525 Gene Therapy, RNA & Peptide Therapy Companies - Worldwide
Order by: Company Name - Location
 |
USA - BPG | This is a Premium Listing - upgrading may double or triple your referrals. Try it out. |
 13therapeutics |
OR - Portland | Peptide Therapeutics |
| 4D Molecular Therapeutics |
CA - Emeryville | Gene Therapy |
| A28 Therapeutics |
CA - La Jolla | Targeted Lytic Peptides for the Treatment of Cancer |
| Aanastra |
CA - Los Angeles | RNA therapeutics |
| AAVantgarde Bio |
Italy - Milan | AAV-Gene Therapy Platform |
| AaviGen |
Germany - Heidelberg | Precision gene therapies for heart failure |
| AAVogen |
MD - Rockville | Gene therapeutics to strengthen muscle and fight disease |
| Abogen |
China - Suzhou | mRNA |
| AcadeMab Biomedical |
Taiwan - Taipei City | Antibody & mRNA Drugs |
| ACON Pharmaceuticals |
NJ - Cranbury | siRNA, LNP |
| ADARx Pharmaceuticals |
CA - San Diego | RNA Therapeutics |
| Addimmune |
MD - Rockville | HIV Gene Therapy |
| AdipoPharma |
France - Strasbourg | Therapeutic Peptide for Diabetes |
| Adolore BioTherapeutics |
FL - Delray Beach | Gene Therapy Analgesic for Pain |
| Adverum Biotechnologies |
CA - Redwood City | Adeno-Associated Virus Delivery, Gene Therapy |
| Affinia Therapeutics |
MA - Waltham | Gene Delivery |
| Aizen Therapeutics |
CA - San Diego | Mirror Peptides |
| Alcyone Therapeutics |
MA - Lowell | Gene Therapy |
| Aligos Therapeutics |
CA - South SF | Therapies for hepatologic diseases and viral infections |
| Alltrna |
MA - Cambridge | tRNA therapies |
| Allysta Pharmaceuticals |
WA - Bellevue | Therapeutic Peptides |
| Alnylam Pharmaceuticals |
MA - Cambridge | RNAi |
| Alnylam Pharmaceuticals |
Switzerland - Zug | RNAi |
| Alnylam Pharmaceuticals |
UK - Maidenhead | RNAi |
| Alpha-1 Biologics |
NY - Stony Brook | Small molecules, peptides, diagnostic, vaccine |
| Altamira Therapeutics |
Bermuda - Hamilton | RNA Therapies |
| Altamira Therapeutics |
DE - Newark | RNA Therapies |
| Altamira Therapeutics |
Switzerland - Basel | RNA Therapies |
| Altheia Science |
Italy - Milan | Cell & Gene Therapy for Autoimmune Disorders |
| AltPep |
WA - Seattle | Alternating Peptides for Neurological Diseases |
| AlveoGene |
UK - Oxford | Inhaled Gene Therapy |
| Alzamend Neuro |
GA - Atlanta | Alzheimer's Treatment |
| Amarna Therapeutics |
Netherlands - Leiden | Viral Gene Delivery Platform |
| Amaroq Therapeutics |
New Zealand - Auckland | lncRNA Therapeutics |
| Ambulero |
FL - Miami | Cell & Gene Therapies |
| 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 |
| Ampersand Biomedicines |
MA - Boston | AND-Body Therapeutics |
| Amplo Biotechnology |
CA - San Diego | Treatment for congenital myasthenic syndromes |
| Amyndas Pharmaceuticals |
Greece - Glyfada | Peptide for various disorders |
| Amytrx Therapeutics |
AL - Birmingham | Peptide Therapies |
| Amytrx Therapeutics |
TN - Nashville | Peptide Therapies |
| Animate Biosciences |
MA - Cambridge | Peptides for Scar Reduction |
| Antag Therapeutics |
Denmark - Copenhagen | Therapeutic peptides for dietary-related metabolic diseases |
| Antion Biosciences |
Switzerland - Plan-les-Ouates | Gene & Cell Therapy |
| Apellis Pharmaceuticals |
Australia - Hawthorn East | Cyclic peptide analog |
| Apellis Pharmaceuticals |
CA - San Francisco | Cyclic peptide analog |
| Apellis Pharmaceuticals |
Germany - Munich | Cyclic peptide analog |
| Apellis Pharmaceuticals |
MA - Waltham | Cyclic peptide analog |
| Apellis Pharmaceuticals |
Switzerland - Zug | Cyclic peptide analog |
| APEPTICO |
Austria - Vienna | Peptide Drugs |
| Apertura Gene Therapy |
NY - New York | AAV vectors for Gene Therapy |
| APIM Therapeutics |
Norway - Rissa | Peptide Drugs |
| Aptadir |
Italy - Milan | RNA Therapeutics |
| Aptahem |
Sweden - Malmö | Aptamer-based Drugs |
| aptaTargets |
Spain - Madrid | Aptamer Drugs |
| Arbutus Biopharma |
PA - Warminster | RNAi |
| Arcturus Therapeutics |
CA - San Diego | Lipid nanoparticle (LNP) mRNA drug delivery systems |
| Argo Biopharma |
China - Shanghai | siRNA Drugs |
| Armatus Bio |
OH - Columbus | RNAi Therapeutics |
| Aro Biotherapeutics |
PA - Philadelphia | Protein Scaffolds, Antisense Oligonucleotide |
| Arrowhead Pharmaceuticals |
CA - Pasadena | RNAi |
| Arrowhead Pharmaceuticals |
CA - San Diego | RNAi |
| Arrowhead Pharmaceuticals |
WI - Madison | RNAi |
| Arrowhead Pharmaceuticals |
WI - Verona | RNAi |
| Art Bioscience |
Israel - Rehovot | DMD Gene Therapy |
| Artery Therapeutics |
CA - San Ramon | Peptide therapies |
| ARTHEx Biotech |
Spain - Paterna | Antisense RNA Compounds |
| Ascidian Therapeutics |
MA - Boston | Mutated exon replacement |
| Aspa Therapeutics |
CA - Palo Alto | Gene Therapy |
| Astellas |
China - Shenyang | Small Molecules, ADC, Cell Therapy, Gene Therapy, Viral, Antibodies |
| Astellas |
IL - Northbrook | Small Molecules, ADC, Cell Therapy, Gene Therapy, Viral, Antibodies |
| Astellas |
Ireland - Dublin | Small Molecules, ADC, Cell Therapy, Gene Therapy, Viral, Antibodies |
| Astellas |
Ireland - Killorglin | Small Molecules, ADC, Cell Therapy, Gene Therapy, Viral, Antibodies |
| Astellas |
Japan - Takahagi | Small Molecules, ADC, Cell Therapy, Gene Therapy, Viral, Antibodies |
| Astellas |
Japan - Tokyo | Small Molecules, ADC, Cell Therapy, Gene Therapy, Viral, Antibodies |
| Astellas |
Japan - Yaizu | Small Molecules, ADC, Cell Therapy, Gene Therapy, Viral, Antibodies |
| Aston Sci. |
Korea - Seoul | Therapeutic cancer vaccines and proteostasis modulators |
| Atalanta Therapeutics |
MA - Boston | RNAi therapeutics |
| Atamyo Therapeutics |
France - Evry | Gene Therapies targeting neuromuscular diseases |
| Atorvia Health Technologies |
ON - Kanata | Kidney-targeted peptide that prevents regulated cell death and kidney failure |
| Atsena Therapeutics |
NC - Durham | Gene Therapy |
| Attralus |
FL - Naples | Amyloidosis Treatments |
| Augelux Therapeutics |
China - Wuhan | AAV-Based Optogenetic Gene Therapies for Ocular Diseases |
| Avellino Laboratories |
CA - Menlo Park | Gene Therapy, Molecular Diagnostics |
| Averna Therapeutics |
Israel - Ness Ziona | Developing genomic medicines that insert therapeutic genes into safe harbors within the DNA |
| Averna Therapeutics |
MA - Watertown | Developing genomic medicines that insert therapeutic genes into safe harbors within the DNA |
| AviadoBio |
UK - London | Gene Therapies for neurodegenerative |
| Avidity Biosciences |
CA - San Diego | Antibody-Oligonucleotide Conjugates |
| Avilex Pharma |
Denmark - Copenhagen | Peptide for ischemic stroke |
| Avista Therapeutics |
PA - Pittsburgh | Gene Therapy Delivery |
| Avrion Therapeutics |
Switzerland - Lausanne | Gene Therapies for Neurodegenerative Diseases |
| Avstera Therapeutics |
PA - Malvern | Cell Therapy, Cancer Vaccine, mRNA |
| Axoltis Pharma |
France - Lyon | Neurological Peptide Drugs |
| Axovia Therapeutics |
UK - London | AAV Gene Therapy |
| Basking Biosciences |
OH - Columbus | Reversible thrombolytic therapy for acute ischemic stroke |
| BDgene Therapeutics |
China - Shanghai | mRNA-based CRISPR editing components |
| Beacon Therapeutics |
MA - Cambridge | Gene Therapy |
| Beacon Therapeutics |
UK - London | Gene Therapy |
| Bedrock Therapeutics |
NC - Chapel Hill | AAV Research and Development Gene Therapy |
| Belief BioMed |
China - Shanghai | AAV-delivered Gene Therapies |
| Benitec Biopharma |
CA - Hayward | DdRNAi |
| Bicycle Therapeutics |
MA - Cambridge | Peptides |
| Bicycle Therapeutics |
UK - Cambridge | Peptides |
| Bio-Path Holdings |
TX - Bellaire | Antisense DNA therapeutics |
| BioGene Therapeutics |
TX - Austin | Bioresponsive lipid nanoparticle technology and innovative gene therapy |
| Biogenera |
Italy - Ozzano dell'Emilia | DNA Oligonucleotide Therapies |
| Biointaxis |
Spain - Barcelona | Gene Therapy for Friedreich Ataxia |
| Biojo Sciences |
Korea - Seoul | Gene Therapy & Others |
| Biojo Sciences |
MD - Bethesda | Gene Therapy & Others |
| Biomonadic |
CA - Berkeley | Klotho Gene Therapy |
| Bionic Sight |
NY - New York | Blindness Treatment Gene Therapy and Device |
| BioNTech |
CA - San Diego | mRNA, Vaccines, Biologics |
| BioNTech |
Germany - Berlin | mRNA, Vaccines, Biologics |
| BioNTech |
Germany - Idar-Oberstein | mRNA, Vaccines, Biologics |
| BioNTech |
Germany - Mainz | mRNA, Vaccines, Biologics |
| BioNTech |
MA - Cambridge | mRNA, Vaccines, Biologics |
| BioNTech |
MD - Gaithersburg | mRNA, Vaccines, Biologics |
| Biorchestra |
Korea - Daejeon | Novel RNA-based Therapeutics |
| Biorchestra |
MA - Cambridge | Novel RNA-based Therapeutics |
| Biosortia Pharmaceuticals |
OH - Columbus | Peptide Drugs |
| Biosyngen |
Singapore - Singapore | Cell & Gene Therapy, Antibodies |
| BioViva Science |
WA - Bainbridge Island | Gene Therapy Delivery |
| Black Swan Pharmaceuticals |
NC - Wake Forest | Antisense RNA-based therapeutics for ALS |
| BlackfinBio |
UK - Cheshire | Gene Therapy |
| Bolden Therapeutics |
RI - Providence | Antisense Oligonucleotides for Neuro Indications |
| Borealis Biosciences |
BC - Vancouver | RNA Medicine for Kidney Disease |
| BreezeBio |
CA - Brisbane | Nonviral CRISPR-delivery system |
| BrightPath Biotherapeutics |
Japan - Tokyo | Peptide Therapies |
| BRIM Biotechnology |
Taiwan - Taipei | Peptides, Recombinant Proteins |
| Brink Therapeutics |
France - Paris | Programmable recombinases |
| BRL Medicine |
China - Shanghai | Cell & Gene Therapy Platforms |
| Broken String Biosciences |
UK - Cambridge | Gene Edited Cell & Gene Therapy |
| C -Biomex |
Korea - Pohang | Radionuclides, peptide |
| Capacity Bio |
CA - Los Angeles | Mitophagy |
| Capsida Biotherapeutics |
CA - Thousand Oaks | AAV Gene Therapy |
| Carbon Biosciences |
MA - Waltham | Gene Therapy |
| Cardior Pharmaceuticals |
Germany - Hanover | Non-coding RNA based therapeutics |
| Caregen Biopharma |
Korea - Anyang | Growth Factors & Biomimetic Peptides |
| Carmine Therapeutics |
Singapore - Singapore | Medicines based on red blood cell extracellular vesicles (RBCEVs), for the delivery of novel therapeutic modalities that include nucleic acids |
| Causeway Therapeutics |
UK - Glasgow | microRNA |
| Cell BioEngines |
NY - New York | Cell-based Immunotherapies |
| Cellastra |
CA - San Francisco | Anti-scarring gene therapy/peptides |
| CellGenTech |
Japan - Chiba | Ex-Vivo Gene Therapy |
| Celosia Therapeutics |
Australia - Sydney | Neurodegenerative Therapies |
| Ceria Therapeutics |
AZ - Tucson | mRNA Delivery |
| Channavix Therapeutics |
NY - Pittsford | Non-opioid analgesics |
| Chimerna Therapeutics |
NY - New York | RNA Therapeutics |
| Chimeron Bio |
PA - Philadelphia | Cancer Immunotherapy |
| Chrysalis BioTherapeutics |
TX - Galveston | Peptide to stimulate tissue regeneration |
| Circle Pharma |
CA - South SF | Macrocyclic Therapeutics |
| CircNova |
MI - Detroit | Circular RNA |
| CirCode Biomed |
China - Shanghai | RNA Therapeutics |
| City Therapeutics |
MA - Cambridge | RNAi Therapies |
| CiVi Biopharma |
MD - Chevy Chase | Novel therapies against cardiometabolic diseases |
| CLOVERNA |
Japan - Okayama | RNA Targeting Oligonucleotides |
| Coave Therapeutics |
France - Paris | Gene Therapy in Ophthalmology |
| Code Biotherapeutics |
PA - Hatfield | DNA-based Gene Therapy Vector |
| Codone Biotechnology |
China - Shanghai | tRNA Therapies |
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| Coegin Pharma |
Sweden - Lund | Inflammatory Targeting, Small Molecules & Peptides |
| Comanche Biopharma |
MA - Concord | siRNA for Preeclampsia |
| Complement Therapeutics |
UK - London | Complement Medicines - AAV Gene Therapy, Biologics |
| Concinnity Genetics |
UK - Glasgow | RNA-control of gene therapies |
| Constant Therapeutics |
MA - Boston | Peptide Treatments |
| Corsera Health |
MA - Boston | RNAi Therapeutics for Cardiovascular |
| Courage Therapeutics |
MA - Newton | Eating Disorder Treatments |
| Creyon Bio |
CA - San Francisco | Oligonucleotide-based Medicines |
| CureGenetics |
China - Suzhou | Gene-editing - Diagnostics, Therapies |
| CuroNZ |
New Zealand - Hamilton | Neural Regeneration Peptides |
| Cytovation |
Norway - Bergen | Peptide for cancer |
| Cytovation |
UK - London | Peptide for cancer |
| Deep Genomics |
ON - Toronto | AI-discovery, Oligonucleotide Therapies |
| Diapin Therapeutics |
MI - Ann Arbor | Peptide for diabetes |
| DiNAQOR |
CA - Laguna Hills | Gene Therapy for Heart Conditions |
| DiNAQOR |
Germany - Hamburg | Gene Therapy for Heart Conditions |
| DiNAQOR |
Switzerland - Schlieren | Gene Therapy for Heart Conditions |
| Diorasis Therapeutics |
MA - Boston | Gene Therapy for Glaucoma |
| Dydo Pharma |
Japan - Osaka | Small Molecules, Gene Therapy |
| Dynavax Technologies |
CA - Emeryville | Synthetic oligonucleotides vaccines, adjuvants |
| E-Star BioTech |
UT - Salt Lake City | Peptide Therapies |
| EA Pharma |
Japan - Tokyo | Amino Acid Treatments |
| East Ocyon Bio |
India - Gurgaon | NK Cells, Gene Therapy |
| EG 427 |
France - Paris | Pinpoint Gene Therapy |
| Eldec Pharmaceuticals |
NC - Durham | Peptide Therapies |
| Eleven Therapeutics |
Israel - Tel Aviv | Small interfering RNAs with ultra-long durability by utilizing high throughput combinatorial chemistry and AI |
| Eleven Therapeutics |
UK - Cambridge | Small interfering RNAs with ultra-long durability by utilizing high throughput combinatorial chemistry and AI |
| Eli Lilly |
CA - San Diego | Biologics Discovery |
| Eli Lilly |
France - Neuilly-sur-Seine | Small Molecules, Biologics |
| Eli Lilly |
IN - Indianapolis | Small Molecules, Biologics |
| Eli Lilly |
Ireland - Kinsale | Small Molecules, Biologics |
| Eli Lilly |
Italy - Sesto Fiorentino | Small Molecules, Biologics |
| Eli Lilly |
MA - Boston | Small Molecules, Biologics |
| Eli Lilly |
Mexico - San José | Small Molecules, Biologics |
| Eli Lilly |
PR - Carolina | Recombinant Insulin |
| Eli Lilly |
PR - Mayaguez | Small Molecules, Biologics |
| Eli Lilly |
Spain - Alcobendas | Small Molecules, Biologics |
| Eli Lilly |
UK - Basingstoke | Small Molecules, Biologics |
| Elicera Therapeutics |
Sweden - Gothenburg | Cell & Gene Therapy Immuno-Oncology |
| Elpida Therapeutics |
CA - Encino | Gene Therapy for Rare Disease |
| Emmaus Life Sciences |
CA - Torrance | Oral amino acid treatment |
| Encoded Therapeutics |
CA - South SF | Gene Therapy |
| Endsulin |
WI - Madison | Genetic therapy for type 1 diabetes |
| Engage Bio |
CA - San Carlos | Tethosome Gene Therapy |
| Envoya |
MA - Boston | Gene Therapy |
| EOM Pharmaceuticals |
NJ - Montvale | Peptide-nucleic Acid Solutions |
| Epics Therapeutics |
Belgium - Gosselies | RNA Therapies |
| EpilepsyGTx |
UK - Waterbeach | Epilepsy Gene Therapy |
| Epsilon Molecular Engineering |
Japan - Saitama | Heavy chain single domain antibodies |
| Esperovax |
MI - Plymouth | Targeted Oral mRNA |
| Esteve |
Spain - Barcelona | Small Molecules, Gene Therapy |
| Ethris |
Germany - Planegg | MRNA-based therapies, vaccines |
| ExCellThera |
QC - Montréal | Growing blood stem cells for therapeutic use |
| ExcepGen |
CA - San Francisco | RNA Therapeutics Platform |
| Exegenesis Bio |
China - Hangzhou | Gene Therapy |
| Exegenesis Bio |
MA - Lexington | Gene Therapy |
| Exegenesis Bio |
PA - Horsham | Gene Therapy |
| Expression Therapeutics |
GA - Tucker | Gene Therapies |
| Feldan Therapeutics |
QC - Quebec City | Peptide-based Intracellular Delivery Technology |
| Fennec Pharmaceuticals |
NC - Durham | Small Molecules, Cyclic Peptides |
| Find Therapeutics |
QC - Saint-Laurent | GPCR-Targeting Therapeutics |
| Flashpoint Therapeutics |
IL - Chicago | Nanoscale design of therapeutic architecture to optimize anti-cancer immune response |
| Fractyl Health |
MA - Burlington | Thermal ablation therapy, Gene Therapy |
| Frontera Therapeutics |
China - Suzhou | Gene Therapy Development |
| Frontera Therapeutics |
MA - Watertown | Gene Therapy Development |
| Fuse Vectors |
Denmark - Copenhagen | Gene Therapy Vectors |
| GEMMA Biotherapeutics |
PA - Philadelphia | Gene Therapy |
| Gemvax & KAEL |
Korea - Seongnam | Peptide Immunotherapy |
| Genascence |
CA - Palo Alto | Musculoskeletal Gene Therapies |
| Geneos Therapeutics |
PA - Philadelphia | Plasmid-based Neoantigen-targeted Cancer Therapies |
| Genervon Biopharmaceuticals |
CA - Pasadena | Synthetic Peptide |
| Genespire |
Italy - Milan | Gene Therapy |
| Genetix Biotherapeutics |
MA - Somerville | Gene Therapy, Cellular Extraction & Reintroduction |
| GeneVentiv Therapeutics |
NC - Raleigh | Gene Therapy |
| Genexine |
Korea - Seoul | Biologics, DNA Plasmid Vaccine |
| Genflow Biosciences |
Belgium - Charleroi | Anti-aging Gene Therapy |
| Genflow Biosciences |
UK - London | Anti-aging Gene Therapy |
| GenKOre |
Korea - Daejeon | Gene-editing therapies |
| GenKOre |
Korea - Seoul | Gene-editing therapies |
| Genocury Biotech |
China - Shenzhen | Off-the-shelf Cell & Gene Therapy |
| Genosera |
OH - Columbus | Gene Therapy |
| Genprex |
TX - Austin | Non Viral Gene Delivery |
| GenSight Biologics |
France - Paris | Ophthalmic Gene Therapy |
| GenVivo |
CA - San Marino | Proprietary Gene Delivery Technology |
| GlioCure |
France - Angers | Peptide |
| Grace Science |
CA - Menlo Park | Therapies based on the function of NGLY1, AAV gene therapy, small molecules |
| Gradalis |
TX - Carrollton | Cancer immunotherapy, RNAi |
| Gritgen Therapeutics |
China - Suzhou | Gene Therapy |
| Guardian Therapeutics |
MA - Lexington | Aptamer Therapeutics |
| Harness Therapeutics |
UK - Cambridge | IncRNA |
| HAYA Therapeutics |
CA - San Diego | RNA-based, RNA-targeting Fibrotic Therapies |
| HAYA Therapeutics |
Switzerland - Lausanne | RNA-based, RNA-targeting Fibrotic Therapies |
| Helix Nanotechnologies |
MA - Boston | DNA Therapy |
| Helixmith |
Korea - Seoul | plasmid DNA based gene therapy |
| Henovcom Bioscience |
China - Guangzhou | Small Molecules, mRNA, 505 (b)(2) |
| Heqet Therapeutics |
Italy - Turin | Noncoding RNA as Therapeutic |
| Highlight Therapeutics |
Spain - Valencia | Immuno-oncology, synthetic dsRNA complex |
| HitGen |
China - Chengdu | DNA encoded library for drug discovery |
| Holostem Tarapie Avanzate |
Italy - Modena | Cell & Gene Therapy |
| Hubble Therapeutics |
NH - Hanover | Gene therapy for Leber Congenital Amaurosis Type 16 |
| HuidaGene Therapeutics |
China - Shanghai | Gene Therapy, Gene editing platform, delivery vector platform |
| Hunterian Medicine |
MA - Cambridge | CRISPR-based drug discovery |
| Hybridize Therapeutics |
Netherlands - Oegstgeest | RNA treatments for kidney disease |
| Ibex Biosciences |
MD - Cumberland | Biologics, Cell Therapy, Gene Therapy |
| iCell Gene Therapeutics |
NY - Stony Brook | Gene Therapy for Cancer |
| Ikarovec |
UK - Norwich | Ocular Gene Therapy |
| ILC Therapeutics |
UK - Glasgow | Synthetic Peptides |
| Immorna Biotherapeutics |
China - Shanghai | Self-Replicating RNA |
| Immuno Cure Biotech |
China - Hong Kong | Therapeutic Antibodies, DNA Vaccines |
| ImmunoVec |
CA - Los Angeles | Gene Therapy for Hematopoietic Diseases |
| Imugene |
Australia - Sydney | Peptide immunotherapy |
| Innovac Therapeutics |
MA - Cambridge | RNA Therapeutics |
| InnoVec Biotherapeutics |
China - Beijing | AAV-Based Gene Therapy |
| Inomagen Therapeutics |
IL - Chicago | Gene Therapies for Atrial Fibrillation |
| Inotrem |
France - Paris | Peptide Therapy |
| Inotrem |
France - Vandoeuvre | Peptide Therapy |
| Inovio Pharmaceuticals |
CA - San Diego | DNA Vaccines |
| Inovio Pharmaceuticals |
PA - Plymouth Meeting | DNA Vaccines |
| InterK Peptide Therapeutics |
Australia - Lane Cove West | Synthetic Peptides to Treat Cancer |
| Invectys |
France - Paris | DNA Vaccine |
| invIOs |
Austria - Vienna | Novel cancer immunotherapies, cell, siRNA, peptides |
| Ionis Pharmaceuticals |
CA - Carlsbad | RNA |
| Jaguar Gene Therapy |
IL - Lake Forest | AAV-Based Gene Therapies |
| Jaya Biosciences |
CA - South SF | CNS-directed gene therapies |
| Jaya Biosciences |
NC - Chapel Hill | CNS-directed gene therapies |
| Judo Bio |
MA - Cambridge | Oligonucleotide Therapies Delivered to Kidney |
| Junevity |
CA - San Francisco | Long-lasting siRNA Therapies |
| Juniper Biologics |
Singapore - Singapore | Small Molecules, Cell Therapy, Gene Therapy |
| Kailera Therapeutics |
CA - San Diego | Peptides, Small Molecules |
| Kailera Therapeutics |
MA - Waltham | Peptides, Small Molecules |
| Kairos Pharma |
CA - Los Angeles | Cell Therapy, Antibodies, Small Molecules |
| Kanglin Biotechnology |
China - Hangzhou | AAV Gene Therapy, Stem Cell Platform |
| Kelonia Therapeutics |
MA - Boston | in vivo CAR T Gene Therapy |
| Kerna Labs |
CA - San Francisco | AI and high-throughput biology - RNA therapeutics |
| Kinea Bio |
WA - Seattle | Gene Therapy for Muscle Disease |
| Kither Biotech |
Italy - Torino | Therapies for the treatment of rare pulmonary diseases |
| Kolon Life Science |
Korea - Seoul | Cell & Gene Therapy, APIs |
| Korro Bio |
MA - Cambridge | RNA-editing Drugs |
| Kriya Therapeutics |
CA - Redwood City | Gene Therapy |
| Kriya Therapeutics |
NC - Morrisville | Gene Therapy |
| Krystal Biotech |
PA - Pittsburgh | Gene Therapy |
| Lactocore |
Germany - Berlin | CNS Peptides |
| Lactocore |
MA - Newton | CNS Peptides |
| Ladder Bio |
PA - Blue Bell | Spiroligomers |
| Lassogen |
CA - San Diego | Lasso peptide-based therapies |
| Lateral Pharma |
Australia - Melbourne | Repurposed Peptide |
| Leal Therapeutics |
MA - Worcester | CNS Therapies |
| Lexeo Therapeutics |
NY - New York | Gene Therapy |
| Ligandal |
NY - Long Island City | Nanomedicine delivery for Genetic Medicines |
| Longevity Biotech |
PA - Philadelphia | Synthetic Peptides |
| Luxna Biotech |
Japan - Osaka | Oligonucleotide Therapeutics |
| Lytica Therapeutics |
MA - Cambridge | Antimicrobial peptide Design |
| Lytix Biopharma |
Norway - Oslo | Oncolytic Peptide |
| Mach5 Therapeutics |
GA - Atlanta | Gene therapies and advanced biologics |
| Mahzi Therapeutics |
CA - San Mateo | Gene therapy for rare genetic neurodevelopmental disorders |
| MAIA Biotechnology |
IL - Chicago | Peptide Cancer Therapies |
| MalarVx |
WA - Seattle | Malaria Vaccine |
| Mammoth Biosciences |
CA - Brisbane | CRISPR, Gene-Editing Therapies |
| Matrix Biomed |
CA - Irvine | Gene therapy for various cancers |
| ||
| Maze Therapeutics |
CA - South SF | Small Molecules, Gene Therapy, Antisense Oligonucleotides, Biologics |
| MBX Biosciences |
IN - Carmel | Hormone Analogues for Rare Endocrine Diseases |
| MDimune |
Korea - Seoul | Drug Delivery Based on Cell-Derived Vesicles |
| Medera |
MA - Boston | Cell & Gene Therapies |
| Medikine |
CA - San Diego | Small Molecules, Peptide |
| Medley Therapeutics |
TX - Houston | Genetic medicines that regenerate tissue and repair organs |
| MeiraGTx |
UK - London | Gene Therapy |
| Merlin Biotech |
PA - Doylestown | RNA Technology |
| MimiVax |
NY - Buffalo | Immunotherapeutics |
| MiNA Therapeutics |
UK - London | Small activating RNA |
| MindMaze Therapeutics |
Switzerland - Geneva | Peptide & Protein Therapies |
| miRecule |
MD - Gaithersburg | microRNA Therapeutics for Cancer |
| Mitotherapeutix |
CT - Farmington | siRNA drugs |
| Modalis Therapeutics |
Japan - Tokyo | Genome Editing Tools for Therapeutic Discovery |
| Modalis Therapeutics |
MA - Waltham | Genome Editing Tools for Therapeutic Discovery |
| Moderna Therapeutics |
Australia - Melbourne | RNA Therapies |
| Moderna Therapeutics |
MA - Cambridge | RNA Therapies |
| Moderna Therapeutics |
MA - Cambridge | RNA Therapies |
| Moderna Therapeutics |
MA - Norwood | RNA Therapies |
| Moderna Therapeutics |
UK - Harwell | RNA Therapies |
| Momotaro-Gene |
Japan - Okayama | Cancer Gene Therapy |
| Mustang Bio |
MA - Worcester | Immunotherapies |
| MyoPax |
Germany - Berlin | Cell & Gene Therapies |
| Myosana Therapeutics |
WA - Seattle | Non-viral Gene Therapy |
| Myrtelle |
MA - Wakefield | Gene Therapy |
| NanoCell Therapeutics |
PA - Berwyn | Gene Therapy |
| Nanogenics |
UK - Selkirk | Gene Therapy |
| Nanoscope Therapeutics |
TX - Dallas | AAV-delivered opsin |
| NapaJen Pharma |
Japan - Tokyo | Oligonucleotide Drugs |
| NapaJen Pharma |
WA - Seattle | Oligonucleotide Drugs |
| Navega Therapeutics |
CA - San Diego | Chronic Pain Treatment |
| NeoMatrix Therapeutics |
NY - Stony Brook | Bioactive Peptides for Burn Healing |
| Nephrogen |
NY - New York | Gene Therapy for Kidney Disease |
| NervGen Pharma |
BC - Vancouver | Peptide inhibitor of protein tyrosine phosphatase sigma |
| NeuExcell Therapeutics |
PA - Philadelphia | Gene Therapy for Neural Regeneration & Repair |
| Neumirna |
Denmark - Copenhagen | RNA Therapeutics |
| Neurexis Therapeutics |
CO - Aurora | Peptide for Cardiac Indications |
| Neurogene |
NY - New York | Genetic Therapies |
| Neurogene |
TX - Houston | Genetic Therapies |
| NeuroGT |
NC - Chapel Hill | Patient-centric Gene Therapy |
| Neurophth Therapeutics |
China - Wuhan | Ophthalmology Gene Therapy |
| NeuroQure |
CA - Dana Point | Gene Therapy for Neurological Disorders |
| Neuroscientific Biopharmaceuticals |
Australia - Cottesloe | Peptide for neurological treatments |
| Neurotez |
NJ - Jersey City | Peptide Treatment for Alzheimer's |
| NeuShen Therapeutics |
China - Shanghai | AAV Gene Therapy, Small Molecules |
| NGGT |
CA - Walnut Creek | Gene Therapies |
| Nitto BioPharma |
CA - San Diego | Drug Delivery of siRNA |
| Novartis |
MA - Cambridge | Small Molecules, Vaccines, Biologics / Diagnostics |
| Novartis |
Mexico - Mexico City | Small Molecules, Vaccines, Biologics |
| Novartis |
NJ - East Hanover | Small Molecules, Vaccines, Biologics |
| Novartis |
Switzerland - Basel | Small Molecules, Vaccines, Biologics |
| Nuevocor |
Singapore - Singapore | AAV-based gene therapy |
| Nuntius Therapeutics |
UK - London | mRNA Therapies |
| Nuravax |
CA - Irvine | DNA Vaccine |
| Nuvie Bio |
CA - San Diego | peptide therapy targeting calcitonin gene-related peptide receptor |
| Nykode Therapeutics |
Norway - Oslo | Therapeutic DNA Vaccine |
| NysnoBio |
CA - Mill Valley | Parkin protein therapeutics |
| Obsidian Therapeutics |
MA - Cambridge | Cell & Gene Therapies |
| Ochre Bio |
UK - Oxford | siRNA liver drugs |
| Oculgen |
China - Shenzhen | Gene and cell therapies, biologics, vaccines |
| Oculogenex |
CA - La Habra | Gene Therapy for Vision |
| Odimma Therapeutics |
France - Strasbourg | Personalized Cancer Immunotherapy |
| OliPass |
Korea - Gyeonggi | Peptide nucleic acid derivatives |
| OliX Pharmaceuticals |
Korea - Seongnam | RNAi Treatments |
| Oncogenuity |
FL - Bay Harbor Islands | Oligonucleotide platform for the treatment of genetically driven cancers |
| Oncolyze |
NY - New York | Peptide Therapy |
| Oncotherapy Science |
Japan - Kawasaki | Small Molecule, peptides, antibody therapies, contract analytical |
| Oncovir |
DC - Washington | dsRNA viral mimic |
| ONL Therapeutics |
MI - Ann Arbor | Small molecule peptide |
| Onodera |
Japan - Kanagawa | Gene Therapy |
| Opus Genetics |
NC - Raleigh | Ophthalmology Gene Therapy |
| Orbital Therapeutics |
MA - Cambridge | RNA technology (not RNAi) |
| Orchard Therapeutics |
MA - Boston | Ex-vivo autologous hematopoietic stem cell gene therapy |
| Orchard Therapeutics |
UK - London | Ex-vivo autologous hematopoietic stem cell gene therapy |
| Orna Therapeutics |
MA - Watertown | oRNA, Delivery |
| OrthoTrophix |
CA - Foster City | Bone, cartilage and teeth therapies |
| Oryn Therapeutics |
CA - West Hollywood | Macrocyclic peptide therapeutics |
| Oryx Translational Medicine |
Germany - Vaterstetten | Cancer Immunotherapy |
| Oxalo Therapeutics |
IL - Chicago | Peptide-based therapeutics to treat rare renal diseases and prevent recurrent kidney stones |
| Pantherna Therapeutics |
Germany - Hennigsdorf | Nanoparticle Delivery for mRNA drugs |
| ParcelBio |
CA - San Francisco | Next-generation mRNA medicines, CAR T |
| Passage Bio |
PA - Philadelphia | Gene Therapy |
| PCI Biotech |
Norway - Oslo | Drug Delivery |
| PEEL Therapeutics |
UT - Salt Lake City | Nature-inspired Cancer Therapies |
| PEP-Therapy |
France - Paris | Peptide Therapies |
| PepKon |
France - Louveciennes | Peptide Therapies for Cancer |
| Peptilogics |
PA - Pittsburgh | Peptide Therapeutics |
| Peptinov |
France - Paris | Peptide-based active immunotherapy |
| Peptomyc |
Spain - Barcelona | Cell Penetrating Peptides |
| Peptron |
Korea - Daejeon | Peptide-based Medicine Platform |
| Peroxitech |
PA - Philadelphia | Peptide treatment for acute lung injury |
| Pfizer |
CA - La Jolla | Peptides, Monoclonal Antibodies |
| Pfizer |
NC - Durham | Small Molecules, Vaccines, Biologics |
| Pfizer |
WA - Bothell | Small Molecules, Vaccines, Biologics |
| Phaim Pharma |
UK - London | Peptide Diabetes Treatment |
| PharmaIN |
WA - Bothell | Subcutaneous peptides, Out licensing |
| Phinomics |
CA - San Carlos | Circular DNA |
| Phio Pharmaceuticals |
PA - King of Prussia | RNAi |
| Phlox Therapeutics |
Netherlands - Naarden | Gene Therapy for Heart Disease |
| Phoenix Nest |
NY - Brooklyn | Sanfilippo syndrome treatment |
| Phylex BioSciences |
CA - Del Mar | Vaccines |
| Pinion Immunotherapeutics |
MD - North Bethesda | mRNA Vaccine Technology |
| PinotBio |
Korea - Suwon | Small molecules, ADC, Nucleoside-based Therapy |
| Plasticell |
UK - Stevenage | Cell, Gene Therapy, Blood Products |
| Poolbeg Pharma |
UK - London | Developing drugs to sell off |
| Precigen |
MD - Germantown | Cell & Gene Therapy |
| Prevail Therapeutics |
NY - New York | AAV-based gene therapies |
| Priavoid |
Germany - Düsseldorf | All-d-peptides |
| Primera Therapeutics |
US - Virtual | mtDNA engineering toolbox for mitochondrial diseases |
| Prolifagen Therapeutics |
PA - Doylestown | microRNA |
| ProQR Therapeutics |
MA - Cambridge | Oligonucleotide Therapy |
| ProQR Therapeutics |
Netherlands - Leiden | Oligonucleotide Therapy |
| Protagenic Therapeutics |
NY - New York | Neuro-Active Peptides |
| Protagonist Therapeutics |
Australia - Brisbane | Oral peptides |
| Protagonist Therapeutics |
CA - Newark | Oral peptides for GI indications |
| Protelight Pharmaceuticals |
China - Jiangyin | Peptide and Small Molecules Therapies |
| ProtoKinetix |
OH - Dalton | Anti-aging Glycopeptide |
| Providence Therapeutics |
AB - Calgary | mRNA Cancer Vaccines |
| PTC Therapeutics |
NJ - Warren | Small Molecules, Gene Therapy |
| PulseSight Therapeutics |
France - Paris | Ophthalmic Treatment Plasmid |
| Purespring Therapeutics |
UK - London | Gene Therapy for Kidney Disease |
| PURMX Therapeutics |
Japan - Hiroshima | Nucleic Acid Drugs |
| PYC Therapeutics |
Australia - Nedlands | Peptide drug discovery |
| Ractigen Therapeutics |
China - Nantong | Small activating RNA |
| Radar Therapeutics |
- | Smart Programmable Medicines Using Molecular RNA Sensors |
| Radella Pharmaceuticals |
UK - Aberdeen | Peptide Therapy |
| Ray Therapeutics |
CA - Berkeley | Optogenetic Gene Therapy |
| ReAlta Life Sciences |
VA - Norfolk | Complement and Inflammation Inhibition drug products |
| Recbio Technology |
China - Taizhou | mRNA Covid Vaccine |
| ReCode Therapeutics |
CA - Menlo Park | Nucleic Acid Therapeutic Delivery |
| Regel Therapeutics |
CA - Berkeley | AAV gene delivery |
| Regel Therapeutics |
MA - Cambridge | AAV gene delivery |
| RegeneRx Biopharmaceuticals |
MD - Rockville | Peptides |
| ReGenTree |
NJ - Princeton | Therapeutic Peptides |
| ReGenX Biosciences |
MD - Rockville | Gene Therapy, Biologics |
| Regenxbio |
MD - Rockville | Gene Therapy |
| Rein Therapeutics |
TX - Austin | Perpetually Active Peptides |
| Rejuve Therapeutics |
MD - Beltsville | Gene therapy drug development company researching senescent cell solutions. |
| Rejuvenate Bio |
CA - San Diego | Gene Therapy for Aging |
| Rejuvenate Biomed |
Belgium - Diepenbeek | Combination Drugs for Aging |
| Rejuvenation Technologies |
CA - Mountain View | Telomere Extension via Nucleoside-modified TERT mRNA |
| Remedium Bio |
MA - Needham | Gene Therapy |
| Renbio |
NY - Long Island City | Gene Therapy Directing Antibody Creation in vivo |
| Renova Therapeutics |
CA - Carlsbad | Gene Therapies |
| Repertoire Immune Medicines |
MA - Cambridge | Cell & Peptide-Based Therapies |
| Replay |
CA - San Diego | HSV Vector Gene Therapies |
| Replicate Bioscience |
CA - San Diego | Preventing oncology drug resistance |
| Replicor |
QC - Montréal | DNA, polymer, small molecule combination Hepatitis B treatment |
| ResoTher Pharma |
Denmark - Holte | Cardiovascular Treatment |
| ResQ Biotech |
Greece - Patra | Therapies for Protein Misfolding Diseases |
| Restore Vision |
Japan - Tokyo | Gene Therapy for Blindness due to Retinal Disorders |
| RheumaGen |
CO - Aurora | Cell & Gene Therapy |
| RhyGaze |
Switzerland - Basel | Gene Therapy Targeting Cone Cells |
| Rhythm Pharmaceuticals |
MA - Boston | Peptide Therapeutics |
| Ribocure Pharmaceuticals |
Sweden - Mölndal | RNA Therapeutics |
| Ribomic |
Japan - Tokyo | Aptamers |
| RiboX Therapeutics |
China - Shanghai | Circular RNA |
| RIGImmune |
CT - Farmington | RNA Immunotherapeutics |
| RinuaGene Biotechnology |
China - Suzhou | mRNA Therapies |
| ||
| Riptide Bioscience |
CA - Vallejo | Peptide Therapeutics |
| Risen Pharma Tech |
China - Shanghai | Small Molecules, siRNA, Combination Therapies |
| Risen Pharma Tech |
China - Suzhou | Small Molecules, siRNA, Combination Therapies |
| Rithim Biologics |
MD - Woodbine | Gene Therapy for Tachycardias |
| RNAimmune |
MD - Germantown | mRNA therapies |
| RNatives |
Finland - Kuopio | RNA-based gene regulating therapies |
| Rocket Pharmaceuticals |
NJ - Cranbury | Gene Therapies |
| Rona Therapeutics |
China - Shanghai | RNA Therapeutics Platform |
| RosVivo Therapeutics |
NV - Reno | miRNA & siRNA Therapies |
| Rznomics |
Korea - Seongnam | RNA Therapy Platform |
| Sagittarius Bio |
CA - Thousand Oaks | Gene Therapy for Cancer |
| San Rocco Therapeutics |
FL - Tampa | Gene Therapy, Orphan Diseases |
| Sana Biotechnology |
WA - Seattle | Cell & Gene Therapies |
| SanegeneBio |
MA - Woburn | siRNA Drug Discovery |
| Sapience Therapeutics |
NY - Tarrytown | Peptide-based Therapies for cancers |
| Sapu BioScience |
CA - San Diego | RNA & Small Molecule Immuno-oncology Therapies |
| Sardocor |
MA - Boston | Gene therapy |
| Sciwind Biosciences |
China - Hangzhou | Oral peptide delivery |
| Scopus BioPharma |
NY - New York | Immuno-oncology Gene Therapy |
| Secarna Pharmaceuticals |
Germany - Planegg | Antisense Oligonucleotide Therapies |
| Senisca |
UK - Exeter | Antisense Oligonucleotides for Senescence |
| Senti Biosciences |
CA - South SF | Gene Circuits; Cell & Gene Therapies |
| Sentia Medical Sciences |
CA - San Diego | Novel peptide therapeutics |
| Seragon Biosciences |
CA - Irvine | Small Molecules, Gene Therapy |
| Serif Biomedicines |
MA - Cambridge | Non-Viral Gene Therapy Delivery |
| Serodus |
Norway - Oslo | Drug Development |
| Serpin Pharma |
VA - Manassas | Peptide drugs |
| Shape Therapeutics |
WA - Seattle | RNA-editing gene therapy |
| Shenogen Pharma |
China - Beijing | Small Molecules, Biologics, Viral |
| Silence Therapeutics |
Germany - Berlin | RNAi |
| Silence Therapeutics |
NJ - Hoboken | RNAi |
| Silence Therapeutics |
UK - London | RNAi |
| Silthera |
Italy - L'Aquilo | siRNA Therapies |
| SineuGene |
China - Beijing | Gene Therapy |
| Siren Biotechnology |
CA - San Francisco | AAV Immuno-Gene Therapy |
| Sirius Therapeutics |
CA - San Diego | RNAi Therapies |
| Sirius Therapeutics |
China - Shanghai | RNAi Therapies |
| siRNAgen |
Korea - ZZ | siRNA Therapeutics |
| Sirnaomics |
China - Suzhou | RNAi Therapeutics |
| Sirnaomics |
MD - Germantown | RNAi Therapeutics |
| SiSaf |
UK - Guildford | RNA Therapies for Rare Diseases |
| Skip Therapeutics |
Israel - Ness Ziona | RNA-based therapies for rare genetic disorders and common diseases |
| Skylark Bio |
MA - Cambridge | Gene Therapy for Hearing Disorders |
| SNIPR BIOME |
Denmark - Copenhagen | CRISPR for microbiome management, genetically-modified bacteriophages |
| SOLA Biosciences |
MA - Natick | Gene Therapies for Protein Misfolding Diseases |
| Solid Biosciences |
MA - Charlestown | Gene Therapy, Devices, Biologics, Small Molecules all for DMD |
| Somagenetix |
Switzerland - Altendorf | Gene therapy for phagocytic disorders |
| Soricimed Biopharma |
NB - Moncton | Proprietary TRPV6-targeting peptide platform |
| Sovargen |
Korea - Daejeon | RNA therapeutics |
| Sovargen |
Korea - Seoul | RNA therapeutics |
| SparingVision |
France - Paris | Retinal Gene Therapy Treatments |
| SparingVision |
MA - Boston | Retinal Gene Therapy Treatments |
| Spine BioPharma |
NY - New York | Amino acid peptide for spinal pain relief |
| Spirovant Sciences |
PA - Philadelphia | Gene Therapy - Lung Diseases |
| Splash Pharmaceuticals |
CA - San Diego | Peptide |
| SpliceBio |
Spain - Barcelona | Intein Technologies - Gene Therapy |
| SpliSense |
Israel - Jerusalem | Antisense Oligonucleotides |
| Spur Therapeutics |
UK - Stevenage | Protein replacement via Gene Therapy |
| SRTD biotech |
Germany - Jülich | seRNA |
| Starna Therapeutics |
China - Suzhou | mRNA vaccine, therapies |
| StemCardia |
CA - Sacramento | Cell & gene therapies engineered to repair the heart |
| Sterna Biologicals & KG |
Germany - Marburg | Antisense Molecule - DNAzyme |
| Stoke Therapeutics |
MA - Bedford | Antisense Oligonucleotide Medicines |
| Strand Therapeutics |
MA - Boston | Gene Therapy |
| STRM.BIO |
MA - Cambridge | Extracellular vesicles (EVs) to deliver gene therapies |
| Stylus Medicine |
MA - Cambridge | Enzymatic Gene Therapy |
| Suzhou Ribo Life Science Company |
China - Kunshan | RNAi |
| Switch Therapeutics |
CA - South SF | RNAi Therapies |
| Sylentis |
Spain - Madrid | RNAi Treatments |
| SynaptixBio |
UK - Oxford | Development for treatment of severe leukodystrophies, ASO |
| SYNG Pharmaceuticals |
ON - Kingston | Diagnostic, Peptide-based Non-Hormonal Treatment for Endometriosis |
| SYTE.bio |
MA - Boston | Precision therapeutics using its proprietary Linear DNA and Circular RNA technology platforms |
| TAGCyx Biotechnologies |
Japan - Tokyo | Oligonucleotide Drug Discovery Platform |
| Takara Bio |
Japan - Shiga | Gene Therapy, Agribiotech, Biotech Lab Technology |
| Taysha Gene Therapies |
TX - Dallas | Adeno-associated virus gene therapies |
| TeamedOn |
MD - Rockville | Gene therapies for rare ocular diseases |
| Telocyte |
MI - Grand Rapids | Telomerase Therapy for Alzheimer's |
| Temple Therapeutics |
Netherlands - Geleen | Amino Acid-based Surgical Treatment |
| Tenaya Therapeutics |
CA - South SF | Cardiovascular Therapeutics |
| Tern Therapeutics |
DC - Washington | Gene Therapy for Batten Disease |
| Tetraneuron |
Spain - Madrid | Gene Therapy |
| Tevard Biosciences |
MA - Boston | tRNA-based Gene Therapies |
| Theragene Pharmaceuticals |
CA - San Diego | Immunotherapy, gene therapy and peptide delivery |
| Therorna |
China - Beijing | RNA Therapies |
| Thylacine Biosciences |
FL - Miami | engineered antiviral peptides |
| TME Therapeutics |
Japan - Tokyo | dsRNA Treatment for Cancer |
| Torque Bio |
NC - Durham | Gene Therapies |
| Trace Neuroscience |
CA - South SF | Medicines to restore UNC13A function |
| TransCode Therapeutics |
MA - Newton | RNA Delivery, Small Molecule |
| Transgene |
France - Illkirch-Graffenstaden | Viral Vector Therapeutics, Gene Therapy |
| Transgene |
MA - Boston | Viral Vector Therapeutics, Gene Therapy |
| Translation-X |
Switzerland - Basel | Autism Therapies |
| Treeway |
Netherlands - Tilburg | Small Molecules, Gene Therapy |
| TuHURA Biosciences |
FL - Tampa | Cancer Therapies |
| Tune Therapeutics |
NC - Durham | Epigenetic modification to modulate gene expression |
| Tune Therapeutics |
WA - Seattle | Epigenetic modification to modulate gene expression |
| Typewriter Therapeutics |
MA - Cambridge | Gene Therapy |
| Tyris Therapeutics |
Spain - Barcelona | Gene Therapies |
| Ultragenyx Pharmaceutical |
CA - Brisbane | Small Molecules, Biologics |
| Ultragenyx Pharmaceutical |
CA - Novato | Small Molecules, Biologics, Gene Therapy, mRNA, Antisense Oligo |
| Ultragenyx Pharmaceutical |
MA - Bedford | Small Molecules, Biologics, Gene Therapy, mRNA, Antisense Oligo |
| Ultragenyx Pharmaceutical |
MA - Somerville | Small Molecules, Biologics |
| Ultragenyx Pharmaceutical |
MA - Woburn | Small Molecules, Biologics |
| uniQure |
Netherlands - Amsterdam | Gene Therapy |
| Unnatural Products |
CA - Santa Cruz | Macrocylic Peptides plus AI |
| Valerio Therapeutics |
France - Paris | Orphan Diseases, Decoy Oligonucleotides |
| Valo Therapeutics |
Finland - Helsinki | Oncolytic Virus, Peptide |
| Vaxxinity |
TX - Dallas | Endobody Vaccines, Synthetic Peptides Vaccine |
| VectorY Therapeutics |
Netherlands - Amsterdam | Vectorized Antibodies |
| Velvet Therapeutics |
TX - Houston | Gene Therapies |
| VeonGen Therapeutics |
Germany - Planegg | AAV Gene Therapy |
| Vernagen |
GA - Tucker | mRNA Therapies |
| Verrica Pharmaceuticals |
PA - West Chester | Topical therapies |
| Versameb |
Switzerland - Basel | RNA Therapeutics |
| Verve Therapeutics |
MA - Boston | Gene-editing Treatments |
| Vico Therapeutics |
Netherlands - Leiden | Oligonucleotide based RNA modulating therapeutics |
| ViferaXS |
Germany - Tübingen | Immunopeptidome-based antigen |
| Vigeo Therapeutics |
MA - Cambridge | Biologics for Cancer |
| Vir Biotechnology |
CA - San Francisco | Infectious disease therapies |
| Vir Biotechnology |
Switzerland - Bellinzona | Infectious disease therapies |
| Vironexis Biotherapeutics |
CA - San Diego | T Cell Immunotherapy |
| Virothera |
UK - Cambridge | Gene Immunotherapeutics |
| Visirna Therapeutics |
China - Shanghai | RNAi Therapeutics: siRNA Drugs |
| Vivet Therapeutics |
France - Paris | Gene Therapy for Orphan Diseases |
| Voyager Therapeutics |
MA - Lexington | AAV capsid Gene Therapy |
| WaVe Life Sciences |
Japan - Kagoshima | Stereopure oligonucleotides |
| WaVe Life Sciences |
MA - Cambridge | Stereopure oligonucleotides |
| Westlake Genetech |
China - Hangzhou | AI-powered Gene Therapy Development |
| Xequel Bio |
Virtual - Virtual | Peptide for Injury Healing |
| Xinnate |
Sweden - Lund | Peptide-based Drugs |
| XyloCor Therapeutics |
PA - King of Prussia | Gene Therapy |
| YouthBio Therapeutics |
ON - Toronto | Gene therapies aimed at restoring more youthful epigenetic profiles with the help of partial reprogramming |
| Zata Pharmaceuticals |
MA - Worcester | RNA, DNA Therapeutics |
| Zealand Pharma |
Denmark - Soborg | Peptide Drugs |
| Ziphius Vaccines |
Belgium - Merelbeke | Self-amplifying RNA Vaccine |
| Zucara Therapeutics |
ON - Toronto | Peptide analog for diabetes treatment |