Gene therapy is up again!

TextTom Lee

A few days ago, AGTC (Applied Genetic Technologies Corporation) broke out a serious safety issue after increasing the therapeutic dose in its pediatric gene therapy ophthalmic trial

AGTC said the gene therapy trial followed a model of increasing dose and decreasing age

Recently, the company recruited 6 pediatric achromatopsia (ACHM) patients with CNGB3 mutations and 5 pediatric ACHM patients with CNGA3 mutations in high-dose groups 5a and 6a

The test results showed that 3 of the 5 pediatric patients who received the highest dose developed severe inflammation about a month after the administration.

CEO Sue Washer said in a conference call with investors, “Part of the reason for the dose escalation study is to determine whether there is a maximum tolerated dose for children

In this regard, Washer did not provide enough ophthalmic gene therapy data to answer questions about dosage

Washer said, "These new data will not change AGTC's plan to continue to develop candidate products for panchromatic blindness, because a large amount of data makes AGTC confident in the plan to proceed with the appropriate dose

Currently, the company is drafting a data briefing for the Phase 2 trial to be submitted to the US Food and Drug Administration (FDA) and expects to receive feedback within the first quarter of 2022

It is worth noting that gene therapy from many companies other than AGTC has frequently exposed safety risks such as patient deaths, and it is not uncommon for gene therapy trials to be suspended

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