Pharma and Biotech M&A in Gene & Cell Therapy: What’s Next in Deal Making in the Emerging Advanced Therapeutics Space?


  • Ed Saltzman, Executive Chairman, Defined Health
  • Michael C. Rice, MS, MBA, Principal, Defined  Health

Novartis’ recently announced $8.7 bln acquisition of the gene therapy company AveXis for their spinal muscular atrophy therapy which is presently in pivotal trials for SMA Type 1 is a bold statement that after years of watching passively from the sidelines, big pharma may at last be ready to embrace advanced therapeutics.

Gene therapy is one of many platforms that make up the emerging field of advanced therapeutics, which is perhaps best understood by what it is not, e.g. traditional “off-the-shelf” products based on conventional concepts of small molecules or biologicals. As such, the field stretches to encompass not only gene therapy but gene editing, cell therapies (both autologous and allogeneic) especially the emerging and increasingly crowded field of CAR-T’s as well as the RNA-based therapeutics space. Though therapeutic applications are as diverse as the underlying science and target both large heterogenic patient populations as well as extremely rare and monogenetic diseases, these approaches share common challenges with delivery platforms, manufacturing complexity, distribution and pricing especially as many, if not most, programs are aiming at cure.

Will more big pharma companies follow Novartis’ lead despite the still untested and uncertain business model challenges posed by advanced therapeutics? This talk will look at the promise and the hurdles of the next phase of therapeutic evolution in Pharma.