BioSpace

FDA’s Bespoke Pathway To Focus on Gene Editing and RNA-Based Treatments for Rare Diseases

The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay ...

FDA proposes new system for approving customized drugs and therapies for rare diseases

The FDA guidelines aim to create a new pathway for bespoke therapies due to the challenges of conducting large studies.
MedPage Today on MSN

FDA Proposes New Approval Pathway for Rare Disease Gene Therapy

Will consider data that includes "the experience of individuals," FDA commissioner says ...
Precision Medicine Online

FDA Expects Plausible Mechanism Pathway to Speed Approval Times of Rare Disease Genetic Medicines

The FDA released a much-anticipated draft guidance on how makers of rare disease genetic medicines can leverage a platform ...

FDA reverses course, will review Moderna's mRNA flu vaccine

Moderna said it met with the FDA on Tuesday to discuss the vaccine and afterward, the U.S. regulator accepted a revised ...

The Munich Security Conference; FDA Rejects Flu Vaccine; The Fall of The Quad God

We get the latest from the Munich Security Conference, where U.S. Secretary of State Marco Rubio delivered a speech before European leaders. We also look at the reasons behind why the FDA rejected ...