Therapeutic Approach

Rescue From Age-Related Loss of ELOVL2

Our strategy is to rejuvenate retinal photoreceptors and RPE cells suffering from macular degeneration by restoring natural healthy levels of ELOVL2 expression, thus “turning back the clock” to a more youthful level of ELOVL2.

ELOVL2 Gene Therapy

Supplement ELOVL2 expression by adding additional copies of the gene to photoreceptor and RPE cells.

Lead ELOVL2 Program

ELOVL2 Retinal Gene Therapy: Suprachoroidal and Subretinal Administration

Visgenx has developed a highly optimized and proprietary ELOVL2 construct that is highly expressed at low MOIs in the target cells (photoreceptors and RPE) and is well tolerated.

Studies in aged mice have demonstrated a single subretinal administration resulted in:

  • Enhanced photoreceptor and Muller cell function
  • Protection from aging induced photoreceptor loss

Studies in non-human primates have demonstrated a single subretinal administration:

  • Was well-tolerated
  • Expressed in the target areas of the retina
  • Increased the target lipid molecules

Research Programs

Synthetic AAV Non-viral gene delivery

As part of our ELOVL2 gene therapy program, Visgenx is developing unique first in class best in class fully synthetic vector constructs that overcome many of the limitations of viral vectors. Advantages of our synthetic vector constructs include:

  • The ability to deliver very large transgenes, much larger than current viral vectors
  • Redosable
  • Durable and robust transgene expression comparable to viral vectors
  • Anti-TLR9 technology
  • Eliminates rate limiting step of second strand synthesis as vectors are dsDNA
  • Unaffected by pre-existing neutralizing anti-capsid antibodies
  • Substantially less immunogenic as there is no protein component within the system
  • No Intrinsic ITR promoter (A/D sequence) activity
  • Reduced CpG signature
  • Sequence not constrained by packaging signal sequences
  • Significantly lower cost of goods, totally synthetic-no eukaryotic cells required in manufacturing

Synthetic AAV: CMV-GFP Expression Kinetics in HeLa Cells

Synthetic AAV-ABCA4 for Stargardt’s Disease Contains an 8.9 Kb Payload

Non-Viral Non-Nuclease Based Gene Editing Using Synthetic AAV as a Homologous Donor for Gene Correction/Addition


Example: GFP placed at ALB locus in HepG2 cells resulting in stably integrated GFP under control of ALB gene locus


Synthetic AAV DNA Displays Efficient Genome Editing




Our Pipeline

Our pipeline includes gene therapies for degenerative retinal disorders using AAV and non-viral systems. Longer term programs target ELOVL2 gene therapy for other aging-related disorders.

Mission In Focus.

Learn more about our goal to develop ELOVL2 based therapeutics to slow or halt aging-related disorders.

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