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ARO-CFB is designed to reduce hepatic expression of complement factor B (CFB), which plays an important regulatory role in amplifying complement alternative pathway activation. It is being evaluated for the treatment of IgA Nephropathy.
Lead Product(s): ARO-CFB
Therapeutic Area: Nephrology Product Name: ARO-CFB
Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 24, 2024
Details:
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 25, 2024
Details:
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 IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 08, 2024
Details:
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Jefferies
Deal Size: $450.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering January 05, 2024
Details:
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Jefferies
Deal Size: $450.0 million Upfront Cash: Undisclosed
Deal Type: Public Offering January 03, 2024
Details:
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 IIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 21, 2023
Details:
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 EnablingProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 17, 2023
Details:
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 EnablingProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 27, 2023
Details:
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 16, 2023
Details:
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 IIProduct 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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