Homology Medicine, a gene editing startup, completes Series A funding and raises $43.5 million through 5AM Ventures and Arch Venture Partners.

Various gene editing techniques, including the CRISPR-Cas9 method, with its promise of curing serious illnesses, have piqued the interest of doctors, patients and scientists alike. Composed of biotech veterans, Homology said that their core science is better than the gene editing techniques previously known. Although the group has no published results yet, its backers are hopeful that the startup will remain true to its claim.

"The company has an aggressive development plan and a significant opportunity to leapfrog current strategies in gene therapy and editing as it builds an expansive and high-value clinical pipeline," said Kush Parmar, a managing partner at 5AM Ventures and board member of Homology.

The startup aims to apply their technology to rare diseases such as sickle cell disease by removing the faulty genes and replacing it with healthy ones without the safety issues faced by present gene therapy and gene editing methods.

In existing gene therapy, adeno-associated viruses (AAV) are used as a delivery tool wherein it goes inside a cell before it expresses a protein until the cell dies.

Homology claims that they can recreate homologous recombination, in which a chromosome can change one short DNA sequence for another one. What Homology hopes to do is to engineer a healthy DNA, embed it into an AAV and infuse it inside the body. This way, the DNA can attach to the affected cell, enter it and subsequently release the healthy DNA and replace the faulty genes.

However, using AAVs to develop homologous recombination is not a novel idea. University of Utah genetics expert Dana Carroll said that use of AAVs have been existing for many years now, and there are both advantages and disadvantages to the approach. Carroll explained that while there is no risk of causing DNA mutations in target genes, the desired outcome for gene swap is significantly lower compared when gene editing homologous recombination was employed.

Homology CEO Arthur Tzianabos is betting on the AAV type discovered by Beckman Research Institute at the City of Hope Virology Professor Saswati Chatterjee, who published the study in Molecular Therapy in 2014.

Tzianabos said that Chatterjee's AAVs has an efficiency rate that is clinically meaningful for patients and is less likely to cause an immune response, as their approach does not cut the DNA but instead causes natural occurring recombination mechanisms.

While no clinical trials exist yet to prove such claims, Homology maintains that their technology is still safer than existing forms of gene editing techniques, and financial backers are holding on to that promise.

"As a scientist and investor, I have evaluated many breakthrough technologies in this field and I believe Homology's technology platform is unique in its ability to leverage naturally occurring genetic mechanisms to both treat and correct diseases in vivo," said ARCH Venture Partners Managing Director and Homology Board Member Steven Gillis.

Photo: Andy Leppard | Flickr

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