TABLE 2.
Pharmaceutical and biotechnology companies with active siRNA drug development programs
| Company | Notes | Indications | Clinical pipeline |
|---|---|---|---|
| Acuity Pharmaceuticals http://www.acuitypharma.com/ | Cand5, siRNA targeting VEGF | Age-related macular degeneration (AMD); diabetic retinopathy (DR) | AMD–siRNA IND filed Aug 10, 2004; Phase II started Oct 14, 2005 |
| AGY Therapeutics http://www.agyinc.com/ | RNAi in neurons and glial cells | CNS | |
| Alnylam Pharmaceuticals http://www.alnylam.com/ | Modified siRNAs, cholesterol conjugated; partnered with Novartis, Merck, and Medtronic; “InterfeRx” ip licensure program | ALN-RSV01–RSV; influenza; cystic fibrosis; Parkinson disease | RSV–siRNA IND filed Nov 1, 2005 |
| Atugen Atugen http://www.atugen.com/ | 2′-O-Me-modified siRNAs; Aventis and Sankyo collaborations | Metabolic diseases; epithelial cancers | Preclinical stage |
| Benitec Benitec http://www.benitec.com/ | Both siRNA and ddRNAi approaches; collaboration with Calando for delivery; InterfeRx partner with Alnylam | Hepatitis C (cocktail of three siRNAs); HIV | Phase I ddRNAi clinical trial for AIDS lymphoma with City of Hope, 2006 |
| Calando Pharmaceuticals http://www.calandopharma.com/ | Cyclodextrin polymer delivery system; collaboration with Benitec | Hepatitis C; cancer | Preclinical stage |
| CombiMatrix http://www.combimatrix.com/ | Parallel chip-based target site optimization | Hepatitis C; HIV | Preclinical stage |
| CytRx http://www.cytrxlabs.com/ | Small-molecule drug program more advanced than RNAi program | Amyotrophic lateral sclerosis; obesity; type II diabetes; CMV | |
| deVGen http://www.devgen.com/ | Collaboration with Genentech; target validation using C. elegans RNAi | Diabetes/obesity | |
| Galapagos http://www.galapagosgenomics.com/ | Adenoviral shRNA/ddRNAi vector approach | Arthritis; osteoporosis; Alzheimer disease; asthma | |
| Galenea http://www.galenea.com/ | Small-molecule CNS drug development with Otsuka Pharmaceuticals; separate RNAi respiratory disease program | Influenza | Preclinical stage |
| GeneCare http://www.we-care-gene.com/ | InterfeRx licensee with Alnylam for two helicase targets | Cancer | |
| Genesis R&D http://www.genesis.co.nz/ | IgE and IgE receptor targeting | Asthma; atopic dermatitis | Preclinical stage |
| Genomica http://www.genomica.es/ | Diagnostics company, recently added RNAi to its R&D program | Undeclared | |
| Genta http://www.genta.com/ | Both antisense and RNAi | Oncology | |
| IC-Vec http://www.icvec.com/ | Lipid nanoparticle delivery system | Hepatitis B and C; cancer | |
| International Therapeutics http://www.internationaltherapeutics.com/ | Collaboration with Rossi lab, City of Hope for HIV | HIV; viral diseases | |
| Intradigm http://www.intradigm.com/ | Synthetic nanoparticle delivery system; many collaborations, including Nucleonics | Cancer; SARS | Preclinical stage |
| Mirus http://www.mirusbio.com/ | Hydrodynamic and polymer-based particle delivery systems | Undeclared | |
| Nastech Pharmaceuticals http://www.nastech.com/ | Peptide based delivery systems; InterfeRx licensee with Alnylam for TNF-α target | Rheumatoid arthritis; metabolic diseases; cancer; others | Preclinical stage |
| NeoPharm http://www.neophrm.com/ | Liposomal delivery platform | Undeclared | |
| Novosom http://www.novosom.com/ | Liposomal delivery platform; collaborations with Nucleonics, Isis, and Santaris | Undeclared | |
| Nucleonics http://www.nucleonicsinc.com/ | ddRNAi, expressed RNAs using a variety of configurations; collaboration with Intradigm | Hepatitis B and C | Preclinical stage |
| Phytovation http://www.phytovation.com/ | Vaccines and other antiviral therapies, including RNAi. | Hepatitis C; influenza; HIV | Preclinical stage |
| Protiva Biotherapeutics http://www.protivabio.com/ | Lipid particle delivery system for both small molecules and siRNA; Sirna collaboration | Viral diseases; cancer; metabolic diseases | Preclinical stage |
| Quark Biotech http://www.quarkbiotech.com/ | Small-molecule, monoclonal antibody, and siRNA programs; using modified siRNAs | AMD, DR; acute renal failure | Preclinical stage |
| Santaris Pharma http://www.santaris.com/ | LNA-based technologies; programs in both antisense and RNAi | Cancer | |
| Sirna Therapeutics http://www.sirna.com/ | Experience with heavily modified RNAs from ribozyme work; collaborations with Eli Lilly and Archemix; AMD with Allergan | AMD–Sirna-027; hepatitis C; asthma; diabetes; Huntington disease; permanent hair removal | Phase I for AMD started Nov 2004, Sirna-027 |
| siRNAsense http://www.sirnasense.com/ | Commercialization of siRNA-related compounds developed in Norwegian institutions | Cancer; tissue factor; aquaporins | |
| ToleroTech http://www.tolerotech.com/ | Variety of technologies relating to organ transplant | Transplantation | |
| TransDerm http://www.pachyonychia.org/ | Founded through the PC project group | Pachyonychia congenital (PC) | |
| TransGenex Nanobiotech http://www.transgenex.com/ | Nanochitosan polymer delivery system | Pulmonary diseases; cancer |