๐Ÿฆ  HIV Therapeutic Vaccine Tracker - Functional Cure

Comprehensive tracking of HIV therapeutic vaccine development for people living with HIV, targeting functional cure (ART-free viral remission). HIV affects 39 million globally (1.2 million US), controlled by antiretroviral therapy (ART) but NOT cured - lifelong daily medication required. HIV establishes latent reservoirs in CD4+ T cells (primarily resting memory T cells) that persist despite ART, viral DNA integrated into host genome dormant but reactivation-competent. Stop ART โ†’ viral rebound within 2-4 weeks from reservoir reactivation. Therapeutic vaccines aim to: (1) "Kick" - reverse latency forcing virus expression, (2) "Kill" - boost HIV-specific immunity eliminating reactivated infected cells, achieving functional cure (virus remains but immune control prevents replication without ART). Leading strategies: Therapeutic vaccines + latency reversal agents (LRAs), broadly neutralizing antibodies (bnAbs), mosaic vaccines, dendritic cell vaccines. Only 2 proven cures: Timothy Ray Brown (Berlin Patient), Adam Castillejo (London Patient) - both via stem cell transplants from CCR5ฮ”32 donors (not scalable). Goal: Scalable functional cure 70%+ achieving ART-free remission years. Complementary to antiretroviral therapy and HIV management.

โš ๏ธ NO CURE AVAILABLE - LIFELONG ART REQUIRED:

HIV affects 39 million people globally (1.2 million US, 1.7 million children worldwide), 38 million on antiretroviral therapy (ART). Modern ART revolutionized HIV from death sentence to chronic manageable disease - life expectancy near-normal (70+ years), viral suppression achieves "undetectable = untransmittable" (U=U, cannot transmit sexually). However ART does NOT cure: (1) Lifelong daily medication required (1-3 pills/day, cost $20,000-40,000/year US, $100-300/year generic developing countries with subsidies), (2) Side effects - GI upset, fatigue, bone loss, cardiovascular risk, neuropsychiatric effects, kidney/liver toxicity (varies by regimen), (3) Adherence challenges - 95%+ adherence needed, missed doses โ†’ resistance, stigma/disclosure issues, (4) Drug-drug interactions complicating other medical care, (5) Long-term toxicities uncertain (decades of ART). Latent HIV reservoir problem: Virus integrated into host CD4+ T-cell DNA (primarily resting memory T cells, also tissue macrophages, microglia), estimated 1 million latently infected cells per person on suppressive ART, reservoir half-life 44 months (4+ years) - would take 70+ years to naturally decay even on perfect ART, cells long-lived memory T cells (decades lifespan). Stop ART โ†’ viral rebound 50% patients within 2 weeks, 90% within 4 weeks from reservoir reactivation (latent provirus transcribes โ†’ virion production โ†’ infects new cells โ†’ exponential replication). Only 2 HIV cures documented: Timothy Ray Brown ("Berlin Patient" 2008) and Adam Castillejo ("London Patient" 2019) - both underwent myeloablative chemotherapy + allogeneic stem cell transplant from CCR5ฮ”32/ฮ”32 homozygous donors for cancer treatment (leukemia), CCR5ฮ”32 mutation (1% European ancestry) makes cells resistant to R5-tropic HIV (CCR5 co-receptor absent), stem cells reconstituted HIV-resistant immune system, reservoir cleared (combination myeloablation killing infected cells + graft-vs-host eliminating residual reservoir), ART-free >15 years (Brown), >5 years (Castillejo), no viral rebound. Not scalable: Requires matching CCR5ฮ”32 donor (rare), dangerous procedure (30% mortality risk myeloablation + transplant), only justified for life-threatening cancer not HIV alone. Functional cure goal: Achieve sustained ART-free viral remission without stem cell transplant - virus remains latent but immune control prevents reactivation/replication, quality of life restoration, eliminate daily medication burden/costs/toxicities.

HIV Therapeutic Vaccines by Phase

39M
Living with HIV Globally
38M
On Antiretroviral Therapy
2
Documented Cures (Transplants)

๐Ÿ”ฌ Phase 2 Clinical Trials - Leading Strategies

"Kick and Kill" - Therapeutic Vaccine + LRA

Most promising functional cure strategy

Phase 2
Strategy Latency reversal + immune boost
Components Vaccine + LRA + bnAbs
Goal Functional cure (ART-free remission)
Leading Trials Multiple Phase 2
Concept - "Shock and Kill" / "Kick and Kill": Two-pronged approach: (1) KICK - Latency reversing agents (LRAs) reactivate latent HIV provirus forcing viral protein expression in infected resting CD4+ T cells ("shock" reservoir out of hiding), (2) KILL - Therapeutic vaccine boosts HIV-specific CD8+ cytotoxic T lymphocytes (CTLs) recognizing/killing reactivated infected cells before they produce infectious virus, broadly neutralizing antibodies (bnAbs) block cell-to-cell spread and enhance immune clearance via ADCC. Rationale: ART prevents new infection but doesn't touch latent reservoir (provirus transcriptionally silent, no viral proteins expressed, invisible to immune system). LRAs activate latent provirus โ†’ viral RNA/protein production โ†’ infected cell displays HIV antigens on MHC-I โ†’ CTLs recognize and lyse cell โ†’ reservoir depletion. Problem: Natural HIV-specific immunity insufficient (exhausted T cells from chronic infection, narrow epitope recognition, immune escape mutations), hence need therapeutic vaccine to broaden/boost HIV-specific responses.
Latency Reversing Agents (LRAs): Multiple classes: (1) HDAC inhibitors (vorinostat, panobinostat, romidepsin) - most studied, inhibit histone deacetylases allowing chromatin opening โ†’ HIV LTR transcription, Phase 1/2 showed increased cell-associated HIV RNA (proof latency reversal) but minimal reservoir reduction (kill component insufficient); (2) PKC agonists (bryostatin-1, ingenol) - activate protein kinase C โ†’ NF-ฮบB โ†’ HIV transcription, potent latency reversal but toxic at high doses; (3) TLR agonists (TLR7/9 agonists - vesatolimod, lefitolimod) - activate innate immunity and HIV transcription, dual benefit: reverse latency + adjuvant effect boosting immune responses; (4) BET inhibitors - block BRD4 protein (silences HIV LTR), paradoxically can reverse latency at certain doses. Challenges: LRAs alone insufficient (reactivate virus but don't eliminate cells - need immune clearance), toxicity (systemic immune activation), incomplete latency reversal (reactivate only fraction of reservoir 5-20%), variable patient response.
Therapeutic Vaccines in Kick-and-Kill: Multiple vaccine platforms combined with LRAs in trials: (1) Dendritic cell vaccines - autologous DCs loaded with HIV antigens (peptides, inactivated virus, mRNA) reinfused IV, powerful T-cell primers, AGS-004 (personalized DC vaccine) + vorinostat Phase 2 showed modest reservoir reduction 20-30%, some patients delayed viral rebound 12+ weeks off ART (not functional cure but encouraging); (2) DNA vaccines (PENNVAX, HVTN DNA vaccines) + LRA, induce broad T-cell responses, limited immunogenicity alone but combination with LRA + bnAbs synergistic; (3) Viral vector vaccines (MVA, adenovirus vectors expressing HIV antigens) + LRA, potent CTL induction, combinations in Phase 2; (4) mRNA vaccines - next-generation, encoding HIV Env/Gag/Pol, combined with LRA + bnAbs in preclinical/Phase 1. Key insight: Vaccines need to induce high-frequency broadly-reactive CTLs targeting conserved HIV epitopes (avoid immune escape), central memory T cells (long-lived responses), polyfunctional T cells (IFN-ฮณ + TNF-ฮฑ + IL-2 production).
Broadly Neutralizing Antibodies (bnAbs): Revolutionary addition to kick-and-kill: Monoclonal antibodies isolated from rare HIV+ individuals ("elite neutralizers") who naturally developed antibodies neutralizing diverse HIV strains. bnAbs target conserved regions of HIV Envelope (gp120/gp41): CD4-binding site (VRC01, 3BNC117, N6), V3-glycan supersite (10-1074, PGT121), V1V2 apex (PG9/PG16), gp41 MFPR (10E8). Mechanisms: (1) Neutralization - block virus entry into cells, (2) ADCC - antibody-dependent cellular cytotoxicity (NK cells kill antibody-coated infected cells), (3) Complement-dependent cytotoxicity. Clinical trials combining bnAbs + LRA ยฑ therapeutic vaccine: 3BNC117 + 10-1074 (dual bnAb) alone delayed viral rebound median 6 weeks post-ART vs. 2 weeks placebo (not cure but proof-of-concept), addition of LRA (romidepsin or TLR7 agonist) + bnAbs showed greater reservoir reduction 40-60% in some studies, triple combination (bnAbs + LRA + therapeutic vaccine) most promising - limited data but some patients achieving 24+ weeks ART-free remission in small trials.
Leading Trials: Fred Hutch / defeatHIV (Seattle) - AGS-004 DC vaccine + vorinostat + analytical treatment interruption (ATI), NIH/NIAID - 3BNC117 + 10-1074 + romidepsin trials, IciStem Consortium (Europe) - combining LRAs + bnAbs + therapeutic vaccines multiple platforms
Mosaic Vaccines - Broad Coverage

Multi-clade coverage targeting global diversity

Phase 2
Developer Janssen/J&J + NIAID
Technology Ad26 vector + gp140 protein
Unique Feature Mosaic antigens (multiple clades)
Details: Originally developed as preventive vaccine but therapeutic potential emerging. Mosaic antigens computationally designed to maximize coverage of global HIV diversity - single antigen contains epitopes from multiple clades (A, B, C, D, etc.), overcomes HIV variability problem. Ad26 (adenovirus serotype 26) vector prime + gp140 protein boost regimen. Therapeutic use: Study of HIV+ individuals on ART receiving mosaic vaccine showed: Enhanced HIV-specific T-cell breadth/magnitude, some delayed viral rebound upon treatment interruption (median 5 weeks vs. 2 weeks historical controls), limited data but Phase 2 therapeutic trials ongoing combining with bnAbs.
Additional Phase 2 Approaches (2)

Novel therapeutic strategies

Phase 2
Candidates: (1) Tat/Rev therapeutic vaccines - targeting HIV regulatory proteins (Tat, Rev) essential for viral replication, Italian HIV-TAT vaccine (protein subunit + alum) Phase 2 showed improved immune parameters, reduced proviral DNA 40% subset patients, combination with ART interruption ongoing; (2) DNA + protein + MVA prime-boost regimens - HVTN trials using DNA prime (PENNVAX) โ†’ protein boost (rgp120) โ†’ MVA boost, maximizes immunogenicity, combined with LRA/bnAbs in therapeutic setting Phase 2.

๐Ÿงช Phase 1 & Preclinical Development

Phase 1 Candidates (5)

Early clinical testing

Phase 1
Approaches: (1) mRNA therapeutic vaccines - encoding HIV Env trimer/Gag/Pol, lipid nanoparticle delivery, leveraging COVID-19 mRNA platform success, potent T-cell/antibody responses preclinical, Phase 1 HIV+ patients starting; (2) Nanoparticle vaccines - HIV antigens on VLPs, self-adjuvanting, enhanced DC uptake; (3) CAR-T cells - not traditional vaccine but immunotherapy, CD4+ CAR-T cells engineered to resist HIV infection + target HIV-infected cells, dual HIV-specific CAR + CCR5 knockout; (4) Therapeutic DNA vaccines + electroporation - improves DNA vaccine immunogenicity 100-fold, multiple HIV antigens, prime for bnAb/LRA combinations; (5) T-cell vaccines - focus on inducing tissue-resident memory T cells (TRM) in lymphoid tissues where reservoir resides, IN injection strategies.
Preclinical Platforms (6)

Next-generation technologies

Preclinical
Technologies: (1) Gene therapy approaches - AAV vectors delivering bnAb genes (vectored immunoprophylaxis VIP), single injection โ†’ lifelong bnAb production bypassing need for infusions, AAV-bnAb constructs in primate trials showing protection; (2) CRISPR excision - gene editing to excise integrated HIV proviral DNA from infected cells, challenging (need delivery to all infected cells), proof-of-concept mice/primates, EBT-101 (Excision BioTherapeutics) using nucleases targeting HIV LTRs in Phase 1/2; (3) Zinc finger nucleases (ZFNs) - disrupting CCR5 gene in patient's own T cells making them HIV-resistant, SB-728-T showed safety, reservoir reduction in subset; (4) Combination immunotherapies - bnAbs + therapeutic vaccine + checkpoint inhibitors (PD-1, CTLA-4 blockade - reverse T-cell exhaustion enhancing anti-HIV responses), dual HIV cure + cancer therapy potential; (5) Replication-competent vectors - CMV vector vaccines (continuous antigen presentation), prime/maintain high-frequency HIV-specific T cells, primate studies show protection; (6) Microbiome manipulation - gut/mucosal microbiome modulation reducing inflammation, reconstituting gut-associated lymphoid tissue (GALT) - major reservoir site.
Key Research Centers: NIH/NIAID, Fred Hutchinson Cancer Center (defeatHIV), Scripps Research, UCSF (delaney-aids.ucsf.edu), University of Pennsylvania (Gene Therapy Program)

๐Ÿ“Š HIV Latency - The Barrier to Cure

Understanding HIV Latency

What is Latent HIV Reservoir? HIV integrates into host CD4+ T-cell chromosomal DNA as provirus. In activated T cells (during acute infection), provirus transcribed โ†’ viral proteins โ†’ virion assembly โ†’ infection spreads. However subset of infected cells transition to resting memory state BEFORE producing virus - provirus remains integrated but transcriptionally silent (no viral RNA/protein production). These latently infected resting memory CD4+ T cells: Invisible to immune system (no viral antigens expressed), not killed by ART (ART blocks new infection and viral replication, doesn't touch integrated proviral DNA), long-lived (memory T cells decades lifespan), reactivation-competent (if cell activated โ†’ provirus transcribes โ†’ virus production โ†’ reinfection cycle resumes). Anatomical sites: Blood (10-20%), lymph nodes (40-60%, major reservoir), gut-associated lymphoid tissue GALT (20-30%), CNS (microglia harbor virus - sanctuary site), genital tract, bone marrow. Cell types: Primarily resting memory CD4+ T cells (central memory TCM, transitional memory TTM), also tissue-resident memory, macrophages (especially CNS microglia - different reservoir characteristics).

Reservoir Size & Dynamics: On suppressive ART, estimated 1 million latently infected cells per person (range 10,000 to 10 million - high variability). Only 1-10% reservoir is replication-competent (produces infectious virus if reactivated), rest defective proviruses (deletions, mutations) but contribute to immune activation. Reservoir half-life 44 months (3.7 years) - extraordinarily stable, would take 70+ years of perfect ART to decay to zero naturally (not feasible). Drivers of reservoir stability: Memory T-cell longevity (decades), homeostatic proliferation (cells divide maintaining pool size - clonal expansion, TCR-driven proliferation in response to antigens), integration sites (proviruses in actively transcribed genes may drive proliferation). Challenge: Even if 99.9% reservoir eliminated, remaining 0.1% (1,000 cells) sufficient for viral rebound - need near-complete clearance for functional cure.

Current ART - Limitations Driving Cure Research

Modern ART Regimens: Highly effective - single tablet once-daily regimens (bictegravir/TAF/FTC, dolutegravir/3TC), viral suppression <50 copies/mL achievable >95% adherent patients within 6 months, U=U (Undetectable = Untransmittable) - suppressed viral load eliminates sexual transmission risk (revolutionary for prevention), life expectancy HIV+ person on ART approaches HIV-negative with early treatment. Classes: NRTIs (nucleoside reverse transcriptase inhibitors - backbone), NNRTIs (non-nucleoside RTIs), PIs (protease inhibitors), INSTIs (integrase strand transfer inhibitors - newest, most potent/tolerable), entry inhibitors (rare - maraviroc CCR5 antagonist).

Why ART Not Cure - Limitations: (1) Lifelong medication - daily pills forever, adherence burden (travel, disclosure, routine), must be taken 95%+ time (missing doses โ†’ resistance risk); (2) Cost - $20,000-40,000/year US without insurance (generic $100-300/year developing countries with subsidies but access barriers), insurance coverage issues; (3) Side effects - vary by regimen: GI upset common (diarrhea, nausea especially PIs), CNS effects (dolutegravir - insomnia, depression, anxiety 10-20%), bone density loss (TDF-based regimens), cardiovascular disease (chronic inflammation despite suppression, some ART contribution), kidney/liver toxicity, weight gain (INSTIs especially - 5-10 lbs average, metabolic effects), lipodystrophy (fat redistribution - older regimens); (4) Drug interactions - many ART drugs CYP450 interactions complicating other medications (statins, antifungals, PPIs, rifampin TB treatment); (5) Resistance - if adherence poor โ†’ viral replication with drug pressure โ†’ resistance mutations, limits future options (though INSTIs high barrier to resistance); (6) Inflammation - chronic immune activation persists despite viral suppression (microbial translocation, persistent low-level viral replication sanctuary sites?), drives comorbidities (cardiovascular disease, cancer, neurocognitive decline); (7) Stigma - lifelong HIV diagnosis, disclosure challenges (dating, employment), internalized stigma mental health; (8) Long-term unknowns - people now living decades on ART, very long-term toxicities (40-50+ years) unknown, aging with HIV accelerated (cardiovascular events, cancers 10-15 years earlier than HIV-negative).

Functional Cure - Definition & Goals

Functional Cure vs. Sterilizing Cure: Sterilizing cure - complete elimination of all HIV DNA from body, no virus remains, theoretically impossible (need eradicate every single infected cell - millions cells scattered throughout body including CNS). Functional cure (remission) - HIV DNA remains latent but immune system controls virus preventing reactivation/replication without ART, no viral load rebound off ART, no disease progression, no transmission, similar to herpesviruses (HSV, CMV, EBV - latent but immune-controlled). Functional cure acceptable goal - achieves all clinical benefits (ART-free, healthy, non-transmissible) without requiring impossible sterilizing cure.

Success Criteria: Consensus definition emerging: (1) Sustained viral suppression (<50 copies/mL) off ART for โ‰ฅ12 months (some propose 24+ months), (2) No AIDS-defining illness, (3) Stable/rising CD4+ count, (4) No HIV-specific treatment required. Measured by: Analytical treatment interruption (ATI) - structured ART cessation under close monitoring (viral load every 1-2 weeks, restart ART if rebound >1,000-10,000 copies/mL or CD4 decline), time to viral rebound, magnitude of rebound, reservoir size reduction (cell-associated HIV DNA/RNA).

Post-Treatment Controllers: Rare individuals (<5% HIV+ stopping ART) who naturally control HIV replication without treatment - viral load remains undetectable/very low off ART for years. Not truly "cured" (reservoir persists, can rebound) but functional cure achieved. Mechanisms: Robust HIV-specific T-cell responses, bnAb development, reservoir restricted to defective proviruses, HLA genetics (protective alleles B*27, B*57), low reservoir size at ART initiation (early treatment). Study: French ANRS VISCONTI cohort - 14 patients treated very early acute infection (weeks post-infection), stopped ART after 3 years median, 70% maintained virologic control >5 years off ART, mechanisms: Early ART limited reservoir seeding (<100 infected cells vs. 1 million typical), preserved HIV-specific immunity. Lesson: Early ART initiation (within weeks-months infection) dramatically increases functional cure odds - drives "test and treat" urgency.

Challenges & Future of HIV Cure

Challenges: Reservoir persistence - most stable latent reservoir of any chronic infection. Anatomical sanctuaries (CNS, genital tract - limited ART/immune penetration). Reservoir diversity - millions proviruses, extensive mutations, immune escape variants. Immune exhaustion - decades HIV infection exhausts HIV-specific T cells (PD-1, CTLA-4, LAG-3 upregulation โ†’ anergy). Balancing act - LRAs induce immune activation (inflammation, cytokine release) risky especially in older individuals. Individual variability - reservoir size varies 10,000-fold, HLA genetics, treatment history, co-infections. Safety paramount - any cure strategy must be safer than current ART (ART low toxicity modern regimens, very effective). Scalability - cure must be accessible globally (not stem cell transplant $1M+ and risky). Ethics of ATI - treatment interruptions risky (rebound can be rapid, high viral load, transmission risk, CD4 decline), require close monitoring, informed consent.

Near-Term (2025-2030): Optimization of kick-and-kill combinations - identify best LRA + bnAb + therapeutic vaccine combinations, Phase 2b/3 trials enrolling hundreds patients. First functional cure demonstrated in larger cohort (current trials 10-30 patients, need 100+). mRNA therapeutic vaccines enter Phase 2 combined with LRAs/bnAbs. Long-acting bnAbs (every 6 months injection) replace monthly infusions improving feasibility. Gene therapy approaches (AAV-bnAbs, CRISPR excision) Phase 2 trials. Biomarkers predicting cure success identified (reservoir size, immune parameters) allowing enrichment strategies.

Mid-Term (2030-2040): First therapeutic vaccine or combination therapy approved achieving functional cure 30-50% patients (ART-free remission 12+ months). Functional cure becomes standard treatment option (not universal but available). Combination: therapeutic vaccine + long-acting bnAb + improved LRA + checkpoint inhibitor = 50-70% functional cure rate. Preventive functional cure - vaccinate acute HIV infection patients (before reservoir established) โ†’ 70-80% never need ART. Gene editing (CCR5 knockout, HIV excision) safe/effective 50%+. Post-treatment controllers increase from <5% to 30-50% with interventions.

Long-Term Vision (2040+): Functional cure achievable 70-80% HIV+ patients stopping ART, durable remission (5+ years) majority, rare viral rebound managed with short ART courses re-suppressing. "Cure" redefined - not elimination (sterile cure) but sustained remission (functional cure) acceptable goal achieved. Sterilizing cure small percentage (~10%) via intensive combinations (multiple bnAbs, multiple LRAs, therapeutic vaccine, gene therapy). Prevention + early treatment near-eliminate HIV - test and treat at acute infection, pre-exposure prophylaxis (PrEP) widespread, new infections <10,000 globally/year (vs. 1.5 million current). AIDS-free generation realized - HIV becomes rare, managed infection, stigma eliminated. Lifelong ART rarity not norm - most achieving functional cure. Vision: HIV transitions from chronic lifelong disease requiring daily medication to acute infection treated acutely (weeks-months early ART) followed by immune control (no ongoing treatment) similar to hepatitis C cure with DAAs.