First genome editing treatment for HIV enters clinical trials

27 September 2021

A CRISPR-based therapy developed as a potential cure for HIV has been approved for human clinical trials by the US Food and Drug Administration (FDA).

Biotechnology company Excision BioTherapeutics Inc., based in San Francisco, has received clearance to initiate a Phase I/II clinical trial of their CRISPR-based treatment for HIV, EBT-101. The trial, commencing in early 2022, will assess the efficacy and safety of EBT-101 in three groups of healthy HIV patients, assessing its potential as a genome editing approach to treat HIV.

‘The clearance of our investigational new drug application for EBT-101 represents an important milestone for Excision and is the result of years of commitment to developing a functional cure for individuals living with HIV,’ said Daniel Dornbusch, chief executive officer of Excision. ‘Although antiviral treatments can manage HIV infection, they require life-long treatment, cause side effects, and do not provide the possibility of a functional cure.’

In contrast to current viral treatments, EBT-101 is a one-time treatment to effectively cure HIV infections, which alleviates the need for patients to take retroviral drugs to prevent HIV from developing into AIDS.

EBT-101 treatment is delivered via an adeno-associated virus, a harmless virus commonly used in gene therapy.  CRISPR/Cas9 targets and cuts out three sites within the HIV genome. Previous non-human primate trials have shown that EBT-101 reached every tissue where HIV reservoirs were present, and successfully removed the pro-viral genome sites.

‘EBT-101 has demonstrated removal of proviral DNA in multiple animal models and offers an opportunity for individuals living with HIV to potentially cease life-long therapies.’ explained Dr Lisa Danzig, Excision’s chief medical officer. ‘The Excision team looks forward to this important collaboration with our principal investigators, scientific advisors and regulators, to conduct a safe and informative trial with this first-in-class approach to a viral disease target previously considered to be incurable.’

The company is also working on using the approach for similar treatments for different viruses, such as herpes and hepatitis B.