Natalia Gomez-Ospina, MD, PhD Awarded $5,444,353 for Late-Stage Preclinical Program by CIRM
January 20, 2023
Stanford University's Natalia Gomez-Ospina, MD, PhD and her team were awarded $5,444,353 by The California Institute for Regenerative Medicine (CIRM) for their late-stage preclinical trial targeting Hurler Syndrome. Hurler Syndrome, caused by a faulty gene, is an inherited condition that results in the lack of an enzyme needed to digest sugar. Undigested sugar then builds up in the body and causes progessive damage to the brain, heart, and other organs.
The goal of this preclinical program is to showcase the team's capability of manufacturing the necessary stem cells to restore the missing enzyme while completing safety studies and applying clinical trial authorization from the FDA. You can read more here.
Joseph C. Wu, MD, PhD Named a Fellow of National Academy of Inventors
December 13, 2022
Stanford University's Joseph C. Wu, MD, PhD was among three faculty that were named Fellows of the National Academy of Inventors. This honor is the highest professional distinction awarded to academic inventors. Dr. Joseph C. Wu was recognized for his lab's work "on biological mechanisms of patient-specific and disease-specific induced pluripotent stem cells." You can read more here.
The National Academy of Inventors states that the 2022 Fellow class is comprised of 110 research universities and governmental and nonprofit research institutions globally. As a whole, the 2022 class holds over 5,000 U.S. patents.
Stanford-born Therapy Reaches Clinical Milestone
November 3, 2022, New York and Cleveland
Abeona Therapeutics has announced positive data in its Phase 3 VIITAL study, which assesses the safety and efficacy of EB-101 for treatment of recessive dystrophic epidermolysis bullosa (RDEB). The study met its two co-efficacy endpoints showing statistically significant, clinically meaningful improvements in healing wounds and reducing pain for large chronic RDEB wounds.
The Phase 3 VIITAL study evaluated the safety, tolerability, and efficacy of EB-101 in 43 large wound pairs across 11 RDEB subjects. At six months, EB-101 significantly improved wound healing and pain reduction when compared to the control.
EB-101 was also shown to be tolerated well by the subjects, with no serious treatment-related adverse events observed. Stanford University’s Jean Tang, M.D., Ph.D, the Principal Investigator of the study, states that while EB-101 has been shown to significantly heal and reduce pain, the Phase 1/2a study will continue to monitor its clinical benefits with up to eight years of follow-up.
Following the positive study results, Abeona plans to submit a Biologics License Application (BLA) for EB-101 to the FDA for approval.
Join us at the 7th Annual CDCM Symposium to hear more about this therapy’s journey from bench to bedside!
Registration for the CDCM 7th Annual Symposium is now Open!
We are excited to announce that registration for the 2023 CDCM 7th Annual Symposium is now open! (Please click here to register) For more information and updates, please visit our events page here.
We look forward to welcoming everyone back to an in-person format after 2 years of virtual conferences!
Anthony Oro, MD, PhD Elected to the National Academy of Medicine
October 24, 2022
Stanford University's Anthony Oro, MD, PhD was among 100 members newly elected to the National Academy of Medicine for "solidifying the first link between Hedgehog signaling and human cancer and building chromatin maps identifying how environmental factors drive tumor epigenetic plasticity and drug-resistance."
Dr. Anthony Oro was among 6 professors at Stanford University to be elected to the National Academy of Medicine, which recognized individuals that have "made major contributions to the advancement of the medical sciences, health care and public health." You can find more information here.
Natalia Gomez-Ospina, MD, PhD Named as a Grantee in the Science Diversity Leadership Program
October 20, 2022
Stanford University's Natalia Gomez-Ospina, MD, PhD has been awarded a grant from the Science Diversity Leadership program for her work in Targeting Macrophage/Microglia Niches with Engineered Stem Cells to Treat Disease. Her work "aims to develop novel conditioning tools that support the safe, efficient, and rapid repopulation of macrophage/microglia niches by bone marrow-derived cells and use them to treat metabolic diseases."
The Chan Zuckerberg Initiative partnered with the National Academies of Science, Engineering, and Medicine to launch the Science Diversity Leadership Program. This program is a funding initiative that recognizes biomedical researchers and awards them grants to further their leadership in "promoting diversity, equity and inclusion in their scientific fields." You can click here for more information.
Registration for the CDCM's 7th Annual Symposium will be Opening Soon
September 13, 2022
Please visit the event's webpage for updates/more information!
Ginger Exley Receives the Donna Schurr Spirit Award
June 30, 2022
The CDCM would like to congratulate and recognize Ginger Exley for receiving the Donna Schurr Spirit Award last Friday. This staff honor is awarded to individuals based on faculty and staff nominations. Everyday, Ginger works to support the CDCM in many administrative facets, which help build the foundation for our work. As such, Ginger is a vital component of the CDCM and instrumental in ensuring that the department operates as smoothly as possible. Thank you Ginger for tirelessly supporting the CDCM and all of us! We greatly appreciate all of your contributions!
Jasper Therapeutics Announces Treatment of First Patient in JSP191 Conditioning Study
May 17, 2022
The CDCM's collaboration with Jasper Therapeutics has resulted in the successful launch of an investigator-initiated clinical trial treating patients with Fanconi Anemia. Read the latest news here.
Rosa Bacchetta, MD Receives $12 Million in CIRM Funding for IPEX clinical trial
Oakland, February 24, 2022
The California Institute for Regenerative Medicine (CIRM) has invested approximately $12 million in a therapy being tested in a clinical trial to help patients that are affected by IPEX Syndrome. IPEX Syndrome is a rare autoimmune condition where an individual’s body cannot control their immune responses; this results in immune cells attacking their own healthy tissue. It is more commonly diagnosed in boys during the first year of life and is often fatal.
Dr. Rosa Bacchetta and her team have developed a therapy that utilizes a patient’s own CD4 T cells, which have been genetically modified to properly express the FOXP3 gene. Because the cells originate from the patient, there are less concerns regarding potential rejection and other fatal transplant complications. Dr. Bacchetta has received approval from the FDA to test this therapy in a Phase 1 clinical trial for affected patients (NCT05241444).
For more information, please contact Abinaya Nathan, Project Manager (firstname.lastname@example.org). The CIRM press release can be found here.
New publication describing cell-based product (T-allo10) enriched in antigen specific regulatory type 1 T (Tr1) cells
October 27, 2021
The Roncarolo lab has published a paper in Science Translational Medicine “Alloantigen-specific type 1 regulatory T cells suppress through CTLA-4 and PD-1 pathways and persist long-term in patients,” describing how antigen specific T regulatory type 1 (Tr1) cells, which are the active component of T-allo10 cell therapy product, can be used to induce prevent graft-versus-host disease, with significant improvement in the outcome of stem/progenitor cell transplantation from HLA incompatible donors. In the paper, lead authors Pauline Chen, MD, Alma-Martina Cepika, MD, PhD, and their colleagues reveal the molecular identity and mechanism of suppression of Tr1 cells, and report immune monitoring data from the first three patients with leukemia treated in a clinical trial with the T-allo10 product prior to stem/progenitor cell transplantation from an HLA mismatched donor. They show that the antigen specific Tr1 cells persist up to one year post-transplant. The type 1 regulatory T (Tr1) cells were originally discovered by the Roncarolo/Bacchetta team by studying tolerant SCID patients after fetal stem cell transplantation. This study is the first to describe the transcriptome and T cell receptor (TCR) repertoire of human alloantigen-specific Tr1 cells, show that these Tr1 cells suppress through IL-10, CTLA-4 and PD-1 pathways, and demonstrate their persistence in the blood of patients treated with T-allo10 cell therapy.
IND Clearance by FDA Received for IPEX Gene Therapy Clinical Trial
September 30, 2021
Congratulations to Rosa Bacchetta, MD, for receiving FDA clearance to use the Investigational New Drug (IND) product of autologous CD4LVFOXP3 in participants with IPEX Syndrome. Dr Bacchetta is the world’s leading expert on Immune-dysregulation polyendocrinopathy-enteropathy-X-linked (IPEX) syndrome, a life-threatening autoimmune disease that she aims to cure using this novel, first-in-human gene therapy. IPEX is a “prototype” genetic autoimmune disease, Dr. Bacchetta notes, because its genetic defect centers on lack of function of regulatory T cells, a critical blood cell type for the immune system. IPEX can reveal causes and underlying mechanisms of many different and more common immune-related diseases, such as Type 1 diabetes, inflammatory bowel disease, and allergies. Her work has been funded in large part by the California Institute for Regenerative Medicine(CIRM) and key studies were enabled by the generous philanthropic gifts of several donors, including Bonnie Uytengsu. This IND was enabled by the Center for Definitive and Curative Medicine’s team that spans bench to bedside, including lab scientists, clinical trials and regulatory experts in the Stem Cell and Gene Therapy Clinical Trials Program (SCGT-CTP), and process development and analytic staff in the Laboratory for Cell and Gene Medicine (LCGM). For more information about this program, please contact Abinaya Nathan, PhD, Project Manager (email@example.com).
Congratulations to Dr. Roncarolo for being a recipient of the 2021 Harrington Scholar-Innovator Award!
July 14, 2021
Dr. Roncaralo was one of eight physician-scientists from U.S. and Canada selected for novel treatments in numerous major disease areas. Recipients receive funding and drug development support from Harrington Discovery Institute to advance their research toward clinical impact.
Congratulations to Dr. Roncarolo for this award in the field of “New Cell Therapy for Transplantation.”
ASGCT: Forum on Gene Therapy for Underserved Populations
April 19, 2021
CDCM Co-Director, Matthew Porteus, is helping to organize a forum sponsored by the American Society of Gene and Cell Therapy (ASGCT) that will bring together key stakeholders and will be discussing a variety of novel models for the development of gene therapy for people with ultra-rare diseases and those in lower-income countries.
ASGCT: Forum on Gene Therapy for Underserved Populations
Drug Development for Ultra-Rare Diseases and Lower-Income Countries
Co-Chairs: Rachel Salzman, D.V.M. and Matthew Porteus, M.D., Ph.D.
Tuesday, June 22, 2021 | 11:30 a.m. - 4 p.m. CT
For more information and how to register for this event, please click here>>
IND Clearance Received for Sickle Cell Disease Gene Editing Clinical Trial
December 14, 2020
FDA Investigational New Drug (IND) clearance has been received to launch a phase I/II clinical trial of gene correction for sickle cell disease. This IND was developed at Stanford and has been transferred to Graphite Bio, a Bay Area start-up that will fund the clinical trial. The protocol was developed by the team led by Matthew Porteus, MD, PhD, Professor of Pediatrics, Stem Cell Transplantation and Regenerative Medicine and Co-Director of the CDCM, and represents what the CDCM aims to do: translate basic discoveries into therapies to cure diseases previously thought to be incurable. The team behind this tremendous milestone include bench scientists, analytic and process development staff in the Laboratory for Cell and Gene Medicine (LCGM), and regulatory experts in the Stem Cell and Gene Therapy Clinical Trials Office (SCGT-CTO). The trial is expected to open in Q1 2021 with Stanford being the lead site as well as the clinical manufacturing site of the cell product.
This project has been funded in part by the California Institute for Regenerative Medicine (CIRM), the generous gift of an anonymous donor, the NIH through the Cure Sickle Cell Initiative, and Graphite Bio. For more information about this program and others, please contact Angelica Martinez (firstname.lastname@example.org).
Read full press release here >>>
California Votes Yes on Proposition 14: California Stem Cell Research, Treatments and Cures Initiative of 2020
November 13, 2020
California voters vote Yes on Prop 14! This proposition provides needed funding to continue investing in the research and discovery of stem cell treatments and cures for chronic illnesses and conditions.
This is great news for stem cell research and finding cures for incurable diseases!
Dr. Porteus' article featured in a Nature Communications Editors’ Highlights webpage
May 18, 2020
The editors at Nature Communications have put together an Editors’ Highlights webpage of recent research on Therapeutics and have chosen to feature Dr. Porteus' article, entitled “Cas9-AAV6-Engineered Human Mesenchymal Stromal Cells Improved Cutaneous Wound Healing in Diabetic Mice”. Congratulations to Dr. Porteus and team!
Read feature and full article here >>>
Kriya Therapeutics announces $80 million in funding to advance gene therapies and Fraser Wright, CDCM's Dir. of Technology Innovation, will be an advisor
May 12, 2020
John Fraser Wright, Ph.D. is the Scientific Co-Founder and Chief Scientific Advisor for Kriya Therapeutics. Kriya Therapeutics announced $80 million in funding to advance gene therapies for highly prevalent serious diseases.
Fraser Wright, currently serves as CDCM's Director of Technology Innovation & Professor of Pediatrics at Stanford University.
Please read Kriya Therapeutics press release here>>>.
Find out more about Fraser Wright here>>> .
Atara Biotherapeutics Appoints Maria Grazia Roncarolo, Director of the CDCM, to the Board of Directors
May 11, 2020
Atara Biotherapeutics announced the appointment of cell & gene therapy expert Maria Grazia Roncarolo, MD to the Board of Directors.
"I am delighted and incredibly proud to join the fantastic team at Atara Biotherapeutics as a member of their Board. It's an exciting time in the field of cell & gene therapy and Atara is at the forefront with its novel allogeneic EBV T-cell platform. I look forward to helping bring transformative cell therapies to patients with serious diseases."--Dr. Roncarolo.
Please read full press release here>>>.
Businesswire.com also wrote a piece on thier website about this great news! Read about here >>>.
Congratulations to Marianne Goodwin for her first author publication!
May 7, 2020
Marianne Goodwin, first author, was a postdoc in the Bacchetta and Roncarolo labs (she is now at AveXis).
The publication titled "CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells" can be read here >>>
New Publication by Dr. Sebastiano
March 24, 2020
CDCM collaborator Vittorio Sebastiano is getting recognized for his work in the rejuvenation of cells, making them more youthful. The research is being published in Nature Communications.
CDCM's Co-Director, Matthew Porteus, is featured in a film: Human Nature
February 25, 2020
Dr. Porteus discusses CRISPR and Sickle Cell disease in the film "Human Nature". "Human Nature" focuses on new technologies that are changing lives.
Starting March 13, 2020 this CRISPR documentary will be playing in movie theaters!
Watch the trailer here.
For tickets to watch the film in the Bay Area visit:
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).