Spark Therapeutics Inc. hit a $1.2 billion valuation on the biotechnology firm’s first day on the market, reflecting growing investor enthusiasm for the once-beleaguered field of gene therapy, a field that has produced the first $1 million drug. The treatments focus on diseases where a single mutation in the genetic code is known to be the cause. Fix or replace the genetic code, and, theoretically, the patient is cured.
Most of the current therapies in development are focused on eye disorders and hemophilia, because drugmakers haven’t figured out how to manufacturer large amounts of the viral vectors. In eye diseases, you only need a very small amount of drug and it’s localized, which gives you safety advantages. Hemophilia B is best suited for an initial proof of concept for systemic delivery because you only need a low dose to make a difference.
Spark, Avalanche and Applied Genetic Technologies Corp. are working on eye diseases, while UniQure and closely held Dimension Therapeutics are among the drugmakers tackling hemophilia. Pfizer Inc., the top U.S. drugmaker, and Biogen Idec Inc., the maker of multiple sclerosis drugs, have also joined the fray, signing pacts in the past two months to help develop hemophilia treatments.
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