Ionis Pharmaceuticals

Metagenomi, Inc. is a genetic medicines company focused on developing innovative gene editing systems to address genetic diseases. Incorporated in 2016 and headquartered in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to discover novel genome editing technologies. By sourcing cellular machinery from previously uncharacterized organisms, the company creates a proprietary toolbox that includes programmable nucleases, base editors, and advanced integration systems like prime editing and CRISPR-associated transposases. These systems are designed to be ultra-small, efficient, and highly specific, minimizing the risk of immune responses. Metagenomi aims to revolutionize gene editing techniques and deliver curative therapeutics to patients worldwide.

Aro Biotherapeutics is a biotechnology company based in Philadelphia, Pennsylvania, focused on developing a novel protein drug platform known as Centyrins. These small, non-antibody protein scaffolds are designed to enhance treatment efficacy and safety for patients with cancer and other serious diseases. Aro Biotherapeutics has established a wholly-owned pipeline of Centyrins targeted at oncology and immunology applications. The unique structure of Centyrins allows for multi-specificity and the targeted delivery of complex drug payloads, including nucleic acids, which may aid in advancing therapeutic mechanisms and unlocking the potential of genetic medicines. Established in 2017, the company aims to innovate treatments that improve patient outcomes in challenging medical conditions.

Akcea Therapeutics, Inc. is a biopharmaceutical company dedicated to the development and commercialization of medicines for serious and rare diseases, particularly those related to lipid disorders. Headquartered in Boston, Massachusetts, Akcea focuses on innovative treatments utilizing antisense technology, originally developed by its parent company, Ionis Pharmaceuticals, Inc. The company’s product portfolio includes TEGSEDI, an antisense therapy aimed at reducing transthyretin protein production, and WAYLIVRA, which has completed Phase III trials for familial chylomicronemia syndrome and is also being studied for familial partial lipodystrophy. Additionally, Akcea is advancing several other candidates through clinical trials, including AKCEA-APO(a)-LRx and AKCEA-ANGPTL3-LRx for lipid-related conditions, and AKCEA-APOCIII-LRx, which targets cardiovascular diseases linked to high triglyceride levels. The company has strategic collaborations with Novartis Pharma AG and PTC Therapeutics International Limited to enhance its market presence and access.

Empirico is a biotechnology company focused on developing an insights platform that leverages biological data derived from human genetics to facilitate the creation of new medicines. The platform integrates extensive biological datasets and employs programmable biology to enhance target discovery and development processes. This enables researchers to systematically generate, analyze, and prioritize therapeutic hypotheses with a high degree of confidence, thereby advancing the search for innovative treatments. Through its technology, Empirico aims to transform the landscape of drug development by providing actionable insights that can lead to effective medical solutions.

Atlantic Healthcare plc is a specialist pharmaceutical company dedicated to developing and commercializing therapeutics that meet unmet patient needs, particularly focusing on rare gastrointestinal diseases. The company is engaged in creating innovative treatments for conditions such as inflammatory bowel disease and gastrointestinal motility disorders. Atlantic Healthcare holds exclusive worldwide rights to alicaforsen, which has the potential to introduce a new class of treatments for inflammatory bowel disease, as well as renzapride, a molecule designed to address various gastrointestinal motility disorders. By targeting these debilitating conditions, Atlantic Healthcare aims to improve the quality of life for individuals affected by gastrointestinal diseases.