Interview Cansu de Muijnck, by Maud van Gerwen (board member) Cansu is a PhD student doing doctoral research on hereditary opticus atrophies (including ADOA). She works with Camiel Boon, Arthur van Bergen and Mies van Genderen, all specialists in this field. Cansu is originally from Turkey and came to the Netherlands four years ago for love. It took her a year to get her medical degree recognised here and she then worked in various positions [...]

Red light therapy Glen Jeffery, email interviewed by Peter Makai Recently, several companies have started to offer red light glasses https://eye-power.co.uk/, www.eyecharger.com.au which supposedly reduces vision loss in ageing eyes, through influencing mitochondria. Since ADOA is a mitochondrial disease, we were naturally curious if it can be safe and possibly beneficial for ADOA patients. ADOA is caused by a damaged gene (OPA1) causing several problems in the mitochondria within the so-called retinal ganglion cells, the [...]

In late 2021, one of our contacts at Stoke Therapeutics sent us their financial results and company updates. Among other things, this states: STK-002 is a proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of Autosomal Dominant Optic Atrophy (ADOA). Stoke believes that STK-002 has the potential to be the first disease-modifying therapy for people living with ADOA. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of [...]

The optic nerve death in ADOA is usually caused by a fault on the OPA1 gene, which takes care of producing the OPA1 protein. Looking into the research around ADOA, we found several researchers who have actually succeeded in editing the OPA1 gene under laboratory conditions. This is very important, as correcting the protein stops the slow cell-death in ADOA and the slow visual decline. The lead geneticist of the study, professor Michael Cheetham from [...]

PYC Therapeutics: Article for ADOA (Autosomal Dominant Optic Atrophy) European Association ADOA remains an area of significant unmet need, and with the advent of precision medicine there is increasing optimism around RNA and DNA therapies for ADOA. PYC Therapeutics, an RNA drug development company, is hoping to apply its RNA technology, known as PPMO, to many inherited retinal diseases. The company recently announced the development of a potential drug for certain sub-types of ADOA. “ADOA [...]

In our last newsletter of February 2021, we reported what kind of studies are currently ongoing. We also reported that there are studies on Glaucoma and Leber, among others, which are expected to be very interesting for us as ADOA patients. This is because the underlying problem has strong similarities. For example, we found an article about a study into Glaucoma and we put a number of questions to the researcher involved. The research [...]