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Aro will use the funds to advance development of its lead program, ABX1100, a novel potential treatment for patients with Pompe Disease and comprised of a CD71 receptor binding Centyrin conjugated to a small interfering RNA.
Lead Product(s): ABX1100
Therapeutic Area: Genetic Disease Product Name: ABX1100
Highest Development Status: Phase IProduct Type: Large molecule
Partner/Sponsor/Collaborator: Johnson & Johnson Innovative Medicine
Deal Size: $41.5 million Upfront Cash: Undisclosed
Deal Type: Series B Financing November 28, 2023
Details:
ABX1100, a novel potential treatment for Pompe Disease, is comprised of a CD71 receptor binding Centyrin conjugated to a siRNA which specifically interferes with expression of Gys1 mRNA, thereby reducing levels and overall activity of the Gys1 enzyme in muscle tissues.
Lead Product(s): ABX1100
Therapeutic Area: Genetic Disease Product Name: ABX1100
Highest Development Status: Phase IProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 25, 2023
Details:
ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in muscle, for the treatment of Pompe disease. ABX1100 is a novel approach that aims to reduce glycogen buildup in the muscles.
Lead Product(s): ABX1100
Therapeutic Area: Genetic Disease Product Name: ABX1100
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 September 15, 2022
Details:
ABX1100 targets the gene for glycogen synthase 1 (Gys1), an enzyme that is responsible for glycogen synthesis in muscle. Inhibition of Gys1 has been shown to reduce glycogen levels and thus represents a novel treatment approach for Pompe disease.
Lead Product(s): ABX1100
Therapeutic Area: Genetic Disease Product Name: ABX1100
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 August 10, 2022
Details:
ABX1100 is a human CD71 specific Centyrin covalently conjugated to the sense strand of a Gys1 specific siRNA via a non cleavable linker, Each component of ABX1100 has been optimized for specificity and potency vs human CD71 or Gys1 mRNA targets.
Lead Product(s): ABX1100
Therapeutic Area: Genetic Disease Product Name: ABX1100
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 May 11, 2022
Details:
Ionis has exercised an option to obtain a license to an antisense oligonucleotide (ASO)-Centyrin drug conjugate. Ionis is using Aro’s CENTYRIN technology to enhance activity of ASOs via tissue-specific delivery for efficient inhibition of disease-causing RNAs and proteins.
Lead Product(s): ASO-Centyrin drug conjugates
Therapeutic Area: Oncology Product Name: Undisclosed
Highest Development Status: UndisclosedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Ionis Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement October 06, 2020
Details:
As per the agreement, the two companies have agreed to jointly work towards advancing a defined number of ASO-Centyrin drug conjugates.
Lead Product(s): Centyrins
Therapeutic Area: Oncology Product Name: Undisclosed
Highest Development Status: UndisclosedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Ionis Pharmaceuticals
Deal Size: $1,400.0 million Upfront Cash: Undisclosed
Deal Type: Collaboration January 09, 2020
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