Disease Focus: Vision Loss
Extracellular Vesicle-Based Therapy for Corneal Scars
Translational Candidate Extracellular vesicles derived from stem cells for the treatment of corneal scars. Area of Impact The candidate aims to restoring vision by reducing the need for corneal transplant and the associated blinding complications. Mechanism of Action The therapeutic candidate is an extracellular vesicle (EV)-based therapy that could regenerate normal cornea tissue and reduce […]
Optogenetic therapy for treating retinitis pigmentosa and other inherited retinal diseases
Translational Candidate Ray-001 is an AAV gene therapy delivering a light sensitive gene to treat patients with advanced Retinitis Pigmentosa. Area of Impact Retinitis pigmentosa (RP) is a genetic disease that causes retinal degeneration leading to near or complete blindness for most patients. Mechanism of Action Ray Therapeutics' therapy (Ray-001) delivers a potent transgene with […]
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 […]
Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models
Translational Candidate Retina organoid sheets (ROs) derived from CSC14 human embryonic stem cells (NIH registry line #0284) manufactured under GMP conditions Area of Impact Retinitis PIgmentosa (RP) (irreversible loss of photoreceptors) due to mutation of photoreceptors and/or other retinal genes Mechanism of Action Proposed mechanism of action is cell replacement, combined with trophic effects. Transplanted […]
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 […]
Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect
Therapeutic Candidate or Device KPI-012 is a human bone marrow mesenchymal stem cell secretome formulated as a topical ophthalmic therapeutic Indication Treatment of persistent corneal epithelial defect, a cornea defect refractory to conventional treatments that can lead to blindness Therapeutic Mechanism KPI-012 is intended to restore normal corneal wound healing, providing a curative solution to […]
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1
Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication Mucopolysaccharidosis I (MPSI) is a rare disease that affects predominantly children. Untreated, these patients typically die by the age of 10. Therapeutic Mechanism The therapeutic […]
Safety and Feasibility of Cultivated Autologous Limbal Stem Cells for Limbal Stem Cell Deficiency
Therapeutic Candidate or Device Autologous cultivated limbal stem cells (cLSC) Indication Limbal stem cell deficiency Therapeutic Mechanism Restoration of a normal corneal surface using cLSC might be achieved by replenishing the LSC population and/or providing trophic factors to stimulate residual LSCs. Unmet Medical Need Therapy using cultivated LSC, which achieves the best clinical outcomes is […]