News Release

Presentations showcase breadth of Vor Bio’s multiplex editing capability including base editing of human hematopoietic stem and progenitor cells (HSPCs) enabling new therapeutic approaches following transplant in patients with acute myeloid leukemia (AML)

CAMBRIDGE, Mass., April 25, 2022 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today announced that two abstracts have been accepted for poster presentations at the Keystone Symposia Precision Genome Engineering meeting, held April 27 to May 1 in Keystone, Colorado.

The presentations include two posters and a workshop discussion featuring data on multiplex gene editing in human hematopoietic stem cells using various genome editing technologies. The data demonstrate how Vor Bio’s novel platform, which 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. This unique approach has the potential to reduce on-target toxicity, allowing new and different therapeutic approaches following transplant in patients with AML.

“As most tumor antigens are also expressed on normal blood cells, traditional multi-targeted immunotherapy increases the risk of severe cytopenia,” explained Tirtha Chakraborty, Ph.D., Vor Bio’s Chief Scientific Officer. “We are excited to present how we can potentially address this challenge with a safer approach, with minimum translocation risk, using our platform that removes two or more target antigens from the donor HSCs. This approach to transplant, coupled with targeted therapies, may enable the delivery of more efficacious and durable treatments.”

Vor Bio is advancing clinical investigation of its lead program, VOR33, an eHSC candidate for patients with AML. VOR33 consists of genome-edited hematopoietic stem and progenitor cells that have been engineered to lack the CD33 protein. It is designed to replace the standard of care in transplant settings for patients suffering from AML and potentially other blood cancers. The company’s Phase 1/2a multi-center, open-label study of VOR33, VBP101, continues to recruit participants with AML who are at risk of relapse.

Details of the abstracts and presentations times are as follows:

Title: Multiplex Editing of Hematopoietic Stem and Progenitor Cells (HSPCs) with CRISPR-Cas Nucleases Achieves High On-Target Editing with Undetectable Translocations
Presenter: Michael A. Pettiglio, Scientist I, Vor Bio
Authors: Michael A. Pettiglio, Nipul Patel, Azita Ghodssi, Timothy Collingsworth, Meltem Isik, Gary Ge, Alejandra Falla, Dane Hazelbaker, Elizabeth Paik, John Lydeard, Tirtha Chakraborty
Date/Time: Saturday, April 30, 2022. Poster session 3: 7:30 PM - 10:00 PM (MST)
Category: Precision Genome Engineering

Title: Multiplex Base Editing in human hematopoietic stem and progenitor cells (HSPCs) enables efficient removal of multiple surface antigens in acute myeloid leukemia (AML) immunotherapy
Presenter: Alejandra Falla, Senior Scientist, Vor Bio
Authors: Alejandra Falla, Caroline McGowan, Meltem Isik, Gabriella Angelini, Sushma Krishnamurthy, Azita Ghodssi, Matthew Ung, Harshal Shah, Huan Qiu, Ruijia Wang, Gary Ge, Kienan Salvadore, Mark B. Jones, Dane Hazelbaker, Elizabeth Paik, John Lydeard, Tirtha Chakraborty
Date/Time: Thursday, April 28, 2022, Poster session I: 7:30 PM - 10:00 PM (MST)
Category: Precision Genome Engineering

This research will also be presented as part of a Keystone Symposia Workshop session as a short oral presentation.
Workshop 1: Genome Editing without DSB
April 28, 2022, 2:30 PM - 4:30 PM (MST)

The posters from these presentations will be available on the Vor corporate website at the start of the meeting on April 27, 2022 at 9:00am Eastern Time at https://ir.vorbio.com/news-and-events/events-and-presentations.

About Vor Bio
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 to enable targeted therapies post-transplant. For more information, visit: www.vorbio.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “aim,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “project,” “should,” “target,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include Vor Bio’s statements regarding the potential of its platform to enable the delivery of more efficacious and durable treatments and the reduction of on-target toxicity. Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Vor Bio’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.

Contacts:

Media:
Sarah Spencer
+1 857-242-6076
sspencer@vorbio.com

Investors:
Chris Brinzey
ICR Westwicke
+1 339-970-2843
chris.brinzey@westwicke.com