9/16/2016

BoostingtheImmuneSystemtoEffectRepairsandFightDisease|Insight&Intelligence|GEN

GEN Exclusives
Boosting the Immune
System to Effect
Repairs and Fight
Disease
Applicationsfrom
RegenerativeMedicinetoGene...

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Top 15 Immuno
Oncology
Collaborations

GEN Roundup:
Immunoassays: Life
Science Critical Tools

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Gene Therapy a
Rewrite

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SpenttheMost...

NothingBeats
ImmunoassaysforSensitivity,
Specificity,...

GeneTherapyIs
ImprovingItsStyle...

GEN Exclusives

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Sep12,2016

Market & Tech Analysis

Trends in Various Segments of the
Circulating Biomarkers Space

Boosting the Immune System to Effect Repairs

and Fight Disease

Evolution of the Exosomes Market

Landscape

ApplicationsfromRegenerativeMedicinetoGeneTherapytoAntiviral
TherapeuticsEmphasizeSelfHealing

Epigenetics Market Landscape: A

Qualitative and Quantitative
Picture

SwapnaSupekar

Growing Outsourcing Market for

Discovery Activities

Race to Find Better Tests Fuels

miRNA Detection Market

More

Source:iStock/Ciripasca

Therapeuticinterventionsofvariouskindstrytoimprovethebodyscapacitytodefend,repair,and
evencureitself.Interventionsthatattempttoenhanceselfhealingspancellbasedtherapy,gene
therapy,smallmoleculedrugs,biologics,andtissueengineering.

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BoostingtheImmuneSystemtoEffectRepairsandFightDisease|Insight&Intelligence|GEN

AdvancesineachoftheseareasarebeingfollowedbyAlliedMarketResearch,whichhasconcluded
thatstemcelltechnologieslookespeciallypromising.Forexample,stemcelltechnologiesaresetto
revolutionizethehumanabilitytoproduceneuralcellsinabundance.
Stemcellsmayovercomealimitationofhumantissuesthatisassoberingasitisfamiliar:theretina
andthecentralnervoussystemhaveextremelypoorregenerativecapabilities.Thislimitationseems
allthemoredirewhenonecontemplatestheprevalenceofLebercongenitalamaurosis,agerelated
maculardegeneration,andotherdiseasesthatleadtovisualimpairment.

Interest in Stem Cell Therapy Is Rising

Ifanything,enthusiasmforstemcelltechnologycanbetoogreat.Forexample,earlierintheyear,the
U.S.Food&DrugAdministrationdecidedtotakeacloserlookatclinicsthatprovidestemcell
treatments.TheFDAhopesthatcloserregulationmaynotonlycurbunproventherapies,butalso

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enhanceconfidenceintrulyeffectiveinterventions.

Affordable Care Act

Researchersareexcitedaboutanewstemcellapproachtorepairingheartdamage.Thisapproach,

DoyouagreethattheAffordableCareActhasgone
fartowardaccomplishingitsgoals?

developedbyAmitPatel,M.D.,director,ClinicalRegenerativeMedicine,UniversityofUtah,involves

Yes

theselectiveexpansionofbonemarrowcells.IntheIxCELLDCMtrial(toassesstheefficacyofthe

No

IxomyocelTcelltherapy),whichincludedUtahSenatorRalphOkerlund,researchersobserveda37%

Notsure

reductioninthenumberofhospitalizationsordeathsrelatedtoheartdiseaseandheartfailure.
Anotherpromisingapproachisthepossibilityofcombiningregenerativemedicineandorgan
transplantation.ThisapproachisbeingdevelopedbyMariaCristinaPiaBarba,Ph.D.,aresearcher
attheInstituteofMaterialResearch,NationalAutonomousUniversityofMexico.Herteamis

More

concentratingonblendingdifferentstemcelltechniquestoregeneratethedamagedpartsofvarious
organslikekidneysandheart.ThenumberofkidneysrequiredeachyearinMexicoalonefor
transplantisapproximately12,431.Whilethedemandforheartsisabout58,thereisanincreasing
needfor7,396corneas,3lungs,and404liverseachyearinthecountry.
Onewaytobeginisbydecellularizingbovinebone,saidDr.PiaBarba.Wehavealso
decellularizedtracheas,kidneys,livers,andhearts.Whencalciumisstrippedfromhardbonetissue,
theresultisaspongeofcollagen.Thisspongemaythenbeseededwithstemcells.
Wegive[theseededsponge]theformoftheorganneeded,andintwomonthsthereistissue
formation,continuedDr.PiaBarba."Thisprocessisapossiblesolutiontotrachealdisorders
causedby...cancer,stenosis(abnormalnarrowing)andtraumasthatcanleadtorespiratory
diseasesandevendeath.Theseinnovationswillopennewavenuesforgrowthintheregenerative
medicinemarket.

Gene Therapy Pipeline is Swelling Fast

Anotherfastgrowingsegmentisgenetherapy.Recentresearch,includingapreclinicaltrial,
demonstratestheimportanceofgenetherapyforthesicklecelldisease.Infact,genetherapyis
inspiringsomuchconfidencethatgenetherapywarranteesarebeingcontemplated.Infact,
GlaxoSmithKlinedecidedtoofferamoneybackguaranteeonagenetherapycalledStrimvelis.
GSKannouncedthatifStrimvelisfailedtocureadisordercalledbubbleboydisease,patientswould
berefundedthetotalamount.SarahSpencer,aspokespersonforthedrugmaker,wasquotedby
CNNtosaythatinpricingStrimvelis,GSKreviewedhowbesttomaketheonetimetreatmentcostfor
themedicineaffordabletohealthcaresystems.
GiventheextremelylowpatientnumbersforStrimvelisandthereforelowbudgetimpact,Spencer
continued,manypayershaveindicatedthatStrimvelisislikelytobefundedonacasebycasebasis
anddoesnotwarrantapotentiallycomplicatedpricingarrangement.StartupsincountrieslikeIsrael
arealsoseeninvestingongenetherapytodiscoveracureforcancer.
Thecompetitivelandscapeforgenetherapyalsolooksbright.Themuchawaitedacquisitionofthe
yearwasAllergansinvestmentinRetrosenseTherapeutics,agenetherapycompany.The
investmenthashelpedAllerganaccessanadvancedtechnologyandexpanditseyecarepipeline.
Whilemanycompanyfocusonpossibilities,Novartishasshuttereditsgenetherapyexperimentthat
promisedacureforcancer.

Small Molecules Can Help Cure Deadly Diseases

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BoostingtheImmuneSystemtoEffectRepairsandFightDisease|Insight&Intelligence|GEN

DeadlyviraldiseasessuchasEbolahavebeenhardtotreat.Butscientistsseearayofhopeinsmall
moleculesthatcanblockandpreventthespreadofviruses.Forexample,acompoundhasbeen
foundthattargetsEbolasAchillesheel.ThecompoundcanblocktheproteinusedbyEbolatobreak
outofthecellsandfurtherinfectthenewcells.
Suchinnovationsareencouragingmanynewentrantstofindnewwaystoharnesstheentireimmune
systemandfightdeadlydiseases.Severalstudieshighlightingmalariasvulnerabilitieshavegot
stakeholdersinterestedinapproachesthatcanreinforcetheimmunesystem,oratleastgiveit
breathingspace.
Earlierstudiesdemonstratedthatsinglemoleculedrugscancommandstemcellstoconstructnew
bones.Thesestudiesshowhowmedicalinterventionscantreatdebilitatingdiseasessuchas
orthopedicdisorders,cardiovasculardiseases,andothers.Moreover,therisingprevalenceofjoint
disordersand,consequently,anincreaseinthenumberofcomplicatedorthopedicsurgerieshas
alsoresultedinthegrowthoftheregenerativemedicinesmarketworldwide.

EXCLUSIVES

Amino Acids in
Goingforward,wewillnotonlyseebigbrandsinvestgenerouslyonregenerativemedicinemarket,
Cancer Metabolism

and Neu...
butwewillalsoseemanystartupsflourish.

Genetic
Engineering & Biotechnology
News
NEWS
THE LISTS
Bayer to Acquire
Monsanto for $66B

Top 20 Biopharma
IPOs of January
June 20...
SwapnaSupekar(swapna.supekar@alliedmarketresearch.com)isaseniordigitalmarketingexecutiveatAlliedMarket
Research.AlliedMarketResearchrecentlyreleasedanewreport,RegenerativeMedicineMarketbyTechnology(Cell

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Therapy,GeneTherapy,TissueEngineering,SmallMoleculesandBiologics),Material(BiodegradableSynthetic
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