From Care to Cure

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Gene therapy is coming of age, offering hope to millions of patients, and offering opportunities for those eager to invest in innovation for health.

AMT is now well positioned to address the urgent needs of many patients suffering from diseases for which no or suboptimal therapeutic solutions exist today. Many, but not all of these diseases are rare, are “orphans”. People affected by these diseases have little prospect of seeing a novel therapy because current drug development processes require too much time and resources to provide pharmaceutical companies with a sufficient return on their investment.

Gene therapy, and the flexibility of AMT’s platform, offer the hope of bringing more curative or causal therapies to patients faster. Furthermore, although orphan drugs target a smaller patient population than non-orphan drugs, “rare” is a relative term. In the United States alone, according to the National Institutes of Health (NIH) as many as 25 million people are affected by one of the 5,000 orphan disease currently diagnosed. This makes orphan diseases – and finding real therapeutic solutions for them – a serious public health concern.

Over the past decade it has become obvious that developing safe and efficacious therapies for orphans may also lead to substantial revenue generation. The best selling drug developed for an orphan indication is Erythropoetin (EPO). EPO has been subsequently expanded to target a number of indications, and in 2008 Amgen sold $ 5.6 billion of EPO products.