Angelina Tadros
November 18, 2025
Genetics
Dr. Barbato
Personalized Gene Editing for All
A recent study discusses how the field of genetic medicine is going through major change, from a one size fits all medication style to more personal and uniquely fixed treatments that can correct someone's specific genetic mutation. The article explains how scientists and the FDA are working together to make personalized gene-editing treatments available to more patients. An important example is an infant named KJ, who was given a custom CRISPR treatment for a severe genetic disorder. Ultimately, his case proved that personalized editing is possible, but it also showed that the traditional drug-approval system is too slow and expensive to support one patient at a time therapies. This caused the FDA to explore new approval pathways that focus on approving entire platforms instead of individual custom drugs.
These platforms depend on the programmable nature of CRISPR technology, scientists can use the same basic treatment while switching out a small part of the RNA to target different mutations. The researchers described platforms for phenylketonuria (PKU) and for seven types of urea-cycle disorders (UCDs-which is what KJ had). After back and forth with the FDA, they found support for a streamlined process where only minimal new testing is required for each new mutation added to a platform. This means many patients with different genetic variants could be treated under one large general clinical trial.
Overall, the article highlights a major shift in genetic medicine and an interesting twist on medicine for once believed untreatable rare diseases. Instead of developing a new therapy from scratch for each patient, platform based editing could make customized treatments faster, safer, and more widely available. With continued support from the FDA and research agencies, personalized gene editing could move from experimental trials like KJ’s to a normal option for patients with rare genetic diseases. This discovery can be life changing for many.
Figure 1
Figure 1 demonstrates how all of the different variants are included efficiently in one trial, by each new IND mostly just reusing the original one and needing only small lab tests for each new variant.
Article + Picture link: https://www.cell.com/ajhg/fulltext/S0002-9297(25)00397-0
Extra source: https://www.sciencedirect.com/science/article/pii/S1465324925007492
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