Seqens Seqens

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2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","amount":"$450.0 million","upfrontCash":"Undisclosed","newsHeadline":"Arrowhead Pharmaceuticals Closes Underwritten Offering with Gross Proceeds of $450.0 Million","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrowhead Pharmaceuticals Initiates Phase 1\/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrowhead Pharmaceuticals Initiates Expanded Access Program for Plozasiran and Announces Upcoming Presentation of Clinical Data","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrowhead Pharmaceuticals Files for Regulatory Clearance to Initiate Phase 1\/2a Study of ARO-CFB for Complement Mediated Kidney Disease","therapeuticArea":"Nephrology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Nephrology","graph2":"Phase I\/ Phase II"}]

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            ARO-APOC3 (plozasiran) is a first-in-class investigational RNAi therapeutic designed to reduce production of APOC3 which is a component of triglyceride rich lipoproteins. It is being evaluated for treatment of familial chylomicronemia syndrome.

            Lead Product(s): Plozasiran

            Therapeutic Area: Genetic Disease Product Name: ARO-APOC3

            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 25, 2024

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            ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene in the muscle. It is being evaluated for the treatment of type 1 myotonic dystrophy.

            Lead Product(s): ARO-DM1

            Therapeutic Area: Genetic Disease Product Name: ARO-DM1

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 08, 2024

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            Arrowhead intends to use the net proceeds for research and development, including ARO-AAT (fazirsiran), being developed to treat the liver disease associated with alpha-1 antitrypsin deficiency (AATD).

            Lead Product(s): Fazirsiran

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Jefferies

            Deal Size: $450.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering January 05, 2024

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            Arrowhead intends to use the net proceeds for research and development, including ARO-AAT (fazirsiran), being developed to treat the liver disease associated with alpha-1 antitrypsin deficiency (AATD).

            Lead Product(s): Fazirsiran

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Jefferies

            Deal Size: $450.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering January 03, 2024

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            ARO-CFB is designed to reduce hepatic expression of complement factor B. Currently, it is being evaluated in the Phase I/II clinical trial studies for the treatment of complement-mediated renal disease, such as immunoglobulin A nephropathy.

            Lead Product(s): ARO-CFB

            Therapeutic Area: Nephrology Product Name: ARO-CFB

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 21, 2023

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            ARO-DUX4 is an investigational RNAi therapeutic designed to reduce expression of the gene that encodes the human double homeobox 4 (DUX4) protein as a potential treatment for FSHD.

            Lead Product(s): ARO-DUX4

            Therapeutic Area: Musculoskeletal Product Name: ARO-DUX4

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 17, 2023

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            ARO-SOD1, an RNAi-based investigational medicine designed to reduce expression of superoxide dismutase 1 (SOD1) in the central nervous system (CNS) as a potential treatment for patients with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations.

            Lead Product(s): ARO-SOD1

            Therapeutic Area: Neurology Product Name: ARO-SOD1

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 27, 2023

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            ARO-APOC3 is an investigational RNAi therapeutic targeting apolipoprotein C-III being developed as a treatment for patients with severe hypertriglyceridemia (SHTG), mixed dyslipidemia and familial chylomicronemia syndrome.

            Lead Product(s): ARO-APOC3

            Therapeutic Area: Genetic Disease Product Name: ARO-APOC3

            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 May 16, 2023

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            ARO-RAGE is the company’s investigational RNA interference (RNAi) therapeutic designed to reduce production of the receptor for advanced glycation end products (RAGE) as a potential treatment for inflammatory pulmonary diseases, such as asthma.

            Lead Product(s): ARO-RAGE

            Therapeutic Area: Pulmonary/Respiratory Diseases Product Name: ARO-RAGE

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 25, 2023

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            Under the terms of the agreement, Arrowhead and Takeda are co-developing TAK-999 (fazirsiran), an investigational RNAi therapy designed to reduce the production of mutant alpha-1 antitrypsin protein (Z-AAT) as the first potential treatment for AATD-LD, a rare genetic disease.

            Lead Product(s): Fazirsiran

            Therapeutic Area: Genetic Disease Product Name: TAK-999

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: $1,040.0 million Upfront Cash: $300.0 million

            Deal Type: Collaboration April 04, 2023

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