Arrowhead Pharmaceuticals, Inc.

Arcturus Therapeutics Holdings Inc. is a biotechnology company focused on RNA-based medicines, developing a pipeline of vaccines for infectious diseases alongside treatments for rare liver and respiratory conditions across the United States. Its notable development programs encompass LUNAR-OTC, designed for ornithine transcarbamylase (OTC) deficiency, and LUNAR-CF, targeting cystic fibrosis lung disease caused by specific CFTR gene mutations. The company also advances vaccine initiatives such as LUNAR-COV19 and LUNAR-FLU. Arcturus has cultivated a range of strategic collaborations, including partnerships with Vinbiocare Biotechnology Joint Stock Company for manufacturing COVID-19 vaccines; Janssen Pharmaceuticals, Inc., to create nucleic acid-based therapies for hepatitis B virus; Ultragenyx Pharmaceutical, Inc., for developing mRNA therapeutics aimed at rare disease targets; CureVac AG, to progress mRNA therapeutic and vaccine candidates across various indications; the Singapore Economic Development Board and Duke-NUS Medical School, specifically for the LUNAR-COV19 vaccine; and Millennium Pharmaceuticals, Inc., focused on discovering siRNA medicines for non-alcoholic steatohepatitis (NASH). Founded in 2013, Arcturus Therapeutics maintains its corporate headquarters in San Diego, California.

ADARx Pharmaceuticals, Inc.ADRX · BiotechnologyUnited States

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ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company located in San Diego, California. The company is committed to developing cutting-edge RNA-targeted therapeutics to treat various genetic and severe diseases. ADARx focuses on translating scientific research into life-saving medicines through its proprietary drug discovery pipeline.

United Therapeutics Corporation, a biotechnology firm, is dedicated to discovering, developing, and bringing to market medical solutions for individuals suffering from chronic and severe, often life-threatening, illnesses. Its operations span both the United States and international markets. The company's current commercial portfolio features several key therapeutics: Remodulin, prescribed for pulmonary arterial hypertension (PAH) to lessen symptoms experienced during physical activity. Tyvaso, an inhaled form of the prostacyclin analogue treprostinil, designed to enhance exercise capacity in patients with PAH and pulmonary hypertension linked to interstitial lung disease (PH-ILD). Orenitram, a treprostinil tablet formulation, also aims to boost the exercise capabilities of PAH patients. Unituxin, a monoclonal antibody utilized in the treatment of high-risk neuroblastoma. Adcirca, an oral PDE-5 inhibitor, which helps improve exercise performance in those with PAH. United Therapeutics also maintains a robust development pipeline, including: Tyvaso DPI, a dry powder inhaler version of Tyvaso. The Remunity Pump, a compact, lightweight, and durable infusion system for treprostinil, accompanied by a separate controller. RemoPro and Ralinepag, both investigational treatments for PAH. Aurora-GT, a gene therapy product focused on regenerating blood vessels within the lungs. Clinical trials such as Tyvaso PERFECT and TETON, which are evaluating Tyvaso's efficacy in patients with World Health Organization (WHO) Group 3 pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD). The company has forged strategic alliances and licensing agreements with various entities, such as DEKA Research & Development Corp. for a semi-disposable subcutaneous treprostinil delivery system; MannKind Corporation for the development and licensing of treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. for the development of Ralinepag. Established in 1996, United Therapeutics Corporation is headquartered in Silver Spring, Maryland.

Arbutus Biopharma Corporation, a clinical-stage biopharmaceutical company, develops novel therapeutics for infectious disease in the United States. Its chronic Hepatitis B virus product pipeline comprises Imdusiran, conjugated GalNAc, subcutaneously-delivered RNAi therapeutic product candidate which is in phase 2a clinical trials that suppresses all HBV antigens, including HBsAg expression; and AB-101, an oral PD-L1 inhibitor, which is in phase 1a/1b clinical trial that has the potential to reawaken patients’ HBV-specific immune response by inhibiting PD-L1. The company has licensing agreement with Alnylam Pharmaceuticals, Inc. to develop and commercialize products with LNP delivery technology. The company was formerly known as Tekmira Pharmaceuticals Corporation and changed its name to Arbutus Biopharma Corporation in July 2015. Arbutus Biopharma Corporation was incorporated in 2005 is headquartered in Warminster, Pennsylvania.

Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.

Matinas BioPharma Holdings, Inc. is a biopharmaceutical firm in the clinical development phase, dedicated to identifying and advancing a range of potential therapeutic products. The company's innovative Lipid Nanocrystal (LNC) platform technology underpins its product development efforts. This sophisticated LNC delivery system leverages lipid nano-crystals to transport a diverse array of therapeutic agents, including small molecules, nucleic acids, gene therapies, vaccines, proteins, and peptides. Its primary experimental drug, LYPDISO, is a proprietary, prescription-grade omega-3 free fatty acid compound engineered to address cardiovascular and metabolic disorders. Matinas BioPharma is also advancing MAT2203, an orally administered version of amphotericin B. This candidate is currently in Phase II clinical trials, where it is being evaluated for its ability to prevent invasive fungal infections in patients undergoing immunosuppressive treatment. Furthermore, MAT2501, an oral formulation of the broad-spectrum aminoglycoside antibiotic amikacin, has successfully completed Phase I clinical trials. Its aim is to combat a spectrum of multidrug-resistant bacterial infections, such as non-tuberculous mycobacterium, multidrug-resistant gram-negative, and intracellular bacterial pathogens. The company actively engages in collaborations, including a research partnership with the National Institute of Allergy and Infectious Diseases (NIAID) to further the development of Gilead's antiviral drug, remdesivir. Additionally, it has a feasibility collaboration with Genentech, Inc. focused on creating new oral drug formulations. Established in 2013, Matinas BioPharma maintains its corporate headquarters in Bedminster, New Jersey.