明眸THE EYE ATLAS

07 — PIPELINE

眼科正在從「反覆打針」走向「一次處理」。

Ophthalmology is moving from repeat injections to one-time biology.

2025–2026 年的主軸：讓眼球自己製造抗 VEGF、用細胞補視網膜色素上皮、用不限基因的光遺傳學，以及把 CRISPR 送進角膜。以下多數仍是試驗，不是診所菜單。

The 2025–2026 arc: teach the eye to make its own anti-VEGF, graft RPE cells, restore light with mutation-agnostic optogenetics, and edit the cornea. Most of this is still a trial, not a menu.

THREE DIRECTIONS

2025–2026 最值得盯的三條線

Three lines worth watching in 2025–2026

點選方向卡片，下面的步驟、試驗與論文連結會跟著切換。多數仍是試驗，不是診所菜單。

Pick a direction; the steps, trials and paper links below follow. Most of this is still a trial, not a menu.

HOW IT WORKS

把抗 VEGF 的基因送進視網膜細胞

Deliver an anti-VEGF gene into retinal cells

接近診所的程度：高（第三期進行中）。目標是用一次玻璃體腔注射，換掉往後數年的每月打針。

Closeness to clinic: high (Phase 3 running). One intravitreal shot aims to replace years of monthly injections.

STEP 1工程病毒（AAV）載入抗 VEGF 或 aflibercept 基因。An AAV vector carries an anti-VEGF or aflibercept gene.
STEP 2注射入眼，細胞開始自己製造藥物。Injected into the eye; cells start making the drug.
STEP 34D-150 還多一條抑制 VEGF-C 的 RNA。4D-150 also encodes RNA against VEGF-C.
Ophthalmology Times · gene therapy 2026 AJMC · Khanani on Phase 3 4FRONT-2 · NCT07064759 4D-150 Phase 1/2 · NCT05197270
TIMELINE

點開節點看來源

Open a mark for sources

2025 AAO

酪胺酸激酶抑制劑與基因治療進入準上市討論。

TKIs and gene therapy treated as pre-launch.

AAO highlight → 2026
2025 Oct–Nov

PRIMA 晶片 NEJM 發表；密歇根 RPE 幹細胞早期字母增加。

PRIMA in NEJM; Michigan RPE stem-cell letter gains.

NEJM PRIMA Michigan stem cells
2026 Q1

OCU410 第二期、Axpaxli SOL-1、角膜 CRISPR IND、OPGx-BEST1 首例。

OCU410 Phase 2, Axpaxli SOL-1, corneal CRISPR IND, first OPGx-BEST1 patient.

OT · Q1 2026 highlights OCU410 NCT06018558
2026–2027

RGX-314、ixo-vec、4D-150 第三期陸續讀出。

RGX-314, ixo-vec and 4D-150 Phase 3 readouts.

4FRONT-2 Khanani interview
PIPELINE

試驗卡

Trial cards

PHASE 3

4D-150

一次注射產 aflibercept + 抑制 VEGF-C。4FRONT 已入組。

One shot making aflibercept + VEGF-C knockdown. 4FRONT enrolled.

PHASE 3

RGX-314 / ixo-vec

AAV 編碼抗 VEGF 抗體或 aflibercept。預計最早讀出之一。

AAV encoding an anti-VEGF fragment or aflibercept. Among the earliest readouts.

NEJM 2025

PRIMA implant

38 人植入，12 個月 81% 中央視力有臨床意義改善。

38 implanted; 81% had a clinically meaningful central-vision gain at 12 months.

PHASE 1–2

RPE cells / OCU410

幹細胞 RPE 移植；OCU410 用 RORA 基因減慢地圖狀萎縮生長。

Stem-cell RPE grafts; OCU410 uses RORA to slow GA growth.

FDA

Encelto

CNTF 細胞植入，目前唯一獲批的 MacTel 2 治療。

CNTF cell implant; currently the only approved MacTel 2 therapy.

PHASE 3

Axpaxli (OTX-TKI)

axitinib 水凝膠。SOL-1：36 周維持視力優於 2 mg aflibercept。

Axitinib hydrogel. SOL-1: vision maintenance at week 36 beat 2 mg aflibercept.

來源：NEJM、ClinicalTrials.gov、Ophthalmology Times、ScienceDaily、FDA、AJMC。試驗結果會改寫結論；本頁不是用藥依據。

Sources: NEJM, ClinicalTrials.gov, Ophthalmology Times, ScienceDaily, FDA, AJMC. Readouts will rewrite this. Not prescribing information.