Engineered viruses are the carriers of choice for many genetic medicines, but they pose several problems. In addition to the risk of dangerous immune responses, these viruses do not spread to all tissue types, and there are limits to the size of the genetic cargo they can carry. Code Biotherapeutics overcomes these problems by completely avoiding viral transmission. The startup, which claims its approach could turn genetic medicines into treating more diseases, now has $75 million to get closer to testing its technology in humans.
This A round The financing announced Tuesday was led by Northpond Ventures.
Code Bio’s approach to delivering genetic medicines is based on synthetic DNA. The company can add targeting molecules—peptides, antibodies, or small molecules—to the structure. The structure also carries genetic cargo. Code Bio, based in Hatfield, Pennsylvania, just outside of Philadelphia, calls its technology 3DNA.
One of the key problems with engineered viruses is that the body sees them as foreign viruses, leading to an immune response. Because the body produces antibodies against the virus, patients cannot be re-treated if necessary. These restrictions limit viral-based genetic medicines to rare diseases. Because its technology does not carry the same risk of immune responses and has a larger genetic payload capacity, Code Bio said it could address a wider range of genetic diseases with repeatable treatments. Additionally, the company claims its technology has manufacturing advantages. 3DNA structures can be fabricated and stored in bulk. When genetic medicine is needed, 3DNA can be pulled off the shelf. All that is required to make the final product is to attach the targeted molecule and add the genetic payload.
Code Bio has two major programs so far, one targeting the rare disease Duchenne muscular dystrophy and the other targeting the more common type 1 diabetes. The gene therapy Duchenne is developing uses an adeno-associated virus to deliver a functional version of a gene that encodes a key protein these patients lack. But AAV’s capacity limitations mean that shortened versions of the genes used in these gene therapies cannot be re-administered. Code Bio aims to overcome these limitations faced by the genetic medicines that Duchenne is developing.
For type 1 diabetes, Code Bio is working with the Juvenile Diabetes Research Foundation to explore ways to increase the amount of beta cell function that produces the insulin these patients lack. The company said the new funding will be used to advance both programs into preclinical studies that can support investigational new drug applications. The cash will also be used to expand the pipeline and platform into other indications as well as manufacturing.
Code Bio isn’t the only company pursuing non-viral delivery methods for genetic medicines. ReCode Therapeutics is developing therapeutics based on lipid nanoparticle (LNP) delivery. While LNPs preferentially enter the liver, ReCode’s technology targets the liver for removal, enabling targeting of other tissue types. The technology also provides greater payload capacity than engineered viruses.
Code Bio emerged from stealth last year, getting $10 million Seed funding. Diana Bernstein, vice president of Northpond Ventures, will join Code Bio’s board of directors with the latest financing.
“Code Bio’s targeted 3DNA delivery platform is designed to extend the utility of genetic medicines beyond what is currently provided by viral gene delivery to support the development of translational therapies,” Bernstein said in the funding announcement.
Other investors in Code Bio’s Series A round include Amgen Ventures, Hatteras Venture Partners and UCB Ventures. Early investors New Enterprise Associates, 4BIO Capital, CureDuchenne Ventures, JDRF T1D Fund, UPMC Enterprises and Takeda Ventures also participated.
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