Retinal Cell Reprogramming Restores Vision in Non-Human Primate Study
Early applications of in vivo cellular reprogramming to medicine are cautiously focused on retinal regeneration. The eye is as close to an isolated system as one is going to find in the body, and only small amounts of a gene therapy vector are required for effective delivery. This very localized, comparatively isolated therapy bypasses or minimizes many of the technical concerns and areas of uncertainty regarding reprogramming, allowing those who are focused on pushing applications to the clinical to forge ahead. The more interesting applications remain those in which reprogramming factors are delivered systemically to much of the body, but a good deal of work remains to answer questions about safety, dosing, and effective delivery systems. Fortunately this part of the industry is very well funded, so answers seem likely to emerge in the years ahead.
Life Biosciences (Life Bio), a biotechnology company advancing innovative cellular rejuvenation technologies to reverse diseases of aging and injury and ultimately restore health for patients, today announced preclinical data in nonhuman primates (NHP) for its novel gene therapy candidate which uses a partial epigenetic reprogramming approach to restore visual function. Life Bio's therapy significantly restored visual function in an NHP model of non-arteritic anterior ischemic optic neuropathy (NAION), a disorder similar to a stroke of the eye that is characterized by painless yet sudden loss of vision.
Life Bio's lead platform reprograms the epigenome of older animals to resemble that of younger animals via expression of three Yamanaka factors, Oct4, Sox2, and Klf4, collectively known as OSK. The approach partially reprograms cells to resemble a more youthful state while retaining their original cellular identity. Previous data have shown that treatment with OSK reverses retinal aging and restores vision in old mice in a mouse model of glaucoma. Now, the company has demonstrated restoration of visual function and increased nerve axon survival in an NHP model that mimics human NAION deficits in retinal ganglion cells.
How do Life Bio actually propose to give the correct level and time period of reprgramming to every cell in the body?