A syndicate of venture capitalists, with support from the European Regional Development Fund, has raised seed financing of €40 million for Aptadir Therapeutics Srl of Italy to support development of a new RNA therapeutic for difficult genetic disorders. The preclinical compound is being investigated for Fragile X syndrome, an inherited generic, neurological disorder.
The US Food and Drug Administration has approved a new small molecule drug for a rare genetic disease that can impair the supply of thyroid hormone to the brain whilst also leaving excessive levels of the hormone in the bloodstream. The drug, Emcitate (tiratricol), was developed by Egetis Therapeutics US Inc, and received multiple designations in the run-up to its approval on 28 September, including a Priority Review designation in March.
Sofinnova Partners SAS has created a new fund to build and support companies developing medical technologies with plans to launch six to eight new companies over the next five years. This is being enabled by an over-subscribed €82 million financing round announced on 28 September. While the target technologies haven’t been identified, the broad approach is to create companies from the ground up. Explaining this strategy, Antoine Papiernik, managing partner, said the investment group builds and supports “every company from day one.” These assets are then scaled up.
Regulatory Strategy
How the US encourages rare disease drug development
By Darex Vera-Rodriguez and Michael Salgaller
Obesity Drug Review
Change is coming to the weight loss sector
By Peter Charlish
Interview
The role played by DEBRA Research in translational medicine
Public Policy
New EU regulation on the use of artificial intelligence
Research News
Shingles vaccine may protect heart
By Rosie Bannister
