ASP7317 / Octapharma, CHA Bio & Diostech, Astellas - LARVOL DELTA

A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration (clinicaltrials.gov) - P1 | N=42 | Recruiting | Sponsor: Astellas Institute for Regenerative Medicine | Trial completion date: Jun 2026 ➔ Dec 2027 | Trial primary completion date: Jun 2026 ➔ Dec 2027

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Subretinal Transplantation of Human Stem Cell-Derived Retinal Pigment Epithelium (ASP7317) for the Treatment of Advanced Geographic Atrophy: Preliminary Phase 1b Results (ARVO 2026) - "On average, patients in all three groups showed improvements in vision and in the damaged areas of the eye after 26 weeks; these improvements were still seen at 52 weeks after treatment. These initial results from the ongoing study suggest that ASP7317 shows promise as a treatment for GA."

Metastases • P1 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Uveitis

A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration (clinicaltrials.gov) - P1 | N=42 | Recruiting | Sponsor: Astellas Institute for Regenerative Medicine | Trial completion date: Jan 2026 ➔ Jun 2026 | Trial primary completion date: Jan 2026 ➔ Jun 2026

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

A PhaseI/IIa Study to Determine the Safety and Tolerability of MA09-hRPE Cells in Patients With Advanced Dry AMD (clinicaltrials.gov) - P1/2 | N=10 | Terminated | Sponsor: CHABiotech CO., Ltd | Unknown status ➔ Terminated; Sponsor decision

Metastases • Trial termination • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Transplantation

Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD) (clinicaltrials.gov) - P1 | N=3 | Completed | Sponsor: CHABiotech CO., Ltd | Unknown status ➔ Completed

Trial completion • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Transplantation

A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration (clinicaltrials.gov) - P1 | N=42 | Recruiting | Sponsor: Astellas Institute for Regenerative Medicine | Phase classification: P1b ➔ P1 | N=18 ➔ 42 | Trial completion date: Aug 2024 ➔ Dec 2025 | Trial primary completion date: Aug 2024 ➔ Dec 2025

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

A Study of the Safety and Tolerability of ASP7317 in Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration (clinicaltrials.gov) - P1b | N=18 | Recruiting | Sponsor: Astellas Institute for Regenerative Medicine | Active, not recruiting ➔ Recruiting | Trial completion date: Nov 2022 ➔ Aug 2024 | Trial primary completion date: Nov 2022 ➔ Aug 2024

Enrollment open • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

A Study of the Safety and Tolerability of ASP7317 in Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration (clinicaltrials.gov) - P1b; N=18; Active, not recruiting; Sponsor: Astellas Institute for Regenerative Medicine; N=150 ➔ 18; Trial completion date: Jan 2027 ➔ Nov 2022; Trial primary completion date: Jan 2027 ➔ Nov 2022

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy (clinicaltrials.gov) - P1/2; N=16; Active, not recruiting; Sponsor: Ocata Therapeutics; Recruiting -> Active, not recruiting ; Trial primary completion date: Nov 2014 ->Aug 2015

Enrollment closed • Trial primary completion date • Biosimilar

Subretinal transplantation of human embryonic stem cell-derived retinal pigment epithelium (MA09-hRPE): A safety and tolerability evaluation in minipigs. (PubMed, Regul Toxicol Pharmacol) - "There were no other changes related to subretinal RPE cell injection. These results suggested that subretinal injection of hESC-derived RPE cells (60 and 120 × 10 cells/150 μL) in minipigs is well-tolerated and safe."

Clinical • Journal

A Phase I/IIa, Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Advanced Dry Age-related Macular Degeneration(AMD) (clinicaltrials.gov) - P1/2; N=12; Active, not recruiting; Sponsor: CHABiotech CO., Ltd; Trial completion date: Apr 2016 ➔ Jun 2020

Trial completion date

Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients (clinicaltrials.gov) - P1/2; N=13; Completed; Sponsor: Astellas Institute for Regenerative Medicine; Active, not recruiting ➔ Completed; Trial completion date: Dec 2019 ➔ Jun 2019; Trial primary completion date: Dec 2019 ➔ Jun 2019

Clinical • Trial completion • Trial completion date • Trial primary completion date

Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Patients With AMD (clinicaltrials.gov) - P1/2; N=11; Completed; Sponsor: Astellas Institute for Regenerative Medicine; Active, not recruiting ➔ Completed; Trial completion date: Dec 2019 ➔ Jul 2019; Trial primary completion date: Dec 2019 ➔ Jul 2019

Clinical • Trial completion • Trial completion date • Trial primary completion date

Transplantation of human embryonic stem cell-derived retinal pigment epithelial cells (MA09-hRPE) in macular degeneration. (PubMed, NPJ Regen Med) - "Given that the technology is still at its infancy for human use, a new clinical regulatory path could aid in cell line validation through small cohort, adaptive clinical trials to accelerate product development toward commercialization. These strategic insights will be invaluable to help both academia and industry, collaboratively shorten the steep learning curve, and reduce large development expenditures spent on unnecessary lengthy clinical trials."

Journal • Review