source
- Beam Therapeutics reports one-year data from early-stage AATD gene therapy trialBeam Therapeutics released new results from its early-stage gene-editing program targeting alpha-1 antitrypsin deficiency (AATD), a rare condition that can damage the lungs and liver. The data com…
- Ionis receives FDA approval for Zanvastro, the first treatment for Alexander disease, as Pfizer partners on new ADC programThe U.S. Food and Drug Administration approved Zanvastro, marking the first approved medicine for the rare and often fatal neurological disorder Alexander disease. Developed by Ionis Pharmaceutical…