Clinical Trials Arena June 6, 2024
Moderna is developing mRNA-3705 for methylmalonic acidemia (MMA), and Myrtelle is developing rAAV-Olig001 for Canavan disease (CD).
Moderna and Myrtelle’s rare programmes will both be included in a pilot programme by the US Food and Drug Administration (FDA) that aims to accelerate the development of novel drugs and biological products for rare diseases.
Announced in September 2023, the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) programme aims to expedite new drug development by providing sponsors with frequent advice and regular communication with the FDA to advance their programme as quickly as possible.
Moderna’s START programme evaluates mRNA-3705, an investigational therapeutic for methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency. The candidate is being investigated in a Phase...
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