Gene Therapy, Pharma

Top 20 players: Everybody loves an orphan — especially when they come with blockbuster sales and a long run of exclusivity

Rare diseases are a big business in the biopharma world. And they’re getting bigger every year.

Advantaged by regulatory groups in the US and the world over, attracted by lower research costs with smaller studies and the prospect of fielding a blockbuster with a small group of reps, biopharma has responded with a burst of new therapies. And now that the gene therapy era is upon us, we’re seeing the big players step up to buy a significant position for themselves in this booming market.

Evaluate’s Karen Pomeranz just wrapped up a deep dive on the topic, and concluded that we’ll continue to see dramatic growth in the field of orphan drugs as these trends continue to play out with a growing pot for the top players to draw from.


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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Associate Director CMC
Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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