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Alnylam’s RNAi Therapy for Acute Hepatic Porphyria Positive in Phase III

Cambridge, Mass.-based Alnylam Pharmaceuticals indicated that the company’s ENVISION Phase III trial of givosiran was positive for acute hepatic porphyria (AHP).

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AskBio Snags $235 Million Investment to Drive Gene Therapy Development

Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.

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FDA Approves ADMA’s Asceniv for Immune Deficiency

The U.S. Food and Drug Administration approved ADMA Biologics’ Asceniv to treat primary humoral immunodeficiency disease (PIDD or PI) in adults and adolescents.

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Dudnyk Joins Fishawack Group of Companies; Expands Service Offering and Global Reach

  March 20, Horsham, PA — Dudnyk leaders announce the next phase in its evolution as an ever-growing organization by joining The Fishawack Group of Companies. Fishawack is one of the world’s leading independent healthcare communication specialists, with offices in the UK (London, Oxford, Knutsford, Brighton), US (San Diego, Philadelphia), and India. “By joining forces […]

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Pfizer buys stake in French gene therapy firm Vivet

Pfizer acquired a 15 percent stake in Vivet Therapeutics as well as an exclusive option to fully acquire the business, a deal that will give the New York-based pharma giant access to the French company’s experimental gene therapy for a rare genetic disorder.

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Alnylam’s Givosiran Looks Positive Overall in Phase III Trial for Rare Disease

Despite releasing positive data on the company’s Phase III ENVISION trial, Alnylam Pharmaceuticals stock dropped 7.5 percent.

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EU panel approves drug for rare genetic disease

An advisory committee of the EMA recommended conditionally approving a drug from Akcea Therapeutics and Ionis Pharmaceuticals that aims to treat a rare genetic disease.

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Ipsen to buy Clementia Pharmaceuticals

French healthcare company Ipsen agreed to buy U.S. peer Clementia Pharmaceuticals in a deal worth up to $1.31 billion, helping to boost Ipsen’s portfolio of products treating rare diseases.

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Roche acquires Spark Therapeutics for $4.3 billion

Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.

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Catalyst Pharmaceuticals defends $375,000 drug price after Bernie Sanders rebuke

Catalyst Pharmaceuticals Inc. – rebuked by U.S. Senator Bernie Sanders for high drug prices – defended the company’s $375,000 treatment for a rare disease, saying the price was in line with similar products in the industry.

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