EXG34217 / Elixirgen Therap - LARVOL DELTA

Clinical Use of ZSCAN4 for Telomere Elongation in Hematopoietic Stem Cells. (PubMed, NEJM Evid) - P1 | "Although definitive conclusions cannot be drawn from the two EXG34217-treated patients, these results warrant further investigation of CD34+ cells exposed to ZSCAN4 for treating TBDs. (Funded by Elixirgen Therapeutics; ClinicalTrials.gov number, NCT04211714.)."

Journal • Aplastic Anemia • Hematological Disorders • Infectious Disease • Musculoskeletal Pain • Pain • CD34

Elixirgen Therapeutics Publishes Early Results Showing Telomere Elongation in First Gene Therapy Trial for Telomere Biology Disorders (GlobeNewswire) - P1 | N=12 | NCT04211714 | Sponsor: Elixirgen Therapeutics, Inc. | "Elixirgen Therapeutics, Inc...today announced publication of encouraging early clinical data from an ongoing Phase 1/2 trial...demonstrating the first-ever successful sustained telomere elongation in two EXG-34217-treated patients with a telomere biology disorder (TBD). No treatment-related safety concerns were observed over a 24-month and 5-month period after EXG-34217 infusion....These data support further investigation into the potential of EXG-34217 to treat patients with TBDs as a potential therapeutic intervention for preventing or treating bone marrow failure."

P1 data • Genetic Disorders

Elixirgen Therapeutics Granted FDA Orphan Drug Designation for EXG-34217 for Treatment of Telomere Biology Disorders (GlobeNewswire) - "Elixirgen Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (TBDs). EXG-34217 is currently being studied in an ongoing Phase 1/2 trial (NCT04211714) for the treatment of patients with TBDs with bone marrow failure. Enrollment is currently open for participants aged 12 and older, regardless of gender or ethnicity."

Orphan drug • Trial status • Genetic Disorders

Elixirgen Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EXG-34217 for Treatment of Telomere Biology Disorders (GlobeNewswire) - "Elixirgen Therapeutics, Inc...today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to EXG-34217, a gene therapy for treatment of Telomere Biology Disorders (TBDs)....EXG-34217 is currently being studied in an ongoing Phase 1/2 trial...for the treatment of patients with TBDs with bone marrow failure."

FDA event • Genetic Disorders

Rollover Study From EXG-US-01 (clinicaltrials.gov) - P1 | N=12 | Enrolling by invitation | Sponsor: Elixirgen Therapeutics, Inc. | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Aplastic Anemia • Hematological Disorders

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Elixirgen Therapeutics, Inc. | Phase classification: P1/2 ➔ P1

Phase classification • Aplastic Anemia • Hematological Disorders

Rollover Study From EXG-US-01 (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Elixirgen Therapeutics, Inc.

New P1 trial • Aplastic Anemia • Hematological Disorders

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Elixirgen Therapeutics, Inc. | Trial completion date: Apr 2024 ➔ Apr 2027 | Trial primary completion date: Apr 2023 ➔ Apr 2027

Trial completion date • Trial primary completion date • Aplastic Anemia • Hematological Disorders

SUCCESSFUL INFUSION OF EXG34217 - AUTOLOGOUS CD34+ CELLS TREATED WITH EXG-001- FOR EX VIVO TELOMERE ELONGATION IN A PATIENT WITH A DYSKERATOSIS CONGENITA (ASPHO 2023) - P1/2 | "Objec ves: Based on this work, we translated this approach into a first-in-human clinical trial (NCT04211714), in which autologous CD34+ hematopoie c stem cells (HSC) were collected by pheresis over two days from an adult subject with dyskeratosis congenita a er mobiliza on with filgras m and plerixafor a er IRB approval and consent. This novel approach uses modified autologous stem cells in the absence of condi oning regimen or immunosuppression and is genotype and muta on independent which is highly desirable in this chemotherapy and radia on sensi ve popula on. These data support further inves ga on and transla on of telomere elonga on via ZSCAN4 exposure by EXG-001 in pa ents with telomere biology disorders as a poten al therapeu c interven on for the preven on or treatment of bone marrow failure."

Clinical • Aplastic Anemia • Hematological Disorders • CD34

Successful Infusion of Autologous CD34+ Cells Treated with Exg-001 for Ex Vivo Telomere Elongation in a Patient with a Dyskeratosis Congenita (TCT-ASTCT-CIBMTR 2023) - P1/2 | "Objectives: Based on this work, we translated this approach into a first-in-human clinical trial (NCT04211714), in which autologous CD34+ hematopoietic stem cells (HSC) were collected by pheresis over two days from an adult subject with dyskeratosis congenita after mobilization with filgrastim and plerixafor after IRB approval and consent...Maintenance of normal differentiation potential after treatment was demonstrated by colony forming unit studies to address the concern of effects on terminal differentiation (Figure 1B; EXG34217)... This novel approach uses modified autologous stem cells in the absence of conditioning regimen or immunosuppression and is genotype and mutation independent which is highly desirable in this chemotherapy and radiation sensitive population. These data support further investigation and translation of telomere elongation via ZSCAN4 exposure by EXG-001 in patients with telomere biology disorders as a potential therapeutic intervention..."

Preclinical • Aplastic Anemia • Gene Therapies • Hematological Disorders • CD34

Successful Ex Vivo Telomere Elongation with Exg-001 in a Patient with a Dyskeratosis Congenita (ASH 2022) - P1/2 | "We translated this work in a first-in-human clinical trial (NCT04211714), in which autologous CD34+ hematopoietic stem cells (HSC) were collected by pheresis from an adult subject with dyskeratosis congenita over two days after mobilization with plerixafor and filgrastim with IRB approval and consent...To address the concern of effects on terminal colony forming unit studies were performed and demonstrated maintenance of normal differentiation potential after treatment (Figure 1B; EXG34217)...This novel approach is genotype and mutation independent and uses modified autologous stem cells in the absence of conditioning regimen or immunosuppression which is highly desirable in this radiation and chemotherapy sensitive population. These data support further investigation and translation of telomere elongation via ZSCAN4 exposure by EXG-001 in patients with telomere biology disorders as a potential therapeutic intervention for the prevention or treatment of bone..."

Preclinical • Aplastic Anemia • Gene Therapies • Hematological Disorders • CD34

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure (clinicaltrials.gov) - P1/2; N=12; Recruiting; Sponsor: Elixirgen Therapeutics, Inc.; Not yet recruiting ➔ Recruiting

Enrollment open • Aplastic Anemia • Hematological Disorders

Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure (clinicaltrials.gov) - P1/2; N=12; Not yet recruiting; Sponsor: Elixirgen Therapeutics, Inc.; Initiation date: Mar 2020 ➔ Sep 2020

Clinical • Trial initiation date • Aplastic Anemia