RP-L102 / Rocket Pharma - LARVOL DELTA

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials (ASH 2024) - "Peripheral blood (PB) mononuclear cells were collected via leukapheresis after mobilization with granulocyte colony-stimulating factor (G-CSF) and plerixafor...Patients are being followed for safety and efficacy (increasing vector copy number [VCN], mitomycin-C [MMC] resistance in BM colony forming cells [CFCs]), and stabilization of cytopenias for 3 years in both the parent study and subsequently in a long-term follow-up (LTFU) study.Results : As of September 2023, 14 patients aged 1 to 6 years at enrollment have undergone successful apheresis and treatment with fanca-cel without significant complications...Fanca-cel has been well-tolerated with no significant safety signals. The risk-benefit profile of fanca-cel appears favorable."

Clinical • Gene therapy • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Myelodysplastic Syndrome • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Respiratory Diseases • Thrombocytopenia • CD34 • FANCA

Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A], is Not Associated with Insertional Mutagenesis (ASGCT 2024) - "Sequential ISA in patients treated with RP-L102 for up to 7.7 years demonstrated no insertional oncogenesis. These data contribute to the continuing favorable overall benefit:risk profile of RP-L102 as an ex vivo LV gene therapy for treatment of FA-A."

Gene therapy • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Oncology • Transplantation • CD34 • FANCA

Gene Therapy for Fanconi Anemia, Complementation Group A (clinicaltrials.gov) - P2 | N=5 | Active, not recruiting | Sponsor: Rocket Pharmaceuticals Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2023 ➔ May 2026 | Trial primary completion date: Jun 2022 ➔ May 2026

Enrollment closed • Gene therapy • Trial completion date • Trial primary completion date • Viral vector • Anemia • Gene Therapies • Hematological Disorders • Pediatrics • CD34

Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia (Businesswire) - "Rocket Pharmaceuticals, Inc...announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102, its lentiviral (LV) vector-based investigational gene therapy for Fanconi Anemia (FA), complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations....MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial....The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024."

BLA • European regulatory • Anemia • Genetic Disorders

Lentiviral-Mediated Gene Therapy for Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical Trials (ASGCT 2023) - "Patients are followed for 3 years for safety assessments (including insertion site analysis [ISA]) and for evidence of efficacy (increasing vector copy number [VCN], mitomycin-C [MMC] resistance in BM colony forming cells [CFCs]), and stabilization/correction of cytopenias). RP-L102 conferred phenotypic correction as demonstrated by sustained increase in BM CFC MMC resistance, genetic correction and hematologic stabilization in at least 6 patients with ≥1 year of follow up. Sustained engraftment, phenotypic correction, and hematologic stability was achieved in the absence of conditioning. RP-L102 represents a potentially curative therapy for FA-related BMF, which can be administered without transplant-conditioning related toxicities."

Clinical • Gene therapy • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hematological Malignancies • Lymphoma • Oncology • Transplantation • CD34 • FANCA

LENTIVIRAL-MEDIATED GENE THERAPY FOR FANCONI ANEMIA [GROUP A]: RESULTS FROM RP-L102 CLINICAL TRIALS (ASPHO 2023) - "Patients are followed for 3 years for safety assessments (including insertion site analysis [ISA]) and evidence of efficacy (increasing vector copy number [VCN], mitomycin-C [MMC] resistance in BM colony forming cells [CFCs]), and stabilization/correction of cytopenias). RP-L102 conferred phenotypic correction as demonstrated by sustained increase in BM CFC MMC resistance, genetic correction and hematologic stabilization in at least 6 patients with ≥1 year of follow up. Sustained engraftment, phenotypic correction, and hematologic stability was achieved in the absence of conditioning. RP-L102 represents a potentially curative therapy for FA-related BMF, which can be administered without transplant-conditioning related toxicities."

Clinical • Gene therapy • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hematological Malignancies • Lymphoma • Oncology • Transplantation • CD34 • FANCA

LENTIVIRAL-MEDIATED GENE THERAPY FOR PATIENTS WITH FANCONI ANEMIA [GROUP A]: UPDATED RESULTS FROM GLOBAL RP-L102 CLINICAL TRIALS (EBMT 2023) - P1, P2 | "Patients are followed for 3 years post-infusion for safety assessments (including replication competent lentivirus [RCL] and insertion site analysis [ISA]) and for evidence of efficacy (increasing PB and BM vector copy number [VCN] and mitomycin-C [MMC] resistance in BM colony forming cells [CFCs]), along with stabilization/correction of cytopenias. RP-L102 conferred phenotypic correction as demonstrated by sustained increase in BM CFC MMC resistance, concomitant genetic correction and hematologic stabilization in at least 6 patients with ≥1 year of follow up. Sustained engraftment, phenotypic correction, and hematologic stability was achieved in the absence of conditioning. RP-L102 represents a potentially curative therapy to prevent FA-related BMF, which can be administered without the need for a suitable allogeneic donor or transplant-conditioning related toxicities."

Clinical • Gene therapy • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hematological Malignancies • Lymphoma • Oncology • Transplantation • CD34 • FANCA

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A (clinicaltrials.gov) - P2 | N=7 | Active, not recruiting | Sponsor: Rocket Pharmaceuticals Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Jan 2023 ➔ Feb 2025 | Trial primary completion date: Jan 2023 ➔ Feb 2025

Enrollment closed • Gene therapy • Trial completion date • Trial primary completion date • Viral vector • Anemia • Gene Therapies • Hematological Disorders • Pediatrics • CD34

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) (Businesswire) - "Rocket Pharmaceuticals...today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022....Details for Rocket's Invited Talk and poster presentations are as follows: Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)...Presenter: Donald B. Kohn, MD..."

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results (ASH 2021) - P1/2 | "HSCs are collected via apheresis after mobilization with granulocyte-colony stimulating factor (G-CSF) and plerixafor and transduced with Chim-CD18-WPRE-LV. Myeloablative therapeutic drug monitoring (TDM) busulfan conditioning precedes RP-L201 infusion...The safety profile of RP-L102 remains highly favorable with no serious adverse events (SAEs) attributed to the investigational product (IP). RP-L201 leads to durable neutrophil CD18 expression and improved clinical course. Additional patient treatment is ongoing in 2021."

P1/2 data • Preclinical • Bone Marrow Transplantation • Gene Therapies • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Pediatrics • Primary Immunodeficiency • Rare Diseases • Transplantation • CD34

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials (TCT-ASTCT-CIBMTR 2023) - "Pts are followed for 3 years post-infusion for safety assessments (including replication competent lentivirus [RCL] and insertion site analysis [ISA]) and for evidence of efficacy (increasing PB and BM vector copy number [VCN] and mitomycin-C [MMC] resistance in BM colony forming cells [CFCs]), along with stabilization/correction of cytopenias. Comprehensive efficacy was identified in 5 of 9 pts with ≥1 year of follow up as demonstrated by increasing BM CFC MMC resistance, concomitant genetic markings and hematologic stabilization. Progressive engraftment of gene-corrected cells was identified in the absence of antecedent conditioning and attendant risks. Updated safety and efficacy data for pts with ≥12 months of follow up will be presented."

Clinical • Gene therapy • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Oncology • Pediatrics • Transplantation • CD34 • FANCA

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials (ASH 2022) - "Peripheral blood (PB) mononuclear cells are collected via leukapheresis on 2 consecutive days after mobilization with granulocyte-colony stimulating factor (G-CSF) and plerixafor...Patients are followed for 3 years post-infusion for safety assessments (including replication competent lentivirus [RCL] and insertion site analysis [ISA]) and for evidence of efficacy (increasing PB and BM vector copy number [VCN] and mitomycin-C [MMC] resistance in BM colony forming cells [CFCs]), along with stabilization/correction of cytopenias... Comprehensive efficacy was identified in 5 of 9 patients with ≥1 year of follow up as demonstrated by increasing BM CFC MMC resistance, concomitant genetic markings and hematologic stabilization. Progressive engraftment of gene-corrected cells was identified in the absence of antecedent conditioning and attendant risks. Updated safety and efficacy data for patients with ≥12 months of follow up will be presented."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hematological Malignancies • Oncology • Solid Tumor • Transplantation • CD34 • FANCA

Hematopoietic and Immunological Assessment in Fanconi Anemia after Ex Vivo Lentiviral FANCA Gene Therapy with RP-L102 (ASH 2022) - "Although our results are based on a small cohort and are interim within ongoing clinical trials, they indicate the need for more thorough assessment of BM and immune characterization in FA patients beyond reliance on blood counts. Importantly, the paucity of HSCs in FA patients even prior to the development of cytopenias supports the need for early intervention for HSC preservation which may be possible with GT upon diagnosis. Additionally, these studies show that RP-L102 GT appears to be a safe approach for hematopoietic preservation in FA without acquisition of detectable mutations in HSPCs to-date."

Preclinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hematological Malignancies • Hepatitis B • Hepatology • Herpes Zoster • Immunology • Infectious Disease • Inflammation • Oncology • Transplantation • Varicella Zoster • CD34 • FANCA

Changing the Natural History of Fanconi Anemia Complementation Group-À with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells (TCT-ASTCT-CIBMTR 2020) - P1; "PBMCs were collected via leucocytapheresis after mobilization with G-CSF and Plerixafor...The investigational drug product (DP) (RP-L102) was infused fresh in patients within 4 hours of release, without any conditioning regimen... DP was successfully manufactured and infused in the Phase I study and both FA-A patients are showing early signs of stabilization of cytopenias. Plans for opening the Phase II study are in progress. Preventing BMF by gene therapy without any exposure to chemo-radiotherapy has the potential to change the natural history of FA."

P1 data • Anemia • Aplastic Anemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Oncology • Thrombocytopenia • CD34 • CSF3

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical Trials (ESGCT 2022) - No abstract available

Clinical • Anemia • Gene Therapies • Hematological Disorders

Ex Vivo Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials (ASGCT 2022) - "Pts are followed for 3 years post-infusion for safety and efficacy evaluations including increasing PB and BM vector copy number (VCN), development of mitomycin-C (MMC) resistance in BM colony forming cells (CFCs), and stabilization/correction of cytopenias. Engraftment and proliferative advantage of transduced HSPCs has been confirmed in 6 of 8 evaluable (≥ 1 year of follow-up) pts in the absence of antecedent conditioning. Increases of ≥10% BM CFC MMC resistance relative to baseline were identified in each of these 6 patients at ≥ 12 months post-infusion (at 1 timepoint or more) with concomitant blood count stabilization identified subsequent to the development of MMC-resistance in at least 5 pts. Additional clinical updates will be presented including initial correlations between BM CFC MMC-resistance, genetic correction and hematologic stabilization."

Preclinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Oncology • Solid Tumor • Transplantation • CD34 • FANCA

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials (ASH 2021) - "Peripheral blood (PB) mononuclear cells are collected via leukapheresis on 2 consecutive days after mobilization with granulocyte-colony stimulating factor (G-CSF) and plerixafor...Patients are followed for 3 years post-infusion for safety assessments (replication competent lentivirus [RCL], insertion site analysis [ISA]) and to ascertain evidence of efficacy (increasing PB and BM vector copy number [VCN] and mitomycin-C [MMC] resistance in BM colony forming units [CFUs]), along with stabilization/correction of cytopenias... RP-L102’s safety profile remains favorable. Increasing evidence of engraftment has been confirmed in 6 subjects as demonstrated by PB VCN ; without conditioning, 12+ months of follow up is likely required to observe the proliferative advantage of transduced HSPCs."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hematological Malignancies • Infectious Disease • Oncology • Respiratory Diseases • Solid Tumor • Transplantation • CD34 • FANCA

[VIRTUAL] Gene therapy for Fanconi anemia [group a]: Interim results of RPL102 clinical trials (ESGCT 2021) - "RPL102's safety profile remains favorable. Increasing evidence of engraftment has been confirmed in 6 subjects as demonstrated by PB VCN; without conditioning, 12+ months of follow up is likely required to observe the proliferative advantage of transduced HSCs."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Infectious Disease • Respiratory Diseases • Transplantation • CD34 • FANCA • RPL10

[VIRTUAL] Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical Trials (ASGCT 2021) - "Peripheral blood (PB) mononuclear cells were collected via leukapheresis on 2 consecutive days after mobilization with granulocyte-colony stimulating factor (G-CSF) and plerixafor. DP has been successfully manufactured for N=9 subjects to meet the required specifications. The safety profile of RP-L102 is excellent. Preliminary evidence of engraftment has been confirmed in 5 subjects as demonstrated by PB VCN; 12+ months of follow-up may be required to observe the proliferative advantage of transduced HSPCs in the absence of conditioning."

Clinical • Anemia • Aplastic Anemia • Gene Therapies • Hematological Disorders • Hematological Malignancies • Infectious Disease • Oncology • Respiratory Diseases • Solid Tumor • Transplantation • CD34 • FANCA

[VIRTUAL] Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102 (ASH 2020) - "Peripheral blood (PB) mononuclear cells were collected via leucocytapheresis on two consecutive days after mobilization with granulocyte-colony stimulating factor (G-CSF) and plerixafor. DP has been successfully manufactured in the Phase I (N=2) and Phase 2 (N=5) to meet the required specifications Safety profile of RP-L102 continues to be highly favorable. Evidence of engraftment has been seen in at least 1 subject with follow up of at least 12 months as indicated by PB genetic markings and increasing BM CFC MMC resistance; 12+ months of follow-up may be required to observe the proliferative advantage of transduced HSPCs."

Clinical • Anemia • Aplastic Anemia • Gene Therapies • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Oncology • Respiratory Diseases • Solid Tumor • Transplantation • CD34 • FANCA

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A (clinicaltrials.gov) - P1; N=2; Active, not recruiting; Sponsor: Rocket Pharmaceuticals Inc.; Trial primary completion date: Sep 2019 ➔ Dec 2020

Clinical • Trial primary completion date • Anemia • Hematological Disorders • Pediatrics

Gene Therapy for Fanconi Anemia, Complementation Group A (clinicaltrials.gov) - P2; N=5; Recruiting; Sponsor: Rocket Pharmaceuticals Inc.; Initiation date: Feb 2020 ➔ Jul 2020; Trial primary completion date: Aug 2023 ➔ Jun 2022

Clinical • Trial initiation date • Trial primary completion date • Anemia • Gene Therapies • Hematological Disorders • Pediatrics

Gene Therapy for Fanconi Anemia, Complementation Group A (clinicaltrials.gov) - P2; N=5; Recruiting; Sponsor: Rocket Pharmaceuticals Inc.

Clinical • New P2 trial