Disease Focus: Age-related macular degeneration
A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy
Phase 1 Safety Assessment of CPCB-RPE1, hESC-derived RPE Cell Coated Parylene Membrane Implants, in Patients with Advanced Dry Age Related Macular Degeneration
Age-related macular degeneration is a progressive disease resulting in death of the retinal pigment epithelium (RPE) causing distortion to central vision and eventually to legal blindness. Regenerative Patch Technologies and scientists at the University of Southern California and UC Santa Barbara, are growing specialized cells of the retina (called retinal pigment epithelium) from embryonic stem […]
An IPSC cell based model of macular degeneration for drug discovery.
Research Objective Towards a cure for age related blindness, we propose to create a human stem cell based model to screen for drugs that are protective against Age related macular degeneration (AMD). Impact With up to 11 million people in the United States affected by AMD, effective treatments against macular degeneration could address vision loss […]
Optogenetic Therapy for Treatment of Geographic Atrophy
Translational Candidate Optogenetic gene therapy for patients with geographic atrophy age related macular degeneration. Area of Impact Blindness from geographic atrophy age related macular degeneration Mechanism of Action Gene therapy to deliver optogenetic protein to the targeted cells of the retina to restore vision. Unmet Medical Need Geographic atrophy (GA) age related macular degeneration is […]
PRPE-SF, polarized hESC-derived RPE Soluble Factors, as a Therapy for Early Stage Dry Age-related Macular Degeneration
Translational Candidate PRPE-SF is a preparation of soluble factors from polarized retinal pigment epithelial cells, to support survival of photoreceptors in dry AMD (dAMD). Area of Impact dAMD with early geographic atrophy (RPE dysfunction/photoreceptor degeneration) that does not involve the fovea, with visual acuity better than 20/80. Mechanism of Action PRPE-SF is composed of multiple […]
NeuBright, a purified allogeneic cell therapy product for treatment of Dry Age-related Macular Degeneration
Translational Candidate NeuBright is an allogeneic cryopreserved neural stem cell therapy product Area of Impact Dry Age-Related Macular Degeneration (AMD) Mechanism of Action Similar to RPE cells, NeuBright cells restore phagocytic function to the retina, secrete anti-inflammatory and trophic factors (VEGF and BDNF), maintain retinal integrity and prevent vision decline. Cells migrate radially from transplant […]
Clinical Translation of Autologous Regenerative Cell Therapy for Blindness
Translational Candidate We are studying autologous induced pluripotent stem cell-derived retinal pigment epithelium (AiPSC-RPE) cells for the treatment of maculopathies. Area of Impact Maculopathies (including AMD, SMD, & MMD) may be treated with AiPSC-RPE cells to replace RPE and support photoreceptors to improve vision. Mechanism of Action AiPSC-RPE cells replace RPE lost to disease, and […]
A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy
Therapeutic Candidate or Device A patch comprised of a layer of stem cell-derived retinal pigmented epithelial (RPE) cells on a supporting matrix that is implanted under the retina Indication Geographic atrophy, the late-stage form of age-related macular degeneration Therapeutic Mechanism The healthy RPE cells on the implant replace dying RPE cells in the eye that […]
Clinical Translation of Autologous Regenerative Pluripotent Stem Cell Therapy for Blindness
Therapeutic Candidate or Device The therapeutic candidate is a patient specific (autologous) induced pluripotent stem cell derived retinal pigment epithelium (AiPSC-RPE) product. Indication AiPSC-RPE cell product will be indicated for the treatment of maculopathies related to RPE atrophy. Therapeutic Mechanism The proposed therapy is an autlogous cell product and intended to be used as a […]
Stem cell based treatment strategy for Age-related Macular Degeneration (AMD)
Retinal degeneration represents a group of blinding diseases that are increasingly impacting the health and well being of Californians. It is estimated that by 2020, over 450,000 Californians will suffer from vision loss or blindness due to the age-related macular degeneration (AMD), the most common cause of retinal degeneration diseases in the elderly. AMD is […]