JNJ
2nd
Johnson & Johnson
$653,395.82 Mn
271.13
+3.32%
+53.83%
The company develops and commercializes cell therapies, specifically CARVYKTI, which is a CAR T cell therapy used for the treatment of relapsed refractory multiple myeloma.
GILD
2nd
Gilead Sciences
$178,009.04 Mn
143.44
+3.25%
+21.57%
The company commercializes CAR T cell therapies, specifically Yescarta and Tecartus, which are engineered cell therapies used to treat cancer.
BMY
2nd
Bristol Myers Squibb
$134,982.59 Mn
66.08
+2.23%
+37.12%
The company commercializes cell and gene therapies, specifically citing products such as Breyanzi and Abecma, which are engineered cell therapies.
VRTX
2nd
Vertex Pharmaceuticals
$133,815.65 Mn
528.19
+2.45%
+35.48%
The company commercializes CASGEVY, a CRISPR-based gene editing therapy for sickle cell disease and transfusion dependent beta thalassemia, which involves modifying genes to treat disease.
INSM
2nd
Insmed
$28,040.39 Mn
128.46
+0.11%
+0.94%
The company has a dedicated 'Neuro & Other Rare' segment that specifically develops gene therapy programs, such as INS1201 for Duchenne muscular dystrophy and INS1202 for amyotrophic lateral sclerosis.
ROIV
2nd
Roivant Sciences
$27,006.10 Mn
37.37
+3.12%
+216.43%
Through Genevant, the company provides lipid nanoparticle and GalNAc delivery platforms specifically designed to enable the formulation of nucleic acid therapeutics, which are core components of gene and cell therapy.
UTHR
2nd
United Therapeutics
$22,118.12 Mn
518.27
+1.93%
+65.00%
United Therapeutics operates organ manufacturing programs that utilize gene editing for pig kidneys and hearts for xenotransplantation, as well as regenerative medicine using decellularized porcine scaffolds.
NBIX
2nd
Neurocrine Biosciences
$15,669.77 Mn
154.23
-0.17%
+15.84%
The company's research and development pipeline explicitly includes the development of gene therapies targeting various neurological and psychiatric disorders.
BMRN
2nd
Biomarin Pharmaceutical
$12,964.95 Mn
66.99
+0.53%
+16.63%
The company developed ROCTAVIAN for severe hemophilia A, which is a gene therapy designed to modify or replace genetic function, fitting the core platform of genetic modification.
KRYS
Krystal Biotech
$9,850.14 Mn
332.82
-0.87%
+127.49%
Krystal Biotech focuses on the discovery and commercialization of genetic medicines using a patented HSV1-based gene therapy platform. Its lead product, VYJUVEK, is a corrective gene therapy for dystrophic epidermolysis bullosa, and its pipeline includes inhaled and ophthalmic gene therapies.
IBRX
2nd
ImmunityBio
$8,130.87 Mn
7.72
-2.53%
+217.70%
The company has a dedicated NK Cell Therapies segment that develops off-the-shelf and autologous NK-cell therapies, including a CAR NK Platform and an Apheresis Platform (M-ceNK) for engineered cell therapy.
TGTX
2nd
Tg Therapeutics
$7,693.69 Mn
50.26
+1.99%
+81.25%
The company is developing azer cel, an allogeneic CAR T therapy targeting CD19 for autoimmune indications, which involves the use of engineered donor-derived T cells.
CRSP
CRISPR Therapeutics AG
$5,120.13 Mn
52.97
-2.41%
-8.02%
CRISPR Therapeutics focuses on developing therapies that modify genes and cells using CRISPR/Cas9 technology, such as CASGEVY for sickle cell disease. Its core business revolves around gene editing platforms, including in vivo gene editing and CAR T cell therapies.
LEGN
Legend Biotech
$4,247.43 Mn
21.92
+6.15%
-41.47%
Legend Biotech focuses on the development and commercialization of CAR T therapies, specifically CARVYKTI for multiple myeloma, which are engineered cell therapies. The company's core platform involves proprietary llama-derived single-domain antibody technology and dual-binding CAR design to modify cells for oncology treatment.
QURE
uniQure
$3,285.08 Mn
47.38
-1.23%
+202.55%
uniQure is a clinical-stage gene therapy company that uses a proprietary adeno-associated virus (AAV) vector platform to develop treatments for genetic diseases. Its pipeline includes gene-based therapies such as AMT-130 for Huntington's disease and AMT-191 for Fabry disease, and it earns revenue from royalties on the gene therapy HEMGENIX.
IOVA
Iovance Biotherapeutics
$3,184.39 Mn
7.03
-0.99%
+181.20%
Iovance develops and commercializes tumor infiltrating lymphocyte (TIL) cell therapies, specifically its lead product Amtagvi (lifileucel), which is an individualized T cell therapy. The company's core platform is based on cellular modification to harness a patient's own immune system to destroy cancer cells.
BEAM
Beam Therapeutics
$2,743.74 Mn
26.57
-1.96%
+55.38%
Beam Therapeutics focuses on precision genetic medicines using a proprietary base editing technology to modify DNA. The company develops therapies for genetic disorders such as sickle cell disease and alpha 1 antitrypsin deficiency, which fits the core platform of genetic modification described in H-03.
RARE
2nd
Ultragenyx Pharmaceutical
$2,513.31 Mn
25.49
-2.45%
-14.18%
The company has a dedicated pipeline and product modalities focused on adeno-associated virus (AAV) gene therapy and nucleic acid candidates to treat rare genetic diseases.
PGEN
Precigen
$2,416.56 Mn
6.75
+3.05%
+128.81%
Precigen's core business is the development and commercialization of gene and cell therapies, utilizing its proprietary AdenoVerse and Ultra CAR T platforms. Its lead approved product, Papzimeos, and its pipeline targeting cancers (PRGN 2009, 3005, 3006, 3007) are based on genetic and cellular modification.
VCEL
Vericel
$2,264.83 Mn
44.26
+1.54%
+21.86%
Vericel's core business is the development and commercialization of autologous cell therapies, specifically MACI for cartilage repair and Epicel for severe burns. These products involve the modification and application of a patient's own cells, which aligns directly with the Gene and Cell Therapy classification.
MESO
Mesoblast
$2,150.93 Mn
16.67
-2.06%
+4.65%
Mesoblast develops allogeneic cellular medicines using a proprietary mesenchymal lineage cell therapy platform to create off-the-shelf therapies. Its lead product, Ryoncil, is a mesenchymal stromal cell therapy approved for pediatric graft versus host disease.
TSHA
Taysha Gene Therapies
$2,013.62 Mn
6.19
-0.32%
+111.26%
Taysha Gene Therapies is a clinical-stage company developing adeno-associated virus (AAV)-based gene therapies, specifically TSHA-102 for Rett syndrome. Its core platform involves modifying genes to treat monogenic diseases of the central nervous system, which aligns directly with the Gene and Cell Therapy classification.
SRPT
Sarepta Therapeutics
$1,903.34 Mn
18.02
-0.88%
-14.15%
Sarepta's core business is the development and commercialization of genetic modalities, specifically gene therapy as evidenced by its product ELEVIDYS and its use of the AAVrh.74 vector. The company focuses on modifying or replacing genes to treat rare diseases like Duchenne muscular dystrophy.
LKFT
2nd
Lakefront Biotherapeutics
$1,844.46 Mn
27.99
+1.71%
-14.95%
The company specifically develops cell therapy initiatives, including CAR T cell therapies GLPG5101 and GLPG5301, which involve the modification of cells for therapeutic use.
NTLA
Intellia Therapeutics
$1,624.21 Mn
11.60
-4.05%
+8.11%
Intellia Therapeutics is a clinical-stage company focused on developing CRISPR-based therapies, specifically utilizing a proprietary CRISPR/Cas9 platform for in vivo and ex vivo gene editing. Its lead programs, lonvo-z and nex-z, are designed to modify genes to treat hereditary angioedema and transthyretin amyloidosis.
MGTX
MeiraGTx Holdings
$1,308.27 Mn
13.89
+0.87%
+74.28%
MeiraGTx is a clinical stage genetic medicines company developing gene therapies for disorders such as Parkinson's disease and inherited retinal diseases. Its core technology involves the delivery of genetic medicines and a proprietary riboswitch platform for controlling gene expression.
SLDB
Solid Biosciences
$1,304.69 Mn
9.08
-0.22%
+50.58%
Solid Biosciences focuses on developing gene therapy candidates using adeno-associated virus (AAV) to deliver functional genes for diseases like Duchenne muscular dystrophy and Friedreich's ataxia. Its core platform is based on genetic modification and proprietary capsid technology for vector delivery.
IMTX
Immatics
$1,242.51 Mn
9.09
-1.73%
+46.38%
Immatics is a clinical-stage company developing T cell receptor (TCR) based immunotherapies, specifically autologous engineered T cell therapies (ACTengine) and bispecific molecules. Its core platform focuses on genetic and cellular modification to target intracellular cancer antigens in solid tumors.
SANA
Sana Biotechnology
$1,086.48 Mn
3.63
-1.36%
+6.76%
Sana Biotechnology focuses on developing engineered cell therapies, specifically using a hypoimmune technology for stem cell derived islet cells (SC451) and a fusogen platform for in vivo CAR T cell generation (SG293). These activities involve modifying and replacing cells and delivering genetic material, which aligns directly with the Gene and Cell Therapy industry.
FDMT
4D Molecular Therapeutics
$1,034.40 Mn
14.72
+1.17%
+102.48%
The company focuses on developing genetic medicines using a proprietary Therapeutic Vector Evolution platform to create adeno-associated virus (AAV) vectors. Its product candidates, such as 4D-150 for retinal diseases and 4D-710 for cystic fibrosis, are designed to deliver transgenes to modify or replace genetic function.
PCRX
2nd
Pacira BioSciences
$955.74 Mn
24.00
+3.27%
-3.58%
The company is developing a gene therapy pipeline, including the candidate PCRX 201, utilizing a high-capacity adenovirus platform to treat osteoarthritis.
CADL
Candel Therapeutics
$917.23 Mn
12.05
-0.90%
+102.18%
Candel Therapeutics develops viral immunotherapies using engineered adenovirus and herpes simplex virus vectors to modify the tumor microenvironment. Its core platforms, including aglatimagene and linoserpaturev, are based on genetic modification of viruses to induce systemic anti-tumor immunity.
IMMX
Immix Biopharma
$844.84 Mn
11.81
+4.14%
+413.48%
Immix Biopharma is a clinical-stage company developing NXC 201, which is an autologous CAR T (chimeric antigen receptor T cell) therapy. Its core business is based on the N GENIUS platform for cell therapy and genetic modification to treat AL amyloidosis.
AUTL
Autolus Therapeutics
$813.08 Mn
2.29
+0.44%
+21.81%
Autolus Therapeutics develops and commercializes T cell therapies, specifically the CD19-directed chimeric antigen receptor T cell therapy AUCATZYL (obecabtagene autoleucel). Its core business is based on a T cell programming technology platform used to engineer cells for the treatment of cancer and autoimmune diseases.
NGNE
Neurogene
$804.43 Mn
35.67
-2.75%
+74.43%
Neurogene is a clinical-stage company developing genetic medicines, specifically using an adeno-associated virus vector to deliver the MECP2 gene for the treatment of Rett syndrome. Its core EXACT technology focuses on regulating transgene expression, which is a hallmark of gene therapy development.
PROK
Prokidney
$705.51 Mn
1.38
-1.43%
-45.67%
ProKidney is developing rilparencel, an autologous cell therapy that involves expanding a patient's own renal cells outside the body and re-injecting them. This core platform of cellular modification and therapy fits the definition of Gene and Cell Therapy.
ALLO
Allogene Therapeutics
$701.05 Mn
2.03
-2.40%
+86.24%
Allogene Therapeutics develops genetically engineered allogeneic T cell product candidates, specifically CAR T therapies, which involve modifying cells to treat cancer and autoimmune diseases. Its core platform relies on gene editing technologies from Cellectis (TALEN) and Arbor Biotechnologies (CRISPR) to create off-the-shelf cellular therapies.
FBIOP
2nd
Fortress Biotech
$608.26 Mn
18.26
+0.72%
+134.40%
Fortress develops therapies that modify or replace genes and cells, specifically through Mustang Bio's CAR T-cell platforms and Cyprium Therapeutics' AAV-ATP7A gene therapy for Menkes disease.
RGNX
Regenxbio
$599.60 Mn
11.07
-2.29%
+27.10%
REGENXBIO is a clinical-stage company focused on developing gene therapies using adeno-associated virus (AAV) vectors for retinal, neuromuscular, and neurodegenerative diseases. Its core business revolves around the NAV Technology Platform for genetic modification and the development of candidates like sura vec.
BNTC
Benitec Biopharma
$592.10 Mn
12.88
-1.45%
+1.34%
Benitec Biopharma is a clinical-stage company developing BB301, an adeno-associated virus (AAV) based gene therapy. Its core business is based on a 'silence and replace' platform designed to modify or replace genes to treat Oculopharyngeal Muscular Dystrophy.
PRME
Prime Medicine
$551.25 Mn
3.05
-3.79%
-12.86%
Prime Medicine focuses on developing genetic medicines using Prime Editing, a next-generation gene editing technology designed to modify genomic DNA. The company is developing in vivo programs for Wilson disease and alpha 1 antitrypsin deficiency, as well as an ex vivo program for chronic granulomatous disease.
KYTX
Kyverna Therapeutics
$523.35 Mn
8.50
-5.03%
+140.11%
Kyverna Therapeutics focuses on developing cell therapies, specifically its lead product candidate mivcel, which is an autologous CD19 directed chimeric antigen receptor (CAR) T cell therapy. The company's core platform is based on genetic and cellular modification to treat autoimmune diseases like stiff person syndrome and generalized myasthenia gravis.
CABA
Cabaletta Bio
$493.38 Mn
2.91
+0.69%
+64.41%
Cabaletta Bio focuses on developing engineered T cell therapies, specifically chimeric antigen receptor (CAR) T cells, to treat autoimmune diseases. Its lead product candidate, rese cel, is a CAR T construct designed to modify the immune system, which fits the definition of gene and cell therapy.
ARTV
Artiva Biotherapeutics
$460.35 Mn
11.68
-0.51%
+276.77%
Artiva Biotherapeutics is a clinical-stage company developing cell-based therapies, specifically allogeneic off-the-shelf natural killer (NK) cell therapies like AlloNK and CAR-NK programs (AB-201 and AB-205). Its core platform is based on cellular modification and the use of cord blood starting material to treat autoimmune diseases and cancers.
OCGN
Ocugen
$454.32 Mn
1.34
-2.90%
+28.85%
Ocugen's core business is centered on its Modifier Gene Therapy Platform, which uses AAV5 vectors to deliver nuclear hormone receptor genes for treating inherited retinal diseases like retinitis pigmentosa and Stargardt disease. This focus on genetic modification for therapeutic purposes aligns directly with the Gene and Cell Therapy industry.
CAPR
Capricor Therapeutics
$408.27 Mn
7.08
-4.97%
-8.05%
Capricor Therapeutics focuses on the development of cell and exosome based therapeutics, specifically its lead candidate Deramiocel for Duchenne muscular dystrophy. The company's core platform is based on cellular modification and delivery, which aligns with the Gene and Cell Therapy classification.
LXEO
Lexeo Therapeutics
$387.61 Mn
4.93
-1.00%
+0.20%
Lexeo Therapeutics is a clinical-stage genetic medicine company developing gene therapy candidates for cardiovascular diseases and Alzheimer's. Its core platform utilizes the AAVrh10 vector to modify or replace genes to treat conditions like Friedreich ataxia and PKP2 mutations.
LYEL
Lyell Immunopharma
$369.44 Mn
15.79
+16.53%
+45.00%
Lyell Immunopharma is a clinical-stage company developing autologous CAR T cell product candidates, which are engineered cell therapies. Its core platform focuses on genetic and cellular modification to treat hematologic malignancies and solid tumors.
RCKT
Rocket Pharmaceuticals
$365.86 Mn
3.34
-1.76%
+10.23%
Rocket Pharmaceuticals focuses on developing genetic therapies using AAV and lentiviral vectors to treat rare diseases. Its product candidates, such as RP-A501 for Danon disease and KRESLADI (RP-L201) for Leukocyte Adhesion Deficiency-I, are designed to modify or replace genes to correct underlying genetic causes.
IPSC
Century Therapeutics
$353.87 Mn
1.96
+0.51%
+243.86%
Century Therapeutics develops allogeneic pluripotent stem cell derived therapies and uses CRISPR-based gene editing and immune evasion technology to create off-the-shelf cell products. Its core platform is based on genetic and cellular modification for treating autoimmune diseases and cancer.
ABEO
Abeona Therapeutics
$352.51 Mn
6.16
+3.70%
-10.85%
Abeona Therapeutics develops and commercializes cell and gene therapies, specifically its lead approved product ZEVASKYN, an autologous cell-based gene therapy. The company also maintains a pipeline of adeno-associated virus (AAV) based gene therapies using its proprietary AIM™ capsid platform.
AGEN
2nd
Agenus
$320.90 Mn
7.53
+5.31%
+66.23%
The company is involved in the development of adoptive cell therapies through its investment in MiNK Therapeutics, which focuses on cellular modification for treatment.
CLLS
Cellectis
$305.60 Mn
3.04
-2.25%
+4.83%
Cellectis is a clinical-stage company focused on gene editing to create allogeneic CAR T cell products using its proprietary TALEN nuclease platform. Its core business involves modifying T cells to create universal therapies for immuno-oncology, which fits the definition of gene and cell therapy.
IRD
Opus Genetics
$304.97 Mn
3.69
+1.10%
+220.87%
Opus Genetics is primarily focused on developing gene therapies using adeno-associated virus (AAV) platforms to treat inherited retinal diseases, specifically targeting genes such as LCA5, BEST1, and RHO. Its core pipeline consists of seven AAV-based gene therapy programs, which align with the genetic modification focus of H-03.
FATE
Fate Therapeutics
$298.74 Mn
2.56
-2.29%
+113.33%
Fate Therapeutics focuses on creating off-the-shelf cellular immunotherapies using an induced pluripotent stem cell (iPSC) platform to generate engineered T cell and NK cell product candidates. Its core business is the development of therapies that modify and replace cells, specifically targeting autoimmune diseases and solid tumors through CAR T and CAR NK cell candidates.
NWBO
Northwest Biotherapeutics
$289.24 Mn
0.18
+0.00%
-28.00%
Northwest Biotherapeutics develops personalized immune therapies using autologous dendritic cells, specifically DCvax®-L and DCvax®-Direct, which are engineered cell therapies for solid tumor cancers. This core platform of cellular modification and therapy fits the definition of Gene and Cell Therapy.
RNAC
Cartesian Therapeutics
$276.35 Mn
9.20
+0.22%
-18.44%
Cartesian Therapeutics develops cell therapy treatments using a proprietary mRNA-based technology to modify patients' own T cells ex vivo. The company's core platform focuses on engineered cell therapies (CAR-T) for autoimmune diseases like myasthenia gravis and lupus.
LCTX
Lineage Cell Therapeutics
$270.61 Mn
1.07
+0.94%
-16.41%
Lineage Cell Therapeutics specializes in cell replacement therapies, specifically developing allogeneic cell therapies like OpRegen and OPC1 that replace damaged or destroyed cells. Its core business is based on a cell therapy platform using pluripotent stem cells to create mature, differentiated cells for transplantation.
EDIT
Editas Medicine
$268.60 Mn
2.88
+1.41%
+5.49%
Editas Medicine is a gene editing company that develops genomic medicines using CRISPR technology, specifically utilizing Cas9 and Cas12a to edit DNA in vivo and ex vivo. Its core business involves developing therapies for sickle cell disease, beta thalassemia, and hyperlipidemia through genetic modification.
ALEC
2nd
Alector
$246.76 Mn
2.21
+7.80%
+3.76%
The company develops siRNA therapeutics and enzyme replacement therapies, such as AL064 and AL050, which involve modifying or replacing cellular functions and delivering genetic material across the blood-brain barrier.
TARA
Protara Therapeutics
$235.15 Mn
3.98
+0.00%
+23.60%
Protara Therapeutics is a clinical-stage company whose core platform, TARA-002, is described as an investigational cell therapy used for treating non-muscle invasive bladder cancer and lymphatic malformations. This aligns with the H-03 classification for companies developing therapies that modify or replace cells.
DTIL
Precision Biosciences
$217.55 Mn
8.23
+11.22%
+66.26%
Precision Biosciences is a clinical-stage gene editing company that develops in vivo therapeutic candidates using its proprietary ARCUS nuclease platform to create permanent genetic treatments. Its focus on DNA editing for disorders like Duchenne muscular dystrophy and hepatitis B aligns directly with the gene and cell therapy classification.
ZSQR
Z Squared
$204.05 Mn
3.85
-0.26%
-73.72%
The company focuses on acquiring and developing cell therapy technologies, specifically utilizing the SNAP-CAR platform and GEAR technology to engineer natural killer (NK) cells for treating cancer and autoimmune diseases. These activities involve modifying cells to create therapies for hematologic malignancies and solid tumors, which aligns directly with the Gene and Cell Therapy classification.
VYGR
Voyager Therapeutics
$202.20 Mn
3.30
+2.17%
-14.06%
Voyager Therapeutics focuses on developing gene therapies and genetic medicines, specifically utilizing adeno-associated virus (AAV) capsids to deliver therapeutic payloads to the brain. Its core business revolves around the TRACER platform for genetic modification and delivery to treat neurological diseases like Alzheimer's and Parkinson's.
NKTX
Nkarta
$172.62 Mn
2.41
-0.82%
+2.55%
Nkarta is a clinical-stage company developing allogeneic engineered natural killer (NK) cell therapies, specifically its lead candidate NKX019. Its core business is based on a proprietary NK cell engineering platform that modifies cells to express chimeric antigen receptors, which fits the definition of cell therapy.
CRBU
Caribou Biosciences
$165.35 Mn
1.56
-0.64%
-16.58%
Caribou Biosciences focuses on developing allogeneic CAR-T cell therapies, such as vispa-cel and CB-011, which are engineered cell therapies. The company's core platform is its proprietary chRDNA genome-editing technology used for precise genomic edits, including TCR knockout and HLA-E insertion.
HUMA
Humacyte
$161.12 Mn
0.58
+3.57%
-67.78%
Humacyte develops and commercializes bioengineered human tissues, specifically acellular tissue engineered vessels like Symvess, which fall under the category of cell and gene therapy/regenerative medicine. The company's core platform focuses on creating universally implantable tissues that remodel with host cells, a hallmark of cellular modification and regenerative therapy.
TNYA
Tenaya Therapeutics
$156.96 Mn
0.70
+0.00%
-44.44%
Tenaya Therapeutics is a clinical-stage company whose core platform focuses on gene therapy candidates, specifically TN-201 and TN-401, which target genetically defined subpopulations with disease-modifying approaches. Its business model relies on platform collaborations, such as the research agreement with Alnylam Pharmaceuticals, and grants for clinical trials of these gene therapies.
HURA
2nd
TuHURA Biosciences
$137.55 Mn
2.16
-5.26%
-30.10%
The company's lead candidate, IFx-2.0, is an intratumoral plasmid DNA encoding a bacterial protein, which falls under the category of gene therapy as it involves the delivery of genetic material to modify the immune response.
ATRA
Atara Biotherapeutics
$131.09 Mn
8.72
+4.31%
-24.04%
Atara Biotherapeutics develops and commercializes allogeneic T cell therapies, specifically its lead product Ebvallo tab cel, which modifies and utilizes cells to treat cancer and autoimmune diseases. The company's core platform is based on cellular modification and the use of allogeneic T cells, fitting the definition of Gene and Cell Therapy.
ANIX
Anixa Biosciences
$116.36 Mn
3.43
-1.72%
+15.10%
Anixa Biosciences focuses on developing chimeric endocrine receptor-T cell therapy (CAR-T) for ovarian cancer and other solid tumors. This core platform involves engineering T cells to target specific receptors, which falls directly under the modification and replacement of cells described in the Gene and Cell Therapy industry.
ENGN
enGene Therapeutics
$115.22 Mn
1.72
+1.18%
-53.76%
enGene is a clinical-stage company developing non-viral genetic therapies, specifically using its DDX platform to deliver genetic cargos (DNA and RNA) to treat bladder cancer. Its lead candidate, detalimogene voraplasmid, is a genetic medicine designed to modify the immune response in mucosal tissues.
GUTS
2nd
Fractyl Health
$114.23 Mn
0.72
+9.09%
-28.71%
The company is developing Rejuva, a pancreas-directed gene therapy platform that uses an adeno-associated virus vector to reprogram pancreatic islet cells. This activity specifically involves the modification of cells to restore hormone production, fitting the gene and cell therapy classification.
TCRX
TScan Therapeutics
$106.56 Mn
0.82
+5.13%
-54.19%
TScan Therapeutics is a clinical-stage company developing TCR T therapies, which are engineered T cell therapies used to treat cancer and autoimmune disorders. Its core platform focuses on cellular modification, specifically using a non-viral transposon-based manufacturing process to insert TCRs and accessory genes into T cells.
PLYX
2nd
Polaryx Therapeutics
$104.18 Mn
2.09
-0.48%
—
The company employs a multimodal approach that specifically includes the development of gene therapy to address the underlying pathophysiology of rare diseases.
ACET
Adicet Bio
$85.28 Mn
9.11
+4.23%
-21.47%
Adicet Bio is a clinical-stage company developing allogeneic gamma delta T cell therapies engineered with chimeric antigen receptors (CARs). Its core platform focuses on cellular modification to create off-the-shelf products for autoimmune diseases and cancer.
INKT
MiNK Therapeutics
$61.16 Mn
12.00
+6.95%
-17.92%
MiNK Therapeutics develops allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies, such as agenT-797, MiNK-215, and MiNK-413. These products are engineered cell therapies designed to modify the immune response to treat cancer and immune-mediated diseases, which fits the core platform of genetic or cellular modification.
INMB
Inmune Bio
$58.35 Mn
2.11
-0.94%
-16.60%
The company's lead program, CORDStrom, is a pooled human umbilical cord derived mesenchymal stromal cell therapy, and its INKmune platform focuses on natural killer cell priming, both of which are cell-based therapies. These activities align with the H-03 classification for companies developing therapies that modify or replace cells.
APTN
Adaptin Bio
$56.76 Mn
6.27
+0.32%
—
Adaptin Bio focuses on integrating gene therapy and cell therapy to treat brain cancers, specifically through its lead candidate APTN-101 which redirects T cells against tumors. The company's core platform is based on cellular modification and the delivery of therapeutics to the CNS.
MGX
Metagenomi Therapeutics
$45.71 Mn
1.21
-0.82%
-36.98%
Metagenomi Therapeutics is an in vivo genome editing company focusing on curative genetic medicines, utilizing proprietary nucleases and platforms like CAST and RIGS for gene insertion and knockout. Its lead program, MGX-001, specifically employs a gene insertion strategy to treat hemophilia A.
SER
2nd
Serina Therapeutics
$44.24 Mn
2.70
-5.59%
-48.28%
The company's POZ platform is specifically applied to gene therapy and lipid nanoparticle formulations for vaccines and cancer immunology, targeting the delivery of genetic materials.
SGMOQ
Sangamo Therapeutics
$42.03 Mn
0.15
-6.25%
-74.58%
Sangamo Therapeutics is a genomic medicine company that develops therapies modifying or replacing genes, specifically using zinc finger protein technology for epigenetic regulation and AAV capsids for delivery. Its pipeline includes gene therapy candidates for Fabry disease (ISARALGAGENE CIVAPARVOVEC) and hemophilia A (GIROCTOCogene FITELPARVOVEC).
ATNM
2nd
Actinium Pharmaceuticals
$34.90 Mn
1.11
+4.72%
-35.09%
The company develops Iomab-ACT, which is specifically designed as a conditioning agent to improve outcomes for cell and gene therapies, including CAR-T and allogeneic hematopoietic stem cell transplants.
ESLA
Estrella Immunopharma
$31.75 Mn
0.73
-3.95%
-20.61%
Estrella Immunopharma is a clinical-stage company developing T-cell therapies, specifically using its ARTEMIS® T Cell Receptor Platform to engineer T cells for treating cancers. Its lead candidates, EB103 and EB104, are engineered cell therapies targeting antigens like CD19 and CD22, which fits the core definition of gene and cell therapy.
GNTA
Genenta Science S.p.A
$27.42 Mn
1.17
-7.14%
-67.32%
Genenta Science is a clinical-stage company developing gene and cell therapies, specifically using autologous hematopoietic stem cells and lentiviral vectors to treat solid tumors. Its lead product candidate, Temferon, is an ex vivo gene therapy designed to deliver an interferon alpha payload to the tumor microenvironment.
TVGN
Tevogen
$27.02 Mn
6.35
-3.50%
-86.83%
Tevogen Bio Holdings develops allogeneic T cell therapies, such as TGVN 489 and TGVN 920, which are engineered cell therapies designed to modify or replace immune cells. The company's core ExacTcell platform focuses on the selection and expansion of CD8 positive T cells for treating infectious diseases and cancers.
GRML
2nd
Greenland Mines
$24.04 Mn
0.19
-5.00%
-75.84%
The company maintains a proprietary gene therapy platform using the Klotho protein to treat neurodegenerative conditions like Alzheimer's and ALS, with expected revenue from licensing and future product sales.
LGVN
Longeveron
$23.91 Mn
0.79
-3.66%
-2.47%
Longeveron is a clinical-stage company developing laromestrocel, an allogeneic cellular therapy derived from human mesenchymal stem cells. Its core business is the development of regenerative medicines for indications such as hypoplastic left heart syndrome and Alzheimer's disease.
INAB
In8Bio
$22.19 Mn
1.14
+2.70%
-52.30%
In8Bio is a clinical-stage company developing therapies that modify or replace cells, specifically focusing on gamma-delta T cellular therapy product candidates like INB-100 and INB-200. Its core business revolves around the DeltEx platform for genetic engineering and ex vivo expansion of T cells to treat cancer and autoimmune diseases.
NEUP
2nd
Neuphoria Therapeutics
$21.46 Mn
3.99
+3.64%
-48.78%
The company has a partnership with Carina Biotech involving the development of a CAR T therapy, which falls under the gene and cell therapy classification.
CELU
Celularity
$21.34 Mn
0.74
+5.71%
-82.17%
Celularity is a cellular and regenerative medicine company developing off-the-shelf placental-derived allogeneic cell therapies, including natural killer cells and mesenchymal-like stromal cells. Its core platform focuses on genetic and cellular modification for age-related degeneration and immune dysfunction.
MRKR
Marker Therapeutics
$19.20 Mn
1.15
-0.86%
-4.96%
Marker Therapeutics develops T cell based immunotherapies, specifically its Multi Antigen Recognizing (MAR) T cell platform, to treat hematological malignancies and solid tumors. The company's core business is the development of engineered cell therapies, which aligns with the H-03 classification for companies whose core platform is cellular modification.
PLUR
Pluri
$16.06 Mn
1.61
+0.62%
-66.11%
Pluri leverages a proprietary 3D cell-based platform to develop cell therapies, specifically placenta-derived PLX PAD and PLX R18 for regenerative medicine and MAIT cells for immunotherapy. These products are designed to modify or replace cells to treat inflammatory conditions and solid tumors.
PASG
Passage BIO
$14.54 Mn
4.52
-0.44%
-37.40%
Passage Bio is a clinical-stage company developing gene therapies, specifically its lead candidate PBFT02, which is an adeno-associated virus (AAV) based gene replacement therapy. Its core business is the modification of genetic expression to treat neurodegenerative diseases like frontotemporal dementia.
CTXR
2nd
Citius Pharmaceuticals
$12.75 Mn
0.57
+1.79%
-58.70%
The company is developing Nove Cite, which is described as a mesenchymal stem cell therapy for acute respiratory distress syndrome, placing this specific product line in the cell therapy category.
ZEOX
Zeo ScientifiX
$12.07 Mn
1.55
+10.71%
+2.65%
Zeo ScientifiX develops and manufactures biological therapeutics focused on regenerative medicine, specifically Zofin™ (derived from amniotic fluid extracellular vesicles) and Patient Pure X™ (autologous biologic nanoparticles). These products are based on cellular and tissue-derived therapies for orthopedic, wound care, and pain management, which aligns with the core platform of genetic or cellular modification described in H-03.
GDTC
CytoMed Therapeutics
$11.12 Mn
0.94
+3.30%
-47.19%
CytoMed Therapeutics focuses on developing cell-based immunotherapies using induced pluripotent stem cells (iPSCs) and healthy donor blood cells to create off-the-shelf products like CTM N2D iPSC gdNKT. Its core platform is based on cellular modification and the use of innate immune cells to treat cancer and degenerative diseases.
ADIA
Adia Nutrition
$10.38 Mn
0.11
-8.33%
+120.00%
The company's core business involves developing and commercialising therapies that modify or replace cells, specifically providing autologous hematopoietic stem cell transplantation and umbilical cord stem cell treatments through its Adia Med clinics.
SNTI
Senti Biosciences Holdings
$9.67 Mn
0.43
+0.00%
-73.13%
Senti Biosciences is a clinical-stage company focused on creating cell and gene therapies using a proprietary gene circuit platform. Its lead product candidate, SENTI-202, is an off-the-shelf CAR NK cell therapy designed for acute myeloid leukemia.
FBLG
FibroBiologics
$7.59 Mn
1.14
+11.76%
-90.76%
FibroBiologics is a clinical-stage company developing allogeneic cell therapies based on a fibroblast technology platform. Its product candidates, such as CYWC628 and CybroCell™, are designed to modify or replace cells to promote tissue regeneration and modulate immune responses, which fits the core definition of cell therapy.
LNAI
Lunai Bioworks
$6.75 Mn
2.16
-4.00%
-88.33%
The company's Renovaro Biosciences segment is dedicated to creating allogeneic cell and gene therapeutic vaccines, specifically dendritic cell platforms like RENB DC11 and RENB DC20 for cancer treatment.
BCAB
2nd
BioAtla
$5.29 Mn
3.18
+3.92%
-83.63%
The company's protein discovery platform is used to generate CAR-T cells, which are engineered cell therapies used to treat solid tumor cancers.
TPST
Tempest Therapeutics
$5.27 Mn
1.07
-5.31%
-89.47%
Tempest's core platform focuses on developing dual targeting chimeric antigen receptor T cell therapies, including autologous and allogeneic programs like TPST 2003 and TPST 3003. This activity of modifying and replacing cells for therapy directly matches the Gene and Cell Therapy classification.
ERNA
Ernexa Therapeutics
$4.97 Mn
4.25
+4.68%
-89.44%
Ernexa Therapeutics develops off-the-shelf cell therapies using induced pluripotent stem cell derived mesenchymal stem cells (iMSCs). Its product candidates, such as ERNA-101 and ERNA-201, are based on a synthetic iMSC platform for treating cancer and autoimmune diseases.
PMCB
PharmaCyte Biotech
$4.36 Mn
0.52
-3.70%
-48.51%
PharmaCyte Biotech focuses on developing cellular therapies for cancer using its proprietary cellulose-based live cell encapsulation technology. Its lead product candidate, CypCaps™, is specifically designed as a cellular therapy for the treatment of pancreatic cancer.
ISCO
International Stem Cell
$3.92 Mn
0.49
+22.50%
+172.22%
The company is a clinical-stage biotechnology firm developing cell-based treatments for Parkinson's disease, traumatic brain injury, and stroke using a proprietary human parthenogenetic stem cell platform. Its lead program, ISC hpNSC, has already completed a Phase 1 clinical trial.
MBIO
Mustang Bio
$3.84 Mn
0.57
-3.39%
-66.27%
Mustang Bio specializes in the development of CAR T cell therapies, specifically MB-101, which involves engineering T cells to target antigens in glioblastoma tumors. This core platform of cellular modification and engineered cell therapy aligns directly with the Gene and Cell Therapy industry.
RNAZ
2nd
Transcode Therapeutics
$2.97 Mn
3.24
+2.53%
-69.14%
The company's Unleash Oncolytic Immunotherapy Platform involves the development of genetically engineered oncolytic adenoviruses (UIO-524, UIO-525, and UIO-526) to treat muscle-invasive bladder cancer, which falls under the modification of genes and cells for therapy.
GOVX
2nd
GeoVax Labs
$2.97 Mn
0.65
+0.00%
-96.49%
The company develops Gedeptin®, an oncolytic solid tumor gene-directed therapy that uses a replication-deficient adenovirus vector to deliver tumor-specific genes, which falls under the scope of gene and cell therapy.
CASIF
2nd
CASI Pharmaceuticals
$2.88 Mn
0.14
-6.67%
-93.81%
The company commercializes CNCT19, which is a CAR-T cell therapy for relapsed and refractory B-cell acute lymphoblastic leukemia, fitting the description of engineered cell therapies.
BRTX
BioRestorative Therapies
$1.86 Mn
0.21
-4.55%
-86.36%
The company's core business is the development of therapies that modify or replace cells, specifically using adult stem cells for its BRTX-100 disc/spine program and the ThermoStem metabolic program. These programs focus on autologous mesenchymal stem cells and brown adipose derived stem cells to treat degenerative disc disease and obesity.
CLDI
Calidi Biotherapeutics
$1.84 Mn
1.30
-5.11%
-68.29%
Calidi Biotherapeutics develops therapies that modify or replace genes using an engineered enveloped oncolytic virus (RedTail platform) and stem cell encapsulation (SuperNova and NeuroNova). Its core business is the development of genetic medicines for metastatic tumors, which fits the definition of gene and cell therapy.
CELZ
Creative Medical Technology Holdings
$1.44 Mn
1.08
-1.82%
-67.21%
The company develops regenerative therapies using stem cells and immune cells, specifically through platforms like ImmCelz™, AlloStem™, and StemSpine®. Its core business is the modification and application of cellular therapies to treat conditions such as type 1 diabetes and disc degeneration.
GNPX
Genprex
$1.25 Mn
3.95
-11.63%
-58.64%
Genprex is a clinical-stage company developing therapies that modify or replace genes, specifically using the ONCOPREX nonviral lipid nanoparticle system to deliver the TUSC2 tumor suppressor gene and an adeno-associated virus vector to deliver Pdx1 and MafA genes for diabetes. Its core business is the development of gene-based treatments for oncology and diabetes.
RMTG
Regenerative Medical Technology
$0.66 Mn
0.05
-16.67%
+25.00%
The company's core business is the manufacturing and commercialization of cell therapy products, specifically mesenchymal stem cell preparations, exosome isolates, and peptide-based formulations through its Celgenic division. These products are sold to both its own clinic network and external customers.