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San Rocco Therapeutics

Clinical trials sponsored by San Rocco Therapeutics, explained in plain language.

Upcoming readouts

Trials from this sponsor expected to report results within the next 12 months.

Gene fix for thalassemia: first human test shows promise Results expected Jul 2027 (estimated)

Browse trials soon ready across all sponsors →

Not yet finished but already full! 1

Gene fix for thalassemia: first human test shows promise

Disease control Ongoing

This early-stage study tests a gene therapy for adults with β-thalassemia major, a severe blood disorder requiring frequent transfusions. Doctors take the patient's own stem cells, fix the faulty gene, and return the corrected cells via an IV. A low dose of busulfan prepares the …

Phase 1 • Sponsor: San Rocco Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:23 UTC