ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia — Stella

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ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia — Stella

Recruiting

Back to Beta-Thalassemia trials

Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(1 site)

United States

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

Key details

Phase

Early testing / Safety & dosing

Sponsor

Children's Hospital of Philadelphia

Enrollment target

~12 participants

Started

April 2025

Primary completion

December 2027

Age range

18 Years – 40 Years

Last updated April 2026

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