Lead Product(s): QR-421a
Therapeutic Area: Genetic Disease
Highest Development Status: Phase I/ Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 31, 2020
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events.