\nVOR33, the lead product candidate from Vor\u2019s novel scientific platform, consists of CRISPR genome-edited hematopoietic stem and progenitor cells that have been engineered to lack CD33. Once infused, VOR33 is designed to protect patients\u2019 healthy cells from anti-CD33 therapies, such as VCAR33 or Mylotarg\u2122 (gemtuzumab ozogamicin). VOR33 is intended to replace standard of care transplants for AML patients who are at high risk of relapse and has the potential to seamlessly integrate into current transplant settings.\n<\/p>\n
\nVor is actively enrolling in its Phase 1\/2a clinical trial for AML patients who currently have limited treatment options. Vor remains on track to report VOR33\u2019s initial clinical data in the first half of 2022. Vor is also currently exploring the use of its genome engineered hematopoietic stem cell platform in combination with multiple therapeutic modalities.\n<\/p>\n
\nThe full text of the announcement from Vor is as follows:\n<\/p>\n
VOR33 Granted U.S. FDA Fast Track Designation for AML<\/b><\/p>\n
CAMBRIDGE, Mass., Sept. 09, 2021<\/b> \u2013 Vor Biopharma (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to VOR33, the Company\u2019s lead engineered hematopoietic stem cell (eHSC) therapeutic candidate for the treatment of acute myeloid leukemia (AML).\n<\/p>\n
\nVOR33 consists of CRISPR genome-edited hematopoietic stem and progenitor cells that have been engineered to lack CD33. Once infused, VOR33 is designed to protect patients\u2019 healthy cells from anti-CD33 therapies, such as VCAR33 or Mylotarg\u2122 (gemtuzumab ozogamicin). VOR33 is intended to replace standard of care transplants for AML patients who are at high risk of relapse and has the potential to seamlessly integrate into current transplant settings.\n<\/p>\n
\n\u201cReceiving Fast Track designation is an important milestone for Vor, which signals the FDA\u2019s recognition of the serious and life-threatening medical condition of patients facing acute myeloid leukemia and the potential of VOR33 to address this unmet medical need,\u201d said Robert Pietrusko, PharmD, Vor\u2019s Chief Regulatory and Quality Officer. \u201cWe will continue to work closely with the FDA to expedite the development of VOR33, which is now actively enrolling in its Phase 1\/2a clinical trial for AML patients who currently have limited treatment options. We continue to remain on-track to report VOR33\u2019s initial clinical data in the first half of 2022.\u201d\n<\/p>\n
\nVOR33 is the lead product candidate of Vor\u2019s novel scientific platform, which has the mission to create next-generation, treatment-resistant transplants that unlock the potential of targeted cancer therapies by leveraging advances in cell therapy and gene editing. Vor is currently exploring the use of its genome engineered hematopoietic stem cell platform in combination with multiple therapeutic modalities.\n<\/p>\n
\nFast Track designation is intended to facilitate development and expedite review of products designed to treat serious and life-threatening conditions with unmet medical needs. The designation is granted upon the FDA\u2019s review of data that demonstrate this potential, along with a product development program that is adequately designed to address the unmet medical need. Therapeutic candidates receiving Fast Track designation may be eligible for priority review and accelerated approval if certain conditions are met.\n<\/p>\n
About Vor Biopharma <\/b><\/p>\n
\nVor Biopharma is a clinical-stage cell and genome engineering company that aims to transform the lives of cancer patients by pioneering an engineered hematopoietic stem cell (eHSC) therapeutic platform that unlocks the potential of targeted therapies. By removing biologically redundant proteins from eHSCs, we design these cells and their progeny to be treatment-resistant to complementary targeted therapies, thereby enabling these therapies to selectively destroy cancer cells while sparing healthy cells.\n<\/p>\n
Forward-Looking Statements<\/b><\/p>\n
\nThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words \u201cbelieve,\u201d \u201ccontinue,\u201d \u201ccould,\u201d \u201cestimate,\u201d \u201cexpect,\u201d \u201cintend,\u201d \u201cmay,\u201d \u201cplan,\u201d \u201cpotential,\u201d \u201cproject,\u201d \u201cshould,\u201d \u201ctarget,\u201d \u201cwill,\u201d \u201cwould,\u201d 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 the Company\u2019s statements regarding the potential efficacy of VOR33, potential benefits VOR33 may receive in connection with its Fast Track designation and reporting initial clinical data from the VOR33 Phase 1\/2a clinical trial in the first half of 2022. The Company 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 the Company\u2019s 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; the impact of the COVID-19 pandemic on the Company\u2019s business, including its preclinical studies and clinical trials and availability of funding sufficient for the Company\u2019s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption \u201cRisk Factors\u201d included in the Company\u2019s most recent annual or quarterly report and in other reports the Company 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 the Company 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.\n<\/p>\n
About PureTech Health<\/b><\/p>\n
\nPureTech is a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, including inflammatory, fibrotic and immunological conditions, intractable cancers, lymphatic and gastrointestinal diseases and neurological and neuropsychological disorders, among others. The Company has created a broad and deep pipeline through the expertise of its experienced research and development team and its extensive network of scientists, clinicians and industry leaders. This pipeline, which is being advanced both internally and through PureTech’s Founded Entities, is comprised of 25 therapeutics and therapeutic candidates, including two that have received both U.S. FDA clearance and European marketing authorization, as of the date of PureTech’s most recently filed Half Year Report and corresponding Form 6-K. All of the underlying programs and platforms that resulted in this pipeline of therapeutic candidates were initially identified or discovered and then advanced by the PureTech team through key validation points based on the Company’s unique insights into the biology of the brain, immune and gut, or BIG, systems and the interface between those systems, referred to as the BIG Axis.\n<\/p>\n
\nFor more information, visit www.puretechhealth.com<\/a> or connect with us on Twitter @puretechh\n<\/p>\n Cautionary Note Regarding Forward-Looking Statements<\/b><\/p>\n \nThis press release contains statements that are or may be forward-looking statements, including statements that relate to expectations regarding the potential efficacy of VOR33, potential benefits VOR33 may receive in connection with its Fast Track designation and the anticipated timing of the initial clinical data from the VOR33 Phase 1\/2a clinical trial in the first half of 2022, and Vor\u2019s future prospects, developments, and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks and uncertainties that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks and uncertainties described in the risk factors included in the regulatory filings for PureTech Health plc. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, neither the Company nor any other party intends to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.\n<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20210909006184\/en\/<\/a><\/span><\/p>\n Investors KEYWORDS:<\/b> United States North America Massachusetts<\/p>\n INDUSTRY KEYWORDS:<\/b> Research Stem Cells Biotechnology Other Health Health Pharmaceutical Other Science Science Oncology<\/p>\n<\/p>\n
\n<\/b>
Allison Mead Talbot
\n
+1 617 651 3156
\n
amt@puretechhealth.com<\/a><\/p>\n
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