Big Pharma bets big on gene therapies: 3 notes

In the last two months several pharma giants announced or completed deals to expand their portfolio of gene therapy treatments.  

The recent flurry of acquisitions and partnerships reveals that Big Pharma wants in on the fast-growing, potentially lucrative gene therapy market.

A breakdown of recent deals:

1. Pfizer agreed to pay $636 million for a 15 percent stake in Vivet Therapeutics March 20, according to The Wall Street Journal. Vivet's most-developed therapy is for Wilson disease, a rare liver disorder that causes copper buildup. Unless treated with a liver transplant, the disease is fatal. Pfizer has also made other strides into the gene-editing space. Rather than acquiring the companies outright, the drugmaker has struck partherships that grant it the rights to specific programs at smaller companies. For example, it is working on a gene therapy for hemophilia A with Sangamo Therapeutics.

2. Roche announced it would buy gene therapy developer Spark Therapeutics for about $4.8 billion in cash Feb. 25. The deal will give Roche access to a gene therapy for blindness priced at $850,000 per patient, as well as other projects for hemophilia and neurodegenerative disorders like Huntington's disease.

3. Biogen announced its intent to acquire Nightstar Therapeutics for about $800 million in early March. Nightstar Therapeutics is a clinical-stage gene therapy company focused on adeno-associated virus treatments for inherited eye disorders. Nighstar's lead asset is for the treatment of choroideremia, a rare degenerative retinal disorder that causes blindness.

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