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1 Disease |  |
5 Trials |
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5 Trials |  |
395 News |  |
| «123456» |
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] RESOLUTION OF SERIOUS VASO-OCCLUSIVE PAIN CRISES: RESULTS FROM THE ONGOING PHASE 1/2 HGB-206 GROUP C STUDY OF LENTIGLOBIN FOR SICKLE CELL DISEASE (SCD) GENE THERAPY (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1616;
P1/2
Complete resolution of VOC/ACS was observed in nearly all patients. The safety profile post-LentiGlobin remains consistent with myeloablative single-agent busulfan conditioning and underlying SCD.
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] RESOLUTION OF SERIOUS VASO-OCCLUSIVE PAIN CRISES: RESULTS FROM THE ONGOING PHASE 1/2 HGB-206 GROUP C STUDY OF LENTIGLOBIN FOR SICKLE CELL DISEASE (SCD) GENE THERAPY (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1615;
P1/2
Complete resolution of VOC/ACS was observed in nearly all patients. The safety profile post-LentiGlobin remains consistent with myeloablative single-agent busulfan conditioning and underlying SCD.
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] RESOLUTION OF SERIOUS VASO-OCCLUSIVE PAIN CRISES: RESULTS FROM THE ONGOING PHASE 1/2 HGB-206 GROUP C STUDY OF LENTIGLOBIN FOR SICKLE CELL DISEASE (SCD) GENE THERAPY (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1614;
P1/2
Complete resolution of VOC/ACS was observed in nearly all patients. The safety profile post-LentiGlobin remains consistent with myeloablative single-agent busulfan conditioning and underlying SCD.
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] RESOLUTION OF SERIOUS VASO-OCCLUSIVE PAIN CRISES: RESULTS FROM THE ONGOING PHASE 1/2 HGB-206 GROUP C STUDY OF LENTIGLOBIN FOR SICKLE CELL DISEASE (SCD) GENE THERAPY (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1613;
P1/2
Complete resolution of VOC/ACS was observed in nearly all patients. The safety profile post-LentiGlobin remains consistent with myeloablative single-agent busulfan conditioning and underlying SCD.
- Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz
[VIRTUAL] INTERIM RESULTS OF BETIBEGLOGENE AUTOTEMCEL GENE THERAPY IN PEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT Β-THALASSEMIA (TDT) TREATED IN THE PHASE 3 NORTHSTAR-2 (HGB-207) AND NORTHSTAR-3 (HGB-212) STUDIES (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1580;
P3
Pediatric patients with diverse TDT genotypes achieved TI rates comparable to adults after beti-cel gene therapy, suggesting that beti-cel is a viable treatment option for patients with TDT across ages. The treatment regimen had a safety profile consistent with busulfan myeloablation.
- Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz
[VIRTUAL] INTERIM RESULTS OF BETIBEGLOGENE AUTOTEMCEL GENE THERAPY IN PEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT Β-THALASSEMIA (TDT) TREATED IN THE PHASE 3 NORTHSTAR-2 (HGB-207) AND NORTHSTAR-3 (HGB-212) STUDIES (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1579;
P3
Pediatric patients with diverse TDT genotypes achieved TI rates comparable to adults after beti-cel gene therapy, suggesting that beti-cel is a viable treatment option for patients with TDT across ages. The treatment regimen had a safety profile consistent with busulfan myeloablation.
- Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz
[VIRTUAL] INTERIM RESULTS OF BETIBEGLOGENE AUTOTEMCEL GENE THERAPY IN PEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT Β-THALASSEMIA (TDT) TREATED IN THE PHASE 3 NORTHSTAR-2 (HGB-207) AND NORTHSTAR-3 (HGB-212) STUDIES (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1578;
P3
Pediatric patients with diverse TDT genotypes achieved TI rates comparable to adults after beti-cel gene therapy, suggesting that beti-cel is a viable treatment option for patients with TDT across ages. The treatment regimen had a safety profile consistent with busulfan myeloablation.
- Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz
[VIRTUAL] INTERIM RESULTS OF BETIBEGLOGENE AUTOTEMCEL GENE THERAPY IN PEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT Β-THALASSEMIA (TDT) TREATED IN THE PHASE 3 NORTHSTAR-2 (HGB-207) AND NORTHSTAR-3 (HGB-212) STUDIES (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1577;
P3
Pediatric patients with diverse TDT genotypes achieved TI rates comparable to adults after beti-cel gene therapy, suggesting that beti-cel is a viable treatment option for patients with TDT across ages. The treatment regimen had a safety profile consistent with busulfan myeloablation.
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] Resolution of sickle cell disease manifestations in patients treated with LentiGlobin gene therapy: updated results from the phase 1/2 HGB‐206 Group C study () - Jan 5, 2021 - Abstract #BSHI2020BSH-I_407;
P1/2
Patients in Group C experienced high‐level, sustained, nearly pancellular expression of anti‐sickling HbAT87Q, with median HbS reduced to ˜50% of total Hb and median total Hb levels of 11·5 g/dl at 6 months. The cessation of clinical complications (no ACS or serious VOCs) and decrease in haemolysis suggest a strong therapeutic effect after LentiGlobin gene therapy in patients with SCD.
- Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz, Defibrotide
[VIRTUAL] Clinical outcomes following autologous haematopoietic stem cell transplantation with LentiGlobin gene therapy in the phase 3 Northstar‐2 and Northstar‐3 studies for transfusion‐dependent β‐thalassaemia () - Jan 5, 2021 - Abstract #BSHI2020BSH-I_98;
P3
Table. Treatment characteristics (median [min–max])
- Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz
[VIRTUAL] Safety and Efficacy Outcomes in Pediatric Patients with Transfusion-Dependent β-Thalassemia (TDT) Receiving Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-thalassemia) Gene Therapy in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies () - Dec 20, 2020 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_691;
P3
Summary After treatment with beti-cel, pediatric patients <18 yrs achieved transfusion independence with comparable rates to adults, suggesting that beti-cel gene therapy represents an effective treatment option across ages. The tolerability profile of treatment with beti-cel is consistent with the known effects of busulfan myeloablation.
- Zynteglo (betibeglogene autotemcel) / bluebird bio
[VIRTUAL] Efficacy and Safety of Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-thalassemia) Gene Therapy in 60 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed for up to 6 Years Post-Infusion () - Dec 20, 2020 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_577;
P=N/A, P1/
- Zynteglo (betibeglogene autotemcel) / bluebird bio
[VIRTUAL] Resolution of Serious Vaso-Occlusive Pain Crises: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy () - Dec 20, 2020 - Abstract #TCTASTCTCIBMTR2021TCT_ASTCT_CIBMTR_214;
P1/2
- | Lyfgenia (lovotibeglogene autotemcel) / bluebird bio
Trial completion date, Trial primary completion date, Gene therapy, Viral vector: HGB-206: A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease (clinicaltrials.gov) - Dec 17, 2020
P1/2, N=50, Active, not recruiting, Sponsor: bluebird bio
The tolerability profile of treatment with beti-cel is consistent with the known effects of busulfan myeloablation. Trial completion date: Feb 2022 --> Mar 2023 | Trial primary completion date: Feb 2022 --> Sep 2022
- || Zynteglo (betibeglogene autotemcel) / bluebird bio
Enrollment closed, Gene therapy: Northstar-3: A Study Evaluating the Efficacy and Safety of the LentiGlobin (clinicaltrials.gov) - Dec 2, 2020
P3, N=18, Active, not recruiting, Sponsor: bluebird bio
Trial completion date: Feb 2022 --> Mar 2023 | Trial primary completion date: Feb 2022 --> Sep 2022 Recruiting --> Active, not recruiting
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy (Channel 20 (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_4467;
P1/2
The safety profile post-LentiGlobin remains generally consistent with myeloablative single-agent busulfan conditioning and underlying SCD. Longer follow-up and additional patients will be presented.
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy (Channel 19 (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_3091;
P1/2
LentiGlobin was infused following myeloablative busulfan conditioning...Summary LentiGlobin for SCD GT improved HRQoL in all domains of PROMIS-57 for patients whose baseline scores were “worse” than the population norm, including clinically meaningful improvements in all evaluable (6/8) domains. Larger sample sizes are required to clarify the impact of LentiGlobin for SCD for some PROMIS-57 domains.
- Zynteglo (betibeglogene autotemcel) / bluebird bio
[VIRTUAL] Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers (Poster Hall (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_2162;
P3
In instances of lower HbAT87Q, higher endogenous HbF might be a determinant in whether TI is achieved. Despite genetic heterogeneity, beti-cel enabled patients to achieve TI regardless of β-thalassemia genotype, TSS, disease genetic modifiers including HBA, and HbF QTL SNP genotypes.
- Mozobil (plerixafor) / Sanofi
[VIRTUAL] Improvement in Erythropoiesis Following Treatment with Betibeglogene Autotemcel Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia in the Phase 3 Hgb-207 Study (Poster Hall (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_1733;
P3
These patients showed improvements in erythropoiesis as assessed by bone marrow biopsies and biomarkers, suggesting that the bone marrow is in the process of normalizing. The treatment regimen comprised of conditioning and beti-cel infusion had a tolerability profile consistent with the known effects of busulfan myeloablation.
- Defitelio (defibrotide) / IRCCS San Raffaele Hospital, Jazz, Defibrotide
[VIRTUAL] Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia (Channel 20 (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_1353;
P3
Summary Interim results in HGB-207 and HGB-212 show that after treatment with beti-cel, pediatric patients <18 yrs achieved transfusion independence with comparable rates as in adults, suggesting that beti-cel gene therapy represents an effective treatment option across ages. The safety profile of gene therapy with beti-cel was consistent with busulfan myeloablation.
- Zynteglo (betibeglogene autotemcel) / bluebird bio
[VIRTUAL] Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up (Channel 20 (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_1342;
P=N/A
Sustained levels of HbAT87Q and effective iron reduction with phlebotomy and/or iron chelation have resulted in improved hematologic parameters and iron burden. The paucity of gene therapy-related AEs observed beyond 2 years post-infusion study suggests a favorable long-term safety profile.
- | Zynteglo (betibeglogene autotemcel) / bluebird bio
European regulatory, Journal: Regulators' Advice Can Make a Difference: European Medicines Agency Approval of Zynteglo for Beta Thalassemia. (Pubmed Central) - Oct 30, 2020
The paucity of gene therapy-related AEs observed beyond 2 years post-infusion study suggests a favorable long-term safety profile. No abstract available
- [VIRTUAL] Market Access Landscape for Advanced Therapy Medicinal Products in the EU-5 () - Oct 3, 2020 - Abstract #ISPOREU2020ISPOR-EU_1648;
Two ATMPs, Zynteglo and Zolgensma are currently being evaluated by NICE. CONCLUSIONS Most ATMPs are granted patient access in EU5 even though HTA bodies have imposed monitoring requirements to ensure the value reflects the price.
- Zynteglo (betibeglogene autotemcel) / bluebird bio
[VIRTUAL] WHEN the German Healthcare System Seems Overwhelmed - Performance of the MOST Expensive Pharmaceuticals in the German HTA (AMNOG)-Procedure () - Oct 3, 2020 - Abstract #ISPOREU2020ISPOR-EU_1082;
The most expensive drug with 1.575.000€ annual therapy costs was betibeglogene autotemcel, a gene therapy... Our results confirm the general German notion, that also in the high-price segment of pharmaceutical drugs, there is no correlation between the negotiated rebate and the category of the additional benefit nor with the cost level of the treatments.
- Zynteglo (betibeglogene autotemcel) / bluebird bio
[VIRTUAL] Betibeglogene Autotemcel GENE Therapy (BETI-CEL) Is Cost-Effective Versus Standard of Care in Patients with Transfusion-Dependent B-Thalassemia (TDT) in France () - Oct 3, 2020 - Abstract #ISPOREU2020ISPOR-EU_1045;
Given a willingness to pay of €100,000/QALY, beti-cel has an 84% cost-effectiveness probability compared to the current SoC. CONCLUSIONS : Even in the context of no official threshold in France, this modeling study suggests that beti-cel can represent a cost-effective treatment option for TDT’ indication patients.
- Zynteglo (betibeglogene autotemcel) / bluebird bio
[VIRTUAL] Rethinking the Health-Economic Evaluation Framework for GENE Therapies- The Betibeglogene Autotemcel (BETI-CEL) Case in Î’-Thalassemia () - Oct 3, 2020 - Abstract #ISPOREU2020ISPOR-EU_144;
Such results are not representative of benefits of gene therapies in real life. It is therefore necessary for health authorities to adapt health-economic evaluation’s framework for gene therapies so that these evaluations can support more realistic public decisions.
- Xtandi (enzalutamide) / Pfizer, Astellas, Zynteglo (betibeglogene autotemcel) / bluebird bio, Tecentriq (atezolizumab) / Roche
[VIRTUAL] Does the Finose Collaboration Improve Access in Sweden and Norway? () - Oct 3, 2020 - Abstract #ISPOREU2020ISPOR-EU_99;
CONCLUSIONS : The FINOSE reports are used by national agencies to inform their decision-making and provides manufacturers with the opportunity to reduce duplication of submissions. Despite that the treatments did not receive a positive recommendation; it could potentially increase equal and faster access to Nordic patients in future collaborations.
- || Zynteglo (betibeglogene autotemcel) / bluebird bio
Review, Journal: Novel genetic therapeutic approaches for modulating the severity of β-thalassemia (Review). (Pubmed Central) - Sep 23, 2020
Gene therapy using the lentiglobin vector is the most recent method for cure without any mortality, graft rejection and clonal dominance issues...CRISPR/Cas9 has can be used for precise transcriptional regulation, genome modification and epigenetic editing. Additional research is required in this regard, as CRISPR/Cas9 may potentially exhibit off-target activity and there are legal and ethical considerations regarding its use.
- | Zynteglo (betibeglogene autotemcel) / bluebird bio
Journal: Recent progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia. (Pubmed Central) - Sep 16, 2020
P3
New development in gene addition strategies by lentiviral vectors and LentiGlobin BB305. Novel advanced approaches target to cut DNA at a targeted site and convert of HbF to HbA during infancy by gene editings such as BCL11A (B cell lymphoma11A) suppression, HPFH (hereditary persistence of fetal hemoglobin) and Zinc-Finger Nucleases.
- autologous hematopoietic stem cell therapy / Northwestern University
[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (Abstract video presentations) - Sep 3, 2020 - Abstract #DGHO2020DGHO_2074;
P3
89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation.
- autologous hematopoietic stem cell therapy / Northwestern University
[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (San Francisco) - Sep 3, 2020 - Abstract #DGHO2020DGHO_1938;
P3
89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation.
- autologous hematopoietic stem cell therapy / Northwestern University
[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (Abstract video presentations) - Sep 3, 2020 - Abstract #DGHO2020DGHO_1370;
P3
89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation.
- autologous hematopoietic stem cell therapy / Northwestern University
[VIRTUAL] Clinical results after autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for ß-thalassemia) gene therapy in phase 3 Nothstar-2 and Northstar-3 studies in transfusion-dependent ß-thalassemia (TDT) (Abstract video presentations) - Sep 3, 2020 - Abstract #DGHO2020DGHO_611;
P3
89% and 75% of evaluable patients in Northstar-2 and -3 achieved TI. Safety is consistent with that of single-agent busulfan myeloablation.
- ||| Zynteglo (betibeglogene autotemcel) / bluebird bio
That’s a great idea. I wonder if @ASTCT is doing something similar in the US. It’s not just CAR T, as you well know. The engineered stem cell products are going to be coming, and Zynteglo is already approved in the EU. It’s going to be the same issue @bluebirdbio @VertexPharma (Twitter) - Aug 30, 2020
- Lenti-D (elivaldogene tavalentivec) / bluebird bio
[VIRTUAL] SAFETY OF AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH GENE ADDITION THERAPY FOR TRANSFUSION-DEPENDENT β-THALASSEMIA, SICKLE CELL DISEASE, AND CEREBRAL ADRENOLEUKODYSTROPHY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_5001;
P=N/A, P1/
Additionally, there were no AEs related to vector integration. While safety beyond 5-years is still being established, these data suggest that HSC gene therapy may be a suitable therapy for patients with TDT, SCD, and CALD, particularly in those who are lacking a suitable donor or at an increased risk of complications from allo-HSCT.
- Lenti-D (elivaldogene tavalentivec) / bluebird bio
[VIRTUAL] SAFETY OF AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH GENE ADDITION THERAPY FOR TRANSFUSION-DEPENDENT β-THALASSEMIA, SICKLE CELL DISEASE, AND CEREBRAL ADRENOLEUKODYSTROPHY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_5000;
P=N/A, P1/
Additionally, there were no AEs related to vector integration. While safety beyond 5-years is still being established, these data suggest that HSC gene therapy may be a suitable therapy for patients with TDT, SCD, and CALD, particularly in those who are lacking a suitable donor or at an increased risk of complications from allo-HSCT.
- Lenti-D (elivaldogene tavalentivec) / bluebird bio
[VIRTUAL] SAFETY OF AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH GENE ADDITION THERAPY FOR TRANSFUSION-DEPENDENT β-THALASSEMIA, SICKLE CELL DISEASE, AND CEREBRAL ADRENOLEUKODYSTROPHY (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4999;
P=N/A, P1/
Additionally, there were no AEs related to vector integration. While safety beyond 5-years is still being established, these data suggest that HSC gene therapy may be a suitable therapy for patients with TDT, SCD, and CALD, particularly in those who are lacking a suitable donor or at an increased risk of complications from allo-HSCT.
- autologous hematopoietic stem cell therapy / Northwestern University
[VIRTUAL] CLINICAL OUTCOMES FOLLOWING AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH LENTIGLOBIN GENE THERAPY IN THE PHASE 3 NORTHSTAR-2 AND NORTHSTAR-3 STUDIES FOR TRANSFUSION-DEPENDENT β-THALASSEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4986;
P3
In Northstar-2, 90% of patients achieved the primary endpoint of TI. The safety profile is consistent with single-agent busulfan myeloablation.
- autologous hematopoietic stem cell therapy / Northwestern University
[VIRTUAL] CLINICAL OUTCOMES FOLLOWING AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH LENTIGLOBIN GENE THERAPY IN THE PHASE 3 NORTHSTAR-2 AND NORTHSTAR-3 STUDIES FOR TRANSFUSION-DEPENDENT β-THALASSEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4985;
P3
In Northstar-2, 90% of patients achieved the primary endpoint of TI. The safety profile is consistent with single-agent busulfan myeloablation.
- autologous hematopoietic stem cell therapy / Northwestern University
[VIRTUAL] CLINICAL OUTCOMES FOLLOWING AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH LENTIGLOBIN GENE THERAPY IN THE PHASE 3 NORTHSTAR-2 AND NORTHSTAR-3 STUDIES FOR TRANSFUSION-DEPENDENT β-THALASSEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4984;
P3
In Northstar-2, 90% of patients achieved the primary endpoint of TI. The safety profile is consistent with single-agent busulfan myeloablation.
- busulfan / Generic mfg.
[VIRTUAL] LENTIGLOBIN GENE THERAPY TREATMENT OF TWO PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3706;
P3
In both patients, improvement of myeloid:erythroid ratio towards normal levels was observed. The safety profile of LentiGlobin was consistent with that of busulfan myeloablation.
- busulfan / Generic mfg.
[VIRTUAL] LENTIGLOBIN GENE THERAPY TREATMENT OF TWO PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3705;
P3
In both patients, improvement of myeloid:erythroid ratio towards normal levels was observed. The safety profile of LentiGlobin was consistent with that of busulfan myeloablation.
- busulfan / Generic mfg.
[VIRTUAL] LENTIGLOBIN GENE THERAPY TREATMENT OF TWO PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_3704;
P3
In both patients, improvement of myeloid:erythroid ratio towards normal levels was observed. The safety profile of LentiGlobin was consistent with that of busulfan myeloablation.
- busulfan / Generic mfg., Mozobil (plerixafor) / Sanofi
[VIRTUAL] BETIBEGLOGENE AUTOTEMCEL (LENTIGLOBIN) IN PATIENTS WITH TRANSFUSION-DEPENDENT Β-THALASSEMIA AND Β0/Β0, B+IVS-I-110/B+IVS-I-110, OR Β0/B+IVS-I-110 GENOTYPES: UPDATED RESULTS FROM THE HGB-212 STUDY () - May 16, 2020 - Abstract #EHA2020EHA_1178;
P3
The treatment regimen comprised of conditioning and beti-cel infusion had a tolerability profile consistent with busulfan myeloablation. Longer follow-up will further establish the efficacy and safety of beti-cel in patients with TDT and β0/β0, β+IVS-I-110/β+IVS-I-110, or β0/β+IVS-I-110 genotypes.
- busulfan / Generic mfg., Mozobil (plerixafor) / Sanofi
[VIRTUAL] IMPROVEMENT IN ERYTHROPOIESIS IN PATIENTS WITH TRANSFUSION-DEPENDENT B-THALASSEMIA FOLLOWING TREATMENT WITH BETIBEGLOGENE AUTOTEMCEL (LENTIGLOBIN FOR B-THALASSEMIA) IN THE PHASE 3 HGB-207 STUDY () - May 16, 2020 - Abstract #EHA2020EHA_580;
P3
These patients showed improvements in erythropoiesis, suggesting that the the bone marrow is in process of normalizing. The treatment regimen comprised of conditioning and beti-cel infusion had a tolerability profile consistent with the known effects of busulfan myeloablation.
- busulfan / Generic mfg., Mozobil (plerixafor) / Sanofi
[VIRTUAL] OUTCOMES IN PATIENTS TREATED WITH LENTIGLOBIN FOR SICKLE CELL DISEASE (SCD) GENE THERAPY: UPDATED RESULTS FROM THE PHASE 1/2 HGB-206 GROUP C STUDY () - May 16, 2020 - Abstract #EHA2020EHA_470;
P1/2
Next, HSC collection via plerixafor mobilization/apheresis was instituted (Group C)...Absence of ACS and serious VOCs, and reduction in hemolysis suggest suppression of sickle-related complications. Longer follow-up and data on additional patients will be presented.
- busulfan / Generic mfg., Defitelio (defibrotide) / Jazz, IRCCS San Raffaele Hospital, Defibrotide
Clinical outcomes following autologous haematopoietic stem cell transplantation with LentiGlobin gene therapy in the phase 3 Northstar‐2 and Northstar‐3 studies for transfusion‐dependent β‐thalassaemia () - May 14, 2020 - Abstract #BSH2020BSH_48;
P3
In Northstar‐2, 90% of evaluable patients achieved TI. The safety profile was consistent with single‐agent busulfan myeloablation.
- busulfan / Generic mfg., Mozobil (plerixafor) / Sanofi
Resolution of sickle cell disease manifestations in patients treated with LentiGlobin gene therapy: updated results from the phase 1/2 HGB‐206 Group C study () - May 14, 2020 - Abstract #BSH2020BSH_12;
P1/2
Patients in Group C experienced high‐level, sustained, nearly pancellular expression of anti‐sickling HbAT87Q, with median HbS reduced to ˜50% of total Hb and median total Hb levels of 11·5 g/dl at 6 months. The cessation of clinical complications (no ACS or serious VOCs) and decrease in haemolysis suggest a strong therapeutic effect after LentiGlobin gene therapy in patients with SCD.
- Zynteglo (betibeglogene autotemcel) / bluebird bio
LentiGlobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from HGB-206 (Fort Lauderdale Marriott Harbor Beach Resort & Spa 3030 Holiday Drive, Fort Lauderdale, FL 33316) - Apr 16, 2020 - Abstract #FSCDR2020FSCDR_52;
- busulfan / Generic mfg.
LENTIGLOBIN GENE THERAPY IN PEDIATRICS, ADOLESCENTS, ADULTS WITH TRANSFUSION-DEPENDENT Β-THALASSEMIA () - Apr 3, 2020 - Abstract #ASPHO2020ASPHO_226;
P3
The safety profile is consistent with busulfan myeloablation. Sponsored by bluebird bio.