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Start-Up Lands $10M for Gene-Editing HIV Treatments

HIV particles infecting human T cell

Scanning electron micrograph of HIV particles infecting a human T cell (NIH.gov)

6 September 2017. A new enterprise developing treatments using gene editing to eliminate HIV infections received $10 million in its seed funding round. Excision BioTherapeutics, a spin-off company from Temple University in Philadelphia, says its technology has the potential to cure people with HIV infections, not just manage the disease as done with current drugs.

According to Centers for Disease Control and Prevention, some 1.1 million people in the U.S. are living with HIV infections. While the number of new HIV cases are declining, more than 39,500 new cases were reported in 2015. Gay and bisexual men, particularly young African-American gay and bisexual men, are most affected. Globally, according to World Health Organization, some 37 million people were living with HIV in 2016, with 1.8 new cases reported. About 1 million people died of AIDS in 2016.

The three year-old Excision BioTherapeutics aims to apply gene editing techniques such as Crispr, or clustered regularly interspaced short palindromic repeats, to remove genes that support the stubborn HIV infections. Crispr is based on bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. The actual editing of genomes with Crispr employs enzymes that cleave DNA strands at the desired points, with Crispr-associated protein 9, or Cas9, being the enzyme used most often.

The company was co-founded by Temple University neuroscientist Kamel Khalili, who studies effects of HIV infections on nervous system functions and leads the university’s NeuroAIDS center. Another founder is Thomas Malcolm, who studied HIV infections and cancer genetics at Mount Sinai School of Medicine and Memorial Sloan Kettering Cancer Center in New York. Malcolm is Excision BioTherapeutics’s CEO, while Khalili is the company’s chief scientific advisor.

In a paper published in July 2017, Khalili outlined a strategy for harnessing gene-editing technologies such as Crispr to eliminate the HIV virus. He argues that HIV remains in a latent state in people with infections, protected by a support system in the body that can be traced to specific regions in the genome. Khalili believes gene editing can deactivate elements in DNA that maintain HIV receptors in a latent form and enable the re-emergence of active HIV infections. In May 2017, Khalili and colleagues reported on research with lab mice where Crispr/Cas9 editing removed genes supporting latent HIV, reducing expression of RNA that maintains the virus, as well as overall infection levels in the animals.

Khalili says the company’s first target is HIV infections, but that’s only the beginning. He notes in a company statement, “what we’re working on is not exclusively focused to the HIV/AIDS virus, but multiple viruses. Our platform will allow us to eradicate permanently the genetic elements of Herpes, Zika, Ebola, Hepatitis, West Nile, and many more viruses.”

The $10 million in seed funding for Excision BioTherapeutics is led by technology venture investor Artis Ventures in San Francisco. Other investors were not identified. Most of the funds are expected to finance early clinical trials of the company’s proposed HIV treatments.

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