Genzyme announced last week that it signed a collaboration deal with Chinese biotech firm Sunway to run clinical trials for—and, if successful, commercialize—the experimental gene therapy Ad2/HIF-1a in China.
Sunway is one of just two companies, both Chinese, to have successfully commercialized a gene therapy product. (Its H101, for the treatment of head and neck cancer, was approved in China in 2005.) Ad2/HIF-1a is an engineered form of the HIF-1a gene employing an adenovirus-based delivery vector. It was developed by Genzyme and is part of a large ongoing study being conducted in US and European medical centers.
As part of the agreement, Genzyme will transfer the technology to Sunway, which will reproduce the product in its facility in China and will take the lead in working with regulatory agencies in China to set up and carry out the clinical trials through Phase II. If the therapy is successful and Genzyme wants to continue into Phase III in China, then Genzyme envisions that the two companies will jointly fund a late-stage-development program and will jointly manage commercialization of the therapy, Genzyme spokesperson Bo Piela told Pharm Exec on Tuesday.
Parallel US Development However, this agreement would pertain only to China—not the United States, where a gene therapy has yet to be approved by FDA for treatment in humans. Genzyme says that there is a parallel US development program ongoing for a slightly different indication. HIF-1a treats peripheral arterial disease (PAD), a disease in which there is not enough oxygen flowing to the limbs. The therapy was designed to promote the growth of blood vessels. It is yet to be determined whether the therapy is effective.
"The principal aim for us is to work with another company that is a leader in the gene therapy field, and it allows us the ability to be part of the Chinese biotech industry," Piela said. "Genzyme has been working on gene therapies since the early '90s and is one of the few companies that is still strongly committed to the field and believes that if it's patient and continues to make the investment, then it will see products approved."
Sunway executives were in Cambridge, MA, last week learning more about how the therapy is made and how it works. The collaboration with Sunway is the first step for Genzyme to participate in the Chinese biotech industry. The company has offices in Shanghai and Beijing and is in discussions with companies for additional collaborations.
Though more than 300 INDs for gene therapies have been filed in the United States and a number of compounds are in Phase II development, progress has been slow due to several research setbacks, including a death that in 2003 led the agency to halt all gene therapy trials of products employing retroviral vectors to insert genetic material into cells.