Ionis Pharmaceuticals

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

Aro Biotherapeutics is a biotechnology company based in Philadelphia, Pennsylvania, focused on developing a novel protein drug platform known as Centyrins. Established in 2017, the company aims to enhance treatment options for oncology and immunology by creating small, non-antibody protein scaffolds. These Centyrins can be engineered as multi-specific therapeutics, enabling innovative mechanisms of action. They are designed to improve efficacy and safety profiles for patients suffering from cancer and other serious diseases, while also facilitating the targeted delivery of complex drug payloads, including nucleic acids. Aro Biotherapeutics is committed to advancing its wholly-owned pipeline of Centyrins, contributing to the field of genetic medicines.

Akcea Therapeutics, Inc. is a biopharmaceutical company dedicated to developing and commercializing medicines for serious and rare diseases, particularly those related to cardiometabolic disorders caused by lipid abnormalities. The company's product portfolio includes TEGSEDI, an antisense medication aimed at reducing transthyretin protein production, and WAYLIVRA, which has completed Phase III clinical studies for familial chylomicronemia syndrome and is also being evaluated for familial partial lipodystrophy. Akcea is advancing several other drug candidates, including AKCEA-APO(a)-LRx and AKCEA-ANGPTL3-LRx, both of which have completed Phase IIb studies for various lipid disorders. Additionally, the company is developing AKCEA-APOCIII-LRx and AKCEA-TTR-LRx for cardiovascular disease and transthyretin amyloidosis, respectively. Akcea Therapeutics is a subsidiary of Ionis Pharmaceuticals and has strategic collaborations with Novartis Pharma AG and PTC Therapeutics International Limited. The company is headquartered in Boston, Massachusetts and was incorporated in 2014.

Empirico is a company focused on developing an insights platform that leverages biological insights derived from human genetics to create new medicines. Its platform utilizes biological datasets and programmable biology to facilitate target discovery and development. This enables scientists to systematically generate, examine, and prioritize high-confidence therapeutic hypotheses, ultimately advancing the field of drug development through innovative approaches grounded in human genetic research.

Atlantic Healthcare plc is a specialist pharmaceutical company dedicated to developing and commercializing therapeutics for unmet patient needs and rare diseases, particularly in the gastrointestinal (GI) sector. The company focuses on creating innovative treatments for GI diseases that significantly impact the lives of millions. Atlantic Healthcare holds exclusive worldwide rights to alicaforsen, a promising drug in development aimed at establishing a new class of treatments for Inflammatory Bowel Disease (IBD) and other GI disorders. Additionally, the company possesses worldwide rights to renzapride, a unique molecule designed to address various GI motility disorders. Through these initiatives, Atlantic Healthcare aims to provide effective solutions for patients suffering from challenging gastrointestinal conditions.