UC San Diego Sanford Stem Cell Institute

What UC San Diego Sanford Stem Cell Institute does

UC San Diego Sanford Stem Cell Institute (SSCI) is a non-profit academic research institute within the University of California San Diego system, governed by the Regents of the University of California, that operates the full pipeline from stem cell discovery to clinical trial delivery. It was established in 2013 through a $100 million philanthropic gift from T. Denny Sanford, formalized as a six-center institute in 2022 with a further $150 million gift, and is one of nine clinics in California's CIRM Alpha Stem Cell Clinics Network. The institute is structured around six interconnected centers: ISSCOR (space-based research aboard the International Space Station), the Stem Cell Fitness and Space Medicine Center, the Innovation Center (genomic technologies), the Discovery Center, the Advanced Therapy Center with cGMP manufacturing, and the Clinical Center housing the Alpha Clinic. Its core technology portfolio includes rebecsinib (the first and only FDA-approved IND for an ADAR1 inhibitor with potential efficacy across 23 cancer types), Cirmtuzumab, brain and tumor organoid modeling, eCLIP and STAMP RNA-mapping technologies, iPSC-derived NK cell therapy, and CRISPR-edited stem cell-derived cancer avatars.

SSCI serves three primary customer sets: patients with life-threatening and degenerative diseases who enroll in clinical trials through its Patient Registry, California-based academic investigators using the CIRM Alpha Clinic infrastructure, and biotechnology and pharmaceutical companies that sponsor clinical trials and contract for cGMP cell therapy manufacturing. The institute lists 17+ named industry sponsors including Regeneron, AstraZeneca, Fate Therapeutics, Lineage Cell Therapeutics, Neurona, Aspen Neuroscience, Aspera Biomedicines, GentiBio, Tr1X, A2 Biotherapeutics, Acepodia, Immunocore, Immatics, and TG Therapeutics, with services spanning project management, regulatory support, trial coordination, data management, and financial management. Revenue is not commercially derived; it is funded through the $250 million cumulative Sanford endowment, CIRM grants (part of $5.5B in California state stem cell funding), industry-sponsored clinical trials, and recharge rates paid by UC San Diego researchers. Clinical trial participation is free for qualifying patients; industry and academic pricing is quote-based via intake request forms.

Name

UC San Diego Sanford Stem Cell Institute

Legal name

Regents of the University of California

Website

https://stemcells.ucsd.edu

Company type

Private

Founded year

2013

Operating status

Operating

Headcount range

11–50 employees

Short description

UC San Diego Sanford Stem Cell Institute (SSCI) is a non-profit academic research institute running six interconnected centers that translate stem cell science and regenerative medicine, including space-based microgravity research, from discovery through cGMP manufacturing to clinical trials for patients and industry sponsors.

Ownership category

akta.pro rank

Product category

Regenerative Medicine Research

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417), Research and Development in the Physical, Engineering, and Life Sciences (54171)

SIC

Services-Commercial Physical & Biological Research (8731), Services-Specialty Outpatient Facilities, Nec (8093)

akta.pro primary industry

Stem Cell Therapies (MSC/iPSC/HSC-derived) (HLAAABAE)

akta.pro secondary industries

Cell Therapy Clinical Services & Treatment Centers (apheresis, administration, monitoring) (HLAAABAJ), Stem Cell–Based Regenerative Therapies (HLAAAGAA), Cell Therapy Manufacturing Services (GMP, autologous/allogeneic) (HLAAAKAC), Cell Therapy Manufacturing Platforms & Automation (closed systems, bioreactors, fill-finish) (HLAAABAG), Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL)

Keywords

• Stem cell research
• Regenerative medicine
• Cell therapy development
• Clinical trial services
• CGMP manufacturing

Headquarters

HQ city

La Jolla

HQ country

United States

HQ region

North America

Offices5 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Technology or R&D, Infrastructure, Operations

Revenue model

1. Philanthropic funding: Primary funding from T. Denny Sanford's $100M gift in 2013 and $150M gift in 2022 establishing and expanding the institute.
2. CIRM funding: California Institute for Regenerative Medicine provides $5.5 billion in funding with 150+ active stem cell programs. UCSD Alpha Clinic is part of CIRM's Alpha Clinic network.
3. Industry partnerships and clinical trial sponsorships: Pharmaceutical and biotech companies sponsor clinical trials conducted at the Alpha Clinic, including Regeneron, AstraZeneca, Fate Therapeutics, and others.
4. Recharge rates for UC San Diego researchers: UC San Diego researchers pay for Alpha Clinic services including project management, regulatory support, trial coordination, data management, and financial management.

Pricing tiers

Go-to-market motion1 record

Distribution channels4 records

Marketing channels9 records

Core offering

The Sanford Stem Cell Institute is a non-profit academic research institute that conducts translational stem cell and regenerative medicine research, runs early-phase cell and gene therapy clinical trials through the CIRM Alpha Clinic, provides cGMP-compliant cell therapy manufacturing at the Advanced Cell Therapy Laboratory, and develops novel therapeutics including the ADAR1 inhibitor rebecsinib. It operates six specialized centers spanning discovery, innovation, clinical delivery, space-based microgravity research aboard the International Space Station, fitness and space medicine, and advanced therapy manufacturing.

Product overview

The UC San Diego Sanford Stem Cell Institute is a global leader in regenerative medicine and hub for stem cell science and innovation, both on Earth and in space. The institute comprises six interconnected centers: the ISSCOR Center (space-based stem cell research), Stem Cell Fitness and Space Medicine Center, Innovation Center (genomic technologies and AI), Discovery Center (translational research), Advanced Therapy Center (GMP production), and Clinical Center/Alpha Clinic (clinical trials). Additional programs include the Brain Tumor & Neurorestoration Program, Advanced Cell Therapy Laboratory (ACTL), and the reGEN Podcast. The institute develops novel therapeutics including Rebecsinib and conducts clinical trials spanning cancer, neurological diseases, diabetes, and spinal cord injury.

Differentiator

Problem solved

Functional benefit

Brands

• reGEN Podcast: Podcast featuring discussions on stem cell science, regenerative medicine, clinical trials, research outcomes, and space-based research
• Rebecsinib
• ISSCOR Center

Products and services

• Alpha Stem Cell Clinic Clinical Trial Services CIRM Alpha Stem Cell Clinic offering inpatient cell and gene therapy clinical trials and outpatient support; provides project management, regulatory support, trial coordination, data management, and financial management services to industry sponsors and academic investigators.
• Advanced Cell Therapy Laboratory (ACTL) cGMP Manufacturing Services cGLP/cGMP-compliant manufacturing facility providing cell banking, process scale-up, product characterization, and cell therapy manufacturing for IND-enabling studies and Phase 1/2/3 clinical trials for investigators and industry sponsors.
• Rebecsinib (ADAR1 Inhibitor Drug Candidate) Investigational anti-cancer drug and ADAR1 inhibitor; first and only ADAR1 inhibitor with an FDA-approved investigational new drug application. Entered Phase 1 clinical trials with potential against 23 cancer types involving ADAR1. Currently being tested aboard Axiom Space Ax-3 and Ax-4 missions using tumor organoid models.
• Brain Tumor and Neurorestoration Program Research and therapeutic program exploiting therapeutic weaknesses of brain cancers through creation of stem cell-derived cancer avatars, CRISPR-edited to include common cancer mutations including glioblastoma and pediatric atypical teratoid rhabdoid tumor models.
• Patient Registry Registry enabling patients to be contacted about qualifying clinical trials and contribute to long-term health outcome tracking for regenerative medicine research advancement.

Quantifiable outcome

• Rebecsinib halted growth of ADAR1-expressing breast cancer organoids in Ax-3 mission
• +4 more outcomes

Named customers6 records

Segments4 records

Ideal customer profiles4 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature10 records

Partnerships

29 partnerships are on record, tiered core and minor.

• California Institute for Regenerative Medicine (CIRM)coreStrategic or Co-development PartnerUCSD Alpha Clinic is one of nine Alpha Clinics in CIRM's network. CIRM was created by California voters in 2004 (Proposition 71) with $5.5 billion in funding and 150+ active stem cell programs.
• Axiom SpacecoreStrategic or Co-development PartnerSpace company conducting missions aboard International Space Station for rebecsinib drug testing on tumor organoids and SASHA stem cell aging studies. Ax-4 mission scheduled for June 2025.
• NASAcoreStrategic or Co-development PartnerNASA funding supports ISSCOR Center research. NASA is working with U.S. Department of Health and Human Services to help cut cancer death rate by 50% in 25 years through space-based research.
• Sanford Consortium for Regenerative MedicinecoreStrategic or Co-development PartnerCo-location of Sanford Stem Cell Institute headquarters at the Sanford Consortium for Regenerative Medicine building at 2880 Torrey Pines Scenic Drive, La Jolla.
• ISS National LabscoreStrategic or Co-development PartnerInternational Space Station National Laboratory supports space-based stem cell and cancer research, awarded Dr. Jamieson the 2024 Compelling Results Award.
• City of Hope Medical CenterminorStrategic or Co-development PartnerTrial sponsor for CMV/HIV-CAR T cell therapy clinical trial at UCSD Alpha Clinic.
• Sumitomo Dainippon PharmaminorStrategic or Co-development PartnerCo-sponsor with UCSD of CT1-DAP001 Parkinson's disease clinical trial using human induced pluripotent stem cell-derived dopaminergic progenitors.
• Aspen NeuroscienceminorStrategic or Co-development PartnerTrial sponsor for ANPD001 sporadic Parkinson disease clinical trial.
• Aspera BiomedicinesminorStrategic or Co-development PartnerCo-sponsor with UCSD of rebecsinib Phase 1 clinical trial for secondary acute myeloid leukemia and myelofibrosis.
• Lineage Cell TherapeuticsminorStrategic or Co-development PartnerTrial sponsor for DOSED spinal cord injury clinical trial using LCTOPC1 cell therapy.
• Regeneron PharmaceuticalsminorStrategic or Co-development PartnerTrial sponsor for ubamatamab (REGN4018) platinum-resistant ovarian cancer clinical trial.
• Neurona TherapeuticsminorStrategic or Co-development PartnerTrial sponsor for NRTX-1001 focal epilepsy clinical trial using inhibitory interneurons.
• TG TherapeuticsminorStrategic or Co-development PartnerTrial sponsor for azercabtagene zaprelecuel B-cell mediated autoimmune disorders clinical trial.
• Tr1X BiominorStrategic or Co-development PartnerTrial sponsor for TRX319 progressive multiple sclerosis clinical trial.
• GentiBiominorStrategic or Co-development PartnerTrial sponsor for GNTI-122 recently diagnosed type 1 diabetes clinical trial.
• AstraZenecaminorStrategic or Co-development PartnerTrial sponsor for AZD5492 B-cell malignancies clinical trial.
• Fate TherapeuticsminorStrategic or Co-development PartnerTrial sponsor for FT836 off-the-shelf CAR T-cell therapy solid tumors clinical trial.
• ImmaticsminorStrategic or Co-development PartnerTrial sponsor for IMC-F103C melanoma clinical trial.
• A2 BiotherapeuticsminorStrategic or Co-development PartnerTrial sponsor for A2B395 logic-gated CAR T solid tumors clinical trial.
• Acepodia BiotechminorStrategic or Co-development PartnerTrial sponsor for ACE2016 allogeneic gamma delta T cell therapy solid tumors clinical trial.
• ImmunocoreminorStrategic or Co-development PartnerTrial sponsor for brenetafusp (IMC-F106C) PRAME-positive cancers clinical trial.
• Space TangominorStrategic or Co-development PartnerSpace company mentioned in 2022 symposium presenting on space-based research capabilities.
• Sierra SpaceminorStrategic or Co-development PartnerSpace company mentioned in 2022 symposium presenting on space-based research.
• Ventrix, Inc.minorStrategic or Co-development PartnerBiotech company co-founded by Dr. Karen Christman (Co-Director, Sanford Advanced Therapy Center) for biomaterials technology commercialization.
• Karios Technologies, Inc.minorStrategic or Co-development PartnerBiotech company co-founded by Dr. Karen Christman for additional biomedical technology development.
• EclipseBiominorStrategic or Co-development PartnerBiotech company where Dr. Gene Yeo serves as scientific advisor for RNA technologies.
• Orbital TherapeuticsminorStrategic or Co-development PartnerBiotech company where Dr. Gene Yeo serves as scientific advisor.
• InsitrominorStrategic or Co-development PartnerBiotech company where Dr. Gene Yeo serves as scientific advisor for AI-driven drug discovery.
• Sanford Burnham PrebysminorStrategic or Co-development PartnerMedical research institute partner represented on SSCI Advisory Council with David Brenner as President and CEO.

Scale indicators7 records

Recent moves9 records

Expansion highlights6 records

Direct peers

• Fred Hutchinson Cancer Center: Pioneer in hematopoietic stem cell transplantation and a major cell therapy research center. Directly comparable as a peer academic research center with extensive cell therapy clinical trial infrastructure and biomanufacturing capabilities.
• City of Hope: Cancer research and treatment center with major cell therapy GMP manufacturing and clinical trial capabilities. Directly comparable as a peer Alpha Clinic member (CIRM network) and a trial sponsor (e.g., CMV/HIV-CAR T trial at UCSD); leader in hematopoietic cell transplantation and CAR-T therapy.
• Gladstone Institutes: San Francisco-based academic research institute with focus on stem cell biology, regenerative medicine, and disease modeling. Comparable as a peer non-profit research institute with shared stem cell research and regenerative medicine focus, and physical co-location with UCSF.
• Harvard Stem Cell Institute: Harvard-affiliated academic stem cell research institute conducting basic and translational stem cell research. Comparable as a peer-tier academic stem cell research center with multi-disease focus and significant funding base.
• Stanford Institute for Stem Cell Biology and Regenerative Medicine: Stanford's academic stem cell research institute with translational programs spanning cancer, neuroscience, and regenerative medicine. Directly comparable as a California-based peer academic institute with industry partnerships and clinical trial infrastructure.
• Yale Stem Cell Center: Yale's academic stem cell research center focused on basic and translational stem cell biology. Comparable as a peer academic stem cell research center with disease focus across multiple indications.

Broad incumbents

• MD Anderson Cancer Center: Largest U.S. cancer center with extensive cell therapy clinical programs and biomanufacturing capabilities. Comparable as a broad incumbent in oncology and cell therapy research, with significantly greater scale and a broader geographic footprint.
• Dana-Farber Cancer Institute: Major cancer research and treatment center with significant cell therapy and immunology research programs. Comparable as a broad incumbent in oncology research and clinical trial infrastructure, with broader oncology focus than SSCI.
• Memorial Sloan Kettering Cancer Center: Major cancer center with extensive cell therapy clinical trial infrastructure and GMP manufacturing. Comparable as a broad incumbent in oncology cell therapy research and academic clinical trial delivery, though operationally larger and more focused on oncology than SSCI.

Others

• California Institute for Regenerative Medicine (CIRM): California's state agency funding stem cell research with $5.5B in funding and 150+ active programs. Comparable as the umbrella ecosystem player that SSCI's Alpha Clinic operates within — a key funding and network partner rather than a direct competitor.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/ucsdstemcell
• YouTubeyoutube.com/@UCSDStemCells
• LinkedInlinkedin.com/company/ucsdstemcells
• Instagraminstagram.com/ucsdstemcells

Revenue estimate

Valuation estimate

Number of profiles

Profiles17 records

Subsidiaries7 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about UC San Diego Sanford Stem Cell Institute