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AKTX vs RARE

Akari Therapeutics plc ADR (0.01 USD) and Ultragenyx Pharmaceutical Inc.

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Akari Therapeutics plc ADR (0.01 USD)

Akari Therapeutics, Plc, an oncology company, develops next-generation antibody-drug conjugates (ADC) for cancer-killing toxins. Its lead payload is PH1 to disrupt the function of spliceosomes and to trigger an immune response that leads to additional cancer cell killing. The company's lead product candidate is AKTX-101, a preclinical stage trop2-targeting ADC that combines PH1 with the trop2 antibody to treat solid tumor cancer types, including lung, breast, colon, and prostate. Its payloads include PH5, which inhibits DNA mismatch repair (MMR) and DNA damage response (DDR) to generate neoepitopes, and PH6, which inhibits DNA transcription in cancer cells and co-opted immune cells. The company also develops AKTX-102, a novel bispecific ADC that utilizes PH1 as its payload to treat various solid tumor cancers. Akari Therapeutics, Plc is headquartered in Boston, Massachusetts.

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Ultragenyx Pharmaceutical Inc.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.

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