Cell Therapies - Gene Editing

Gene and RNA therapies for rare diseases have opened a vast field of potential breakthroughs

…if and when regulatory approval is ultimately granted

The selection - a limited sample in a specialized field of research - will be updated as more information becomes available

The large healthcare firms – often strong contenders in rare disease R&D in their own right – such as AbbVie , Astra Zeneca , Bayer ,  Eli Lilly , Gilead Sciences , Johnson and Johnson , Novartis , Pfizer , Regeneron Pharmaceuticals  or Roche Holding – have been set aside from this selection limited to smaller market caps

Success stories in the biotechnology space, such as Vertex or Alnylam , are included

Saddled with uncertain outcomes, most firms in the theme have had a poor performance record over the past year

Identify turn-around stories early on by tracking their performance over very short periods (from 2 weeks on, by selecting your preferred time frame on the drop-down menu, top right)

Insights on gene and RNA therapy collaborations with global healthcare firms can be found in the companion theme "Healthcare - Collaboration in CRISPR Expertise"