News

Researchers from the Seoul National University (Korea) have applied for the first an improved genome-editing methodology, named prime editing to correct the genetic defects of two animal models of human disease: a liver disease (hereditary tyrosinemia) and an eye disease (Leber congenital amaurosis). This high-precision genome-editing tool, developed only two years ago by the team of David R. Liu from...

Encouraging advances in therapies for Leber congenital amaurosis

05/07/21 - Leber Congenital Amaurosis, Therapy, Gene Therapy,

Editas Medicine CRISPR therapy for CEP290 gene Editas Medicine, a company specialising in the development of gene editing therapies, has announced the recruitment of paediatric patients to the Brilliance trial. This phase 1/2 clinical trial began last year and is based on the CRISPR gene editing methodology to excise the intron mutation c.2991+1655A>G, which produces an aberrant messenger RNAs (see...

DBGen awarded an EASI-Genomics grant for innovative long-read sequencing of inherited retinal dystrophies

28/06/21 - DBGen, Genetic Diagnosis,

The Transnational Access Committee of the European Advanced infraStructure for Innovative Genomics (EASI-Genomics) has selected the project PID15251 submitted by DBGen Ocular Genomics in the third call for projects of this initiative. DBGen's proposal has successfully passed the scientific and technical evaluation steps of this highly competitive call. EASI-Genomics is an initiative, funded with 10 million euros by the European...

Promising results for a first-in-class Usher syndrome IIA therapy

13/04/21 - Retinitis pigmentosa, Usher syndrome, Therapy,

The Dutch biotechnology company ProQR has just presented (March 2021) the first results of a clinical trial, named Stellar, with QR-421a, an antisense oligonucleotide designed to stop vision loss or restore vision in Usher syndrome IIA and non-syndromic retinitis pigmentosa patients due to USH2A exon 13 mutations. The therapeutical approach aims to exclude the exon 13 coding region in the...

LHON gene therapy affords sustained improvement in visual acuity paving the way to approval

17/03/21 - Gene Therapy,

The biopharma company GenSight Biologics, specialized in developing gene therapies for retinal degenerative diseases, recently published results of two Phase III clinical trials on the effect of LUMEVOQ gene therapy for Leber Hereditary Optic Neuropathy (LHON) patients. In September 2020, GenSight requested approval of LUMEVOQ to the European Medicines Agency (EMA) to treat LHON patients carrying a mutation in the...

Optogenetic therapy to restore advanced vision loss in retinal dystrophies

08/10/20 - Retinal Dystrophy, Gene Therapy,

Gene therapies currently under study for retinal dystrophies are administered when viable photoreceptor able to be rescued at the functional level still remain. Most therapies are gene-specific and, in some cases, mutation-specific. In advanced disease stages, when a large number of photoreceptor cells have degenerated, the probability of success of these therapies is very low. Therefore, researchers are exploring methodologies...

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