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RegenxBio and Pfizer Partner on Gene Therapy

Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.

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Silence Therapeutics and Mallinckrodt announce RNAi therapeutics collaboration

Mallinckrodt plc and Silence Therapeutics plc announced a collaboration that will allow the companies to develop and commercialize RNAi drug targets designed to inhibit or ‘silence’ the complement cascade, a group of proteins that are involved in the immune system and that play a role in the development of inflammation.

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Study confirms heart benefit of soy as FDA reviews this claim

U.S. regulators should continue to recommend soy protein as part of a heart-healthy diet because it helps lower cholesterol, researchers conclude from an analysis of dozens of studies done in the past two decades.

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Genentech Strikes Three Drug Discovery Deals

In a deal-making morning for South San Francisco-based Genentech, the Roche subsidiary struck three separate transactions with small companies to boost drug development.

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FDA Approves Symdeko to Treat Underlying Cause of CF in Children Ages 6-11 Years with Certain Mutations in CFTR Gene

The U.S. FDA approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the Vertex Pharmaceuticals Inc. medicine.

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Research Roundup: A New Protein Linked to Alzheimer’s and Other Highlights

Researchers at the RIKEN Center for Brain Science identified a new protein that might make the connection between amyloid plaques and tau in Alzheimer’s patients.

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Rare Cancer Treatments and the Power of Checkpoint Inhibitors Among Hot Topics at ASCO

With the books closed on another ASCO meeting, it’s clear to see that checkpoint inhibitors keep showing a promise.

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Moderna Reports Influenza Vaccine Study Results

Cambridge, Mass.-based Moderna announced the publication of results from two Phase I clinical trials of mRNA vaccines against two strains of influenza, H10N8 and H7N0.

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AstraZeneca-Daiichi breast cancer treatment shows promise in latest study

An experimental breast cancer drug being developed by British drugmaker AstraZeneca and Japan’s Daiichi Sankyo’s met the main goal in a mid-stage study, bolstering the companies’ position in a highly competitive oncology market.

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Waylivra Approved In EU For Treating FCS

Akcea Therapeutics Inc. and Ionis Pharmaceuticals Inc. announced that Waylivra received conditional marketing authorization from the European Commission as the only therapy for FCS.

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