cell & gene therapy bioprocessing & commercialization 2020 · every year, informa connect invites...

Cell & Gene Therapy Bioprocessing &Commercialization 2020Post-event Report

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Introduction & Contents


Cell, gene, and tissuetherapies continue tobe the fastest growingfields in thebiopharmaceuticalindustry.

These advancedmedicines are treatingcomplex diseases andconditions such ascancer, tissue injuries,and revolutionaryvaccines.

But significantprocessing andanalytical challenges

processing andanalytical challengesremain, however.

Every year, InformaConnect invites cell,gene, and tissuetherapy industryrepresentatives meet todiscuss best practicesand best strategies forovercoming thosechallenges.

Like most meetings in2020, the Cell andGene Bioprocessingand Commercializationconference was offered

and Commercializationconference was offeredentirely online.

During 19–22 October,participants wereinvited to listen topresentations,plenaries, speakerpanels, and even livevirtual laboratorytours.

The five tracks werecell therapies, in vivogene therapies, ex vivogene-edited celltherapies, andadvanced tools for cell

therapies, andadvanced tools for celland tissuemanufacturing.

This report highlights afew presentations andspeaker panels (bytopic).

Maribel Rios,

ManagingEditor atBioProcessInternational

Welcome,

Jump to any article using the contents on the following page, or at any time using the Contents menu in thetop left. There you can also download this report as a PDF.

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Contents

1. Panels and Plenaries

From investment trends and standardization toimmunotherapy and the impacts of COVID-19, Maribel Riosreflects on the most popular live sessions across the week.

2. Cell Therapy

Maribel Rios reviews the top sessions from the biggesttrack of the event, including automated analytics andautologous CAR-T cell therapy.

3. In Vivo Gene Therapy

We look back at a popular talk exploring next generationcell-line development and recombinant adenoassociatedvirus production.

4. Gene-Edited Ex Vivo Cell Therapy

Maribel Rios highlights key takeaways from the track'shottest panel, which discussed gene modifiedallogeneic MSCs and NK Cells.

5. Advanced Tools for Cell and TissueManufacturing

We explore presentations showcasing diverse tissueengineering applications and automated cellharvesting.

Panels & PlenariesInvestment, viral vectors, CAR-T and COVID-19


The discussion began withprojections about the futureof allogeneic andautologous cell therapiesand whether “off-the-shelf”allogeneic products wouldeventually be the mostpreferred platform overautologous formats.

Schmidt and others agreed

that both types would be apart of the industry in thelong term. For example,autologous chimeric antigenreceptor (CAR) T celltherapies are beneficial forcertain indications.

“Ultimately what matters isefficacy and safety forpatients,” said Schmidt.

Forte also noted that thechoice between autologousand allogeneic depends onthe indication, the unmetmedical need, cost,scalability, the value beingbrought by the final product,and other factors.

Mason added thatmanufacturing also is a

critical factor. “There isn’t avein-to-vein process that canbring down the cost of thesetherapies, which are tooexpensive regardless of howthey are created.”

So new manufacturingtechnologies are neededthat could reduce costs. Thevalue of using contract

Panels and Plenaries

Some of the most exciting talks were found in the live agenda, as hundreds of attendees tuned into join the panel discussions and plenaries. Here, Maribel Rios, Managing Editor at BioProcessInternational, reflects on the most popular live sessions across the week.

Investment in the CGT space: Trends, Technologies, and Early Stage Success

Led by Mike Zhao (MSQ Ventures), the panel included Miguel Forte (Bone Therapeutics), Jak Knowles (Leapsby Bayer), Joey Mason (M Ventures), and Dominic Schmidt (Syncona Investment Management Ltd.).

manufacturing organizations(CMOs) was anotherdiscussion point.

Knowles acknowledged thatusing CMOs would behelpful depending on thetechnology. “Mostautologous therapies can bemade using lentivirusplatforms, for example, andthere are many wellestablished CMOs that candeliver GMP [goodmanufacturing practice]lentiviral transduction.

On the allogeneic side,people tend to prefer geneediting for allogeneicproducts, which is notachievable with lentivirus.

So companies that want tomake allogeneic productstend to do the gene editing

in-house, and theautologous programs tendto use CMOs to produce thelentivirus for transduction.

From a pharma perspective,you prefer companies tobuild and own theirtechnology so that you canget follow-on products.

But there is value in workingwith well-validated CMOsbecause most of them canscale-up for commerciallaunch.

The big difference that I’venoticed is that most

allogeneic companies tendto focus on in-house geneediting, but autologousprograms can use externalproduction methods forlentivirus.”

Zhao asked panelists whatthey look for what “earlysuccess” looks like to them.Knowles acknowledged thatthe definition of earlysuccess depends ontherapeutic modality andthe underlying technology.

For example, in one case hegained confidence when atechnology “climbed the

species ladder” and hadsuccess in different animalmodels during earlydevelopment.

Forte said the mostimportant element is when atherapy progresses tohuman clinical trials and theinitial clinical data becomesavailable.

“If you’re in the early stagesand you get the earlysuccesses, you need to honethe technology that youhave because you are still inprocess development andbuilding your product,” said

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"Most allogeneic companies tend to focus on in-house gene editing, butautologous programs can use external production methods for lentivirus."

Jak Knowles, VP Venture Investments, Head of Pharma Investments, Leaps by Bayer

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Knowles.

“Later, if you’re able to scaleup, you may be able topartner and outsource. Thatis late-stage success. Forearly stage success, youneed to hone yourtechnology and have yourclinical data to support it.”Mason provided a“macroview” of what is likelyto be successful in acompany.

Investors have seen a lot gowrong over their careers. Soif you’re trying to ‘givecomfort’ to your investorbase, you need tounderstand the journey andwhere the critical pieces arethat are missing, or need tobe fixed, or need to beaddressed as time passes.

You need to know howmuch it’s going to cost youto get to those criticalmilestones in the clinic.

All of that would be veryimportant as we look atdeals. Early understandingof what your competition islikely to be and whattechnologies are out thereare critical.”

One of the main discussionpoints centered on theopportunities andchallenges thatbiopharmaceutical scientistsare facing regarding theCOVID-19 pandemic.

All areas of the industry,including supply chain, havebeen facing uncertaintiessuch as determining how tostreamline R&D, production,and commercialization,

optimizing processes, andstaying in time.

These difficulties have beena part of all phases of celland gene therapydevelopment.

Ricordi noted that scalabilitywas as one of thechallenges, from hundredsof doses to thousands,specifically moving from two-dimensional expansion to

PANEL: COVID-19: Trailblazers in the CGT Industry

Led by Miguel Forte (Bone Therapeutics), the panelincluded Claudia Berron (Avantor), Chris Gemmiti(Sentien Biotechnologies), John Lewis (Aegis LifeInc. and Entos Pharmacetuticals), Racheli Ofir(Pluristem Therapeutics), Lawrence Thompson(Pfizer), and Camilo Ricordi (National Academy ofInventors, Italian Supreme Council of Health).

three dimensionalbioreactors.

Other challenges were in thesupply chain, includingobtaining enough materialto expand cells to the levelsneeded.

Panelists generally agreedthat such “challenges” wereresults of the opportunitiesand the successes of arapidly growing field and theneeds to generate highvolumes.

Other noted opportunitiesincluded the ability to obtain

clinical readouts in shorttimes, which allowsresearchers to obtain “goodclinical proof” that therapiesare working, and goodcooperation with regulatoryagencies.

Panelists also talked aboutthe challenges brought onas a result of lockdowns(and other pandemicrestrictions) and theopportunities to applytechnologies used inresponse to COVID-19 forother indications.

Lewis pointed out that

biopharmaceuticaldevelopers rely on a lot ofpartners, and many tasksmust be completed in ashort time.

Safety and product qualityare not compromised butsome risks are taken whenmultiple tasks must be donein parallel.

One challenge fordevelopers has been toidentify industry partnersthat are still used working in“typical” timeframe fromdiscovery to clinic.

He also noted that thisprocess also can be anopportunity because manypotential partners (industryand regulatory) around theworld are motivated towardthe same goals.

Thompson chimed in on thediscussion about theimportance and challenge ofhaving enough supplies (e.g.,enzymes, lipids, and evensyringes) to generatehundreds of millions ofdoses of a vaccine in a veryshort time.

“It’s amazing to do so muchat the same time. When youare doing clinical trials butalso doing late-stagemanufacturing activities as ifeverything is going to work.Typically, we (Pfizer) doesn’ttake these risks, but now weare doing everything at thesame time.”


"Safety and product quality are not compromisedbut some risks are taken when multiple tasksmust be done in parallel."

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The discussion began witheveryone stating theirbiggest supply chain issuewith either starting or rawmaterials.

One of the main difficultiesthat was noted isdetermining the differencebetween starting and rawmaterials from a regulatoryperspective (such as formaking viral vectors).

Panelists pointed out thatthe European Union hassome documentation on thedifference between both

terms, but the US Food andDrug Administration doesnot.

Other supply chainchallenges includedminimizing variability incellular starting material(especially for autologouscell therapy products),mitigating risk with single-and multiple-sourcedsuppliers, and maximizingcontrol and consistency ofan apheresis process.

Panelists pointed out thateven before the pandemic,

biotherapeutic developersfaced problems withchanging lead times anddelays, especially for single-use assemblies and bulkmedia materials.

Other discussion pointsfocused on the shortage ofcertified reference materialsand the challenge of

handling variability of activityin those materials.

Suppliers must either buyreference materials orprepare them in-housewhen a reference product isnot available (which, as onepanelist pointed out, is thecase for most items in a billof materials).

Raw Material, Viral Vector and Supply Chain Considerations

Led by Christopher Bravery (Advbiols), the panel included Scott Burger (Advanced Cell and GeneTherapy), Christiane Niederlaender (formerly MHRA, AMBR Consulting Ltd), Max Sellman (Aldevron), andTom Walls (Spark Therapeutics).


"Suppliers must either buy reference materials orprepare them in-house when a referenceproduct is not available"

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And GMP facilities typicallyconduct biological activityassays on materials whenreceived (if a standardizedassay is available).

Many materials, however,cannot be tested againstcompendial standards, sothose materials can bedifficult to source.

How to ensure activity andconsistency of a materialssuch as a cytokines are“open questions for whichwe don’t have answers for,”said Bravery. “But they aresomething that we need.”

Of the different topologicalforms, the supercoiledplasmid is recognized byFDA as the mosttherapeutically effective.

Plasmid DNA (pDNA) vectorsare used to produce viralvectors and vaccines (e.g.,mRNA-based such as thecurrent COVID-19 vaccines).

Parker presented anoverview of all unitoperations of plasmid DNA(pDNA) purification, from cellharvest to sterile filtration.

In gene therapy transienttransfection, four plasmids(including the plasmidcoating for the transgene)are used to make a viralvector encapsulating a geneof interest.

Parker presented a list ofunique challenges of pDNApurification. They includedlow productivity of microbialfermentation, high viscosityfees, shear sensitivity, andlow-resolution separation.

Parker showed a generalplasmid process,

highlighting whereMilliporeSigma technologiesand services can beimplemented to provide an“integrated approach fromharvest to final fill.”

He also reviewed theapproaches and keyconsiderations for eachpDNA processing step,providing case studies andoperating parameters forsome steps.

Data-Driven Strategies for Downstream Processing ofPlasmid DNA In Viral Vector Production

Thomas Parker (MilliporeSigma) explained that plasmids are double-helix DNAmolecules that are found naturally in bacteria and that replicate intracellularly.

The TIM platform is beingused for the development ofthe company’s allogeneicchimeric antigen receptorCAR T-cell candidate formetastatic colorectal cancer.

“TCR complex is responsiblefor graft versus host disease(GvHD) and attenuation ofthe TCR complex isnecessary for creating CAR-Ttherapies,” said Parone.

When TIM is overexpressedfrom a T cell, it significantlyattenuates CD3-zeta. So,TIM-based allogeneic CAR T-cells do not exhibit in vitro

and in vivo alloreactivity.

The company also hasdeveloped its NKG2Dreceptor, which iscoexpressed by thecompany’s CYAD-101allogeneic candidate (formetastatic colorectal cancer)along with TIM and selectionmarker for an “all in one”vector approach.

The company’s shRNAplatform is being used forthe development of anallogeneic CAR-T candidatefor multiple myeloma.

The company collaboratedwith Horizon DiscoveryGroup, which provided itsSMARTvector technologythat mimics the endogenousnature of microRNA. shRNAenables knockdown geneexpression through RNAinterference.

Parone showed that shRNAtargeting CD3-zeta reducesTCR expression with noovert transcriptomedisturbance. Expression of asingle shRNA also providesprolonged TCR knockdownwithout inducing GvHD.

The size taken up by theshRNA on the vector also isrelatively minimal (250 bp).

Parone showed results ofthe platform’s capability inmultiple gene knockdownand concluded with anoverview of the company’sCYAD-200 series of shRNA-based allogeneic CAR-Tcandidates.

Non-Gene Edited Approaches to Allogeneic CAR-T Cell Therapy

Philippe Parone (Celyad) highlighted two platforms developed by Celyad: a T-cell receptor (TCR) inhibitorymolecule (TIM) technology and a short hairpin RNA (shRNA)-based allogeneic platform.

They include limited access(half of patients require ICUmanagement), failure totreat (manufacturingfailures can delay orprohibit treatment), andcomplex supply chain (as aresult of patient-specificmanufacturing).

The company partneredwith MD Anderson CancerCenter to develop CAR-NK(natural killer) cell therapiesfor patients with non-Hodgkin’s lymphoma. Corzohighlighted the company’sTAK-007 candidate based onthat platform.

The allogeneic therapy isengineered with interleukin(IL)-15, which enablesmultiple ways of recognizingtumors. The first target isCD19. Corzo showed earlyclinical results and futuredevelopment plans.

She concluded byhighlighting the company’sprioritization of data scienceand digital tools for rapiddecision-making, includingdata governance, machinelearning, and datavisualization.

She discussed the issues ofgrade designations forstarting and raw materials,and how realistic it is toprovide internationalstandards to the ATMP(advanced therapy medicinal

products) field.

In the European Unionlegislation, starting materialsare defined as “all materialsfrom which the activesubstance is manufactured

The Potential of CAR-NK

Kathryn Corzo (Takeda) presented the limitations ofcurrent CAR-T platforms.

Starting Material Standardization

Christiane Niederlaender (formerly MHRA, AMBRConsulting Ltd.) focused on internal (within amanufacturing site) standards to ensure qualitycontrol and product consistency and highlighted thebenefits of standardization of procedures andacceptance criteria, especially for starting materials.


Characterization is less challenging fornoncellular starting materials for gene therapymedicinal products. However, quality and purityof such materials are major concerns."

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or extracted.” In the UnitedStates, such materials aretypically referred to as“critical raw materials.”

EU regulations also defineraw materials as “any othersubstances used formanufacturing or extractingthe active substances, butfrom which this activesubstance is not directlyderived (e.g., reagentsculture media, buffers).

Niederlaender focused onregulations pertaining togenetically modified cells (ECDirective, Part IV Annex 1,paragraphs 3.2.1).

Although there are externalstandards for procurementand collection procedures ofsome cells (e.g., bonemarrow, apheresis products,

and blood-derived sources).

“However, even in thesesituations there needs to besome patient-specific andproduct-specific flexibilities.”

Characterization is lesschallenging for noncellularstarting materials for genetherapy medicinal products(e.g., viral vectors, plasmids,gene-editing components).

However, quality and purityof such materials are majorconcerns.

She explained how the useof “supplier grades” have noclear regulatory definitionand proposed thatmanufacturers instead relyon in-house testing, changecontrol, recognized qualitysystems, and qualify

suppliers based on risk.

Niederlaender said thatideally, regulators wouldcertify starting and rawmaterials, but she said thereare difficulties with thisconcept, including how therequired attributes would bedefined and the increasedresource constraints thatwould be put on regulatoryagencies.

Cell TherapyAutomated Analytics and Autologous CAR-T Cell Therapy


However, most biochemicalchanges that are important for cellgrowth occur on a subcellular level,including subcellular molecularoxygen, protons in the form of pH,carbon dioxide, andmacromolecular structures such as

cytokines and proteins.

New measurement technologiesare needed to enable subcellularmeasurements, and automatedanalytics are needed to enhanceefficacy of cell culture.

Such sensors ideally should benoninvasive and highly sensitiveand provide high spatial andtemporal resolutions.

Fluorescent nanosensors havebeen developed that can bedelivered to subcellular spaces andmake continual long-termmeasurements.

The compose of a biologicallyfriendly (inert) matrix that protects

Cell Therapy

Arguably the biggestconference track of theevent, Cell Therapyprovided live and on-demand sessionsexploring topics fromprocess developmentand manufacturing toanalytical technologies.

Maribel Rios looks backat two of the mostpopular sessions acrossthe week.

Augmenting Automated Analytics for Cell and Gene Therapy UsingFluorescent Nanosensors

Veeren Chauhan, (University of Nottingham) explained that traditional bioreactor sensorsmeasure extracellular parameters such as pH, cellular oxygen, and metabolites to ensure thatcells are growing effectively.


"New measurement technologies are needed to enablesubcellular measurements"

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cells from dyes and fluorophoresfrom cellular interferents.

They also contain an analyte and areference fluorophore. Thesensors permit silent report of keybiological parameters, accurateratiometric measurements, andhigh spatial and temporalresolutions.

Chuahan provided details of astudy that used nanosensorssensitive to pH ( acid test.

The sensors contained apolyacrylamide matrix (50-nm indiameter) and composed to twopH-sensitive fluorphores (OregonGreen and carboxyfluorescein), andpH-sensitive rhodamine.

With all three fluorophores, thesensors could make pH sensitivemeasurements pH 3.5 to pH 7.5.

A calibration curve could begenerated by taking a ratio of theintensities of Oregon Green andrhodamine at specific pH.

Chuahan suggested that theaccuracy of the nanosensors (+/-0.17 pH units) makes them ideal foronline measurements in biologicalsystems.

They also can provide real-timesubcellular analytics and contributeto the optimization of each stage ofcell and gene therapymanufacturing.

Chuahan also provided examples ofhow fluorescent nanosensors canbe used to make complexmeasurements in C. elegan andeukaryotic cell lines.

They also can be used to augmentautomated analytics in cell andgene manufacturing processes (off-

line, on-line, and in-line) bymonitoring biochemical subcellularchanges.

After an overview of ICH Q5E andEMA guidelines, she focused onphase-dependent approaches tocomparability, taking into theaccount the lifecycle of drugproducts.

For example, first-in-human studiestypically do not requirecomparability studies, but duringphase 2 and 3, a risk-basedapproach should be taken todetermine the scope and tiers forcomparability assessment.

For paired run studies, risk-developers typically “split either atthe source material or further

downstream, depending on thenature of the change.”

Burst presented a general risk-based comparability strategyflowchart based on assessment ofprocess control and critical qualityattribute assessment and a generalapproach to beginning acomparability assessment.

One approach to comparability riskassessment is scoring, in whichattributes are assigned a numberbased on potential risk of a changeto cause variation in processperformance.

Those scores are used to prioritizewhich aspects to assess in thecomparability assessment.

A three-tier approach tocomparability risk assessment alsohas been a successful strategy.

Brust provided and overview of thetiers (generally: tier 1 isequivalence test approach, tier 2 isquality range, tier 3 is visualassessment approach), andconcluded with a case study ofrisk-based comparabilityassessment for drug productmanufacturing site transfer.

Risk-Based Approaches for Autologous CAR T-Cell Therapy Comparability

Erica Brust (Bristol Myers-Squibb) emphasized that the goal of comparability as defined in the ICH Q5Eguidance is “to ensure quality, safety, and efficacy of drug products . . . produced through collectionand evaluation of relevant data.”

In Vivo Gene TherapyNext Generation Cell-line Development and Recombinant Adenoassociated Virus Production


In a collaboration with Bayer, thecompany has conductedengineering improvements to itsHeLa PCL platform.

Specifically, the company mademolecular changes to the plasmidcomponents and multiple processand screening changes to theoriginal “HeLa 1.0” platform.

The improved “HeLa 2.0” stage hasa 10× increase in yield and a

streamlined clonal selection.

The technology now is used for thecompany’s work in finding a genetherapy for Wilson’s disease.

Wadsworth showed how furtherimprovements to the platform(“HeLa 3.0”) could be used toincrease production ofadenoassociated virus (AAV) vectoryield and how the use of a siRNAknockdown of specific genes could

improve AAV production titer.

He highlighted work on Wilson’sdisease potency assays and the useof clustered regularly interspacedshort palindromic repeats (CRISPR)CRISPR-associated protein 9 (Cas 9)to delete ATP7B (the loss of thisfunction causes copper toxicity inWilson’s disease) in the HepG2 cellline.

In Vivo Gene Therapy

From an informativeselection of insightsacross upstream anddownstreambioprocessing andvector manufacturing,we review the mostwatched presentationin the In Vivo GeneTherapy track.

Next Generation Cell Line Development and Large-Scale BioprocessingTechnology

Sam Wadsworth (Ultragenx Gene Therapy) began by highlighting the features of his company’s genetherapy manufacturing processes based on human embryonic kidney (HEK)293 suspension/plasmidtransfection and HeLa producer cell line (PCL) suspension/adenovirus helper platforms.

Gene-Edited Ex Vivo Cell TherapyGene Modified Allogeneic MSCs and NK Cells


Lee also discussed a case studyfocused on the use engineeredmesenchymal stem cells (MSCs) totreat solid tumors through lentiviralvector transduction.

The cells were derived from bonemarrow and engineered to expresstwo immune-stimulated payloads(interleukins 12 and 21).

Data from preclinical trials thatshowed that the combination ofthose payloads is highly potent andthat it yields a robust antitumor

response across every tumormodel evaluated.

Lee presented his company’sstrategy for transferring its geneticmodification process to goodmanufacturing practice (GMP)scales.

He also showed how the MSCplatform can be expanded to othergene-modified cell therapies. Thesecond study focused on the earlystage development an allogeneicnatural killer (NK) cell treatment for

cancer.

Lee provided an overview of theSenti Biosciences’s method forrobust cell isolation andcryopreservation and emphasizedthe company’s continued work inoptimizing its viral vector processand NK cell transduction andexpansion strategies.

Senti Bio Case Study: Gene Modified Allogeneic MSCs and NK Cells

Gene-Edited Ex Vivo Cell Therapy

Philip Lee (Senti Biosciences) introduced his company’s “gene circuit” platform, which is a genetic“logic system” created by a method of combining DNA.

For overviews of more presentations from the GeneTherapy tracks, check out BioProcess International’sGene Therapy Featured Report published in theNovember/December 2020 issue.

https://bioprocessintl.com/category/november-december-2020-featured-report/https://bioprocessintl.com/category/november-december-2020-featured-report/

Advanced Tools for Cell and Tissue ManufacturingDiverse Tissue Engineering Applications and Automated Cell Harvesting


He presented datasupporting the applicationof tissue-engineered musclerepair (TEMR) constructs inanimal models and tissueengineering to repairvolumetric muscle lossinjuries (face and hand) andthe use of sheet-basedtissue-engineered medicalproducts (TEMPs) for musclerepair.

He presented data showingthe benefits of addingseeding cellular material onan extracellular matrixscaffold (2-cm square area),comparing TEMR (with cells)results (e.g., volume quality,composition, vascularization,contractile force) with thoseof applying bladder acellularmatrix (BAM) alone.

Christ reviewed his group’sTEMR implant process and

manual engineering process,including taking a biopsyfrom a patient, expandingprogenitor cells, matrixseeding (manually), tissuestretching, andpreconditioning of skeletalmuscle constructs in abioreactor for in vitromaturation.

“The idea is that we are notimplanting mature muscle.We are implanting

myoblasts and myotubes(mainly myoblasts) to createa regenerative template,which enhances themicroenvironment for tissuerepair that normally wouldnot exist in adult mammalsto improve tissue repair.”

Christ compared thetraditional manual TEMRcreation process withautomated bioprinting.

Advanced Tools for Cell and Tissue Manufacturing

Development of Modular Biofabrication Platforms for Diverse Tissue EngineeringApplications

George Joseph Christ (University of Virginia) began with an overview of the importance of treating volumetricmuscle loss injuries.

“We envision an automatedbioprinting method wherebywe can take the sameconstruct, put it in a holderin the same bioreactor usedfor the TEMR approach, andbioprinting instead ofmanually seeding at a higherinitial cell density with fewercells in a rapid process, andthen placing the systemback into the bioreactor.”

He summarized the TEMRapproach as being a“hybrid” biofabricationprocess betweenbioprinting and cell sheetsand showed a process (andchallenges) for bioprintingtissue-engineered medicinalproducts (TEMPs).

She focused on capabilitiesand applications of theSepax C and Sefia cellprocessing and isolationinstruments.

The former is composed ofthe hardware, protocolsoftware, and cell processingkit.

The automated system isclosed for compliance togood manufacturingpractice (GMP) applications.The Sepax instrument usescentrifugation for cellseparation, as specified by aprotocol.

The optical detection tosense the transition of layersbetween the cells that havebeen separated so that thecells can be pulled to theappropriate bag.

Applications includeharvesting of mesenchymalstems cells (MSCs) and Tcells.

The Sefia instrument wasdeveloped to address scaleup to handle larger volumes(up to 10 L) and highercapacities.

It includes sensors forweight, temperature andenables eight fluid pathways.The instrument can beimplemented in continuousflow processes.

Applications for the Sefiasystem include isolation inupstream manufacturingworkflow (PremierCellprotocol) and automatedcell harvest (FlexCellprotocol).

Automated Cell Harvesting Isolation Technologies

Sonia Bulsara (Cytiva) presented the company’s technology for each stage of acell therapy workflow.


See you at

Cell TherapyDigital Week inFebruary 2021

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Cell & Gene Therapy Bioprocessing & Commercialization 2020Post-event Report

Introduction & ContentsRead onWelcome,Jump to any article using the contents on the following page, or at any time using the Contents menu in the top left. There you can also download this report as a PDF.Maribel Rios, Managing Editor at BioProcess International

Contents1. Panels and Plenaries2. Cell Therapy3. In Vivo Gene Therapy4. Gene-Edited Ex Vivo Cell Therapy5. Advanced Tools for Cell and Tissue Manufacturing

Panels & PlenariesRead onInvestment, viral vectors, CAR-T and COVID-19Read onPanels and PlenariesInvestment in the CGT space: Trends, Technologies, and Early Stage Success

"Most allogeneic companies tend to focus on in-house gene editing, but autologous programs can use external production methods for lentivirus."Jak Knowles, VP Venture Investments, Head of Pharma Investments, Leaps by BayerPANEL: COVID-19: Trailblazers in the CGT Industry

"Safety and product quality are not compromised but some risks are taken when multiple tasks must be done in parallel."Raw Material, Viral Vector and Supply Chain Considerations

"Suppliers must either buy reference materials or prepare them in-house when a reference product is not available"Data-Driven Strategies for Downstream Processing of Plasmid DNA In Viral Vector ProductionNon-Gene Edited Approaches to Allogeneic CAR-T Cell TherapyThe Potential of CAR-NKStarting Material Standardization

Characterization is less challenging for noncellular starting materials for gene therapy medicinal products. However, quality and purity of such materials are major concerns."

Cell TherapyRead onAutomated Analytics and Autologous CAR-T Cell TherapyRead onCell TherapyAugmenting Automated Analytics for Cell and Gene Therapy Using Fluorescent Nanosensors

"New measurement technologies are needed to enable subcellular measurements"Risk-Based Approaches for Autologous CAR T-Cell Therapy Comparability

In Vivo Gene TherapyRead onNext Generation Cell-line Development and Recombinant Adenoassociated Virus ProductionRead onIn Vivo Gene TherapyNext Generation Cell Line Development and Large-Scale Bioprocessing Technology

Gene-Edited Ex Vivo Cell TherapyRead onGene Modified Allogeneic MSCs and NK CellsRead onSenti Bio Case Study: Gene Modified Allogeneic MSCs and NK Cells

Gene-Edited Ex Vivo Cell Therapy

Advanced Tools for Cell and Tissue ManufacturingRead onDiverse Tissue Engineering Applications and Automated Cell HarvestingRead onAdvanced Tools for Cell and Tissue ManufacturingDevelopment of Modular Biofabrication Platforms for Diverse Tissue Engineering ApplicationsAutomated Cell Harvesting Isolation Technologies

Cell Therapy Digital Week in February 2021

cell & gene therapy bioprocessing & commercialization 2020 · every year, informa connect invites...

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