Ophthalmology

The CGTLive™ Ophthalmology specialty topic page includes the most recent clinical care news related to the development of cell therapies, gene therapies, and engineered and regenerative medicines for disorders and diseases of the eye. It also contains video interviews with key opinion leaders in the field of ophthalmology discussing related FDA actions, clinical guideline updates, and clinical trial findings in the field of gene and cell therapy for ophthalmic conditions.

Latest News

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – August 14, 2025

August 15th 2025

Review top news and interview highlights from the week ending August 15, 2025.

EMA's CHMP Gives Ocugen the Green Light to Submit MAA for OCU410ST Based on GARDian3 Trial
EMA's CHMP Gives Ocugen the Green Light to Submit MAA for OCU410ST Based on GARDian3 Trial

August 14th 2025

Around the Helix: Cell and Gene Therapy Company Updates – August 13, 2025
Around the Helix: Cell and Gene Therapy Company Updates – August 13, 2025

August 13th 2025

AAVantgarde’s Stargardt Gene Therapy AAVB-039 Garners FDA Fast Track Designation
AAVantgarde’s Stargardt Gene Therapy AAVB-039 Garners FDA Fast Track Designation

August 12th 2025

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – August 8, 2025

August 8th 2025

Latest CME Events & Activities

More News

Around the Helix: Cell and Gene Therapy Company Updates – February 26, 2025

Around the Helix: Cell and Gene Therapy Company Updates – February 26, 2025

Noah Stansfield
February 26th 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – February 19, 2025

Around the Helix: Cell and Gene Therapy Company Updates – February 19, 2025

Noah Stansfield
February 19th 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – February 12, 2025

Around the Helix: Cell and Gene Therapy Company Updates – February 12, 2025

Noah Stansfield
February 12th 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – February 5, 2025

Around the Helix: Cell and Gene Therapy Company Updates – February 5, 2025

Noah Stansfield
February 5th 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Data Roundup: January 2025 Features Updates for Sarepta's Elevidys in DMD, Tenaya's Hypertrophic Cardiomyopathy Gene Therapy, and More

Data Roundup: January 2025 Features Updates for Sarepta's Elevidys in DMD, Tenaya's Hypertrophic Cardiomyopathy Gene Therapy, and More

CGTLive Staff
February 3rd 2025

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

FDA Activity Recap: January 2025 Features CRL, Multiple Clinical Holds, and More

FDA Activity Recap: January 2025 Features CRL, Multiple Clinical Holds, and More

CGTLive Staff
February 2nd 2025

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

Around the Helix: Cell and Gene Therapy Company Updates – January 29, 2025

Around the Helix: Cell and Gene Therapy Company Updates – January 29, 2025

Noah Stansfield
January 29th 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – January 22, 2025

Around the Helix: Cell and Gene Therapy Company Updates – January 22, 2025

Noah Stansfield
January 22nd 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

NGGT Biotechnology’s Gene Therapy NGGT001 Continues to Show Encouraging Safety in Bietti’s Crystalline Dystrophy

NGGT Biotechnology’s Gene Therapy NGGT001 Continues to Show Encouraging Safety in Bietti’s Crystalline Dystrophy

Noah Stansfield
January 22nd 2025

At 12 months of follow-up, there were no dose-limiting toxicities and no severe adverse events related to the treatment reported.

Around the Helix: Cell and Gene Therapy Company Updates – January 15, 2025

Around the Helix: Cell and Gene Therapy Company Updates – January 15, 2025

Noah Stansfield
January 15th 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Bart P. Leroy, MD, PhD, the head of the Department of Ophthalmology at the Center for Medical Genetics at Ghent University Hospital

ViGeneron Snags FDA Rare Pediatric Disease Designation for CNGA1-Associated Retinitis Pigmentosa Gene Therapy VG901

Noah Stansfield
January 14th 2025

In addition to the new designation, ViGeneron also announced that the data safety monitoring board for the trial has given the green light for dose escalation.

Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen

Ocugen’s Retinitis Pigmentosa Gene Therapy OCU400 Sustains or Improves Visual Function in 2-Year Follow-Up Data

Noah Stansfield
January 13th 2025

Among 9 patients treated in the study who were evaluable at 2 years posttreatment, all 9 showed improved or preserved visual function in comparison to their untreated fellow eyes.

Atsena Therapeutics’ Makes Progress With X-Linked Retinoschisis Gene Therapy Trial

Atsena Therapeutics’ Makes Progress With X-Linked Retinoschisis Gene Therapy Trial

Noah Stansfield
January 12th 2025

The company’s phase 1/2 LIGHTHOUSE clinical trial for ATSN-201 has moved onto its Part B.

Around the Helix: Cell and Gene Therapy Company Updates – January 8, 2025

Around the Helix: Cell and Gene Therapy Company Updates – January 8, 2025

Noah Stansfield
January 8th 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

CGTLive's 2024 Pillars of Progress: Top News in Ophthalmology

CGTLive's 2024 Pillars of Progress: Top News in Ophthalmology

CGTLive Staff
December 29th 2024

Take a look what stood out as pillars of progress and success from all of CGTLive's most popular ophthalmology stories in 2024.

Aurion Biotech’s Cell Therapy AURN001 Demonstrates Efficacy and Safety in Phase 1/2 ABA-1, CLARA Clinical Trial

Aurion Biotech’s Cell Therapy AURN001 Demonstrates Efficacy and Safety in Phase 1/2 ABA-1, CLARA Clinical Trial

Noah Stansfield
December 22nd 2024

The trial is evaluating AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.

Around the Helix: Cell and Gene Therapy Company Updates – December 18, 2024

Around the Helix: Cell and Gene Therapy Company Updates – December 18, 2024

Noah Stansfield
December 18th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – December 11, 2024

Around the Helix: Cell and Gene Therapy Company Updates – December 11, 2024

Noah Stansfield
December 11th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – December 4, 2024

Around the Helix: Cell and Gene Therapy Company Updates – December 4, 2024

Noah Stansfield
December 4th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – November 27, 2024

Around the Helix: Cell and Gene Therapy Company Updates – November 27, 2024

Noah Stansfield
November 27th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024

Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024

Noah Stansfield
November 20th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – November 13, 2024

Around the Helix: Cell and Gene Therapy Company Updates – November 13, 2024

Noah Stansfield
November 13th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024

Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024

Noah Stansfield
November 6th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

HuidaGene Therapeutics’ CRISPR/Cas13 RNA-Editing Therapy HG202 Gets Greenlight for US Trial in Age-Related Macular Degeneration

HuidaGene Therapeutics’ CRISPR/Cas13 RNA-Editing Therapy HG202 Gets Greenlight for US Trial in Age-Related Macular Degeneration

Noah Stansfield
November 6th 2024

According to HuidaGene, it is the first CRISPR/Cas13Y RNA-targeting therapy to receive clearance for a clinical trial from the FDA or any other regulatory agency.

Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial

Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial

Noah Stansfield
November 5th 2024

The randomized, controlled clinical trial treated patients with either a high dose of MCO-010, a low dose of MCO-010, or a sham–control.

Data Roundup: October 2024 Features Update for TCR-Based Autologous Cell Therapy in Melanoma, the First Clinical Demonstration of Therapeutic RNA Editing in Humans, and More

Data Roundup: October 2024 Features Update for TCR-Based Autologous Cell Therapy in Melanoma, the First Clinical Demonstration of Therapeutic RNA Editing in Humans, and More

CGTLive Staff
November 1st 2024

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Around the Helix: Cell and Gene Therapy Company Updates – October 30, 2024

Around the Helix: Cell and Gene Therapy Company Updates – October 30, 2024

Noah Stansfield
October 30th 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Ocugen Gets Green Light to Move Onto Phase 2 Portion of Trial Evaluating Stargardt Gene Therapy OCU410ST

Ocugen Gets Green Light to Move Onto Phase 2 Portion of Trial Evaluating Stargardt Gene Therapy OCU410ST

Noah Stansfield
October 24th 2024

The DSMB’s decision was made with reference to safety data from the GARDian trial’s phase 1 portion.

Around the Helix: Cell and Gene Therapy Company Updates – October 23, 2024

Around the Helix: Cell and Gene Therapy Company Updates – October 23, 2024

Noah Stansfield
October 23rd 2024

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Arshad Khanani, MD, the director of clinical research and director of Fellowship at Sierra Eye Associates

REGENXBIO’s ABBV-RGX-314 Reduces Need for AntiVEGF Injections After Application in Fellow Eyes of Patients With Wet AMD

Noah Stansfield
October 22nd 2024

The data comes from a phase 2 substudy (NCT03999801) that treated the fellow eyes of patients who had previously been treated with ABBV-RGX-314.

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