Eloxx Pharmaceuticals, Inc.

Ardelyx, Inc. is a biopharmaceutical firm dedicated to the discovery, development, and commercialization of innovative medicines. The company primarily focuses on addressing gastrointestinal (GI) and cardiorenal conditions, serving patients both domestically in the United States and across international markets. Its flagship product candidate is tenapanor. This drug has successfully completed Phase III clinical trials for treating irritable bowel syndrome with constipation (IBS-C) and is also currently in Phase III development to manage hyperphosphatemia (high serum phosphorus) in adult patients with chronic kidney disease (CKD) who are undergoing dialysis. Ardelyx is also advancing other key pipeline assets, including RDX013, a potassium secretagogue designed to combat hyperkalemia (elevated serum potassium), a significant issue for individuals with compromised kidney and/or heart function. Furthermore, the company is in the early stages of developing RDX020, which targets metabolic acidosis, a serious electrolyte disorder frequently seen in CKD patients. To broaden the reach of tenapanor, Ardelyx has established strategic partnerships with Kyowa Kirin in Japan, Fosun Pharmaceutical Industrial Development Co. Ltd. in China, and Knight Therapeutics, Inc. in Canada, granting them rights for the drug's development and commercialization in their respective regions. Originally incorporated in 2007 as Nteryx, Inc., the company adopted its current name, Ardelyx, Inc., in June 2008. Its headquarters are located in Waltham, Massachusetts.

Sarepta Therapeutics, Inc. operates as a biopharmaceutical firm with products already available on the market. Its primary objective is the research and advancement of genetic treatment modalities, encompassing RNA-targeted therapeutics and gene therapies, specifically for the management of rare diseases. The company's commercial offerings include EXONDYS 51, an injectable medication prescribed for Duchenne muscular dystrophy (DMD) patients whose genetic mutation in the dystrophin gene is responsive to exon 51 skipping. Similarly, VYONDYS 53 is provided for individuals with DMD who can benefit from exon 53 skipping. Sarepta is also actively developing several investigational therapies: AMONDYS 45, a product candidate that utilizes phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to bypass exon 45 of the dystrophin gene. SRP-5051, a peptide-conjugated PMO engineered to bind to exon 51 of dystrophin pre-mRNA. SRP-9001, a gene therapy initiative focused on micro-dystrophin for DMD. SRP-9003, a gene therapy program targeting limb-girdle muscular dystrophies. The company has established collaborative relationships with various partners, including F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio. Sarepta was incorporated in 1980 and maintains its corporate headquarters in Cambridge, Massachusetts.

THX Pharma is a biopharmaceutical company that develops therapeutic solutions for rare neurological diseases such as Batten disease, Niemann-Pick type C, and Gaucher disease. The company utilizes antisense oligonucleotides and scientific research to create targeted drug candidates for patients with limited treatment options.

Xilio Therapeutics, Inc. is a clinical-stage biotechnology firm dedicated to crafting immunotherapies aimed at strengthening the immune systems of cancer patients. A key part of their pipeline is XTX101, a tumor-selective anti-CTLA-4 monoclonal antibody that is currently undergoing Phase 1/2 clinical trials for various solid tumors. The company also develops a range of cytokine programs, including XTX202 (a modified IL-2), XTX301 (an IL-12 product candidate), and XTX401 (an IL-15 product candidate). These cytokine therapies are ingeniously engineered with a protein domain that keeps them inactive until proteases specific to the tumor microenvironment (TME) cleave this mask, thereby initiating their therapeutic activity. Xilio Therapeutics, Inc. was founded in 2016 and is based in Waltham, Massachusetts.

Alnylam Pharmaceuticals0HD2 · PharmaceuticalsCambridge, United States

66% matchPublic

Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the exploration, advancement, and marketing of innovative therapies rooted in RNA interference (RNAi) technology. The company's research pipeline for RNAi treatments targets a diverse array of medical conditions, encompassing inherited disorders, cardiovascular and metabolic diseases, hepatic infections, and illnesses affecting the central nervous system (CNS) and eyes. Alnylam's commercialized products include ONPATTRO (patisiran), an injectable lipid complex prescribed for adults experiencing polyneuropathy due to hereditary transthyretin-mediated amyloidosis. Also marketed are GIVLAARI, used to treat acute hepatic porphyria (AHP) in adult patients, and OXLUMO (lumasiran), indicated for primary hyperoxaluria type 1 (PH1). In terms of its developmental programs, the company is progressing several candidates: Givosiran, for the treatment of AHP in adolescent patients. Patisiran, being investigated for transthyretin amyloidosis (ATTR amyloidosis) when accompanied by cardiomyopathy. Cemdisiran, aimed at complement-mediated diseases. ALN-AAT02, for liver disease associated with AAT deficiency. ALN-HBV02, targeting chronic hepatitis B virus infection. Zilebesiran, for the management of hypertension. ALN-HSD, for non-alcoholic steatohepatitis (NASH). Fitusiran, addressing hemophilia and various rare bleeding disorders. Inclisiran, developed to treat hypercholesterolemia. Lumasiran, for advanced PH1 and recurrent kidney stones. Vutrisiran, currently in Phase 3 clinical trials for ATTR amyloidosis. Alnylam Pharmaceuticals maintains strategic alliances with several key players in the industry. It collaborates with Regeneron Pharmaceuticals, Inc. on the discovery, development, and commercialization of RNAi therapeutics for ocular and CNS targets. Another significant partnership exists with Sanofi Genzyme, focusing on the broader development and commercialization of RNAi treatments. Furthermore, the company holds licensing and collaboration agreements with Novartis AG, Vir Biotechnology, Inc., Dicerna Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., and PeptiDream, Inc. The company was established in 2002 and operates from its headquarters in Cambridge, Massachusetts.

Apellis Pharmaceuticals, Inc.PharmaceuticalsUnited States

66% matchPrivate

Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company headquartered in Waltham, Massachusetts. Founded in 2008, the public company discovers, develops, and commercializes targeted therapeutic treatments for autoimmune and inflammatory diseases.