2022 Year in Review: Top News and Insights in Gene Therapy

2022 was a big year for gene therapies, with FDA approvals coming in for indications including hemophilia B, transfusion-dependent beta-thalassemia, bladder cancer, and active cerebral adrenoleukodystrophy. With clinical trials for many more therapeutic areas underway, 2023 is shaping up to potentially offer even more advances in gene therapy.

CGTLive has brought you coverage of key advancements throughout the year, including the latest study data and clinical trial updates. Let's review some of our most-viewed coverage of gene therapies from 2022 below:

1. Running With Scissors: The FDA Moves to Tame Gene Therapy Drug Development

Looking to homogenize expectations around clinical trial design and execution, the FDA released 3 separate guidance documents in 2022 focused on the following topics: gene editing, gene therapy for neurodegenerative diseases, and gene therapy umbrella trials.

2. Stargardt Disease Gene Therapy IND Cleared to Proceed

Nanoscope Therapeutics received approval from the FDA for its investigational new drug (IND) application for MCO-010, a gene therapy for the potential treatment of Stargardt disease, a type of macular degeneration.

3. First Patient Dosed in Limb-Girdle Muscular Dystrophy Gene Therapy Trial

The first patient with FRKP-related limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) was treated with Atamyo Therapetuics’ ATA-100 (GNT0006), an investigational gene therapy, in a phase 1/2 clinical trial (EudraCT 2021-004276-33, NCT05224505).

4. Real-World Luxturna Data Shows Good Efficacy, New Adverse Reaction in Retinal Dystrophy

Findings from the ongoing PERCEIVE study demonstrated continued safety and efficacy of voretigene neparvovec (Luxturna; Spark Therapeutics) in patients with RPE65-mediated inherited retinal dystrophy in the real-world setting. The findings, which included up to 2 years of data, were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting, and also shed light on a new treatment-related adverse reaction.

5. Improvements in Developmental Milestones Observed With GM1 Gangliosidosis Gene Therapy

Data from the ongoing Imagine-1 clinical trial (NCT04713475) demonstrated good safety and impressive improvements in clinically meaningful end points in patients with GM1 gangliosidosis treated with PBGM01, an investigational gene therapy from Passage Bio.