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Targeted Therapy

Targeted therapies are drugs designed to switch off a specific broken protein that a cancer depends on.

Small-molecule kinase inhibitors, monoclonal antibodies, hormone-pathway agents, PARP inhibitors, degraders (PROTACs, molecular glues), and synthetic-lethality approaches. Matched to a molecular alteration by a companion diagnostic. Resistance is the central problem; next-generation and combination strategies are the response.

Targeted Therapy: how this front works · animated schematic, not to scale

Technologies

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Standard of care
Anti-angiogenic therapy

Anti-angiogenic therapy cuts off the tumour's blood supply; it is now mostly used to help immunotherapy work better.

Preclinical
Antibody-oligonucleotide conjugates

An ADC that carries a gene-silencing strand instead of a chemotherapy, so it can switch a protein off rather than poison the cell.

Approved
Bispecific antibodies

A bispecific antibody is one antibody with two different grabbing arms, so it can block two targets at once or pull an immune cell onto a cancer cell.

Phase 2
Cancer neuroscience: cutting the nerve supply

Tumours recruit nerves and use nerve signals to grow. Blocking that traffic, with beta-blockers or botulinum toxin, is being tested.

Approved
CDK4/6 inhibitors

Pills that stop the cell-division engine, added to hormone therapy for the most common type of breast cancer.

Phase 1
De novo designed protein binders

Designing a protein from scratch on a computer to grip a chosen target, instead of finding one in an animal or a library.

Phase 1
Degrader-antibody conjugate (DAC)

An ADC that delivers a protein-destroying molecule instead of chemotherapy, hitting targets inside the cell that were previously unreachable.

Preclinical
DNA origami nanorobots

Folded DNA machines that open only when they touch a tumour, releasing a payload or clotting the tumour's blood supply.

Phase 1
Engineered exosomes as drug carriers

Loading the tiny vesicles cells naturally use to talk to each other with a cancer drug, so the body treats the carrier as its own.

Concept
Epigenetic editing (durable gene silencing)

Switching a gene off for good without changing the DNA sequence, by writing chemical marks onto it.

Phase 2
Hypoxia-activated prodrugs

A harmless molecule that turns into a poison only where there is no oxygen, which in the body means inside a tumour.

Concept
In vivo base and prime editing for cancer

In vivo base and prime editing would rewrite a cancer's DNA letter by letter inside the body. It works in the liver for inherited disease; nobody has yet corrected a cancer this way in a person.

Approved
KRAS & RAS inhibitors

Drugs against the most common cancer gene, considered impossible to target until sotorasib in 2021.

Phase 1
Logic-gated therapeutics (AND, NOT gates)

Cells or drugs that fire only when two conditions are true at once, so healthy tissue expressing just one of them is spared.

Phase 2
Metabolic therapy: starving the tumour of a nutrient

Removing an amino acid or nutrient that certain tumours cannot make for themselves, while normal cells can.

Phase 1
Molecular glue discovery platforms

Molecular glues are small molecules that stick two proteins together so the cell destroys one of them. They are smaller and more drug-like than bifunctional degraders.

Standard of care
Monoclonal antibodies

Lab-made immune proteins that lock onto one target, either blocking it or flagging the cell for destruction.

Phase 2
Oligonucleotide therapeutics

Oligonucleotide therapeutics are short synthetic strands of genetic code that silence a specific cancer gene.

Approved
PARP inhibitors

Pills that block a DNA repair backup, killing cancer cells that already lost their main repair system (BRCA).

Preclinical
Programmable DNA-targeting therapeutics

Programmable DNA-targeting therapeutics are an experimental idea: a drug that reads a cell's DNA, recognises a cancer-specific sequence, and kills only cells that carry it. Change the guide, and the same drug becomes a new drug.

Approved
PROTACs & molecular glues (targeted protein degradation)

Instead of blocking a protein, these drugs tag it for the cell's own garbage disposal, removing it entirely.

Phase 1
Radioligand plus DNA-repair inhibitor combinations

Adding a PARP or ATR inhibitor to a radioactive drug so the tumour cannot repair the damage the radiation causes.

Preclinical
Senolytics and senescence-directed therapy

Chemotherapy leaves behind zombie cells that will not divide but poison their neighbours. Senolytics aim to clear them.

Standard of care
Small-molecule kinase inhibitors

Pills that block the specific enzyme a cancer relies on. Imatinib in 2001 proved a cancer could be switched off by design.

Approved
Synthetic lethality approaches

Finding a second gene that a cancer needs only because its first gene is broken, then hitting the second one.

Preclinical
Targeting the tumour's own microbes

Some tumours contain bacteria and fungi that shelter cancer cells and break down chemotherapy. Killing them may make treatment work.

Preclinical
Targeting tumour mechanics and pressure

Stiff, high-pressure tumours squeeze their own blood vessels shut, keeping drugs out. Softening them is a way in.

Key papers

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institutions

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