
Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Aro Biotherapeutics Gets FDA IND Clearance for ABX1100 in Pompe Disease
Details : ABX1100 targets CD71, a specific receptor, to transport siRNA therapy into muscle tissues, effectively inhibiting the GYS1 enzyme, being investgated in late-onset Pompe disease (LOPD) patients.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
February 05, 2025
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Aro Bio Doses First Patient in Phase 1b Trial of ABX1100 in Late-Onset Pompe Disease
Details : ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a siRNA that specifically interferes with expression of GYS1 mRNA.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
January 23, 2025
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Aro Biotherapeutics Opens Phase 1b Trial for ABX1100 in Late-Onset Pompe Disease
Details : ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a siRNA that specifically interferes with expression of GYS1 mRNA.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
October 29, 2024
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Johnson & Johnson Innovative Medicine
Deal Size : $41.5 million
Deal Type : Series B Financing
Aro Biotherapeutics Secures $41.5M Series B Financing
Details : 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.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
November 28, 2023
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Johnson & Johnson Innovative Medicine
Deal Size : $41.5 million
Deal Type : Series B Financing

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Study to Assess the Safety, Tolerability, PK and PD of ABX1100
Details : ABX1100 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Glycogen Storage Disease Type II.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
October 31, 2023
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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 ...
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
October 25, 2023
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
September 15, 2022
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
August 10, 2022
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
May 11, 2022
Lead Product(s) : ABX1100
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Undisclosed
Therapeutic Area : Oncology
Study Phase : Undisclosed
Sponsor : Ionis Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Collaboration
Aro Collaboration with Ionis Advances with Option Exercise
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-causi...
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
October 06, 2020
Lead Product(s) : Undisclosed
Therapeutic Area : Oncology
Highest Development Status : Undisclosed
Sponsor : Ionis Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Collaboration
