Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug…
By daniellenierenberg
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced that new results from its CD117-ADC patient preparation program were presented at the 61st Annual Meeting of the American Society of Hematology (ASH). These results, which were highlighted in an oral presentation at ASH by John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, showed the first-ever successful transplant of gene-modified cells in non-human primates using a targeted, single-agent antibody-drug conjugate (ADC), without the use of chemotherapy or radiation.
Todays conditioning regimens involve high doses of chemotherapy, often paired with radiation, to remove the disease-causing cells. As a result, patients undergoing gene therapy or stem cell transplant are all faced with a difficult choice: whether to endure severe toxicity and risk infertility and cancer for the chance for a cure. Magentas portfolio of targeted ADCs represents an extremely promising new option to prepare patients for gene therapy or transplant with no need for toxic chemotherapy or radiation, said Dr. Tisdale. The results presented today show that a single dose of single agent CD117-ADC achieves the same level of depletion as four doses of busulfan chemotherapy to enable successful engraftment and persistence of stem cells modified with the -globin gene, the gene that causes sickle cell disease and -thalassemia when mutated. Importantly, the animals undergoing preparation with CD117-ADC showed none of the damaging toxicities associated with busulfan conditioning.
Magenta is the only company with the people, platforms and a product engine committed to comprehensively transforming immune and blood system reset, which includes revolutionizing the toxic methods that are used to prepare patients for gene therapy and transplant today. said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. The gene therapy field has learned that higher levels of stem cell depletion, which meant higher doses of busulfan, were needed to ensure long-term engraftment of the gene-modified cells and persistence of gene therapy. Across all the modalities we have tested, we have seen that ADCs are most effective at achieving these high levels of stem cell depletion without chemotherapy to enable engraftment and long-term durability of the transplant. Todays impressive results provide important validation of the ADC approach as well as the CD117 target for patient preparation and underscore Magentas leadership in the field of conditioning.
Results from the CD117-ADC Patient Preparation Program
Title: A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates (Abstract #610)Presenter: John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, Bethesda, Md.
Magentas most advanced patient preparation program, CD117-ADC, targets CD117, a protein expressed on hematopoietic stem cells. CD117-ADC is designed to remove the genetically mutated cells in the bone marrow that cause certain genetic diseases, such as sickle cell disease, enabling curative stem cell transplant or gene therapy.
Results presented by Dr. Tisdale showed:
About Magenta Therapeutics
Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.
Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.
Follow Magenta on Twitter: @magentatx.
Forward-Looking Statement
This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Registration Statement on Form S-1, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.
View source version on businesswire.com: https://www.businesswire.com/news/home/20191209005253/en/
Go here to read the rest:
Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug...
- 001 Expansion of Stem Cells by Valproic Acid [Last Updated On: June 24th, 2011] [Originally Added On: June 24th, 2011]
- 002 Cord Blood and Bone Marrow Stem Cells for Liver Failure [Last Updated On: June 25th, 2011] [Originally Added On: June 25th, 2011]
- 003 Science behind Enhancing Adult Stem Cells for wellbeing [Last Updated On: June 26th, 2011] [Originally Added On: June 26th, 2011]
- 004 Stem cells Transplatation in Completed Paralyze Dog. [Last Updated On: June 26th, 2011] [Originally Added On: June 26th, 2011]
- 005 STEM CELLS - Bone Marrow Stem Cells (Balzitt).flv [Last Updated On: June 27th, 2011] [Originally Added On: June 27th, 2011]
- 006 Bone Marrow Stem Cells/Gene Therapy [Last Updated On: June 28th, 2011] [Originally Added On: June 28th, 2011]
- 007 Stem cells used for medical treatment [Last Updated On: June 30th, 2011] [Originally Added On: June 30th, 2011]
- 008 STEM CELLS FOR OTHER USES Interview with Sam Smith.wmv [Last Updated On: July 1st, 2011] [Originally Added On: July 1st, 2011]
- 009 Stem Cells Reversing Endothelial Senescence [Last Updated On: July 2nd, 2011] [Originally Added On: July 2nd, 2011]
- 010 Bone Marrow Stem Cells [Last Updated On: July 3rd, 2011] [Originally Added On: July 3rd, 2011]
- 011 Bone marrow transplantation HD, ENG subtitles [Last Updated On: July 5th, 2011] [Originally Added On: July 5th, 2011]
- 012 Adult Stem Cells May Target and Repair Heart Attack Damage [Last Updated On: July 6th, 2011] [Originally Added On: July 6th, 2011]
- 013 Bone Marrow Stem Cell Applications [Last Updated On: July 8th, 2011] [Originally Added On: July 8th, 2011]
- 014 Stem cells in bone marrow are being used to treat EB [Last Updated On: July 13th, 2011] [Originally Added On: July 13th, 2011]
- 015 STEM CELLS FOR MACULAR DEGENERATION Sam Smith's story.wmv [Last Updated On: July 14th, 2011] [Originally Added On: July 14th, 2011]
- 016 MS Cure - Progression [Last Updated On: July 14th, 2011] [Originally Added On: July 14th, 2011]
- 017 Blind Girl get's cure you need to see to believe" [Last Updated On: July 17th, 2011] [Originally Added On: July 17th, 2011]
- 018 From Surgical Repair to Stem Cell Repair: A Surgeon's Journey by Leonard Smith MD, FACS [Last Updated On: July 19th, 2011] [Originally Added On: July 19th, 2011]
- 019 Bone Marrow Stem Cell Donation [Last Updated On: July 19th, 2011] [Originally Added On: July 19th, 2011]
- 020 The potential of stem cells [Last Updated On: July 28th, 2011] [Originally Added On: July 28th, 2011]
- 021 LifeCell in Kalaignar Seithigal,Sun News [Last Updated On: July 31st, 2011] [Originally Added On: July 31st, 2011]
- 022 Spirulina DLA Naturals [Last Updated On: August 6th, 2011] [Originally Added On: August 6th, 2011]
- 023 Bill Lahti; stem cell nutrition stimulates wound repair for diabetic stroke victim [Last Updated On: August 6th, 2011] [Originally Added On: August 6th, 2011]
- 024 Christian Drapeau Talk About - Adult Stem Cells and StemEnhance./StemTech [Last Updated On: August 7th, 2011] [Originally Added On: August 7th, 2011]
- 025 "Bone Marrow Stem Cells" Donald Kohn [Last Updated On: August 14th, 2011] [Originally Added On: August 14th, 2011]
- 026 Cancer Update: Autologus Stem Cell (Bone Marrow) Transplant [Last Updated On: September 4th, 2011] [Originally Added On: September 4th, 2011]
- 027 Bone Marrow / Stem Cell Transplant Recovery Fund [Last Updated On: September 6th, 2011] [Originally Added On: September 6th, 2011]
- 028 Bone Marrow/Stem Cell Transplantation: An Introduction, With Sonali Smith, MD [Last Updated On: September 9th, 2011] [Originally Added On: September 9th, 2011]
- 029 Bone Marrow Stem Cell Transplant Live from Top US Hospital [Last Updated On: September 12th, 2011] [Originally Added On: September 12th, 2011]
- 030 Adult Stem Cell Mobilization from Bone Marrow (Animation) [Last Updated On: September 12th, 2011] [Originally Added On: September 12th, 2011]
- 031 MS Cure - Introduction to stem cell bone marrow transplant in Australia [Last Updated On: September 13th, 2011] [Originally Added On: September 13th, 2011]
- 032 Immune Modulation by Bone Marrow Mesenchymal Stem Cells [Last Updated On: September 14th, 2011] [Originally Added On: September 14th, 2011]
- 033 Bone Marrow/Stem Cell Transplant [Last Updated On: September 15th, 2011] [Originally Added On: September 15th, 2011]
- 034 Bone Marrow and Stem Cell Transplant Patients Share Their Stories [Last Updated On: September 20th, 2011] [Originally Added On: September 20th, 2011]
- 035 Stem Cell Transplant India,Bone Marrow Transplant India,Sickle Cell Anemia Treatment India [Last Updated On: September 20th, 2011] [Originally Added On: September 20th, 2011]
- 036 Bone Marrow Stem Cell Expansion by HOXB4 and p21 Knock Out [Last Updated On: September 21st, 2011] [Originally Added On: September 21st, 2011]
- 037 Multiple Sclerosis, Stem Cells, and Hope, Part 2 [Last Updated On: September 22nd, 2011] [Originally Added On: September 22nd, 2011]
- 038 Becoming a Blood Stem Cell Donor [Last Updated On: September 24th, 2011] [Originally Added On: September 24th, 2011]
- 039 LittleBigPlanet 2 - Stem Cell Sackboy Quarter 4 Update (Bone Marrow Bugaloo) [Last Updated On: September 25th, 2011] [Originally Added On: September 25th, 2011]
- 040 Can Stem Cell Prolotherapy or Bone Marrow Prolotherapy help articular cartilage defects? [Last Updated On: September 27th, 2011] [Originally Added On: September 27th, 2011]
- 041 Mesenchymal stem cells and marrow stromal cells [Last Updated On: October 1st, 2011] [Originally Added On: October 1st, 2011]
- 042 Bone Marrow Transplant and Stem Cell Transplant Treatment in India for International patients [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 043 Bone Marrow Stem Cell Aspiration and Re-Injection with PRP for Osteoarthritis by Dr Adelson [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 044 Selected Donor for Stem Cell, Bone Marrow [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 045 Stem Cell Injections - Bone Marrow Prolotherapy - treatment for arthritis [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 046 Nurses Discuss Special Bonds With Bone Marrow and Stem Cell Transplant Patients [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 047 Hagen's bone marrow collection part 1 [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 048 Samsparks after bone marrow collection for stem cell treatm [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 049 Chat w/ Dr. Maharaj, founder of S. FL. Bone Marrow/Stem Cell Transplant Institute [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 050 Bone Marrow - Stem Cell Prolotherapy [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 051 MS Cure - Hickman line Insertion for stem cell/bone marrow transplant [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 052 Bone Marrow Transplant Program Continues to Grow, Make a Difference [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 053 Stem cell transplant in children: what to expect | Dana-Farber Cancer Institute [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 054 LittleBigPlanet 2 - (WIP) Stem Cell Sackboy Bone Marrow Bugaloo [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 055 Calum's stem cell donation for Anthony Nolan [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 056 Stem Cell Treatment in New Jersey- Colon's story (Dr Damon Noto) [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 057 How to be an Anthony Nolan blood stem cell donor [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 058 Dr Steve talks about stem cells.mov [Last Updated On: October 5th, 2011] [Originally Added On: October 5th, 2011]
- 059 Stem Cells extracted from bone marrow [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- 060 Webinar 1, Stem cell therapy basics, what is available today? [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- 061 Mesenchymal stem cells and marrow stromal cells---2nd--- [Last Updated On: October 8th, 2011] [Originally Added On: October 8th, 2011]
- 062 Harvesting stem cells from horse bone marrow at UT [Last Updated On: October 12th, 2011] [Originally Added On: October 12th, 2011]
- 063 Bone Marrow Stem Cells - Video [Last Updated On: October 13th, 2011] [Originally Added On: October 13th, 2011]
- 064 Stem Cell Transplant Apheresis Method - Video [Last Updated On: October 14th, 2011] [Originally Added On: October 14th, 2011]
- 065 Mantak Chia: Sumup stem cell (Berlin,Germany 2009) Part I : Bone marrow activate Stem cell - Video [Last Updated On: October 16th, 2011] [Originally Added On: October 16th, 2011]
- 066 Stem Cell, Bone Marrow Transplants / Donations - Easy and life saving proceedure - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- 067 HIV/AIDS: Spotlight on Stem Cell Research - Loren Leeds - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- 068 Sickle Cell Anemia: Stem Cell Gene Therapy - Donald Kohn - Video [Last Updated On: October 21st, 2011] [Originally Added On: October 21st, 2011]
- 069 Do you need a large number of stem cells injected for Bone Marrow Prolotherapy to work? - Video [Last Updated On: October 23rd, 2011] [Originally Added On: October 23rd, 2011]
- 070 What are stem cells? - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- 071 What is in situ Stem Cell Injection Therapy or Bone Marrow Prolotherapy? - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- 072 Stem Cells Research at Hadassah - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- 073 Blood or Bone Marrow Cells May Provide a Route to Healing Blood Vessels - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- 074 Hematopoietic stem cell transplantation #2 - Video [Last Updated On: October 28th, 2011] [Originally Added On: October 28th, 2011]
- 075 Radiation - Sickle Cell Half Match Stem Cell Bone Marrow Transplant 2 - Video [Last Updated On: November 7th, 2011] [Originally Added On: November 7th, 2011]
- 076 Sickle Cell Stem Cell Bone Marrow Transplant 3 - Video [Last Updated On: November 7th, 2011] [Originally Added On: November 7th, 2011]
- 077 Macular degeneration - Stem Cell therapy (English subtitles) - Video [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- 078 Where Do The Stem Cells Come From? Hollywood | Los Angeles - Video [Last Updated On: November 13th, 2011] [Originally Added On: November 13th, 2011]
- 079 Bone Marrow Stem Cell Injections - Video [Last Updated On: November 14th, 2011] [Originally Added On: November 14th, 2011]
- 080 Diabetes- Stem cell therapy (english) - Video [Last Updated On: December 2nd, 2011] [Originally Added On: December 2nd, 2011]
