StemCell Therapy

1Bluebird BioSomerville, MA, USAGene therapies for genetic diseasesZynteglo, Skysona, LyfgeniaPublic (BLUE)Multiple approved gene therapies for rare diseases. Patents (20202024): 15 total (10 gene, 1 vector, 4 cancer)2NovartisBasel, SwitzerlandPioneer: CAR-T (Kymriah), Zolgensma (SMA)Kymriah, ZolgensmaPublicRobust pipeline, global CGT leader. Patents (20202024): 95 total (47 gene, 8 vector, 40 cancer)3Gilead Sciences/Kite PharmaFoster City, CA, USACAR-T therapies for oncologyYescarta, TecartusPublic (GILD)Leading CAR-T franchise in oncology. Patents (20202024): 40 total (6 vector, 34 cancer). 2024 sales data strong: Q2: $521M combined, Q3: ~$485M, steady performance.4Bristol Myers SquibbNew York, NY, USAMultiple CAR-T approvalsAbecma, BreyanziPublic (BMY)Deep CAR-T pipeline, oncology focus. Patents (20202024): 164 total (92 gene, 12 vector, 60 cancer). Q3 2024 total revenue +8% YoY to $11.9B; growth portfolio (incl. Breyanzi, Abecma) +18% to $5.8B.5RocheBasel, SwitzerlandInherited retinal disease gene therapyLuxturnaPublicIntegrated CGT pipeline, global presence. Patents (20202024): 61 total (18 gene, 40 vector, 3 cancer). Elevidys sales Q3 2024: ~30M CHF, exceeding analyst expectations.6PfizerNew York, NY, USAGene therapy in hemophilia B, othersBeqvezPublic (PFE)Partnering in gene therapies, robust R&D. Patents (20202024): 25 total (6 gene, 5 vector, 14 cancer)7BioMarin PharmaceuticalSan Rafael, CA, USARare disease gene therapy (hem A)RoctavianPublic (BMRN)Pioneer in rare disease gene therapy. Patents (20202024): 13 total (13 gene)8Vertex PharmaceuticalsBoston, MA, USAExa-cel for SCD/-thalassemiaCasgevy (Exa-cel)Public (VRTX)Strong CF base, gene editing collaborations. Patents (20202024): 19 total (16 gene, 3 vector)9Sarepta TherapeuticsCambridge, MA, USADMD gene therapyElevidysPublic (SRPT)Leader in RNA/gene for neuromuscular disease. Patents (20202024): 11 total (11 gene)10Adaptimmune TherapeuticsAbingdon, UKEngineered T-cells for solid tumorsTecelraPublic (ADAP)SPEAR T-cell therapies. Patents (20202024): 22 total (3 vector, 19 cancer)11Autolus TherapeuticsLondon, UKNext-gen CAR-TAucatzylPublic (AUTL)Modular CAR-T platforms. Patents (20202024): 45 total (5 gene, 40 cancer)12Krystal BiotechPittsburgh, PA, USAGene therapy for DEBVyjuvekPublic (KRYS)Topical gene therapy for rare skin disorders. Patents (20202024): 9 total (9 gene)13Orchard TherapeuticsLondon, UKEx vivo gene therapies (rare)LenmeldyPublic (ORTX)Lentiviral ex vivo for inherited disorders14Gamida CellBoston, MA, USACell therapy for BMT (Omisirge)OmisirgePublic (GMDA)Improving bone marrow transplant outcomes15EnzyvantDurham, NC, USAAllogeneic processed thymus tissueRethymicPrivate/SubsidiaryThymus-based regenerative medicine16MesoblastMelbourne, AustraliaAllogeneic MSC therapy (GvHD)RyoncilPublic (MESO)MSC platform for inflammatory diseases17Astellas Gene TherapiesBoston, MA, USANeuromuscular & ocular gene therapiesNonePublic (ALPMY)Expanding CNS/ocular pipeline. Patents (20202024): 19 total (14 gene, 5 cancer)18BayerLeverkusen, GermanyHemophilia gene therapy pipelineNonePublicInvested heavily in CGT (AskBio, BlueRock integrated). Patents (20202024): 12 total (11 gene, 1 vector)19Intellia TherapeuticsCambridge, MA, USACRISPR in vivo editingNonePublic (NTLA)First systemic in vivo CRISPR trial. Patents (20202024): 17 total (14 gene, 3 vector)20Beam TherapeuticsCambridge, MA, USABase editing gene therapiesNonePublic (BEAM)Base editing precision. Patents (20202024): 11 total (1 gene, 10 vector)21REGENXBIORockville, MD, USAAAV vector platformNonePublic (RGNX)NAV AAV vectors licensed broadly. Patents (20202024): 10 total (8 gene)22uniQureLexington, MA, USAAAV gene therapiesHemgenix (with CSL Behring)Public (QURE)Hemophilia B gene therapy23Freeline TherapeuticsStevenage, UKAAV for hemophilia & FabryNonePublic (FRLN)Next-gen AAV capsids, metabolic/bleeding disorders24AvrobioCambridge, MA, USALentiviral gene therapies for lysosomal disordersNonePublic (AVRO)Ex vivo lentiviral for metabolic diseases. Funding Status: M&A, Last Funding Date: Feb 1, 2018, $60,000,000 (Series B), Total Funding Amount: $85,000,000, Top Investors: Cormorant Asset Management, Citadel, Atlas Venture, SV Health Investors, Leerink Partners25Rocket PharmaceuticalsNew York, NY, USALentiviral & AAV for rare diseasesNonePublic (RCKT)Hematologic and cardiac gene therapy pipeline26Passage BioPhiladelphia, PA, USACNS AAV gene therapiesNonePublic (PASG)UPenn partnership, rare CNS disorders27Solid BiosciencesCambridge, MA, USADMD gene therapyNonePublic (SLDB)Microdystrophin gene therapy for DMD28Homology MedicinesBedford, MA, USAIn vivo gene editing & gene therapyNonePublic (FIXX)AAVHSC platform for gene correction. Patents (20202024): 8 total (8 gene)29ElevateBioWaltham, MA, USACGT innovation centerNonePrivate ($401M Series D)Enabling platform for multiple CGTs. Funding Status: Late Stage Venture, Last Funding Date: May 24, 2023, Last Funding Amount: $401,000,000 (Series D), Total Funding Amount: $1,246,000,000, Top Investors: Novo Nordisk, Fidelity, Vertex Ventures, Invus, Surveyor Capital30Sana BiotechnologySeattle, WA, USAEx/in vivo CGTNonePublic (SANA)Fusogenix, hypoimmune platforms31Fate TherapeuticsSan Diego, CA, USAiPSC immunotherapiesNonePublic (FATE)Off-the-shelf NK & T-cell therapies. Patents (20202024): 7 total (7 cancer)32Poseida TherapeuticsSan Diego, CA, USAGene editing allogeneic CAR-TNonePublic (PSTX)* Roche is acquiringNon-viral gene editing (piggyBac). Patents (20202024): 20 total (11 gene, 3 vector, 6 cancer)33Caribou BiosciencesBerkeley, CA, USACRISPR-edited cell therapiesNonePublic (CRBU)chRDNAs for precision CRISPR editing. Patents (20202024): 48 total (4 gene, 33 vector, 11 cancer)34CellectisParis, FranceTALEN-based allogeneic CAR-TNonePublic (CLLS)TALEN gene editing, off-the-shelf CAR-T. Patents (20202024): 91 total (12 gene, 20 vector, 59 cancer)35MeiraGTxLondon, UKAAV for ocular & neuroNonePublic (MGTX)Vertically integrated gene therapy company. Patents (20202024): 15 total (10 vector, 5 gene*). Additional Funding Info (for Tmunity Therapeutics Rank #35): M&A, Last Funding Date: Oct 31, 2019, $75,000,000 (Series B), Total Funding Amount: $220,000,000, Top Investors: Gilead Sciences, Andreessen Horowitz, University of Pennsylvania, Kleiner Perkins, Lilly Asia Ventures364D Molecular TherapeuticsEmeryville, CA, USAEngineered AAV vectorsNonePublic (FDMT)Custom AAV capsids for multiple indications. Patents (20202024): 34 total (34 gene)37Abeona TherapeuticsNew York, NY, USARare disease gene therapiesNonePublic (ABEO)Focus on RDEB and CLN138Adverum BiotechnologiesRedwood City, CA, USAAAV ocular gene therapyNonePublic (ADVM)Wet AMD gene therapy. Patents (20202024): 26 total (23 gene, 3 vector)39Generation BioCambridge, MA, USANon-viral genetic medicinesNonePublic (GBIO)Closed-ended DNA platform40Dyno TherapeuticsCambridge, MA, USAAI-driven AAV engineeringNonePrivateAI to optimize AAV capsids41MetagenomiEmeryville, CA, USANovel CRISPR systemsNonePublic (MGX)Metagenomic enzyme discovery for gene editing. Patents (20202024): 11 total (1 gene, 10 vector)42EdiGeneBeijing, ChinaGene editing & therapyNonePrivateCRISPR-based therapies in development. Patents (20202024): 6 total (6 vector)43CelularityFlorham Park, NJ, USAPlacental-derived cell therapiesNonePublic (CELU)Off-the-shelf NK cell therapies44Rubius TherapeuticsCambridge, MA, USARed Cell TherapeuticsNonePublic (RUBY)Engineering RBCs for immuno-oncology. Patents (20202024): 8 total (7 gene, 1 cancer)45ArcellxGermantown, MD, USAControllable CAR-TNonePublic (ACLX)ARC-sparX platform. Patents (20202024): 5 total (5 cancer)46AstraveusParis, FranceCGT manufacturing solutions (with innovation)NoneSeed 10.4MMicrofluidic platform for scalable manufacturing. Funding Status: Seed, Last Funding Date: Oct 24, 2023, 10,400,000 (Grant), Total Funding Amount: 28,872,000, Top Investors: Bpifrance, EASME EU Executive Agency for SMEs, Johnson & Johnson Innovation JJDC, Bpifrance Large Venture, M Ventures47Inceptor BioRaleigh, NC, USACell therapy for tough cancersNoneEarly Stage VentureDiversified cell therapy pipeline. Funding Status: Early Stage Venture, Last Funding Date: Oct 20, 2022, $15,875,000 (Debt Financing), Total Funding Amount: $87,645,000, Top Investors: Kineticos Ventures, Kineticos Disruptor Fund48Tevogen BioWarren, NJ, USAOff-the-shelf T-cell therapiesNonePublic (TVGN)Allogeneic T-cells for viral infections/cancer. Funding Status: IPO, Last Funding Date: May 10, 2024, $36,000,000 (Post-IPO Debt), Total Funding Amount: $58,000,000, Top Investors: HMP Partners49CSL BehringMelbourne, AustraliaGlobal biotech in immunology/heme/CGTNonePublic (CSL)Hemophilia B gene therapy & plasma products50CRISPR TherapeuticsZug, Switzerland & Boston, MACRISPR/Cas9 gene editing therapiesExa-cel (with Vertex)Public (CRSP)Pipeline in hemoglobinopathies, oncology. Patents (20202024): 57 total (46 gene, 6 vector, 5 cancer)51ImmunoACTMumbai, IndiaAdvanced CGT startupNone800M CorporateCAR-T development for emerging markets. Funding Status: Corporate Round (Unlisted as IPO/M&A), Last Funding Date: May 31, 2023, 800,000,000, Total Funding Amount: 1,397,500,000, Top Investors: Laurus Labs52Coave TherapeuticsParis, FranceGene therapies for ocular/CNSNonePrivate (33.1M Series B)AAV-ligand conjugates53Celyad OncologyBelgiumAllogeneic CAR-T for oncologyNonePublic (CYAD)shRNA-based allogeneic CAR-T54Chimeron BioPhiladelphia, PA, USASelf-amplifying RNA therapeuticsNone$4.33M fundingChaESAR RNA platform55Deep GenomicsToronto, CanadaAI-driven RNA therapeuticsNone$241M totalBigRNA AI for RNA drug design56DendreonSeal Beach, CA, USACellular immunotherapy for prostate cancerPROVENGECommercial revenueAutologous cell therapy pioneer57Editas MedicineCambridge, MA, USACRISPR-based gene editing therapiesNone~$931.6M raisedIn vivo & ex vivo CRISPR platform. Patents (20202024): 48 total (21 gene, 26 vector, 1 cancer)58eGenesisCambridge, MA, USAGene-edited xenotransplantationNone$191M Series DMulti-gene-edited porcine organs59EyevensysParis, FranceNon-viral gene therapy for eye diseasesNone$44.1M fundingElectroporation-based ocular gene delivery60Ferring PharmaceuticalsSaint-Prex, SwitzerlandADSTILADRIN for bladder cancerADSTILADRINPrivate; $500M royalty dealGene therapy in oncology61Forte BiosciencesDallas, TX, USAAnti-CD122 mAb for autoimmuneNone$53M private placementTargeting IL-2 pathway disorders62GenascencePalo Alto, CA, USAGene therapy for osteoarthritisNoneFDA Fast Track GNSC-001IL-1Ra gene therapy for OA63GenSight BiologicsParis, FranceGene therapies for retinal diseasesNonePublic (SIGHT.PA)Mitochondrial targeting, optogenetics64Cabaletta BioPhiladelphia, PA, USAT-cell therapies for autoimmune diseaseNonePublic (CABA)CAART for B-cell-mediated conditions65Capricor TherapeuticsSan Diego, CA, USACell & exosome-based therapies (DMD)NonePublic (CAPR)StealthX exosome platform66RegeneronTarrytown, NY, USAExpanding in CGT, advanced biologicsNone (CGT)Public (REGN)Acquired cell therapy programs, DB-OTO gene therapy67RiboKunshan, ChinaRNAi therapeuticsNoneMultiple RMB roundsGalNAc RNAi for liver/CV/metabolic68TakedaTokyo, JapanGlobal pharma investing in CGTNonePublic (TAK)Cell therapy facility, gene therapy collaborations69Ultragenyx PharmaceuticalNovato, CA, USARare & ultra-rare diseases (gene therapy)NonePublic (RARE)Diverse biologics/gene therapy pipeline70VinetiSan Francisco, CA, USAPersonalized therapy management platformNoneSeries A-C fundingSoftware for CGT supply chain71VericelCambridge, MA, USACell therapies for cartilage & burnsMACI, Epicel, NexoBridPublic (VCEL)Strong revenue, expanding indications72Verve TherapeuticsBoston, MA, USAGene editing for cardiovascular diseaseNonePublic (VERV)Base editing for LDL-C/triglycerides73Vivet TherapeuticsParis, FranceGene therapies for metabolic diseasesNonePrivateVTX-801 for Wilson disease (Phase 1/2)74Voyager TherapeuticsCambridge, MA, USAGene therapies for neurological diseasesNonePublic (VYGR)TRACER capsids, partnered CNS programs75Sangamo TherapeuticsBrisbane, CA, USAZinc finger & AAV gene therapiesNonePublic (SGMO)ZFN platform, Fabry, hemophilia programs76SQZ BiotechnologiesWatertown, MA, USACell therapy for HPV16+ tumorsNonePublic (SQZ)AAC & eAPC platforms, strategic restructuring77Arrowhead PharmaceuticalsPasadena, CA, USARNAi therapeutics (cardiometabolic & more)NonePublic (ARWR)SiRNA pipeline, strong partnerships78Alnylam PharmaceuticalsCambridge, MA, USARNAi therapies for various diseasesOnpattro, Givlaari, Oxlumo, AmvuttraPublic (ALNY)RNAi pioneer with multiple approved products. Patents (20202024): 73 total (73 gene)79Aspen NeuroscienceSan Diego, CA, USAAutologous iPSC for Parkinsons diseaseNonePrivate (> $220M raised)iPSC-based personalized cell therapy80American Gene Technologies (AGT)Rockville, MD, USAGene therapy for HIV & rare diseasesNone~$78M fundingHIV functional cure in Phase 181Ionis PharmaceuticalsCarlsbad, CA, USAAntisense therapiesSpinraza, Tegsedi, WaylivraPublic (IONS)Antisense leader, broad pipeline. Patents (20202024): 62 total (60 gene, 2 vector)82Allogene TherapeuticsSouth San Francisco, CA, USAAllogeneic CAR T therapiesNonePublic (ALLO)Off-the-shelf CAR T for hematologic & solid tumors83NexImmuneGaithersburg, MD, USAAIM nanoparticle immunotherapyNonePublic (NEXI)Artificial Immune Modulation platform84NextCureBeltsville, MD, USAImmunomedicines for oncologyNonePublic (NXTC)B7-H4 ADC, immunotherapy pipeline85Oxford BiomedicaOxford, UKVector platform (Lentiviral), integrated innovationNonePublic (LSE:OXB)Leading lentiviral vector developer & collaborator86Pluri Inc.Haifa, Israel3D cell-based technology across industriesNonePublic (PLUR)3D cell expansion platform87ReNeuronBridgend, UKExosome-based therapeuticsNonePublic (RENE.L)CustomEX platform for targeted delivery88Arcturus TherapeuticsSan Diego, CA, USAmRNA medicines & vaccinesNonePublic (ARCT)Self-amplifying mRNA vaccines/therapies. Patents (20202024): 23 total (23 gene)89Silence TherapeuticsLondon, UKRNAi therapeutics targeting liver genesNonePublic (SLN)GalNAc-siRNA pipeline90BioNTechMainz, GermanymRNA-based immunotherapiesComirnaty (COVID-19 vaccine)Public (BNTX)mRNA cancer immunotherapies, global leader. Patents (20202024): 36 total (29 gene, 7 vector)91ModernaCambridge, MA, USAmRNA therapeutics & vaccinesSpikevax (COVID-19 vaccine)Public (MRNA)Expanding mRNA platform to rare diseases. Patents (20202024): 140 total (124 gene, 16 vector)92CureVacTbingen, GermanymRNA therapeutics & vaccinesNonePublic (CVAC)mRNA platform for prophylactic & therapeutic use. Patents (20202024): 65 total (56 gene, 9 vector)93Inovio PharmaceuticalsPlymouth Meeting, PA, USADNA medicines (cancer, infectious diseases)NonePublic (INO)Electroporation delivery of DNA plasmids. Patents (20202024): 28 total (25 gene, 3 vector)94Senti BiosciencesSouth San Francisco, CA, USAGene circuit-engineered cell & gene therapiesNonePublic (SNTI)Programmable gene circuits for cell therapies95Graphite BioSouth San Francisco, CA, USACRISPR gene editing therapiesNonePublic (GRPH)Precise gene correction for genetic diseases96Century TherapeuticsPhiladelphia, PA, USAAllogeneic iPSC-derived cell therapiesNonePublic (IPSC)iPSC platform for NK & T-cell therapies97ArsenalBioSan Francisco, CA, USAProgrammable cell therapies for cancerNonePrivateEngineered T-cells with synthetic biology98Homestead BioPharmaHouston, TX, USAGene therapies targeting AML & cancersNonePrivateEarly-stage gene therapy for oncology99Anew MedicalOmaha, NE, USACGT for cancer/neurologicalNoneIPO $15MDeveloping gene/cell therapies for CNS & cancer. Funding Status: IPO, Last Funding Date: May 26, 2023, $15,000,000 (Post-IPO Debt), Total Funding Amount: $15,000,000, Top Investors: Gaensel Energy Group100AmbuleroMiami, FL, USAVascular gene therapiesNoneSeed $5.5MGene therapies for vascular/ischemic diseases. Funding Status: Seed, Last Funding Date: Jan 25, 2021, $5,500,000 (Seed), Total Funding Amount: $5,500,000, Top Investors: Orphinic Scientific

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100 cell and gene therapy leaders to watch in 2025

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