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NTLA vs PTCT

Intellia Therapeutics, Inc and PTC Therapeutics, Inc.

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Intellia Therapeutics, Inc

Intellia Therapeutics, Inc., a clinical-stage gene editing company, focuses on the development of curative genome editing treatments. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; and NTLA-2002 that is in Phase 1/2 study for the treatment of hereditary angioedema. It also focusses on the research of proprietary programs focused on developing engineered cell therapies to treat various cancers and autoimmune diseases. The company has license and collaboration agreement with AvenCell Therapeutics, Inc. to develop allogeneic universal CAR-T cell therapies; Kyverna Therapeutics, Inc. for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of various of B cell-mediated autoimmune diseases; ONK Therapeutics, Ltd. for the development of engineered NK cell therapies to cure patients with cancer; and ReCode Therapeutics, Inc. to develop novel genomic medicines for the treatment of cystic fibrosis. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.

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PTC Therapeutics, Inc.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd., Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.

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