Evaluation of the safety and neuroprotective properties of the VCP inhibitor ML240 in large-animal and human retinal explants: a preclinical ex vivo investigation - Summary - MDSpire

Evaluation of the safety and neuroprotective properties of the VCP inhibitor ML240 in large-animal and human retinal explants: a preclinical ex vivo investigation

  • By

  • Ana-Cristina Almansa-García

  • Angela Armento

  • Bowen Cao

  • Anne-Sophie Petremann-Dumé

  • Stefano Salmaso

  • Paolo Caliceti

  • Cristine Henes

  • Sylvie Bolz

  • Ellen Kilger

  • Daniela Süsskind

  • Marius Ueffing

  • Blanca Arango-Gonzalez

  • January 10, 2026

  • 0 min

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Objective:

To evaluate the safety and neuroprotective potential of the VCP inhibitor ML240 using organotypic retinal explants from pigs, macaques, and humans, highlighting its relevance for retinal degenerative diseases.

Key Findings:
  • ML240 demonstrated a comprehensive preclinical safety profile across species, indicating its potential for broader application.
  • The treatment preserved photoreceptor structure and function in retinal explants, suggesting a protective mechanism.
  • Microglial reactivity was effectively managed with ML240 treatment, which may have implications for inflammatory responses in retinal diseases.
Interpretation:

The findings support the potential of ML240 as a mutation-independent therapeutic candidate for retinal degenerative diseases, showing safety and efficacy in a relevant ex vivo model, which could translate into clinical settings.

Limitations:
  • The study is limited to ex vivo models, which may not fully replicate in vivo conditions, potentially affecting the translatability of results.
  • The long-term effects of ML240 were not assessed in this study, which is crucial for understanding its therapeutic viability.
Conclusion:

ML240 shows promise as a therapeutic agent for retinal degenerative diseases, warranting further investigation in clinical settings, particularly focusing on long-term safety and efficacy.

Original Source(s)

Evaluation of the safety and neuroprotective properties of the VCP inhibitor ML240 in large-animal and human retinal explants: a preclinical ex vivo investigation

  • by Ana-Cristina Almansa-García, Angela Armento, Bowen Cao, Anne-Sophie Petremann-Dumé, Stefano Salmaso, Paolo Caliceti, Cristine Henes, Sylvie Bolz, Ellen Kilger, Daniela Süsskind, Marius Ueffing, Blanca Arango-Gonzalez

  • January 10, 2026

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