Articles from Aro Biotherapeutics
Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced interim data from a phase 1b study of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD). ABX1100 was well tolerated in the study of normal healthy volunteers (NHVs, n=29) and patients with LOPD (n=9). The data were presented in an oral session at the 22nd annual WORLDSymposium in San Diego, California. (The session poster is available on Aro’s website, here.)
By Aro Biotherapeutics · Via Business Wire · February 5, 2026
Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced that it will present interim clinical data from its ongoing Phase 1b clinical trial of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD), at the 22nd annual WORLDSymposium in San Diego, Calif.
By Aro Biotherapeutics · Via Business Wire · January 20, 2026
Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced that it has completed enrollment in its ongoing Phase 1b clinical trial of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD). The trial is evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics of ABX1100 in patients with LOPD who are currently receiving enzyme replacement therapy (ERT).
By Aro Biotherapeutics · Via Business Wire · September 24, 2025

Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent, targeted short-interfering RNA (siRNA) medicines, today announced that its co-founder, Susan Dillon, Ph.D., will transition from her position as president and chief executive officer (CEO) and continue as chair of the Board of Directors and advisor to the company. As part of the planned leadership transition, Aro appointed industry veteran Purnanand Sarma, Ph.D., as the CEO and a member of the Board, effective immediately.
By Aro Biotherapeutics · Via Business Wire · February 11, 2025

Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced that it has received U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug Application (IND) for ABX1100, a novel therapy currently being investigated in a phase 1 study in late-onset Pompe disease (LOPD) patients.
By Aro Biotherapeutics · Via Business Wire · February 5, 2025

Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced the dosing of the first patient in its clinical trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD).
By Aro Biotherapeutics · Via Business Wire · January 23, 2025

Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent, tissue-targeted medicines, today announced the appointment of Glenn D. Crater, MD, as chief medical officer (CMO). Dr. Crater will lead the development of Aro’s pipeline of Centyrin-based, tissue-targeted medications, including the company’s lead product candidate ABX1100, a novel potential treatment for Pompe disease.
By Aro Biotherapeutics · Via Business Wire · January 7, 2025

Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced the initiation of the Phase 1b portion of its clinical trial of ABX1100, a novel potential treatment for late-onset Pompe disease (LOPD). By targeting a known receptor called CD71, ABX1100 delivers siRNA therapy to muscle tissue, where it inhibits the production of the GYS1 enzyme, which is responsible for synthesizing glycogen. In the planned 1b study, investigators seek to enroll adults with LOPD to evaluate the safety and bioactivity of ABX1100. More information about the trial is available at ClinicalTrials.gov using the identifier NCT06109948.
By Aro Biotherapeutics · Via Business Wire · October 29, 2024

Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced upcoming data presentations highlighting the therapeutic potential of its proprietary CD71 Centyrin – Gys1 siRNA conjugate ABX1100, for the treatment of Pompe Disease. The presentations will take place at the WORLD Symposium 2024 in San Diego, CA, from February 4-6, 2024.
By Aro Biotherapeutics · Via Business Wire · February 6, 2024

Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced it has secured $41.5M in a Series B funding round. The round was led by Cowen Healthcare Investments, with participation from existing investors from Aro’s Series A financing round in 2020, including Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Northpond Ventures, Healthcap and BVF Partners, L.P. In conjunction with the financing, Christopher Picariello, President, JJDC, has joined Aro’s Board of Directors.
By Aro Biotherapeutics · Via Business Wire · November 28, 2023

Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced the enrollment of the first subject in its Phase 1 trial of ABX1100, Aro’s lead Centyrin-siRNA program for the treatment of Pompe Disease. ABX1100 is a first-in-class molecule representing a new approach to reducing toxic levels of glycogen that build up in muscles of patients with Pompe Disease.
By Aro Biotherapeutics · Via Business Wire · October 25, 2023

Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Drug (RPD) designation for ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in muscle, for the treatment of Pompe disease.
By Aro Biotherapeutics · Via Business Wire · September 15, 2022

Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in the muscle, for the treatment of Pompe disease.
By Aro Biotherapeutics · Via Business Wire · August 10, 2022

Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced upcoming data presentations highlighting the therapeutic potential of its proprietary Centyrin-siRNA conjugate therapies for the treatment of Pompe disease. During the TIDES USA 2022 meeting taking place in Boston from May 9 to 12, Aro Biotherapeutics Co-Founder and CSO Karyn O’Neil, PhD, will deliver an oral presentation alongside two poster presentations by Aro Biotherapeutics’ scientists.
By Aro Biotherapeutics · Via Business Wire · May 11, 2022

Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced upcoming data presentations highlighting the therapeutic potential of its proprietary Centyrin oligonucleotide conjugate therapies. The presentations will take place at the 2022 European Laboratory Research & Innovation Group (ELRIG) Research & Innovation Conference, the 3rd Annual RNAi – Based Therapeutics Summit, and TIDES USA.
By Aro Biotherapeutics · Via Business Wire · March 30, 2022

Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced the appointment of Sukumar Sakamuri, Ph.D. as Chief Technology Officer and a member of the company’s executive leadership team. Dr. Sakamuri will play a lead role in the growth and expansion of Aro’s technology platforms driving the continued discovery and development of Centyrin-oligonucleotide conjugate therapeutics that transform the treatment of life-threatening genetic and immune-mediated diseases.
By Aro Biotherapeutics · Via Business Wire · January 12, 2022

Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced the appointment of Mittie Doyle, MD, FACR as Chief Medical Officer and a member of the company’s executive leadership team.
By Aro Biotherapeutics · Via Business Wire · October 7, 2021

Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced the appointment of three new executive leaders including Scott Greenberg as Chief Operating Officer, Jeffrey Staiger as Senior Vice President of Finance and Business Development, and Michael Tortorici, PharmD, Ph.D. as Vice President of Clinical Pharmacology and Non-Clinical Development. Mr. Greenberg most recently served as Chief Business Officer of Aro, while Mr. Staiger and Dr. Tortorici are new additions to the leadership team. The Company also announced plans to move its headquarters to The Curtis in Philadelphia to accommodate ongoing growth of its operations and staff.
By Aro Biotherapeutics · Via Business Wire · August 4, 2021