Pharma Invests in Gene Therapy Programs

Published on: 
BioPharm International, BioPharm International-08-01-2013, Volume 26, Issue 8

Pharmaceutical companies have stepped up gene therapy development in the wake of the approval of Glybera in 2012.

When the European Medicines Agency granted marketing approval for uniQure's Glybera (alipogene tiparvovec)—the first gene therapy in Europe or the United States—in November 2012, it was a milestone for gene therapy research. The approval also encouraged investment and development activity by pharmaceutical companies, said Elemer Piros, managing director and senior equity research analyst for Burrill Securities, in a May 16, 2013 podcast, "Why Gene Therapy is Coming of Age," on

In recent months, pharmaceutical companies made the following announcements on their respective websites.

In July, uniQure announced a collaboration agreement with Chiesi Farmaceutici SpA (Parma, Italy) for the commercialization of Glybera. Chiesi recieves exclusive rights to commercialize Glybera in Europe and selected countries; uniQure retains commercial rights for the US, Japan, and parts of Latin America and Asia, and Australasia.


The US Food and Drug Adminstration (FDA) in June granted Breakthrough Therapy designation to GlaxoSmithKline's investigational compound drisapersen, an antisense oligonucleotide, for the potential treatment of patients with Duchenne Muscular Dystrophy (DMD). GSK's clinical development plan will evaluate the effect of drisapersen in ambulant and non-ambulant young male patients with DMD who have dystrophin gene mutations amenable to an exon 51 skip.

Oxford BioMedica signed an agreement with Novartis in May to manufacture clinical grade material using its LentiVector gene-delivery technology. Oxford BioMedica will manufacture several batches of a lentiviral vector encoding CTL019 technology, which will be used to transduce patients' T-cells in an ex vivo process before they are reinfused into patients. CTL019 targets a protein called CD19 that is associated with a number of B-cell malignancies.

Baxter International Inc. and Chatham Therapeutics, an affiliate of Asklepios BioPharmaceutical, Inc., will collaborate on the development and commercialization of potential treatments for hemophilia B. Baxter will investigate Chatham's Biological Nano Particles (BNP), a recombinant adeno-associated virus-(rAAV) based gene therapy technology that has shown potential therapeutic benefit in early clinical studies.

Ambrx and Bristol-Myers Squibb agreed in May to collaborate on the discovery and development of novel antibody drug conjugates using Ambrx's protein medicinal chemistry technology. Ambrx will receive an upfront payment of $15 million; funding for discovery and research activities; and potential development, regulatory, and sales-based milestone payments of up to $97 million per product resulting from the collaboration. Bristol-Myers Squibb will receive worldwide rights to develop and commercialize products resulting from the collaboration.

In March, AstraZeneca announced an upfront payment of $240 million to Moderna Therapeutics to discover, develop, and commercialize messenger RNA therapeutics. AstraZeneca will have exclusive access to select any target of its choice in cardiometabolic diseases, as well as selected targets in oncology, over a period of up to five years.

In February, BioMarin Pharmaceutical licensed a Factor VIII gene therapy program for hemophilia A from University College London and St. Jude Children's Research Hospital. The company reports that it expects to select a development candidate in 2013, initiate and complete IND-enabling toxicology studies next year, and initiate proof of concept human studies by the end of 2014.