Novel universal stem cell-based therapy for the treatment of retinal diseases

Unmet Need / Invention Novelty: Gene augmentation therapy, the current technology for treatment of inherited retinal diseases, is limited by its 1) small cargo capacity which challenges the delivery of larger genes 2) ability to target only a single genetic mutation, and thus disease subtype, at a time, and 3) applicability only to recessive diseases or dominant diseases with a haploinsufficiency mechanism.  These limitations hinder the ability of gene therapy to be a cure for all inherited retinal diseases and patients. There is an unmet need to develop a novel therapeutic strategy for inherited retinal diseases that addresses these limitations and universally repairs all dysfunctional retinal cells in all patients. 

Technical Details: Johns Hopkins researchers have developed a novel stem cell therapy that universally treats all subtypes of retinal diseases. The therapy, called Cytoplasmic Cell Transfer (CTC) therapy, repairs dysfunctional retinal photoreceptor cells by transferring healthy cytoplasmic materials into them. CTC is envisioned to be a universally curative strategy for retinal diseases delivered to the retina using previously disclosed technologies (C15239, C15596). The in vitro potency of CTC cells has been validated.

Value Proposition: 

·       Stem cell-based therapy that repairs dysfunctional retinal photoreceptors   

·       Universally therapeutic in the majority of degenerative retinal diseases

·       Mechanism of CTC is independent of genetic mutation, gene size, and inheritance type 

·       May not require long-term systemic immune suppression 

Looking for Partners to: Develop & commercialize as a novel cell-based therapy for retinal degenerations. 

Stage of Development: Pre-Clinical 

Data Availability: in vitro and in vivo

Inventors: Mandeep Singh, Seth Blackshaw, Robert Johnston & Jian Qian 

Patent Status: Pending PCT application.  

Related Publication(s): Singh MS, Balmer J, Barnard AR, et al. Transplanted photoreceptor precursors transfer proteins to host photoreceptors by a mechanism of cytoplasmic fusion. Nat Commun. 2016;7:13537. Published 2016 Nov 30. doi:10.1038/ncomms13537

Related Technologies: C15239, C15596, C16290