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Vertex will obtain a non-exclusive license for Editas Medicine’s Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of sickle cell disease and beta thalassemia, including Casgevy (exagamglogene autotemcel).


Lead Product(s): Exagamglogene Autotemcel

Therapeutic Area: Hematology Product Name: Casgevy

Highest Development Status: ApprovedProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement December 13, 2023

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EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, which demonstrated a sustained increase in fetal hemoglobin production in patients with with severe SCD and TDT.


Lead Product(s): EDIT-301,Busulfan

Therapeutic Area: Hematology Product Name: EDIT-301

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 16, 2023

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The partnersip aims to support the scaling of EDIT-301, Editas Medicine’s experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), from approval to commercialization.


Lead Product(s): EDIT-301

Therapeutic Area: Hematology Product Name: EDIT-301

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Recipient: Azzur Group

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Partnership July 27, 2023

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EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, which demonstrated a sustained increase in fetal hemoglobin production in patients with with severe SCD and TDT.


Lead Product(s): EDIT-301,Busulfan

Therapeutic Area: Hematology Product Name: EDIT-301

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 27, 2023

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Under the terms of the agreement, Shoreline will acquire EDIT-202, Editas Medicine’s preclinical multiplexed edited iNK cell medicine for the potential treatment of solid tumors and an additional iNK program under development and certain related manufacturing technologies.


Lead Product(s): EDIT-202

Therapeutic Area: Oncology Product Name: EDIT-202

Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Shoreline Biosciences

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Acquisition January 19, 2023

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EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).


Lead Product(s): EDIT-301,Busulfan

Therapeutic Area: Genetic Disease Product Name: EDIT-301

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 06, 2022

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EDIT-101 is a CRISPR/Cas9-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), by deleting the IVS26 CEP290 mutant allele.


Lead Product(s): EDIT-101

Therapeutic Area: Rare Diseases and Disorders Product Name: EDIT-101

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 17, 2022

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EDIT-103 is a CRISPR/Cas9-based experimental medicine in preclinical development for the treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), a progressive form of retinal degeneration.


Lead Product(s): EDIT-103

Therapeutic Area: Genetic Disease Product Name: EDIT-103

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 13, 2022

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Details:

EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).


Lead Product(s): EDIT-301,Busulfan

Therapeutic Area: Genetic Disease Product Name: EDIT-301

Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 27, 2022

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By combining Editas’s gene editing technology with Immatics’ ACTallo® allogeneic, off-the-shelf adoptive cell therapy platform based on gamma-delta T cells, that can be redirected to cancer cell targets with the goal of creating cells with enhanced tumor recognition.


Lead Product(s): Gamma-delta T Cell Adoptive Cell Therapies

Therapeutic Area: Oncology Product Name: Undisclosed

Highest Development Status: DiscoveryProduct Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Immatics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration June 07, 2022

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