Unlocking the power of genetically modified mesenchymal stem cells

Unlocking the power of genetically modified mesenchymal stem cells


Kyttaro Therapeutics is one of the few companies with a patent estate across a portfolio of genetically-modified mesenchymal stem cells (MSCs), offering a powerful tool to enhance the viability, homing, and therapeutic function in naïve MSCs.

These genetically-modified MSCs provide more effective and targeted, off the shelf, allogeneic, cell-based therapies in large markets with unmet need, and longevity. 

Kyttaro was founded with a singular vision:

Kyttaro was founded with a singular vision:




To improve the quality of life for patients suffering from diseases with high unmet medical needs by developing unique treatments based on genetically-modified MSCs.

To improve the quality of life for patients suffering from diseases with high unmet medical needs by developing unique treatments based on genetically-modified MSCs.

Accomplishments to Date


Kyttaro Therapeutics is headquartered in Philadelphia PA, with scientific roots in Munich, Germany.


The Company was founded to unlock the full therapeutic potential of MSCs through genetic engineering. Kyttaro’s platform generates three proprietary, patent-protected, genetically modified MSC cell lines — MSC-AAT, MSC-Cytokine (MSC-Cyt), and MSC-Klotho — each of which addresses significant unmet medical needs across multiple large-market indications.  The Company has also developed patents on naïve MSCs with CAR-T cells designed to enhance the anti-tumor activity of immunotherapy.

Kyttaro’s intellectual property and preclinical data were originally generated over more than a decade at Apceth Biopharma GmbH (Munich), representing over $40 million in R&D investment. That work established the genetic modification protocols, bioreactor manufacturing processes, and initial clinical evidence that form the foundation of Kyttaro’s programs today. This data is available under NDA to qualified investors and partners.

We have developed a robust patent portfolio across three varieties of genetically-modified MSC's.


Swipe sideways to view the full patent portfolio →

46 patents MSC-Klotho MSC-AAT MSC-Cytokines MSC for CAR-T GRANTED 41 patents Granted (8) US EP IT GB FR ES DE CH Granted (16) AU JP CA IL EP SE IT IE HK GB FR ES DE CH BE AT Granted (8) US JP CN CA EP GB FR DE Granted (9) JP EP IT HK GB FR ES DE CH PENDING 5 patents Pending (2) US CN Pending (3) US CA AU TOTALS 8 granted 16 granted · 2 pending 8 granted 9 granted · 3 pending

Kyttaro wholly owns the entire patent estate created by Apceth Biopharma GmbH. The IP was assigned to Kyttaro by contract from JuntuCell (which had received it when Apceth's manufacturing business was sold to Hitachi Chemical/Minaris in 2019). Certain patent registers may still list a prior holder pending administrative re-recording of assignments; however, beneficial and legal ownership rests with Kyttaro Therapeutics, Inc.


All Apceth scientific and operational documentation has been assembled into a Kyttaro Document Database comprising 8 years of patented R&D and pre-clinical work that is now readily accessible via a retrieval augmented generative AI system named Kyttaro Document Intelligence.



Leadership

Kyttaro is led by an internationally recognized executive team and scientific / strategic advisory boards with deep industry knowledge and experience.The founding team combines deep expertise in cell and gene therapy, clinical development, regulatory strategy, healthcare investment, and corporate law. Our Scientific Advisory Board includes leading physicians from Children’s Hospital of Philadelphia, Johns Hopkins University, and Albert Einstein College of Medicine / Montefiore.


Meet the Team

Operations

Kyttaro’s operations are headquartered at BioLabs Philadelphia (co-working and shared lab space with ancillary operations in Munich), and will collaborate with Thermo Fisher’s Advanced Therapies Collaboration Center there to create comprehensive, end- to-end manufacturing workflows.


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Infrastructure

Kyttaro has formed a Supply Agreement with the New York Blood Center and has access to its FDA approved Master Cell Bank of umbilical cord-derived mesenchymal stem cells already being used for cell transplantation procedures with appropriate preparative regimens.

Kyttaro's manufacturing platform begins with a well-defined, cord blood–derived MSC source, established through our partnership with the New York Blood Center. Each of our three MSC lines is engineered using lentiviral transfection. The process steps for MSC propagation, lentivirus construction, and lentivirus transduction have been optimized over 10+ years of development and reviewed by European regulators, Germany's Paul-Ehrlich-Institut. The production process has been developed to GMP standards.  Protocols for the characterization and phenotyping of MSCs, and for measuring protein production levels, have also been established to GMP standards. This foundation in manufacturing, characterization, and potency underpins every program in the Kyttaro pipeline.

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Pipeline

The Company has chosen MSC-Klotho for neurodegenerative disease and longevity as its initial product candidate and has established a partnership to perform in-vivo, proof of concept studies for ALS within nine months of commencement.

MSC-AAT was approved by the German authorities and prepared to initiate a Phase I/II clinical study in steroid-refractory GvHD in Europe, demonstrating long-term survival in over 50% of the research subjects and anti-inflammatory activity compared to naïve MSCs. Kyttaro is now prepared to commence discussions with the FDA in the US. The FDA recently approved Mesoblast’s Ryoncil for the pediatric population in GvHD, the first MSC therapy ever approved in the US, which validates the modality.


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MSC-Klotho is a patent protected ALS program designed to reach its next major value inflection over 24 months.


0-6 months


Preclinical Workup

Randomized design, route / regimen, comparator arms

6-12 months


Blinded efficacy readout

Survival, motor function, biomarkers

12-18 months


Second ALS model

Translatability + direct add-on value

18-24 months


Translation package

Potency, release, biodistribution, FDA-ready path

24 months


Results
  • Direct efficacy
  • Biomarkers
  • Series A package

Outcome:

A financeable orphan ALS program with a Phase 1/2-ready clinical path

Next steps


Kyttaro is currently seeking seed stage financing which will lead to a larger Series A focused on Phase I clinical initiation within 24 months. Kyttaro is poised to conclude preclinical partnerships for MSC-Klotho and commence discussions with the FDA to initiate a Phase I/ II clinical study in GvHD in the US. We invite interested partners to contact us for more details.

Next steps

Next steps


Kyttaro is poised to commence clinical trials in Europe and the US for COVID-19 and GvHD applications. We invite interested partners to contact us for more details.

Kyttaro is currently seeking seed stage financing which will lead to a larger Series A focused on Phase I clinical initiation within 24 months. Kyttaro is poised to conclude preclinical partnerships for MSC-Klotho and commence discussions with the FDA to initiate a Phase I/ II clinical study in GvHD in the US. We invite interested partners to contact us for more details.