Director's Letter
In 2025, the UCSF Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research (Broad Center) drove major advances in our strategic vision — launching bold new genome-editing initiatives, strengthening industry partnerships, expanding community outreach, and accelerating therapies to help patients. These milestones represent our mission for stem cell biology and regenerative medicine, setting the stage for a campaign to fuel the future. Guiding our work is the vision to: Discover – Engineer – Heal. This framework shapes everything we do:
- Discover: Science explaining how cells and tissues develop, age, and repair.
- Engineer: Cutting-edge tools that harness biology to solve complex problems.
- Heal: Translating discoveries into therapies that change lives.
I am proud of all we accomplished this year and excited to see how we will continue to grow in the year to come.
Tippi C. MacKenzie, MD
Professor of Surgery
Benioff UCSF Professor of Children's Health
Director, The Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research
Co-Director, Center for Maternal-Fetal Precision Medicine
John G. Bowes Distinguished Professor of Stem Cell and Tissue Biology
Director's Letter * Strategic Planning * Research Highlights * Our Accomplishments * People * Growing the Vision
Strategic Planning: Building Toward the Future
Starting in late 2023 and continuing today, the Broad Center has been focused on a strategic planning initiative to strengthen research, education, and innovation in stem cell and regenerative medicine. Building on our strong foundation — including the state-of-the-art Dolby Regeneration Medicine Building and our multidisciplinary community — we are working on four key growth areas: People, Research, Education, and Development.
The vision emerging from this process, “Discover – Engineer – Heal,” highlights our commitment to advancing discovery science, integrating genome editing and cell engineering, and translating these efforts into life-saving therapies. Key actions we’re taking:
Advance Genome Editing in Stem Cells
- Recruit Expertise: Hire two faculty members specializing in hematopoietic stem cell biology and genome editing. Recruitment is ongoing.
- Build Research Infrastructure: Retrofit and transform lab spaces previously used for older research technologies in the Dolby Building to enable new work in high-throughput experiments in stem cell culture and genome editing.
- Support Collaborative Projects: Provide small seed funding mechanisms to aid towards fostering collaborations among investigators.
Strengthen Industry Partnerships
- Identify and Grow Connections: Create an Industry Advisory Board to guide partnerships for our faculty with pharmaceutical and biotech companies in the Bay Area.
- Host Innovation Forums: Facilitate opportunities for faculty and industry leaders to network and collaborate on advancing stem cell research.
- Launch Collaborative Projects: Develop initiatives like organoid-based drug screening and imaging platforms to align academic research with industry needs.
Expand Education and Mentorship
- Increase visibility for trainees’ research activities, enhance mentorship programs, and engage the public with the work ongoing at UCSF to highlight the impact of stem cell research.
Assess and Optimize Infrastructure
- Repurpose and transform space for genome editing and stem cell research.
- Survey equipment needs to ensure researchers have access to cutting-edge technology.
Some initiatives are already underway, including a partnership with the Innovative Genomics Institute in Berkeley to develop a Center for Genome Surgery focused on advancing stem cell engineering and therapies. This Center will support additional efforts in stem cell genome editing and foster industry partnerships, as well as create a referral service to discuss potential genomic therapies for patients with defined single-gene conditions. Other long-term goals, such as faculty recruitment and facility upgrades, require additional funding and careful financial planning, but remain important goals in the immediate future.
Priorities to financially support our strategic efforts include:
- Faculty recruitment in hematopoietic stem cell biology and genome editing.
- Retrofitting lab spaces for high-throughput experiments.
- Pilot grants to support new research teams.
- Building regulatory and clinical infrastructure for tissue biobanking.
The strategic planning implementation involves extensive community engagement and is already driving sustained growth, innovation, and impactful collaborations through 2026 and beyond.
Director's Letter * Strategic Planning * Research Highlights * Our Accomplishments * People * Growing the Vision
Research Highlights: Discover – Engineer – Heal
Guided by a core vision — Discover – Engineer – Heal — the Broad Center leads pioneering research that bridges fundamental discovery with real-world impact. From uncovering the essential mechanisms of biology to engineering transformative tools and translating these breakthroughs into life-changing therapies, we are shaping the future of regenerative medicine and stem cell science.
Discover
- Mapping brain development to reveal potential genetic origins of autism and cancers.
- Using advanced models (“assembloids” and “organoids”) to explore traumatic brain injury and psychiatric disorders.
- Advancing immune cell therapies that activate the body’s own defenses against tumors.
- Discovering fat–joint communication that could lead to new treatments for arthritis.
Engineer
- Optimizing gene delivery tools for greater precision.
- Demonstrating the promise of in-utero therapy for spinal muscular atrophy.
- Designing synthetic receptors to reduce the cost of cell-based therapies.
Heal
- Pioneering genome editing for blood disorders like α-thalassemia.
- Developing fetal interventions that could prevent disease before birth.
- Creating new cell therapy platforms that overcome the complexity of cancer
Publication Highlights
This year, Broad Center researchers published nearly 50 studies in leading scientific journals, driving major advances in brain development, Alzheimer’s disease, gene editing, and regenerative medicine. Below are selected highlights from this body of work.
Per-Ola Carlsson et al., including Broad member Tobias Deuse, published “Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression” in the New England Journal of Medicine. This breakthrough shows that insulin-producing cells can be transplanted without lifelong immune-suppressing drugs, making a curative cell therapy for diabetes much closer to reality.
Jiapei Chen et al., including Broad members Elizabeth Crouch, Edward Valenzuela, Janeth Ochoa Birrueta, Loukas Diafos, Kaylee Wedderburn-Pugh, Arnold Kriegstein, Michael Oldham, and Eric Huang, published “Proinflammatory immune cells disrupt angiogenesis and promote germinal matrix hemorrhage in prenatal human brain” in Nature Neuroscience. This study shows how harmful immune activity can damage fragile blood vessels in the developing brain, helping to explain a major cause of life-threatening brain bleeding in premature infants.
Karissa Hansen et al., including Broad members Ryan Boileau and Robert Blelloch, published “Synergy between regulatory elements can render cohesin dispensable for distal enhancer function” in Science. This landmark mechanistic genomics paper changes how scientists understand long-distance gene control.
Jae-Jun Kim et al., including Broad members Simone Kurial, Pervinder Choksi, Jan Bartel, Julia Driscoll, Aras Mattis, and Holger Willenbring, published “AAV capsid prioritization in normal and steatotic human livers maintained by machine perfusion” in Nature Biotechnology. This study helps identify the safest and most effective gene-therapy delivery vehicles for healthy and fatty human livers, a crucial step toward improving treatments for liver disease.
Homa Majd et al., including Broad members Ryan Samuel, Andrius Cesiulis, Jonathan Ramirez, Ali Kalantari, Kevin Barber, Sina Farahvashi, Alireza Majd, Angeline Chemel, Mikayla Richter, Samyukta Bhat, Tomasz Nowakowski, and Faranak Fattahi, published “Engrafted nitrergic neurons derived from hPSCs improve gut dysmotility in mice” in Nature. This work shows that lab-grown nerve cells can restore normal gut movement in mice, pointing toward a potential new treatment for serious digestive disorders caused by nerve damage.
Sachin Sharma et al., including Broad members Holger Willenbring, Aras Mattis, and Valerie Weaver, published “Resolving fibrosis by stimulating HSC-dependent extracellular matrix degradation” in Science Translational Medicine. This study shows that stem cells can actively break down scar tissue rather than just slow its formation, opening the door to new treatments for chronic organ scarring.
Li Wang et al., including Broad members Cheng Wang, Juan Moriano, Songcang Chen, Guolong Zuo, Arantxa Cebrián-Silla, Tanzila Mukhtar, Shaohui Wang, Mengyi Song, Lilian Gomes de Oliveira, Qiuli Bi, Jonathan Augustin, Mercedes Paredes, Eric Huang, Arturo Alvarez-Buylla, Jingjing Li, and Arnold Kriegstein, published “Molecular and cellular dynamics of the developing human neocortex” in Nature. This work creates one of the most detailed maps ever of how the human brain forms before birth, providing a critical foundation for understanding brain disorders and normal brain development.
Irene Munoz-Blat et al., including Broad member Susan Fisher, published “Multi-omics-based mapping of decidualization resistance in patients with a history of severe preeclampsia” in Nature Medicine. This study reveals why the uterus fails to properly support pregnancy in women who develop severe preeclampsia, and points toward better ways to predict and prevent dangerous pregnancy complications.
Tomasz Nowakowski et al. published a package of developing brain atlases (multiple Nature papers) in Nature. This international effort builds a set of detailed brain development maps across species and technologies, setting a new global standard for how the developing brain is studied.
David Shin et al., including Broad members Bryan Pavlovic, Alex Pollen, and Tomasz Nowakowski, published “Thalamocortical organoids enable in vitro modeling of 22q11.2 microdeletion” in Cell Stem Cell. This work builds lab-grown brain tissue that mimics key brain circuits affected in autism and schizophrenia, allowing researchers to study how this genetic disorder disrupts brain wiring directly.
Beika Zhu et al., including Broad members Andi Wangzhou, Yonatan Perez, Arnold Kriegstein, Tom Nowakowski, and Xianhua Piao, published “ADGRG1 drives a protective microglial state in Alzheimer’s disease” in Neuron. This research identifies a switch that helps brain immune cells protect neurons in Alzheimer’s disease, suggesting a new way to slow disease progression, not just treat symptoms.
Director's Letter * Strategic Planning * Research Highlights * Our Accomplishments * People * Growing the Vision
Our Accomplishments
Strengthening Scientific Community
The Broad Center faculty and trainees span the breadth of regenerative medicine — from developmental biology and aging to clinical translation. A collaborative culture, combined with UCSF’s unique position in the Bay Area, supports bold innovation and impact.
Teamwork Unlocks New Insights into Female Fertility and Ovarian Aging
A unique collaboration between reproductive and neuroscientists at the Broad Center led to groundbreaking insights into female fertility, revealing the hidden role of peripheral nerves in the growth of eggs and changes during aging. Sparked by the complementary interests and skills of trainees — Eliza Gaylord and Mariko Foecke from Diana Laird’s lab and Ryan Samuel from Faranak Fattahi’s lab — this collaboration evolved organically during their time in the Ray and Dagmar Dolby Regeneration Medicine Building.
Their findings, published in Science, reveal that fertility decline isn’t just about egg quality. Eggs cluster in “pockets” throughout the ovary, surrounded by nerves, blood vessels and supportive tissues that age alongside them, creating a deteriorating ecosystem. This discovery sheds light on the broader impacts of ovarian aging and offers hope for extending both fertility and health span. The partnership highlights the power of shared curiosity and teamwork in driving scientific breakthroughs.
Securing Research Support
Despite the challenges posed by the rapid changes and delays in federal funding, the Broad Center continues to adapt and deliver outstanding progress, fueled by the dedication of the faculty and success in securing alternative funding sources. Broad Center faculty demonstrated extraordinary success in 2025 in California Institute for Regenerative Medicine (CIRM) grants across discovery, translational, and clinical programs — fueling research even as federal funding remains uncertain. In early 2025, Mercedes Parades, Tomasz Nowakowski, and Alex Pollen were all recipients of ReMIND (DISC4) funding totaling over $32M in funding to their research programs. In September 2025, Holger Willenbring, Jingjing Li, Bruce Conklin, Benoit Bruneau, and Tippi MacKenzie were recipients of the next round of Discovery (DISC0) funding, with $11.4 million in support for their research. These grants allow Broad Center faculty to continue to support novel research in rare diseases and therapeutics.
Refining Focus Leads to Breakthrough Support for Rare Disease Research
Neuroscientist Mercedes Paredes and her team began their CIRM Discovery Stage (DISC4) proposal with an ambitious plan to study multiple rare genetic brain disorders. Reviewer feedback urged them to narrow their scope, prompting the team to focus on 22q microdeletion syndrome, a rare condition linked to developmental delays. This sharper focus allowed them to connect genes to brain circuitry and unite experts in genetics, neurology, and CRISPR editing. The result: a funded proposal and a powerful example of how collaboration and focus fuel scientific progress.
Excellence Across the Pipeline
From breakthrough gene-editing strategies to global leadership in stem cell science, Broad Center faculty continue to shape the future of regenerative medicine at every career stage. This year’s honors — spanning transformative early-career awards, landmark translational funding, and international recognition for public service — reflect both the depth and breadth of that impact:
Kyle Cromer received one of NIH’s most competitive early-career awards to advance transformative gene-editing approaches for treating sickle cell disease.
Mercedes Paredes received the U.S. government’s highest honor for early-career researchers in recognition of her exceptional scientific leadership and innovation.
Mort Cowan and Jennifer Puck secured a landmark $15 million CIRM award to advance a potentially curative gene therapy for infants with Artemis-deficient SCID toward standard-of-care use.
Tomasz Nowakowski received a Vilcek Prize for Creative Promise and was named a national finalist for the Blavatnik Awards, placing him among the top young scientific innovators in the United States.
Carolyn Sangokoya was selected for the inaugural ISSCR Public Service Award, recognizing extraordinary leadership in advancing stem cell science worldwide.
Empowering the Next Wave of Discovery
The Broad Center continues to build an efficient operational framework to support research and resource management, ensuring scientists can remain focused on advancing discovery, even in the face of uncertainty.
Building the Future of Breakthrough Science
Through our Strategic Planning process, we’ve identified key areas for growth, including creating a core facility for genome editing, enabling high-throughput stem cell culture, and preparing research-grade materials like lipid nanoparticles — critical tools for modern biomedical research. To bring this vision to life, Director of Research Operations Cassandra Belair has repurposed two large rooms, previously dedicated to Drosophila fly culture, into specialized tissue culture facilities. These updated spaces are now equipped to support new faculty recruitment and serve as a hub for innovative research efforts. The newly established Stem Cell Reserve further strengthens collaboration and efficiency, acting as a centralized resource hub that connects scientists across the Broad Center and UCSF to essential tools and equipment.
Our updates extend beyond the labs — our rooftop gardens have become a symbol of renewal and community, offering a space for connection and inspiration as we continue pushing the boundaries of discovery. The Dolby Building itself remains an enduring testament to innovation. Designed to rise from a steep 60-degree slope and spanning 68,926 square feet, its four split-level Pods offer sweeping views of the Pacific. Engineered with cutting-edge seismic isolation technology, the building continues to attract architects and engineers who study its pioneering design.
Engaging the Community
From scientific retreats and seminars to public school visits and lab tours, the Broad Center fostered connections between science and the community. Highlights included:
- A microscopy art contest showcasing the beauty of biology. First place went to Eliza Gaylord for her portrait of a 30-year-old human ovary, where magenta oocytes, developing follicles, and cyan-stained neurons revealed the organ’s hidden architecture. Second place to David Scheel for Ectopic Pretzel, a striking image capturing non-dividing cells in the developing gut of an embryo. The People’s Choice Awards went to Marco De Leon, whose fluorescently labeled heart highlighted the remarkable regenerative capacity of cardiac cells, and to postdoctoral scholar Yonatan Perez, whose GFP-labeled human cortical neurons image reflected efforts to better understand autism spectrum disorder through single-cell genomics. This year’s entries demonstrated once again how science and art together can illuminate worlds unseen.
- A summer internship program with California public and private schools. For the past four years, the Broad Center has partnered with the Claremont Schools to offer a dynamic summer internship program for students from public and private schools across California. A highlight of the program is the trainee-led CIRM Days celebration, designed to inspire interns to explore careers in stem cell and gene therapy research and clinical practice. During CIRM Days, trainees share their research through presentations and hands-on demonstrations. This year’s program opened with a keynote by CIRM Science Officer Uta Grieshammer, a former UCSF trainee, who offered insights into her scientific journey and the impact of regenerative medicine.
- TED-style trainee talks transformed complex research into engaging narratives about how life begins, grows, and heals. Insights into how early embryonic cells maintain genetic precision (Deniz Goekbuget), how the placenta takes root to nourish a developing baby (Matthew Gormley), and how gene-editing tools delivered before birth may correct diseases (Beltran Borges). Simone Kurial revealed the liver’s ability to regenerate missing bile ducts by converting one cell type into another. Caroline Doherty showed how surrounding cells fuel the growth of developing eggs, Tanzila Mukhtar explained why certain brain cells are vulnerable to SARS-CoV-2, and Reyna Villa revealed how diet may shape cartilage health in osteoarthritis. Christoforos Meliadis demonstrated how exercise may help preserve the delicate structures of the inner ear.
Training the Next Generation
The Broad Center is a training ground for tomorrow’s leaders. Graduate students, postdocs, and clinical fellows benefit from mentorship, summer internships, and courses in ethics. Our trainees are pushing boundaries in cancer vaccines, metabolism research, and organ models of disease. They are also active ambassadors for science—mentoring students, reading STEM stories in classrooms, and participating in the Bay Area Science Festival.
Through programs like the CIRM Trainee Network Conference and our Getting to Patients series, they engage directly with patients and clinicians, ensuring their science is both cutting-edge and compassionate. These interactions foster understanding of the lived experiences behind the diseases they study, helping bridge the gap between bench research and bedside care.
Getting to Patients | Grounding Innovation in Real-World Impact
Program Director of the UCSF Pediatric Diabetes Program Stephen Gitelman presented “Altering the Course of Type 1 Diabetes” during Getting to Patients. He shared insights alongside a patient who described the profound impact of living with diabetes and discovering her child was also developing the disease. After joining Dr. Gitelman’s clinical trial, they discussed what truly defines a “cure” — whether delaying child-onset diabetes or stopping its progression can be considered one. The conversation highlighted the challenges of managing chronic diseases, the toll of standard treatments on quality of life, and the hope offered by emerging therapies. This dialogue emphasized the importance of innovation rooted in real-world patient experiences, ensuring scientific advancements lead to meaningful improvements. These exchanges demonstrate how UCSF is not only advancing science but also listening to the voices of patients, ensuring innovations are grounded in empathy and real-world impact.
Lilian Gomes de Oliveira, PhD — Pew Latin American Fellow
Trainee Lilian Gomes de Oliveira, PhD, was named a 2025 Pew Latin American Fellow in the Biomedical Sciences, recognizing exceptional postdoctoral researchers from Latin America. Working in Arnold Kriegstein’s lab at the Broad Center, Gomes de Oliveira studies how the immune system’s response to Zika virus affects the developing human brain. Her work bridges immunology and neuroscience, with implications for understanding how emerging pathogens affect fetal neurodevelopment.
Director's Letter * Strategic Planning * Research Highlights * Our Accomplishments * People * Growing the Vision
Our People
This year, in addition to Broad Center researchers publishing nearly 50 studies in top journals such as Nature and Science, they were featured in public forums, ranging from podcasts to international conferences, sharing discoveries that resonate far beyond UCSF — on brain development, Alzheimer’s disease, and regenerative therapies.
Highlights included:
When women scientists connect and lead, the whole field moves forward. Carolyn Sangokoya saw a gap and built UCSF’s Women Physician-Scientists Supergroup to change that. Through mentorship and collaboration, she’s creating a stronger future for women in science.
Tippi MacKenzie delivered the George Stamatoyannopoulos Memorial Keynote Lecture at the American Society of Gene & Cell Therapy conference, “Prenatal Therapies for Severe, Early-Onset Genetic Diseases,” highlighting her pioneering work in translational science and gene editing.
From his roots on a cattle farm in rural Virginia to pioneering advancements in gene editing, Kyle Cromer’s story is one of dedication, discovery, and scientific curiosity. Recently awarded a New Innovator Award, part of the NIH’s High-Risk, High-Reward Research Program, the People Behind the Science podcast shares Cromer's perspective on the future of gene editing and advice for aspiring scientists.
In a Freakonomics podcast, Cradle to Grave: Why Aren't We Having More Babies? Diana Laird, starting at 32:48, emphasizes the significant gaps in our understanding of reproductive biology, particularly regarding egg development and embryo implantation.
In the film Technologies Transforming Precision Medicine, Arnold Kriegstein explores the use of human brain organoids to model brain development and disease, including how three-dimensional cellular structures allow researchers to track early neural formation, understand the origins of neurological disorders, and test potential therapeutics.
In a short Abcam film, Robert Blelloch shares how an early fascination with how life begins sparked his passion for science and shaped his career. He also reflects on how science ultimately helped save his own life and connect him to a wider community.
Director's Letter * Strategic Planning * Research Highlights * Our Accomplishments * People * Growing the Vision
Growing the Vision
The Broad Center outreach has expanded to supporters and alumni through tours, newsletters, and events. We continue to look for new opportunities for friends of the Broad Center to engage with patient advocates.
Looking Ahead
As the Broad Center moves into another year of innovation and growth, core goals include:
- Recruiting faculty in stem cell biology and genome editing.
- Expanding lab space for high-throughput research.
- Launching new pilot grants and strengthening clinical research.
- Building partnerships with industry to accelerate therapies to patients.
The Broad Center is ready to lead the next chapter of regenerative medicine — bringing discoveries from the lab bench to the bedside, and delivering hope to patients worldwide.
Thank you for partnering with us to lead the next era of innovations in stem cell and gene therapy. Please consider supporting our research.
Director's Letter * Strategic Planning * Research Highlights * Our Accomplishments * People * Growing the Vision