SUMO2 conjugation therapy for restoring cognitive function in Alzheimer's disease

This technology is a biologic platform that enhances SUMO2 conjugation to preserve or restore synaptic function and memory in Alzheimer’s disease (AD) without altering amyloid burden.

Unmet Need: Non-amyloid therapy to reverse cognitive decline

Current therapies for Alzheimer’s disease (AD) primarily aim to reduce amyloid-β plaques and tau tangle formation or enhance their clearance; however, these approaches have shown limited success in reversing cognitive decline associated with AD. Many patients continue to experience progressive dementia in the form of synaptic dysfunction and memory loss, even as the amyloid burden is reduced. There remains a critical need for therapies that directly preserve or restore synaptic function, particularly therapies that are effective in later stages of disease.

The Technology: Synapse-restoring biologic effective in advanced Alzheimer’s disease stages

This technology is a therapeutic platform that utilizes a modified small ubiquitin modifier 2 (SUMO2) peptide to prevent and reverse synapse loss and cognitive deficits associated with Alzheimer’s disease (AD). SUMO2 conjugation plays a critical role in long-term potentiation, a synaptic mechanism fundamental to learning and memory. Through systemic administration of a recombinant biologic, SUMO2 conjugation is selectively elevated in the brain without altering amyloid production or clearance. The biologic is designed for efficient brain penetration and localizes to synaptic compartments, where it promotes stability and function by modulating protein interactions and stress responses linked to neurodegeneration.

This technology has been validated in vivo using AD mouse models.

Applications:

  • Therapeutic for Alzheimer’s disease (AD)
  • Drug screening for synaptic protection and repair
  • Research tool for studying SUMOylation in neurodegeneration
  • Adjunct therapy in combination with amyloid-targeting drugs
  • Preclinical platform for evaluating late-stage AD interventions
  • Research tool for cell type behavior in disease
  • Cell line production

Advantages:

  • Effective in both early and late-stage AD
  • Preserves and restores synaptic function without targeting amyloid levels
  • Compatible with systemic administration and crosses the blood-brain barrier
  • Demonstrates a strong safety profile in preclinical testing
  • Suitable for combination with existing amyloid-clearing therapies

Lead Inventor:

Ottavio Arancio, M.D., Ph.D.

Patent Information:

Patent Pending(US[20260132176])

Related Publications:

Tech Ventures Reference:

Quick Facts:
Tags
Adjuvant therapyAmyloid betaBlood–brain barrierDementiaGenetically modified mouseGliaHigh-throughput screeningLong-term potentiationMemoryMutationNeurodegenerative diseasePeptideProteinSUMO proteinSynapseUbiquitin
Inventors
Luana Fioriti Ph.D.Ottavio ArancioPaul Edward Fraser
Manager
Jerry Kokoshka
Departments
Mortimer B. Zuckerman Mind Brain Behavior InstitutePathology
Divisions
Columbia University Medical Center (CUMC)
Reference Number
CU23265
Release Date
2025-06-26