[{"orgOrder":0,"company":"Graphite Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Graphite Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Inapplicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Graphite Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Inapplicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Graphite Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Inapplicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Graphite Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Inapplicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Graphite Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Inapplicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Kamau Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Agreement","leadProduct":"Nulabeglogene Autogedtemcel","moa":"CRISPR\/HiFi Cas9","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Graphite Bio \/ Kamau Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Graphite Bio \/ Kamau Therapeutics"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Versant Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Graphite Bio \/ Versant Ventures","highestDevelopmentStatusID":"5","companyTruncated":"Graphite Bio \/ Versant Ventures"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"GPH102","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Graphite Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Graphite Bio \/ Inapplicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"LENZ Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Merger","leadProduct":"Aceclidine Hydrochloride","moa":"||Muscarinic receptor","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Graphite Bio \/ LENZ Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Graphite Bio \/ LENZ Therapeutics"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Versant Ventures","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Private Placement","leadProduct":"Aceclidine Hydrochloride","moa":"||Muscarinic receptor","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Graphite Bio \/ Versant Ventures","highestDevelopmentStatusID":"10","companyTruncated":"Graphite Bio \/ Versant Ventures"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Versant Ventures","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Private Placement","leadProduct":"Aceclidine Hydrochloride","moa":"||Muscarinic receptor","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Graphite Bio \/ Versant Ventures","highestDevelopmentStatusID":"10","companyTruncated":"Graphite Bio \/ Versant Ventures"}]

Find Clinical Drug Pipeline Developments & Deals by Graphite Bio

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : The net proceeds will advance LENZ’s lead programs, LNZ100 and LNZ101, preservative-free, single-use, once-daily eye drops for treating presbyopia.

                          Product Name : LNZ101

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          March 21, 2024

                          Lead Product(s) : Aceclidine Hydrochloride,Brimonidine Tartrate

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase III

                          Sponsor : Versant Ventures

                          Deal Size : $53.5 million

                          Deal Type : Private Placement

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                          02

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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.

                          Product Name : LNZ101

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          November 15, 2023

                          Lead Product(s) : Aceclidine Hydrochloride,Brimonidine Tartrate

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase III

                          Sponsor : LENZ Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          03

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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 presbyo...

                          Product Name : LNZ101

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          November 15, 2023

                          Lead Product(s) : Aceclidine Hydrochloride,Brimonidine Tartrate

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase III

                          Sponsor : Versant Ventures

                          Deal Size : $53.5 million

                          Deal Type : Private Placement

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                          04

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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.

                          Product Name : GPH101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 12, 2023

                          Lead Product(s) : Nulabeglogene Autogedtemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Kamau Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          05

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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).

                          Product Name : GPH101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 01, 2023

                          Lead Product(s) : Nulabeglogene Autogedtemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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).

                          Product Name : GPH101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 08, 2022

                          Lead Product(s) : Nulabeglogene Autogedtemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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.

                          Product Name : GPH102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 16, 2022

                          Lead Product(s) : GPH102

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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 ...

                          Product Name : GPH101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 10, 2021

                          Lead Product(s) : Nulabeglogene Autogedtemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : 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.

                          Product Name : GPH101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 17, 2021

                          Lead Product(s) : Nulabeglogene Autogedtemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Iran Expo
                          Not Confirmed
                          Iran Expo
                          Not Confirmed

                          Details : GPH101 harnesses the power of CRISPR and natural DNA repair mechanisms to precisely insert the correct DNA sequence, with the aim of directly correcting the disease-causing sickle mutation and leading to the production of normal red blood cells.

                          Product Name : GPH101

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 14, 2020

                          Lead Product(s) : Nulabeglogene Autogedtemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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