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The top 100 cell and gene therapy companies to watch in 2023

By Brian Buntz | March 10, 2023

Pharma 50: Cell and Gene Therapy CompaniesThe cell and gene therapy sector is poised to deliver a wave of new therapies with the potential to cure rare and common diseases. As many as 13 new cell or gene therapies could be approved for use in the U.S., Europe, or both by the end of 2023.

While manufacturing and regulatory challenges remain, the cell and gene therapy industry continues to offer significant therapeutic potential and commercial opportunities.

We have beefed up our 2022 list of leading cell and gene therapy companies, doubling the number of organizations from 50 to 100.

In selecting the companies for the latest list, we weighed public companies’ degree of involvement in cell and gene therapy, focusing primarily on including drug developers specializing in the area.

For private companies, we factored in their funding levels, pipeline and degree of history for the list.

Because the field of cell and gene therapy remains relatively small, this list is a fairly comprehensive overview of companies active in the industry.

We have a separate list of cell and gene therapy vendors and suppliers here.

Company Headquarters Description Product/pipeline examples
1 4D Molecular Therapeutics Emeryville, California Focuses on developing targeted and customizable adeno-associated virus (AAV) vectors for gene therapy. 4D-125, 4D-110, 4D-310
2 Abeona Therapeutics New York City Develops gene and cell therapies for rare genetic diseases. ADVM-022, EB-101, ABO-102 (AAV-SGSH), ABO-101 (AAV-NAGLU)
3 Adaptimmune Therapeutics Abingdon, UK Pipeline includes SPEAR T-cell therapies for solid tumors. Afamitresgene autoleucel or “afami-cel”, ADP-A2M4CD8 SPEAR T-cell therapy
4 Adverum Biotechnologies Redwood City, California Developing ADVM-022, a gene therapy for wet AMD, a leading cause of blindness in the elderly. ADVM-022, ADVM-062
5 Allogene Therapeutics South San Francisco, California Specializes in developing allogeneic CAR-T products for cancer ALLO-501, ALLO-715
6 Alnylam Cambridge, Massachusetts Develops RNA interference (RNAi) therapies for rare genetic diseases. Onpattro (patisiran), Givlaari (givosiran), Oxlumo (lumasiran)
7 Altor BioScience Miramar, Florida Altor has a pipeline of immunotherapies for cancer and infectious diseases. Develops cell and gene therapies for cancer and inflammatory diseases
8 American Gene Technologies Rockville, Maryland This gene therapy company specializes in developing treatments for HIV and genetic diseases. AGT103-T, AGT103-iNKT
9 Amgen Thousand Oaks, California Amgen’s gene therapy and cell therapy products focus on oncology and rare diseases. AMG 510, AMG 330, AMG 596, AMG 757, AMG 673, AMG 119, AMG 592
10 Arcellx Germantown, Maryland Arcellx specializes in developing ARC T-cell therapies for cancer. ACLX-001 and ACLX-002
11 ArrowHead Pharmaceuticals Pasadena, California ArrowHead focuses on developing RNA interference (RNAi) therapies. ANPDR001, ARO-AAT
12 Aspen Neuroscience San Diego, California Aspen focuses on developing gene therapies for neurodegenerative diseases such as Parkinson’s disease. AAV-based gene therapies
13 Astellas Gene Therapies Boston, Massachusetts This clinical-stage biotechnology company focuses on developing gene therapies for rare genetic diseases. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271
14 AstraZeneca/Alexion Boston, Massachusetts Develops gene therapies for rare diseases such as metabolic disorders and lysosomal storage disorders. LentiGlobin, MEDI9197, MEDI9198
15 Atara Biotherapeutics South San Francisco, California Focuses on developing T-cell immunotherapies for cancer and autoimmune diseases. ATA188, ATA2271, ATA3271, ATA3219
16 Autolus Therapeutics London Autolus specializes in developing CAR-T cell therapies for the treatment of cancer. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8
17 AVROBIO Cambridge, Massachusetts Focuses on developing lentiviral-based gene therapies for rare diseases. AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05
18 Axovant Gene Therapies New York City Develops gene therapies for neurological and neuromuscular diseases such as Parkinson’s and Tay-Sachs disease. AXO-Lenti-PD, AXO-AAV-GM1, AXO-AAV-GM2, AXO-AAV-OP
19 Batavia Biosciences Leiden, Netherlands Provides viral vector and mammalian cell line technologies for the development of biopharmaceuticals and vaccines. Viral vector and mammalian cell line technologies
20 Bayer Leverkusen, Germany Bayer’s gene therapy efforts focus on hemophilia and other rare genetic diseases. It has also developed a manufacturing platform for AAV vectors. BAY 2599023, BAY 2599023 + emicizumab, BAY 2599023 + BMN 307, BAY 2599023 + etranacogene dezaparvovec
21 Bean Therapeutics Boston, Massachusetts Develops genetic medicines for inherited diseases such as PKU and Friedreich’s ataxia. Development of genetic medicines
22 BioMarin Pharmaceutical San Rafael, California BioMarin develops therapies for rare genetic diseases such as PKU, MPS I, MPS IIIB, Batten disease and hemophilia. Brineura, Kuvan, Naglazyme, Palynziq, Vimizim
23 Bluebird Bio Cambridge, Massachusetts Develops gene therapies for genetic diseases such as sickle cell anemia, beta-thalassemia and cerebral adrenoleukodystrophy. Zynteglo, LentiGlobin
24 BlueRock Therapeutics Cambridge, Massachusetts BlueRock specializes in developing induced pluripotent stem cell (iPSC) therapies for cardiovascular, neurology and immunology. Induced Pluripotent Stem Cell (iPSC) Technology
25 Bristol-Myers Squibb New York City Uses gene editing and cell engineering technologies to develop next-gen immunotherapies for cancer. BMS-986249, BMS-986253, BMS-986258, Liso-cel (JCAR017), bb21217, bb21222
26 Cabaletta Bio Philadelphia Develops gene therapies for rare autoimmune diseases such as mucosal pemphigus vulgaris and muco-cutaneous pemphigus. Engineered Autologous T-cell therapy
27 Capricor Therapeutics Los Angeles Capricor develops cell and gene therapies for heart disease. CAP-1002, CAP-1001, CAP-2003, CAP-3001
28 Caribou Biosciences Berkeley, California Develops gene editing technologies using CRISPR/Cas9 with a focus on developing therapies for genetic diseases. Pipeline of off-the-shelf genome-edited CAR-T and CAR-NK cell therapies
29 Celgene (now Bristol-Myers Squibb) Summit, New Jersey This Bristol Myers Squibb company has developed a CAR-T cell therapy that recognizes the B-cell maturation antigen (BCMA) and kills BCMA-expressing tumor cells. Abecma (idecabtagene vicleucel)
30 Cellectis Paris Focuses on developing immunotherapies based on gene-edited allogeneic CAR T-cells. UCART19, UCART22, UCART123, ALLO-501, ALLO-715
31 Celularity Florham Park, New Jersey Celularity develops allogeneic placental-derived cells for use in cellular therapeutics. CYNK-001, CYNK-101, COVID-specific immune cells
32 Celyad Oncology Mont-Saint-Guibert, Belgium Develops chimeric antigen receptor T cell (CAR T) therapies for cancer. CYAD-101, CYAD-211, CYAD-02
33 Chimeron Bio Foster City, California Focuses on developing gene therapies for rare diseases. CB-101, ChaESAR RNA therapy
34 Coave Therapeutics San Diego, California Develops targeted, non-viral gene therapies using its proprietary nanoparticle platform. Preclinical programs for inherited retinal diseases and cancer immunotherapies
35 CRISPR Therapeutics Zug, Switzerland A gene-editing company specialized in developing transformative gene-based medicines for serious diseases. CTX001 and therapies for blood stem cell, immuno-oncology and regenerative medicine
36 CSL Behring King of Prussia, Pennsylvania Focuses on adeno-associated virus (AAV)-based gene therapy and stem-cell gene therapies for primary immunodeficiency diseases. Hemgenix (etranacogene dezaparvovec)
37 Deep Genomics Toronto, Canada AI drug development company’s focus includes the discovery and development of genetic therapies. AI Workbench for RNA biology and discovery of novel genetic therapies
38 Dendreon Seal Beach, California Develops immune-oncology therapies for the treatment of cancer. PROVENGE (sipuleucel-T) for prostate cancer
39 Dyno Therapeutics Cambridge, Massachusetts Develops novel gene therapies with its proprietary AI-powered platform. AAV vectors for gene therapies
40 Editas Medicine Cambridge, Massachusetts The company develops CRISPR-based therapies for genetic diseases. EDIT-101, EDIT-301, EDIT-201
41 eGenesis Cambridge, Massachusetts The company’s lead product is a genetically modified pig kidney that is intended for use in humans. Porcine organs for human transplantation
42 ElevateBio Cambridge, Massachusetts The firm’s subsidiaries include AlloVir, a cell therapy company and HighPassBio, a gene therapy company. AlloVir, HighPassBio, RegenxBio and others
43 Enzyvant Durham, North Carolina Company scored FDA approval for Rethymic, is a regenerative tissue therapy for the ultra-rare condition of congenital athymia. Rethymic (allogeneic processed thymus tissue-agdc)
44 Eyevensys Paris, France Specializes in electro-transfection technology for the sustained intraocular production of therapeutic proteins. EYS606 for non-infectious uveitis
45 Fate Therapeutics San Diego, California Develops programmed cellular immunotherapies for cancer and immune disorders. FT516, FT596, FT538 for cancer, FT576
46 Ferring  Pharmaceuticals Saint-Prex, Switzerland The company has won FDA approval for Adstiladrin, is a novel adenovirus vector-based gene therapy, for adults with high-risk, Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer NMIBC) with carcinoma in situ. Adstiladrin (nadofaragene firadenovec-vncg)
47 Forte Biosciences South San Francisco, California The company’s lead product candidate, FB-401, is a topical live biotherapeutic product for the treatment of atopic dermatitis. FB-401 for atopic dermatitis, FB-201 for acne
48 Freeline Therapeutics Stevenage, UK Freeline develops gene therapies for inherited diseases using adeno-associated viruses. FLT180a for Hemophilia B, FLT190 for Fabry disease
49 Fresh Tracks Therapeutics San Francisco, California The company’s lead product candidate, FT-101, is a gene therapy for phenylketonuria (PKU) that uses the CRISPR/Cas9 gene editing system. FRTX-02, FRTX-10, FRTX-03
50 Gamida Cell Boston, Massachusetts The company’s lead product candidate, omidubicel, is for bone marrow transplantation. Omidubicel
51 GeneQuine Biotherapeutics GmbH Berlin, Germany Specializes in developing gene therapies for musculoskeletal disorders. Gene therapies for osteoarthritis and other musculoskeletal diseases
52 Genascence Palo Alto, California Aims to treat osteoarthritis with a gene therapy that is injected directly into the affected joint. GNSC-001
53 Generation Bio Cambridge, Massachusetts The company’s pipeline includes gene therapies for hemophilia A, phenylketonuria and sickle cell disease. Gene therapies for diseases such as phenylketonuria (PKU), hemophilia A and sickle cell disease
54 GenSight Biologics Paris, France The company’s pipeline includes gene therapies for Leber hereditary optic neuropathy and retinitis pigmentosa. LUMEVOQ for Leber Hereditary Optic Neuropathy (LHON) and GS030 for Retinitis Pigmentosa (RP)
55 Gilead Sciences Foster City, California Gilead subsidiary Kite Pharma focuses on developing CaliforniaR-T cell therapies for cancer. Yescarta
56 Graphite Bio San Francisco, California Graphite Bio’s pipeline includes gene therapies for sickle cell disease and other genetic disorders. Gene therapies for genetic diseases, such as sickle cell disease and genetic blindness
57 Homestead BioPharma Houston The company’s pipeline includes gene therapies for the treatment of acute myeloid leukemia and other cancers. Cancer immunotherapies.
58 Homology Medicines Bedford, Massachusetts A gene therapy company that focuses on developing therapies for rare genetic diseases. Gene therapies for phenylketonuria (PKU) and metachromatic leukodystrophy (MLD).
59 Intellia Therapeutics Cambridge, Massachusetts The company’s pipeline includes gene editing therapies for transthyretin amyloidosis and sickle cell disease. Pipeline focuses on genetic diseases such as transthyretin amyloidosis (ATTR), hemophilia and sickle cell disease
60 Juno Therapeutics Seattle Develops cell-based immunotherapies based on its chimeric antigen receptor and T cell receptor technologies to genetically engineer T cells for oncology indications. Lisocabtagene maraleucel (Breyanzi)
61 Krystal Biotech Pittsburgh The company’s lead product candidate, KB103, is a gene therapy for dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. Gene therapies for rare skin diseases, including epidermolysis bullosa (EB)
62 LogicBio Therapeutics Cambridge, Massachusetts Developing a gene editing platform that uses transposons to integrate therapeutic transgenes into the genome without the use of nucleases. LB-001 for methylmalonic acidemia and LB-10 for phenylketonuria
63 Lysogene Paris, France Lysogene is focused on developing gene therapies for rare central nervous system (CNS) diseases. The company’s lead program is a gene therapy for the treatment of Sanfilippo syndrome type A. LYS-SAF302 for Sanfilippo syndrome type A
64 MeiraGTx London MeiraGTx is developing gene therapies for rare genetic diseases, with a focus on diseases of the eye and salivary gland. AAV-CNGB3 for achromatopsia
65 Mesoblast Melbourne, Australia The company’s lead product is a mesenchymal stem cell therapy for the treatment of graft versus host disease (GvHD). Remestemcel-L for GvHD
66 Metagenomi Emeryville, California Metagenomi is developing a platform for the discovery and development of novel CRISPR systems. The company’s lead program is a novel CRISPR system for the treatment of genetic diseases. The company has attracted $275M in Series B financing to progress their lead therapeutic programs into the clinic. The company has an internal and partnered pipeline of in vivo genetic medicines. Scant information is available about them, however.
67 NexImmune Gaithersburg, Maryland NexImmune is developing cell therapies for the treatment of cancer and autoimmune diseases. The company’s lead product is a T cell therapy for the treatment of acute myeloid leukemia (AML). NEXI-001 for AML
68 NextCure Beltsville, Maryland NextCure is focused on discovering and developing next-generation immuno-oncology products. NC318, NC410
69 Nightstar Therapeutics London Nightstar Therapeutics is developing gene therapies for inherited retinal diseases. NSR-REP1, NSR-RPGR
70 Novartis Basel, Switzerland Novartis AG (formerly AveXis) is focused on developing gene therapies for rare genetic diseases. Zolgensma
71 Orchard Therapeutics London Specializes in gene therapies for rare diseases, with a focus on primary immune deficiencies, metabolic disorders and inherited blood diseases. Strimvelis, OTL-101
72 Oxford Biomedica Oxford, UK Develops lentiviral vector-based gene therapies for rare and life-threatening diseases, including cancer and Parkinson’s disease. LentiGlobin, OXB-102, OXB-202, AXO-Lenti-PD, OXB-301
73 Passage Bio Philadelphia The company’s technology platform enables the precise editing of genes in vivo. PBGM01, PBFT02
74 Pfizer New York City  Focused on diseases with single-gene defects, including certain neuromuscular and hematologic diseases. VTX-801
75 Pluristem Therapeutics Haifa, Israel Pluristem Therapeutics is focused on improving the health and well-being of patients by developing innovative cell therapies that can transform the treatment of serious diseases. PLX-R18, PLX-PAD
76 Poseida Therapeutics San Diego, California The company’s technology platform allows for the precise editing of genes in vivo, enabling the development of gene therapies that can address the root cause of genetic diseases. P-BCMA-ALLO1, P-PSMA-101
77 Regeneron Tarrytown, New York The company’s focus includes developing therapies for genetic diseases caused by a single gene mutation. RGN-352, RGX-121, REGN1979, REGN5458, REGN5459, REGN5678
78 Regenxbio  Rockville, Maryland Regenxbio specializes in the development of AAV gene therapies for a range of diseases. RGX-121, RGX-314
79 ReNeuron Bridgend, UK ReNeuron’s core focus is on its CustomEx exosome technology platform. ReN0001, ReN0000
80 Roche  Basel, Switzerland Roche has a significant presence in the field of cell and gene therapy. It has a number of partnerships and collaborations with companies working in the space. Cell and gene therapy programs for hemophilia A, Huntington’s disease, spinal muscle atrophy and ophthalmology
81 Rocket Pharmaceuticals New York City Advancing an integrated pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. RP-A501, RP-L102, RP-A201, RP-A301
82 Rubius Therapeutics Cambridge, Massachusetts Company is genetically engineering red blood cells to develop a new class of cellular medicines called Red Cell Therapeutics. RTX-240, RTX-224, RTX-T1D
83 Sana Biotechnology Seattle Working to repair and control genes in cells or replace missing or damaged cells to address the underlying cause of diseases. Sana is building differentiated capabilities across the spectrum of cell and gene therapy SG295, SG242, SG221, SG239, SG328
84 Sangamo Therapeutics Brisbane, California The company develops genomic therapies for cancer, monogenic diseases and infectious diseases. SB-525, ST-400, SB-318, ST-920, TX200, BIVV003, ST-101, KITE-037
85 Sarepta Therapeutics Cambridge, Massachusetts Develops treatments for rare neuromuscular diseases, including Duchenne muscular dystrophy, Limb-girdle muscular dystrophy and Charcot-Marie-Tooth. Exondys 51, Vyondys 53, golodirsen, casimersen, SRP-9001, SRP-9003, SRP-5051, SRP-707, SRP-9004
86 Solid Biosciences Cambridge, Massachusetts The company’s lead product candidate is a microdystrophin gene therapy that aims to restore functional dystrophin protein expression in Duchenne muscular dystrophy. SGT-001
87 Spark Therapeutics Philadelphia Specializes in adeno-associated viral (AAV) vectors for debilitating genetic diseases. Luxturna (voretigene neparvovec-rzyl)
88 SQZ Biotechnologies Watertown, Massachusetts Develops cellular therapies for cancer, infectious diseases and other serious conditions. SQZ-PBMC-HPV for human papillomavirus-associated tumors and SQZ-AAC for solid tumors
89 StrideBio Durham, NC Develops viral vector technologies for gene therapy. STR101, STR102, STR103
90 Suzhou Ribo Life Science Suzhou, China The company’s pipeline includes RNA therapies for cancer and infectious diseases. Eight products in development include assets for hepatitis B and non-arteritic anterior ischemic optic neuropathy
91 Takeda Pharmaceutical Tokyo, Japan Focuses on developing cell and gene therapies addressing significant unmet medical needs in oncology, rare genetic diseases and neurological disorders. Adynovate, Ninlaro, Alunbrig, Gattex/Revestive, Takhzyro, Natpara, Firazyr, Iclusig, Velcade
92 Tmunity Therapeutics Philadelphia Develops T cell therapies for the treatment of cancer and other diseases. Tmunity CD19, Tmunity NY-ESO, Tmunity WT1, Tmunity EGFRvIII
93 Ultragenyx Novato, California The company’s pipeline includes therapies for genetic diseases, metabolic diseases and rare cancers. Dojolvi, Crysvita, Mepsevii
94 UniQure Lexington, Massachusetts UniQure’s technology platform allows for the targeted delivery of therapeutic genes to specific cells. AMT-130, AMT-150, AMT-190
95 Veneti South San Francisco, California The company’s pipeline includes cell therapies for hematologic malignancies and solid tumors. VET-001 (acute myeloid leukemia), VET-002 (solid tumors), VET-003 (solid tumors)
96 Vericel Cambridge, Massachusetts The company’s product portfolio includes cell therapies for cartilage repair and burns. MACI (cartilage regeneration), Epicel (skin regeneration)
97 Vertex Pharmaceuticals Boston, Massachusetts Specializes in the development of small molecule drugs for the treatment of serious diseases, including cystic fibrosis, sickle cell disease and beta thalassemia. Trikafta (cystic fibrosis), Kaftrio (cystic fibrosis), Symdeko (cystic fibrosis)
98 Verve Therapeutics Cambridge, Massachusetts The company’s technology platform allows for the precise editing of genes in vivo. VERVE-101, VERVE-201
99 Vivet Therapeutics Paris, France The company’s lead program is a gene therapy for Wilson disease, a rare genetic disorder that causes the accumulation of copper in the liver and other organs. VTX-801, VTX-803
100 Voyager Therapeutics Cambridge, Massachusetts Focused on developing gene therapies for neurological diseases, including Parkinson’s disease, Huntington’s disease and ALS. VY-HTT01, VY-SOD102, VY-AADC01

Filed Under: Cell & gene therapy, Pharma 50, Special Feature
Tagged With: 4D Molecular Therapeutics, Abeona Therapeutics, Adaptimmune Therapeutics, Adverum Biotechnologies, Allogene Therapeutics, Alnylam, Altor BioScience, American Gene Technologies, Amgen, Arcellx, Arrowhead Pharmaceuticals, Aspen Neuroscience, Astellas Gene Therapies, AstraZeneca/Alexion, Atara Biotherapeutics, Autolus Therapeutics, AVROBIO, Axovant Gene Therapies, Batavia Biosciences, Bayer, Bean Therapeutics, BioMarin Pharmaceutical, Bluebird Bio, BlueRock Therapeutics, Bristol-Myers Squibb, Cabaletta Bio, Capricor Therapeutics, Caribou Biosciences, Celgene, Cellectis, Celularity, Celyad Oncology, Chimeron Bio, Coave Therapeutics, CRISPR Therapeutics, CSL Behring, Deep Genomics, Dendreon, Dyno Therapeutics, Editas Medicine, eGenesis, ElevateBio, Enzyvant, Eyevensys, Fate Therapeutics, Ferring Pharmaceuticals, Forte Biosciences, Freeline Therapeutics, Fresh Tracks Therapeutics, Gamida Cell, Genascence, GeneQuine Biotherapeutics GmbH, Generation Bio, GenSight Biologics, Gilead Sciences, Graphite Bio, Homestead BioPharma, Homology Medicines, Intellia Therapeutics, Juno Therapeutics, Krystal Biotech, LogicBio Therapeutics, Lysogene, MeiraGTx, Mesoblast, Metagenomi, NexImmune, NextCure, Nightstar Therapeutics, Novartis, Orchard Therapeutics, Oxford Biomedica, Passage Bio, Pfizer, Pluristem Therapeutics, Poseida Therapeutics, Regeneron, Regenxbio, ReNeuron, Roche, Rocket Pharmaceuticals, Rubius Therapeutics, Sana Biotechnology, Sangamo Therapeutics, Sarepta Therapeutics, Solid Biosciences, Spark Therapeutics, SQZ Biotechnologies, StrideBio, Suzhou Ribo Life Science, Takeda Pharmaceutical, Tmunity Therapeutics, Ultragenyx, UniQure, Veneti, Vericel, Vertex Pharmaceuticals, Verve Therapeutics, Vivet Therapeutics, Voyager Therapeutics
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

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