Biotechnology company MeiraGTx has announced encouraging news from its phase 1/2 clinical trial of botaretigene sparoparvovec (previously known as AAV-RPGR), a gene therapy aimed at X-linked retinitis pigmentosa caused by faults in the RPGR gene.
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Prof Mariya Moosajee at Moorfields Eye Hospital has asked us to share the message below about the clinical trial she is running. This trial is for a treatment that targets a particular section of the USH2A gene.
Retina UK is delighted to have awarded a new three-year research grant to Prof Majlinda Lako at Newcastle University for a project entitled: To establish AAV.PRPF31 gene augmentation in PRPF31-deficient RPE and photoreceptor cells and assess its efficacy in restoring RPE and photoreceptor function.
A blog from the research coal face
Jing Yu is a bioinformatician with the Eye Research Group at Oxford University, and is part of the UK Inherited Retinal Dystrophy Consortium (UKIRDC) team, funded by a Retina UK grant.
In April 2022 we joined with another seven leading sight loss organisations to make a public commitment to increasing the number of blind and partially sighted people employed within our sector, including at senior management level.
In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
With all the interest in exciting gene and cell therapies for inherited sight loss, it’s easy to forget that more traditional drugs can also hold promise.
This year the AGM will be held on Saturday, 9 July at 4.00pm:
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