Industry News
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Infant with Rare Genetic Disorder Successfully Treated with Customized mRNA-based CRISPR Gene Editing Therapy at CHOP and Penn Medicine
May 15 2025 -
Genespire Presents Positive Preclinical Dosing Data for In Vivo Gene Therapy for Methylmalonic Acidemia
May 14 2025 -
Latus Bio Announces Novel AAV Capsid AAV-Ep+ Demonstrates Robust Brain Transduction and Therapeutic Protein Expression in Preclinical Studies
May 14 2025 -
Rgenta’s RSwitch Platform Reported to Regulate Gene Therapy Transgene Expression
May 13 2025 -
REGENXBIO Announces FDA Acceptance and Priority Review of BLA for RGX-121 Gene Therapy in Hunter Syndrome
May 13 2025 -
Sarepta Therapeutics’ Delandistrogene Moxeparvovec Receives Conditional Approval in Japan for Younger Duchenne Patients
May 13 2025 -
Arcturus Shifts Focus to mRNA Therapeutics to Propel Rare Disease Pipeline
May 13 2025 -
Beam Therapeutics Receives FDA RMAT Designation for BEAM-302 Base Editing Therapy for Alpha-1 Antitrypsin Deficiency
May 12 2025 -
Capsida Biotherapeutics Receives FDA Clearance for IND Application for CAP-002 Gene Therapy for STXBP1-DEE
May 12 2025