《Evaluate Pharma：2024細胞與基因療法市場格局研究報告：機遇及挑戰（英文版）（26頁）.pdf》由會員分享，可在線閱讀，更多相關《Evaluate Pharma：2024細胞與基因療法市場格局研究報告：機遇及挑戰（英文版）（26頁）.pdf（26頁珍藏版）》請在三個皮匠報告上搜索。

1、Click here or press enter for the accessibility optimised versionCell&GeneTherapies CI PerspectivePaul DSouza-Bento and Alex BourClick here or press enter for the accessibility optimised versionKey Takeawaysfor CI TeamsKey Takeaways for CI TeamsThe landscape for cell and gene therapies has been evol

2、ving over the past few yearswith a rise in available treatments for rare and life-threatening diseases in the lastyear alone.This is despite generally high costs of R&D and manufacturing,coupledwith the infancy of scientific understanding compared to other areas of science.Despite the increasing vol

3、ume of gene therapy assets targeting hematological malignancies,significantmarket potential exists,driven by the complexities in treatment-related adverse events among approvedproducts opportunities for further differentiation thus exists for novel therapies.As cell and gene therapies move into indi

4、cations with larger disease burdens,manufacturers will have amyriad of factors to consider,including the holistic value of a potentially curative therapy on already strainedhealthcare systems,payer price-sensitivity,and a potentially depleting patient population.The curative potential of cell and ge

5、ne therapies presents paradigm-shifting opportunities and challenges tothe biopharma industry,requiring CI teams with deep therapy area knowledge and access to robustsecondary and primary insights to support critical strategic decision-making at key inflection points.Click here or press enter for th

6、e accessibility optimised versionGene TherapyLandscapeGene Therapy LandscapeSince 2004,32 gene therapies,including genetically modified cell therapies,have been registered covering oncology indications,including non-Hodgkinslymphoma(NHL),acute lymphocytic leukaemia(ALL),and chronic lymphocytic leuka

7、emia(CLL).Key players have mainly been top pharma,althoughBluebird Bio biotech tops the list with 4 assets(Figure 1 and Table 1).Figure 1:Top players(by number of approved gene therapies)Source:PharmaprojectsTable 1:Most prevalent diseases targeted by approved gene therapiesSource:PharmaprojectsEigh

8、t of these treatments had been approved for the first time since the beginning of 2023 alone for a range of oncology and rare indications(Table 2).CRISPR Therapeutics/Vertexs Casgevy became the first CRISPR-edited therapy approved in November 2023,specifically for sickle cell anaemia andtransfusion-

9、dependent beta thalassemia.The approach consists of genetically-modified autologous CD34+cells containing haematopoietic stem andprogenitor cells edited ex vivo by CRISPR gene editing technology.Another treatment for sickle cell anaemia,Lyfgenia,provides a functional beta-globingene to a patients ow

10、n haematopoetic stem cells,was approved in December 2023.Table 2:Gene therapies approved since the beginning of 2023Source:PharmaprojectsThe pipeline stage is also progressing with 2,092 assets in preclinical pre-registration.Within late-stage(Phase 3 Pre-registration),38 candidates havebeen identif

11、ied with smaller players Regenxbio and Ultragenyx Pharmaceutical leading the way with 4 assets each,whilst Novartis and Pfizer have a couplea piece.Most prevalent indications include solid cancer,bladder,and brain cancer.Figure 2:Top players(by number of gene therapies inPhase 3 pre-registration)Sou

12、rce:PharmaprojectsOverall,a combination of both large pharma and biotech top the list of players with the highest volume of gene therapies,with Roche capping the charts with28 assets,followed by AstraZeneca and Sarepta with 21 each(Figure 3).With approved treatments under their belt,Bluebird and CAR

13、sgen also havesignificantly sized portfolios.The majority of diseases included solid cancer,myeloma,acute lymphocytic leukaemia and non-Hodgkins lymphoma(Table 3).Figure 3:Top players(by number of gene therapies(preclinical approved)Source:PharmaprojectsTable 3:Most prevalent diseases targeted by ge

14、ne therapies(preclinical approved)Source:PharmaprojectsClick here or press enter for the accessibility optimised versionCell TherapyLandscapeCell Therapy LandscapeIn contrast to gene therapies,there are currently 74 cell therapies approved,excluding gene-modified cell therapies,treating conditions s

15、uch as burns,orthopaedic lesions and osteoarthritis.Key players are mainly non-top pharma or biotech,including Smith&Nephew,Teijin,and Bluebird Bio,with GSK andSanofi having three registered treatments each(Figure 4 and Table 4).Figure 4:Top players(by number of approved cell therapies,excludinggene

16、-modified cell therapies)Source:PharmaprojectsTable 4:Most prevalent diseases targeted by approved cell therapies,excluding gene-modified cell therapiesSource:PharmaprojectsSince 2023,4 non-modified cell therapies have received first approval targeting various diseases(Table 5).In June 2023,Lantidra

17、,a treatment consisting ofallogenic pancreatic cells derived from deceased patients,was approved becoming the first ever cell therapy for type 1 diabetes,potentially removing theneed for insulin.Amtagvi became the first approved cell treatment for advanced melanoma,in March 2024.The approach involve

18、s use of tumour-infiltrating lymphocytes derived from a tumour sample,strengthened,and infused back into the patient.Table 5:Non-modified cell therapies approved since the beginning of 2023Source:PharmaprojectsThe number of cell therapies in the pipeline is around half of that for gene therapies,wit

19、h 1,323 in preclinical pre-registration,however,there are morecandidates(59)in late-stage(Phase 3 or pre-filing),predominantly led by non-large pharma(2 to 3 assets each).In contrast to gene therapies,topindications focus on non-oncology diseases such as COVID-19,osteoarthritis,heart,and respiratory

20、 conditions(Figure 5 and Table 6).Figure 5:Top players(by number of non-modified cell therapies in late-stage development(Phase 3 pre-registration)Source:PharmaprojectsTable 6:Most prevalent diseases targeted by non-modified cell therapiesin late-stage development(Phase 3 pre-registration)Source:Pha

21、rmaprojectsAstellas and Takeda have an active portfolio with 14 and 11 cell therapies,respectively,although top competitors are predominantly biotech,includingAnocca(15),Aavocyte,and Adaptimmune(12 each),whilst oncology once again dominates the disease areas of focus,including solid cancer,and NSCLC

22、,although osteoarthritis and type I disease feature predominantly(Table 7 and Figure 6).Figure 6:Top players(by number of non-modified therapies(preclinical approved)Source:PharmaprojectsTable 7:Most prevalent diseases targeted by non-modified cell therapies(preclinical approved)Source:Pharmaproject

23、sClick here or press enter for the accessibility optimised versionGene and Non-modified CellTherapiesGene and Non-modified Cell TherapiesInternal innovation engines established by top pharmaExpertise and innovation in emerging fields of science such as cell and gene therapies had been predominantlya

24、ssociated with biotech,however,more and more of top pharma are investing in developing their internal capabilitieseither organically or inorganically.Novartis Gene Therapies focuses on AAV(adeno-associated virus)-based therapiesthat deliver functional genes into human cells for neurology and ophthal

25、mology and CRISPR-based technologies thatmanipulate DNA for haematology and ophthalmology.Set up in 2013 and bolstered by the$8.7bn acquisition ofAveXis and spinal muscular neuron gene therapy,Zolgensma for spinal muscular atrophy in 2018,further significantinvestments were made including a$3bn lice

26、nse for Voyager Therapeutics AAV capsids.Roche has its PharmaTechnical Cell&Gene Therapy unit and has made large investments in the space,including a$4.8bn acquisition ofSpark Therapeutics in 2019 with Luxturna,a one-time product,in approved for retinal dystrophy,a rare form ofblindness,being a key

27、part of the deal.Roche has also utilised partnerships to bulk up its capabilities in neoantigen-targeting T-cells for oncology(Adaptive Biotechnologies),engineered T-cells for solid tumours(Arsenal Biosciences),CAR-T therapy for haematological cancer(Poseida Therapeutics),allogeneic approaches start

28、 with inducedpluripotent stem cells(Adaptimmune),and Regenerative cell therapies for eye diseases(Lineage Cell Therapeutics).Click here or press enter for the accessibility optimised versionDealsTrendsDeals TrendsAppetite for partnerships around cell and genetherapies has varied over the past few ye

29、ars.Genetherapies had attracted around$14.5bn deal dollarsand 23 deals in 2021 across a variety of therapy areas,with Takeda showing hunger with a$2.7bn licensewith Poseida Therapeutics to develop gene therapiesfor liver-and hematopoietic stem cell(HSC)-directedindications and CSLs$2.1bn license to

30、uniQuresFactor IX gene therapy,Hemgenix for haemophilia B.Deal volume dropped to eight in 2022,while total dealvalue reduced to$4.7bn,although sizeable deals werestill made with Novartiss partnering with VoyagerTherapeutics for$1.8bn to use its adeno-associatedvirus(AAV)capsid discovery platform to

31、developgene therapies against CNS diseases.Voyager was atthe heart of 2023 once again,outlicensing its GBA1gene therapy programme for Parkinsons disease andthree gene therapy programmes targeting rare CNStargets for$1.7bn.Deal volume remained aroundseven with an increase in dollars spent to$8.7bn.Fi

32、gure 7:Total deal dollars spent in licenses and M&A for gene therapies(2021 2023)Source:Evaluate PharmaIn contrast to gene therapies,oncology has been thepriority for gene-modified therapies,with almost noother therapy area to call out(Figure 8).In 2022,therewas a significant$18bn spent across eight

33、 dealslargely driven by Roches$6.2bn license of PoseidaTherapeutics CAR-T technology for haematologicalmalignancies and BMSs$3.1bn license to CenturyTherapeutics chimeric antigen receptor naturalkiller(CAR-NK)cell therapy,CNTY-104,for acutemyeloid leukaemia.However,2023 witnessed asignificant drop i

34、n investment to$2.3bn for a similardeal volume,with the largest deal valued at just over$500m;Amgens use of TScan Therapeutics targetdiscovery platform for T-cell therapies targetingCrohns disease.Although,2024 shows a potentialrevival in multi-billion dollar partnerships with AbbVieinvesting 1.4bn

35、in biotech Umojoa Biopharma toleverage its lentiviral gene delivery technology and thedevelopment of up to five CAR-T cell therapies,including UB-VV11 which is in Phase 1 forhaematological malignancies.Figure 8:Total deal dollars spent in licenses and M&A for gene-modified therapies(2021 2024)Source

36、:Evaluate PharmaInvestment in partnerships within the cell therapyspace has been fairly flat with total deal dollarshovering between$4bn and$6.8bn over the past fewyears.2022 saw$6.8bn spent across 10 deals withRoche partnering with Jnana Therapeutics for$2bn toleverage its tsRAPID chemoproteomics p

37、latform todiscover novel small molecule drugs targeting cancer,immune-mediated and neurological diseases.AstraZeneca led 2023 spending$2bn for QuellTherapeutics to use its multi-modular T-regulatory cellengineering platform to develop treatments for T1diabetes and inflammatory bowel disease.Six deal

38、swere formed with$4.5bn spent.Figure 9:Total deal dollars spent in licenses and M&A for cell therapies(2021 2024)Source:Evaluate PharmaClick here or press enter for the accessibility optimised versionFuture RevenueDriversCAR T-cell therapies are projected to be amongthe highest revenue drivers in th

39、e gene therapyclass in 2028 with large pharma topping the list.The multiple myeloma market is expected to seesignificant revenue,including$4.7B from J&JsCarvytki and BMS Abecma also a keycontributor.Non-Hodgkins lymphoma is alsoexpected to be a key disease area with$2.4bnand$640m forecasted from Gil

40、eads Yescartaand Tecartus,respectively,and$1.9bn fromBMS Breyanzi.Outside of CAR T-cells and oncology,genetargeting therapies are also expected to be keyrevenue drivers in 2028.Duchennes musculardystrophy treatments Elevidysand PF-06939926(both dystrophin genetargeting)are forecasted to return just

41、over$3bn for Sarepta and its partner Roche,and$383M for Pfizer,respectively.Novartiscurrently available treatment Zolgensma(survival motor neurone gene)has$1.7bnprojected for spinal muscular atrophy,whileKrystal Biotechs Vyjuvek for epidermolysisbullosa is expected to pull in$643m.$515m forRocket Ph

42、armaceuticals RP-A501(lysosome-associated membrane glycoprotein 2 genetransference)in cardiovascular disease.Haematology seems to be a popular therapyarea with$971m expected forBioMarins Roctavian in haemophilia A,and$412m for Rockets RP-L102(FAcomplementation group A GENE)for anaemia.Non geneticall

43、y-modified cell therapies,particularly T-cells and stem cells,are expectedto generate significant revenue in 2028 forsmaller players.Iovance Biotherapeutics,aDutch-based biotech with its HQ in the US,hastwo anti-PD-1,tumour-infiltrating lymphocyte(TIL)T-cell therapies Amtagvi,approved formelanoma an

44、d LN-145,in Phase 2 for cervicalcancer and NSCLC,which are expected togenerate$572m and$387m,respectively.Another T-cell therapy,commercial-stagebiotechs Immunocores IMC-F106C,that targetsa specific antigen associated with melanoma,preferentially expressed antigen of melanoma(PRAME),is expected to y

45、ield$313m formelanoma.Mesenchymal stem cells areexpected to be large revenue drivers forMesoblast with Revascor and MPC-06-D andRyoncil expected to yield a total of just over$1bn for coronary heart failure,lower back painand graft-vs-host disease,respectively.Brainstorm also has NurOwn,a mesenchymal

46、bone marrow stromal stem cell therapy foramyotrophic lateral sclerosis and primary-progressive multiple sclerosis,forecasted togenerate around$350m.Future Revenue DriversClick here or press enter for the accessibility optimised versionConsultantsViewThe number of cell&gene therapy(C>)-basedtreatme

47、nts in development have increased significantlyover the last two decades and can be expected tocontinue,driven by the modality-specific marketpotential and the breadth of applicability acrossindications.Although early innovations in C> have beenspearheaded by small biotechs,Big Pharma quicklyfollo

48、wed suit with major deals to jumpstart their own in-house programmes.Whilst this means that deal volumein the space may slow in the medium-to-long term,theirvalue may continue to increase as Big Pharmas searchfor high value disease portfolio lynchpins also continues.Big Pharmas competitive edge over

49、 biotech marketentrants will be most evident in how it deploys its in-house capacity and capabilities to accelerate time tomarket or acquire complementary therapies tostrategically bolster its portfolios.New biotech entrants to the C> space thereforeneed to remain vigilant of Big Pharma and other

50、biotechcompetitors alike,but for different reasons.Otherbiotech companies pose an inherent threat as they seekto manage their relatively smaller budgets to maximumvalue.Biotech C> companies may benefit fromtracking competitors earnings calls and generalcorporate communications,senior/executive hir

51、es andoverall headcount,and funding plus general deal-makingactivities.Monitoring Big Pharma deal trends andappetite for specific C> modalities will also providevaluable directional intelligence on which fellowbiotechs might be next up for a landscape-shifting deal.Big Pharma players in C> face

52、identical CI challenges,but often with the additional need to monitorcompetitors portfolios where there is significant overlapin pipeline and portfolio strategies.In addition tocompetitive intelligence tracking,Big Pharma playersmay also benefit from monitoring for businessdevelopment purposes,ensur

53、ing regular updates on keylandscape events and catalysts to inform potential M&Aactivities.As the cell and gene therapy space continues to growand expand,so will the need for a well-organised andwell-resourced CI function to ensure successful andsustainable growth for Big Pharma and small biotechpla

54、yers alike.Consultants ViewBY ALEX BOURClick here or press enter for the accessibility optimised versionAbout Evaluates CIConsulting PracticeYour Tech-Enabled Partner inCompetitive IntelligenceStrategic competitive intelligence(CI)in the pharma industry is a crucial but time consuming and complex ta

55、sk.Tracking R&D and marketed productsacross multiple indications,monitoring competing companies and assets,and identifying gaps in the market require a combination of data,intelligence,insight and therapy area expertise.Evaluates CI practice provides you with a technology-enabled extension of your t

56、eam to ensure your team are free tofocus on the areas that will achieve your strategic goals.How we helpExpert-Driven CI InsightsOur team of 70+consultants and analysts areexperienced in a wide range of therapy areas from oncology to vaccines to cell and genetherapies and beyond.Their expertise ensu

57、reswe provide you with timely,accurate andactionable insights,supported bycomprehensive data from across the widerNorstella group.Tech-enabled SolutionsOur customisable,tech-enabled analyticscombine key CI data points to release CI teamsfrom the burden of manual reporting.Weprovide best in class ins

58、ights based on the mostrobust data sets overlaid with domainexpertise from our consultants.Collaborative Partnership ModelAs your needs evolve,our team is on-hand toexpand coverage,develop customisedvisualisations and deliver strategic insights thatanswer the questions that keep your executivesawake

59、 at night.Never prescriptive,we work tomeet you where you are providing an outsideperspective on your market,to strengthen yourstrategic decision-making.Click here or press enter for the accessibility optimised versionThank you for readingCell&Gene Therapies CI PerspectiveCookies Terms Privacy 1 2 P O W E R E D B Y