US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Huntington Disease
VICO Therapeutics,
a Leiden Bio Science Park, the Netherlands, based biotech company focusing on the development of RNA modulating therapies for rare neurological disorders, today announced that the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for VO659, VICO s investigational antisense oligonucleotide (AON) therapy, for the treatment of Huntington s disease (HD). This news comes only one month after VICO announced that VO659 had received orphan-drug designation for the treatment of spinocerebellar ataxia (SCA). This new designation completes the quartet of orphan designations for VO659 in HD and SCA in both the US and EU.
Gene and Cell Therapies Expected to Offer New Treatment Paradigm in CNS Disorders; Market Expected to Increase at a CAGR of 45% During the Study Period 2018-2026 in the 7MM
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US FDA grants VICO Therapeutics Orphan-Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia
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Două tinere au moştenit de la tatăl lor boala Huntingdon ce poate fi mortală
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The sweeping changes to the Embryo Protection Act in 2018 enabled in-vitro fertilisation to be unshackled from some of the constraints introduced six years earlier.
The changes allowed doctors to be able to fertilise up to five eggs instead of only two, increasing the chances of success. They also made embryo freezing an integral part of the treatment process.
But government was very careful back then to steer clear from introducing pre-implantation genetic diagnosis (PGD) on embryos in those cases where prospective parents carry the risk of transmitting a hereditary disease or genetic disorder to their child.
In parliament, several government MPs at the time made it a point to emphasise that PGD will not be on offer.