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[{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy\u2122 for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Reviews Recent Portfolio Progress and Reports Third Quarter 2020 Financial Results","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces FDA Clearance of IND Application for OTL-200 for Metachromatic Leukodystrophy (MLD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Seeks UK MHRA Marketing Approval for Chronocort to Treat Patients with Rare Condition Congenital Adrenal Hyperplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Group plc - 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            OTL-203 is an investigational hematopoietic stem cell gene therapy which uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells. It is being evaluated in Phase 3 clinical trials for MPS-I Hurler Syndrome.

            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: OTL-203

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: San Raffaele-Telethon Institute for Gene Therapy

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 05, 2024

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            The Company intends to use the net proceeds to accelerate commercialization of Libmeldy (atidarsagene autotemcel), a gene therapy treatment for metachromatic leukodystrophy, and advance the company’s HSC gene therapy R&D portfolio and approval of OTL-200 in the U.S. for MLD.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Libmeldy

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Guggenheim Securities, LLC

            Deal Size: $188.0 million Upfront Cash: Undisclosed

            Deal Type: Private Placement June 26, 2023

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            ION-682884 (eplontersen) is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of TTR protein. It is being developed as a monthly self-administered subcutaneous injection to treat all types of ATTR.

            Lead Product(s): Eplontersen

            Therapeutic Area: Genetic Disease Product Name: ION-682884

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 27, 2023

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            The Company intends to use the net proceeds to accelerate commercialization of Libmeldy and advance the company’s HSC gene therapy R&D portfolio and approval of OTL-200 in the U.S. for metachromatic leukodystrophy (MLD), advancing its clinical and pre-clinical stage programs.

            Lead Product(s): OTL-200

            Therapeutic Area: Genetic Disease Product Name: OTL-200

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: RA Capital Management

            Deal Size: $188.0 million Upfront Cash: Undisclosed

            Deal Type: Private Placement March 06, 2023

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            ALXN1840 (WTX101) is a potential new once-daily, oral medicine in development for the treatment of Wilson disease. This investigational, novel molecule is designed to selectively and tightly bind to and remove copper from the body’s tissues and blood.

            Lead Product(s): Tiomolibdate Choline

            Therapeutic Area: Genetic Disease Product Name: ALXN1840

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Alexion Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 27, 2022

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            Results from positive FoCus trial in Wilson disease showed that WTX101 (ALXN1840), met its primary endpoint demonstrating three-times greater copper mobilisation from tissues compared to SoC including in patients who had been treated previously for an average of 10 years.

            Lead Product(s): Tiomolibdate Choline

            Therapeutic Area: Genetic Disease Product Name: WTX101

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 23, 2022

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            Eplontersen (IONIS-TTR-LRX) is a LICA investigational medicine designed to reduce the production of transthyretin, or TTR protein, to treat all types of ATTR, a systemic, progressive and fatal disease.

            Lead Product(s): Eplontersen

            Therapeutic Area: Genetic Disease Product Name: IONIS-TTR-LRX

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 21, 2022

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            Details:

            ALXN1840, a potential new once-daily, oral medicine, demonstrated approximately three times greater copper mobilisation than SoC. ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments.

            Lead Product(s): Tiomolibdate Choline

            Therapeutic Area: Genetic Disease Product Name: WTX101

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 26, 2021

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            Details:

            The Phase 3 study results published by the JCEM found that although the standard-deviation-score-focused primary endpoint of the study was missed, Chronocort® improved morning and early afternoon biochemical control for adults with CAH over standard glucocorticoid therapy.

            Lead Product(s): Hydrocortisone

            Therapeutic Area: Genetic Disease Product Name: Chronocort

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 02, 2021

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            Details:

            The submission for the MHRA is based on the data from the Company’s Phase 3 study, an open-label safety extension study of Chronocort® and written formal Scientific Advice received in April 2019 confirming the clinical and regulatory pathway for Chronocort® for CAH treatment.

            Lead Product(s): Hydrocortisone

            Therapeutic Area: Genetic Disease Product Name: Chronocort

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 12, 2021

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