Gene Therapy Delivery
Nanocarriers for gene editing and gene therapy
Arrowhead Research
Pasadena, United States
Biopharmaceutical company developing targeted RNAi therapeutics using proprietary TRiM platform
Selecta Biosciences
Watertown, United States
Selecta Biosciences, Inc., founded in 2007 and based in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company developing its proprietary ImmTOR nanoparticle platform to address a critical challenge in biologics therapy—unwanted immune responses that reduce treatment efficacy. Publicly traded on NASDAQ (SELB) with over $300 million in funding and 100-200 employees, Selecta has pioneered synthetic vaccine particles (SVP) that can selectively modulate immune responses, potentially enabling repeat administration of therapeutic proteins and gene therapies. The company emerged from research on how nanoparticle properties influence immune system activation, discovering that specific nanoparticle designs could induce immune tolerance rather than immunogenicity. Selecta's ImmTOR platform consists of biodegradable polymer nanoparticles incorporating immunomodulatory small molecules (typically rapamycin analogs) that are taken up by immune cells, inducing tolerance to co-administered therapeutic agents. This approach addresses a major limitation of protein therapeutics and gene therapies—the development of neutralizing antibodies that reduce or eliminate treatment efficacy with repeated dosing. For example, many patients with gout receiving enzyme replacement therapy develop anti-drug antibodies that neutralize the therapeutic enzyme, requiring treatment discontinuation. Selecta's lead program, SEL-212, combines ImmTOR nanoparticles with pegsiticase enzyme therapy for severe gout, with clinical trials demonstrating reduced immunogenicity and improved sustained therapeutic response. The company has expanded its platform to address immunogenicity challenges in gene therapy, where immune responses against viral vectors or therapeutic proteins can limit treatment durability. Selecta has established partnerships with major pharmaceutical companies including Sarepta Therapeutics and Asklepios BioPharmaceutical to apply ImmTOR technology to gene therapy programs for muscular dystrophies and other genetic diseases. The company's nanoparticles are engineered with precise sizes (typically 200-500 nanometers), controlled drug loading, and surface properties optimized for uptake by specific immune cell populations. Selecta has developed manufacturing processes producing clinical-grade ImmTOR nanoparticles at scales required for therapeutic applications, addressing a key challenge in nanomedicine translation. The company's approach represents sophisticated application of nanotechnology principles—using nanoparticle properties to program specific biological responses rather than simply delivering drugs. Selecta's clinical programs have generated important data on how synthetic nanoparticles can modulate human immune systems, contributing to fundamental understanding of nanoparticle immunology. As gene therapies and protein biologics become increasingly important in medicine, addressing immunogenicity challenges will be crucial for enabling repeat dosing and expanding patient populations. Selecta's ImmTOR platform offers a potentially broadly applicable solution to this critical problem, positioning the company at the intersection of nanomedicine and immunotherapy, two of the most promising areas in biopharmaceutical development.
Tarveda Therapeutics
Watertown, United States
Developer of miniature drug conjugates for targeted cancer therapy
Moderna
Cambridge, United States
Pioneer in mRNA therapeutics using lipid nanoparticle (LNP) delivery technology for vaccines and medicines
BioNTech
Mainz, Germany
Immunotherapy company leveraging lipid nanoparticle delivery for mRNA vaccines and cancer therapies
Acuitas Therapeutics
Vancouver, Canada
Leading developer of lipid nanoparticle (LNP) delivery systems for nucleic acid therapeutics, partner for COVID-19 vaccines
Precision NanoSystems
Vancouver, Canada
Provider of instruments for scalable nanoparticle manufacturing for gene therapies and mRNA vaccines
Alnylam Pharmaceuticals
Cambridge, United States
Pioneer in RNAi therapeutics using lipid nanoparticle delivery for treatment of genetic diseases
Intellia Therapeutics
Cambridge, United States
Gene editing company using lipid nanoparticle delivery for in vivo CRISPR therapeutics
Nanocure
Tel Aviv, Israel
Nanocure, established in 2015 in Tel Aviv, Israel, is an innovative nanomedicine company focused on developing next-generation cancer therapies through advanced nanoparticle-based drug delivery systems. The company's proprietary platform leverages the unique properties of engineered nanoparticles to achieve precise targeting of cancer cells while minimizing damage to healthy tissue, addressing one of the most significant challenges in oncology treatment. Nanocure's targeted nanoparticle therapeutics are designed to overcome the limitations of conventional chemotherapy, which often causes severe side effects due to non-selective distribution throughout the body. The company's nanoparticle formulations utilize sophisticated surface modifications, including targeting ligands and stealth coatings, to enhance circulation time, improve tumor accumulation through the enhanced permeability and retention effect, and facilitate active targeting to specific cancer cell markers. This multifaceted approach enables higher drug concentrations at tumor sites while reducing systemic exposure and associated toxicities. The technology platform is versatile, capable of encapsulating various therapeutic agents including small molecule drugs, peptides, and nucleic acids, making it applicable to multiple cancer types and treatment modalities. Nanocure's nanoparticles are engineered at the nanoscale (typically 10-200 nanometers) to optimize biological interactions, cellular uptake, and intracellular drug release. The company has secured over $15 million in funding to advance its pipeline through preclinical and clinical development stages. Israel's strong biotechnology ecosystem and expertise in nanomedicine provide Nanocure with access to world-class research facilities, clinical networks, and regulatory support. As cancer treatment increasingly moves toward personalized medicine and targeted therapies, Nanocure's nanoparticle platform represents a significant advancement in delivering more effective and safer cancer treatments, positioning the company as an important player in the rapidly evolving field of cancer nanotherapy.
Codiak BioSciences
Cambridge, United States
Codiak BioSciences, founded in 2015 in Cambridge, Massachusetts, is a pioneering biotechnology company at the forefront of exosome-based therapeutics. The company has developed the proprietary engEx platform, which engineers exosomes—naturally occurring nanovesicles (30-150 nanometers) secreted by cells—as sophisticated drug delivery vehicles. Unlike synthetic nanoparticles, exosomes offer inherent biocompatibility, low immunogenicity, and natural ability to cross biological barriers, making them ideal carriers for therapeutic payloads. Codiak's engEx technology precisely modifies exosomes to display specific targeting molecules on their surface while loading therapeutic cargo inside, creating programmable nanomedicines with enhanced tissue specificity and therapeutic efficacy. The company's lead programs include exoSTING, an exosome-delivered STING agonist for cancer immunotherapy, and exoIL-12, which delivers interleukin-12 for tumor treatment. These engineered exosomes leverage the body's natural intercellular communication system to deliver potent immunomodulatory agents directly to target cells, potentially revolutionizing cancer treatment with improved safety profiles compared to conventional delivery methods. Codiak has raised over $300 million in funding, reflecting significant investor confidence in exosome therapeutics. The Cambridge location places Codiak at the heart of the biotech innovation ecosystem, with access to world-renowned research institutions and clinical infrastructure. The company employs between 100-200 specialists in molecular biology, nanotechnology, and clinical development. Exosomes represent a convergence of cell biology and nanotechnology, and Codiak's platform addresses critical challenges in drug delivery including tumor penetration, immune evasion, and payload stability. As the field of nanomedicine evolves beyond synthetic nanoparticles, Codiak's focus on biological nanovesicles positions it as a leader in next-generation therapeutic delivery, with potential applications extending beyond oncology to neurodegenerative diseases, rare disorders, and vaccines.
NanoVation Therapeutics
Vancouver, Canada
Developer of next-generation lipid nanoparticle delivery systems for RNA therapeutics
Verve Therapeutics
Boston, United States
Gene editing company using lipid nanoparticle delivery for cardiovascular disease treatment
Beam Therapeutics
Cambridge, United States
Pioneer in base editing using lipid nanoparticle delivery for precision genetic medicines
Sirnaomics
Gaithersburg, United States
Clinical-stage biopharmaceutical company developing RNAi therapeutics using polypeptide nanoparticle delivery
CureVac
Tubingen, Germany
Pioneer in mRNA technology using lipid nanoparticle delivery for vaccines and therapeutics
Genevant Sciences
Cambridge, United States
Developer of lipid nanoparticle and ligand conjugate delivery systems for genetic medicines
Arcturus Therapeutics
San Diego, United States
Developer of LUNAR lipid nanoparticle delivery platform for mRNA medicines
Silence Therapeutics
London, United Kingdom
Pioneer in precision RNAi medicines using mRNAi GOLD platform with GalNAc delivery
Dicerna Pharmaceuticals
Lexington, United States
Developer of GalXC RNAi platform for targeted gene silencing therapeutics
Nanovex Biotechnologies
Oviedo, Spain
Biotech company developing extracellular vesicle and exosome-based delivery systems
Arrowhead Pharmaceuticals
Pasadena, United States
Leader in RNAi therapeutics using targeted delivery
Translate Bio
Lexington, United States
mRNA therapeutics company with proprietary LNP technology, acquired by Sanofi
Etherna Immunotherapies
Niel, Belgium
mRNA immunotherapy company with proprietary LNP delivery technology
eTheRNA Immunotherapies
Niel, Belgium
mRNA immunotherapy with proprietary delivery platform
Strand Therapeutics
Boston, United States
Programmable mRNA therapeutics with logic gates
Prime Medicine
Cambridge, United States
Prime editing therapeutics with LNP delivery
Mina Therapeutics
London, United Kingdom
Small activating RNA therapeutics with LNP delivery
Tidal Therapeutics
Cambridge, United States
LNP-based in vivo gene therapy for immune cells, acquired by Sanofi
ReNAgade Therapeutics
Cambridge, United States
RNA delivery technology for extrahepatic targets
Exicure
Chicago, United States
Spherical nucleic acid therapeutics platform
SciSparc
Tel Aviv, Israel
Nano-drug delivery for CNS disorders
NanoTherapeutics
Alachua, United States
Biopharmaceutical nanotechnology development
Ionis Pharmaceuticals
Carlsbad, United States
Antisense oligonucleotide therapeutics for genetic diseases
CRISPR Therapeutics
Zug, Switzerland
CRISPR-based gene editing therapeutics for blood disorders and cancer
Editas Medicine
Cambridge, United States
CRISPR-based medicines for genetic diseases including eye disorders
LNP Japan
Tokyo, Japan
Lipid nanoparticle manufacturing services for RNA therapeutics
Nanobio2Pharma
Bordeaux, France
Nanoparticle drug formulation and CDMO services
Nanomerics
London, United Kingdom
Molecular envelope technology for enhanced drug delivery
Ethris GmbH
Planegg, Germany
SNIM RNA stabilized mRNA therapeutics
Entos Pharmaceuticals
Edmonton, Canada
Fusogenix nucleic acid delivery platform
Arrowhead Pharmaceuticals
Pasadena, United States
TRiM RNAi therapeutics platform
Phio Pharmaceuticals
Marlborough, United States
INTASYL self-delivering RNAi technology
Nitto Denko Avecia
Milford, United States
Oligonucleotide and nucleic acid CDMO services
Ajinomoto Bio-Pharma Services
San Diego, United States
CDMO for oligonucleotides and ADCs
Samyang Biopharmaceuticals
Seoul, South Korea
Genexol-PM polymeric micelle nanoparticle drugs
WuXi AppTec
Shanghai, China
Global CRO/CDMO with nanomedicine manufacturing capabilities
Sixfold Bioscience
London, United Kingdom
AI-driven nanoparticle delivery optimization
NanoSyrinx
Sheffield, United Kingdom
Bacterial contractile injection system for protein delivery
Lonza
Basel, Switzerland
Life sciences and specialty ingredients
Alnylam Pharmaceuticals
Cambridge, United States
Leader in RNAi therapeutics using lipid nanoparticle delivery for genetic diseases
Arbutus Biopharma
Vancouver, Canada
Develops lipid nanoparticle technology for hepatitis B and other infectious diseases
Arcturus Therapeutics
San Diego, United States
RNA medicines company using LUNAR lipid nanoparticle delivery platform
Acuitas Therapeutics
Vancouver, Canada
Develops lipid nanoparticle delivery systems for nucleic acid therapeutics
Genevant Sciences
Cambridge, United States
Lipid nanoparticle and ligand conjugate delivery platforms for genetic medicines
Intellia Therapeutics
Cambridge, United States
CRISPR gene editing company using lipid nanoparticle delivery for in vivo therapeutics
Editas Medicine
Cambridge, United States
Genome editing company using CRISPR with lipid nanoparticle delivery systems
Verve Therapeutics
Cambridge, United States
Gene editing company for cardiovascular disease using base editing and LNP delivery
Ionis Pharmaceuticals
Carlsbad, United States
Pioneer in antisense technology and RNA-targeted therapeutics
Arrowhead Pharmaceuticals
Pasadena, United States
RNAi therapeutics company developing targeted delivery systems for genetic medicines
Dicerna Pharmaceuticals
Lexington, United States
RNAi therapeutics using GalXC delivery platform for liver-targeted gene silencing
Celator Pharmaceuticals
Vancouver, Canada
Liposomal nanoparticle drug delivery for combination cancer therapies
Lipocine
Salt Lake City, United States
Lipid-based oral drug delivery platform for poorly bioavailable compounds
Liquidia Technologies
Morrisville, United States
PRINT nanoparticle technology for precisely engineered drug particles
Afrigen Biologics
Cape Town, South Africa
African mRNA vaccine hub developing lipid nanoparticle delivery technology
Catalent
Somerset, United States
Contract manufacturer with lipid nanoparticle and advanced drug delivery capabilities
Lonza Pharma
Basel, Switzerland
Life sciences company with lipid nanoparticle manufacturing for mRNA therapeutics
Evonik Health Care
Essen, Germany
Specialty chemicals for pharmaceutical drug delivery including lipid nanoparticles
Croda Health Care
Snaith, United Kingdom
Specialty ingredients including lipids for nanoparticle drug delivery
Curia Global
Albany, United States
Contract research and manufacturing with nanoparticle drug delivery expertise
Piramal Pharma Solutions
Mumbai, India
CDMO with capabilities in lipid nanoparticle and advanced drug delivery systems
Recipharm
Stockholm, Sweden
Contract manufacturer with inhaled nanoparticle and specialty drug delivery
Corden Pharma
Basel, Switzerland
CDMO specializing in lipids and lipid nanoparticle manufacturing
Exelead
Indianapolis, United States
CDMO focused on liposomes and lipid nanoparticle drug delivery systems
Polymun Scientific
Klosterneuburg, Austria
CDMO specializing in liposomal and lipid nanoparticle formulations
Northern Lipids
Vancouver, Canada
CDMO for liposomal and lipid nanoparticle drug delivery formulations
NOF Corporation
Tokyo, Japan
PEG-lipid conjugates and functional lipids for liposome drug delivery
Merck Life Science
Darmstadt, Germany
Life science products including lipids and nanomaterials for drug delivery research
University College London Nano
London, United Kingdom
Major UK university with London Centre for Nanotechnology
Alnylam Pharmaceuticals
Cambridge, United States
Pioneer in RNAi therapeutics with lipid nanoparticle delivery
Denali Therapeutics
South San Francisco, United States
Neurodegeneration drugs using Transport Vehicle BBB-crossing technology
Passage Bio
Philadelphia, United States
AAV gene therapies for CNS diseases with novel delivery
Voyager Therapeutics
Cambridge, United States
AAV capsid engineering for CNS gene therapy delivery
4D Molecular Therapeutics
Emeryville, United States
Therapeutic vectors using Directed Evolution AAV capsid discovery
Spark Therapeutics
Philadelphia, United States
Gene therapy pioneer (Luxturna) now part of Roche
Bluebird Bio
Somerville, United States
Gene therapy using lentiviral vectors for genetic diseases
LogicBio Therapeutics
Lexington, United States
Gene insertion technology using AAV and GeneRide platform
Generation Bio
Cambridge, United States
Non-viral gene therapy using lipid nanoparticle delivery
Stoke Therapeutics
Bedford, United States
Antisense oligonucleotides to upregulate protein expression
Dicerna Pharmaceuticals
Lexington, United States
RNAi therapeutics using GalNAc delivery (acquired by Novo Nordisk)
Curia Global
Albany, United States
CDMO with nanoparticle and complex formulation capabilities
Croda International
Snaith, United Kingdom
Specialty chemicals with Avanti lipids for LNP formulations
Entos Pharmaceuticals
Edmonton, Canada
Fusogenix DNA delivery platform for gene therapy
Kernal Biologics
Cambridge, United States
Self-amplifying mRNA with immune-silent delivery
Replicate Bioscience
San Diego, United States
Self-replicating RNA therapeutics platform
Laronde
Cambridge, United States
Endless RNA (eRNA) technology for persistent protein expression
Orna Therapeutics
Cambridge, United States
Circular RNA platform for cell therapy and protein replacement
Chimeron Bio
Philadelphia, United States
Chimeric viral vectors for improved gene therapy delivery
Entrada Therapeutics
Boston, United States
Endosomal Escape Vehicle (EEV) platform for intracellular delivery
PeptiDream
Kawasaki, Japan
Peptide Drug Discovery Platform System for macrocyclic therapeutics
Mersana Therapeutics
Cambridge, United States
Antibody drug conjugates using Dolasynthen platform
Bioasis Technologies
Guilford, Canada
xB3 platform for blood-brain barrier crossing drug delivery
AngioChem
Montreal, Canada
Peptide-drug conjugates for CNS delivery across BBB
Annexon Biosciences
Brisbane, United States
Complement cascade inhibitors for neurological diseases
Prevail Therapeutics
New York, United States
Gene therapies for neurodegenerative diseases (Eli Lilly)
Sangamo Therapeutics
Brisbane, United States
Zinc finger nuclease gene editing and gene regulation
Applied Genetic Technologies
Alachua, United States
AAV gene therapies for rare ophthalmologic diseases
Regenxbio
Rockville, United States
AAV gene therapy using NAV Technology Platform
MeiraGTx
New York, United States
Gene therapy for inherited retinal diseases and beyond
Krystal Biotech
Pittsburgh, United States
Gene therapies using HSV-1 vectors for skin diseases
Sarepta Therapeutics
Cambridge, United States
Gene therapy and RNA therapeutics for muscular dystrophies
Solid Biosciences
Charlestown, United States
Gene therapy for Duchenne muscular dystrophy
Ultragenyx Pharmaceutical
Novato, United States
Gene therapy and biologics for rare genetic diseases
Rocket Pharmaceuticals
Cranbury, United States
Lentiviral and AAV gene therapies for rare diseases
Genethon
Evry, France
Non-profit gene therapy research laboratory
Lysogene
Paris, France
CNS-targeted gene therapy for lysosomal storage diseases
Freeline Therapeutics
Stevenage, United Kingdom
AAV gene therapy for liver-targeted diseases
Orchard Therapeutics
London, United Kingdom
Hematopoietic stem cell gene therapy for rare diseases
Synthego
Redwood City, United States
CRISPR reagent supplier and genome engineering platform
Poseida Therapeutics
San Diego, United States
Gene editing and CAR-T cell therapy using piggyBac system
Precision BioSciences
Durham, United States
ARCUS genome editing for cell therapy and gene correction
Cellectis
Paris, France
TALEN gene editing for allogeneic CAR-T cell therapy
Fate Therapeutics
San Diego, United States
iPSC-derived cell therapies including NK and T cells
Ultragenyx Pharmaceutical
Novato, United States
Biopharmaceutical company developing novel products for rare and ultra-rare diseases
Biomarin Pharmaceutical
San Rafael, United States
Global biotechnology company developing therapies for rare genetic diseases
Vertex Pharmaceuticals
Boston, United States
Global biotechnology company focused on cystic fibrosis and gene editing therapies
Kite Pharma
Santa Monica, United States
Cell therapy company developing engineered autologous CAR T-cell cancer immunotherapies
Bristol Myers Squibb Cell Therapy
Summit, United States
Global biopharmaceutical company with leading CAR T cell therapy franchise
Legend Biotech
Somerset, United States
Global biotechnology company developing novel cell therapies for oncology
Allogene Therapeutics
South San Francisco, United States
Clinical-stage biotechnology company pioneering allogeneic CAR T cell therapies
Caribou Biosciences
Berkeley, United States
Clinical-stage CRISPR genome editing company developing transformative therapies
Lyell Immunopharma
South San Francisco, United States
T cell reprogramming company developing next-generation cell therapies for solid tumors
Adicet Bio
Menlo Park, United States
Clinical-stage biotechnology company developing allogeneic gamma delta T cell therapies
Century Therapeutics
Philadelphia, United States
Biotechnology company developing iPSC-derived allogeneic cell therapies for cancer
Takeda Cell Therapy
Tokyo, Japan
Global pharmaceutical company with advanced cell therapy programs
Resilience
San Diego, United States
Biomanufacturing company building sustainable network for complex medicine production
Lonza Cell and Gene
Basel, Switzerland
Global CDMO providing cell and gene therapy manufacturing services
Catalent Cell and Gene
Somerset, United States
Global CDMO offering cell therapy, gene therapy, and viral vector manufacturing
Charles River Cell Solutions
Wilmington, United States
Contract research organization providing cell and gene therapy development services
WuXi Advanced Therapies
Shanghai, China
Global contract development and manufacturing organization for advanced therapies
Oxford Biomedica
Oxford, United Kingdom
Gene and cell therapy group specializing in lentiviral vector development and manufacturing
uniQure
Amsterdam, Netherlands
Gene therapy company developing AAV-based treatments for severe genetic diseases
Rocket Pharmaceuticals
Cranbury, United States
Gene therapy company developing treatments for rare childhood diseases
Asklepios BioPharmaceutical
Research Triangle Park, United States
Gene therapy company developing AAV-based treatments for genetic diseases
Abeona Therapeutics
New York, United States
Clinical-stage biopharmaceutical company developing gene therapies for rare diseases
LogicBio Therapeutics
Lexington, United States
Genome editing company developing gene insertion medicines for pediatric diseases
Lexeo Therapeutics
New York, United States
Gene therapy company developing treatments for cardiac and neurological diseases
Neurogene
New York, United States
Gene therapy company developing treatments for rare neurological diseases in children
Sana Biotechnology
Seattle, United States
Biotechnology company engineering cells to treat disease using in vivo and ex vivo approaches
Taysha Gene Therapies
Dallas, United States
Gene therapy company developing treatments for monogenic CNS diseases
Inari Agriculture
Cambridge, United States
Seed design company using gene editing for sustainable crop improvement
ARIZ Precision Medicine
San Diego, United States
Nanotechnology for targeted cancer drug delivery using siRNA nanoparticles
SeraGene Therapeutics
London, United Kingdom
RNA and nanomedicine treatments for blood disorders
Madrigal Mental Care
Boston, United States
Organic nanoparticle drug delivery for mental health
Aurora Biosynthetics
Singapore, Singapore
GMP manufacturing of plasmid DNA, mRNA and lipid nanoparticles for Asia Pacific
Exsilio Therapeutics
Boston, United States
mRNA platform for revocable genetic medicines
Helex
Boston, United States
Lipid nanoparticle technology for targeted drug delivery for kidney diseases
DNA Nanobots
Cambridge, United States
DNA-based nanodevices for targeted therapeutics delivery, vaccines, and diagnostics
BioPact
Delft, Netherlands
Nanoformulation technology for drug delivery applications
Evox Therapeutics
Oxford, United Kingdom
DeliverEX platform for engineered exosome therapeutics
Aruna Bio
Athens, United States
Neural-derived exosomes for CNS drug delivery crossing BBB
ExonanoRNA
Boston, United States
RNA nanoparticles and exosomes for cancer therapeutics
Phosphorex
Hopkinton, United States
CDMO for lipid nanoparticle and liposome drug delivery