List of RNAi Companies with Phase 3 Active Clinical Trial - 5
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Alnylam Pharmaceuticals Cambridge, Massachusetts, United States of America | Alnylam is the world's leading RNAi therapeutics company and the first and only company to bring RNAi-based medicines to market. We are developing RNAi (RNA interference) as an innovative, entirely new class of medicines to treat rare genetic, cardio metabolic, acute hepatic infectious and central nervous system (CNS) and ocular diseases. Alnylam was founded in 2002 based on a Nobel Prize-winning breakthrough in biology – the discovery of RNAi, and a bold vision that this discovery could be used to silence disease-causing genes upstream of today’s medicines. We are relentless in our pursuit of new treatments because we believe that RNAi therapeutics can be used to treat many diseases for which treatment options do not exist or are inadequate. Alnylam is turning scientific possibility into reality - in 2018, the FDA and EMA approved of our first product, ONPATTRO (patisiran) which is also the first-ever approved RNAi therapeutic. We now have three additional medicines on the market: GIVLAARI (givosiran), OXLUMO (lumasiran) and LEQVIO (inclisiran) in partnership with Novartis). Our robust pipeline of investigational medicines includes multiple programs in late-stage and early-stage clinical development. We are a global and diverse company of more than 1,700 people. We pride ourself on fostering a fun, inclusive and dynamic work environment where employees can recognize their fullest potential. That's why we've been named a Science Magazine Top Employer 3 years in a row ('19-'21), a Boston Globe Top Place to Work 7x in a row ('15-'21) and one of Fast Company's Best Workplaces for Innovators for 2021. |
Arrowhead Pharmaceuticals Pasadena, California, United States of America | Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. |
Dermata Therapeutics, Inc. Lexington, Massachusetts, United States of America | Dermata Therapeutics, Inc. (Nasdaq: DRMA) is a clinical-stage biotechnology company focusing on the treatment of medical and aesthetic skin conditions. The Company's lead product candidate, DMT310, is the first product candidate being developed from its Spongilla technology platform. DMT310 is a once-weekly topical product candidate derived from a naturally sourced freshwater sponge with multiple unique mechanisms of action. DMT310 is currently under clinical development for the treatment of acne, psoriasis, and rosacea. Our second product candidate, DMT410, uses our Spongilla technology as a new method for topical intradermal delivery of botulinum toxin for the treatment of multiple aesthetic skin conditions. |
Leuko 8 St Marys St, Boston, Massachusetts 02215, US | Leuko is developing the first non-invasive white blood cell device. White blood cell assessment is a first-line indicator for various medically-relevant situations, ranging from chemotherapy management to the detection of life threatening infections worldwide. This test is currently invasive and not readily accessible - it requires patient travel, blood draws and laboratory infrastructure. Based on MIT research, Leuko is re-imagining the way to perform these tests without extracting blood and in a portable device. |
Oneness Biotech Taipei, Taiwan | Oneness Biotech is dedicated to fulfilling unmet medical needs by developing new drugs focused on chronic disorders in dermatology and immunology |