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Amicus Therapeutics’ Gene Therapy for Rare, Fatal Childhood Disease Shows Promise in Study

Amicus Therapeutics announced positive interim results from the company’s CLN6 Batten disease gene therapy program.

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Amicus and Penn Broaden Gene Therapy Collaboration

Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.

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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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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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Amicus Therapeutics Buys Celenex

Amicus Therapeutics inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex.

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FDA Requests Another Trial for Pharming’s Ruconest Label Expansion

The U.S. FDA issued a Complete Response Letter to Netherlands-based Pharming Group for the company’s supplemental Biologics License Application for Ruconest.

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FDA Shaves Four Months Off Amicus’ Fabry Drug Review Date

The FDA accepted Amicus Therapeutics’ NDA for migalastat to treat Fabry disease in patients with amenable mutations.

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Three Small CRISPR Biotechs That Could Cure 10,000 Diseases

Investor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.

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Shire’s Hunter Syndrome Treatment Fails Study Goals

Shire’s experimental treatment SHP609 to address cognitive impairment in pediatric patients diagnosed with the rare disease known as Hunter syndrome failed a key trial.

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Sangamo Scores Bragging Rights for First-in-Human Gene Editing Trial

Barely a week after announcing it was moving into a new corporate headquarters, Sangamo Therapeutics announced that the company has dosed the first patient in its Phase I/II gene therapy trial.

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