2minutemedicine.com5h
Beti-cel gene therapy achieves high rates of transfusion independence in severe β-thalassemia
There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: Transfusion-dependent β-thalassemia (TDT) is a condition that results in iron ...
precisionmedicineonline12h
Fondazione Telethon Seeking Approval for Wiskott-Aldrich Syndrome Gene Therapy in Europe
Thirty patients have received the gene therapy within a clinical trial and early-access program, and the foundation also hopes to seek approval in the US.
Viralgen and CTNNB1 Foundation partner to manufacture a new gene therapy for CTNNB1 Syndrome
Viralgen and the CTNNB1 Foundation have come together to manufacture an investigational gene therapy designed to treat CTNNB1 ...
ALS News Today14h
AMT-162 gene therapy trial for SOD1-ALS set for next dose group
Qure is planning soon to start enrolling patients in a second dose group as part of a clinical trial testing AMT-162 for SOD1 ...
Appointment of Joint Corporate Broker and Joint Financial Adviser
Oxford, UK – 5 February 2025: OXB (LSE: OXB), a quality and innovation-led cell and gene therapy CDMO, today announces the appointment of Jefferies International Limited ("Jefferies") as its Joint ...