Treatment for retinal degenerative diseases

Pharmaceutical, Life sciences

UNMET NEED

Millions of North Americans suffer from irreversible vision loss due to retinal degenerative diseases such as retinitis pigmentosa, age-related macular degeneration, cone-rod dystrophies, Leber congenital amaurosis, Stargardt disease, and Usher syndrome. The common cause sight impairments in these diseases is progressive death of the light-sensing cells of the retina, the rod and cone photoreceptors. While rod photoreceptor degeneration leads to night blindness and reduced peripheral vision, it is the loss of cones that is the most devastating to patients because these cells provide the most-important daylight and high acuity macular vision in humans. Although there are currently some treatments available to slow disease progression and cone loss in some conditions, there are no cures available for any retinal degenerative diseases.

TECHNOLOGY OVERVIEW

Regeneration of photoreceptor from mammalian Müller glia, an endogenous source of stem cells. Pr. Michel Cayouette laboratory have shown that the co-expression of specific genes can reprogram Müller glia into cone-like cells in retinal explants.  The co-expression induced clear morphological changes and the majority of the cells moved to the apical side of the retina in the outer nuclear layer (ONL) where cone photoreceptors are usually located. As compared to the control conditions, the majority of the morphologically programmed cells lost Müller glia markers and gain cone specific markers.

Animal experiments have confirmed the migration of the reprogram Müller glia toward the ONL and revealed the presence of an inner and outer segment and of axon reaching towards the inner nuclear layer (INL). These cells also showed normal cone nuclear architecture with multiple chromocenters associated to the nuclear lamina. Immunofluorescence studies have revealed the presence of several mature cone markers in the reprogram Müller glia cells.

 

COMPETITIVE ADVANTAGES

  • In vitro, in vivoand ex-vivodatas
  • Novel targets
  • Known mechanism of action

 

BUSINESS OPPORTUNITY

  • Eligibility to government financing for industry/academic maturation program
  • Seeking for industrial co-development partner and research partnering
  • Technology available for in-licensing

 

MARKET APPLICATIONS

  • Treatment/cure for retinal degenerative diseases
  • Cure for blindness

 

IP PROTECTION

  • US patent application

CONTACTS

Michel Cayouette, Ph. D.

PRINCIPAL INVESTIGATOR
Professor, Department of Cellular Neurobiology
University of Montreal

Benoit Doré, Ph. D.

CONTACT PERSON
Project Manager, Life Sciences
Axelys
benoit.dore@axelys.ca