Best Gene Therapy Companies in California, United States
The 82 best Gene Therapy companies in California, United States in 2026, ranked by funding and momentum. Funding, investors, founders, valuation, tech stack and live signals. Top: Kriya Therapeutics.
Ranked: Best Gene Therapy companies in California
| # | Company | Disclosed funding | Last round | Employees | Founder | Tech stack | Patents & IP focus | Recent signals |
|---|---|---|---|---|---|---|---|---|
| 1 | 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 | — | — |
| 2 | 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 | — | — |
| 3 | Arrowhead Pharmaceuticals Developer of therapeutics to treat multiple diseases | $290M | 12 mo ago | 427 427 → 809 (+89%) | Christopher Anzalone CEO & President | DrupalApple iCloud MailMicrosoft 365+25 | 1,202 patents RNAi therapeutics and targeted antibody/lipid conjugates for disease gene silencing | — |
| 4 | Tenaya Therapeutics Developer of regenerative and gene therapeutics for heart failure | $248M | 16 mo ago | 141 141 → 93 (-34%) | Benoit Bruneau Co-Founder | WordPressDrupalMySQL+24 | 207 patents Engineered AAV gene‑therapy vectors and CRISPR guides for cardiac PLN mutation treatment | — |
| 5 | 4D Molecular Therapeutics Developer of gene therapeutics for genetic disorders | $187M | 72 mo ago | 147 147 → 216 (+47%) | David Kirn Md Co-Founder & CEO | WordPressBugHerdMySQL+23 | 246 patents Patents concentrated in chemistry & materials | — |
| 6 | 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 | — | — |
| 7 | AudentesTherapeutics Developer of therapeutics to treat rare genetic diseases | $138M | 128 mo ago | 404 | — | — | — | — |
| 8 | Tocagen Developer of gene therapies for the treatment of cancer | $124M | 105 mo ago | 1 | — | — | 161 patents Gene therapy and CAR‑T delivery vectors with cytosine deaminase prodrug activation | — |
| 9 | Kate Therapeutics Developer of AAV-based gene therapies to treat genetically defined muscle disease | $124M | 25 mo ago | 46 | — | — | — | — |
| 10 | DTx Pharma Provider of RNA-based therapeutics for multiple health purposes | $113M | 63 mo ago | 50 | — | — | — | — |
| 11 | 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 | — | — | — |
| 12 | 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 | 2 mo ago | 10 | Paul Bresge Co-Founder & CEO | WordPressMySQLMariaDB+21 | — | — |
| 13 | Sangamo BioSciences Developing Zinc finger nuclease based therapeutics for monogenic diseases and HIV | $90M | 87 mo ago | 337 | — | WordPressDrupalApple iCloud Mail+30 | 1,158 patents Patents concentrated in chemistry & materials | — |
| 14 | Spotlight Therapeutics Developer of cancer therapeutics using gene editing | $81M | 33 mo ago | 32 | — | — | — | — |
| 15 | Novome Biotechnologies Developer of gene therapies to treat GI diseases | $77M | 45 mo ago | 44 | Will DeLoache Co-Founder | — | 37 patents Engineered microbial therapeutics and recombinant protein secretion for gut and bile‑acid treatments | — |
| 16 | Switch Therapeutics Developer of therapeutics for neurological disorders | $72M | 39 mo ago | 25 | Dee Datta Co-Founder & CEO | — | — | — |
| 17 | Ceregene Develops AAV-based therapies for neurodegenerative and eye disorders | $72M | 187 mo ago | 30 | Stephen Sherwin Co-Founder | — | 25 patents Gene therapy delivery vectors for retinal and dopaminergic neurodegenerative disease treatment | — |
| 18 | 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 | — | — |
| 19 | CODA Biotherapeutics Developer of gene therapy-based analgesics for the treatment of neuropathic pain disorder | $62M | 54 mo ago | 22 | Mitchell H Finer Co-Founder | — | 32 patents Therapeutic peptide/protein compositions targeting neurological disorders. | — |
| 20 | Adverum Biotechnologies Developer of gene therapy based therapies for wet age-related macular degeneration & ophthalmologic disorders | $61M | 28 mo ago | 123 123 → 138 (+12%) | — | WordPressApple iCloud MailMicrosoft 365+21 | 319 patents Gene‑therapy AAV vectors for treating ocular neovascular and inflammatory eye diseases | — |
| 21 | Capsida Developer of next-generation AAV platform for gene therapies | $50M | 61 mo ago | 134 | Nicholas Flytzanis Co-Founder & VP | WordPressMySQLPHP+24 | 64 patents Engineered adeno-associated virus vectors optimized for brain, spinal cord, and heart gene delivery | — |
| 22 | Jasper Therapeutics Developer of therapeutics for chronic diseases by targeting mast cells and stem cells | $49M | 77 mo ago | — | Judith Shizuru Co-Founder | WordPressApple iCloud MailMicrosoft 365+21 | — | — |
| 23 | Creyon Bio Provider of drug design and oligonucleotide-based drug development platform | $40M | 51 mo ago | 38 | Swagatam Mukhopadhyay Chief Scientific Officer & Cofounder & Board Member | WordPressMySQLPHP+15 | — | — |
| 24 | Solstice Biologics Providing solutions for targeting and delivering nucleic acid therapeutics | $40M | 126 mo ago | 4,443 | — | — | 9 patents Modified oligonucleotide synthesis using chiral phosphoramidite auxiliaries for therapeutic nucleic acids | — |
| 25 | Intradigm Corporation Develops RNA interference (RNAi) therapeutics for the treatment of cancer. | $37M | 209 mo ago | 17 | — | — | 136 patents RNAi‑based gene silencing therapeutics for cancer, angiogenesis, and scar‑free wound healing | — |
| 26 | Quark Pharmaceuticals Late stage pharmaceutical company developing RNAi-based therapeutics | $37M | 192 mo ago | 30 | Daniel Zurr Founder & CEO | — | 475 patents Therapeutic nucleic acid agents for preventing ischemia‑reperfusion injury in organs | — |
| 27 | Regulus Therapeutics Innovative medicines company reimagining medicine to improve and extend people’s lives | $35M | 27 mo ago | 30 | — | WordPressMySQLPHP+11 | 403 patents Antisense oligonucleotide therapeutics for polycystic kidney disease and neurological disorders | — |
| 28 | Refuge Biotech Company using CRISPR activation and CRISPR interference to develop novel cancer treatments | $34M | 91 mo ago | 16 | David Parkinson Co-Founder | — | — | — |
| 29 | Modis Therapeutics Provider of therapies for rare genetic diseases | $30M | 92 mo ago | — | Daniel Pietro Co-Founder | — | — | — |
| 30 | 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 | — |
| 31 | SomaGenics Developer of RNA-based therapeutics and diagnosis for multiple disease conditions | $26M | 78 mo ago | 5 | — | — | 82 patents Nucleic acid sequencing library prep and RNA‑based therapeutics for gene silencing and wound healing | — |
| 32 | Siren Biotechnology Developer of gene therapies for cancer treatments | $20M | 26 mo ago | 8 | Nicole Paulk Founder & CEO | WordPressMySQLPHP+10 | 15 patents AAV gene therapy vector design, production, and CpG-modified viral manufacturing for oncology | — |
| 33 | 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 | — | — |
| 34 | Navega Therapeutics Developer of gene therapy products for chronic pain management | $15M | 13 mo ago | 10 | Prashant Mali Co-Founder | — | — | — |
| 35 | Radar Therapeutics Developer of precision-expressed mRNA-based therapeutics for intractable diseases | $13M | 25 mo ago | — | Eerik Kaseniit Co-Founder | WebflowGSAPjQuery+7 | — | — |
| 36 | ParcelBio Developer of programmable technology to deliver mRNA to diverse cell types in the body | $13M | 4 mo ago | — | Christopher Carlson Co-Founder | — | — | — |
| 37 | Genascence Developer of gene therapies for musculoskeletal and joint diseases | $10M | 49 mo ago | 6 | Thomas Chalberg Founder/CEO | WordPressMySQLPHP+18 | 9 patents Patents concentrated in health & consumer products | — |
| 38 | Arrogene Developer of cancer therapeutics based on a nanotechnological targeting platform | $9M | 79 mo ago | — | Larry Couture Chief Executive Officer | — | 5 patents Polymeric drug delivery systems and antisense glioma therapeutics using polymalic acid. | — |
| 39 | Visgenx Developer of gene therapy for age related muscular degeneration | $9M | 36 mo ago | 9 | Marty Emanuele Co-Founder & CSO | — | 16 patents ELOVL2-based gene therapy vectors for treating ocular diseases | — |
| 40 | Eiger BioPharmaceuticals Developer of antiviral agents against targets in the treatment of hepatitis | $9M | 85 mo ago | 56 | — | — | 181 patents Peptide therapeutics for metabolic disorders and antiviral treatments, especially exendin derivatives. | — |
| 41 | Renova Therapeutics Developer of gene therapies for congestive heart failure and type 2 diabetes | $8M | 23 mo ago | 7 | Jack Reich Chief Executive Officer | WordPressMySQLPHP+17 | — | — |
| 42 | Particella Developer of gene therapies for gastrointestinal diseases | $7M | 50 mo ago | 4 | Timothy Day Co-Founder | — | 3 patents Advanced biologic drug delivery systems and formulations for therapeutic agents | — |
| 43 | Traversa Therapeutics Development and commercialization of RNAi Delivery Technologies | $7M | 206 mo ago | — | — | — | — | — |
| 44 | ImmunoVec Developer of gene therapies for hematopoietic diseases | $4M | 55 mo ago | 6 | Katelyn Masiuk Co-Founder | — | 7 patents Design of enhanced viral vectors for gene therapy and transgene expression | — |
| 45 | Celestial Therapeutics Developer of mRNA-based vaccines and therapies for multiple diseases | $1M | 14 mo ago | — | Leontis Teryazos Co-Founder | — | — | — |
| 46 | Arcturus Therapeutics Developer of RNAi technologies for the treatment of rare orphan diseases | $1M | 32 mo ago | 337 | — | DrupalPHPAmazon Web Services+25 | 488 patents Lipid nanoparticle formulations for RNA/mRNA delivery and vaccine therapeutics | 1 partnership |
| 47 | Eos Neuroscience Developer of treatments for blindness and other neurological conditions | $1M | 165 mo ago | — | Ed Boyden Co-Founder | — | 29 patents Neuro-modulation therapies for chronic pain and ocular gene‑therapy using engineered vectors. | — |
| 48 | Ariz Precision Medicine Developer of RNA therapeutics for cancer | $685K | 70 mo ago | 3 | Lonnie Bookbinder Founder | — | — | — |
| 49 | OncoTelic Developer of RNA therapeutics to treat cancer and infectious diseases | $575K | 6 mo ago | 22 22 → 24 (+9%) | — | WordPressApple iCloud MailMySQL+11 | 6 patents Patents concentrated in health & consumer products | — |
| 50 | Cistern Developer of a platform to build mRNA for therapeutics | $125K | 22 mo ago | — | Hari Bhaskaran Founder | — | — | — |
Top 12 Gene Therapy companies in California
#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
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
#4
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
#5
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
#6
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
#7
Public · San Francisco, California · founded 2016 · 141 employees$248Mraised▾
Developer of regenerative and gene therapeutics for heart failure. The company's pipeline include MYBPC3 for genetic hypertrophic cardiomyopathy, PKP2 for genetic arrhythmogenic right ventricular cardiomyopathy, DWORF for dilated cardiomyopathy, and HDAV6i for heart failure with preserved ejection fraction.
- Feb 2025Grant (prize money)$8M
- Mar 2021Series C$106M
- Oct 2019Series B$92M
- Dec 2016Series A$50M
#8
Seed · San Francisco, California · founded 2023$13.2Mraised▾
Developer of programmable technology to deliver mRNA to diverse cell types in the body. The company has proprietary nanoparticle technology, which is ‘STAmP’, stabilizes the mRNA and utilizes an interchangeable, lock-and-key-like targeting mechanism which enables fully customizable mRNA medicines for kidney disorders, chronic liver diseases, and immune system dysregulation.
- Feb 2026Seed$13.2M
#9
Series B · San Francisco, California · founded 2018$68Mraised▾
Developer of therapeutics for multiple diseases. It offers gene therapies using ultracompact Cas proteins which modulate gene expression without cutting DNA. The company's platform leverages the power of CRISPR/Cas9 gene editing to target specific genes and achieve therapeutic outcomes. The pipeline includes EPI-321 for facioscapulohumeral muscular dystrophy, EPI-221, EPI-224 for genetic disorder, EPI 114 and EPI 111 for petinas pigmentosa.
- Mar 2025Series B$68M
#10
Funding Raised · San Diego, California · founded 2018 · 10 employees$14.5Mraised▾
Developer of gene therapy products for chronic pain management. The company's novel gene therapy platform to target and imitate the patients with a mutation in the genome where no pain is felt., and apply it to treat pain.
- Apr 2025Grant (prize money)$4.3M
- Apr 2025Grant (prize money)$4.3M
- Apr 2025Grant (prize money)$2.2M
- Sep 2023Grant (prize money)$400K
#11
Acquired · San Diego, California · founded 2020 · 46 employees$123.8Mraised▾
Developer of AAV-based gene therapies to treat genetically defined muscle disease
- May 2024Series A$21.8M
- Jun 2023Series A$51M
- Aug 2021Series A$35M
- Feb 2021Series A$16M
#12
Seed · San Diego, California · founded 2021$26Mraised▾
Developer of AAV-delivered T-cell immunotherapies for the treatment of cancer. The company has TransJoin AAV gene therapy platform enables the creation of immunotherapies, including CAR-T for blood-based cancers, solid tumor metastasis prevention, and a cancer vaccine. The company's product include VNX-101, for the treatment of CD19+ acute lymphoblastic leukemia.
- Sep 2024Seed$26M
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 California is moving
A read on the academic research around Gene Therapy in California — which themes are gaining ground, the institutions and researchers driving them, and how the tone is shifting. Based on 591 papers.
- University of California, Los Angeles118
- University of California, Berkeley86
- University of California, San Francisco79
- California Institute of Technology58
- University of California, Davis54
- Genome editing2→17
- CRISPR3→16
- Gene delivery36→48
- Cas92→11
- Capsid1→8
- Vaccination1→6
- In vivo4→8
- Genome editing2→5
- CRISPR1→3
- Cytotoxic T cell1→3
- Immune system2→5
- Virology2→3
- Nanotechnology2→3