Genzyme Forges Chinese Connection to Speed Gene Therapy to World Market

Genzyme (NASDAQ: GENZ) and China are probably the world’s biggest players in gene therapy, and now the two are working together, thanks to a deal announced last Friday around Genzyme’s lead gene therapy candidate.

The deal—with Shanghai Sunway Biotech—should put Genzyme’s gene therapy program on the fast track, but also into the midst of some controversy. While gene therapy has faced big setbacks here in the U.S., Chinese companies have moved aggressively into the field with support from their government. As a result, today China alone offers the only two approved gene therapies in the world. However, at least one of those two treatments, and possibly both, sprang not from Chinese innovation, but from copying U.S. research. And because the Chinese government both invests in and regulates its drug industry, there has been concern about how safe and effective these treatments really are.

Under the terms of the agreement, Genzyme’s lead gene therapy candidate, called Ad2/HIF-1a, will now be jointly developed by Genzyme and Sunway in China. (Genzyme will also continue to develop it on its own in the U.S.) The product promotes new blood vessel growth and is being tested in patients with circulation problems in their legs. The goal is to grow new vessels, thereby preventing the pain, disability, and need for amputation that sometimes arise from this condition. The plan is for Genzyme to tell Sunway how to make the product, so it can start pumping it out in China and testing it on patients there. If the trials succeed, the two companies “envision jointly commercializing a therapy in China,” according to a press release.

In the U.S., gene therapy research initially took off like a rocket in the 1990s after a child’s genetic immune-system disease was treated with an experimental gene therapy. But trouble soon followed, including the highly publicized death of a clinical trial volunteer in another study, which raised questions about oversight of gene therapy and other cutting-edge treatments. The field has also been hampered by worries that some methods of introducing new “good” genes into a sick person can accidentally trigger cancers.

As a result, gene therapy is now considered an extremely high-risk venture from a business standpoint here. Genzyme is one of a few companies that stuck with it, and one of the best positioned as well, with gene-therapy treatments in the works for, among other things, Parkinson’s disease, eye disorders, and genetic diseases. Most other gene therapy companies are small startups with no other products of any kind yet on the market. Several have gone out of business before their products got close to market.

China has in some ways proven to be more fertile ground for gene therapy, but its role in the field is controversial. Some observers feel the Chinese are more open-minded about experimental therapies and that they offer a new pathway for development of cutting-edge treatments, as this Nature news article suggests. But the Chinese government has potentially conflicting interests. Not only does it oversee the approval of new treatments, it is also trying to encourage a flourishing biotech industry and actually invests in some of these companies.

Questions about intellectual property rights are also swirling around. China’s first approved gene therapy was Gendicine, produced by Shenzhen SiBiono GeneTech in 2003. That treatment exploits a famous cancer-linked gene called p53. It is approved, only in China, to treat head and neck cancer. That’s a particularly tough-to-treat cancer, and the treatment is very expensive.

Austin, TX-based Introgen has been developing a similar product, though, and in a recent Washington Post article its CEO was quoted claiming the Chinese company stole Introgen’s idea. Notably, it took only about seven years to get Gendicine launched in China, whereas Introgen’s scientists have been laboring for about 15 years on their product, which has just completed phase 3 trials for head and neck cancer in the U.S.

Shanghai Sunway Biotech, Genzyme’s new partner, was also accused of stealing the idea for its first gene therapy product—Oncorine, another cancer treatment which China approved in 2005. Emeryville, CA-based Onyx stopped development of the product in 2003 and was caught by surprise when it discovered Sunway had “revived” and appropriated it by copying Onyx scientists’ published research. Sunway and Onyx eventually inked a licensing deal.

Sunway has thus done the right thing by doing the licensing deal first this time. As long as the Chinese gene therapy industry doesn’t run into any (further) troubles, the new deal could help usher a wave of gene therapy products to the clinic. The big question is whether the U.S. will open its doors to those products, or whether China will remain the only place in the world to get gene therapy.

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One response to “Genzyme Forges Chinese Connection to Speed Gene Therapy to World Market”

  1. Lesley Hartland says:

    My child has Infantile Tay-Sachs /The oldest with her disease 6 years4 months we are looking into Gene Therapy as you can imagine with great interest.Any information would be greatfully accepted .