Insights

BioHeights has launched a new tool that allows to forecast revenue for an investigational therapeutic. In the main menu, go to Tools – Revenue Forecast Calculator. Why should you give it a try? Building your own drug revenue forecast model from scratch is time consuming. Instead, you can test your product and market assumptions using…

The First CAR T Patient Story Revisited The only child’s cancer diagnosis, remission and then relapse, oncology ward, remission again, lost cure, relapse again, hospice recommendations, coma, long coma – these were 2 years in the life of Tom Whitehead seven years ago. Tom is the father of the first child who received CAR T…

Two anti-CD19 CAR T cell therapies are currently approved by the FDA to treat hematologic malignancies: Kymriah and Yescarta. Both therapies have the same mechanism of action and treat relapsed/refractory B-cell malignancies such as acute lymphoblastic leukemia (ALL) and large B-cell lymphoma (LBCL). Click to zoom in. Anti-CD19 CAR T Race to Market The infographic…

On June 2, I attended the Entrepreneurial Bootcamp at the BIO Convention in Philadelphia. The Keynote speaker was Jeff Marrazzo, the CEO of Spark Therapeutics. The goal of his talk was to tell us about the early days of Spark, when it was the company of 1 (or of 1.5). Building a biopharma/biotech startup from…

The American Society of Gene and Cell Therapy (ASGCT) annual meeting on Apr 28 – May 2 in Washington D.C. was superb. It brought together several thousands attendees. Prof. Steven Gray wittily noted that the number of attendees was similar to the number of orphan diseases. Hence, each of us should get to work. Arguably,…

This article discusses diseases targeted in AAV gene therapy clinical trials initiated since 2003 through January 2019. It includes interventional, long-term follow up, and some observational studies. The review also covers industry sponsor involvement, volume of trials in each disease, and most advanced clinical development achieved in each indication to date. As of January 2019,…