The US Food and Drug Administration (FDA) has given the nod for Excision BioTherapeutics to begin trials testing CRISPR gene editing as a treatment for HIV.

EBT-101 will be a first-in-human, CRISPR-based one-time gene therapy to be evaluated in individuals with HIV.

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On 15 September Excision announced that the FDA had accepted its Investigational New Drug (IND) application for EBT-101 as a potential functional cure for chronic HIV.

The IND clearance will allow the firm to start a first-in-human Phase I/II trial to evaluate the safety, tolerability and efficacy of EBT-101 in healthy individuals living with HIV.

“The clearance of our IND 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 Excision CEO Daniel Dornbusch.

“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. We are grateful for the FDA’s engaged review and acceptance of the IND for EBT-101 and look forward to initiating the Phase I/II clinical trial later this year.”

EBT-101 uses CRISPR to cut out or excise HIV that has wrapped around the DNA in cells. It has been HIV’s ability to coil itself into DNA that has made it so difficult to treat and largely the reason that past curative efforts have fallen flat.

The therapy harnesses an adeno-associated virus (AAV) at a relatively low dose to deliver the one-time treatment.

Excision said that the investigational programme employs CRISPR-Cas9 and two guide RNAs that target three sites within the HIV genome, “thereby excising large portions of the HIV genome and minimizing potential viral escape”.

The upcoming trial will be one of the first attempts to directly extract the latent virus from DNA.

In preclinical studies, the therapy demonstrated the ability to excise HIV proviral DNA in human primary cells as well as multiple animal models including non-human primates.

In later studies, after evaluating the safety and tolerability of the therapy, Excision hopes to take patients off their regular antiviral meds to test EBT-101 as a cure.

“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,” said Excision’s CMO Lisa Danzig.

“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 news of the trial comes just weeks after it was announced that J&J’s recent efforts at an HIV vaccine had failed and news broke that Moderna is set to begin trials of its HIV vaccine based on mRNA technology.

HIV can now be well managed with antiretroviral medications, but these are a lifelong commitment and cause side effects. For the last few decades, an elusive HIV vaccine has been considered ‘the holy grail’ in stopping the spread of the highly stigmatised disease, but CRISPR-based gene editing would represent a brand-new therapeutic approach.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

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