
Atsena Therapeutics ’ AAV gene therapy is being evaluated in 15 patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D across 5 cohorts of various doses. Additional data are expected to be presented later this year.


Atsena Therapeutics ’ AAV gene therapy is being evaluated in 15 patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D across 5 cohorts of various doses. Additional data are expected to be presented later this year.

The clinical trial of HG004 for RPE65-LCA2 dosed its first patient in February 2023.

Review top news and interview highlights from the week ending March 31, 2023.

Most patients with RP treated with MCO-010 had clinically meaningful improvements in vision.

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

OCU400 is also being evaluated in other retinal degeneration indications.

Intergalactic Therapeutics’ IG-002 utilizes a proprietary non-viral delivery method.

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

GenSight Biologics reported that 73% of patients treated bilaterally with Lumevoq improved at least +15 ETDRS letters relative to their worst recorded BCVA.

Serious adverse events of reduced visual acuity and eye inflammation were observed in 2 clinical trials.

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

NR082 is being evaluated in a phase 1/2/3 clinical trial in patients with Leber hereditary optical neuropathy.

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

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

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

A third price model to be tested will limit the price of generic drugs for chronic conditions to $2 under Medicare plan D.

No serious adverse events deemed related to RGX-314 occurred in the treated patients.

Encouraging efficacy signals have also been observed in treated participants.

Review top news and interview highlights from the week ending February 10, 2023.

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

The new findings are consistent with previous research, in which ophNdi1 demonstrated benefit in AMD models.

The company is developing a one-time, non-AAV2 gene replacement therapy to restore, treat, and prevent blindness of patients with RPE65 mutation-associated retinopathies.

Review top news and interview highlights from the week ending February 3, 2023.

The clinical hold comes a few weeks after the company announced it was stopping enrollment in the phase 1/2 clinical trial.

The phase 1 clinical trial is the second of Frontera’s trials to begin dosing in 2023.