hemophilia | GenomeWeb

hemophilia

The free genetic testing program has amassed genetic data and samples from more than 5,000 patients that researchers can now apply to access.

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.

The therapy is the company's first to feature an optimized version of its GalNAc conjugate delivery technology.

NEW YORK (GenomeWeb) – As Phase I testing of its hemophilia therapy ALN-AT3 continues, Alnylam Pharmaceuticals is setting its sights broadly for the program, taking aim at both patients receiving standard medical care as well as those who have become refractory to such treatment, company official

NEW YORK (GenomeWeb) – Although Alnylam Pharmaceuticals has focused on rare, genetically defined diseases in recent years, the company sees opportunities to someday move back into the broader indications that once populated its research and development initiatives, a top company official said las

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week announced that its Phase I hemophilia drug ALN-AT3 has received orphan drug status from European regulators.

Alnylam Pharmaceuticals this week announced that it has begun a phase I trial of ALN-AT3, an RNAi-based treatment for hemophilia and other bleeding disorders.

Alnylam Pharmaceuticals this week announced that it has filed a clinical trial application with UK regulators to begin human testing of an siRNA-based treatment for hemophilia A and B.

Alnylam Pharmaceuticals this week announced that it has received an orphan drug designation from the US Food and Drug Administration for its preclinical siRNA-based drug ALN-AT3 as a treatment for hemophilia A.

Officials from Alnylam Pharmaceuticals last week provided updates on the company’s pipeline programs, highlighting efforts involving its GalNAc conjugation technology, which enables subcutaneous siRNA delivery, and a fledgling effort in complement-mediated diseases.

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The White House has created a list of cuts, including to the NIH, that could be in a budget bill for this year.

The US National Institutes of Health is to allow applicants to cite preprints just as they would any other research paper, ScienceInsider reports.

Two manuscript pages handwritten by Charles Darwin are going on the auction block, according to the Los Angeles Times.

In PNAS this week: tool to track transcriptome-wide binding, evidence of balancing selection on behavior-linked genes, and more.