Tag: hemophilia

28
Mar 2022

Bionest

ASH 2021 Update: Part 2

A look at new hematology therapy developments for hemophilia, beta thalassemia and sickle cell disease presented at ASH 2021.
17
Sep 2018

Bionest

FDA Issues Set of Guidelines on Gene Therapy

Back in December, the U.S. Food and Drug Administration (FDA) granted its first approval for a gene therapy product, Spark Therapeutics’ Luxturna. According to the Alliance for Regenerative Medicine, as many as 50 more such treatments — addressing a diverse range of health problems from genetic, autoimmune, and heart diseases, to cancer and HIV/AIDS —...
25
Jun 2018

Bionest

First Up For The New FDA RMAT Designation: Gene Therapeutics for Hemophilia

We recently wrote about the U.S. Food and Drug Administration’s (FDA) creation of a Regenerative Medicine Advanced Therapy (RMAT) designation in an effort to speed up the approval process for certain types of treatments, including gene therapies. The agency has specifically chosen gene therapy for hemophilia as its first focus, due to the considerable effort...