EMMI Intensive Programme "Design, Synthesis and Validation of Imaging Probes Torino, 19-30 September 2011



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EMMI Intensive rogramme "Design, Synthesis and Validation of Imaging robes Torino, 19-30 September 2011 Basic principles and procedures of solid phase peptide synthesis Lorenzo Tei, hd Dipartimento di Scienze dell Ambiente e della Vita Università del iemonte rientale Amedeo Avogadro Viale T. Michel 11, 15121, Alessandria Contents: Introduction istorical background Different strategies for SS ossible problems Aminoacids and protections Amide bond formation Solid supports and linkers Analytical tests eptide isolation and purification

WY ETIDES? Immune peptides: synthetic antigens; vaccines diagnostic tools immunostimulator peptides; muramyl dipeptide tuftsin derivatives ormones: oxytocin vasopressin insulin somatostatin Gn etc. europeptides: cholecystokinin neurotensin Antibiotics: tachikinin gramicidine S Transporter peptides: penetratin oligoarginine IV-Tat protein eptides for structural studies: turn mimicking cyclic peptides Applications of synthetic peptides Carriers: deliver diagnostic or therapeutic agents to targeted cells Toxins: conotoxins spider toxins snake toxins ionchanel blockers Enzymes and enzyme inhibitors: ibonuclease A Advantages of utilizing peptides as therapeutic molecules: igh activity igh specificity Unique 3D characteristics o accumulation in organs Low toxicity Less immunogenic than antibodies They can be made synthetically, by recombinant methods or by chemical modification of an isolated natural product.

eptides developed for therapeutic applications: Allergy/Asthma Arthritis Cancer Diabetes Cardiovascular diseases Inflammation Vaccines phtalmology Central ervous System diseases Antiviral Antibacterial eptide synthesis: Solution synthesis: ptimisation of reaction conditions and yields. urification of every step. Time consuming. Synthesis of small peptides. Useful for large-scale manufacturing. Solid phase synthesis: Anchoring of the peptide chain on a resin. Use of excess reagents removed by filtration and washing procedures. Simple, rapid and repetitive steps. igh yields. ossible automation Synthesis of long peptides.

Beginnings of the SS Merrifield in 1963 introduced the use of a polystyrenic support for the synthesis of peptides. Development of the Boc strategy (use of quite strong acidic conditions) In the 70 different polymeric supports and more labile protecting groups were developed. In 1978 the Fmoc-based SS was introduced. In the last 15 years the Fmoc approach has prevailed over the Merrifield SS. Solid phase peptide synthesis The idea: T AA 1 X anchoring T AA 1 T AA 2 AA 1 T AA 2 coupling (- 2 ) deprotection AA 1 = resin T = 2 protecting group (boc, Fmoc) = side chain protecting group

T AA 2 AA 1 deprotection coupling repetitive cycle T AA n AA 2 AA 1 cleavage + final deprotection AA n AA 2 AA 1 Fmoc strategy in SS Fmoc ' olimer ' =, 2, Cl Attachment Fmoc olimer Deprotection Fmoc " olimer epeat deprotection and coupling Fmoc Coupling 2 " Activated olimer Fmoc " n olimer Cleavage eptide and olimer

Fmoc deprotection + + + C 2 + 2 _ 2 + Boc Strategy in SS Boc Boc ' olimer olimer epeat deprotection and coupling Attachment Coupling ' Boc Boc olimer Deprotection 2 olimer Activated The Boc approach needs to use highly toxic liquid F for cleavage from the resin. Boc n ' olimer Cleavage eptide and olimer

Boc deprotection 2 + C 2 + + Common AA side-chain protecting groups used in Fmoc SS Side-chain fuctionality of amino acids Commonly used protecting group Abbreviation Arg 2 S bf Asp, Glu, Ser, Thr, Tyr tbu Lys, Trp (2) () Boc Asn, Gln, Cys, is X X trt

Coupling methods in Fmoc SS re-activation method: re-activation of the acid via activated esters. -hydroxysuccinimide (Su); slow formation of the amide bond. entafluorophenyl (fp) ester; in the presence of Bt the rate of reaction is very rapid. Fmoc Bt esters Fmoc fp esters Su esters F F F F F Fmoc Coupling methods in Fmoc SS In situ - activation method: Carbodiimides (DCC and DIC) Benzotriazole F 6 salts (BTU, yb, ATU) ( 3 C) 2 + (C 3 ) 2 C Dicyclohexylcarbodimide (DCC) + _ F 6 - BTU F 6 - yb ( 3 C) 2 + + _ (C 3 ) 2 F 6 - ATU

Coupling using Diisopropylcarbodiimide The use of Bt minimises the formation of unreactive -acylurea Mechanism of amide bond formation using yb/bt/diea Fmoc Fmoc Fmoc (DIEA) - - ( - Bt ) (yb) F 6-2 2 u Fmoc + + - Bt 3

Solid supports: Dry polystyrene beads have an average diameter of 50 µm, but in DMF or DCM they swell 2.5 to 6.2 fold in volume. SS takes place within a well solvated gel containing mobile and reagent accessible peptide chains. Small particle sized resins with low cross linking allows rapid diffusion of reagents inside the beads. Solid support and peptide backbone may be of comparable polarities (polyamides, EG). esins for SS esin Composition Functional group Chloro/Aminomethyl polystyrene Tentagel 1% divinylbenzene cross-linked polystyrene olystyrene-polyethylene glycol graft polymer C 2 Cl, C 2 2, benzhydrylamino C 2, C 2 2 Cl n Cl Cl Cl n Merrifield Tentagel

esins for SS EGA ovagel TM Bis 2-acrylamidoprop-1-ylEG, 2-acrylamidoprop- 1-yl[2-aminoprop-1-yl]EG dimethylacrylamide co-polymer (4 --methylpolyethylene glycoxycarbonylaminomethyl)polystyrene C 2 2 C 2 2 Me 2 n 2 n C 3 2 2 2 EGA ovagel TM Linkers: The purpose of the linker are: provide a reversible linkage between the synthetic peptide chain and the solid support. rotect the C-terminal α-carboxyl group during the process of chain extension. Most linkers release peptide acids or amides upon treatment with TFA. Some linkers permit the cleavage with nucleophiles, used for the preparation of C-terminal modified peptides such as esters secondary amides, aldehydes, alcohol.

ydroxymethylbased linkers Linkers: Aminomethyl-based linkers Trityl chloridebased linkers Me 2 Wang linker (ydroxymethylphenoxy) Me Me ink linker (trialcoxybenzhydrylamine) Me Cl Cl 2-Chlorotrityl chloride Sasrin linker 2 Me AL linker (trialcoxybenzylamine) 2 MBA linker C 3 Sieber linker (9-Aminoxanthenyl) Bayer's 4-carboxytrityl linker 3C ink acid linker esin handling procedures Apart from EGA resin, all resins have to be swollen before use. Underivatizedpolystyrene resins swell only in dichloromethane. All the other are swollen in DMF. esins (except EGA) can be dried under vacuum without damage. esins are fragile and has to be handled carefully (do not use magnetic stirrers!)

Attachment of 1 st amino acid For hydroxymethyl based linkers quite harsh conditions has to be employed: 1. use of the symmetrical anhydride of the Fmoc amino acid and DMA in DMF. 2. Use of the Fmoc amino acid and dichlorobenzoyl chloride in DMF/pyridine Fmoc 1) Fmoc 0.1 eq. DMA, DMF Fmoc 5 eq. 2) Fmoc 5 eq. DCB (5 eq.), pyridine (8.25 eq.) DCM Fmoc Attachment of 1 st amino acid 1) Trityl based linkers: Fmoc 1.2 eq 3 eq. DIEA, DCM Cl Cl Fmoc 2) Toluene, AcCl 60 C 3h 3 eq. DIEA, DCM Fmoc Fmoc 1.2 eq. Aminomethyl-based linkers: Generally the amino function is Fmoc protected Standard piperidine deprotection and subsequent coupling of the 1st amino acid

Synthesis protocol: Swelling of the resin 1 st Fmoc amino acid attachment Deprotection Fmoc group with piperidine in DMF Washings with DMF (between 5 to 10 times) Fmoc amino acid coupling (Activators/DIEA/DMF) Washings with DMF Deprotection Fmoc and so on After last coupling the resin has to be dried in vacuo before cleavage Analytical tests Qualitative amine tests: Kaiser test (ninhydrin test) http://www.natureprotocols.com/2007/10/24/monitoring_solid_phase_peptide.php TBS test Chloranil test http://www.chempep.com/chemep-fmoc-solid-hase-eptide-synthesis.htm Fmoc determination for estimation of first residue attachment: The loading of the 1 st AA can be calculated determining the absorbance at 290 nm (dibenzofulvene formed in the Fmoc deprotection)

ossible problems Aggregation eptide chains on resin can form secondary structures or aggregates either with other peptide chains or with the polymer support. Mainly caused by -bonding and hydrophobic interactions (high proportion of Ala, Ile, Asn or Gln) Enantiomerization During coupling the acidity of the atom on the α-carbon is enhanced by the carboxy group activation. May occur on attachment of Cys, is, ro, Met, or Trp on hydroxymethyl-based resins Side reactions are if the AA are protected and the correct procedures are followed. Aspartimide formation can occur if Asp is coupled to Gly, Ser, Asn, Thr or Arg. Aspartimide formation X u u u ydrolysis Base catalyzed aspartimide formation involves attack by the nitrogen atom attached to the α-carboxy group of either aspartic acid or asparagine on the side chain ester or amide group, respectively. ucleophilic attack then causes subsequent ring opening, which gives rise to a mixture of α-aspartyl and β-aspartyl peptides.

Diketopiperazine formation eptides containing proline or -alkylated amino acids attached via benzyl ester to the resin may have the problem of diketopiperazine formation This not only causes a yield reduction but may also lead to the generation of truncated sequences. In these cases is better to use a more hindered trityl resin ' ' Cleavage from the resin 95% TFA for 1-3h cleaves the peptide from the resin and deprotect t-butyl groups, bf group from Arg, Trt group from Cys, Gln, Asn and is. ighly reactive cationic species are generated that can react with amino acids containing electron rich functional groups: Tyr, Trp, Met and Cys. ucleophilic reagents (scavengers) are added to the TFA to quench these ions: water, 1,2-Ethandithiol (EDT), triisopropylsilane (TIS), thioanisole.

Cleavage cocktails If Cys is absent we normally use TFA, TIS 95:5 v/v. With Cys we add EDT (TFA, TIS, EDT; 95:3:2 v/v). With Met we add thioanisole (TFA, TIS, Thioanisole; 95:3:2 v/v) eagent K (TFA, thioanisole, 2, phenol, EDT; 82.5:5:5:5:2.5 v/v). eagent (TFA, thioanisole, anisole, EDT; 90:5:3:2). With Chlorotrityl chloride resins protections on the AA sidechains can be mantained using DCM, Ac, trifluoroethanol 80:10:10 v/v. With Trp a further step with a 5% water solution of Ac is required to obtain the fully deprotected peptide. eptide isolation and purification recipitated by addition of cold diethyl ether Directly from TFA solution or following evaporation of TFA and volatile scavengers. Centrifuge and wash with Et 2 3 times Dry the solid in vacuo urification by LC Characterisation by MALDI-TF mass spectrum, M spectrometry.

Multisyntech SY II Syro II automated peptide synthesizer CEM Liberty Liberty automated peptide synthesizer shown with single-mode microwave reaction vessel