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Prof. Agnieszka Czechowicz is a physician-scientist and biotech entrepreneur with 20 years of experience in stem cell biology and translational research. Dr. Czechowicz is a faculty member in Stanford University’s Department of Pediatrics, Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine Division and is also a member of Stanford's Institute for Stem Cell Biology and Regenerative Medicine.Dr. Czechowicz’s primary clinical interests are in bone marrow failure (including Fanconi anemia and aplastic anemia) and other diverse diseases that necessitate or could benefit from stem cell transplantation. Her primary research interests are in hematopoietic stem cell biology, transplantation, gene-therapy/gene-editing and regenerative medicine. Multiple pre-clinical and clinical therapies are in development based upon her studies.Dr. Czechowicz’s pioneering work has led to critical developments within the life sciences ecosystem. She was the scientific co-founder of Magenta Therapeutics which amongst other things was developing antibody-drug-conjugates for conditioning for hematopoietic stem cell transplantation, and parallel antibody-based conditioning also based upon her work are additionally being pursued by Jasper Therapeutics and Forty Seven Inc (acquired by Gilead in 2020). Her efforts have also led to important patents that are assigned to Editas Medicine and Decibel Therapeutics. Previously, Dr. Czechowicz spent nearly a decade as a consultant at Third Rock Ventures, where she assisted and started companies including Global Blood Therapeutics, Bluebird Bio, Editas Medicine, Decibel Therapeutics and Magenta Therapeutics. She has also advised Prime Medicines, Beam Therapeutics, and Spotlight Therapeutics.Dr. Czechowicz completed her Ph.D. work in Developmental Biology at Stanford University with Prof. Irv Weissman. She then completed her residency in Pediatrics at the Boston Children’s Hospital, and subsequently pursued subspecialty training in Pediatric Hematology and Oncology at the Dana Farber Cancer Institute while simultaneously conducting postdoctoral research with Prof. Derrick Rossi in collaboration with Prof. David Scadden. She received her M.D., Ph.D., and Bachelor’s Degree from Stanford University.Dr. Czechowicz is a strong physician-scientist and advocate of translational research. She has done pioneering work showing that hematopoietic stem cell depletion is a critical component to donor hematopoietic stem cell engraftment. She has also led several clinical trials exploring new cell and gene therapy treatments for Fanconi Anemia. She is passionate about mentoring and training future generations of physicians and scientists, and is very supportive of helping diverse trainees on various traditional and non-traditional career paths. Dr. Czechowicz can best be reached through her administrative assistant Paula Vasquez (firstname.lastname@example.org).Recent press releases on her efforts can be found here:https://med.stanford.edu/news/all-news/2019/02/antibody-drug-combo-may-obviate-need-for-tissue-matching.htmlhttp://med.stanford.edu/news/all-news/2019/02/antibody-could-increase-cure-rate-for-blood-immune-disorders.html
Dr. Czechowicz’s research is aimed primarily at understanding how hematopoietic stem cells interact with their microenvironment in order to subsequently modulate these interactions to ultimately improve bone marrow transplantation and unlock biological secrets that further enable regenerative medicine broadly. She is interested in increasing our basic science understanding of these interactions and also developing new novel therapies that stem from this work to expand treatment options for a wide variety of pediatric and adult diseases. Her group is primarily focused on studying the cell surface receptors on hematopoietic stem/progenitor cells and bone marrow stromal cells, and is actively learning how manipulating these can alter cell state and cell fate. Her group is using cells and serum from both mice and primary specimens from healthy and diseased patients for these studies and using a variety of exciting new tools and methods to unlock future discoveries. There are many exciting opportunities that stem from her work across a variety of disease states ranging from rare genetic diseases, autoimmune diseases, solid organ transplantation, microbiome and cancer. While her group is primarily focused on blood and immune diseases, the expanded potential of this work is much broader and can be applied to other organ systems as well and she is very eager to develop collaborations across disease areas.Dr. Czechowicz has also been part of the initial founding team of several companies including Global Blood Therapeutics, Editas Medicine, Decibel Therapeutics and Magenta Therapeutics and advises multiple other transformative companies. As a true physician scientist, she has done pioneering work showing that hematopoietic stem cell depletion is a critical component to donor hematopoietic stem cell engraftment, and multiple pre-clinical and clinical therapies are in development based upon her studies. Stanford has had several clinical trials derived from Dr. Czechowicz’s research.Research Interests: Hematopoietic Stem Cells (HSC), Hematopoietic Stem Cell Transplantation (HSCT), Monoclonal Antibodies, Immunotoxins, Cell Cycle, Cell Fate, Cell Membrane, Cell Surface Antigens, Microenvironment, Stem Cell Niche, Cell Proliferation, Stem Cell Quiescence, DNA Damage, DNA Repair, Rare Genetic Diseases, Bone Marrow Failure, Aplastic Anemia, Genomics, Fanconi Anemia (FA), Immunodeficiency (SCID), Gastrointestinal Stromal Tumors (GIST), Rhabdomyosarcoma, Neuroblastoma, Myelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML), Graft vs Host Disease (GVHD), Immune Tolerance, Histocompatibility Testing, Immunologic Deficiency Syndromes, Hemoglobinopathies, Transplantation Conditioning, Immune Tolerance, Gene Therapy, Gene-Editing, Base-Editing, Cytokines, Cytokine Receptors, Serum, Clinical Trials, Autoimmune diseases, Multiple Sclerosis, Microbiome, Cancer, Cell Therapy, Allogenic Bone Marrow Transplantation (BMT), Metabolic Diseases, Hurler SyndromeFor more information, please visit our lab webpage: http://med.stanford.edu/czechowiczlab.html
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which
enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by
transplanting depleted stem cells from a donor after using an experimental antibody treatment
called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause
fewer side effects than chemotherapy (the current standard of care method).
Participants will be administered the conditioning regimen, are assessed until they receive
the depleted stem cell infusion, and will be followed for up to 2 years after the cell
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Gene Therapy for Fanconi Anemia, Complementation Group A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic
cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A).
Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be
transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After
transduction, the corrected stem cells will be infused intravenously back to the patient with
the goal of preventing bone marrow failure.
A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
The objective of this study is to assess the therapeutic safety and preliminary efficacy of a
hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells
transduced with a lentiviral vector carrying the FANCA gene in subjects with Fanconi anemia
subtype A (FA-A).