Courtney Tito’s Post

Herein authors discuss the similarities and differences between #TB and nontuberculous mycobacteria (#NTM) #lungdiseases, compare the #preclinical and #clinicaladvances, and identify major knowledge gaps and areas of cross-fertilization. https://lnkd.in/gMkS6sss

A high polygenic #risk score in combination with being factor V Leiden and #prothrombin variant carrier resulted in the highest risk of #venous #thromboembolism during the first two years of #oral #contraceptive use (hazard ratio, 14.8 [95% CI, 9.28 - 23.6]). #Women with a high genetic liability also had an increased risk during continued use but less pronounced, with the highest risk in combination with being carriers of both factor V Leiden and prothrombin variant (hazard ratio, 4.93 [95% CI, 3.16 - 7.7]).

Polycystic ovary syndrome (PCOS) - a serious genetic, metabolic and reproductive disorder affecting women - is the leading cause of female infertility and a precursor to many other diseases. A main goal of PCOS Awareness Month is to help improve the lives of those affected by: 1. Helping them overcome their symptoms 2. Preventing and reducing their risk for life-threatening related diseases To learn more about our successful experience advancing treatments in the women's health field, please see our Women's Health Fact Sheet.

Exciting progress in leukemia treatment with Bleximenib (JNJ-75276617) by Janssen Inc.! This selective, orally bioavailable small molecule targets the menin-KMT2A interaction, showing potential in treating leukemias with KMT2A and NPM1 alterations. A Phase Ib study combined Bleximenib with venetoclax and azacitidine in relapsed/refractory KMT2A-altered and NPM1-mutated AML. Results presented at EHA 2024 showed a 79% overall response rate (ORR), with 90% ORR in NPM1m and 62% in KMT2Ar AML groups. Bleximenib demonstrated compatibility with standard doses and a favorable safety profile. Ongoing Phase I dose escalation studies aim to determine the recommended Phase II dose (RP2D). Bleximenib continues to be explored in combination with AML therapies for newly diagnosed and relapsed patients. These promising results highlight a significant step forward in addressing unmet medical needs in leukemia treatment. Read the analysis in detail here @ https://lnkd.in/g8vgVqTi #LeukemiaResearch #Bleximenib #Janssen #EHA2024 #ClinicalTrials #venetoclax #azacitidine #ClinicalResearch #OncologyInnovations #healthcare #business #clinicalstudies #clinicaltrials #results #phases #top #abstracts #multiplemyeloma #pharmacautical #hematology #leukemia

Exciting progress in leukemia treatment with Bleximenib (JNJ-75276617) by Janssen Inc.! This selective, orally bioavailable small molecule targets the menin-KMT2A interaction, showing potential in treating leukemias with KMT2A and NPM1 alterations. A Phase Ib study combined Bleximenib with venetoclax and azacitidine in relapsed/refractory KMT2A-altered and NPM1-mutated AML. Results presented at EHA 2024 showed a 79% overall response rate (ORR), with 90% ORR in NPM1m and 62% in KMT2Ar AML groups. Bleximenib demonstrated compatibility with standard doses and a favorable safety profile. Ongoing Phase I dose escalation studies aim to determine the recommended Phase II dose (RP2D). Bleximenib continues to be explored in combination with AML therapies for newly diagnosed and relapsed patients. These promising results highlight a significant step forward in addressing unmet medical needs in leukemia treatment. Read the analysis in detail here @ https://lnkd.in/g_ZWWs5k #LeukemiaResearch #Bleximenib #Janssen #EHA2024 #ClinicalTrials #venetoclax #azacitidine #ClinicalResearch #OncologyInnovations #healthcare #business #clinicalstudies #clinicaltrials #results #phases #top #abstracts #multiplemyeloma #pharmacautical #hematology #leukemia

New European regulation for Substances of Human origin (SoHO): Standardization and new challenges On 14 December 2023, a political agreement was reached on the Commission’s proposal for a Regulation on standards of quality and safety for substances of human origin intended for human application, which will then apply as from 2027. The term SoHO includes blood, tissues, cells and any other substances of human origin like human breast milk and microbiota. Only solid organs intended for transplantation will remain regulated separately under the Organs Directive (Directive 2010/45/EU). This is an opportunity for companies and professionals. It will facilitate innovation; establishes authorisation and registry of SoHO clinical studies, and each authorised clinical study will be published on the EU SoHO Platform; and the obligation for all entities that carry out any activity that may affect their quality or safety to designate a Person responsible for ensuring that the entity complies with the Regulation and has a quality management system. For their part, SoHO establishments (entities that store and process, release, import or export SoHO) will require specific authorization and will be subject to periodic inspections by the authorities. In addition to having a Responsible Person, the SoHO establishment must designate a SoHO Release Manager and have a physician. https://lnkd.in/d5bTfZFX

Social Pharma Patient-Centric Marketing socialpharma.net >>> Gene&Cell Therapy >> FDA hits Mesoblast with CRL for remestemcel-L, wants adult data in acute GVHD #lucidquest #genetherapy #celltherapy

External controls can augment low populations in rare disease trials In an exclusive interview with Clinical Trials Arena, Dr Will Maier, VP of scientific affairs at ICON plc discusses the benefits and challenges of using historical or synthetic controls and what sponsors should keep in mind before planning trials that use these controls. This interview has been edited for length and clarity. - https://lnkd.in/dFdCvFYv #raredisease #clinicaltrials #clinicaldata

In this episode of Certara Talks, we continue to highlight Rare Disease Day. In this chat, our experts Oxana Iliach, Rajesh Krishna, David Wesche, and Paul Martin share their thoughts on the importance of clinical pharmacology programs in supporting rare disease drug development. ▶️ Watch now: https://ow.ly/l2zX50QH9wv Ready to explore the intersection of clinical pharmacology and rare disease drug development? Dive deeper with us! https://ow.ly/yLyF50QH9ww #RareDiseaseDay #ClinicalPharmacology #CertaraTalks

As I look forward to the hopeful prospects of a new year, I am mindful of families in our MPS community who face a very cruel future. Such is the case of this Sanfilippo type B syndrome family from the UK that had their story highlighted in The Guardian over the holidays. https://lnkd.in/dSCciEzD A Sanfilippo syndrome diagnosis is a tragic one, in part because there is no approved therapy. But increasingly, preclinical research has provided a viable and effective approach to treating this and similar disorders. However, the FDA, and in particular, their Center for Drug Evaluation and Research have a regulatory process that makes drug approval for ultra rare pediatric neurodegenerative disease difficult in the extreme. This family had access to an effective therapy for years as part of a clinical trial. Now, because of drawn out approval process and timeline, the current company involved with this therapy can no longer move forward. Before joining the National MPS Society, I spent my last years at Iowa State University generating the long-term pre-clinical treatment data for this program in the large animal model of MPS IIIB. I know, as do these families, that this program uses science and technology that work to prevent MPS IIIB disease! We simply must figure out a viable regulatory path forward for these and similar families. It is only a failure of will and imagination that stand in our way. #mucopolysaccharidosis #sanfilipposyndrome #drugdevelopment #drugapproval #acceleratedapproval #rarediseases #geneticdisorders