Enrollment complete in two more cohorts in X-linked retinitis pigmentosa gene therapy trial – Healio

February 21, 2020

Add Topic To Email Alerts

Enrollment is complete in the two highest dose groups in a phase 1/2 clinical trial evaluating an adeno-associated virus-based gene therapy for the treatment of X-linked retinitis pigmentosa, according to a press release from Applied Genetic Technologies Corporation.

The open-label, dose escalation trial is evaluating the safety and efficacy of AGTC-501 in patients with XLRP due to a mutation in the RPGR gene.

We expect that the information that will be obtained from the two additional dose groups will help to reinforce the data generated to date and previously reported in September 2019 and January 2020, Sue Washer, president and CEO of AGTC, said in the release. We plan to report interim data from these two new dose groups and to report top-line 12-month data for the first four dose groups in the second half of 2020 and intend to initiate a pivotal trial by the end of the year.

In January, the company announced 6-month preliminary data showing a favorable safety profile in all 25 patients treated with AGTC-501.

Enrollment is complete in the two highest dose groups in a phase 1/2 clinical trial evaluating an adeno-associated virus-based gene therapy for the treatment of X-linked retinitis pigmentosa, according to a press release from Applied Genetic Technologies Corporation.

The open-label, dose escalation trial is evaluating the safety and efficacy of AGTC-501 in patients with XLRP due to a mutation in the RPGR gene.

We expect that the information that will be obtained from the two additional dose groups will help to reinforce the data generated to date and previously reported in September 2019 and January 2020, Sue Washer, president and CEO of AGTC, said in the release. We plan to report interim data from these two new dose groups and to report top-line 12-month data for the first four dose groups in the second half of 2020 and intend to initiate a pivotal trial by the end of the year.

In January, the company announced 6-month preliminary data showing a favorable safety profile in all 25 patients treated with AGTC-501.

Continue reading here:
Enrollment complete in two more cohorts in X-linked retinitis pigmentosa gene therapy trial - Healio