Drug Development & Regulation

Drug Discovery

TRİlaç Keşfi

The early research process of identifying a disease-relevant biological target and finding and optimizing a molecule that modulates it, to yield a candidate suitable for development.

In depth

Classic stages are target identification and validation, hit identification (high-throughput, fragment-based or virtual screening), hit-to-lead work and lead optimization, in which potency, selectivity, ADME properties and safety are improved iteratively. The process ends with selection of a development candidate that enters preclinical testing.

Approaches include phenotypic screening, target-based and structure-based (rational) design and natural-product sources. Imatinib, designed against the BCR-ABL tyrosine kinase, and captopril, modeled on ACE-inhibiting snake-venom peptides, are classic examples of rational design. Only a small fraction of candidates reach the market, and the path from discovery to approval usually takes more than a decade.

Example drugs

Imatinib Captopril
TRIn Turkish

İlaç Keşfi

Hastalıkla ilişkili bir biyolojik hedefin belirlenmesini ve bu hedefi etkileyen bir molekülün bulunup optimize edilerek geliştirmeye uygun bir aday ilaca dönüştürülmesini kapsayan erken araştırma sürecidir.

Klasik aşamalar; hedefin tanımlanması ve doğrulanması, vuruş (hit) bileşiklerinin belirlenmesi (yüksek verimli, fragman temelli ya da sanal tarama), vuruştan öncü bileşiğe geçiş ve potens, seçicilik, ADME özellikleri ile güvenliliğin döngüsel olarak iyileştirildiği öncü optimizasyonudur. Süreç, klinik öncesi çalışmalara alınacak bir geliştirme adayının seçilmesiyle tamamlanır.

Başlıca yaklaşımlar fenotipik tarama, hedef temelli ve yapı temelli (rasyonel) tasarım ile doğal ürün kaynaklarıdır. BCR-ABL tirozin kinazına yönelik tasarlanan imatinib ve ACE'yi inhibe eden yılan zehiri peptitlerinden yola çıkılarak geliştirilen kaptopril, rasyonel tasarımın klasik örnekleridir. Adayların yalnızca küçük bir bölümü piyasaya ulaşır; keşiften onaya kadar geçen süre genellikle on yılı aşar.

Updated 24.09.2026 0 views Educational reference — verify against current sources before clinical use.

Read next

Lead CompoundÖncü Bileşik

A molecule with confirmed, reproducible activity at a chosen target and acceptable drug-like properties that serves as the chemical starting point for optimization into a drug candidate.

 Preclinical StudyKlinik Öncesi Çalışma

Laboratory and animal investigation of a drug candidate's pharmacology, pharmacokinetics and toxicity, carried out before first administration to humans to justify and design safe clinical trials.

 in silicoin siliko

Experiments or studies performed using computer simulations, modeling, or computational methods to predict biological processes without physical testing.

 Mechanism of ActionEtki Mekanizması

The specific biochemical interaction through which a drug produces its pharmacological effect, usually binding to a defined molecular target such as a receptor, enzyme, ion channel or transporter.

 SelectivitySeçicilik

The degree to which a drug acts on a particular target (receptor subtype, enzyme or tissue) rather than on others, usually expressed as the ratio of its affinities or potencies at different targets.

 Phase I Clinical TrialFaz I Klinik Araştırma

The first stage of testing an investigational drug in humans, usually in a small number of healthy volunteers, designed mainly to assess safety, tolerability, pharmacokinetics and, where possible, pharmacodynamics.