New Funding Announcement! Dr. Bacchetta, recieves CLIN_1 funding from CIRM for the LVFOXP3 IND-enabling studies!
October 31, 2019
CIRM awarded $5.53 million to Dr. Rosa Bacchetta at Stanford to complete work necessary to conduct a clinical trial for IPEX syndrome, a rare disease caused by mutations in the FOXP3 gene. Dr. Rosa Bacchetta and her team at Stanford will use gene therapy to insert a normal version of the FOXP3 gene into the patient’s own T Cells to restore the normal function of regulatory T Cells.
Dr. Porteus, Co-Director of CDCM, obtains a Sickle Cell Disease/Advancing Award from the Doris Duke Charitable Foundation!
September 10, 2019
Dr. Porteus recieved an award for his project: Optimization of gene correction using genome editing in human hematopoietic cells.
Published Paper: "Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I" by one of CDCM's gene editing platform leaders, Natalia Gomez Ospina, MD, PhD
September 6, 2019
Congratulations to Dr. Gomez-Ospina and team for publishing "Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I".
CDCM Partner, Rocket Pharmaceuticals Announces Registration-Enabling Phase 2 Plans for RP-L102 Gene Therapy for Fanconi Anemia Following a Supportive End-of-Phase 1 FDA Meeting.
September 4, 2019
Based on feedback from a recent End-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA), Rocket plans to open enrollment for the U.S. Phase 2 trial of RP-L102 for FA in the fourth quarter of 2019.
The CDCM serves as the lead clinical site in the US for this study.
New Faculty Announcement!
July 1, 2019
We are delighted to announce that J. Fraser Wright has joined Stanford University as Professor of Pediatrics at the Center for Definitive and Curative Medicine (CDCM) beginning July 1, 2019. Dr. Wright will contribute expertise in vectorology, translational research, and clinical manufacturing and quality systems towards successful development of transformative stem cell and gene therapies discovered at the CDCM and the Institute for Stem Cell Biology and Regenerative Medicine.
For Dr. Wright's bio please visit: http://med.stanford.edu/cdcm/about/Faculty.html
Interview with Dr. Oro
Abril 17, 2019
Dr. Oro was recently interviewed on "The Future of Everything" by Stanford School of Engineering. Dr. Oro discussed the CDCM and the work being done to ethically bring cell and gene therapies to those affected by genetic diseases.
3rd Annual CDCM Symposium huge success!
March 19, 2019
The Center for Definitive and Curative Medicine (CDCM) held its 3rdannual symposium on March 19, breaking previous years’ attendance records with over 600 registrants. Berg Hall was at capacity most of the day with attendees from academia, biotech and pharma. The keynote addresses included Nobel laureate and former President of Caltech David Baltimore, President, Chief Executive Officer and Co-Founder of Allogene Therapeutics David Chang, as well as head of the FDA’s Center for Biologics Evaluation and Research (CBER) Peter Marks and founder of the Fanconi Anemia Research Fund, Lynn Frohnmayer. The agenda included a broad spectrum of talks that covered topics from new cell-based medicine discoveries to pre-clinical development to clinical trials, as well as the challenges in development and commercialization. The event highlighted the growth in the clinical trial pipeline of the CDCM with the innovative cell and gene therapies both open and soon-to-be-open at Stanford. The team looks forward to setting new records again next Spring.
Rocket FA Clinical Trial
Stanford’s Center for Definitive and Curative Medicine (CDCM) has treated the first patient in an open-label, Phase 1 clinical trial of RP-L102, Rocket Pharmaceuticals’ lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA). This is the first stem cell gene therapy trial to be opened at Stanford, which serves as the lead U.S. clinical site. The trial is expected to enroll two FA pediatric patients at the CDCM with the goal to evaluate “Process B” RP-L102 which incorporates a modified cell enrichment process, transduction enhancers, and commercial-grade vector manufacturing and cell processing.
New Discoveries in Blood Stem Cell Transplantations!
Hope for organ transplantation without a matching donor and one-time treatment/lifetime cure for many blood and immune diseases
February 6, 2019. Agnieszka Czechowicz, MD, PhD, and her colleagues at the Stanford Institute for Stem Cell Biology and Regenerative Medicine (ISCBRM) published a pair of papers that demonstrate a new method for safely preparing for blood stem cell transplantation in mice, opening the door for a much wider use of the method.
NPR: The Human Connection: Featuring a story about Dr. Roncarolo and one of her patients.
February 5, 2019. Please listen to this wonderful story told by Suzette Stone regarding the human connection Dr. Roncarolo prioritizes while treating her patients.
--Suzette Stone is a Pediatric Nurse Practitioner who works with children and their families undergoing Stem Cell Transplant on the Peninsula.
"Human Nature": New Documentary Featuring Dr. Porteus!
A CRISPR documentary film that will feature Dr. Matthew Porteus will premiere at the SXSW Film Festival in Austin, TX in early March 2019!
Please visit the "Human Nature" website for official listings and screening dates.
Rocket Pharmaceuticals Announces FDA Clearance of IND Application for RP-L102 Gene Therapy for Fanconi Anemia
NEW YORK – November 7, 2018 - Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT)
- Patients to Be Treated with RP-L102 under “Process B” Incorporating Higher Cell Doses, Transduction Enhancers, and Commercial-grade Vector Manufacturing and Cell Processing –
- U.S. Trial to Commence Early 2019; No Conditioning Required-
- Center for Definitive and Curative Medicine at Stanford University School of Medicine to Lead U.S. Clinical Studies -
California Institute for Regenerative Medicine (CIRM) Funding Announcement
July 20, 2018
The project, Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome, aims to develop a platform to generate transplantable thymus organoids derived from human pluripotent stem cells. These tissues are designed to treat severe immunodeficiencies in children affected by this rare disease. There are currently no treatments available for patients with 22q11DS, which means there is an urgent need for alternative therapies. These children remain in isolation given their compromised immune systems, and if not treated, have a life expectation of just two years. This research project sets the preclinical scientific foundation to develop a curative therapy for patients while also significantly advancing the field of iPS-derived tissue engineering.
Congratulations to Vittorio Sebastiano and Katja Weinacht for being awarded a CIRM grant to develop a therapeutic product for children with 22q11 Deletion Syndrome!
Maria Grazia-Roncarolo, MD, appointed President of FOCIS
June 22, 2018
Maria Grazia-Roncarolo, MD appointed President of FOCIS! The Federation of Clinical Immunology Societies (FOCIS) exists to improve human health through immunology by fostering interdisciplinary approaches to both understand and treat immune-based diseases.
Rocket Pharma Collaboration
May 17, 2018
The Center for Definitive and Curative Medicine (CDCM) at Stanford Medicine has entered into a collaborative research agreement with Rocket Pharmaceuticals, Inc. (www.rocketpharma.com) to further the CDCM’s mission of curing the seemingly incurable. The agreement outlines how the two entities will collaborate in their shared mission to advance gene therapy research, with Stanford named as the lead US-based clinical trial research center for two programs targeting rare orphan diseases: Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD).