The Eyes Have It: FDA Approves Phase 1 Clinical Trial of Life Biosciences’ Reprogramming Therapy for Vision Loss

The core message for longevity-focused biotechnology companies is promising. The FDA is showing openness to the clinical testing of rejuvenation-style technologies when sponsors anchor them to a recognized disease, use accepted clinical outcomes, and build strong safety controls. The FDA has not reclassified aging itself as a disease, and it has not endorsed aging biomarkers as substitutes for patient outcomes. Even so, this step shines a light on how longevity and healthspan innovators can move through the existing regulatory framework.

What the FDA Approved

The FDA approved an Investigational New Drug application (IND) by Life Biosciences for ER-100, an epigenetic therapy to test a partial cellular reprogramming gene therapy in humans, starting in patients with optic neuropathies such as glaucoma and non-arteritic anterior ischemic optic neuropathy. The clinical trial will start small and focus on safety, tolerability, immune reactions, and standard measures of vision. 

This “approval” enables the developer to start a Phase 1 clinical trial, in which the therapy will be administered to one eye, use a well-known viral delivery method, and control activity through a simple drug on and off switch. A Phase 1 trial is the first test of a new therapy in people, and will focus on safety, tolerability, immune reactions, standard measures of vision, and appropriate dosage. This step only permits research use and does not allow sales, which would require a separate approval showing the product is safe and effective for a specific use. 

Importantly, the FDA’s decision to approve Life Biosciences’ IND for ER-100 shows the agency is open to reprogramming science when tied to a specific disease, using accepted outcomes, and has strong controls. It does not approve whole-body rejuvenation or treat aging as a standalone condition but does provide a clear entry path for similar clinical trials.

The FDA’s View of Aging and Why This Approval Is Significant

The FDA has long held that aging by itself is not a disease that can be listed as a drug indication. Further, the FDA has not validated a universal aging biomarker that can replace clinical outcomes. In other words, the FDA has not found any single lab test or score that can stand in for real patient benefit across aging. An aging biomarker is something you measure that estimates “biological age.” To replace clinical outcomes, a change in that number would need to reliably predict things that matter to people, like better vision, fewer hospital stays, slower memory loss, or longer life, across many settings. The FDA does not yet see enough proof that any aging biomarker does that. Companies can measure these biomarkers in studies, but the FDA decisions still depend on disease-specific results that show how patients feel, function, or survive. Sponsors must choose a defined disease, pick endpoints that regulators accept for that disease, and show the benefits outweigh the risks. 

Many in the longevity field want regulators to allow trials that target the root causes of aging and measure success either by combining several key health events into one outcome or by using a set of lab tests merged into a single biological age or risk score. Life Biosciences’ trial approval helps bridge the gap by keeping the disease-first rule while allowing research and development (R&D) to proceed. 

The FDA is establishing a measured playbook for longevity-focused developers. It focuses on specific diseases, local delivery, clear on and off control of gene activity, and standard outcomes, with aging biomarkers used as supporting data. Some will say this is still too narrow. In practice, it is a workable starting point that can build trust, produce the data needed to advance R&D, and create room for broader study designs over time. Sponsors that follow this approach and show rigor and transparency can help the FDA and the public get comfortable with reprogramming, paving the way for a new era of life science innovation.