Moderna (Nasdaq: MRNA) has announced that the US Food and Drug Administration has selected mRNA-3705 for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program.
mRNA-3705 is being developed for methylmalonic acidemia (MMA), a rare, inherited metabolic disorder which can lead to toxic acid buildup in the body, causing significant mortality and morbidity.
Currently, there are no approved therapies for the condition, with standard care including dietary and palliative measures. Liver or combined liver and kidney transplants are the only effective treatments available.
This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.
Login to your accountTry before you buy
7 day trial access
Become a subscriber
Or £77 per month
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed
Chairman, Sanofi Aventis UK
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze