Gene therapy made a commercial reality

At the beginning of April 2007 Oxford BioMedica plc signed a US$690 million deal with the Swiss-based multinational pharmaceutical company Sanofi Aventis for TroVax, a gene therapy-based cancer treatment.

This was a significant breakthrough, as it was the first time a major pharmaceuticals company had publicly embraced gene therapy.

2006 was an excellent year for UK gene therapy.

Professor Martin Gore

Chair

GTAC

Oxford BioMedica’s chief executive officer Alan Kingsman commented, “The financial terms are good and competitive, and to a significant extent that is because we had several [potential] partners at the table.”

Kingsman believes the licence with such a prominent partner will unlock the potential of gene therapy treatments for cancer.

Ark gets endorsements

Also in April, Ark Therapeutics Group plc received the endorsement of the European Medicines Agency (EMEA) for Cerepro, its gene therapy treatment for brain cancer.

In May, Ark’s technology received further backing when the US Recombinant DNA Advisory Committee gave clearance to a phase III trial of another gene therapy product, Trinam.

Ark had not expected a judgement until late in June and chief executive officer Nigel Parker says the rapid granting of the approval, “illustrated just how far gene-based medicine has advanced in the last year or two.”

The company now expects to get final approval of the protocol and start the 200-patient trial in the second half of 2007.

Committee provides clarity

There are complex ethical, clinical, technical and regulatory issues behind the premise of using a virus (or other vector) to deliver a beneficial gene.

Early on in the development of the technology, in 1993, the UK formed the Gene Therapy Advisory Committee (GTAC).

As the sole body that can approve gene therapy trials, the committee has provided clarity for companies working in the field.

The validation that Oxford BioMedica and Ark have provided will help other products through to commercialisation.

In the latest annual review Professor Martin Gore, chair of GTAC, says that “2006 was an excellent year for UK gene therapy”.

The most striking element was a marked increase in applications to develop gene therapy treatments for single-gene defects, which Gore attributes to new funding made available by the UK Government in 2003.

Validation helps other products through

GTAC reports there are 43 live gene therapy trials in the UK at present.

Like monoclonal antibodies before it, gene therapy has seen many ups and downs en route to market.

The validation that Oxford BioMedica and Ark have provided will help other products through to commercialisation.

Next step: stem-cell therapy

It is acknowledged that it will take some time to translate stem-cell research to new therapies.

However, the UK’s ability to bring gene therapy through to market shows the elements needed to support long development cycles in complex and novel technologies like stem cells are now in place.