• Live Feeds
    • Press Releases
    • Insider Trading
    • FDA Approvals
    • Analyst Ratings
    • Insider Trading
    • SEC filings
    • Market insights
  • Analyst Ratings
  • Alerts
  • Subscriptions
  • Settings
  • RSS Feeds
Quantisnow Logo
  • Live Feeds
    • Press Releases
    • Insider Trading
    • FDA Approvals
    • Analyst Ratings
    • Insider Trading
    • SEC filings
    • Market insights
  • Analyst Ratings
  • Alerts
  • Subscriptions
  • Settings
  • RSS Feeds
Dashboard
    Quantisnow Logo

    © 2025 quantisnow.com
    Democratizing insights since 2022

    Services
    Live news feedsRSS FeedsAlerts
    Company
    AboutQuantisnow PlusContactJobs
    Legal
    Terms of usePrivacy policyCookie policy

    Successful Primary Engraftment of Trem-cel in First Five AML Patients Demonstrates Promise of Vor Bio's Platform

    6/9/23 7:30:00 AM ET
    $VOR
    Biotechnology: Biological Products (No Diagnostic Substances)
    Health Care
    Get the next $VOR alert in real time by email
    • Patients transplanted with trem-cel achieved primary neutrophil engraftment and high levels of myeloid donor chimerism.

    • Strong investigator enthusiasm and continued robust enrollment; additional data updates expected by year-end 2023.
    • U.S. FDA clears VCAR33ALLO IND.
    • Conference call scheduled for today, June 9 at 8:30am ET.

    CAMBRIDGE, Mass., June 09, 2023 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today presented updated clinical data from patients treated in VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (formerly VOR33) in patients with acute myeloid leukemia (AML). These data were presented by Guenther Koehne, MD, PhD, Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida in a poster presentation at the European Hematology Association (EHA) 2023 Congress in Frankfurt, Germany.

    "Based on this interim update of five patients treated, we remain confident in the potential of our approach to enable targeted therapies post-transplant. Investigator enthusiasm is strong and proposal of patients for enrollment in the study currently exceeds the enrollment stagger. We look forward to sharing further engraftment and hematologic protection data from additional patients treated by year-end," said Eyal Attar, MD, Vor Bio Chief Medical Officer.

    "The results being presented today are very encouraging. The unmet medical need, particularly for high-risk AML, is significant and this approach, if approved, could potentially transform outcomes for these patients," said Dr. Koehne.

    The time for neutrophil engraftment in all five patients treated with trem-cel (10-11 days) was similar to unedited transplants, suggesting that CD33 may be biologically dispensable. All patients achieved high levels of myeloid donor chimerism by day 28.

    After achieving timely neutrophil engraftment and platelet recovery, patient 2 experienced secondary graft failure coincident with a detected coronavirus hKU1 infection and following administration of antimicrobial agents including trimethoprim-sulfamethoxazole, both of which may be associated with graft failure. A backup graft was administered, and neutrophil engraftment and platelet recovery were observed.

    Patient 3 achieved timely neutrophil engraftment at Day 10, however platelets are still recovering. A platelet-reactive antibody was identified, and the patient is being treated for autoimmune thrombocytopenia. Platelets are steadily increasing and are at 15,000 per µL independent of transfusions.

    Patients 4 and 5 achieved normal neutrophil engraftment and platelet recovery, providing further confidence in the Company's platform and approach. These patients have not experienced any unexpected adverse events to date.

    Cell doses for all five patients were successfully manufactured and met release criteria, and all had a high level of CD33 editing efficiency.

    As previously reported, neutrophil and platelet cell counts were maintained in patient 1 who received multiple Mylotarg doses at 0.5 mg/m2, suggesting protection from Mylotarg-induced hematotoxicity.

    U.S. FDA clears VCAR33ALLO IND

    The Company also announced today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for VCAR33ALLO, a T-cell therapy derived from allogeneic healthy donors using a chimeric antigen receptor (CAR) specifically binding to CD33. VCAR33ALLO is planned to be studied in the VBP301 clinical trial, which will focus on patients who have relapsed following allogeneic stem cell transplant where T cells harvested from the original donor are used as starting material for the drug product.

    Conference Call & Webcast Information

    Members of the Vor Bio management team, joined by Dr. Guenther Koehne, will conduct a live conference call and webcast today at 8:30 am Eastern Time.

    Listeners can register for the webcast via this LINK.

    Analysts wishing to participate in the Q&A session should use this LINK.

    A replay of the webcast will be available via the investor section of the Company's website at www.vorbio.com approximately two hours after the call's conclusion.

    About AML

    AML is the most common type of acute leukemia in adults and one of the deadliest and most aggressive blood cancers, affecting 20,000 newly diagnosed patients each year in the United States. Approximately half of patients with AML who receive a hematopoietic cell transplant (HCT) suffer a relapse of their leukemia, with two-year survival rates of less than 20%, and relapse rates are higher for patients with certain adverse risk features. The fragility of engrafted hematopoietic stem cells prevents treatment following transplant, giving the cancer a chance to return.

    About the VBP101 Clinical Trial

    VBP101 is a Phase 1/2a, multicenter, open-label, first-in-human study of trem-cel (VOR33) in participants with AML who are undergoing human leukocyte antigen (HLA)-matched allogeneic HCT. Trem-cel is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product, lacking the CD33 protein. It is being investigated for participants with CD33+ AML at high risk for relapse after HCT to allow post-HCT targeting of residual CD33+ acute AML cells using Mylotarg (gemtuzumab ozogamicin) without toxicity to engrafted cells. Participants undergo a myeloablative HCT with matched related or unrelated donor CD34-selected HSPCs engineered to remove CD33 expression (trem-cel drug product). Mylotarg is given after engraftment for up to four cycles. The primary endpoint is the incidence of successful engraftment, defined as the first day of 3 consecutive days of absolute neutrophil count (ANC) 500 cells/mm2 by day 28. Part 1 of this study is evaluating the safety of escalating Mylotarg dose levels to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose. Part 2 will expand the number of participants to evaluate the Mylotarg recommended Phase 2 dose. For more information, visit: https://clinicaltrials.gov/ct2/show/NCT04849910

    About Trem-cel

    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. Transplant with trem-cel is designed to replace standard of care transplants for patients suffering from AML and potentially other blood cancers. Trem-cel has the potential to enable powerful targeted therapies in the post-transplant setting including CD33-targeted CAR-T cells.

    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," "anticipate," "can," "continue," "could," "design," "enable," "expect," "initiate," "intend," "may," "on-track," "ongoing," "plan," "potential," "should," "target," "update," "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 product candidates to positively impact quality of life and alter the course of disease in the patients it seeks to treat, the timing and pace of patient enrollment in clinical trials and the availability of data therefrom, the expected safety profile of its product candidates, the potential of trem-cel to enable targeted therapies in the post-transplant setting including Mylotarg and CD33-targeted CAR-Ts. 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; whether successful engraftment and platelet recovery will ultimately lead to efficacy of trem-cel; expectations for regulatory approvals to conduct trials or to market products; the success of Vor Bio's in-house manufacturing capabilities and efforts; 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.

    Contact:

    Investors & Media

    Sarah Spencer

    +1 857-242-6076

    [email protected]



    Primary Logo

    Get the next $VOR alert in real time by email

    Chat with this insight

    Save time and jump to the most important pieces.

    Recent Analyst Ratings for
    $VOR

    DatePrice TargetRatingAnalyst
    7/26/2022$18.00Outperform
    Wedbush
    4/27/2022$6.00Neutral
    Goldman
    12/17/2021$26.00Buy
    HC Wainwright & Co.
    12/3/2021$38.00Outperform
    Robert W. Baird
    12/3/2021$38.00Outperform
    Baird
    12/2/2021$30.00Outperform
    Oppenheimer
    10/19/2021$40.00Market Outperform
    JMP Securities
    More analyst ratings

    $VOR
    Large Ownership Changes

    This live feed shows all institutional transactions in real time.

    See more
    • Amendment: SEC Form SC 13G/A filed by Vor Biopharma Inc.

      SC 13G/A - Vor Biopharma Inc. (0001817229) (Subject)

      11/14/24 4:33:50 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • SEC Form SC 13D/A filed by Vor Biopharma Inc. (Amendment)

      SC 13D/A - Vor Biopharma Inc. (0001817229) (Subject)

      2/14/24 4:17:24 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • SEC Form SC 13G/A filed by Vor Biopharma Inc. (Amendment)

      SC 13G/A - Vor Biopharma Inc. (0001817229) (Subject)

      2/14/24 4:08:06 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $VOR
    SEC Filings

    See more
    • Vor Biopharma Inc. filed SEC Form 8-K: Costs Associated with Exit or Disposal Activities

      8-K - Vor Biopharma Inc. (0001817229) (Filer)

      5/8/25 8:35:56 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • SEC Form DEFR14A filed by Vor Biopharma Inc.

      DEFR14A - Vor Biopharma Inc. (0001817229) (Filer)

      4/30/25 4:04:48 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • SEC Form 8-K filed by Vor Biopharma Inc.

      8-K - Vor Biopharma Inc. (0001817229) (Filer)

      4/25/25 4:01:13 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $VOR
    Financials

    Live finance-specific insights

    See more
    • Vor Bio Announces Exploration of Strategic Alternatives to Maximize Shareholder Value

      CAMBRIDGE, Mass., May 08, 2025 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today announced that, based on currently available clinical data from its key clinical programs and a challenging fundraising environment, the Board of Directors has approved the initiation of a process to explore a range of strategic alternatives aimed at maximizing both near- and long-term shareholder value.   These strategic alternatives could include, among other alternatives, a potential sale of assets of the Company, a potential licensing of assets of the Company, a sale of the Company, a business combination, a merger or other strategic action. The Company

      5/8/25 8:30:15 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Vor Bio Provides Clinical Update Further Validating Approach of Using Shielded Transplants to Deliver Targeted Therapies; Receives Supportive Feedback from FDA Regarding Registrational Trial Design

      Preliminary data suggests improved relapse-free survival compared to published groups of acute myeloid leukemia (AML) patients at high risk of relapse post-transplantTrem-cel + Mylotarg continue to demonstrate engraftment, shielding, and broadened therapeutic windowCompany has received supportive feedback from the FDA regarding a registrational clinical trial design CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today announced updated clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™. The data, which was presented in a post

      12/9/24 7:30:00 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • New Clinical Data Validates Vor Bio's Approach of Using Shielded Transplants to Deliver Targeted Therapies

      Trem-cel + Mylotarg demonstrated engraftment, shielding, broadened therapeutic window, and patient benefitVCAR33ALLO demonstrates encouraging biomarker data at lowest dose New asset VADC45 with significant potential opportunities across oncology, gene therapy, and autoimmune disorders CAMBRIDGE, Mass., Sept. 05, 2024 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today announced new clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™. The data demonstrated reliable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic wind

      9/5/24 4:01:00 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $VOR
    Insider Purchases

    Insider purchases reveal critical bullish sentiment about the company from key stakeholders. See them live in this feed.

    See more
    • Large owner Reprogrammed Interchange Llc bought 38,974,101 shares (SEC Form 4)

      4 - Vor Biopharma Inc. (0001817229) (Issuer)

      1/2/25 7:42:04 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $VOR
    Press Releases

    Fastest customizable press release news feed in the world

    See more
    • Vor Bio Announces Exploration of Strategic Alternatives to Maximize Shareholder Value

      CAMBRIDGE, Mass., May 08, 2025 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today announced that, based on currently available clinical data from its key clinical programs and a challenging fundraising environment, the Board of Directors has approved the initiation of a process to explore a range of strategic alternatives aimed at maximizing both near- and long-term shareholder value.   These strategic alternatives could include, among other alternatives, a potential sale of assets of the Company, a potential licensing of assets of the Company, a sale of the Company, a business combination, a merger or other strategic action. The Company

      5/8/25 8:30:15 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • PureTech Announces Annual Results for Year Ended December 31, 2024

      Innovation engine drives meaningful clinical, regulatory, and financial milestones, including positive Phase 2b results for wholly-owned deupirfenidone (LYT-100) in IPF, compelling Phase 1b data for wholly-owned LYT-200 in AML and solid tumors, FDA approval of PureTech-invented Cobenfy™1 for schizophrenia, and rapid growth of Founded Entity2, Seaport Therapeutics, which raised over $325 million Capital-efficient operations support robust balance sheet with PureTech level cash, cash equivalents, and short-term investments of $366.8 million3 and consolidated cash, cash equivalents, and short-term investments of $367.3 million4 as of December 31, 2024, with operational runway into at least 20

      4/30/25 2:00:00 AM ET
      $PRTC
      $VOR
      Biotechnology: Pharmaceutical Preparations
      Health Care
      Biotechnology: Biological Products (No Diagnostic Substances)
    • Vor Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Company Update

      VCAR33 clinical data update planned for first half of 2025 and trem-cel + Mylotarg clinical data update planned for second half of 2025 Anticipate initiation of the first trem-cel+VCAR33 Treatment System clinical trial in second half of 2025$55.6 million private placement completed in December 2024 CAMBRIDGE, Mass., March 20, 2025 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period and full year ended December 31, 2024, and provided a business update.  "We continue to make significant strides in advancing our novel cell and genome engineering platform. The continued progress in our cl

      3/20/25 4:05:00 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $VOR
    Insider Trading

    Insider transactions reveal critical sentiment about the company from key stakeholders. See them live in this feed.

    See more
    • PRESIDENT AND CEO Ang Robert covered exercise/tax liability with 2,361 shares, decreasing direct ownership by 0.26% to 911,318 units (SEC Form 4)

      4 - Vor Biopharma Inc. (0001817229) (Issuer)

      5/7/25 4:05:57 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Chief Sci Off & Head Tech Ops Chakraborty Tirtha covered exercise/tax liability with 876 shares, decreasing direct ownership by 0.40% to 217,416 units (SEC Form 4)

      4 - Vor Biopharma Inc. (0001817229) (Issuer)

      5/7/25 4:05:00 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Chief Financial Officer Choi Han Wook covered exercise/tax liability with 2,061 shares, decreasing direct ownership by 4% to 51,280 units (SEC Form 4)

      4 - Vor Biopharma Inc. (0001817229) (Issuer)

      5/2/25 4:30:56 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $VOR
    Leadership Updates

    Live Leadership Updates

    See more
    • Vor Bio Appoints Life Sciences and Technology Investor and Entrepreneur, Mr. Erez Kalir to its Board of Directors

      CAMBRIDGE, Mass., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today announced the appointment of Mr. Erez Kalir to its Board of Directors. The appointment represents a new seat on Vor Bio's Board of Directors, which has been created in connection with the recent private investment in public equity financing (PIPE) which was led by Reid Hoffman. Mr. Kalir will serve as the director designee of Reprogrammed Interchange, LLC, Mr. Hoffman's investment fund. Mr. Kalir brings a wealth of experience as an investor, entrepreneur, and thought leader with a deep focus on life sciences and technology. Currently the Managing Member of

      1/8/25 9:00:00 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Vor Bio Appoints Seasoned Healthcare Institutional Investor, Han Choi, M.D., LL.M., as Chief Financial Officer

      CAMBRIDGE, Mass., Sept. 30, 2024 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today announced the appointment of Han Choi, M.D., LL.M., as its new Chief Financial Officer, effective immediately. Dr. Choi will join the Vor Bio leadership team and will report directly to the company's President and Chief Executive Officer, Dr. Robert Ang. Dr. Choi brings to Vor Bio over twenty-five years of experience in public and private investment management, business development, and corporate strategy within the pharmaceutical and biotechnology industry. His deep expertise in structuring corporate partnerships and investments, managing complex transact

      9/30/24 7:00:00 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Vor Bio Appoints Accomplished Oncology and Cancer Immunotherapy R&D Executive, Fouad Namouni, M.D., to its Board of Directors

      CAMBRIDGE, Mass., May 02, 2024 (GLOBE NEWSWIRE) -- Vor Bio (NASDAQ:VOR), a clinical-stage cell and genome engineering company, today announced the appointment of Fouad Namouni, M.D., to its Board of Directors. Dr. Namouni currently serves as President of Research & Development at Blueprint Medicines, bringing a wealth of industry experience and expertise to Vor Bio's Board. In his role at Blueprint Medicines, Dr. Namouni has demonstrated exceptional leadership in building the company's fully integrated business and advancing a broad pipeline of innovative medicines to address significant medical needs in oncology/hematology and allergy/inflammation. With over two decades of experience in

      5/2/24 4:01:00 PM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care

    $VOR
    Analyst Ratings

    Analyst ratings in real time. Analyst ratings have a very high impact on the underlying stock. See them live in this feed.

    See more
    • Wedbush initiated coverage on Vor Biopharma with a new price target

      Wedbush initiated coverage of Vor Biopharma with a rating of Outperform and set a new price target of $18.00

      7/26/22 8:25:46 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • Goldman initiated coverage on Vor Biopharma with a new price target

      Goldman initiated coverage of Vor Biopharma with a rating of Neutral and set a new price target of $6.00

      4/27/22 6:14:19 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care
    • HC Wainwright & Co. initiated coverage on Vor Biopharma with a new price target

      HC Wainwright & Co. initiated coverage of Vor Biopharma with a rating of Buy and set a new price target of $26.00

      12/17/21 6:23:13 AM ET
      $VOR
      Biotechnology: Biological Products (No Diagnostic Substances)
      Health Care