OTL-300 / Kyowa Kirin - LARVOL DELTA

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL) (clinicaltrials.gov) - P=N/A | N=9 | Active, not recruiting | Sponsor: Orchard Therapeutics | Phase classification: P1/2 ➔ P=N/A

Phase classification • Beta-Thalassemia • Genetic Disorders

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL) (clinicaltrials.gov) - P1/2; N=9; Active, not recruiting; Sponsor: Orchard Therapeutics; Enrolling by invitation ➔ Active, not recruiting

Clinical • Enrollment closed • Beta-Thalassemia • Genetic Disorders

Orchard Therapeutics Announces Global License Agreements for Stable Cell Line Technology from GSK (GlobeNewswire) - "Orchard Therapeutics...today announced that the company has entered into two worldwide royalty-bearing license agreements with GlaxoSmithKline...for Wiskott Aldrich syndrome (OTL-103 for WAS) and transfusion-dependent beta thalassemia (OTL-300 for TDT)."

Licensing / partnership • Beta-Thalassemia

Orchard Therapeutics announces eight presentations across gene therapy portfolio at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting (GlobeNewswire) - "The presentations also include...full results of the clinical proof-of-concept clinical trial for OTL-300 for the treatment of transfusion-dependent beta thalassemia, which achieved its primary outcome measures;"

Clinical data • Anemia • Beta-Thalassemia • Hematological Disorders

A follow-on study in patients who have received gene therapy for Beta-thalassemia in the TIGET-BTHAL study to assess safety and effectiveness of treatment. Uno studio successivo su pazienti chehanno ricevuto terapia genica per la beta-talassemia nello studio TIGET-BTHAL per valutare la sicurezza e l'efficacia del trattamento (clinicaltrialsregister.eu) - P2; N=10; Ongoing; Sponsor: GLAXOSMITHKLINE RESEARCH AND DEVELOPMENT

Clinical • New P2 trial

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL) (clinicaltrials.gov) - P1/2; N=9; Enrolling by invitation; Sponsor: Orchard Therapeutics; Recruiting ➔ Enrolling by invitation

Clinical • Enrollment status

Orchard Therapeutics announces clinical proof-of-concept data for gene therapy OTL-300 demonstrating efficacy in transfusion-dependent beta-thalassemia (GlobeNewswire) - "Orchard Therapeutics...announced the presentation of the full clinical proof-of-concept data from its trial of...OTL-300, for the treatment of transfusion-dependent beta-thalassemia (TDT). The data will be featured today in an oral presentation at the 22nd American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Washington, D.C...All nine patients met the safety endpoint of survival with follow-up ranging from 16 to 43 months (3.6 years). No adverse events related to the product were reported."

P1/2 data

OTL-300: “8/9 patients with reduced or eliminated need for transfusions (5 pediatrics / 3 adults) at 12 months”; Transfusion-dependent beta-thalassemia (Orchard Therapeutics) - Corporate Presentation

P1/2 data

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL) (clinicaltrials.gov) - P1/2; N=9; Recruiting; Sponsor: Orchard Therapeutics; Phase classification: P2/3 ➔ P1/2; Trial primary completion date: May 2026 ➔ Dec 2025

Clinical • Phase classification • Trial primary completion date

Orchard Therapeutics Announces Presentation of Clinical Proof-of-Concept for OTL-300 for the Treatment of Transfusion-Dependent Beta-Thalassemia at the 22nd American Society of Gene & Cell Therapy Annual Meeting (GlobeNewswire, Orchard Therapeutics Limited) - "Orchard Therapeutics...today announced the achievement of clinical proof-of-concept based on data from the trial of its ex vivo, autologous, hematopoietic stem cell (HSC) gene therapy, OTL-300, for the treatment of transfusion-dependent beta-thalassemia (TDT). The data will be published online today in an abstract via the American Society of Gene & Cell Therapy (ASGCT) website, and the full data set will be featured in an oral presentation at the 22nd ASGCT Annual Meeting to be held on April 29 – May 2, 2019 in Washington, D.C."

Clinical data

OTL-300: Data from proof-of-concept trial for transfusion-dependent beta-thalassemia in 2019 (Orchard Therapeutics) - Corporate Presentation

Clinical data

OTL-300: “5/7 patients with reduced need for transfusions (4 pediatrics / 3 adults)”; Transfusion-dependent beta-thalassemia (Orchard Therapeutics) - Corporate Presentation

Clinical data