doktorska disertacija
Povzetek
Protitelesa in rekombinantne učinkovine druge generacije, kamor sodijo tudi Fc-fuzijski
proteini, predstavljajo visok delež na svetovnem trgu bioloških učinkovin, saj obstaja široko
področje njihove uporabe za številne indikacije. Ker biološka zdravila proizvajajo živi
organizmi, ki so po svoji naravi spremenljivi, lahko zdravilna učinkovina v končnem biološkem
zdravilu izraža določeno manjšo stopnjo variabilnosti (mikroheterogenosti), ki pa mora biti
znotraj sprejemljivega razpona, da bi dosledno zagotavljali varnost in učinkovitost. Ena izmed
pomembnejših posttranslacijskih modifikacij, ki ključno vpliva na njihovo strukturo, topnost,
stabilnost, funkcijo, lokalizacijo, zvijanje in na interakcije z drugimi proteini, je glikozilacija.
Proizvodnja terapevtskih glikoproteinov tako poteka pod nadzorovanimi pogoji, ki jih je treba
konstantno spremljati, da bi zagotovili dosleden, ponovljiv in predvidljiv vzorec glikozilacije.
S tem namenom smo želeli razviti kromatografske tehnike za določanje različnih izooblik
protiteles, ki bi jih lahko vključili v sledenje bioprocesa. V prvem delu doktorske disertacije
smo okarakterizirali lektinske ligande za afinitetno kromatografijo. V ta namen smo lektine
modificirali za uspešnejšo vezavo na nosilce in boljšo izpostavljenost aktivnega mesta.
Biološko aktivnost smo preverjali z interferometrijo z biološkimi plastmi (ang. Biolayer
interferometry oz. kratica BLI). Modificiran lektin rPA-ILNME6, ki smo ga poimenovali rPE6,
je, imobiliziran na nosilec, izkazoval boljšo kinetiko vezave v primerjavi s prvim. V drugem
delu doktorske disertacije smo v nadaljevanju določali pogoje za delno razvijanje protiteles, ki
je potrebno, da se ogljikovi hidrati izpostavijo in s tem postanejo dostopni za vezavo lektinov.
Protitelesa smo inkubirali pri povišanih temperaturah ob prisotnosti reducenta in detektirali
spremembe s pomočjo merjenja intrinzične fluorescence triptofana, DLS-meritev, gelsko
izključitvene kromatografije in ionsko izmenjevalne kromatografije. Glede na rezultate meritev
intrinzične fluorescence in BLI-meritev je do razvijanja protiteles sicer prišlo, vendar sta bila
delež razvitih molekul, pa tudi njihova stabilnost nizka, saj jih nismo uspeli zaznati s
kromatografskimi metodami. Vzporedno smo zato kot predstavnike glikoproteinov pri razvoju
kromatografskih nosilcev z lektini uporabljali Fc-fuzijske proteine, z znanimi glikanskimi
strukturami, ki so izpostavljene na zunanji strani proteina. Na ta način smo lahko v tretjem delu
doktorske disertacije okarakterizirali poliHIPE-nosilec z vezanim lektinom rPE6. Kapaciteto
nosilca smo ocenili na 0,57 mg/ml, mejo detekcije pa med 0,19 mg/ml in 8 mg/ml. S pomočjo
BLI-metode smo pokazali, da je vezavna kinetika imobiliziranega lektina rPE6 dovolj hitra, da
nima vpliva na vezavo Fc-fuzijskega proteina. To omogoča uporabo razvitega nosilca za hitro
in učinkovito ločbo izooblik glikoproteinov. Čas ločbe smo optimizirali na 10 min, pri pretoku
mobilne faze 8 ml/min. PoliHIPE-nosilec z vezanim lektinom rPE6 je ohranjal stabilnost vsaj
še pet mesecev po izvedeni imobilizaciji. V četrtem delu doktorske disertacije smo prikazali še
konkretni primer uporabe tega nosilca za spremljanje perfuzijskega bioprocesa proizvodnje Fc-
fuzijskega proteina. Vzorec dobljenih koncentracij s poliHIPE-nosilcem se je ujemal z rezultati,
dobljenimi s pomočjo izolacije s kolono, z imobiliziranim protienom A. Komponente gojišča,
kot so HCP-ji in DNK-molekule, niso motila detekcije, zaradi česar je razviti nosilec primeren
za ločevanje izooblik glikoproteinov, ki imajo v glikanski strukturi mono-/disaharide, ki jih
veže lektinski ligand, v našem primeru terminalno vezani galaktozo in N-acetillaktozamin.
Nosilec smo uporabili za metodo določanja debeline sloja z merjenjem padca tlaka, ki omogoča
sledenje nastajanja agregatov Fc-fuzijskega proteina, vendar je bila njihova koncentracija pod
mejo detekcije, smo pa s to metodo pokazali agregacijo lektina med imobilizacijo.
Ključne besede
glikozilirani proteini;monoklonska protitelesa;Fc-fuzijski proteini;lektini;kromatografija;lektinske kromatografske kolone;interferometrija z biološkimi plastmi;doktorske disertacije;
Podatki
Jezik: |
Slovenski jezik |
Leto izida: |
2021 |
Tipologija: |
2.08 - Doktorska disertacija |
Organizacija: |
UL FKKT - Fakulteta za kemijo in kemijsko tehnologijo |
Založnik: |
[M. Stantič] |
UDK: |
66.081.3(043.3) |
COBISS: |
90194435
|
Št. ogledov: |
261 |
Št. prenosov: |
41 |
Ocena: |
0 (0 glasov) |
Metapodatki: |
|
Ostali podatki
Sekundarni jezik: |
Angleški jezik |
Sekundarni naslov: |
Development of chromatographic techniques of process analytical technology for determination of monoclonal antibody isoforms |
Sekundarni povzetek: |
Antibodies and recombinant active substances of the second generation, which also include the
Fc-fusion protein, represent a high percentage of the global market of biologically active
substances as there is a wide range of their use in numerous applications. Because biological
medicines are produced by living organisms, the healing substance in the final biological
medicine can express a certain lower stage of variability (microheterogeneity), which has to be
within a certain acceptable range. One of the more important post-translation modifications,
that has a vital influence on their structure, solubility, stability, function, locality, folding, and
interactions with other proteins, is glycosylation. The production of therapeutic glycoproteins
thus takes place under controlled conditions, which must be constantly monitored to ensure a
consistent, reproducible and predictable pattern of glycosylation. With this aim, we wanted to
develop chromatographic techniques for determining different isoforms o antibodies, which we
could include in a bioprocess analysis. In the first part of the doctorate dissertation, we
characterized lectin ligands for affinity chromatography. For this purpose, we modified the
lectins for a more successful immobilization to carriers and better exposure of the active region
for binding. Biological activity of lectins was checked by biolayer interferometry (BLI). The
modified lectin rPA-ILNME6, which we named rPE6, bonded to a carrier showed better
bonding kinetics in comparison to the first. In the second part of the doctorate dissertation, we
further determined the conditions for partial antibody unfolding, which is needed, for the
carbohydrates to expose themselves and with that become accessible to bond with lectins. We
exposed the antibodies to temperature stress with the presence of a reductant and detected
changes with the help of tryptophane intrinsic fluorescent measurements, DLS measurements,
size-exclusion chromatography, and ionic exchange chromatography. According to the results
of intrinsic fluorescence and BLI measurements, the unfolding of antibodies was achieved, but
the proportion of unfolded molecules, as well as their stability, was low, as they could not be
detected by chromatographic methods. Because of the unsuccessful unfolding of the anty bodies
we used Fc-fusion proteins, as representatives of glycoproteins, with known glycan structures
exposed for bonding, for the development of chromatographic carriers with lectins. This way
we were able to characterize a poliHIPE carrier with the bonded lectin rPE6 in the third part of
the doctorate dissertation. We estimated the carrier capacity to 0.57 mg/ml and the limit of
detection between 0.19 mg/ml and 8 mg/ml. We showed that the binding kinetics of the
immobilized lectin rPE6 is fast enough so that it doesn't affect the Fc-fusion protein binding.
This allows us to use the developed carrier for fast and efficient separation of glycoprotein
isoforms. We optimized the separation time to 10 min, at a mobile phase rate of 8 ml/min, and
the carrier maintained stability for a further four months after immobilization. In the fourth part
of the doctorate dissertation, we showed an example of the use of this carrier for monitoring a
perfusion bioprocess production of an Fc-fusion protein. The concentration pattern obtained
with poliHIPE carrier matched with the results obtained with the protein A column isolation.
The components of the medium, HCPs and DNA molecules did not interfere with the detection
with our carrier, which makes it suitable for separating isoforms of glycoproteins that have
oligosaccharides bound by lectin ligand in the glycan structure. The carrier was also used to
determine layer thickens by measuring pressure drop, which allows us to track the aggregate
formation of the Fc-fusion protein, but their concentration was below the limit of detection.
However, we used this method to show lectin aggregation during immobilization. |
Sekundarne ključne besede: |
lectins;monoclonal antibody;Fc-fusion protein;chromatography;biolayer interferometry;Kromatografija;Disertacije; |
Vrsta dela (COBISS): |
Doktorsko delo/naloga |
Študijski program: |
1000381 |
Konec prepovedi (OpenAIRE): |
1970-01-01 |
Komentar na gradivo: |
Univ. v Ljubljani, Fak. za kemijo in kemijsko tehnologijo |
Strani: |
XVIII, 100 str. |
ID: |
13854611 |