Novel stem-cell based therapeutic for retinal and macular repair

Unmet Need / Invention Novelty: Currently, there are limited options for patients diagnosed with degenerative macular diseases, with available options reducing the rate of degeneration rather than repairing the diseased retinal tissue. There is an unmet need to develop a novel therapeutic for macular degenerations that repairs diseased tissue. 

Technical Details: Johns Hopkins researchers have developed a novel reparative stem-cell based therapeutic for macular degeneration. The therapeutic, Macular Repair Cells (MARC), consists of human stem cells derived from human retinal organoid culture. MARC is optimized in terms of its content of retinal cells including cone subtypes. MARC delivery is optimized for transplantation into the maculas to ensure optimal maturation, integration, and cone cell regeneration. MARC transplantation is envisioned to preserve and/or improve vision in people affected by macular diseases. The in vitro characteristics of MARC cells have been established.

Value Proposition: 

·       Stem-cell based therapeutic for macular degeneration derived from human retinal organoid culture

·       Repairs diseased retinal tissue by regenerating cone cell subtypes   

·       Optimized for transplantation, maturation, and integration into the macula

·       Broadly applicable to various diseases of macular degeneration including AMD, Stargardt disease, cone dystrophy, achromatopsia, Best disease, pattern dystrophy, mitochondrial macular degeneration, RDS-associated macular degeneration, and other inherited macular dystrophies 

Looking for Partners to: Develop & commercialize as a novel cellular therapeutic for retinal and macular repair. 

Stage of Development: Pre-Clinical 

Data Availability: in vitro 

Inventors:  Mandeep Singh & Robert Johnston  

Patent Status: Pending provisional patent application. 

Related Publication(s): Eldred KC, Hadyniak SE, Hussey KA, et al. Thyroid hormone signaling specifies cone subtypes in human retinal organoids. Science. 2018;362(6411):eaau6348. doi:10.1126/science.aau6348

Related Technologies: C15239, C15596, C16070,

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