[{"orgOrder":0,"company":"BioLineRx","sponsor":"Washington University School of Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BioLineRx Announces Clinical Trial Collaboration with Washington University School of Medicine to Evaluate Motixafortide for CD34+ Hematopoietic Stem Cell Mobilization for Gene Therapies in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"ISRAEL","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Peptide","graph2":"BioLineRx"},{"orgOrder":0,"company":"Pluri","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pluristem Reports Positive Topline Phase I Results in Innovative Hematology Program, Which Is First to Study PLX-R18 in Humans","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pluri"},{"orgOrder":0,"company":"SpliSense","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SpliSense Successfully Completed Phase 1 Study of SPL84, RNA-Based Therapy, for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"SpliSense"}]
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SPL84, the Company's lead inhaled anti sense oligonucleotide (ASO) product for the treatment of patients with CF, carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.
The aim of the collaboration is to advance a Phase 1 clinical trial that will evaluate the safety and feasibility of BL-8040 (motixafortide) to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in sickle cell disease (SCD).
The results demonstrate that PLX-R18 has the potential to stimulate the implanted hematopoietic cells to realize their therapeutic potential and generate improved blood counts over the long term in all three blood cell lineages at once.