Methods and compositions for treatment of retinal degeneration
Inventors
Case, Casey C. • Kawanishi, Toru • Kuno, Noriyuki
Assignees
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Publication Number
US-9326999-B2
Publication Date
2016-05-03
Expiration Date
Abstract
Disclosed herein are methods and compositions for treating retinal degeneration, such as occurs in retinitis pigmentosa and age-related macular degeneration, using descendents of marrow adherent stem cells that have been engineered to express an exogenous Notch intracellular domain.
Core Innovation
The document describes a method for treating retinal degeneration, including retinitis pigmentosa and age-related macular degeneration (AMD), by administering SB623 cells. SB623 cells are marrow adherent stem cell (MASC/MSC) descendants that have been engineered to express an exogenous Notch intracellular domain (NICD). The approach is positioned as enhancing photoreceptor function in a subject in need thereof.
The document further describes administration concepts directed to the eye, including transplantation into the eye with intravitreal or subretinal delivery. It describes local formulations/kits and optional repeated dosing within the claimed administration concepts. The outcomes are stated as increasing photoreceptor activity/function and visual signal transmission.
The document includes experimental support using an RCS rat model, reporting preserved electroretinography (ERG) a-/b-waves and retained azide responses after SB623 injection by intravitreal or subretinal delivery. It also describes histological preservation of the outer nuclear layer (ONL), persistence of transplanted cells, and restored visually evoked potentials (VEPs) at later timepoints.
Claims Coverage
The document includes one independent claim directed to a method of treating retinal degeneration by enhancing photoreceptor function using engineered MASC/MSC descendant cells expressing an exogenous Notch intracellular domain (NICD). The dependent claims further narrow the method by specifying transplantation into the eye, including intravitreal or subretinal delivery, and by limiting the retinal degeneration indication to particular named diseases/syndromes and additional subgroup selections for pigment pattern dystrophy.
Engineered MASC descendants expressing an exogenous Notch intracellular domain to enhance photoreceptor function
Administering, to the subject, cells descended from marrow adherent stem cells (MASCs) that have been engineered to express an exogenous Notch intracellular domain.
Transplantation into the eye delivery
Transplanting the claimed cells into the eye of a subject.
Intravitreal transplantation
Transplantation performed intravitreally.
Subretinal transplantation
Transplantation performed subretinally.
Treatment for retinitis pigmentosa
Applying the method to retinal degeneration occurring in retinitis pigmentosa.
Selected pigment pattern dystrophy subgroup
Selecting the pigment pattern dystrophy from a defined group of named dystrophy types.
Overall, the claim set is centered on administering MASC-derived SB623 cells engineered to express an exogenous NICD to enhance photoreceptor function, with delivery narrowed to transplantation into the eye (intravitreal or subretinal) and treatment indication refined to specific named retinal degeneration disorders and selected pigment pattern dystrophy subgroups.
Stated Advantages
Increasing photoreceptor activity/function.
Increasing visual signal transmission.
Documented Applications
Treating retinal degeneration, including retinitis pigmentosa and age-related macular degeneration (AMD).
Use in an RCS rat experimental support context reporting preserved ERG responses, ONL preservation, transplanted cell persistence, and restored VEPs at later timepoints.
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