RESTORING VISION WITH NEW CELLS

REGENERATIVE MEDICINE FOR RESTORING VISION in PATIENTS AFFECTED by GEOGRAPHIC ATROPHY (GA)

Our team is developing TPT-005, a first-in-class cell therapy for restoring vision in subjects effected by Geographic Atrophy (GA), to be administered via sub-retinal injection.  GA is a late stage of macular degeneration eventually resulting in a permanent loss of the central vision with no treatment available to halt the progression of the disease or restore the vision. Our investigational therapy TPT005 has the potential to offer a durable and curative solution by replacing the lost RPEs with heathy ones and restore the vision.

WHY REGENERATIVE MEDICINE FOR OPHTHALMOLOGY?

WHAT IS IT?

  • TPT-005 is an allogenic cell suspension of Retinal Pigment Epithelium (RPE) cells derived from human embryonic stem cells (hESC).
  • TPT-005 RPE cells are differentiated and expanded in vitro and exhibit the functions, the morphology, the markers and the pigmentation profile characteristic of endogenous RPE cells.
  • TPT005 is an off-the-shelf cell suspension developed specifically for sub-retinal injection.

WHAT IS THE MECHANISM?

  • We are born with a defined number of RPE and Photoreceptors which are remarkable cells able to live as long as we live, however not able to naturally regenerate.
  • RPE cells atrophy is a hallmark of GA which is accompanied by the death of RPE and consequently photoreceptors, resulting in a permanent loss of vision.
  • Replacement of lost cells through cell-based therapy is the only treatment modality with the potential to dramatically improve vision.
  • Following transplantation into the sub-retinal space, TPT-005 RPE cells have the potential to engraft, form a functional monolayer, re-establish the functions of the permanently lost endogenous RPE cells and restore the vision.

WHAT IS THE INDICATION?

  • TPT-005 is targeting the treatment of subjects effected by Geographic Atrophy (GA), an advanced form of dry age-related macular degeneration (dry-AMD) in which large, well-demarcated sections of the retina stop functioning due to the death of retinal cells.
  • Geographic Atrophy affects 5 million people worldwide and 1 million in USA and accounts for 20% of all legal blindness attributed to AMD
  • TPT-005 has the potential to offer a durable and curative solution by replacing lost endogenous RPE cells and restore vision.

WHAT IS THE CAUSE?

  • The causes of Geographic Atrophy (GA) are multifactorial, arising from a combination of genetic predispositions, biological processes, and environmental influences.
  • Key biological mechanisms that lead to RPE cell death include oxidative stress, over-activation of complement pathway, inflammation, mitochondrial dysfunction, accumulation of lipofuscin and drusen, impaired cellular waste removal and blood supply.
  • Risk of developing GA increases with AMD progression (early to intermediate), age, and there are some risk factors that contribute to GA development such as: smoking or history of smoking, specific genetic polymorphisms.
  • These mechanisms ultimately lead to the permanent loss of retinal cells, including RPE.

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