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Sep 03, 2020

Blood Cell Traits Linked to Individual Loci, Polygenic Contributors in International Studies

Two new papers have identified thousands of genetic loci with ties to more than two-dozen blood cell traits in individuals from up to five ancestry groups.

Aug 20, 2020

Asks for More

Reuters reports that the Food and Drug Administration has rejected BioMarin's gene therapy for hemophilia A and has asked for longer-term data.

Jul 20, 2020

A High Price

A gene therapy for hemophilia could cost millions of dollars, NPR writes.

Aug 07, 2018

New Hemophilia Treatment Data

Spark Therapeutics reports new data on its investigational hemophilia A treatment that says it reduces bleeds, according to Stat News.

May 23, 2018

Quickly, Quickly

Gene therapies could qualify for a faster US Food and Drug Administration approval process, according to Stat News.

Mar 09, 2017

Hemophilia Genetic Testing Program Opens Data Repository for Research Applications in US

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

Feb 13, 2015

Alnylam Aims to Release Multi-program Clinical Data in 1H 2015

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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.

Dec 10, 2014

Alnylam Releases Preliminary Phase I Data on Hemophilia Drug

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

Nov 06, 2014

Alnylam Aims Broadly with Hemophilia Drug, Advances HBV Program toward Clinic

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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

Oct 09, 2014

Alnylam Sees a Return to Big Indications amid Advances in Rare Diseases

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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

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The Scan

This Time, Novavax

Novavax has begun a phase III trial of its SARS-CoV-2 vaccine, according to the New York Times.

Shortened Visas for Some Students

Vox reports that the Trump Administration may limit student visas for individuals from some countries to two years.

New York's Own Review

The governor of New York says the state will conduct its own review of any SARS-CoV-2 vaccine, NPR reports.

Science Papers Examine Y Chromosomes of Archaic Humans, Schistosomiasis Therapeutic Targets

This week in Science: Neanderthal Y chromosomes replaced by Homo sapiens Y chromosomes, and more.

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