Study of EBT-101 in Aviremic HIV-1 Infected Adults on Stable ART

Prior treatment

• Documented prior HIV-1 drug resistance to ≥2 or more classes of ART defined as single key mutations or an accumulation of minor mutations that result in resistance to entire respective drug classes within the past 5 years.
• History of \>1 change in ART due to virologic failure during preceding 2 years prior to screening.
• Received in the preceding 12 months or HCP plans to prescribe long-acting injectable ART.
• History of AIDS-defining opportunistic infection prior to screening.
• Receipt of any investigational HIV vaccine (prophylactic and/or therapeutic) within the year prior to screening.
• Receipt of any gene therapy product approved or experimental, at any time.
• Known positive SARS-CoV-2 test within 48 hours prior to planned dosing date.

Other illness

• History of HIV dementia.
• History of progressive multifocal leukoencephalopathy.
• History of significant cardiac disease in last 2 years.
• History of HIV-related kidney disease with abnormal renal function.
• Evidence of acute or chronic hepatitis B and/or hepatitis C.
• Known history of positive tuberculin skin test.

Lab values

• Known history and/or documented: pre-HIV treatment nadir CD4+ T cell count \<200 cells/mm3 or post-suppressive HIV treatment confirmed CD4+ T cell \<200 cells/mm3 prior to screening
• Predefined abnormal laboratory values within 42 days of dosing per protocol
• Anti-AAV9 serum neutralizing antibodies (Nabs) \>1:20 titer.

Other

• Known history or diagnosis of liver cirrhosis.
• Diagnosis of nonalcoholic fatty liver or advanced nonalcoholic steatohepatitis.

• Willing to enroll and sign the written informed consent for EBT-101-001 (current study) and EBT-101-002, the LTFU study.
• Age between 18 and 70 years (both inclusive).
• Weight: Cohort A ≥55 and ≤110 kg, Cohort B ≥55 and ≤90 kg.
• Cohorts A and B will only enroll male subjects (sex at birth).
• Chronic HIV-1 with known subtype B infection
• On stable regimen defined as continuous ART suppressive treatment with HIV RNA level below the level of quantitation for \>1 years prior to screening
• Plasma HIV-1 RNA levels below the limit of quantitation during screening.
• Peripheral blood CD4 T cell count ≥500 cells/mm3 for at least 2 years prior to screening
• Willing and able to comply, as assessed by the Investigator, with all study-related procedures.
• Have previously been vaccinated for N. meningitidis with documented history and/or received a N. meningitidis vaccination prior to dosing.
• Willing to stop ART if eligible for analytical treatment interruption.
• Willing to comply with the measures to prevent HIV transmission and reinfection required by the protocol.
• Must have received a COVID-19 vaccination series and boosters as specified by current federal (CDC) recommendations, with the last dose ≥30 days prior to dosing.