Close Menu

Huntington's disease

The company also disclosed that it and partner Cubist Pharmaceuticals have decided to put on hold the development of a second-generation version of Alnylam's respiratory syncytial virus treatment, although no details regarding the decision were provided.

Alnylam said that the non-profit CHDI Foundation has agreed to pay up to 50 percent of the costs of advancing a treatment for the disease up through an investigational new drug application filing.

The combined grants are worth nearly $650,000 and are meant to support work on treatments for cancer and Huntington's disease.

ISB and partner Complete Genomics will perform whole-genome sequencing on DNA samples provided by Gladstone's Taube-Koret Center for Huntington's Disease Research.

The goal is to identify inherited variants that affect the manifestation of Huntington's disease and that could be used as drug targets.

The grants will fund projects seeking to validate novel molecular targets for disease-modifying interventions for Huntington's disease.

The Galapagos subsidiary will conduct high-content screening and a range of other services for CHDI's Huntington's disease drug development programs.

The method is currently available from Asuragen as a research-use-only PCR kit, but the company is hoping that the publication is an important step toward developing a routine PCR-based diagnostic for the disorder.

Complete Genomics will sequence for ISB 100 genomes from Huntington's disease patients, family members, and matched controls to study modifiers of disease presentation and progression.

The newly released preclinical data shows that direct delivery of huntingtin-targeted siRNAs induced "robust silencing" of the huntingtin mRNA, with the effect observed at "substantial distances from the infusion site," the companies said.

Pages

According to Science, the US National Academy of Sciences is poised to eject two members accused of sexual harassment.

A UK study on mixing SARS-CoV-2 vaccines is expanding to include vaccines developed by additional companies, according to the Guardian.

In Nucleic Acids Research this week: machine learning approach to detect DNA-bound proteins, CRISPR-based method for activating specific gene targets, and more.

The FDA and CDC call for a pause in administering Johnson & Johnson's SARS-CoV-2 vaccine while reports of rare blood clots are looked into, reports the Wall Street Journal.