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Registration for the CDCM 9th Annual Symposium is Now Open!

October 22, 2024

The Center for Definitive and Curative Medicine (CDCM) hosts an annual symposium revolving around the cell and gene therapy field. We invite speakers from academia, not-for-profits, the federal government, and industry to present bench-to-bedside research discoveries and innovations. Our 2025 symposium will be held on February 26, at Berg Hall (LKSC) on Stanford campus. You can register for the symposium here

 

This year's host of renowned speakers (so far) include: 

  1. George Church, PhD, Professor of Genetics, Harvard Medical School (virtual)
  2. Everett Meyer, MD, Associate Professor of Medicine, Pediatrics – Stem Cell Transplantation, Stanford University
  3. Kapil Bharti, PhD, NEI Scientific Director, Ocular and Stem Cell Translational Research Section, National Institutes of Health
  4. Peter Marks, MD, PhD, Director, The Center for Biologics Evaluation and Research, Food and Drug Administration
  5. Siddhartha Jaiswal, MD, PhD, Assistant Professor of Pathology, Stanford University
  6. David Miklos, MD, PhD, Chief of BMT and Cell Therapy Program, Stanford University
  7. Joseph C. Wu, MD, PhD, Director, Stanford Cardiovascular Institute, Stanford University
  8. Mark A. Skylar-Scott, PhD, Assistant Professor of Bioengineering, Stanford University
  9. Anthony Atala, MD, W. Boyce Professor and Chair of Urology, Wake Forest University
  10. Birgitt Schuele, MD, Associate Professor (Research) of Pathology, Stanford University

Cystic Fibrosis Team Wins Top Award at American Rhinologic Society 70th Annual Meeting

October 10, 2024

At the ARS 70th Annual Meeting, the Cystic Fibrosis (CF) team won the prize for "Top Basic Science Abstract and Paper". Huge Congratulations to Dr. Dawn Bravo and the Nayak Lab for their achievement! Currently, the team is also working on a CF therapy with recent funding from CIRM.


Join Us for a Breath of Fresh Air Gala With Dr. Matt Porteus

September 20, 2024

On October 6th, the Cystic Fibrosis Research Institute will be honoring Dr. Matt Porteus for his work in the field of genome editing. His research on developing genome editing by homologous recombination has helped many children with genetic diseases, including those with cystic fibrosis. From 2PM - 7PM, please join us in sipping local wines, enjoying food, and exploring the estate of Nestldown. Other exciting activities include bidding on luxurious items, enjoying live music, and connecting with Dr. Matt Porteus. We hope to see you there! 


Stanford Labs and Researchers Among Contributors to Article Publication on New In Vitro Platform to Model Formation of Human Pharyngeal Endoderm

August 8, 2024

A collaborative effort between multple universities involving Stanford researchers and labs have led to a new in vitro platform in which the formation of human pharyngeal endocerm (PE cells) can be modeled. The PE, which is a vital developmental tissue, serves as the precursor to the esophagus, parathyroids, thyroids, lungs, and thymus. Despite its importance, an in-depth characterization of PE cells was not available to us yet, due to technical and ethical limitations surrounding their early formation.

Members of the Sebastiano and Tartaglia Labs were credited with contributions from their scientific discussion. Additionally, this work is part of the 22q11 Deletion Syndrome Consortium of the CDCM, which is a donor-funded program studying the the biology and immunology of the disease. 

The full article can be accessed here

 


Editorial: Cell and Gene Therapy Accessibility

August 2, 2024

Rayne H. Rouce* and Matthew H. Porteus

 

Patients with devastating illnesses demonstrate incredible courage in battling their disease. Innovative cell and gene therapies (CGTs), built on decades of research, are changing the lives of those who suffer from conditions ranging from cancer to sickle cell disease to neurologic diseases. Although hailed for their promise and recognized for benefits that will exceed the costs, the high prices of CGTs ($300 thousand to $4 million per dose) leave these therapies out of reach for many. This accessibility problem will only be solved if academia, industry, investors, funders, regulators, and advocacy groups work together to put CGT breakthroughs in the hands of all who stand to benefit.

In CGT research and development, newer, more complex techniques are more likely to be funded, published, and confer prestige than discoveries that simplify older methods. Accessibility is hardly considered in such “discovery” science. Process engineering, which aims to improve productivity in biopharmaceutical manufacturing, is ripe for rethinking and could be creatively focused on lowering patient cost. As well, clinical care innovation can be a game changer, particularly in low- and middle-income countries. For example, India has decreased the cost of hematopoietic stem cell transplantation by 30- to 50-fold compared to the US while maintaining excellent clinical results.

CGTs are fundamentally custom-made therapies, with small batches or even single doses manufactured, which is why they are inherently expensive. Further, supply chain costs can account for up to 50% of total manufacturing costs. The supply chain and manufacturing for CGTs have not had their “assembly line” moment and thus lack the “economies of scale” required to make goods available to global populations. Automated manufacturing is essential to reduce costs while maintaining quality and reproducibility. Whether automation will be applied through point of care and/ or through large, centralized factory manufacture to increase accessibility is not yet clear. Similarly, successful leveraging of logistics infrastructure to create reliable accessibility remains to be determined. Nevertheless, these are issues that require ongoing exploration in a quest to enhance accessibility to CGTs.

Although regulatory agencies recognize the promise and importance of CGTs, requirements throughout the process of bench-to-bedside discovery add substantialcosts. Analytically identical reagents, for example, can cost 10 to 100 times more to meet the specifications required for use in humans, while performing no better than those agents with a “research use only” label. These costs contribute to the high list prices of approved CGTs and consequently create a barrier to development. Regulatory bodies could rethink this approach and answer affirmatively to sponsor requests for use of identical reagents that include scientific justification of equivalent performance, thus lowering costs without compromising safety.

Companies prioritizing shareholder value have been successful at developing safe and effective drugs, but complementary approaches are needed. Many CGTs that will affect a small number of patients are halted in their clinical development or even removed from market owing to limited commercial viability. Complementary approaches in which various institutions—not-for-profit foundations, privately held corporations, governmentsupported entities, or benefit corporations—take on clinical development and commercial distribution for CGTs with low revenue streams are being considered. The next step is to develop and test these other business models.

Only a subset of patients who could benefit from CGTs receive them because their complexity limits the number of institutions that can safely provide them, thus creating geographical barriers to access. Also, high prices place the patient and treating institution at financial risk, which means that in most cases, treatment does not proceed without prior payor authorization. Unfortunately, delayed or even denied authorization can make timely treatment a challenge in many countries. In the United States, it makes the treatment of uninsured patients nearly impossible. These financial and logistical factors closely mirror the well-studied social determinants of health (such as income and education) that lead to poor outcomes and disproportionately affect the most vulnerable.

Despite these issues, there is hope that CGTs will become globally accessible because potential solutions can be envisioned and because stakeholders have the creativity to make it happen. This future will not occur unless innovation and courage can disrupt the traditional CGT ecosystem. After all, the patients who show tremendous courage and resilience deserve nothing less.

 

*R.H.R. is a consultant for Pfizer and has received honoraria from Novartis Pharmaceuticals. M.H.P. holds equity in CRISPR Therapeutics, Kamau Therapeutics, Allogene Therapeutics, and Arbor Biotechnologies and is on the scientific advisory board of Allogene Therapeutics and Orthobio.


Stanford's Matthew Porteus, MD, PhD Delivers Keynote at the 11th International Symposium of Cell and Gene Therapy Institute 

June 28, 2024

Every year, the Samsung Medical Center (SMC) in Korea hosts a symposium with discussion in fields such as gene therapy, genetic modification, immuno-cancer cell therapy, stem cell exosome treatment, 3D bioprinting, and organoids. This year's theme placed a focus on cell and gene therapy with high clinical unmet needs as well as an efficient transfer of laboratory studies to the clinic.

Dr. Matthew Porteus was invited to the symposium as a speaker and delivered the keynote, presenting his work and research within the cell and gene therapy field. The full program for the symposium can be viewed here


Stanford's Matthew Porteus, MD, PhD Helps Develop Technique to Genetically Modify HSPCs

June 20, 2024

Matthew Porteus MD, PhD, along with UCSF researcher M. Kyle Cromer, PhD, have utilized CRSIPR-Cas9 to modify HSPCs genetically, enabling them to increase red blood cell production without losing HSPC functionality. 

HSPCs (hematopoietic stem and progenitor cells) play a critical role in treating blood disorders such as sickle cell disease. This new technique could help in eliminating the toxic conditioning process currently required for HSPC transplant. Dr. Porteus's lab work suggests that combining precise genome editing with human genetics can result in safer/more effective genome-editing therapies. 

You can read the full article here


Stanford's Dr. Maria Grazia Roncarolo, MD Bestowed the Assobiotec Award at 2024 Assembly of Federchimica Assobiotec

June 25, 2024

The 2024 Assembly of Federchimica Assobiotec was recently held on June 25. The  conference discussed the role of the private sector in supporting research progress and the challenges of modern society with scientific evolution. The Conference also was the setting for the Assobiotec Award, an achievement given to individuals/organizations that have distinguished themselves in the "promotion of innovation, scientific research, and technological transfer". 

This year, Dr. Maria Grazia Roncaralo, MD was the recipient of the award. To read more about this event, please click here


Stanford's Matt Porteus, PhD and Natalia Gomez-Ospina, MD, PhD awarded $6 Million and $5 Million Respectively for Their Programs

May 30, 2024

This month, $6 million was awarded to Dr. Matt Porteus and his team to develop a stem cell-based gene correction therapy for Cystic Fibrosis (chronic sinusitis). Cystic Fibrosis currently has no cure and can only be treated with symptom management. The proposed new treatment hopes to serve as an alternative for patients who have mutations or cannot tolerate existing therapies. While not directly tied with mortality, sinus disease in cystic fibrosis significantly impacts the quality of life and may also impact lower respiratory tract disease.

Additionally, Dr. Natalia Gomez-Ospina and her team were awarded $5 million as part of CIRM's Translational program. This award serves to support stem cell-based or gene projects to advance clinical study or broader uses. This grant was awarded for her Gaucher Disease Program. 

To read more about both awards, please visit the CIRM press release here.


CDCM's Matt Porteus, PhD Appointed Vice President of the American Society of Gene + Cell Therapy 

May 2, 2024

Starting June 1, Matt Porteus, PhD will assume the role of vice president for the American Society of Gene + Cell Therapy. He will be ascending through the presidential succession before service as the president in the 2016-2027 year. For more information, please visit the ASGCT site here.  


CDCM 7 Year Recap

May 7, 2024

Seven years after its establishment in 2017, the Center for Definitive and Curative Medicine (CDCM) has proven to be successful in translating bench-to-bedside therapies. Some such examples include Stanford led programs such as the iPS-derived skin grafts for Epidermolysis Bullosa, Sickle Cell Disease Gene Correction, IPEX Syndrome Gene Correction, and the Fanconi Anemia Treatments. These programs were featured and extensively discussed in Stanford’s 7th and 8th Annual Symposium. Additionally, Stanford CDCM’s Ami Shah and her team recently had two Vertex trials published in the New England Journal of Medicine, both of which represent the strength of CDCM-industry partnerships with Stanford as a primary clinical trial site.

In December 2022, Dr. Matthew Porteus took over the role of Director, previously held by Dr. Maria-Grazia Roncarolo, who founded the CDCM. Since then, the CDCM has continued to follow its original vision of bridging the divide between laboratory discovery and clinical translation to patients. The CDCM’s new video highlights the foundation of the organization and offers different perspectives on how several faculty members strive to embody the vision in their research and daily work.


Natalia Gomez-Ospina, MD, PhD Receives 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award

March 13, 2024

Stanford University's Natalia Gomez-Ospina was presented the "Watson Award" by the ACMG Foundation for Genetic and Genomic Medicine earlier last Wednesday.

The foundation, a nonprofit organization that focuses on medical genetics and genomics in healthcare, recognized Dr. Gomez-Ospina for her work and dedication to advancing genetic and genomic medicine. 

Nancy J. Mednelsohn, MD, FACMG, president of the ACMG Foundation, stated, "Dr. Gomez-Espina is a promising physician-scientist whose work is befitting the Dr. Michael Watson Genetic and Genomic Medicine Innovation Award, an award that honors an individual whose work has had a significant impact on genetic and genomic medicine. Dr Gomez-Espina's work is innovative, collaborative, and novel, recognizing several newfound genetic conditions. Her research focuses on advancing cell-based therapies for genetic disorders, investigating genome editing to engineer hematopoietic stem cells for the treatment of lysosomal storage diseases."

Read the full story here


The Rhythm of Life

January 29, 2024

Quenton Rashawn Bubb is a sixth-year MD-PhD student in Stanford with a focus on stem cell biology and regenerative medicine. Inspired and motivated by his mother, who was a nurse, he developed a passion for the field of medicine.

Bubb’s work in Dr. Agnieszka Czechowicz’s lab is focused on the development oftherapies that would eliminate the need for chemotherapy and lead to a cure for childhood acute myeloid leukemia.

You can read more about his story here.


Dr. Rajni Agarwal-Hashmi Awarded the Fanconi Anemia Research Fund (FARF)'s Lifetime Achievement Award

October 1, 2023

Over the weekend of September 28 – October 1, Stanford’s Dr. Rajni Agarwal-Hashmi was awarded the Fanconi Anemia Research Fund (FARF) Lifetime Achievement Award at their 2023 Scientific Symposium. For more than 35 years of her career, she has been dedicated to the improvement of the care of patients with Fanconi Anemia (FA), including spearheading impactful and innovative clinical treatment strategies that have led to meaningful advances in the quality of life for FA patients and their families.

Dr. Agarwal-Hashmi’s work and diligence toward the care of FA is exemplified by the 70+ patients, that have come from all over the nation, treated by her at Stanford Children’s. Recognizing the extreme sensitivity that FA patients deal with regarding conventional pre-transplant conditioning strategies, she has pioneered efforts to advance non-toxic antibody-based conditioning which challenges the status quo. Specifically, her leadership in a first-in-human trial for utilizing non-toxic-antibody-based conditioning over genotoxic irradiation and busulfan is field-defining. This is the first-time this antibody is being combined with immunosuppression and alpha/beta T-cell depleted haploidentical grafts, the later providing greater access to donors as well as decreasing non-transplant related morbidities

Dr. Agarwal-Hashmi’s impact on the FA field is unequivocally vital toward the advancement and improvement of treatments and patient care. Her leadership and innovation in the field are reflected in her reception of this prestigious FARF honor.


South Korean Minister of Health Visits CDCM to Learn More About Stanford's Cell and Gene Therapies

July 7, 2023

Following a visit by the Samsung Medical Center’s Cell and Gene Therapy Institute (CGTI) in April, the CDCM hosted the South Korean Minister of Health and Welfare, Kyoo Hong Cho, on July 7, 2023. The purpose of the visit was to learn more about Stanford’s cell and gene therapy activities, including research, clinical care, and training.

During Minister Cho's visit, he met with Lloyd Minor, MD, Dean of the School of Medicine; David Entwistle, President and CEO of Stanford Health Care; Paul King, President and CEO of Stanford Medicine Children's Health; and Matthew Porteus, MD, PhD, Director of the CDCM. They discussed Stanford Medicine's advanced practices in various practices such as precision medicine through cell and gene therapy, the Medical Scientist Training Program (MSTP), and the Clinical and Translation Science Awards (CTSA). Opportunities for future collaborations between Stanford Medicine and research-focused hospitals in Korea were also explored. 

In total, 17 people from the Korean Embassy, the Korea Health Industry Development Institute (KHIDI), and the CEOs of the Korea Drug Development Fund (KDDF) and Korea Medical Device Development Fund (KMDF) etc. came to the visit.


CIRM Awards Stanford University $2,000,ooo in their First Phase of Building California Cell & Gene Therapy Manufacturing Network

May 31, 2023

The California Institute for Regenerative Medicine (CIRM) awarded Stanford University $2,000,000 as part of their $10,000,000 Phase One Initiative to build a California Cell and Gene Therapy Manufacturing Network. Steven Feldman, PhD, was the recipient of this award with his work in Stanford's LCGM and his INFR5-14663 application. The full program title is the "Laboratory for Cell and Gene Medicine: A Partner in the California Cell and Gene Therapy Manufacturing Network". 

You can read more here.


Stanford Medicine Children's Health Receives "Rare Disease Centers of Excellence" Designation

May 11, 2023

The National Organization for Rare Disease (NORD) has named Stanford Medicine Children's Health as one of its "Rare Disease Centers of Excellence", which are dedicated to diagnosing, treating, and researching all rare diseases. 40 hospitals nationwide make up this healthcare network that is at the forefront of cutting edge scientifc/medical breakthroughs and cares for thousands of rare disease patients.

Natalia Gomez-Ospina, MD, PhD will serve as the director for Stanford Children's NORD Center of Excellence for Rare Disorders. You can read more here.


CIRM Awards Stanford University $11,813,964 for their CLIN2-14315 Program headed by Matthew Porteus, MD, PhD

April 27, 2023

Principal investigator Matthew Porteus, MD, PhD, along with fellow investigators Rajni Agarwal, MD and Agnieszka Czechowicz, MD, PhD were awarded $11,813,964 by The California Institute for Regenerative Medicine (CIRM) for their CLIN2-14315 program. The program's full title is "Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients". You can read more about the awards here.

 

Other active CIRM grants:

  • Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy - $6,056,713 (TRAN1-14609)
  • Genome Editing of Autologous Hematopoietic Stem Cells to Treat Severe Mucopolysaccharidosis type 1 (Hurler Syndrome) - $5,444,353 (CLIN1-13988)
  • Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant - $11,998,188 (CLIN2-14024)
  • Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome - $11,999,179 (CLIN2-13259)
  • Phase 1/1b study of  T-allo10 infusion after HLA-partially matched ab depleted-HSCT in children and young adults with hematologic malignancies - $10,563,822 (CLIN2-12563)

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 (abnathan@stanford.edu). 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 (abnathan@stanford.edu).

 


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.”

Read full press release here>>>


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 (angelic5@stanford.edu). 

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!

Read full press release here >>>


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.

Read here for more information. 


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:

https://www.humannaturefilm.com/tickets/

 


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.

Read here for more information.


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.

Read here for more information. 


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". 

Read the entire paper here>>


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. 

Read more about this exciting news here>>


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. 

Click here to see the entire interview>>


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.

Additional information can be found here >>


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.  

 

Learn More>>


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.

To listen to the full story click here>>


"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 - 

                                                Learn More>>


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.

Learn More About This Appointment >>


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).

Learn More About This Collaboration >>