ATA-100 / Atamyo Therap - LARVOL DELTA

Two-year biological and clinical effects following ATA-100, an AAV-FKRP, gene transfer in LGMDR9: dose-escalation results from a phase 1/2 study (ESGCT 2026) - No abstract available

Clinical • P1/2 data • Gene Therapies

Whole-Body Oxygen-Enhanced MRI of Tumor-Bearing Mice Under Hyperbaric Oxygenation. (PubMed, Magn Reson Med) - "Whole-body pre-clinical OE-MRI on a clinical 3 T system is feasible and sensitive under hyperbaric oxygenation, capturing both tissue-level pressure effects and heterogeneous tumor oxygen responses. This non-invasive HBO-OE-MRI platform provides a valuable pre-clinical tool for assessing oxygen delivery and identifying oxygen-refractory tumor zones, supporting oxygen-guided therapeutic strategies."

Journal • Preclinical • Brain Cancer • Oncology

Challenges and solutions in the development of gene therapy for muscular dystrophies (ASGCT 2026) - "We present the key challenges encountered throughout the preclinical and clinical development of ATA-100, an AAV9-mediated gene therapy candidate for LGMD-R9, and the innovative solutions that enabled us to address and bypass these obstacles...Therefore; FKRP gene therapy for LGMDR9 demonstrates that safe and effective gene transfer is achievable at doses substantially lower than those historically used in MDs. Conclusion Altogether, these results illustrate the importance of controlling transgene expression, optimizing vector design, and leveraging disease biology and evolution."

Gene therapy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Muscular Dystrophy • MIR208A

ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9 (clinicaltrials.gov) - P1 | N=6 | Active, not recruiting | Sponsor: Atamyo Therapeutics | Recruiting ➔ Active, not recruiting | Phase classification: P1/2 ➔ P1 | N=39 ➔ 6 | Trial completion date: Oct 2030 ➔ Sep 2029 | Trial primary completion date: Oct 2025 ➔ Feb 2025

Enrollment change • Enrollment closed • Phase classification • Trial completion date • Trial primary completion date • Gene Therapies • Muscular Dystrophy

Gene therapy for LGMDR9: preliminary results of a dose‐escalation study (ESGCT 2023) - P1/2 | "Patients with neutralizing anti-AAV9 antibodies are excluded. Status of the study, including preliminary clinical data and muscle biopsy results will be presented."

Gene therapy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Muscular Dystrophy • FKRP

Advanced tetra amino (ATA-100) cobalt(II) phthalocyanine-based metallo-covalent organic polymer for sensitively detecting volatile organic compounds. (PubMed, Turk J Chem) - "ATA-100 showed the highest sensitivity for ethyl butyrate. The results have confirmed the possibility of utilizing ATA-100 COP-based surface acoustic wave (SAW) sensors for a wide variety of applications, including indoor air quality and environmental monitoring of volatile organic compounds (VOCs)."

Journal • Metastases

GNT0006 Gene Therapy Trial in Patients With LGMDR9 (clinicaltrials.gov) - P1/2 | N=39 | Recruiting | Sponsor: Atamyo Therapeutics | Not yet recruiting ➔ Recruiting | Initiation date: May 2022 ➔ Aug 2022

Enrollment open • Trial initiation date • Gene Therapies • Muscular Dystrophy