This email brought to mind Linus Pauling....who won a Nobel prize... twice. Double luck? And if I remember correctly, he was eventually thrown under the bus when he started using vitc and lysine for heart health issues.
I ran into Pauling in a hallway waiting to be interviewed on WBEZ (local public radio in Chicago) in the late 80s I think. Recognized him immediately and spoke briefly to him. If memory serves, he was there hawking his latest book about vitamin C. Had a copy in his lap. No entourage, just him sitting alone in a bleak hallway. Probably took the bus to get there.
Vinay is simply stating the obvious. Normally, the awarding of a prize is based on the quality of the recipient, but the choice among many eligible candidates depends on many accidental factors. Thus, I also consider that the overwhelming majority of people who receive the Nobel Prize are those who have been most favored by the combination of accidental factors. And in the case of the Nobel Peace Prize, this is glaringly evident!
However, there have been a small number of Nobel laureates who received it due to absolute merit. But there are also a small number of people with absolute demerit who have received the Nobel Prize.
In short, all activities must have references to admire, just as ideologies and religions have their saints.
The criticism is outdated. Optogenetics (light-based control of genetically modified cells, typically using channelrhodopsins or similar opsins) has produced real clinical progress in humans, primarily for vision restoration in advanced retinal degenerations, with multiple trials showing functional improvements and one therapy now under FDA review for potential approval.
Direct clinical applications in humans
The strongest evidence is in retinitis pigmentosa (RP) and related inherited retinal diseases (e.g., Stargardt disease), where photoreceptors die but remaining retinal cells (bipolar or ganglion cells) can be made light-sensitive via gene therapy (usually AAV vectors delivered by intravitreal injection). This is mutation-agnostic, so it does not require knowing the specific genetic cause.
Key developments (as of late 2025–2026):
Nanoscope Therapeutics’ MCO-010 (sonpiretigene isteparvovec / Mogenry): Multi-characteristic opsin therapy targeting bipolar cells, activatable by ambient light. Phase 2b/3 RESTORE trial (randomized, sham-controlled) met primary endpoints with clinically meaningful vision gains (e.g., multi-luminance mobility/shape discrimination tests; visual acuity improvements equivalent to ~3 lines on an eye chart in longer-term follow-up). Safety was favorable (no serious drug-related ocular/systemic adverse events in key reports). The FDA accepted the Biologics License Application (BLA) in 2026, with a decision expected in the first half of 2027. If approved, it would be the first FDA-approved optogenetic therapy.
GenSight Biologics’ GS030: Channelrhodopsin-based (ChrimsonR) approach combined with specialized light-projecting goggles. Phase 1/2 PIONEER data showed partial recovery of object perception/localization in previously blind RP patients; longer-term follow-up continues.
Other programs: Zhongmou’s ZM-02 (positive first-in-human data with vision gains vs. sham; FDA IND clearance for multinational trials); Restore Vision’s RV-001 (early signals of light perception recovery); trials for Stargardt disease and geographic atrophy; additional candidates from Ray Therapeutics and others. Multiple Phase 1/2 studies (e.g., NCT02556736, NCT04919473, NCT04945772) report safety and functional gains such as improved mobility, object recognition, and (in some cases) color vision.
A 2021 proof-of-principle case and subsequent trials demonstrated that optogenetic gene + light delivery to the human retina can restore usable visual perception in end-stage disease. No major safety signals have derailed the programs to date in published reports.
Broader impact and indirect benefits
Optogenetics’ biggest contribution so far has been as a research tool that generated causal circuit knowledge now informing other therapies (deep brain stimulation refinements, drug targeting, neuromodulation protocols). A 2025 Nature Neuroscience roadmap paper explicitly notes both direct (retinal) translation and these indirect pathways for conditions such as Parkinson’s, epilepsy, pain, and others.
Direct brain applications remain preclinical or early-concept due to delivery, specificity, long-term safety, and ethical hurdles (invasive gene + light delivery into the CNS). No approved treatments exist yet for psychiatric or most neurological disorders outside the eye.
Context on the criticism
Early critiques (e.g., ~2013) correctly noted the gap between laboratory circuit mapping and human therapies, overselling risks for mental illness treatments, and practical barriers (gene delivery, light sources, specificity). Those points were fair at the time. Progress has been deliberately focused on the retina first because light delivery is straightforward, the target cells are accessible, downstream circuitry is often intact, and the unmet need (irreversible blindness) is high. Clinical translation for other organs/systems is slower and faces greater technical/ethical barriers, but the retinal results refute claims of “no real-world human impact.”
In short, optogenetics has moved beyond pure research into human trials with measurable benefits for vision loss, and regulatory review of the first therapy is underway. Further applications will depend on contin
Reformed scientist here; this article is absolutely on the button. Needed to be said -- well done.
This email brought to mind Linus Pauling....who won a Nobel prize... twice. Double luck? And if I remember correctly, he was eventually thrown under the bus when he started using vitc and lysine for heart health issues.
I ran into Pauling in a hallway waiting to be interviewed on WBEZ (local public radio in Chicago) in the late 80s I think. Recognized him immediately and spoke briefly to him. If memory serves, he was there hawking his latest book about vitamin C. Had a copy in his lap. No entourage, just him sitting alone in a bleak hallway. Probably took the bus to get there.
Vinay is simply stating the obvious. Normally, the awarding of a prize is based on the quality of the recipient, but the choice among many eligible candidates depends on many accidental factors. Thus, I also consider that the overwhelming majority of people who receive the Nobel Prize are those who have been most favored by the combination of accidental factors. And in the case of the Nobel Peace Prize, this is glaringly evident!
However, there have been a small number of Nobel laureates who received it due to absolute merit. But there are also a small number of people with absolute demerit who have received the Nobel Prize.
In short, all activities must have references to admire, just as ideologies and religions have their saints.
The criticism is outdated. Optogenetics (light-based control of genetically modified cells, typically using channelrhodopsins or similar opsins) has produced real clinical progress in humans, primarily for vision restoration in advanced retinal degenerations, with multiple trials showing functional improvements and one therapy now under FDA review for potential approval.
Direct clinical applications in humans
The strongest evidence is in retinitis pigmentosa (RP) and related inherited retinal diseases (e.g., Stargardt disease), where photoreceptors die but remaining retinal cells (bipolar or ganglion cells) can be made light-sensitive via gene therapy (usually AAV vectors delivered by intravitreal injection). This is mutation-agnostic, so it does not require knowing the specific genetic cause.
Key developments (as of late 2025–2026):
Nanoscope Therapeutics’ MCO-010 (sonpiretigene isteparvovec / Mogenry): Multi-characteristic opsin therapy targeting bipolar cells, activatable by ambient light. Phase 2b/3 RESTORE trial (randomized, sham-controlled) met primary endpoints with clinically meaningful vision gains (e.g., multi-luminance mobility/shape discrimination tests; visual acuity improvements equivalent to ~3 lines on an eye chart in longer-term follow-up). Safety was favorable (no serious drug-related ocular/systemic adverse events in key reports). The FDA accepted the Biologics License Application (BLA) in 2026, with a decision expected in the first half of 2027. If approved, it would be the first FDA-approved optogenetic therapy.
GenSight Biologics’ GS030: Channelrhodopsin-based (ChrimsonR) approach combined with specialized light-projecting goggles. Phase 1/2 PIONEER data showed partial recovery of object perception/localization in previously blind RP patients; longer-term follow-up continues.
Other programs: Zhongmou’s ZM-02 (positive first-in-human data with vision gains vs. sham; FDA IND clearance for multinational trials); Restore Vision’s RV-001 (early signals of light perception recovery); trials for Stargardt disease and geographic atrophy; additional candidates from Ray Therapeutics and others. Multiple Phase 1/2 studies (e.g., NCT02556736, NCT04919473, NCT04945772) report safety and functional gains such as improved mobility, object recognition, and (in some cases) color vision.
A 2021 proof-of-principle case and subsequent trials demonstrated that optogenetic gene + light delivery to the human retina can restore usable visual perception in end-stage disease. No major safety signals have derailed the programs to date in published reports.
Broader impact and indirect benefits
Optogenetics’ biggest contribution so far has been as a research tool that generated causal circuit knowledge now informing other therapies (deep brain stimulation refinements, drug targeting, neuromodulation protocols). A 2025 Nature Neuroscience roadmap paper explicitly notes both direct (retinal) translation and these indirect pathways for conditions such as Parkinson’s, epilepsy, pain, and others.
Direct brain applications remain preclinical or early-concept due to delivery, specificity, long-term safety, and ethical hurdles (invasive gene + light delivery into the CNS). No approved treatments exist yet for psychiatric or most neurological disorders outside the eye.
Context on the criticism
Early critiques (e.g., ~2013) correctly noted the gap between laboratory circuit mapping and human therapies, overselling risks for mental illness treatments, and practical barriers (gene delivery, light sources, specificity). Those points were fair at the time. Progress has been deliberately focused on the retina first because light delivery is straightforward, the target cells are accessible, downstream circuitry is often intact, and the unmet need (irreversible blindness) is high. Clinical translation for other organs/systems is slower and faces greater technical/ethical barriers, but the retinal results refute claims of “no real-world human impact.”
In short, optogenetics has moved beyond pure research into human trials with measurable benefits for vision loss, and regulatory review of the first therapy is underway. Further applications will depend on contin