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April 26, 2021

Curing the Uncurable Through Gene Editing

The COVID-19 pandemic has reawakened the world to the unique and ever-present dangers of novel viruses and the pandemics they cause. Novel viral pandemics have the potential not only to kill millions, but also to paralyze society and the normal social interactions we take for granted.

Forty years ago, in 1981, a novel viral illness swept through the world – killing millions of people in gruesome fashion. In 1983, scientists identified and isolated the virus we now know of as HIV for the first time. Since 1983, more 33 million people have died from this disease and more than 38 million people worldwide live with HIV today. The news is not all doom and gloom; however. Powered by science, ingenuity, and a ruthless desire to improve patients’ lives, scientists and biopharmaceutical companies have developed a better understanding of HIV and also an armamentarium of treatments for HIV that enable HIV+ individuals to live lives equal in lifespan to HIV- people.

We still lack a cure for HIV; however, and while there have been case studies of individuals cured of HIV, most people require lifelong treatment to keep the virus at bay.

Excision and the Excision Platform:

In February of this year, we joined an investment in Excision BioTherapeutics, a biotechnology company committed to curing HIV and other chronic viral diseases including: Hepatitis B, Herpes Simplex Virus, JC Virus, and more. 

The Excision platform leverages CRISPR (clustered regularly interspaced short palindromic repeats), Cas9/X/Y, and viral vectors to develop therapies that are able to directly attack viral DNA and provide lasting cures to patients. Not unlike a pair of “molecular scissors” the Company aims to “cut” a virus (and only virus) at precise locations. In the case of viruses like HIV, Excision’s drugs remove the virus from the host genome enabling durable cures. For viruses like HBV and HSV, Excision’s drugs attack “hard to reach” reservoirs of virus inside host cells.

The Company is not yet in the clinic, but will be soon. However, it has demonstrated numerous scientific achievements on the way there. In July 2019, in Nature Communications, the Company and its collaborators published the ability of its product to clear HIV-1 reservoirs from the genomes of HIV-1 infected humanized mice – the first ever time this has been demonstrated in animals. In November 2020, also in Nature Communications, the Company and its collaborators demonstrated the ability to edit SIV (a virus closely related to HIV) from the genomes of non-human primates. Some of the Company’s collaborators and advisors have also demonstrated similar data in mouse models of Herpes virus infection.

A World Class Team and Scientific Heritage:

The Excision team’s work stands on the back of progress made by an uncountable number of dedicated scientists. It has scientific roots and beginnings in the research of Kamel Khalili, PhD, Laura H. Carnell Professor and Chair of the Department of Neuroscience, Director of the Center for Neurovirology, and Director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine at Temple University (LKSOM). An incredible profile of Kamel’s journey with this technology can be found here. The Company has also licensed the discoveries of and works with Nobel-laureate and CRISPR-discoverer Jennifer Doudna, PhD (who shares these honorees with Emmanuelle Charpentier, PhD) and is working with some of her new Cas nuclease editors (X,Y and others) in the field of infectious disease.

As a Company, Excision is helmed by Daniel Dornbusch (CEO) and TJ Cradick, PhD (CSO). Daniel is an accomplished biotech executive with experience building and scaling companies from idea through to clinical product. TJ is the former Head of Genome Editing at CRISPR Therapeutics and a pioneer in developing genome editing science for more than 16 years. As the 2nd US CRISPR Therapeutics employee, TJ stewarded CRISPR’s pipeline from scientific idea to a clinical stage.

We’re thrilled to announce our participation in Excision’s $60M Series A financing alongside: Great Point Ventures, Artis Ventures, Adjuvant Capital, Anzu Partners, Cota Capital, WRVI Capital, IndusAge Partners, Loreda Holdings, and Olive Tree Capital.

We could not be more optimistic about the potential for Excision to usher in a new era of cures for patients with uncurable viral diseases. The platform will not only change the lives of patients in developed countries, but also those in developing countries as well – who rarely see access to such novel therapies. The potential for this platform is nearly limitless, 8 percent of human DNA consists of remnants of ancient viruses and these genes have been implicated in a variety of disease pathologies. We could not be more excited for the entire Excision team and cannot wait to see what the future holds!