Tag Archives: rare disease

Orphan Drug Investors Discuss the Current Rare Disease Environment during RESI@JPM

21 Dec

By Cole Bunn, Senior Research Analyst, LSN


A large number of early stage biotechs are pursuing orphan disease indications.  This trend is driven by the large number of rare diseases with either no current treatment options or severely inadequate therapies as well as the unique regulatory/development advantages available for these therapeutics. However, orphan drugs also face some unique challenges, such as clinical trial recruitment, pricing, and small market sizes. Recent developments in the space should make for an interesting year to come with the new FDA head, Dr. Scott Gottlieb, planning to eliminate the backlog of orphan drug designation requests and the halving of the orphan drug tax credit.

In the highly anticipated RESI @ JPM conference on January 9, 2018, a varied group of investors active in the orphan/rare disease space will discuss the financing and business development environment for startups working on developing drugs for orphan indications.

Moderated by Ken Kengatharan, General Partner, Atheneos Capital panelists include:

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