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ESR10: Optimisation, delivery and tolerability of antisense oligonucleotides to treat STGD1 patients with the most common splice mutations in ABCA4

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Partner
ProQR Therapeutics, Leiden, The Netherlands (www.proqr.com)
Supervisor
Prof. Dr. P. Adamson
Melita Kaltak
My name is Melita Kaltak and I will be joining the StarT crew the 1st of September as a PhD student within ProQR located in Leiden, the Netherlands. It will be the first time for ProQR to be involved in a Marie Curie Network, and I am super happy to be part of that! I am 24 years old, originally coming from Croatia, but I lived for many years in Italy and Sweden while studying. Recently I graduated in Functional Genomics at the University of Trieste in Italy, and, since then, I am working as a Laboratory Engineer within a research group at the Sahlgrenska Cancer Center in Gothenburg, Sweden. I have many hobbies, but I would say that I am most passionate about music. In my free time I play the drums, and I like to attend concerts and music festivals. Most of the time you can spot me among the first rows checking the drum player’s technique. Beside music, I love sports and spending time outdoors in the nature. Before discovering science, for many years I played tennis and was part of a volleyball club in the first Croatian league. I am very excited for having the opportunity to work with ProQR and being part of an important European project such as StarT, and I will be more than happy for contributing with my knowledge in all missions and challenges together with other 13 PhD students and their supervisors.
Abstract
ProQR is currently in Phase IIa clinical development with a splice modulating AON (QR-110) targeting the deep-intronic c.2991+1655A>G mutation in LCA type 10. In addition, they have two additional AON programs in Usher syndrome targeted all exon-13 mutations (QR-421a) and c.7595-2144A>G (QR-411a), respectively, in pre-IND/CTA enabling studies. ProQR is also in the discovery phase with regards to an intronic ABCA4 mutation (c.5461-10T>C), one of the most frequent mutations underlying STGD1 that results in the exclusion of exon 39, or exon 39 and exon 40 together, from the mRNA, leading to a frameshift. ESR10 will continue this program and optimise the sequence and chemistry of the final molecule to generate a clinical development candidate. This lead candidate will be further optimised by determining the extent of target engagement using digital-droplet PCR, immune-profiling against human donor T-lymphocyte panels, delivery to photoreceptors tolerability and pharmacokinetic parameters following intravitreal injection of mouse and rabbit models.
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This project has received funding from the European Union's Horizon 2020 research and innovation programme Marie Sklodowska-Curie Innovative Training Networks (ITN) under grant No.
813490
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