Recorded on the final day of ARVO 2026 in Denver, John Kitchens, MD, invites Marci English and Erin Kimbrel, PhD, from Astellas to review some of the most significant cell therapy data to emerge in retina in recent years: the Phase 1b interim results for ASP7317, an investigational RPE cell therapy that showed functional visual acuity gains in patients with advanced GA. The conversation covers what made earlier cell therapies fail, what's different now, the path to a registrational trial, and two additional pipeline programs: ASP2767 (a gene therapy for glaucoma) and ASP2020 (an immune-evasive RPE cell therapy for Stargardt disease).

The Vitreoretinal View segment features Murtaza Adam, MD, who has been performing IV-free vitreoretinal surgery since 2018, now accounting for roughly 70% of his cases. He breaks down patient selection, practical logistics, and why the approach could serve as a bridge to office-based surgery.

John and Scott then cover the realities of office-based surgery and speculate on what needs to change for retina surgeons to embrace it.

Hosts: John Kitchens, MD, Scott Krzywonos

Topics Covered

ASP7317 Phase 1b Interim Results

• ASP7317: investigational RPE cell therapy derived from human embryonic stem cells, delivered via subretinal transplantation in advanced GA secondary to AMD

• Three dose cohorts with severity stratification; BCVA improvements observed in multiple patients, including gains of 2 lines

• GA lesion area stabilized in treated eyes; only 1 month of immunosuppression required

• Mechanism: likely a combination of cell replacement, phagocytic debris clearance, and trophic factor secretion

• Why earlier RPE transplantation attempts failed: engraftment and immune rejection; advances in differentiation technique and surgical delivery have changed the picture

Pipeline & Path Forward

• Astellas plans to engage regulators in the coming months with the goal of moving directly to Phase 3; functional BCVA endpoints expected to be central to the registrational design

• Complement inhibitors and ASP7317 seen as complementary across the GA spectrum: slowing progression in early disease, potentially restoring function in advanced disease

• ASP2020: iPS-derived RPE cell therapy for Stargardt disease; Phase 1 expected in 2026; EVOLVE natural history study ongoing

• ASP2767: AAV gene therapy for neuroprotection of retinal ganglion cells in glaucoma; IND filing anticipated shortly

Vitreoretinal View: IV-Free Surgery (Murtaza Adam, MD)

• Oral sedation plus sub-Tenon's block replaces IV anesthesia in approximately 70% of Dr. Adam's cases; in practice since 2018 with published safety data

• Patient benefits: no fasting, no anesthesiologist copay, no GLP-1 or anticoagulation concerns; appropriate patients can drive themselves home

• Patient selection is key, and active patient communication during surgery is essential

Office-Based Surgery in 2026

• The financial model isn't there yet: no facility fee reimbursement in the office setting, and organized ophthalmology advocacy has stalled

Key Takeaways

• ASP7317 Phase 1b data showed cell-based therapy led to functional BCVA gains in advanced GA.

• Astellas views complement inhibitors and ASP7317 as complementary tools, not competitors, across the GA disease spectrum.

• IV-free vitreoretinal surgery is practical, patient-friendly, and already in routine use — but the path to office-based surgery still requires a reimbursement solution.

Credits Production & Marketing: Laura Brown | Business Operations: Liz Hogan

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