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Beam Gets Important Proof Of Concept For AATD Therapy

 
• By Alaric DeArment

The company announced early proof-of-concept data for BEAM-302, its base-editing medicine for the genetic liver and lung disease alpha-1 antitrypsin deficiency, showing a clean safety profile.

DNA bases
• Source: Shutterstock

Beam Therapeutics sees BEAM-302 as having potential for eventual accelerated approval from the US Food and Drug Administration in alpha-1 antitrypsin deficiency (AATD) after announcing positive early data from its Phase I/II study of the therapy that provide an important proof of concept for the company’s base editing technology.

According to data released 10 March, the third dose level of 60mg achieved levels of mean total AAT that exceed the protective therapeutic threshold while reducing Z-AAT, the mutant form of the AAT protein

Key Takeaways
  • Beam announced promising early data for BEAM-302, its base editing therapy for AATD, showing a clean safety profile and signs of efficacy.
  • The company’s president said Beam sees potential for the therapy to qualify for accelerated approval, though more data are necessary

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