BioMarin up on positive gene therapy data in hemophilia A : fiercebiotech

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BioMarin up on positive gene therapy data in hemophilia A

BioMarin up on positive gene therapy data in hemophilia A

BioMarin Pharmaceutical ($BMRN) gained almost $1 billion in market cap on positive data for its gene therapy to treat severe hemophilia A.If approved, the company expects that BMN 270 could deliver more than $1 billion in annual revenue.The company expects that BMN 270 will be one of "four pillars of growth" that will drive BioMarin to profitability next year.


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Pfizer to help Western Oncolytics move gene therapy through Phase I trials

Pfizer to help Western Oncolytics move gene therapy through Phase I trials

Pfizer plans to help Western Oncolytics move its gene therapy through Phase I clinical trials — and it could buy the company if those trials go well.Under the agreement, Pfizer will work with the small Westlake-based company to complete animal trials and Phase I clinical trials.Afterward it would have the exclusive option to acquire the drug, according to a news release from the two companies.


besides eurekalert

Penn study verifies human gene therapy in model of rare metabolic disorder

Penn study verifies human gene therapy in model of rare metabolic disorder

PHILADELPHIA -- Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I.It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in cells.MPS I eventually leads to the abnormal accumulation of sugar debris and cell death.