TTR amyloidosis | GenomeWeb

TTR amyloidosis

The firm also said it aims to soon begin human studies for its hepatic porphyria treatment ALN-AS1 and to select a development candidate for its program in primary hyperoxaluria type 1.

All of the adverse events were deemed mild and are not expected to affect the drug's future clinical development.

 

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals announced this week that it has completed enrollment in a Phase II trial of its subcutaneously administered TTR-mediated amyloidosis (ATTR) drug revusiran, formerly known as ALN-TTRsc.

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week released data from an ongoing Phase II open-label extension study of its TTR-mediated amyloidosis therapy patisiran, which showed that the drug treatment was associated with a stabilization of neurological impairment progression in patients

NEW YORK (GenomeWeb) – RNAi startup Arcturus Therapeutics this week released new preclinical data on its lead transthyretin-mediated amyloidosis (ATTR) candidate LUNAR-101, showing that a single dose of the drug could cut levels of its target by more than 90 percent in non-human primates (NHPs) f

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week announced that it has initiated a screening study, called DISCOVERY, to examine the prevalence of transthyretin (TTR) mutations in patients believed to have cardiac amyloidosis, in support of its continued development of an RNAi-based treat

NEW YORK (GenomeWeb) – Officials from Alnylam Pharmaceuticals last week provided updates on the two drug candidates from the company's flagship transthyretin-mediated amyloidosis program, stating that the intravenously delivered agent patisiran is proceeding toward a possible market approval in t

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week unveiled new Phase II data on its transthyretin-mediated amyloidosis (ATTR) therapy patisiran, which showed that the drug is capable of knocking down its disease-causing protein target by about 80 percent in patients — in line with previous

Alnylam Pharmaceuticals this week announced that its transthyretin-mediated amyloidosis drug ALN-TTRsc has received orphan drug status from European regulators.

Arcturus Therapeutics this week announced new data showing that its proprietary lipid nanoparticles are capable of functionally delivering messenger RNA into the livers of mice, which a company official said opens the door for the use of the technology with CRISPR-based drugs.

Pages

In Science this week: genetic target for urothelial bladder cancer treatment, and more.

At the Conversation, the University of Oxford's Michael Macklay writes that learning genetic risk of disease is a personal decision.

Two dozen scientific organizations have endorsed the March for Science, according to ScienceInsider.

Researchers in Japan describe a chimpanzee with a chromosomal abnormality similar to human Down syndrome, Mashable reports.