Cell Source, Inc.

Company overview

• Legal name: Cell Source, Inc. (Nevada corporation)
• Focus: cell therapy company specializing in immunotherapy
• Core technology: Veto Cell platform (anti-third-party central memory CD8 T cells) licensed from Yeda Research & Development Co., Ltd.
• Primary R&D hub: University of Texas MD Anderson Cancer Center, Houston, TX
• Business objective: enable selective tolerance of transplanted cells and organs to support allogeneic transplants and off-the-shelf cell therapies while reducing immune suppression and related complications

Technology and approach

Veto Cell platform

• CD8 central memory T-cell–based approach that selectively attenuates host T-cell clones that target donor cells
• Key properties:
  • surface features trigger targeted immune engagement
  • can eliminate attacking host T cells
  • recognizes third-party/viral peptides to reduce GvHD and rejection risk
  • long-lived in the body
  • migrates to thymus and lymph nodes
• Mechanism is intended to support chimerism and sustained donor–host tolerance with mild immune suppression

Intended applications

• Hematopoietic stem cell transplantation (HSCT) with haploidentical donors under reduced-intensity conditioning (RIC)
• Enabling allogeneic CAR-T and other gene-modified cell therapies (VETO CAR-T, NK, TCR approaches) by improving persistence and reducing rejection/GvHD
• Tolerance therapies for organ transplantation (starting with kidney, then liver) and for non-malignant indications such as sickle cell disease, aplastic anemia, and Type 1 diabetes risk reduction

Proprietary scope

• Exclusive worldwide license from Yeda for the Licensed Information and Patents
• Patents and applications cover Veto Cell technology and combinations with CAR-T/TCR/NK therapies
• Royalty obligations: 4% of net product sales payable to Yeda; $50,000 annual license fee until royalties commence
• License terms extend for the life of the patents with country-by-country expiration and milestones tied to clinical development

Products and programs (planned)

1. VETO CAR-T HSCT cell therapy for donor-mismatched allogeneic HSCT (haploidentical donors) for blood cancers
2. Veto Cell–based haploidentical organ transplantation (kidney first, then liver) using donor-derived stem cells and HSCT
3. Off-the-shelf VETO CAR-T therapy using Veto Cells to enhance persistence of allogeneic CAR-T products
4. Veto Cell–based tolerance therapy for non-malignant disorders (e.g., sickle cell disease, aplastic anemia, Type 1 diabetes prevention in at-risk individuals)

Target indications and market context

• Initial focus: hematologic malignancies — lymphoma, leukemia, multiple myeloma; haploidentical allogeneic HSCT candidates are a primary target
• Additional targets: organ transplantation (kidney, liver), blood disorders (sickle cell disease, beta-thalassemia, aplastic anemia), and autoimmune disease contexts such as Type 1 diabetes risk reduction
• Market context:
  • Company references a broad cancer immunotherapy market exceeding $250 billion with growth projections
  • More than 1,800 transplantation centers worldwide; roughly 900 centers in North America and Western Europe; over 300 centers in the US provide CAR-T therapy
  • Initial commercialization strategy targets about 100 leading centers, followed by broader adoption through publications, conferences, and clinician engagement

Development status and clinical trials

• Anti-Viral Veto Cells (lead candidate)
  • Phase 1/2 clinical trial in the US sponsored by Cell Source at MD Anderson
  • Interim results reported: the Phase 1/2 trial completed the first five treatment cohorts with 15 patients each (haploidentical HSCT under reduced-intensity conditioning with Veto Cells)
  • The trial is reported as continuing under the existing protocol toward a total of 24 patients (per the document)
  • MD Anderson Principal Investigator: Prof. Richard Champlin
• CAR-T and combined approaches
  • Collaboration with Zelig Eshhar (inventor of CAR-T); initial collaboration completed and license remains in place
  • Preclinical data indicate potential for combining Veto Cells with CAR-T to enable allogeneic CAR-T with reduced rejection/GvHD and improved persistence
• Other development notes
  • Preclinical data cited for engraftment under mild conditioning, reduced GvHD, and anti-viral protection
  • Roadmap includes advancing VETO CAR-T HSCT programs and exploring expansion to solid tumors and other indications

Intellectual property and licensing

• Primary IP: Veto Cell platform licensed exclusively from Yeda
• Portfolio summary:
  • 14 patent families; 73 granted patents; 1 allowed; 29 pending
  • Patent coverage across the US, Europe, China, Israel, India, Japan, Korea, Canada, Australia, and other jurisdictions, addressing topics such as anti-third-party central memory T cells, central memory T cells for leukemia/lymphoma treatment, genetically modified Veto Cells, anti-viral Veto Cells, and Veto CAR-T Cells
• License terms with Yeda:
  • Exclusive worldwide license to Licensed Information and Patents
  • Royalty: 4% of product sales; $50,000 annual license fee until royalties commence
  • Milestones tied to clinical progress and commercialization (e.g., Phase 2 commencement, marketing approval, first commercial sale)
  • Sublicense restrictions and consent requirements (with certain China affiliate exceptions)
  • Yeda retains ownership of the patents; Cell Source holds exclusive development, manufacturing, and sales rights under the license; default could result in loss of licensed rights

Corporate structure and operations

• Employees: only the CEO is a full-time employee; the company relies on independent contractors and consultants
• Facilities and production:
  • Headquarters in New York
  • Initial cell processing facilities expected to be colocated with major transplantation centers (for example, near MD Anderson)
  • Later plan to develop stand-alone GMP production facilities; estimated capital cost per stand-alone facility at least $10 million
  • Veto Cells prepared at Cell Source facilities or partner CMOs adjacent to transplant centers and infused at the time of transplantation
• Regulatory approach: pursuing FDA regulatory approvals (IND pathway) and EMA processes; fast-track and other expedited mechanisms are being discussed as possible options

Market access and commercialization approach

• Short-term revenue options being explored prior to full regulatory approval, including treating patients after Phase 2 under expedited pathways and pursuing upfront and milestone-based licensing agreements with third parties
• The company expects reimbursement to be favorable once clinical efficacy and regulatory approvals are established

Financial highlights (historical)

• Revenue: none generated to date
• Net losses:
  • 2024: $(4,512,642)
  • 2023: $(5,321,212)
• Cash and liquidity (Dec 31, 2024):
  • Cash on hand: $74,631
  • Working capital deficiency: $(18,537,836)
  • Accumulated deficit: $(46,180,030)
• Past-due liabilities:
  • Notes payable past due: $2,588,593 (current) and $11,015,939 (long-term) as of Dec 31, 2024
• Financing and liquidity:
  • Historically financed through equity and debt; the company reports an ongoing need for additional capital
  • Management projects total financing requirements of at least $50 million to reach commercialization milestones
• Going concern:
  • The company reports substantial doubt about its ability to continue as a going concern for at least one year from the filing date given cash, deficits, and past-due notes

Market position and competitive landscape

• Competes in HSCT, GvHD management, anti-viral post-transplant strategies, and allogeneic CAR-T development
• Competitors include developers pursuing reduced-intensity conditioning approaches, other GvHD-focused programs, and autologous/allogeneic CAR-T companies
• Value proposition: combine T-cell depletion HSCT under mild conditioning with anti-viral protection and potential integration with CAR-T to create a safer, end-to-end allogeneic transplantation and cancer therapy strategy

Key takeaways

• Cell Source is an early-stage biotechnology company developing the Veto Cell immunotherapy platform under an exclusive license from Yeda, with MD Anderson as a core clinical partner
• The company aims to enable safer allogeneic HSCT, reduce GvHD and viral complications, and support off-the-shelf CAR-T and other allogeneic cell therapies
• No products have been approved or commercialized and no revenue has been generated to date
• Substantial financing and regulatory progress are required to advance development and commercialization plans
• The company depends on a single primary license (Yeda) for core IP and on MD Anderson for clinical development, with limited internal staffing and reliance on contractors and partners