Immune checkpointImmuno-oncologyUniProt P16410

CTLA-4 antibodies discovery

CTLA-4 is a validated immune checkpoint. The challenge in 2026 is no longer validation — it’s designing differentiated anti-CTLA-4 antibodies with a clear edge on efficacy, tolerance and IP. MAbSilico engineers next-generation CTLA-4 candidates from 1,147 annotated antibodies, 450+ affinity measurements and 22 INNs.

Target card · CTLA-4

UniProt IDP16410
Molecular weight41–43 kDa
StructureHomodimer · IgV domain
ExpressionActivated T cells · Tregs
LigandsCD80 · CD86
FunctionInhibitory receptor
Therapeutic areaImmuno-oncology
Approved drugsIpilimumab · Tremelimumab

On this page

  1. CTLA-4 biology
  2. Why target CTLA-4
  3. Approved indications
  4. Challenges
  5. 1st-gen antibodies
  6. Next-gen antibodies
  7. MAbSilico platform
  8. Workflow
  9. Talk to us

In 2026, how do you design anti-CTLA-4 antibodies that stand out on efficacy, tolerance and IP? Monoclonal, bispecific or innovative formats — which one positions a candidate against first-generation drugs and the rising wave of next-gen molecules?

1. What is CTLA-4 and its role in immune response?

CTLA-4 (cytotoxic T-lymphocyte-associated protein 4) is an inhibitory receptor expressed on activated T cells and constitutively on regulatory T cells (Tregs). It competes with CD28 for binding CD80 and CD86, acting as a key negative regulator of T-cell activation.

Dual inhibition mechanism

  • Intrinsic: phosphatase recruitment dampening TCR/PI3K signaling in T cells.
  • Extrinsic: trans-endocytosis of CD80/CD86 from antigen-presenting cells.
Structure. Type I transmembrane glycoprotein · 41–43 kDa homodimer · IgV extracellular domain recognizing CD80/CD86 · C-terminal domain recruiting phosphatases.

2. Why target CTLA-4 in therapy?

Blocking CTLA-4 restores T-cell activation in lymph nodes and reduces Treg suppression in the tumor microenvironment. As the first immune checkpoint to be therapeutically validated (ipilimumab, 2011), CTLA-4 blockade remains a cornerstone of combination immunotherapy strategies.

3. Approved indications & combinations

Ipilimumab (Yervoy, BMS) received FDA approval in 2011 for unresectable or metastatic melanoma, expanding to multiple solid tumors in combination with nivolumab. Tremelimumab (Imjudo, AstraZeneca) received approval in 2022 for hepatocellular carcinoma in the STRIDE regimen.

4. Challenges of anti-CTLA-4 projects

First-generation CTLA-4 antibodies carry dose-limiting toxicity from systemic immune activation. The key engineering challenges are: (1) improved Fc-mediated Treg depletion at the tumor site, (2) selective activity to reduce irAEs, and (3) differentiated epitope engagement beyond the ipilimumab footprint.

5. Panorama of anti-CTLA-4 antibodies

AntibodyIsotypeMechanismIndication
IpilimumabHuman IgG1CTLA-4/B7 blockade · Treg depletion via ADCC/ADCPMelanoma + anti-PD-1
TremelimumabHuman IgG2Lower Fc effector functionHCC (STRIDE)

7. The MAbSilico platform for CTLA-4

MAbSilico’s computational platform processes the entire annotated CTLA-4 antibody landscape — 1,147 antibodies, 450+ affinity measurements, 22 INNs — to identify differentiated binding modes, model Fc effector profiles and generate optimized candidate sequences ready for CRO transfer in 21 days.

Competitive landscape

  • Ipilimumab (BMS)
  • Tremelimumab (AstraZeneca)
  • Zalifrelimab (Agenus)
  • BMS-986218 (BMS)
  • MK-1308 (Merck)

Related targets

  • PD-1 / PD-L1
  • LAG-3
  • TIGIT
  • TIM-3
  • VISTA

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CTLA-4 in MAbSilico’s database

The deepest annotated anti-CTLA-4 antibody dataset, engineered for computational design.

1,147
Anti-CTLA-4 antibodies
450+
Affinity measurements
22
INNs catalogued
70+
Curated publications
3D
Crystal structure available

How MAbSilico engineers CTLA-4 candidates

Our in silico workflow applied to CTLA-4, from epitope strategy to ready-to-clone sequences.

01
Target characterization
Full structural analysis of CTLA-4 · epitope landscape · IgV domain binding regions · IP freedom-to-operate assessment.
02
Epitope & format strategy
mAb vs bispecific vs nanobody · Fc engineering options (IgG1/IgG4/LALA) · Treg depletion vs pure blockade strategy.
03
Binding region selection
Identification of CD80/CD86 binding interface residues · non-overlapping epitopes vs ipilimumab.
04
Candidate identification
AI screen of 1,147 annotated antibodies + in silico generation of novel VH/VL sequences.
05
Multiparametric optimization
Affinity · developability · Fc effector tuning · pH stability · bio-better profiling · PI-friendly design.
06
Selection & transfer
Top candidate sequences in standard format · ready-to-clone for your CRO or internal team · full computational report.

CTLA-4 project scenarios

How clients engage MAbSilico for their anti-CTLA-4 programs.

De novo design
Next-generation CTLA-4 mAb
Design a differentiated IgG1 CTLA-4 antibody with enhanced Treg depletion and improved tolerability vs ipilimumab.
Optimization
Bio-better program
Improve affinity and developability of an existing anti-CTLA-4 scaffold with multiparameter in silico optimization.
Bispecific
CTLA-4 × PD-1 bispecific
Engineer a dual checkpoint inhibitor combining CTLA-4 and PD-1 blockade in a single molecule.
Feasibility
IP landscape analysis
Map the CTLA-4 antibody IP landscape and identify freedom-to-operate windows for your novel candidate.

Ready to start your CTLA-4 program?

Get a feasibility note on your anti-CTLA-4 project within 48h. Phase-I-ready candidates in 21 days.

Clinical Evidence

CheckMate trials & pivotal data

Ipilimumab's approvals rest on a landmark series of randomized trials that established immune checkpoint blockade as a curative-intent strategy in melanoma and beyond.

Phase III • FDA Approval
MDX010-20 (CA184-002)
Ipilimumab vs. gp100 peptide vaccine in previously treated melanoma. First RCT to show OS benefit in advanced melanoma (HR=0.66). Led to FDA approval in 2011.
10.1 mo
Median OS (ipi)
6.4 mo
Median OS (ctrl)
0.66
Hazard Ratio
Phase III • NSCLC
CheckMate 227
Nivolumab + ipilimumab in first-line NSCLC (high TMB ≥10 mut/Mb). Dual PD-1/CTLA-4 blockade broadened into thoracic oncology.
17.1 mo
Median OS (combo)
14.9 mo
Median OS (chemo)
FDA 2020
Approval
Phase III • STRIDE
HIMALAYA (tremelimumab)
Single-dose tremelimumab 300 mg (STRIDE) + durvalumab vs. sorafenib in uHCC. First CTLA-4 + PD-L1 approval in liver cancer (2022).
16.4 mo
Median OS (STRIDE)
13.8 mo
Median OS (ctrl)
0.78
Hazard Ratio
⚠️ irAE mechanism — endosomal CTLA-4 & LRBA
Grade 3/4 immune-related adverse events (irAEs) occur in ~20-30% of patients on ipilimumab monotherapy and >50% on combination therapy. The extrinsic mechanism underlies toxicity: in normal tissues, Treg-mediated transendocytosis of B7 ligands dampens peripheral T cells. Antibody blockade removes this brake. Critical insight: LRBA (LPS-responsive beige-like anchor protein) recycles CTLA-4 into endosomes after internalization — patients with LRBA deficiency phenocopy ipilimumab toxicity, confirming the endosomal trafficking pathway as a key determinant of irAE severity. Next-gen pH-sensitive antibodies exploit this biology to spare normal tissue.
Ipi + Nivo
Ipilimumab 3 mg/kg + Nivolumab 1 mg/kg Q3W × 4 → Nivo 3 mg/kg Q2W
STRIDE
Single-dose Tremelimumab 300 mg + Durvalumab 1500 mg Q4W × 1 → Durvalumab
Low-dose Ipi
Ipilimumab 1 mg/kg (reduced dose) + Nivolumab to improve tolerability vs. 3 mg/kg
Next-Generation Engineering

Beyond ipilimumab: rational design to widen the therapeutic window

The clinical success of CTLA-4 blockade is limited by dose-limiting toxicities. Next-generation antibodies use structure-guided engineering to dissociate anti-tumor efficacy from systemic irAEs.

🔔
pH-selective mAb
Gotistobart (BNT316 / ONC-392)
pH-sensitive IgG1 engineered to bind CTLA-4 tightly in the acidic tumor microenvironment (pH 6.0-6.5) but release in normal tissues (pH 7.4). Exploits endosomal CTLA-4 trafficking via LRBA. Avoids Treg depletion in peripheral tissues. Phase II/III ongoing in NSCLC + melanoma.
Phase II/III
🍷
Probody / Masking
Probody anti-CTLA-4
CTLA-4-targeting probodies use peptide masks that block the binding site and are removed by tumor-associated proteases (MMP-2/9, uPA) only within the TME. CytomX approach: systemic inactivation until tumor-selective protease activation reduces peripheral irAE burden.
Preclinical / Phase I
🌪
Bispecific
CTLA-4 × LAG-3 / TIM-3 bispecifics
Dual-checkpoint bispecifics target CTLA-4 in combination with LAG-3 or TIM-3, enabling simultaneous blockade of two non-redundant inhibitory axes with a single molecule. Potential to replace doublet-mAb combinations and reduce toxicity via tumor co-localization.
Emerging
💊
ADC approach
Anti-CTLA-4 ADC (Treg depletion)
Antibody-drug conjugates targeting CTLA-4 leverage CTLA-4 over-expression on intratumoral Tregs vs. peripheral Tregs. Selective Treg depletion within tumor without systemic immunosuppression. Payloads: MMAE, DXd. Offers a cytotoxic mechanism independent of PD-1 blockade.
Preclinical
🧬
IgG1 ADCC / ADCP
Treg-depleting IgG1 variants
IgG1 Fc-enhanced variants (GASDALIE, SELF) with improved FcγRIIIA affinity for superior ADCC-mediated intratumoral Treg depletion. Distinct MoA from ipilimumab IgG1: the effector-cell killing of CTLA-4+ Tregs drives tumor control in heavily Treg-infiltrated tumors (RCC, ovarian).
Phase I/II
😅
Half-life extended
YTE / Half-life engineering
FcRn-mediated half-life extension via M252Y/S254T/T256E (YTE) or LS mutations applied to CTLA-4 antibodies to enable less frequent dosing (Q8W or Q12W). Particularly relevant for combination regimens where ipilimumab Q6W is already used in CheckMate 9ER (RCC).
Clinical use

How pH-selective CTLA-4 blockade works

At tumor pH (6.0–6.5), histidine residues in the antibody CDR loops become protonated, stabilizing a high-affinity conformation that tightly engages CTLA-4 on intratumoral T cells and Tregs. At systemic pH (7.4), the same histidines deprotonate, reducing affinity ~100-1000× and sparing peripheral CTLA-4+ cells. This mechanism—pioneered in gotistobart—produces tumor-selective checkpoint blockade with dramatically lower irAE rates in early-phase data.

6.0–6.5
Tumor pH range
7.4
Normal tissue pH
~100×
Affinity difference
His-swap
CDR engineering