In Vivo Gene Therapy for Mainstream Diseases - Making Inroads Beyond the Rare and Orphan

The years 2018 and 2019 have provided a glimpse of the promising future of in vivo gene therapy. The US launches of LUXTURNA ® and ZOLGENSMA ® , and high profile acquisitions suggest this field will forge ahead into new indications and continue to break boundaries. The continued growth and change indicates that invested parties need to revitalize perspectives. A holistic view of the market, agencies and competitors is needed to truly optimize a portfolio and maintain a favorable market presence. We review where in vivo gene therapy stands today, examine the potential shift in indications, from monogenic to complex, and discuss why it is time to review market and portfolio analyses in the field.

Our conclusion is that gene therapy is going well beyond monogenetic disease to bigger indications such as Parkinson's and Ovarian Cancer. However, the targets these therapies are going after are not necessarily innovative or unique. Other mechanisms of therapeutic delivery may or may not be just as effective. The jump to larger indications is not yet clear at this time.