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Start-Up Designing Synthetic DNA for Gene Therapies

DNA puzzle

(Arek Socha, Pixabay)

20 Apr. 2021. A new biotechnology company is underway creating gene therapies for inherited diseases delivered with synthetic DNA instead of benign viruses. Code Biotherapeutics Inc. in Hatfield, Pennsylvania began operations, staked to $10 million in seed funding from venture capital companies and genetic disease advocacy organizations.

Code Bio is developing therapies that deliver working genes to cells that replace or repair damaged or missing genes responsible for genetic disorders. The company is spun-off from, and appears to succeed another company, Genisphere LLC, also in Hatfield, Pa. Genisphere developed a therapy delivery technology called 3DNA that uses synthetic DNA as a nanoscale delivery mechanism for small molecule drugs, biologics, and nucleic acids, targeted with antibodies or peptides.

Code Bio is founded by Robert and Lori Getts, brother and sister who served in Genisphere’s scientific leadership, along with Brian McVeigh, Genisphere’s board chair. Robert and Lori Getts led development of 3DNA at Genisphere, and occupy similar roles at Code Bio. McVeigh is Code Bio’s CEO and board chair.

The company is developing gene therapies based on the 3DNA platform to fill unmet needs for complex inherited disease treatments. Most gene therapies, says Code Bio, use benign viruses like adeno-associated viruses that can penetrate cells and can deliver genetic payloads. The adeno-associated virus is a benign and naturally occurring microbe that infects cells, but does not integrate with the cell’s genome or cause disease, other than at most mild reactions. As a result, the virus has become a workhorse for delivering healthy genes to treat inherited diseases.

Deliver larger and more complex payloads

With 3DNA, Code Bio aims to overcome limitations of adeno-associated viruses, particularly delivery of larger and more complex genetic payloads needed by some rare inherited diseases and more common disorders such as type 1 diabetes. The company is developing gene therapies based on 3DNA for Duchenne muscular dystrophy, or DMD, caused by mutations in the dystrophin gene. DMD occurs mainly in males and leads to progressive muscle weakness and loss, including heart and respiratory muscles.

People with type 1 diabetes have an inherited autoimmune disorder where beta or islet cells in the pancreas do not produce insulin. Type 1 diabetes, or T1D, is diagnosed primarily in children or young adults, where the immune system is tricked into attacking healthy cells and tissue, in this case, insulin-producing islet cells.

Code Bio is raising $10 million in its seed funding round. The financing is led by life science venture investors 4BIO Capital in London and UPMC Enterprises in Pittsburgh. Taking part in the round are venture investors New Enterprise Associates and Takeda Ventures, with CureDuchenne Ventures and JDRF T1D Fund, the venture philanthropy arms of advocacy organizations CureDuchenne and JDRF.

McVeigh notes in a Code Bio statement that each seed investor brings “specialized areas of expertise and capabilities to the company that will enable us to rapidly deliver on the promise of our novel non-viral gene therapy approach.”

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