Boston is a modern biotech Milan where old and young pharmaceutical companies have established fashion runways to attract the eyes and wallets of investors with their biotech wares. I will focus my attention on the Gene Therapy runway. And what a runway it is, so much potential and so much drama. Gene therapy is an old idea from the 70s(1) that finally made it to clinical trials in the early 90s(2) when 2 girls were treated for Adenosine Deaminase – Severe Combined Immunodeficiency (ADA-SCID). (1, 2, 3) The early triumphs of gene therapy however were shattered by the death of a young boy in 1999, an X-linked severe combined immunodeficiency (XSCID) patient. (4) Gene therapy hit an ice age, the future was in jeopardy. Today as a result there is just a single drug approved and only in Europe [2012, Glybera (alipogene tiparvovec) for lipoprotein lipase deficiency (LPLD) by UniQure]. The price for being ‘the only couture therapy’, is also high, 1.1 millions Euro per patient (5,6).
I will concentrate my attention on 12 US companies (Table 1, Map) that are today the supermodels of the gene therapy fashion show in and around my hometown of Boston.
Gene therapy has blossomed in Boston regardless of bad publicity and the dark ages of 1999-2007. During this period, gene therapy was not a ‘safe field’ to invest in. That maybe the reason why Sarepta (1980), Bluebird Bio (1992), Advantagene (1999), Alnylam (2002) needed almost 15~30 years until they actually became successful. In fact, both Sarepta (formerly AVI BioPharma) and Bluebird bio (formerly Genetix Pharmaceuticals) underwent name changes just to get ready for stardom. The golden age of gene therapy begin after 2006. Companies like Dicerna (2007) and later Hemera (2010), Moderna (2010), RXi (2011), RaNA (2012), Dimension (2013) and Voyager (2014), hit the runway and brought their drug candidates for testing in clinical trials. In a relatively short span Alnylam, Dicerna, Bluebird Bio, RXi and Sarepta have all gone public (Table 2). While other companies, like Moderna, are probably en route to their own IPO(s).
The early successes of gene therapy were arguably serendipitous. They remind me of my baby, when he rolled over for the first time; one day out of the blue, he rolled over onto his tummy. My husband and I got very excited, days passed and he never turned again. Weeks later his muscles were strong enough to allow him to turn in earnest, since then he has not stopped rolling. That’s what happened with gene therapy. It needed almost 10 years before ‘the muscles’ of gene therapy: molecular biology techniques, development of nano vectors, easy access to the human genome, bioimaging; gained enough strength to sustain the demands of modern drug design. It was necessary for an army of techniques to be developed in order to support this novel platform that promises to change ‘the way’ we think about drugs. Targeted therapy at the site of the disease was an early selling point before personalized medicine could tailor drugs to specific patient needs. 21st century drugs are at nanoscale and we need to think about drug delivery and challenges from a nanoscale perspective for the paradigm shift to occur.
Today, the potential of gene therapy is recognized and recompensed by government, venture capital and private foundations.
This post is just a brief introduction to the Bostonian companies that develop products based on gene therapy, next post will analyze the vectors and gene payloads that make gene correction possible and bring hope to improving patient lives.
References
(1) http://www.ncbi.nlm.nih.gov/pubmed/5061866
(2) http://www.sciencemag.org/content/256/5058/808
(3) http://primaryimmune.org/first-gene-therapy-patients-attend-conference
(4) https://en.wikipedia.org/wiki/Jesse_Gelsinger
(6) http://news.yahoo.com/exclusive-first-gene-therapy-drug-sets-million-euro-173717146–finance.html