[{"orgOrder":0,"company":"Graphite Bio","sponsor":"Versant Ventures","pharmaFlowCategory":"D","amount":"$45.0 million","upfrontCash":"Undisclosed","newsHeadline":"Graphite Bio Launches with $45 Million Series A Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Graphite Bio Receives IND Clearance to Initiate Clinical Trial for Next-Generation Gene Editing Therapy GPH101 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 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2024","url1":"","url2":"","graph1":"Ophthalmology","graph2":"Phase III"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Kamau Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Kamau Therapeutics Emerges from Stealth With Clinical-Stage Novel Gene Correction Technology Targeting Life-Threatening Genetic Diseases","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Hematology","graph2":"Phase I\/ Phase II"}]
Find Clinical Drug Pipeline Developments & Deals by Graphite Bio
The net proceeds will be used on the advancement of LENZ’s lead programs, LNZ100 and LNZ101, which are preservative-free, single-use, once-daily eye drops containing aceclidine and aceclidine plus brimonidine, respectively, for the treatment of presbyopia.
The combined will focus on the advancement of LENZ’s lead programs, LNZ100 and LNZ101, which are preservative-free, single-use, once-daily eye drops containing aceclidine and aceclidine plus brimonidine, respectively, for the treatment of presbyopia.
Under the agreement, Kamau will focus on the clinical development of lead program Nula-Cel (nulabeglogene autogedtemcel), which is being evaluated in Phase I/II clinical trial studies for the treatment of sickle cell disease.
The combined will focus on the advancement of LENZ’s lead programs, LNZ100 and LNZ101, which are preservative-free, single-use, once-daily eye drops containing aceclidine and aceclidine plus brimonidine, respectively, for the treatment of presbyopia.
The net proceeds will be used on the advancement of LENZ’s lead programs, LNZ100 and LNZ101, which are preservative-free, single-use, once-daily eye drops containing aceclidine and aceclidine plus brimonidine, respectively, for the treatment of presbyopia.
GPH101 (nula-cel), is an investigational next-generation gene editing autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD).
Nula-cel, formerly known as GPH101, is an investigational next-generation gene-editing autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD).
GPH102 is designed to replace the mutated beta-globin gene with a functional gene and restore adult hemoglobin (HbA) expression to levels similar to individuals who do not have the disease.
First patient expected to be treated by GPH101 in first half of 2022, GPH101 an investigational next-generation gene-edited autologous hematopoietic stem cell therapy designed to directly correct the genetic mutation that causes sickle cell disease.
The preclinical data support CEDAR clinical trial, a Phase 1/2 study evaluating the safety, pharmacodynamics, engraftment success, gene correction rates and total hemoglobin, as well as other clinical and exploratory endpoints of GPH101 in patients with severe SCD.