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RU A new step forward in the search for a cure: UMass Chan Licenses siRNA Technology
UMass Chan Medical School has licensed a promising siRNA-based technology to M2DS, a startup company dedicated to advancing the technology as a treatment for MECP2 Duplication Syndrome. This marks a major step forward in translating cutting-edge research into clinical reality. The technology was developed in the lab of Dr. Khvorova, with the project led by […]
Promising early results from the HERO Trial for MDS
Earlier this year, Alvin Luk attended the CRISPR Medicine Conference in Copenhagen to provide an update on the HERO clinical trial for MECP2 Duplication Syndrome. Alvin’s presentation is available for the community to watch on YouTube. In this video, you’ll learn about Huidagene’s experimental CRISPR-Cas13 therapy, HG204, and see the progress in the first treated […]
Start på rekruttering til ATTUNE-klinisk forsøg
ATTUNE-forsøget er en fase 1/2 klinisk undersøgelse sponsoreret af Ionis. Formålet er at evaluere ION440, en eksperimentel antisense-oligonukleotidterapi (ASO), der er udviklet til at behandle årsagen til MECP2-duplikationssyndrom (MDS).
Rekruttering starter til HERO: CRISPR cas13-klinisk forsøg med MDS
Det kliniske HERO-forsøg er et banebrydende studie, der evaluerer HG204, en ny CRISPR Cas13 RNA-redigeringsterapi, til MECP2 Duplication Syndrome (MDS). Forsøget, der er sponsoreret af HuidaGene, markerer et vigtigt skridt i jagten på en behandling af MDS. I november 2024 fik den første patient HG204 med succes, hvilket giver fornyet håb til MDS-samfundet.
ION440: IONIS bringer MDS-fællesskabet op på næste niveau
Written by David and verified by Ionis Following Ionis’ recent announcement about the upcoming clinical trial, we aim to provide a clearer understanding of this trial. It is especially important for families considering enrolling their child to know what a clinical trial is, particularly in the context of rare diseases like MECP2 Duplication Syndrome (MDS). […]
HuidaGene: På vej mod kliniske forsøg
In February 2024, David Covini, President of DupMECP2, visited the innovative biotech company HuidaGene Therapeutics in Shanghai. The visit provided an exciting glimpse into the future for a treatment for MECP2 duplication syndrome (MDS). Led by Dr. Alvin Luk, CEO at HuidaGene, the Company is at the forefront of developing a groundbreaking CRISPR technology that […]
Huidagene: En lovende CRISPR-CAS13-teknologi fra Shanghai (Kina)
Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based medicines. The company focuses on muscular, ophthalmic and neurological disorders. Huidagene has developed HG204, an adeno-associated viral (AAV) vector delivering a novel CRISPR-Cas13 complex into cells. Designed for MECP2 duplication syndrome, HG204 aims to regulate the […]