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QURE vs VOR

uniQure N.V. and Vor Biopharma Inc. Common Stock

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uniQure N.V.

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in Phase I/II clinical study for the treatment of Huntington's disease. The company also develops AMT-260, which is in Phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in Phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

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Vor Biopharma Inc. Common Stock

Vor Biopharma Inc. operates as a clinical-stage cell and genome engineering company. The company offers the Vor Bio platform, a technology platform for the treatment of acute myeloid leukemia (AML) and other blood cancers. It develops tremtelectogene empogeditemcel (trem-cel), a genome-edited hematopoietic stem and progenitor allogeneic donor product candidate to treat acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and other blood cancers; trem-cel in combination with Mylotarg to treat patients in VBP101, which is in phase 1 / 2; and VCAR33, a CAR-T therapy designed to target CD33, which is under preclinical studies to treat AML post-transplant. The company also develops trem-cel + VCAR33 treatment system, CD33-directed CAR-T program, which is under pre-clinical studies for the treatment of AML; VADC45 for the treatment of hematologic malignancies, conditioning, and immune reset, which is under preclinical studies; and CD33-CLL1 treatment system made with CD33-CLL1 multiplex-edited HSC therapy and a CD33-CLL1 multi-specific CAR-T therapy, which is under preclinical studies for the treatment of AML. It has a license agreement with Columbia University and the National Institutes of Health for patents; and with Editas Medicine, Inc. for ex-vivo Cas9 gene-edited HSC therapies for the treatment and/or prevention of hematological malignancies. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

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