Krisztina M. Zsebo, CEO of San Diego’s Celladon, went to the annual meeting of the American Society of Gene Therapy to bust some scientific myths about the experimental technology.
The conference is meeting this week in San Diego, and when I caught up with her before her talk yesterday, she wasted no time busting myths about the corporate side of gene therapy as well.
Zsebo rejected the notion that venture financing for gene therapy companies has completely dried up. She pointed out that Celladon, which is working on a gene therapy for heart failure patients, received $9 million in venture funding earlier this year and stands to get $5.6 million more if certain milestones are met. What’s more, Celladon is in discussions with several pharmaceutical companies and expects to announce a development partner in the near future, she said.
Celladon is not an anomaly, she noted. This month, Applied Genetic Technologies, a Florida company working on a gene therapy for age-related macular degeneration with Cambridge-based Genzyme, received $11.8 million in venture funding.
There’s no question the economic environment is difficult, she acknowledged. But, “if you have a solid program, a strong mechanism of action and strong data … there are potential investors out there.”
Not that Celladon is immune to the misfortunes of other gene therapy firms. Seattle’s Targeted Genetics, which faces possible bankruptcy, manufactured Celladon’s drug. But Zsebo said Celladon has stockpiled enough of the drug to complete its clinical trials, and is working with Sigma-Aldrich, a contract manufacturer based in St. Louis, to produce the commercial product.
Gene therapy involves using a healthy gene to replace a defective or missing gene to relieve symptoms and cure chronic disease. A benign virus is used as vector to carry the healthy gene into a patient’s cells.
Celladon’s gene therapy uses a virus to deliver a gene that encodes for the SERCA2a enzyme, a key factor in maintaining a healthy heartbeat that is deficient in heart failure patients. The gene therapy is administered directly to the heart through a catheter that