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hemophilia

Originally published Oct. 8

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.

Alnylam Pharmaceuticals this week announced that it has received an orphan drug designation from the US Food and Drug Administration for its subcutaneous hemophilia B treatment ALN-AT3.

Alnylam Pharmaceuticals this week released preclinical data showing that ALN-AT3, its investigational treatment for hemophilia and other bleeding disorders, can normalize thrombin generation and improve hemostasis in hemophilia mice and fully correct thrombin generation in a non-

Alnylam Pharmaceuticals this week announced that it plans to report initial data from an ongoing phase II trial of its lead drug candidate, the TTR-mediated amyloidosis treatment ALN-TTR02, this summer at an upcoming scientific meeting, and that it will launch an expansion study

Alnylam Pharmaceuticals this week provided an update to its research and development activities, adding a new preclinical program to its pipeline with ALN-AS1 for acute intermittent porphyria while dropping its refractory anemia drug ALN-HPN in order to focus on “higher priority”

Alnylam Pharmaceuticals this week presented preclinical data from its hemophilia and bleeding disorder program, showing that its subcutaneously administered drug candidate ALN-AT3 could trigger potent, dose-dependent, durable target knockdown in non-human primates.

This article has been updated from a version posted Nov. 13 to correct the name of the National Hemophilia Foundation.

NEW YORK (GenomeWeb News) – Two patient advocacy groups, Biogen Idec, and a medical center have teamed up to offer free or low-cost genetic testing to people with hemophilia and their families to identify ways to personalize patient care, generate new genetic information, and help create a databa

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