Arrowhead Pharmaceuticals, Inc. competitors & alternatives

Biotechnology

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

71% matchPublic

Biotechnology

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.

Biotechnology

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.

Biotechnology

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.

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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.

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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.

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Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for significant unmet medical needs in both viral and liver diseases. Key candidates in its pipeline include ALG-010133, an s-antigen transport-inhibiting oligonucleotide polymer, which is currently undergoing Phase Ib clinical trials for chronic hepatitis B (CHB). Another asset for CHB, ALG-000184, a capsid assembly modulator, is progressing through Phase I clinical trials. Additionally, Aligos is advancing ALG-020572, an antisense oligonucleotide designed to stop HBsAg translation and secretion. Its portfolio also features siRNA drug candidates, including ALG-125755, ALG-125097, and ALG-125819, which have demonstrated potent capabilities in inhibiting HBsAg release from HBV-infected cells. For non-alcoholic steatohepatitis (NASH), the company is developing ALG-055009, a small molecule THR-ß agonist, now in Phase 1a/1b studies. Aligos has also forged several strategic alliances. These include licensing and collaboration with Luxna Biotech Co., Ltd. for HBV genome-targeting oligonucleotides, a partnership with Emory University for HBV capsid assembly modulator technology, an agreement with Katholieke Universiteit Leuven for the development of coronavirus protease inhibitors, and a deal with Merck to research and develop NASH-focused oligonucleotides. Established in 2018, Aligos Therapeutics is headquartered in South San Francisco, California.

Biotechnology

aTyr Pharma, Inc., a biopharmaceutical firm established in San Diego, California, in 2005, is dedicated to the discovery and advancement of therapeutic solutions. Operating within the United States, the company's research focuses on pioneering novel immunological pathways to address various medical conditions. Its primary investigational drug, efzofitimod, functions as a selective modulator of NRP2. This compound is currently undergoing a Phase III clinical trial for pulmonary sarcoidosis. Additionally, efzofitimod is being evaluated in a Phase 1b/2a clinical study for the treatment of other interstitial lung diseases (ILDs), including conditions such as chronic hypersensitivity pneumonitis and ILDs linked to connective tissue diseases. Beyond its lead candidate, aTyr Pharma's pipeline features ATYR0101, a fusion protein derived from a domain of aspartyl-tRNA synthetase, which is in preclinical stages of development for combating fibrosis. The company is also progressing with ATYR0750, a domain sourced from alanyl-tRNA synthetase, intended for the management of liver disorders. Furthermore, aTyr Pharma has entered into a strategic collaboration and licensing agreement with Kyorin Pharmaceutical Co., Ltd. This partnership is focused on the development and commercialization of efzofitimod for interstitial lung diseases specifically within the Japanese market.

Biotechnology

Aro Biotherapeutics is a U.S. biotechnology company developing targeted therapeutics using its Centyrin protein platform, including delivery of genetic medicines to specific tissues and cells. It serves patients with serious diseases and partners or works with biopharmaceutical organizations to advance targeted drug-development programs.

Wave Life Sciences Ltd.WVE · BiotechSingapore

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Wave Life Sciences Ltd., a clinical-stage biotechnology company, designs, develops, and commercializes ribonucleic acid (RNA) medicines through PRISM, a discovery and drug development platform. The company’s medicines platform, PRISM combines multiple modalities, chemistry innovation, and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. It is developing WVE-006, a GalNAc-conjugated RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; WVE-007, a GalNAc-conjugated small interfering RNA designed to silence INHBE mRNA targeting obesity; WVE-008, a GalNAc-conjugated RNA editing oligonucleotide for the treatment of liver disease; WVE-N531, an exon splicing oligonucleotide for the treatment of Duchenne muscular dystrophy; and WVE-003, an allele-selective oligonucleotide for the treatment of Huntington’s disease (HD). The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was founded in 2012 and is based in Singapore.

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Atea Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers, develops, and commercializes oral antiviral therapeutics for patients with serious viral infections. Its lead product candidate is the regimen of bemnifosbuvir, a nucleotide NS5B inhibitor, and ruzasvir, an NS5A inhibitor, which is in Phase 3 clinical trial for the treatment of hepatitis C virus (HCV). The company also developing AT-587, that is in Phase 1 clinical trial for the treatment of chronic HEV infection. In addition, it offers a proprietary platform of nucleosides and nucleotides for virology, medicinal chemistry, and antiviral drug development. The company has a license agreement with MSD International GmbH for the development, manufacture, and commercialization of ruzasvir. Atea Pharmaceuticals, Inc. was incorporated in 2012 and is headquartered in Boston, Massachusetts.

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Annexon, Inc. is a biopharmaceutical company currently engaged in clinical trials, dedicated to the discovery and advancement of therapies for a variety of autoimmune, neurodegenerative, and ophthalmic conditions. Central to its approach is the C1q molecule, which initiates the classical complement pathway. The company targets this pathway to address specific disease mechanisms, such as those seen in antibody-mediated autoimmune disorders and complement-driven neurodegeneration. The company's developmental pipeline features several promising candidates. ANX005, a monoclonal antibody, is in advanced clinical testing, undergoing Phase II/III trials for Guillain-Barré syndrome, as well as Phase II trials for warm autoimmune hemolytic anemia, Huntington's disease, and amyotrophic lateral sclerosis. Another candidate, ANX009, is currently in Phase Ib studies for lupus nephritis. Additionally, ANX007 is progressing through Phase II clinical trials for the treatment of geographic atrophy, an eye disease. Further investigational assets include ANX105, a monoclonal antibody being developed for neurodegenerative indications, and ANX1502, an oral small molecule under investigation for specific autoimmune conditions. Annexon, Inc. was established in 2011 and operates from its headquarters in Brisbane, California.