Moderna and Myrtelle Chosen for FDA's Rare Disease Drug Development Initiative

Moderna and Myrtelle Chosen for FDA's Rare Disease Drug Development Initiative

Katarina Novákova
1 min read

Moderna and Myrtelle Chosen for FDA's START Pilot Program

Moderna and Myrtelle have been selected for the FDA's Strategic Targeted Research Acceleration Program (START) pilot initiative, which aims to accelerate the development of innovative drugs for rare diseases. Moderna is currently conducting Phase I/II clinical trials for mRNA-3705 to address methylmalonic acidemia, caused by a deficiency in methylmalonyl-CoA mutase, while Myrtelle's rAAV-Olig001 is being evaluated for Canavan disease, a fatal genetic disorder affecting brain white matter.

Key Takeaways

  • Moderna is developing mRNA-3705 for methylmalonic acidemia (MMA).
  • Myrtelle is developing rAAV-Olig001 for Canavan disease (CD).
  • Both Moderna and Myrtelle are part of the FDA's START program to accelerate rare disease therapeutics.
  • mRNA-3705 is in Phase I/II for MMA, and rAAV-Olig001 is in Phase I/II for CD.
  • The FDA's START program provides frequent advice to sponsors to expedite drug development.


The selection of Moderna and Myrtelle for the FDA's START program indicates a strategic shift towards expedited development of rare disease treatments. This move could significantly impact patients with MMA and CD, offering hope for improved therapies. It may also lead to increased regulatory support for biotech firms, influencing investment and R&D strategies. This collaboration could streamline drug approval processes, encouraging more biotech involvement in rare disease research.

Did You Know?

  • mRNA-3705: mRNA-3705 is an mRNA-based therapeutic drug that aims to treat MMA, a condition where the body's inability to break down certain proteins and fats leads to a deficiency in the enzyme methylmalonyl-CoA mutase.

  • rAAV-Olig001: Myrtelle's gene therapy, rAAV-Olig001, focuses on addressing CD by delivering a functional version of the ASPA gene, crucial for myelin synthesis and maintenance in the brain's white matter.

  • FDA's START Program: Launched in September 2023, this program provides regulatory advice and support to expedite the development of novel drugs for rare diseases, aiming to accelerate the approval of therapies with significant impact on patients' lives.

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