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Genetic Neurodegenerative Diseases / CRISPR and Genetic Engineering · Journal article
Trends in Biotechnology · August 1, 2026
A consensus or society position rather than new primary data.
This is a perspective article arguing that as CRISPR therapeutics advance from in vitro research tools to clinical applications—exemplified by two FDA-approved therapies (exagamglogene autotemcel for hemoglobinopathies and kayjayguran abengcemeran for a metabolic disorder)—the field must develop a more sophisticated understanding of on-target and off-target mutational activities across diverse biological contexts. The authors frame this as a challenge requiring evolution in how CRISPR optimisation is approached, rather than presenting new data.
Journal article. Patients with hemoglobinopathies and ultra-rare metabolic disorders (as context for approved therapies discussed).
Two FDA-approved CRISPR therapies illustrate spectrum of molecular approaches: exagamglogene autotemcel (ex vivo, Cas9 nuclease for hemoglobinopathies) and kayjayguran abengcemeran (in vivo, PAM-altered base-editing Cas9 variant for ultra-rare metabolic disorder)
No original data, effect sizes, safety metrics, or efficacy comparisons are presented
This opinion frames an agenda for CRISPR therapeutic development and optimisation rather than providing actionable clinical evidence. Clinicians and researchers should interpret this as identifying an area where improved understanding of off-target effects and on-target optimisation is needed as CRISPR moves into clinical practice.
An opinion article addressing scientific and translational challenges in CRISPR therapeutics rather than reporting new empirical evidence or clinical outcomes.
This opinion frames an agenda for CRISPR therapeutic development and optimisation rather than providing actionable clinical evidence. Clinicians and researchers should interpret this as identifying an area where improved understanding of off-target effects and on-target optimisation is needed as CRISPR moves into clinical practice.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
What is missing. This record has no reported figures. That is a gap in the analysis, not a judgement about the study.
Recent FDA-approved gene-editing therapies illustrate not only the transformative potential of biotechnologies using CRISPR (clustered regularly interspaced short palindromic repeats)-derived ribonucleoproteins in treating a broad range of diseases but also the spectrum of possible molecular variations CRISPR therapeutics can adopt. These include exagamglogene autotemcel, an ex vivo therapy for hemoglobinopathies using CRISPR nuclease Cas9, and kayjayguran abengcemeran, an in vivo therapy using a protospacer adjacent motif-altered base-editing Cas9 variant for an ultra-rare metabolic disorder. Together, these therapies underscore how far CRISPR has advanced beyond its original use as a tool in biological/biomedical research. In this opinion article, we argue that as CRISPR biotechnologies advance beyond the relative simplicity of in vitro applications, our understanding must also evolve to address the challenges of optimizing ‘on-target' and ‘off-target' mutational activities across the diverse contexts in which they occur.
Taken from the source record, never inferred. Follow any of these and new work involving them reaches your briefing.