Synbal’s mission is to develop safer, non-viral, cell-based gene medicine treatments of rare genetic diseases using its innovative next generation gene editing platform: ACTIVE GENETICS

  • We're at a key point when it comes to cell and gene therapy. These therapies have the potential to address hundreds, if not thousands, of different rare and common diseases. For a long time, they were largely theoretical constructs. Now they're a therapeutic reality. And it's my expectation that they will soon become the mainstay of how we treat a wide range of illness.

    Scott Gottlieb, May 22, 2018 FDA Commissioner

OUR TECHNOLOGY

Synbal is a biotech company using a proprietary gene-editing platform, Active Genetics, developed at UC San Diego.  Our technology allows the development of next-generation laboratory animal models and autologous cell-based treatments for patients suffering from rare genetic diseases.  Our platform combined with innovative proprietary technology resolves current problems associated with autologous cell therapy and dramatically improves patient safety.

Synbal’s unique advantage is its proprietary technology platform which delivers large DNA cargos (>7 kb) to one chosen DNA site improving safety.  These capabilities allow us to incorporate patient safety elements into our gene edits such as control promoters-operators and kill switches.

 

This groundbreaking technology provides the following advantages:

  1. Higher efficiency: it results in increased patient cell transformation rate.
  2. Large payload: it allows for a site-directed delivery of large genetic cargos.
  3. No virus: it avoids random integration and associated oncology concerns.
  4. Kill switch: it allows for unprecedented control of therapeutic strength.

 

Synbal’s technology originated in the laboratories of Dr. Ethan Bier and Valentino Gantz of UCSD. The unique benefits of their technology innovation recruits Homology-Directed Repair (HDR) coupled with CRISPR/Cas9 (or other nucleases), a site-specific gRNA, and other proprietary technologies making HDR much more efficient in somatic cells.

Synbal’s Active Genetics Technology Applied to Laboratory Animals and Cell-Based Therapuetics

Download Synbal’s Executive Summary >>

 

Synbal’s Progress:

  • September 2018: Synbal was awarded a Small Business Innovation Research (SBIR) Fast Track award for $2.3M to develop a humanized rat model. This rat will express 9 different human Cytochrome P450 genes responsible for metabolizing 80% of commercial small molecule drugs. If successful, this will provide an invaluable resource for studying human drug metabolism in rat models for drug pharmacokinetics validation of study of human diseases.
  • October 2018: Synbal opened its laboratories in the BioLabs facility in San Diego, CA. Currently there are two scientific staff at the PhD level along with the CSO and the CEO.
  • October 2018: A second contract ($2.6M) has also been awarded to Synbal by a Biopharmaceutical company to develop a humanized, multigene animal model in the mouse.  The funds from both sources are exclusively for the development of laboratory animal models and provide the company with sufficient operating funds for approximately three years.

Synbal’s proprietary TETHRing technology

 

Synbal’s proprietary TETHRing technology making homology directed repair (HDR) more efficient in somatic cells.

Key:  Clamp Unique proprietary clamp technology, Left/Right Homology ~1Kb of exact match DNA to genome, gRNA, or guide RNA; Cargo, DNA sequence to be inserted at the target site.  The Clamp & Cas9 (or similar enzyme) will be delivered in trans, and removed before any product is administered to patients.

OUR TEAM

Management

  • David Webb CEO and Board Member

    Former VP of Research, Celgene
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  • Kurt Jarnagin Chief Scientific Officer

    Former Pfizer, Anacor, Iconix
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Founders

  • Valentino Gantz Board Member & Founder

    Assistant Research Scientist, UC San Diego
    Co-Inventor of Active Genetics: Gene Drive Technology
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  • Tsvi Goldenberg Chairman of the Board & Founder

    Former CEO at Itherx (Immusol)
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  • Ethan Bier Board Member & Founder

    Distinguished Professor, UC San Diego
    Co-Inventor of Active Genetics: Gene Drive Technology
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Scientific Advisory Board

  • Valentino Gantz Board Member & Founder

    Assistant Research Scientist, UC San Diego
    Co-Inventor of Active Genetics: Gene Drive Technology
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  • Ethan Bier Board Member & Founder

    Distinguished Professor, UC San Diego
    Co-Inventor of Active Genetics: Gene Drive Technology
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  • Stephen Hedrick Distinguished Professor, UC San Diego

    Co-discoverer of the T-Cell Receptor
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  • Rick Morrissey President Biotech Development

    Former VP of Development, Celgene
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  • Kim Cooper, PhD Scientific Advisory Board

    Professor, UCSD
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Business Advisors

  • Dan Koeppen
    Dan Koeppen Legal Affairs

    Wilson Sonsini Goodrich Rosati

  • Sibylle Hauser Fundraising & Corporate Business Development Strategist

    Hauser Strategy Consulting
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Board of Directors

  • Valentino Gantz Board Member & Founder

    Assistant Research Scientist, UC San Diego
    Co-Inventor of Active Genetics: Gene Drive Technology
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  • Tsvi Goldenberg Chairman of the Board & Founder

    Former CEO at Itherx (Immusol)
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  • Ethan Bier Board Member & Founder

    Distinguished Professor, UC San Diego
    Co-Inventor of Active Genetics: Gene Drive Technology
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  • David Webb CEO and Board Member

    Former VP of Research, Celgene
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  • Clyde Shores Board Member

    Former Abbott, Amgen, Baxter
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CONTACT US

For questions about press releases please contact: IR@synbal.com
For all other inquiries please contact: 858-771-3788

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