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...
- Bone Marrow Donors Can Be Hard to Find. One Company Is Turning to ... - November 15th, 2024
- Hematopoietic Stem Cells and Their Niche in Bone Marrow - November 15th, 2024
- Bone Marrow Transplant Program - Overview - Mayo Clinic - November 15th, 2024
- Bone Marrow Donors Can Be Hard to Find. One Company Is Turning to Cadavers - WIRED - November 15th, 2024
- More stem cells for sickle cell gene therapy readied with motixafortide - Sickle Cell Disease News - November 15th, 2024
- Skull bone marrow expands throughout life and remains healthy during aging, researchers discover - Medical Xpress - November 15th, 2024
- Adult skull bone marrow is an expanding and resilient haematopoietic reservoir - Nature.com - November 15th, 2024
- Evaluation of standard fludarabine dosing and corresponding exposures in infants and young children undergoing hematopoietic cell transplantation -... - November 15th, 2024
- Stem cells grown in space show super powers but theres a catch - Study Finds - November 15th, 2024
- Getting a Stem Cell or Bone Marrow Transplant - October 21st, 2024
- Acquisition of durable insulin-producing cells from human adipose tissue-derived mesenchymal stem cells as a foundation for cell- based therapy of... - October 21st, 2024
- 1.5 Lakh Indians Register To Save Lives: Join the Mission To Fight Blood Cancer - The Better India - October 21st, 2024
- How Stem Cell and Bone Marrow Transplants Are Used to Treat Cancer - October 13th, 2024
- Stem Cell (Bone Marrow) Transplants - MD Anderson Cancer Center - October 13th, 2024
- Donating Bone Marrow and Stem Cells: The Process and What To Expect - October 13th, 2024
- What to expect as a stem cell or bone marrow donor - October 13th, 2024
- Structural organization of the bone marrow and its role in ... - October 13th, 2024
- Stem cell donor from down the road saved my life after global search - BBC.com - September 23rd, 2024
- Awaiting the call: family hopes to find blood stem cell donor - Claremont Courier - September 23rd, 2024
- Michigan woman one of first in world to successfully receive bone marrow from deceased donor - WDIV ClickOnDetroit - September 23rd, 2024
- Next-generation stem cell transplant: Revolutionizing a lifesaving cancer therapy - The Business Journals - September 23rd, 2024
- Sophie's life was saved by a stranger. Some in her position have an 'unfair' disadvantage - SBS News - September 23rd, 2024
- What Are Leukemia and Lymphoma and How Are They Treated? - LVHN News - September 23rd, 2024
- Giralt on MDS Transplant Timing and Candidacy - Targeted Oncology - September 14th, 2024
- Aging is associated with functional and molecular changes in distinct hematopoietic stem cell subsets - Nature.com - September 14th, 2024
- A practical guide to therapeutic drug monitoring in busulfan: recommendations from the Pharmacist Committee of the European Society for Blood and... - September 14th, 2024
- ISU researcher blown away by blood cell replication discovery - Radio Iowa - September 14th, 2024
- Pausing biological clock could give boost to lab-produced blood stem cells - Phys.org - September 14th, 2024
- 9-year-old gets successful bone marrow transplant - The Times of India - September 14th, 2024
- Dr. Crandall: Stem Cell Treatment Heals the Heart - Newsmax - September 3rd, 2024
- Orion Corporation: Managers’ transactions – Hao Pan - August 19th, 2024
- BioCorRx Reports Business Update for the Second Quarter of 2024 - August 19th, 2024
- Tevogen Bio Reports Second Quarter 2024 Financial Results, Eliminates Doubt About Company’s Ability to Continue as a Going Concern, Eliminates... - August 19th, 2024
- Aligos Therapeutics Announces Reverse Stock Split - August 19th, 2024
- Lumos Pharma to Participate in H.C. Wainwright 26th Annual Global Investment Conference - August 19th, 2024
- Protect Pharmaceutical Corp. (PRTT) Announces New CEO and New Director; Moves to Finalize the Karinca Logistics Merger - August 19th, 2024
- OKYO Pharma Participates in H.C. Wainwright 4th Annual Ophthalmology Virtual Conference - August 19th, 2024
- CORRECTION – Tevogen Bio Reports Second Quarter 2024 Financial Results, Eliminates Doubt About Company’s Ability to Continue as a Going Concern,... - August 19th, 2024
- NurExone Biologic Achieves Key Milestone in Support of Robust Exosome Manufacturing Process - August 19th, 2024
- Silexion Therapeutics Ltd. and Moringa Acquisition Corp Announce Closing of their Business Combination - August 19th, 2024
- Vericel Announces FDA Approval of NexoBrid for the Treatment of Pediatric Patients with Severe Thermal Burns - August 19th, 2024
- Codexis Publishes FY2023 Sustainability Disclosures - August 19th, 2024
- MediWound Announces U.S. Food and Drug Administration Approval of NexoBrid® for the Treatment of Pediatric Patients with Severe Thermal Burns - August 19th, 2024
- First Successful Paediatric Allogeneic Bone Marrow Transplant In Bengaluru; Know All About The Procedure - Onlymyhealth - August 4th, 2024
- Is Stem Cell Transplant Often The Only Treatment Option For Blood Cancer Patients? Why So? - News18 - June 2nd, 2024
- This Swedish startup wants to reduce the cost, and controversy, around stem cell production - TechCrunch - March 10th, 2024
- Bone Marrow Transplantation | Johns Hopkins Medicine - December 20th, 2023
- Mansour bin Zayed witnesses inauguration of ADSCC Bone Marrow Transplant & Cellular Therapy Congress 2023 - ZAWYA - November 26th, 2023
- ADSCC Bone Marrow Transplant and Cellular Therapy Congress 2023 to take place in Abu Dhabi - ZAWYA - November 18th, 2023
- Orchard Therapeutics Reports First Quarter 2023 Financial Results and Announces Initiation of Rolling Submission for Biologics License Application of... - May 16th, 2023
- Family of 7-month-old in need of bone marrow transplant hosting donor registration event - CBS Pittsburgh - May 8th, 2023
- Anika Continues to Expand Addressable Market for Tactoset Injectable Bone Substitute with Additional 510(k) Clearance from FDA - Marketscreener.com - April 5th, 2023
- MorphoSys Completes Enrollment of Phase 3 MANIFEST-2 Study of Pelabresib in Myelofibrosis with Topline Results Expected by End of 2023 -... - April 5th, 2023
- VOR BIOPHARMA INC. Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-K) - Marketscreener.com - March 25th, 2023
- BioRestorative Therapies to Seek FDA Approval to Expand the Clinical Application of BRTX-100 - Marketscreener.com - March 17th, 2023
- BioSenic delivers a new post-hoc analysis of its Phase III JTA-004 trial on knee osteo-arthritis with positive action on the most severely affected... - March 17th, 2023
- JASPER THERAPEUTICS, INC. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS (form 10-K) - Marketscreener.com - March 9th, 2023
- For a range of unmet medical needs, India offers a fantastic opportunity to push cell and gene therapies: B .. - ETHealthWorld - March 9th, 2023
- NGM BIOPHARMACEUTICALS INC Management's Discussion and Analysis of Financial Condition and Results of Operations. (form 10-K) - Marketscreener.com - March 1st, 2023
- Bone health: Tips to keep your bones healthy - Mayo Clinic - January 27th, 2023
- Bone marrow drive held for military wife with cancer - January 27th, 2023
- Bone cancer - Symptoms and causes - Mayo Clinic - January 27th, 2023
- Bone | Definition, Anatomy, & Composition | Britannica - January 19th, 2023
- Bone Definition & Meaning - Merriam-Webster - January 19th, 2023
- What Is Bone? | NIH Osteoporosis and Related Bone Diseases National ... - January 19th, 2023
- Anatomy of the Bone | Johns Hopkins Medicine - January 19th, 2023
- Bone Health: Is Eating Meat Healthy For Your Bones? - January 19th, 2023
- Bone Keeper | Deepwoken Wiki | Fandom - January 19th, 2023
- With blood and plasma donations in short supply, uniting communities to give the gift of life - Toronto Star - January 3rd, 2023
- Side Effects of a Bone Marrow Transplant (Stem Cell Transplant) - December 25th, 2022
- 28-year-old cancer patient at Nebraska Medicine advocates for diversity in bone marrow registry - KMTV 3 News Now Omaha - December 17th, 2022
- Stem Cell Technologies and Applications Market Report 2022-2032 - Yahoo Finance - December 9th, 2022
- Fred Hutch at ASH: Global insights on AML outcomes, COVID-19 and cancer, CD19 CAR T-cell therapy updates, latest on precision oncology and more -... - December 9th, 2022
- Types of Stem Cell and Bone Marrow Transplants - American Cancer Society - December 1st, 2022
- Getting a Stem Cell or Bone Marrow Transplant - American Cancer Society - December 1st, 2022
- Woman, 41, With Bubbles In Her Urine Dismissed By Doctors. Turns Out To Have The Blood Cancer Multiple Myeloma. - SurvivorNet - December 1st, 2022
- Stem cell and bone marrow transplants - Cancer Research UK - November 22nd, 2022
- Donating Bone Marrow Experience | Be The Match - November 22nd, 2022
- Learn How to Donate Bone Marrow | Be The Match - October 29th, 2022
- Stem Cell Transplantation Program - DanaFarber Cancer Institute - October 29th, 2022