502 Gene Therapy, RNA & Peptide Therapy Companies - Worldwide

Perpetual Medicines	-	Computational peptide therapeutics
Providence Therapeutics	AB - Calgary	mRNA Cancer Vaccines
Amytrx Therapeutics	AL - Birmingham	Peptide Therapies
Protagonist Therapeutics	Australia - Brisbane	Oral peptides
Neuroscientific Biopharmaceuticals	Australia - Cottesloe	Peptide for neurological treatments
InterK Peptide Therapeutics	Australia - Lane Cove West	Synthetic Peptides to Treat Cancer
Lateral Pharma	Australia - Melbourne	Repurposed Peptide
PYC Therapeutics	Australia - Nedlands	Peptide drug discovery
Imugene	Australia - Sydney	Peptide immunotherapy
Apellis Pharmaceuticals	Australia - Victoria	Cyclic peptide analog
APEPTICO	Austria - Vienna	Peptide Drugs
invIOs	Austria - Vienna	Novel cancer immunotherapies, cell, siRNA, peptides
Susavion Biosciences	AZ - Tempe	Bio-active Peptides
Ceria Therapeutics	AZ - Tucson	mRNA Delivery
Symvivo	BC - Burnaby	Bacterial-based gene delivery platform
Nanovation Therapeutics	BC - Vancouver	Gene Therapy
NervGen Pharma	BC - Vancouver	Peptide inhibitor of protein tyrosine phosphatase sigma
Flamingo Therapeutics	Belgium -	RNA Therapeutics
Genflow Biosciences	Belgium - Charleroi	Anti-aging Gene Therapy
Rejuvenate Biomed	Belgium - Diepenbeek	Combination Drugs for Aging
Aelin Therapeutics	Belgium - Leuven	Peptide-based Biologics
Ziphius Vaccines	Belgium - Merelbeke	Self-amplifying RNA Vaccine
Aelin Therapeutics	Belgium - Zwijnaarde	Peptide-based Biologics
Sapu BioScience	CA - Agoura Hills	RNA & Small Molecule Immuno-oncology Therapies
Regel Therapeutics	CA - Berkeley	AAV gene delivery
Mammoth Biosciences	CA - Brisbane	CRISPR, Gene-Editing Therapies
Ultragenyx Pharmaceutical	CA - Brisbane	Small Molecules, Biologics
Attralus	CA - Burlingame	Amyloidosis Treatments
Creyon Bio	CA - Carlsbad	Oligonucleotide-based Medicines
Ionis Pharmaceuticals	CA - Carlsbad	RNA
PYC Therapeutics	CA - Carlsbad	Peptide drug discovery
NeuroQure	CA - Coto de Caza	Gene Therapy for Neurological Disorders
4D Molecular Therapeutics	CA - Emeryville	Gene Therapy
Dynavax Technologies	CA - Emeryville	Synthetic oligonucleotides vaccines
Benitec Biopharma	CA - Hayward	DdRNAi
Spotlight Therapeutics	CA - Hayward	In vivo Gene Editing
Matrix Biomed	CA - Irvine	Gene therapy for various cancers
Nuravax	CA - Irvine	DNA Vaccine
Oculogenex	CA - La Habra	Gene Therapy for Vision
Pfizer	CA - La Jolla	Peptides, Monoclonal Antibodies
DiNAQOR	CA - Laguna Hills	Gene Therapy for Heart Conditions
Kairos Pharma	CA - Los Angeles	Cell Therapy, Antibodies, Small Molecules
Vault Pharma	CA - Los Angeles	Peptide payloads for immune signaling
Avellino Laboratories	CA - Menlo Park	Gene Therapy, Molecular Diagnostics
CohBar	CA - Menlo Park	Mitochondrial-Derived Peptides
Grace Science	CA - Menlo Park	Therapies based on the function of NGLY1, AAV gene therapy, small molecules
NysnoBio	CA - Mill Valley	Parkin protein therapeutics
Rampart Bioscience	CA - Monrovia	Non-viral, durable gene therapy
Rejuvenation Technologies	CA - Mountain View	Telomere Extension via Nucleoside-modified TERT mRNA
Turn Biotechnologies	CA - Mountain View	mRNA Therapies
Protagonist Therapeutics	CA - Newark	Oral peptides for GI indications
Alzamend Neuro	CA - Newport Beach	Alzheimer's Treatment
Ultragenyx Pharmaceutical	CA - Novato	Small Molecules, Biologics, Gene Therapy, mRNA, Antisense Oligo
OrthoTrophix	CA - Oakland	Bone, cartilage and teeth therapies
Aspa Therapeutics	CA - Palo Alto	Gene Therapy
Genascence	CA - Palo Alto	Musculoskeletal Gene Therapies
Arrowhead Pharmaceuticals	CA - Pasadena	RNAi
Duet BioTherapeutics	CA - Pasadena	Bifunctional oligonucleotides for immuno-oncology
Genervon Biopharmaceuticals	CA - Pasadena	Synthetic Peptide
Adverum Biotechnologies	CA - Redwood City	Adeno-Associated Virus Delivery, Gene Therapy
Kriya Therapeutics	CA - Redwood City	Gene Therapy
Ocelot Bio	CA - Redwood City	Peptide therapeutic to treat complications of end-stage liver disease
Oryn Therapeutics	CA - Redwood City	Macrocyclic peptide therapeutics
StemCardia	CA - Sacramento	Cell & gene therapies engineered to repair the heart
Abintus Bio	CA - San Diego	Gene Therapy
ADARx Pharmaceuticals	CA - San Diego	RNA Therapeutics
Amplo Biotechnology	CA - San Diego	Treatment for congenital myasthenic syndromes
Arcturus Therapeutics	CA - San Diego	Lipid nanoparticle (LNP) mRNA drug delivery systems
Avidity Biosciences	CA - San Diego	Antibody-Oligonucleotide Conjugates
BioNTech	CA - San Diego	mRNA, Vaccines, Biologics
Cidara Therapeutics	CA - San Diego	Small molecules, peptide
Eli Lilly	CA - San Diego	Biologics Discovery
HAYA Therapeutics	CA - San Diego	RNA-based, RNA-targeting Fibrotic Therapies
Impilo Therapeutics	CA - San Diego	ASOs
Inovio Pharmaceuticals	CA - San Diego	DNA Vaccines
Kate Therapeutics	CA - San Diego	AAV-based gene therapy for muscle disease
Lassogen	CA - San Diego	Lasso peptide-based therapies
Medikine	CA - San Diego	Small Molecules, Peptide
Navega Therapeutics	CA - San Diego	Chronic Pain Treatment
Nitto BioPharma	CA - San Diego	Drug Delivery of siRNA
Novartis	CA - San Diego	Small Molecules, Vaccines, Biologics
Peptide Logic	CA - San Diego	Peptide-Antibody Conjugates
Poseida Therapeutics	CA - San Diego	Genome Editing Technologies
Regulus Therapeutics	CA - San Diego	MicroRNAs
Replay	CA - San Diego	HSV Vector Gene Therapies
Replicate Bioscience	CA - San Diego	Preventing oncology drug resistance
Sentia Medical Sciences	CA - San Diego	Novel peptide therapeutics
Sirius Therapeutics	CA - San Diego	RNAi Therapies
Splash Pharmaceuticals	CA - San Diego	Peptide
Theragene Pharmaceuticals	CA - San Diego	Immunotherapy, gene therapy and peptide delivery
Apellis Pharmaceuticals	CA - San Francisco	Cyclic peptide analog
Cellastra	CA - San Francisco	Anti-scarring gene therapy/peptides
DNARx	CA - San Francisco	DNARx specializes in developing gene delivery technologies for cancer, heart disease, and neurodegenerative diseases including Alzheimer’s
ExcepGen	CA - San Francisco	RNA Therapeutics Platform
Excision BioTherapeutics	CA - San Francisco	Gene Editing
Ligandal	CA - San Francisco	Nanomedicine delivery for Genetic Medicines
Ray Therapeutics	CA - San Francisco	Optogenetic Gene Therapy
Siren Biotechnology	CA - San Francisco	AAV Immuno-Gene Therapy
Vir Biotechnology	CA - San Francisco	Infectious disease therapies
Mahzi Therapeutics	CA - San Mateo	Gene therapy for rare genetic neurodevelopmental disorders
Artery Therapeutics	CA - San Ramon	Peptide therapies
Unnatural Products	CA - Santa Cruz	Macrocylic Peptides plus AI
Visgenx	CA - Santa Cruz	Therapeutics for Macular Degeneration and Other Age-Related Disorders
Aligos Therapeutics	CA - South SF	Therapies for hepatologic diseases and viral infections
Circle Pharma	CA - South SF	Macrocyclic Therapeutics
Encoded Therapeutics	CA - South SF	Gene Therapy
GenEdit	CA - South SF	Nonviral CRISPR-delivery system
Maze Therapeutics	CA - South SF	Small Molecules, Gene Therapy, Antisense Oligonucleotides, Biologics
Senti Biosciences	CA - South SF	Gene Circuits; Cell & Gene Therapies
SonoThera	CA - South SF	Ultrasound-Guided Gene Therapy
Switch Therapeutics	CA - South SF	RNAi Therapies
Tenaya Therapeutics	CA - South SF	Cardiovascular Therapeutics
Capsida Biotherapeutics	CA - Thousand Oaks	AAV Gene Therapy
Emmaus Life Sciences	CA - Torrance	Oral amino acid treatment
Riptide Bioscience	CA - Vallejo	Peptide Therapeutics
Shenogen Pharma	China - Beijing	Small Molecules, Biologics, Viral
Therorna	China - Beijing	RNA Therapies
HitGen	China - Chengdu	DNA encoded library for drug discovery
Exegenesis Bio	China - Hangzhou	Gene Therapy
Sciwind Biosciences	China - Hangzhou	Oral peptide delivery
Immuno Cure Biotech	China - Hong Kong	Therapeutic Antibodies, DNA Vaccines
Suzhou Ribo Life Science Company	China - Kunshan	RNAi
Ractigen Therapeutics	China - Nantong	Small activating RNA
Argo Biopharma	China - Shanghai	siRNA Drugs
BDgene Therapeutics	China - Shanghai	mRNA-based CRISPR editing components
Belief BioMed	China - Shanghai	AAV-delivered Gene Therapies
HuidaGene Therapeutics	China - Shanghai	Gene Therapy, Gene editing platform, delivery vector platform
Immorna Biotherapeutics	China - Shanghai	Self-Replicating RNA
NeuShen Therapeutics	China - Shanghai	AAV Gene Therapy, Small Molecules
Rona Therapeutics	China - Shanghai	RNA Therapeutics Platform
RVAC Medicines	China - Shanghai	mRNA, vaccines, and lipid nanoparticle technologies
Sirius Therapeutics	China - Shanghai	RNAi Therapies
Skyline Therapeutics	China - Shanghai	AAV Gene Therapy
StemiRNA Therapeutics	China - Shanghai	mRNA Treatments
Abogen	China - Suzhou	mRNA
CureGenetics	China - Suzhou	Gene-editing - Diagnostics, Therapies
Frontera Therapeutics	China - Suzhou	Gene Therapy Development
Gritgen Therapeutics	China - Suzhou	Gene Therapy
Sirnaomics	China - Suzhou	RNAi Therapeutics
Starna Therapeutics	China - Suzhou	mRNA vaccine, therapies
Recbio Technology	China - Taizhou	mRNA Covid Vaccine
Neurophth Therapeutics	China - Wuhan	Ophthalmology Gene Therapy
Neurexis Therapeutics	CO - Aurora	Peptide for Cardiac Indications
Viridian Therapeutics	CO - Boulder	MicroRNA
Mitotherapeutix	CT - Farmington	siRNA drugs
RIGImmune	CT - Farmington	RNA Immunotherapeutics
Oncovir	DC - Washington	dsRNA viral mimic
Altamira Therapeutics	DE - Newark	RNA Therapies
Antag Therapeutics	Denmark - Copenhagen	Therapeutic peptides for dietary-related metabolic diseases
Avilex Pharma	Denmark - Copenhagen	Peptide for ischemic stroke
IO Biotech	Denmark - Copenhagen	Immuno-Oncology
SNIPR BIOME	Denmark - Copenhagen	CRISPR for microbiome management, genetically-modified bacteriophages
ResoTher Pharma	Denmark - Holte	Cardiovascular Treatment
Zealand Pharma	Denmark - Soborg	Peptide Drugs
Valo Therapeutics	Finland - Helsinki	Oncolytic Virus, Peptide
Beacon Therapeutics	FL - Alachua	Gene Therapy
Oncogenuity	FL - Bay Harbor Islands	Oligonucleotide platform for the treatment of genetically driven cancers
Adolore BioTherapeutics	FL - Delray Beach	Gene Therapy Analgesic for Pain

Ambulero	FL - Miami	Cell & Gene Therapies
San Rocco Therapeutics	FL - Tampa	Gene Therapy, Orphan Diseases
TuHURA Biosciences	FL - Tampa	Cancer Therapies
GlioCure	France - Angers	Peptide
InnoSkel	France - Biot	Therapies for Rare Skeletal Diseases
Amolyt Pharma	France - Ecully	Therapeutic Peptides
Atamyo Therapeutics	France - Evry	Gene Therapies targeting neuromuscular diseases
Généthon	France - Evry	Gene Therapy
Vaxeal	France - Evry	Cancer Immunotherapies, Long Synthetic Peptides
PeptiMimesis	France - Illkirch	Therapeutic Peptides
Transgene	France - Illkirch-Graffenstaden	Viral Vector Therapeutics, Gene Therapy
Enterosys	France - Labege	Oral Peptides
Axoltis Pharma	France - Lyon	Neurological Peptide Drugs
Nosopharm	France - Lyon	Anti-infectives, Peptide
Transgene	France - Lyon	Viral Vector Therapeutics, Gene Therapy
Eli Lilly	France - Neuilly-sur-Seine	Small Molecules, Biologics
Coave Therapeutics	France - Paris	Gene Therapy in Ophthalmology
EG 427	France - Paris	Pinpoint Gene Therapy
GenSight Biologics	France - Paris	Ophthalmic Gene Therapy
Inotrem	France - Paris	Peptide Therapy
Invectys	France - Paris	DNA Vaccine
Jalon Therapeutics	France - Paris	Cell-Penetrating Peptide
PEP-Therapy	France - Paris	Peptide Therapies
PulseSight Biosciences	France - Paris	Ophthalmic Treatment Plasmid
SparingVision	France - Paris	Retinal Gene Therapy Treatments
Valerio Therapeutics	France - Paris	Orphan Diseases, Decoy Oligonucleotides
Vivet Therapeutics	France - Paris	Gene Therapy for Orphan Diseases
Odimma Therapeutics	France - Strasbourg	Personalized Cancer Immunotherapy
Inotrem	France - Vandoeuvre	Peptide Therapy
Expression Therapeutics	GA - Tucker	Gene Therapies
Vernagen	GA - Tucker	mRNA Therapies
Berlin Cures	Germany - Berlin	Aptamer for Cardio Indication
BioNTech	Germany - Berlin	mRNA, Vaccines, Biologics
MyoPax	Germany - Berlin	Cell & Gene Therapies
Silence Therapeutics	Germany - Berlin	RNAi
CureVac	Germany - Frankfurt	MRNA treatments
DiNAQOR	Germany - Hamburg	Gene Therapy for Heart Conditions
GQ Bio	Germany - Hamburg	Gene Therapy for Osteoarthritis
Cardior Pharmaceuticals	Germany - Hanover	Non-coding RNA based therapeutics
AaviGen	Germany - Heidelberg	Precision gene therapies for heart failure
Pantherna Therapeutics	Germany - Hennigsdorf	Nanoparticle Delivery for mRNA drugs
BioNTech	Germany - Idar-Oberstein	mRNA, Vaccines, Biologics
BioNTech	Germany - Mainz	mRNA, Vaccines, Biologics
Sterna Biologicals & KG	Germany - Marburg	Antisense Molecule - DNAzyme
Apellis Pharmaceuticals	Germany - München	Cyclic peptide analog
Ethris	Germany - Planegg	MRNA-based therapies, vaccines
Freeline Therapeutics	Germany - Planegg	Protein replacement via Gene Therapy
Secarna Pharmaceuticals	Germany - Planegg	Antisense Oligonucleotide Therapies
ViGeneron	Germany - Planegg	AAV Gene Therapy
CureVac	Germany - Tübingen	MRNA treatments
Oryx Translational Medicine	Germany - Vaterstetten	Cancer Immunotherapy
Amyndas Pharmaceuticals	Greece - Glyfada	Peptide for various disorders
Flashpoint Therapeutics	IL - Chicago	Nanoscale design of therapeutic architecture to optimize anti-cancer immune response
MAIA Biotechnology	IL - Chicago	Peptide Cancer Therapies
Oxalo Therapeutics	IL - Chicago	Peptide-based therapeutics to treat rare renal diseases and prevent recurrent kidney stones
Jaguar Gene Therapy	IL - Lake Forest	AAV-Based Gene Therapies
MBX Biosciences	IN - Carmel	Hormone Analogues for Rare Endocrine Diseases
Eli Lilly	IN - Indianapolis	Small Molecules, Biologics
Altamira Therapeutics	Ireland - Dublin	RNA Therapies
Ovoca Bio	Ireland - Dublin	Nasally Delivered Synthetic Peptide
Eli Lilly	Ireland - Kinsale	Small Molecules, Biologics
SpliSense	Israel - Jerusalem	Antisense Oligonucleotides
Skip Therapeutics	Israel - Ness Ziona	RNA-based therapies for rare genetic disorders and common diseases
Art Bioscience	Israel - Rehovot	DMD Gene Therapy
Eleven Therapeutics	Israel - Tel Aviv	Small interfering RNAs with ultra-long durability by utilizing high throughput combinatorial chemistry and AI
Altheia Science	Italy - Milan	Cell & Gene Therapy for Autoimmune Disorders
Genenta Science	Italy - Milan	Gene Therapy
Genespire	Italy - Milan	Gene Therapy
Holostem Tarapie Avanzate	Italy - Modena	Cell & Gene Therapy
Eli Lilly	Italy - Sesto Fiorentino	Small Molecules, Biologics
Kither Biotech	Italy - Torino	Therapies for the treatment of rare pulmonary diseases
Heqet Therapeutics	Italy - Turin	Noncoding RNA as Therapeutic
CellGenTech	Japan - Chiba	Ex-Vivo Gene Therapy
WaVe Life Sciences	Japan - Kagoshima	Stereopure oligonucleotides
Gene Therapy Research Institute (GTRI)	Japan - Kanagawa	Gene Therapy
Oncotherapy Science	Japan - Kawasaki	Small Molecule, peptides, antibody therapies, contract analytical
Momotaro-Gene	Japan - Okayama	Cancer Gene Therapy
Luxna Biotech	Japan - Osaka	Oligonucleotide Therapeutics
Epsilon Molecular Engineering	Japan - Saitama	Heavy chain single domain antibodies
Takara Bio	Japan - Shiga	Gene Therapy, Agribiotech, Biotech Lab Technology
BrightPath Biotherapeutics	Japan - Tokyo	Peptide Therapies
EA Pharma	Japan - Tokyo	Amino Acid Treatments
Modulus Discovery	Japan - Tokyo	Peptide Discovery?
NapaJen Pharma	Japan - Tokyo	Oligonucleotide Drugs
Restore Vision	Japan - Tokyo	Gene Therapy for Blindness due to Retinal Disorders
Ribomic	Japan - Tokyo	Aptamers
TAGCyx Biotechnologies	Japan - Tokyo	Oligonucleotide Drug Discovery Platform
TME Therapeutics	Japan - Tokyo	dsRNA Treatment for Cancer
Caregen Biopharma	Korea - Anyang	Growth Factors & Biomimetic Peptides
Biorchestra	Korea - Daejeon	Novel RNA-based Therapeutics
Gemvax & KAEL	Korea - Daejeon	Peptide Immunotherapy
GenKOre	Korea - Daejeon	Gene-editing therapies
Peptron	Korea - Daejeon	Peptide-based Medicine Platform
OliPass	Korea - Gyeonggi	Peptide nucleic acid derivatives
OliX Pharmaceuticals	Korea - Gyeonggi	RNAi Treatments
PinotBio	Korea - Gyeonggi-do	Small molecules, ADC, Nucleoside-based Therapy
Aston Sci.	Korea - Seoul	Therapeutic cancer vaccines and proteostasis modulators
Curamys	Korea - Seoul	Stem Cell Fusion Technology
Genexine	Korea - Seoul	Biologics, DNA Plasmid Vaccine
GenKOre	Korea - Seoul	Gene-editing therapies
Helixmith	Korea - Seoul	plasmid DNA based gene therapy
Kolon Life Science	Korea - Seoul	Cell & Gene Therapy, APIs
MDimune	Korea - Seoul	Drug Delivery Based on Cell-Derived Vesicles
siRNAgen	Korea - ZZ	siRNA Therapeutics
Frontera Therapeutics	MA - Bedford	Gene Therapy Development
Homology Medicines	MA - Bedford	Gene-editing via homologous recombination
Stoke Therapeutics	MA - Bedford	Antisense Oligonucleotide Medicines
Ultragenyx Pharmaceutical	MA - Bedford	Small Molecules, Biologics
Aileron Therapeutics	MA - Boston	Perpetually Active Peptides
Akouos	MA - Boston	Gene Therapy for Hearing Disorders
Ampersand Biomedicines	MA - Boston	AND-Body Therapeutics
Ascidian Therapeutics	MA - Boston	Mutated exon replacement
Atalanta Therapeutics	MA - Boston	RNAi therapeutics
Constant Therapeutics	MA - Boston	Peptide Treatments
CureVac	MA - Boston	MRNA treatments
Envoya	MA - Boston	Gene Therapy
hC Bioscience	MA - Boston	tRNA-based Therapeutics
Inozyme Pharma	MA - Boston	Enzyme Replacement Therapy
Kelonia Therapeutics	MA - Boston	in vivo CAR T Gene Therapy
Orchard Therapeutics	MA - Boston	Ex-vivo autologous hematopoietic stem cell gene therapy
QLD Biotherapeutics	MA - Boston	Cell & Gene Therapies
Rhythm Pharmaceuticals	MA - Boston	Peptide Therapeutics
Strand Therapeutics	MA - Boston	Gene Therapy
SYTE.bio	MA - Boston	Precision therapeutics using its proprietary Linear DNA and Circular RNA technology platforms
TransCode Therapeutics	MA - Boston	RNA Delivery, Small Molecule
Transgene	MA - Boston	Viral Vector Therapeutics, Gene Therapy
Verve Therapeutics	MA - Boston	Gene-editing Treatments
Aera Therapeutics	MA - Cambridge	Genetic Medicines
Alltrna	MA - Cambridge	tRNA therapies
Alnylam Pharmaceuticals	MA - Cambridge	RNAi
AVROBIO	MA - Cambridge	Cellular & Gene Therapies
Beacon Therapeutics	MA - Cambridge	Gene Therapy
Bicycle Therapeutics	MA - Cambridge	Peptides
BioNTech	MA - Cambridge	mRNA, Vaccines, Biologics
Biorchestra	MA - Cambridge	Novel RNA-based Therapeutics
Eli Lilly	MA - Cambridge	Small Molecules, Biologics
Generation Bio	MA - Cambridge	Gene Therapy
Helix Nanotechnologies	MA - Cambridge	DNA Therapy
Hunterian Medicine	MA - Cambridge	CRISPR-based drug discovery
Innovac Therapeutics	MA - Cambridge	RNA Therapeutics
Intergalactic Therapeutics	MA - Cambridge	Non-viral Gene Therapy
Korro Bio	MA - Cambridge	RNA-editing Drugs
Lytica Therapeutics	MA - Cambridge	Antimicrobial peptide Design
Matice Biosciences	MA - Cambridge	Peptides for Scar Reduction
Moderna Therapeutics	MA - Cambridge	RNA Therapies
Novartis	MA - Cambridge	Small Molecules, Vaccines, Biologics / Diagnostics
Obsidian Therapeutics	MA - Cambridge	Cell & Gene Therapies
Omega Therapeutics	MA - Cambridge	Gene Editing
Orbital Therapeutics	MA - Cambridge	RNA technology (not RNAi)
Orna Therapeutics	MA - Cambridge	oRNA, Delivery
ProQR Therapeutics	MA - Cambridge	Oligonucleotide Therapy
Regel Therapeutics	MA - Cambridge	AAV gene delivery
Repertoire Immune Medicines	MA - Cambridge	Cell & Peptide-Based Therapies
SalioGen Therapeutics	MA - Cambridge	Gene Therapy Platform Technology
SmartPharm Therapeutics	MA - Cambridge	Production of biologics within patients via gene therapy
STRM.BIO	MA - Cambridge	Extracellular vesicles (EVs) to deliver gene therapies
Tevard Biosciences	MA - Cambridge	tRNA-based Gene Therapies
Ultragenyx Pharmaceutical	MA - Cambridge	Small Molecules, Biologics
Vigeo Therapeutics	MA - Cambridge	Biologics for Cancer

WaVe Life Sciences	MA - Cambridge	Stereopure oligonucleotides
Solid Biosciences	MA - Charlestown	Gene Therapy, Devices, Biologics, Small Molecules all for DMD
Comanche Biopharma	MA - Concord	siRNA for Preeclampsia
SynerK	MA - Concord	RNA-targeted Therapies
Carbon Biosciences	MA - Lexington	Gene Therapy
Exegenesis Bio	MA - Lexington	Gene Therapy
Fractyl Health	MA - Lexington	Thermal ablation therapy, Gene Therapy
Guardian Therapeutics	MA - Lexington	Aptamer Therapeutics
LogicBio Therapeutics	MA - Lexington	Gene Therapy
uniQure	MA - Lexington	Gene Therapy
Voyager Therapeutics	MA - Lexington	AAV capsid Gene Therapy
Alcyone Therapeutics	MA - Lowell	Gene Therapy
Phio Pharmaceuticals	MA - Marlborough	RNAi
RVAC Medicines	MA - Medford	mRNA, vaccines, and lipid nanoparticle technologies
Avenge Bio	MA - Natick	Cell & Gene Therapies
SOLA Biosciences	MA - Natick	Gene Therapies for Protein Misfolding Diseases
Remedium Bio	MA - Needham	Gene Therapy
Courage Therapeutics	MA - Newton	Eating Disorder Treatments
Lactocore	MA - Newton	CNS Peptides
Moderna Therapeutics	MA - Norwood	RNA Therapies
bluebird bio	MA - Somerville	Gene Therapy, Cellular Extraction & Reintroduction
Myrtelle	MA - Wakefield	Gene Therapy
Affinia Therapeutics	MA - Waltham	Gene Delivery
Apellis Pharmaceuticals	MA - Waltham	Cyclic peptide analog
Hemera Biosciences	MA - Waltham	Gene Therapy
OncXerna Therapeutics	MA - Waltham	Cancer Therapeutics
Viridian Therapeutics	MA - Waltham	MicroRNA
Sonata Therapeutics	MA - Watertown	Small Molecules, Gene Therapy
SanegeneBio	MA - Woburn	siRNA Drug Discovery
Ultragenyx Pharmaceutical	MA - Woburn	Small Molecules, Biologics
Leal Therapeutics	MA - Worcester	CNS Therapies
Mustang Bio	MA - Worcester	Immunotherapies
Zata Pharmaceuticals	MA - Worcester	RNA, DNA Therapeutics
Viramatix Biotechnology	Malaysia - Kajang	Peptide based anti-viral therapeutics
AsclepiX Therapeutics	MD - Bethesda	Peptide drugs
CiVi Biopharma	MD - Chevy Chase	Novel therapies against cardiometabolic diseases
Ibex Biosciences	MD - Cumberland	Biologics, Cell Therapy, Gene Therapy
BioNTech	MD - Gaithersburg	mRNA, Vaccines, Biologics
miRecule	MD - Gaithersburg	microRNA Therapeutics for Cancer
RNAimmune	MD - Gaithersburg	mRNA therapies
Precigen	MD - Germantown	Cell & Gene Therapy
Sirnaomics	MD - Germantown	RNAi Therapeutics
AAVogen	MD - Rockville	Gene therapeutics to strengthen muscle and fight disease
Addimmune	MD - Rockville	HIV Gene Therapy
American Gene Technologies	MD - Rockville	Gene Therapy and Immunotherapy Company. Lentiviral Vector Platform and Indication-Specific Technologies. Lead Therapeutics for an HIV Cure, Liver Cancer Cure, and PKU therapy
Primera Therapeutics	MD - Rockville	mtDNA engineering toolbox for mitochondrial diseases
RegeneRx Biopharmaceuticals	MD - Rockville	Peptides
ReGenX Biosciences	MD - Rockville	Gene Therapy, Biologics
Rithim Biologics	MD - Woodbine	Gene Therapy for Tachycardias
Novartis	Mexico - Mexico City	Small Molecules, Vaccines, Biologics
Eli Lilly	Mexico - San José	Small Molecules, Biologics
Diapin Therapeutics	MI - Ann Arbor	Peptide for diabetes
ONL Therapeutics	MI - Ann Arbor	Small molecule peptide
CircNova	MI - Detroit	Circular RNA
Esperovax	MI - Plymouth	Targeted Oral mRNA
Vir Biotechnology	MO - St. Louis	Infectious disease therapies
Soricimed Biopharma	NB - Moncton	Proprietary TRPV6-targeting peptide platform
Bedrock Therapeutics	NC - Chapel Hill	AAV Research and Development Gene Therapy
Atsena Therapeutics	NC - Durham	Gene Therapy
CLAIRIgene	NC - Durham	Viral Vector Gene Therapy
Eldec Pharmaceuticals	NC - Durham	Peptide Therapies
Fennec Pharmaceuticals	NC - Durham	Small Molecules, Cyclic Peptides
Opus Genetics	NC - Durham	Ophthalmology Gene Therapy
Pfizer	NC - Durham	Small Molecules, Vaccines, Biologics
StrideBio	NC - Durham	Antibody-Evading Gene Therapy
Torque Bio	NC - Durham	Gene Therapies
Tune Therapeutics	NC - Durham	Epigenetic modification to modulate gene expression
Enzerna Biosciences	NC - Morrisville	RNA Editing Gene Therapy
Kriya Therapeutics	NC - Morrisville	Gene Therapy
Adrenas Therapeutics	NC - Raleigh	Gene Therapy
GeneVentiv Therapeutics	NC - Raleigh	Gene Therapy
RainBIO	NC - Raleigh	Cornea Gene Therapy
Black Swan Pharmaceuticals	NC - Wake Forest	Antisense RNA-based therapeutics for ALS
uniQure	Netherlands - Amsterdam	Gene Therapy
VectorY	Netherlands - Amsterdam	Vectorized Antibodies
Temple Therapeutics	Netherlands - Geleen	Amino Acid-based Surgical Treatment
Amarna Therapeutics	Netherlands - Leiden	Viral Gene Delivery Platform
ProQR Therapeutics	Netherlands - Leiden	Oligonucleotide Therapy
Vico Therapeutics	Netherlands - Leiden	Oligonucleotide based RNA modulating therapeutics
Chondropeptix	Netherlands - Maastricht	Peptide for cartilage improvement
Phlox Therapeutics	Netherlands - Naarden	Gene Therapy for Heart Disease
Hybridize Therapeutics	Netherlands - Oegstgeest	RNA treatments for kidney disease
DegenRx	Netherlands - Tilburg	Gene Therapy, Antibodies
Treeway	Netherlands - Tilburg	Small Molecules, Gene Therapy
Amaroq Therapeutics	New Zealand - Auckland	lncRNA Therapeutics
CuroNZ	New Zealand - Hamilton	Neural Regeneration Peptides
Neurotez	NJ - Bridgewater	Peptide Treatment for Alzheimer's
IVERIC Bio	NJ - Cranbury	Aptamers, Biologics
Rocket Pharmaceuticals	NJ - Cranbury	Gene Therapies
Novartis	NJ - East Hanover	Small Molecules, Vaccines, Biologics
Silence Therapeutics	NJ - Hoboken	RNAi
EOM Pharmaceuticals	NJ - Montvale	Peptide-nucleic Acid Solutions
ReGenTree	NJ - Princeton	Therapeutic Peptides
PhageNova Bio	NJ - Summit	Gene Delivery Vector
Cytovation	Norway - Bergen	Peptide for cancer
Hubro Therapeutics	Norway - Oslo	Cancer Immunotherapies
Lytix Biopharma	Norway - Oslo	Oncolytic Peptide
Nykode Therapeutics	Norway - Oslo	Therapeutic DNA Vaccine
PCI Biotech	Norway - Oslo	Drug Delivery
Ultimovacs	Norway - Oslo	Immunotherapy
APIM Therapeutics	Norway - Rissa	Peptide Drugs
RosVivo Therapeutics	NV - Reno	miRNA & siRNA Therapies
Phoenix Nest	NY - Brooklyn	Sanfilippo syndrome treatment
MimiVax	NY - Buffalo	Immunotherapeutics
Renbio	NY - Long Island City	Gene Therapy Directing Antibody Creation in vivo
Apertura Gene Therapy	NY - New York	AAV vectors for Gene Therapy
Cell BioEngines	NY - New York	Cell-based Immunotherapies
Chimerna Therapeutics	NY - New York	RNA Therapeutics
Lexeo Therapeutics	NY - New York	Gene Therapy
Nephrogen	NY - New York	Gene Therapy for Kidney Disease
Neurogene	NY - New York	Genetic Therapies
Oncolyze	NY - New York	Peptide Therapy
Orphion Therapeutics	NY - New York	Gene Therapy
Prevail Therapeutics	NY - New York	AAV-based gene therapies
Protagenic Therapeutics	NY - New York	Neuro-Active Peptides
Scopus BioPharma	NY - New York	Immuno-oncology Gene Therapy
Spine BioPharma	NY - New York	Amino acid peptide for spinal pain relief
Channavix Therapeutics	NY - Pittsford	Non-opioid analgesics
Alpha-1 Biologics	NY - Stony Brook	Small molecules, peptides, diagnostic, vaccine
iCell Gene Therapeutics	NY - Stony Brook	Gene Therapy for Cancer
NeoMatrix Therapeutics	NY - Stony Brook	Bioactive Peptides for Burn Healing
Sapience Therapeutics	NY - Tarrytown	Peptide-based Therapies for cancers
Abeona Therapeutics	OH - Cleveland	Therapies for Sanfilippo (SF) Syndrome Type A and Type B.
Basking Biosciences	OH - Columbus	Reversible thrombolytic therapy for acute ischemic stroke
ExonanoRNA	OH - Columbus	RNA nanoparticles, therapies
Genosera	OH - Columbus	Gene Therapy
ProtoKinetix	OH - Dalton	Anti-aging Glycopeptide
Biosortia Pharmaceuticals	OH - Dublin	Peptide Drugs
Atorvia Health Technologies	ON - Kanata	Kidney-targeted peptide that prevents regulated cell death and kidney failure
SYNG Pharmaceuticals	ON - Kingston	Diagnostic, Peptide-based Non-Hormonal Treatment for Endometriosis
Deep Genomics	ON - Toronto	AI-discovery, Oligonucleotide Therapies
YouthBio Therapeutics	ON - Toronto	Gene therapies aimed at restoring more youthful epigenetic profiles with the help of partial reprogramming
Zucara Therapeutics	ON - Toronto	Peptide analog for diabetes treatment
13therapeutics	OR - Portland	Peptide Therapeutics
Vir Biotechnology	OR - Portland	Infectious disease therapies
SwanBio Therapeutics	PA - Bala Cynwyd	Gene Therapy
Code Biotherapeutics	PA - Hatfield	DNA-based Gene Therapy Vector
Exegenesis Bio	PA - Lower Gwynedd	Gene Therapy
Avstera Therapeutics	PA - Malvern	Cell Therapy, Cancer Vaccine, mRNA
Aro Biotherapeutics	PA - Philadelphia	Protein Scaffolds, Antisense Oligonucleotide
Chimeron Bio	PA - Philadelphia	Cancer Immunotherapy
Interius BioTherapeutics	PA - Philadelphia	Novel methods for in vivo cell-specific gene delivery
Longevity Biotech	PA - Philadelphia	Synthetic Peptides
NeuExcell Therapeutics	PA - Philadelphia	Gene Therapy for Neural Regeneration & Repair
Passage Bio	PA - Philadelphia	Gene Therapy
Peroxitech	PA - Philadelphia	Peptide treatment for acute lung injury
SparingVision	PA - Philadelphia	Retinal Gene Therapy Treatments
Spark Therapeutics	PA - Philadelphia	Singe Dose Gene Therapy
Spirovant Sciences	PA - Philadelphia	Gene Therapy - Lung Diseases
Krystal Biotech	PA - Pittsburgh	Gene Therapy
NeuBase Therapeutics	PA - Pittsburgh	Oligonucleotides
Peptilogics	PA - Pittsburgh	Peptide Therapeutics
Geneos Therapeutics	PA - Plymouth Meeting	Plasmid-based Neoantigen-targeted Cancer Therapies
Inovio Pharmaceuticals	PA - Plymouth Meeting	DNA Vaccines
Prolifagen Therapeutics	PA - Villanova	microRNA
Arbutus Biopharma	PA - Warminster	RNAi
Windtree Therapeutics	PA - Warrington	Peptides
NanoCell Therapeutics	PA - Wayne	Gene Therapy
XyloCor Therapeutics	PA - Wayne	Gene Therapy

Verrica Pharmaceuticals	PA - West Chester	Topical therapies
ExploRNA Therapeutics	Poland - Warsaw	mRNA Therapies
Eli Lilly	PR - Carolina	Recombinant Insulin
Eli Lilly	PR - Mayaguez	Small Molecules, Biologics
ExCellThera	QC - Montréal	Growing blood stem cells for therapeutic use
Inspire Biotherapeutics	QC - Montréal	AAV Gene Therapy Platform
Replicor	QC - Montréal	DNA, polymer, small molecule combination Hepatitis B treatment
Feldan Therapeutics	QC - Quebec City	Peptide-based Intracellular Delivery Technology
Bolden Therapeutics	RI - Providence	Antisense Oligonucleotides for Neuro Indications
Sinfonia Biotherapeutics	RI - Warren	Nervous System Therapies
Xequel Bio	SC - Mount Pleasant	Peptide for Injury Healing
Biosyngen	Singapore - Singapore	Cell & Gene Therapy, Antibodies
Carmine Therapeutics	Singapore - Singapore	Medicines based on red blood cell extracellular vesicles (RBCEVs), for the delivery of novel therapeutic modalities that include nucleic acids
Juniper Biologics	Singapore - Singapore	Small Molecules, Cell Therapy, Gene Therapy
Lerna Biopharma	Singapore - Singapore	Nucleic Acid Therapeutics
Nuevocor	Singapore - Singapore	AAV-based gene therapy
RVAC Medicines	Singapore - Singapore	mRNA, vaccines, and lipid nanoparticle technologies
Eli Lilly	Spain - Alcobendas	Small Molecules, Biologics
Esteve	Spain - Barcelona	Small Molecules, Gene Therapy
Peptomyc	Spain - Barcelona	Cell Penetrating Peptides
SpliceBio	Spain - Barcelona	Intein Technologies - Gene Therapy
Tyris Therapeutics	Spain - Barcelona	Gene Therapies
aptaTargets	Spain - Madrid	Aptamer Drugs
Sylentis	Spain - Madrid	RNAi Treatments
ARTHEx Biotech	Spain - Paterna	Antisense RNA Compounds
Highlight Therapeutics	Spain - Valencia	Immuno-oncology, synthetic dsRNA complex
Elicera Therapeutics	Sweden - Gothenburg	Cell & Gene Therapy Immuno-Oncology
Sinfonia Biotherapeutics	Sweden - Huddinge	Nervous System Therapies
CombiGene	Sweden - Lidingö	Gene Therapy
Coegin Pharma	Sweden - Lund	Inflammatory Targeting, Small Molecules & Peptides
WntResearch	Sweden - Lund	Peptide Therapies
Xinnate	Sweden - Lund	Peptide-based Drugs
Aptahem	Sweden - Malmö	Aptamer-based Drugs
Cyxone	Sweden - Malmö	Immunomodulating cyclotides for the treatment of autoimmune diseases
InDex Pharmaceuticals	Sweden - Solna	DNA-Based Drugs, Diagnostics
Altamira Therapeutics	Switzerland - Basel	RNA Therapies
Novartis	Switzerland - Basel	Small Molecules, Vaccines, Biologics
Versameb	Switzerland - Basel	RNA Therapeutics
Vir Biotechnology	Switzerland - Bellinzona	Infectious disease therapies
Relief Therapeutics	Switzerland - Geneva	Peptide & Protein Therapies
Avrion Therapeutics	Switzerland - Lausanne	Gene Therapies for Neurodegenerative Diseases
HAYA Therapeutics	Switzerland - Lausanne	RNA-based, RNA-targeting Fibrotic Therapies
Antion Biosciences	Switzerland - Plan-les-Ouates	Gene & Cell Therapy
Anjarium Biosciences	Switzerland - Schlieren	Non-viral Gene Therapy
DiNAQOR	Switzerland - Schlieren	Gene Therapy for Heart Conditions
Redbiotec	Switzerland - Schlieren	Various Biologic Modalities
Vaxeal	Switzerland - Vevey	Cancer Immunotherapies, Long Synthetic Peptides
Alnylam Pharmaceuticals	Switzerland - Zug	RNAi
Apellis Pharmaceuticals	Switzerland - Zug	Cyclic peptide analog
BRIM Biotechnology	Taiwan - Taipei	Peptides, Recombinant Proteins
Amytrx Therapeutics	TN - Nashville	Peptide Therapies
Genprex	TX - Austin	Immunogene Therapy
Bio-Path Holdings	TX - Bellaire	Antisense DNA therapeutics
Gradalis	TX - Carrollton	Cancer immunotherapy, RNAi
Nanoscope Therapeutics	TX - Dallas	AAV-delivered opsin
ReCode Therapeutics	TX - Dallas	Nucleic Acid Therapeutic Delivery
Taysha Gene Therapies	TX - Dallas	Adeno-associated virus gene therapies
Vaxxinity	TX - Dallas	Endobody Vaccines, Synthetic Peptides Vaccine
Xequel Bio	TX - Fort Worth	Peptide for Injury Healing
Chrysalis BioTherapeutics	TX - Galveston	Peptide to stimulate tissue regeneration
Neurogene	TX - Houston	Genetic Therapies
Twister Biotech	TX - Houston	Gene Therapies
Eli Lilly	UK - Basingstoke	Small Molecules, Biologics
Bicycle Therapeutics	UK - Cambridge	Peptides
Broken String Biosciences	UK - Cambridge	Gene Edited Cell & Gene Therapy
Eleven Therapeutics	UK - Cambridge	Small interfering RNAs with ultra-long durability by utilizing high throughput combinatorial chemistry and AI
Harness Therapeutics	UK - Cambridge	IncRNA
VaxEquity	UK - Cambridge	Self-amplifying RNA
Senisca	UK - Exeter	Antisense Oligonucleotides for Senescence
Causeway Therapeutics	UK - Glasgow	microRNA
Sisaf	UK - Guildford	RNA Therapies for Rare Diseases
ILC Therapeutics	UK - Lanarkshire	Synthetic Peptides
AviadoBio	UK - London	Gene Therapies for neurodegenerative
Axovia Therapeutics	UK - London	AAV Gene Therapy
Beacon Therapeutics	UK - London	Gene Therapy
Complement Therapeutics	UK - London	Complement Medicines - AAV Gene Therapy, Biologics
Cytovation	UK - London	Peptide for cancer
DiNAQOR	UK - London	Gene Therapy for Heart Conditions
Genflow Biosciences	UK - London	Anti-aging Gene Therapy
MeiraGTx	UK - London	Gene Therapy
MiNA Therapeutics	UK - London	Small activating RNA
Orchard Therapeutics	UK - London	Ex-vivo autologous hematopoietic stem cell gene therapy
Poolbeg Pharma	UK - London	Developing drugs to sell off
Purespring Therapeutics	UK - London	Gene Therapy for Kidney Disease
Silence Therapeutics	UK - London	RNAi
Viramatix Biotechnology	UK - London	Peptide based anti-viral therapeutics
Alnylam Pharmaceuticals	UK - Maidenhead	RNAi
Ikarovec	UK - Norwich	Ocular Gene Therapy
AlveoGene	UK - Oxford	Inhaled Gene Therapy
Ochre Bio	UK - Oxford	siRNA liver drugs
Oxular	UK - Oxford	Gene Therapy
Nanogenics	UK - Selkirk	Gene Therapy
Freeline Therapeutics	UK - Stevenage	Protein replacement via Gene Therapy
Plasticell	UK - Stevenage	Cell, Gene Therapy, Blood Products
PEEL Therapeutics	UT - Salt Lake City	Nature-inspired Cancer Therapies
Serpin Pharma	VA - Manassas	Peptide drugs
ReAlta Life Sciences	VA - Norfolk	Complement and Inflammation Inhibition drug products
BioViva Science	WA - Bainbridge Island	Gene Therapy Delivery
Allysta Pharmaceuticals	WA - Bellevue	Therapeutic Peptides
PharmaIN	WA - Bothell	Subcutaneous peptides, Out licensing
AltPep	WA - Seattle	Alternating Peptides for Neurological Diseases
Kinea Bio	WA - Seattle	Gene Therapy for Muscle Disease
Myosana Therapeutics	WA - Seattle	Non-viral Gene Therapy
NapaJen Pharma	WA - Seattle	Oligonucleotide Drugs
OncoSenX	WA - Seattle	Proteo-Lipid Vehicle (PLV) and plasmid DNA for cancer
Sana Biotechnology	WA - Seattle	Cell & Gene Therapies
Shape Therapeutics	WA - Seattle	RNA-editing gene therapy
Tune Therapeutics	WA - Seattle	Epigenetic modification to modulate gene expression
Arrowhead Pharmaceuticals	WI - Madison	RNAi
Endsulin	WI - Madison	Genetic therapy for type 1 diabetes
Nimble Therapeutics	WI - Madison	Photoprotected Amino Acids