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Vor Biopharma
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VCAR33 is a CD33-directed CAR-T cell therapy made from healthy cells obtained from the same donor from which the patient was previously transplanted. It is under phase 1/2 clinical development for the treatment of relapsed/refractory acute myeloid leukemia.


Lead Product(s): VCAR33

Therapeutic Area: Oncology Product Name: VCAR33

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 January 17, 2024

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Trem-cel (tremtelectogene empogeditemcel), is a genome-edited hematopoietic stem and progenitor allogeneic donor product candidate in order to remove the CD33 surface target protecting them from a targeted therapy post-transplant.


Lead Product(s): Tremtelectogene Empogeditemcel,Ozogamicin

Therapeutic Area: Oncology Product Name: Trem-cel

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 February 16, 2023

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VOR33 (trem-cel) is our lead eHSC product candidate created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target which is involved in causing AML and other blood related cancers thus, protecting them from a targeted therapy post-transplant.


Lead Product(s): Trem-cel,Ozogamicin

Therapeutic Area: Oncology Product Name: VOR33

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 January 19, 2023

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Vor Bio intends to use the net proceeds from the offering and concurrent private placement primarily to fund the continued clinical development of Trem-cel (tremtelectogene empogeditemcel) and pipeline products and for working capital and general corporate purposes.


Lead Product(s): Tremtelectogene Empogeditemcel,Ozogamicin

Therapeutic Area: Oncology Product Name: Trem-cel

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

Partner/Sponsor/Collaborator: Evercore Group L.L.C.

Deal Size: $65.8 million Upfront Cash: Undisclosed

Deal Type: Public Offering December 07, 2022

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Vor Bio intends to use the net proceeds from the offering and concurrent private placement primarily to fund the continued clinical development of Trem-cel (Tremtelectogene Empogeditemcel) and pipeline products and for working capital and general corporate purposes.


Lead Product(s): Tremtelectogene Empogeditemcel,Ozogamicin

Therapeutic Area: Oncology Product Name: Trem-cel

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

Partner/Sponsor/Collaborator: RA Capital Management

Deal Size: $50.0 million Upfront Cash: Undisclosed

Deal Type: Private Placement December 07, 2022

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Tremtelectogene empogeditemcel (trem-cel), formerly VOR33, is a genome-edited hematopoietic stem and progenitor allogeneic donor product candidate where CD33 has been deleted using genome engineering.


Lead Product(s): Tremtelectogene Empogeditemcel,Ozogamicin

Therapeutic Area: Oncology Product Name: VOR33

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 07, 2022

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VOR33 (Engineered Hematopoietic Stem Cell) is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell therapy product that lacks the CD33 myeloid protein.


Lead Product(s): Engineered Hematopoietic Stem Cell,Ozogamicin

Therapeutic Area: Oncology Product Name: VOR33

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 11, 2022

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The pre-clinical data of VOR33 (Engineered Hematopoietic Stem Cell), demonstrates that multiplex deletion by CRISPR/Cas9 of CD33 and CLL-1 from human CD34+ HSPCs maintained cell function and persisted long-term post engraftment in vivo.


Lead Product(s): Engineered Hematopoietic Stem Cell,Ozogamicin

Therapeutic Area: Oncology Product Name: VOR33

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 June 10, 2022

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The data demonstrated Vor Bio’s novel platform, VOR33, genetically modifies healthy donor hematopoietic stem cells (HSCs) to remove select cell surface targets, enables targeted therapies to selectively destroy cancer cells while sparing the healthy cells.


Lead Product(s): Engineered Human Stem Cell Therapy,Ozogamicin

Therapeutic Area: Oncology Product Name: VOR33

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 25, 2022

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This collaboration strengthens Akron’s portfolio of off-the-shelf gene editing technologies while broadening Vor Bio’s collection of nucleases to engineer gene modified cell therapies including VOR33.


Lead Product(s): Engineered Human Stem Cell Therapy,Ozogamicin

Therapeutic Area: Oncology Product Name: VOR33

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

Recipient: Akron Biotech

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration March 22, 2022

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