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

Share

  • 1

    The study evaluates the safety and neuroprotective effects of the VCP inhibitor ML240 in retinal explants from pigs, macaques, and humans.

  • 2

    ML240 demonstrated a comprehensive preclinical safety profile, supporting its potential as a therapeutic candidate for retinal degenerative diseases.

  • 3

    The research highlights the importance of using large-animal and human-derived models to assess drug efficacy and toxicity in retinal therapies.

  • 4

    ML240 was tested both as a free compound and encapsulated in nanoparticles to enhance drug efficacy and reduce toxicity.

  • 5

    The findings confirm that pharmacological inhibition of VCP can preserve photoreceptor structure and function across different species.

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

Related Content

Inner retinal layer thickness reflects plasma biomarkers in preclinical Alzheimer’s disease

AI Falls Short on Differential Dx

New PrIME-LLM benchmark shows strong diagnostic accuracy but persistent gaps in clinical reasoning across 21 large language models

Could Blood Flow Reveal Glaucoma Earlier?

A University of Maryland study finds that retinal blood-flow autoregulation may signal early glaucoma before structural damage or vision loss is detectable.