — | 4 patents Therapeutic biotech targeting myelin repair and neurodegenerative disease treatment | — |
| 32 | XyloCor Therapeutics Developer of gene therapies for the treatment of coronary arterial disease | $43M | 17 mo ago | 18 | Ronald Crystal Co-Founder | Apache HTTP ServerjQueryGoogle Hosted Libraries | — | — | — |
| 33 | Kernal Biologics Developer of mRNA therapeutics for immuno-oncology using deep learning technology | $35M | 8 mo ago | 18 18 → 22 (+22%) · sector median -8% | Burak Yilmaz Co-Founder | — | — | 27 patents Targeted lipid-based delivery systems for oncoselective cancer therapeutics | — |
| 34 | Vironexis Biotherapeutics Developer of AAV-delivered T-cell immunotherapies for the treatment of cancer | $26M | 21 mo ago | — | Timothy Cripe Co-Founder | — | — | 15 patents AAV-based gene therapy vectors targeting metastatic cancer prevention and treatment | — |
| 35 | Opus Genetics Developer of gene therapies to treat retinal degenerative diseases 42Patents top 29%Funding top 30%Moat top 32%Innovation top 34% All 11 signals ▾Patentstop 29% Fundingtop 30% Moattop 32% Innovationtop 34% Talenttop 38% Growthtop 47% Techtop 50% Newstop 50% Globalbottom 36% Marketbottom 31% Fin. healthbottom 28% Patents: 26 patents matched (strict assignee match) · 26 filings since 2022 vs 0 in 2017-21 Funding: Raised $23.5M across 4 rounds · 4 rounds over 5 yrs (0.8/yr) rank vs industry peers · Fliar Intelligence Index 42/100 | $24M | 10 mo ago | 14 | Jason Menzo Co-Founder | WordPressMySQLPHP+24 | 4 signals ▼20% Regulatory · Earnings · Funding | 26 patents Ophthalmic alpha‑adrenergic antagonist drugs for mydriasis, presbyopia, and AMD treatment | — |
| 36 | Rejuvenate Bio Developer of gene therapies for treating chronic and age-related diseases | $15M | 23 mo ago | 39 | Noah Davidsohn Co-Founder & CSO | Cart FunctionalitySquarespace CommerceSquarespace+12 | — | — | — |
| 37 | Navega Therapeutics Developer of gene therapy products for chronic pain management | $15M | 13 mo ago | 10 | Prashant Mali Co-Founder | — | — | — | — |
| 38 | ParcelBio Developer of programmable technology to deliver mRNA to diverse cell types in the body | $13M | 4 mo ago | — | Christopher Carlson Co-Founder | — | — | — | — |
| 39 | Expression Therapeutics Provider of gene therapy technology for the treatment of hemophilia A | $13M | 16 mo ago | 21 | — | — | — | — | — |
| 40 | Cirrus Therapeutics Developer of therapies designed to extend ocular healthspan and restore vision | $11M | 10 mo ago | — | Andrew Dick Co-Founder | — | — | — | 1 award |
| 41 | NeuroGT Developer of gene therapies for rare neurogenetic diseases | $10M | 9 mo ago | — | Haiyan Fu Founder | — | — | — | — |
| 42 | Hunterian Provider of gene-editing platform based on CRISPR delivery technology | $8M | 16 mo ago | 6 | Vinod Ranga Founder & CEO | — | — | — | — |
| 43 | 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 | 11 11 → 13 (+18%) · sector median -8% | Minoru Ko Co-Founder | WordPressMySQLPHP+11 | — | — | — |
| 44 | 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 | — | — | 1 job opening |
| 45 | miRecule Developing microRNA therapies for the treatment of cancers | $5M | 9 mo ago | 8 | Anthony Saleh Co-Founder & CEO | WordPressMySQLPHP+14 | — | 52 patents Muscle‑targeted imaging agents and nucleic‑acid therapeutics for neuromuscular disorders | 1 award |
| 46 | Nionyx Bio Developer of gene therapy solutions designed for precision and safety | $4M | 8 mo ago | — | Leszek Lisowski Co-Founder | — | — | — | — |
| 47 | Armatus Developer of precision gene therapies for rare diseases | $3M | 13 mo ago | 9 | Scott Harper Co-Founder | WordPressMySQLPHP+19 | — | — | — |
| 48 | Bedrock Therapeutics Developer of gene therapy using AAV capsids and immune modulators | $2M | 15 mo ago | — | Bedrock Inc Owner | — | — | — | — |
| 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 | OncoTelic Developer of RNA therapeutics to treat cancer and infectious diseases | $575K | 6 mo ago | 22 22 → 24 (+9%) · sector median -8% | — | WordPressApple iCloud MailMySQL+11 | 5 signals ▲400% Announcements · Earnings · Contracts | 6 patents Patents concentrated in health & consumer products | — |
Top 12 Gene Therapy companies in United States
#1
Public · San Francisco, California · founded 2016 · 122 employees$158Mraised▾
Developer of cell and gene therapies for patients living with incurable diseases. The company has developed its proprietary synthetic biology platform to produce cell and gene therapies for cancer treatment. The company's pipeline is comprised of cell therapies engineered with Gene Circuits to target difficult to treat liquid and solid tumors. It offers CAR-NK-based therapies for multiple cancer and tumors.
- May 2026Post IPO$40M
- Dec 2024Post IPO$37.6M
- Dec 2024Post IPO$10M
- Jun 2024Grant (prize money)$8M
#2
Series B · Berkeley, California · founded 2021 · 10 employees$106Mraised▾
Developer of therapeutics for degenerative retinal diseases. The lead candidate RAY-001 for Retinitis pigmentosa, RAY-002 for geography atrophy, and for other inherited retinal diseases.
- Apr 2026Series B$125M
- Apr 2025Grant (prize money)$8M
- Nov 2023Grant (prize money)$4M
- May 2023Series A$100M
#3
Public · Durham, North Carolina · founded 2006 · 236 employees$135.7Mraised▾
Developer of gene editing therapies to treat rare genetic diseases. The company utilizes novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion, excision, and elimination. The company's pipeline include therapies for Hepatitis B, duchenne muscular dystrophy, and more.
- Mar 2026Post IPO$7.5M
- Jun 2022Post IPO$75M
- Mar 2019Conventional Debt$39.5M
- Jun 2018Series B$110M
#4
Series C · Alachua, Florida · founded 2023 · 76 employees$290.7Mraised▾
Developer of therapeutics for retinal disorders. It offers gene therapies which include AGTC-501, to treat X-linked retinitis pigmentosa (XLRP), and other pipeline candidates for dry age-related macular degeneration and cone-rod dystrophy.
- Jan 2026Series C$75M
- Jul 2024Series B$170M
- Jun 2023Series A$120.7M
#5
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
#6
Series A · Philadelphia, Pennsylvania · founded 2022$54Mraised▾
Developer of gene therapy medicines for neurological diseases. The company leverages precision delivery through Adeno-Associated Virus to target specific cells and tissues, to improve the lives of patients suffering from various central nervous system disorders.
- May 2026Series A$97M
- May 2024Series A$54M
#7
Series B · Menlo Park, California · founded 2015 · 105 employees$342Mraised▾
Developer of precision medicines for treating lung-related diseases. The pipeline includes lead programs which includes RCT1100 an inhaled mRNA-based investigational therapy for primary ciliary dyskinesia caused by mutations in the DNAI1 gene and RCT2100 is an inhaled mRNA-based investigational therapy for cystic fibrosis. It has proprietary non-viral lipid nanoparticle platform that enables the delivery of a variety of payloads, and organ targeting of nucleic acid and protein payloads via both systemic and local delivery.
- Sep 2025Series B$29M
- Sep 2023Series B$50M
- Jan 2023Series B$10M
- Jun 2022Series B$120M
#8
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—
#9
Series D · Redwood City, California · founded 2012 · 159 employees$617.6Mraised▾
Developer of therapeutic solutions for the treatment of diabetes. The company has developed AAV-based gene therapies for the treatment of type 1 and type 2 diabetes. Its drug candidate KT-A112 contains insulin and glucokinase proteins that help in treating Type 1 & Type 2 diabetes mellitus. It also provides KT-A522 with glucagon-like peptide (GLP-1) receptor agonist for the treatment of Type 2 diabetes, KT-A832 insulin growth factor that helps in treating diabetes mellitus.
- Jul 2025Series D$320.8M
- May 2022Series C$430M
- Jun 2021Series B$100M
- May 2020Series A$87.6M
#10
Public · Gaithersburg, Maryland · founded 2007 · 88 employees$267Mraised▾
Developer of RNA-based therapeutics for the treatment of cancer and fibrosis disease. The company has proprietary delivery technologies, a polypeptide nanoparticle RNAi platform and GalNAc RNAi platform, GalAhead, to develop drug candidate pipeline which include STP122G for cardiovascular disease treatment. It also, develops pipeline of drugs for cancers, skin conditions, fibrosis-related diseases, antiviral and cardiometabolic diseases, and medical aesthetics.
- Sep 2025Post IPO$17.7M
- Jul 2021Series E$105M
- Oct 2020Series D$105M
- Jul 2019Series C$25M
#11
Series A · San Francisco, California · founded 2021$106.5Mraised▾
Developer of RNA-mediated genetic medicines. The company uses PRINT technology to develop RNA-only therapeutics to transform genetic medicine.
- Dec 2025Series A$106.5M
#12
Acquired · Pasadena, California · founded 1989 · 427 employees$290.2Mraised▾
Developer of therapeutics to treat multiple diseases. The company develops medicines that treat intractable diseases by silencing the genes that cause them. The company’s pipeline includes ARO-AAT to treat liver diseases, ARO-APOC3 for Hypertriglyceridemia, ARO-ANG3 for Dyslipidemia, and more.
- May 2025Grant (prize money)$305K
- Dec 2017Post IPO$7.2M
- Jan 2017Post IPO$9.7M
- Sep 2016Post IPO$56.5M
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 is moving in the United States
A read on the academic research around Gene Therapy — which themes are gaining ground, the institutions and researchers driving them, and how the tone is shifting. Based on 6,961 United States-affiliated papers.
- Harvard University265
- University of North Carolina at Chapel Hill221
- University of Pennsylvania213
- University of Florida209
- Johns Hopkins University182
- Genome editing46→103
- CRISPR41→97
- Messenger RNA3→37
- Cas924→55
- In vivo88→117
- Genome editing13→50
- CRISPR9→38
- Messenger RNA2→17
- Vaccination26→37
- Nasal administration2→13
- Genome editing7→23
- CRISPR6→17
- Immune system33→41
- Immunotherapy2→9
- Capsid7→14
Where gene therapy is researched across United States
Location clusters by institution affiliation — each city's volume, stance tone, and a quick read on its hot topic, what's advancing, the opening opportunity, and the main concern.
Who's filing gene therapy patents in the United States
United States gene therapy patent filing is accelerating — 6,200 filings from 1,958 assignees (2016–2025), led by Genzyme, UNIV Pennsylvania, UNIV Florida; annual filings 281→830.
- Genzyme585
- UNIV Pennsylvania534
- UNIV Florida344
- UNIV California245
- UNIV Massachusetts241
- RES INST Nationwide Childrens Ho163
- Genvec156
- Spark Therapeutics155