Gene Therapy
18 Jul 2025
18 Jul 2025
Health Canada Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)
Drug ApprovalLicense out/in
License out/inPROTACs
Piston Bio Receives Endorsement from Leading Oncologists for its Tumor Antigen-Inducing Therapeutic
Medera and University of Kansas Medical Center Announce First Patient Treated in Pioneering Gene Therapy Trial for Duchenne Muscular Dystrophy Associated Cardiomyopathy
Gene TherapyAcquisitionFast TrackOrphan Drug
KALA BIO Announces Completion of Enrollment in CHASE Clinical Trial Evaluating KPI-012 for the Treatment of Persistent Corneal Epithelial Defect (PCED)
Fast TrackOrphan DrugClinical Result
Atsena Therapeutics Announces Alignment with FDA on Regulatory Pathway to Approval for ATSN-201 in X-Linked Retinoschisis (XLRS)
Gene TherapyOrphan DrugFast TrackClinical Study
License out/in
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