The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy.
“Having spoken to some clinicians, they realized that only a handful of people in the world have been cured of HIV ever, so they understand the transformative nature of the therapy. And we believe that the trial will provide important information on the path to a potential functional cure for people living with HIV and meets an area of high unmet medical need. It’s really exciting that we have this groundbreaking therapy for infectious diseases. This is the first time it's been administered to a participant and I think it is really exciting to be moving forward."
Excision BioTherapeutics has dosed the first patient in the phase 1/2 clinical trial (NCT05144386) of EBT-101, an in vivo CRISPR-based gene editing approach intended for the treatment of human immunodeficiency virus type 1 (HIV-1). The patient was dosed in July of this year and has tolerated the therapy well since then. Excision expects that the patient will qualify for analytical treatment interruption of background anti-retroviral therapy.
EBT-101 delivers CRISPR-Cas9 and dual guide RNAs via an adeno-associated virus (AAV), targeting 3 sites in the HIV genome for excision, with the intention of minimizing viral escape.
CGTLive spoke with TJ Cradick, chief scientific officer, Excision BioTherapeutics, to learn more about the trial and its goals. He discussed the potential of a 1-time infusion to change the HIV treatment landscape.