Sector News

Janssen/MeiraGTx showcase nine-month data for sight loss gene therapy

October 5, 2020
Life sciences

MeiraGTx and Janssen Pharmaceuticals have announced nine-month data from the ongoing phase I/II trial of their investigational gene therapy for X-linked retinitis pigmentosa (XLRP).

XLRP is a progressive condition that causes deterioration of the visual field and is the most severe form of retinitis pigmentosa – a group of inherited retinal diseases. The most frequent cause of XLRP is disease-causing variants in the RPGR gene, accounting for more than 70% of XLRP cases.

In nine-month data from the phase I/II trial, MeiraGTx and Janssen’s investigational gene therapy AAV-RPGR continued to demonstrate significant improvement in retinal sensitivity in treated eyes, across both the low and intermediate dose cohorts.

In addition, data from the assessment of vision-guided mobility carried out at the nine-month timepoint demonstrated a significant improvement in walk time, compared to baseline in treated eyes compared to untreated eyes in the low and intermediate dose cohorts.

“There are currently no treatment options for XLRP, and vision in patients suffering from this disease inevitably declines over time,” said Michel Michaelides, trial investigator, consultant pphthalmologist, Moorfields Eye Hospital and professor of Ophthalmology, University College London.

“Data from this clinical trial demonstrate that patients treated with AAV-RPGR had significant and sustained improvement in retinal sensitivity, as well as improved ability to navigate in low light conditions. These exciting results continue to suggest that AAV-RPGR has the potential to be a much-needed and important treatment option for those living with XLRP,” he added.

MeiraGTx and Janssen are jointly developing AAV-RPGR as part of a wider collaboration to develop and commercialise gene therapies for the treatment of inherited retinal diseases.

By: Lucy Parsons

Source: Pharma Times

Join the discussion!

Your email address will not be published. Required fields are marked *

Related News

January 17, 2021

SII expects WHO emergency approval of Covid-19 vaccine soon

Life sciences

The Serum Institute of India (SII) expects to soon receive World Health Organisation (WHO) emergency use authorisation for the Oxford University/AstraZeneca Covid-19 vaccine, produced for mid and low-income countries.

January 17, 2021

Sanofi to acquire clinical-stage biopharma firm Kymab for $1.45bn

Life sciences

According to the deal, Sanofi will gain full global rights to Kymab’s fully human monoclonal antibody, KY1005 that attaches to OX40-Ligand and can potentially treat various immune-mediated diseases and inflammatory ailments.

January 17, 2021

Moderna poaches Amgen vet as CCO, jump-starting commercial team as it rolls out COVID-19 vaccine

Life sciences

Moderna tapped veteran Amgen executive Corinne Le Goff to spearhead that effort as chief commercial officer.

Send this to a friend