Orsini - Rare Disease Pharmacy Solutions’ Post

Check out Orsini’s latest white paper, exploring the dynamic world of classical hematology. From hemophilia to sickle cell disease, we showcase groundbreaking hematology advancements, uncover trends and hurdles in treatment, and spotlight key payor considerations. Download the white paper now! https://hubs.la/Q02tY91v0 #ClassicalHematology #SpecialtyPharmacy #PatientCare #Hemophilia #SickleCellDisease

Check out Orsini’s latest white paper, exploring the dynamic world of classical hematology. From hemophilia to sickle cell disease, we showcase groundbreaking hematology advancements, uncover trends and hurdles in treatment, and spotlight key payor considerations. Download the white paper now! https://hubs.la/Q02tY9z80 #ClassicalHematology #SpecialtyPharmacy #PatientCare #Hemophilia #SickleCellDisease

Check out Orsini’s latest white paper, exploring the dynamic world of classical hematology. From hemophilia to sickle cell disease, we showcase groundbreaking hematology advancements, uncover trends and hurdles in treatment, and spotlight key payor considerations. Download the white paper now! https://hubs.la/Q02tYb110 #ClassicalHematology #SpecialtyPharmacy #PatientCare #Hemophilia #SickleCellDisease

We are excited to provide updated clinical safety and efficacy data from 18 sickle cell disease patients treated with renizgamglogene autogedtemcel (reni-cel) in the Phase 1/2/3 RUBY clinical trial at European Hematology Association (EHA) annual congress. Treated patients show early normalization of total hemoglobin with a mean within the normal range at >14 g/dL, as well as rapid and sustained improvements in fetal hemoglobin and percentage of F-cells of >40 and >90%, respectively. Additionally, all treated patients have been free of vaso-occlusive events for up to 22.8 months of follow-up. Reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. #Biotechnology #Cas12a #CellTherapy #ClinicalTrials #CRISPR #EHA #GeneEditing #Hematology #SickleCellDisease

We are very excited to provide updated clinical safety and efficacy data from 28 individuals with sickle cell disease treated with renizgamglogene autogedtemcel (reni-cel) in the Phase 1/2/3 RUBY clinical trial at the American Society of Hematology (ASH) annual congress. Participants were at a median (range) of 9.5 (0.7-25.2) months post-reni-cel infusion, with 11 having >1 year follow-up. Of the 28 participants, 27 were VOE-free post-reni-cel infusion. Early total hemoglobin normalization was observed, with mean total hemoglobin increasing from 9.8 g/dL at baseline to 13.8 g/dL at Month 6 (n=18), as well as rapid and sustained improvements in fetal hemoglobin (HbF) to ≥40% and mean corpuscular HbF concentration per F-cells well above the anti-sickling threshold. Allelic editing levels in both peripheral blood nucleated cells and bone marrow–derived CD34+ cells were high, with mean editing levels of 75.1% (n=8) and 87.8% (n=7) at Month 12, respectively. Markers of hemolysis, including absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, and haptoglobin, improved or normalized by Month 6 and were generally maintained over time. Finally, meaningful improvements in patient-reported outcome domains for pain, physical function, and social roles and activities were observed. Reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant.   If you have any questions or want more information, please reach out or check the links below!   Poster can be found here - https://lnkd.in/eUSdVYyG   More information on the Ruby Trial can be found here - https://lnkd.in/emSvRPqe   Long-term follow up information can be found here - https://lnkd.in/ebuETUd7   #ASH24 #Biotechnology #Cas12a #CellTherapy #ClinicalTrials #CRISPR #GeneEditing #GeneTherapy #Hematology #SickleCellDisease

Research showcased at this year's 6th European CAR-T Cell Meeting highlighted many of the incredible scientific developments that have occurred over the past year, including... 🔹 Promising rCAR-T outcomes for generalized myasthenia gravis 🔹 CD19 CAR-T success in systemic lupus erythematosus 🔹 Recommendations for cell therapy use in autoimmune diseases The conference, hosted by @ The EBMT and @ European Hematology Association (EHA), showed encouraging results across many disease areas, and highlighted the importance of keeping patient access and safety front of mind. Only then can we ensure that these therapies are not only approvable, but accessible and affordable too. Read the conference summary ➡️ https://lnkd.in/eC3cFRJE #ManufacturingBrighterFutures #CellTherapy #GeneTherapy #PatientAccess #Biotechnology

Unlock critical physician insights in allogeneic hematopoietic cell transplant (HCT) for high-risk patients with myelodysplastic syndrome (MDS) with our on-demand roundtable. You'll hear from a panel of hematology/oncology experts as they discuss best practices, current research and specific patient cases and clinical scenarios. Access this CME learning activity here 👉 https://lnkd.in/gZyX-BTj #NMDP #HCT #MDS

"Our results show that we can provide a basis for more objective clinical decisions for our patients with #MDS. Furthermore, our study shows that incorporating molecular information can significantly improve the decision-making process in #hematology." The Decision Support System for transplantation in #MyelodysplasticSyndromes, devised by Prof. Matteo G Della Porta and his team from Humanitas Research Hospital, has been spotlighted by American Society of Hematology Clinical News! Read the full article written by Anna Azvolinsky to learn more about how this revolutionary system powered by #ClinicalData, #Genomics and #NextGen Technologies aims to advance #PrecisionMedicine for MDS patients. 🔗https://lnkd.in/eqng_uni

NEW from #ESMOGI2024 | Advancements in second-line #immunotherapy for #cholangiocarcinoma 🎥 🇩🇪 Do-Youn Oh, MD, PhD, Seoul National University College of Medicine, discusses second-line therapies and #immunotherapies for #cholangiocarcinoma Currently, the standard first-line treatment includes #nivolumab or #pembrolizumab with #chemotherapy If the disease progresses, second-line therapies such as #FOLFOX are used in the absence of #genetic alterations It is emphasized that the next step should be to develop new regimens that incorporate #immunecheckpointinhibitors in this line of treatment Learn more 👉 https://lnkd.in/ehntdDMQ #VJOncology #Oncologynews

Clinical flow cytometry is crucial in diagnosing L&L. It allows for rapid identification and classification of abnormal cells in blood, bone marrow, lymph nodes... by analyzing their surface markers. This technique helps distinguish between different subtypes of L&L, guiding targeted treatments. Additionally, it provides insights into treatment response and disease progression. Flow cytometry's speed, precision, and ability to analyze multiple cell properties simultaneously make it an essential tool for accurate and timely L&L diagnostics.