Vor

Engineering hematopoietic stem cell therapies to enable targeted therapies for patients with blood cancers

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Engineering hematopoietic stem cells to enable targeted therapies for patients with blood cancers

4.0% Equity

PHASE 1/2a

Description

Engineering hematopoietic stem cells to enable targeted therapies for patients with blood cancers

Puretech Ownership¹

4.0% Equity

Stage of Development

PHASE 1/2a

¹ As of August 4, 2023, PureTech’s ownership percentage of Vor Bio was approximately 4.0% on an outstanding voting share basis. This calculation includes outstanding shares, options, and warrants, but excludes unallocated shares authorized to be issued pursuant to equity incentive plans.

Engineering hematopoietic stem cell therapies to enable targeted therapies for patients with blood cancers

Vor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells (HSC) to unlock the potential of Vor’s highly potent targeted therapies which have an improved safety profile for patients, several of which Vor is also developing.

Program Discovery Process by the PureTech Team
- We were interested in approaches to treat hematological malignancies that currently have poor response rates or poor adverse event profiles despite recent advances in cell therapies and targeted therapies. We worked with Vor Bio Scientific Board Chair, Siddhartha Mukherjee, M.D., Ph.D., on key intellectual property, which Vor Bio exclusively in-licensed from Columbia in April 2016, and on advancing this concept through critical POC experiments.
Milestones Achieved & Developmental Status
- In December 2022, Vor announced initial clinical data from VBP101, Vor’s Phase 1/2a multicenter, open-label, first-in-human study of tremtelectogene empogeditemcel or “trem-cel” (formerly VOR33) in patients with AML. The data observed that the first AML patient transplanted with trem-cel demonstrated durable engraftment through three cycles of Mylotarg (gemtuzumab ozogamicin), which was well tolerated at the initial dose level.
- In February 2023, Vor announced a second patient also successfully received a trem-cel transplant and engrafted normally.
- In August 2023, Vor announced the U.S. FDA has cleared their Investigational New Drug (IND) application for a Phase 1/2 clinical trial of VCAR33^ALLO. The clinical trial will enroll patients who have relapsed following allogeneic stem cell transplant, which uses lymphoid cells harvested from the original donor as starting material for the drug product.
- In August 2023, Vor announced a world-wide non-exclusive license from Editas Medicine for ex-vivo Cas9 gene-edited HSC therapies for the treatment and/or prevention of hematological malignancies.
Expected Milestones
- Vor Bio expects additional trem-cel engraftment and hematologic protection data updates by year-end 2023.

Note: Vor Bio has an active IND on file with the FDA for VOR33 and an active IND is on file for VCAR33. PureTech does not have a direct interest in Vor Bio’s therapeutic candidates or its proprietary platform. PureTech’s interest in Vor Bio’s therapeutic candidates and proprietary platforms is limited to its non-controlling equity interest in Vor Bio and any potential appreciation in the value of such equity interest and PureTech does not control the clinical or regulatory development of Vor Bio’s therapeutic candidates. Vor Bio is well-protected with a robust intellectual property portfolio. Vor Bio was incorporated in December 2015.

³ The VCAR33 construct is being studied in a Phase 1/2 clinical trial sponsored by the National Marrow Donor Program (“NMDP”), and the timing of data release is dependent on the investigators conducting the trial.

Vor Bio’s proprietary platform leverages its expertise in HSC biology and genome engineering to remove surface targets expressed by cancer cells by genetically modifying HSCs. This allows Vor Bio to advance its goal of replacing the patient’s HSCs with next-generation, treatment-resistant eHSCs that unlock the potential of highly-potent targeted therapies. Vor Bio intends to develop Trem-cel (formerly VOR33) as an HSC transplant therapeutic candidate to replace the standard of care in transplant settings. Once the Trem-cel cells have engrafted, patients can potentially be treated with anti-CD33 therapies, such as Mylotarg or a CAR-T therapy therapeutic candidate, with limited on-target toxicity. The combination of Trem-cel and CD33-directed therapies has the potential to lead to durable anti-tumor activity.