After rocky beginnings, there is increasing optimism about the potential for gene therapy to make an impact in rare disease. Promising data from late-stage clinical trials, improved safety, and the first Western regulatory approval of a gene therapy are now driving numerous initial public offerings, venture capital financings, and tie-ups with major pharmaceutical companies.

While an increasing number of South Korean biotech firms are looking to advance into overseas gene and cell therapy markets as part of the government's plan to develop these areas as a new growth engine, a U.S. regulatory expert has cautioned it is risky for bioventures to focus only on the gene and cell sectors while they still face major regulatory and commercialization hurdles.

While an increasing number of South Korean biotech firms are looking to advance into overseas gene and cell therapy markets as part of the government's plan to develop these areas as a new growth engine, a U.S. regulatory expert has cautioned it is risky for bioventures to focus only on the gene and cell sectors while they still face major regulatory and commercialization hurdles.