BioMedTracker is part of the Business Intelligence Division of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC's registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

Informa
Menu

Report Library

All Reports
Future Treatment Approaches for Rare Congenital and Genetic Diseases

September 08, 2020

Many biotech and pharmaceuticals companies have prioritized drug development for rare congenital and genetic diseases over the past few years given the high unmet need, rapidly advancing science, and favorable clinical development paths. Rare congenital and genetic diseases often have severe or even fatal manifestations, with few treatments available, but emerging genetic data and new treatment modalities, such as gene therapy, are rendering monogenic diseases more tractable. What’s more, regulatory and legislative initiatives such as Breakthrough Therapy designation, which makes it easier to work with the FDA on tailored trial designs, and the Orphan Drug Act, which provides seven years of regulatory exclusivity, have encouraged development in this space.

For the full report, please download the PDF version at the top of this page.
Back to the top Back to the top