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Organisation › Details

4D Molecular Therapeutics LLC (4DMT)

4DMT is focused on the discovery and development of targeted and proprietary AAV gene therapy products for use in patients with severe genetic diseases with high unmet medical need. Our robust discovery platform, termed Therapeutic Vector Evolution, empowers us to create customized gene delivery vehicles to deliver genes specifically to any tissue or organ in the body, by optimal clinical routes of administration, at manageable doses and with resistance to pre-existing antibodies. These proprietary and targeted products may be developed to treat both rare genetic diseases and large market complex diseases. 4DMT is creating a diverse and deep product pipeline through its own internal 4D products, as well as through partnered programs. 4DMT patient advocacy organization partners in ophthalmology include Foundation Fighting Blindness and Choroideremia Research Foundation. About 4DMT’s Therapeutic Vector Evolution Gene therapy has shown promise for the treatment of rare diseases, yet current clinical stage gene therapy products are not targeted and are generally based on one of a few AAV vectors that are “naturally-occurring” or “wild-type”, meaning they were found in nature (e.g. as laboratory contaminants or as monkey infections). These first-generation AAV vectors, while generally safe and well-tolerated in patients, do not have targeted or optimized delivery properties and often require aggressive and/or invasive injection at high doses to attempt the desired transduction of target cells in the body. 4DMT is advancing the field of AAV vector technology by deploying principles of evolution and natural selection to create vectors that efficiently and selectively target the desired cells within the diseased human organ via clinically optimal routes of administration, at manageable doses and with resistance to pre-existing antibodies in the population. Our Therapeutic Vector Evolution platform deploys an estimated 100 million unique AAV variants with extensive diversity, from over 35 unique and proprietary 4DMT AAV vector libraries. After defining the Target Product Profile, and the associated Target Vector Profile, 4DMT then applies proprietary methods to identify lead vectors for the specific Target Vector Profile from within our AAV libraries. The result is a customized, novel, and proprietary pharmaceutical-grade product uniquely designed for targeted therapeutic gene delivery and efficacy in humans. *

 

Period Start 2014-01-19 existent
Products Industry adeno-associated viral vector (AAV vector)
  Industry 2 gene therapy
Person Person Kirn, David (4D Molecular Therapeutics 201804 CEO + Co-Founder)
     
Region Region Berkeley, CA
  Country United States (USA)
  Street 5980 Horton Street
Suite 460
  City 94608 Emeryville, CA
  Tel +1-510-505-2680
    Address record changed: 2020-12-02
     
Basic data Employees n. a.
     
    * Document for »About Section«: 4D Molecular Therapeutics. (4/30/18). "Press Release: 4DMT and Roche Expand Ophthalmology Partnership to Develop and Commercialize Multiple AAV Gene Therapy Products". Emeryville, CA.
     
   
Record changed: 2023-06-26

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