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100 cell and gene therapy leaders to watch in 2025

By Brian Buntz | December 20, 2024

Bright green stem cell colony of embryo under microscope

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As 2024 drew to a close, the cell and gene therapy sector has witnessed a string of recent approvals and some positive clinical trial data such as Mesoblast’s Ryoncil receiving FDA authorization for pediatric graft-versus-host disease in December, Adaptimmune’s Tecelra gaining approval for synovial sarcoma in August, and the landmark clearance of Casgevy (Exagamglogene Autotemcel) as the first CRISPR-based therapy for sickle cell disease in late 2023 and a second FDA approval in January for transfusion-dependent beta-thalassemia (TDT).

In 2024, established players like Gilead/Kite and Bristol Myers Squibb (BMS) have reported strong quarterly revenues for their CAR-T portfolios. Meanwhile, pioneering companies such as Bluebird Bio, despite their competitive CGT standing, face mounting pressure from rivals with broader patient populations and more rapid market uptake. Although Bluebird’s ultra-rare disease therapies have begun to produce revenue, larger rivals such as Gilead/Kite and BMS could see more momentum as they tap into larger patient populations.

The broader cell and gene therapy continues to diversify with the third-quarter 2024 edition of the Gene, Cell, + RNA Therapy Landscape Report from ASGCT and Citeline noting a shift toward non-oncology indications, with 51% of newly initiated gene therapy trials targeting conditions beyond cancer. This momentum extends beyond individual approvals — the report identifies 4,099 therapies in development across the pipeline, with gene therapies accounting for 49% of all cell, gene, and RNA therapeutics in development.

The report noted that deal activity remained steady with 101 transactions recorded in Q3 2024 and early-stage financing nearly doubled quarter-over-quarter. The CDMO sector saw prominent acquisitions such as Agilent’s $925M purchase of Biovectra, and global players like ImmunoACT in Mumbai and EdiGene in Beijing advanced new platforms. Major firms like Novartis and Cellectis continued to expand their patent portfolios.

The following table provides a data-driven snapshot of the CGT landscape, including a mix of well-established companies and emerging ventures:

Rank Organization Name Headquarters Description FDA-Approved CGT Products Funding Status / Recent Funding Notes
1 Bluebird Bio Somerville, MA, USA Gene therapies for genetic diseases Zynteglo, Skysona, Lyfgenia Public (BLUE) Multiple approved gene therapies for rare diseases. Patents (2020–2024): 15 total (10 gene, 1 vector, 4 cancer)
2 Novartis Basel, Switzerland Pioneer: CAR-T (Kymriah), Zolgensma (SMA) Kymriah, Zolgensma Public Robust pipeline, global CGT leader. Patents (2020–2024): 95 total (47 gene, 8 vector, 40 cancer)
3 Gilead Sciences/Kite Pharma Foster City, CA, USA CAR-T therapies for oncology Yescarta, Tecartus Public (GILD) Leading CAR-T franchise in oncology. Patents (2020–2024): 40 total (6 vector, 34 cancer). 2024 sales data strong: Q2: $521M combined, Q3: ~$485M, steady performance.
4 Bristol Myers Squibb New York, NY, USA Multiple CAR-T approvals Abecma, Breyanzi Public (BMY) Deep CAR-T pipeline, oncology focus. Patents (2020–2024): 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.
5 Roche Basel, Switzerland Inherited retinal disease gene therapy Luxturna Public Integrated CGT pipeline, global presence. Patents (2020–2024): 61 total (18 gene, 40 vector, 3 cancer). Elevidys sales Q3 2024: ~30M CHF, exceeding analyst expectations.
6 Pfizer New York, NY, USA Gene therapy in hemophilia B, others Beqvez Public (PFE) Partnering in gene therapies, robust R&D. Patents (2020–2024): 25 total (6 gene, 5 vector, 14 cancer)
7 BioMarin Pharmaceutical San Rafael, CA, USA Rare disease gene therapy (hem A) Roctavian Public (BMRN) Pioneer in rare disease gene therapy. Patents (2020–2024): 13 total (13 gene)
8 Vertex Pharmaceuticals Boston, MA, USA Exa-cel for SCD/β-thalassemia Casgevy (Exa-cel) Public (VRTX) Strong CF base, gene editing collaborations. Patents (2020–2024): 19 total (16 gene, 3 vector)
9 Sarepta Therapeutics Cambridge, MA, USA DMD gene therapy Elevidys Public (SRPT) Leader in RNA/gene for neuromuscular disease. Patents (2020–2024): 11 total (11 gene)
10 Adaptimmune Therapeutics Abingdon, UK Engineered T-cells for solid tumors Tecelra Public (ADAP) SPEAR T-cell therapies. Patents (2020–2024): 22 total (3 vector, 19 cancer)
11 Autolus Therapeutics London, UK Next-gen CAR-T Aucatzyl Public (AUTL) Modular CAR-T platforms. Patents (2020–2024): 45 total (5 gene, 40 cancer)
12 Krystal Biotech Pittsburgh, PA, USA Gene therapy for DEB Vyjuvek Public (KRYS) Topical gene therapy for rare skin disorders. Patents (2020–2024): 9 total (9 gene)
13 Orchard Therapeutics London, UK Ex vivo gene therapies (rare) Lenmeldy Public (ORTX) Lentiviral ex vivo for inherited disorders
14 Gamida Cell Boston, MA, USA Cell therapy for BMT (Omisirge) Omisirge Public (GMDA) Improving bone marrow transplant outcomes
15 Enzyvant Durham, NC, USA Allogeneic processed thymus tissue Rethymic Private/Subsidiary Thymus-based regenerative medicine
16 Mesoblast Melbourne, Australia Allogeneic MSC therapy (GvHD) Ryoncil Public (MESO) MSC platform for inflammatory diseases
17 Astellas Gene Therapies Boston, MA, USA Neuromuscular & ocular gene therapies None Public (ALPMY) Expanding CNS/ocular pipeline. Patents (2020–2024): 19 total (14 gene, 5 cancer)
18 Bayer Leverkusen, Germany Hemophilia gene therapy pipeline None Public Invested heavily in CGT (AskBio, BlueRock integrated). Patents (2020–2024): 12 total (11 gene, 1 vector)
19 Intellia Therapeutics Cambridge, MA, USA CRISPR in vivo editing None Public (NTLA) First systemic in vivo CRISPR trial. Patents (2020–2024): 17 total (14 gene, 3 vector)
20 Beam Therapeutics Cambridge, MA, USA Base editing gene therapies None Public (BEAM) Base editing precision. Patents (2020–2024): 11 total (1 gene, 10 vector)
21 REGENXBIO Rockville, MD, USA AAV vector platform None Public (RGNX) NAV AAV vectors licensed broadly. Patents (2020–2024): 10 total (8 gene)
22 uniQure Lexington, MA, USA AAV gene therapies Hemgenix (with CSL Behring) Public (QURE) Hemophilia B gene therapy
23 Freeline Therapeutics Stevenage, UK AAV for hemophilia & Fabry None Public (FRLN) Next-gen AAV capsids, metabolic/bleeding disorders
24 Avrobio Cambridge, MA, USA Lentiviral gene therapies for lysosomal disorders None Public (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 Partners
25 Rocket Pharmaceuticals New York, NY, USA Lentiviral & AAV for rare diseases None Public (RCKT) Hematologic and cardiac gene therapy pipeline
26 Passage Bio Philadelphia, PA, USA CNS AAV gene therapies None Public (PASG) UPenn partnership, rare CNS disorders
27 Solid Biosciences Cambridge, MA, USA DMD gene therapy None Public (SLDB) Microdystrophin gene therapy for DMD
28 Homology Medicines Bedford, MA, USA In vivo gene editing & gene therapy None Public (FIXX) AAVHSC platform for gene correction. Patents (2020–2024): 8 total (8 gene)
29 ElevateBio Waltham, MA, USA CGT innovation center None Private ($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 Capital
30 Sana Biotechnology Seattle, WA, USA Ex/in vivo CGT None Public (SANA) Fusogenix, hypoimmune platforms
31 Fate Therapeutics San Diego, CA, USA iPSC immunotherapies None Public (FATE) Off-the-shelf NK & T-cell therapies. Patents (2020–2024): 7 total (7 cancer)
32 Poseida Therapeutics San Diego, CA, USA Gene editing allogeneic CAR-T None Public (PSTX)* Roche is acquiring Non-viral gene editing (piggyBac). Patents (2020–2024): 20 total (11 gene, 3 vector, 6 cancer)
33 Caribou Biosciences Berkeley, CA, USA CRISPR-edited cell therapies None Public (CRBU) chRDNAs for precision CRISPR editing. Patents (2020–2024): 48 total (4 gene, 33 vector, 11 cancer)
34 Cellectis Paris, France TALEN-based allogeneic CAR-T None Public (CLLS) TALEN gene editing, off-the-shelf CAR-T. Patents (2020–2024): 91 total (12 gene, 20 vector, 59 cancer)
35 MeiraGTx London, UK AAV for ocular & neuro None Public (MGTX) Vertically integrated gene therapy company. Patents (2020–2024): 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 Ventures
36 4D Molecular Therapeutics Emeryville, CA, USA Engineered AAV vectors None Public (FDMT) Custom AAV capsids for multiple indications. Patents (2020–2024): 34 total (34 gene)
37 Abeona Therapeutics New York, NY, USA Rare disease gene therapies None Public (ABEO) Focus on RDEB and CLN1
38 Adverum Biotechnologies Redwood City, CA, USA AAV ocular gene therapy None Public (ADVM) Wet AMD gene therapy. Patents (2020–2024): 26 total (23 gene, 3 vector)
39 Generation Bio Cambridge, MA, USA Non-viral genetic medicines None Public (GBIO) Closed-ended DNA platform
40 Dyno Therapeutics Cambridge, MA, USA AI-driven AAV engineering None Private AI to optimize AAV capsids
41 Metagenomi Emeryville, CA, USA Novel CRISPR systems None Public (MGX) Metagenomic enzyme discovery for gene editing. Patents (2020–2024): 11 total (1 gene, 10 vector)
42 EdiGene Beijing, China Gene editing & therapy None Private CRISPR-based therapies in development. Patents (2020–2024): 6 total (6 vector)
43 Celularity Florham Park, NJ, USA Placental-derived cell therapies None Public (CELU) Off-the-shelf NK cell therapies
44 Rubius Therapeutics Cambridge, MA, USA Red Cell Therapeutics None Public (RUBY) Engineering RBCs for immuno-oncology. Patents (2020–2024): 8 total (7 gene, 1 cancer)
45 Arcellx Germantown, MD, USA Controllable CAR-T None Public (ACLX) ARC-sparX platform. Patents (2020–2024): 5 total (5 cancer)
46 Astraveus Paris, France CGT manufacturing solutions (with innovation) None Seed €10.4M Microfluidic 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 Ventures
47 Inceptor Bio Raleigh, NC, USA Cell therapy for tough cancers None Early Stage Venture Diversified 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 Fund
48 Tevogen Bio Warren, NJ, USA Off-the-shelf T-cell therapies None Public (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 Partners
49 CSL Behring Melbourne, Australia Global biotech in immunology/heme/CGT None Public (CSL) Hemophilia B gene therapy & plasma products
50 CRISPR Therapeutics Zug, Switzerland & Boston, MA CRISPR/Cas9 gene editing therapies Exa-cel (with Vertex) Public (CRSP) Pipeline in hemoglobinopathies, oncology. Patents (2020–2024): 57 total (46 gene, 6 vector, 5 cancer)
51 ImmunoACT Mumbai, India Advanced CGT startup None ₹800M Corporate CAR-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 Labs
52 Coave Therapeutics Paris, France Gene therapies for ocular/CNS None Private (€33.1M Series B) AAV-ligand conjugates
53 Celyad Oncology Belgium Allogeneic CAR-T for oncology None Public (CYAD) shRNA-based allogeneic CAR-T
54 Chimeron Bio Philadelphia, PA, USA Self-amplifying RNA therapeutics None $4.33M funding ChaESAR RNA platform
55 Deep Genomics Toronto, Canada AI-driven RNA therapeutics None $241M total BigRNA AI for RNA drug design
56 Dendreon Seal Beach, CA, USA Cellular immunotherapy for prostate cancer PROVENGE Commercial revenue Autologous cell therapy pioneer
57 Editas Medicine Cambridge, MA, USA CRISPR-based gene editing therapies None ~$931.6M raised In vivo & ex vivo CRISPR platform. Patents (2020–2024): 48 total (21 gene, 26 vector, 1 cancer)
58 eGenesis Cambridge, MA, USA Gene-edited xenotransplantation None $191M Series D Multi-gene-edited porcine organs
59 Eyevensys Paris, France Non-viral gene therapy for eye diseases None $44.1M funding Electroporation-based ocular gene delivery
60 Ferring Pharmaceuticals Saint-Prex, Switzerland ADSTILADRIN for bladder cancer ADSTILADRIN Private; $500M royalty deal Gene therapy in oncology
61 Forte Biosciences Dallas, TX, USA Anti-CD122 mAb for autoimmune None $53M private placement Targeting IL-2 pathway disorders
62 Genascence Palo Alto, CA, USA Gene therapy for osteoarthritis None FDA Fast Track GNSC-001 IL-1Ra gene therapy for OA
63 GenSight Biologics Paris, France Gene therapies for retinal diseases None Public (SIGHT.PA) Mitochondrial targeting, optogenetics
64 Cabaletta Bio Philadelphia, PA, USA T-cell therapies for autoimmune disease None Public (CABA) CAART for B-cell-mediated conditions
65 Capricor Therapeutics San Diego, CA, USA Cell & exosome-based therapies (DMD) None Public (CAPR) StealthX exosome platform
66 Regeneron Tarrytown, NY, USA Expanding in CGT, advanced biologics None (CGT) Public (REGN) Acquired cell therapy programs, DB-OTO gene therapy
67 Ribo Kunshan, China RNAi therapeutics None Multiple RMB rounds GalNAc RNAi for liver/CV/metabolic
68 Takeda Tokyo, Japan Global pharma investing in CGT None Public (TAK) Cell therapy facility, gene therapy collaborations
69 Ultragenyx Pharmaceutical Novato, CA, USA Rare & ultra-rare diseases (gene therapy) None Public (RARE) Diverse biologics/gene therapy pipeline
70 Vineti San Francisco, CA, USA Personalized therapy management platform None Series A-C funding Software for CGT supply chain
71 Vericel Cambridge, MA, USA Cell therapies for cartilage & burns MACI®, Epicel®, NexoBrid® Public (VCEL) Strong revenue, expanding indications
72 Verve Therapeutics Boston, MA, USA Gene editing for cardiovascular disease None Public (VERV) Base editing for LDL-C/triglycerides
73 Vivet Therapeutics Paris, France Gene therapies for metabolic diseases None Private VTX-801 for Wilson disease (Phase 1/2)
74 Voyager Therapeutics Cambridge, MA, USA Gene therapies for neurological diseases None Public (VYGR) TRACER capsids, partnered CNS programs
75 Sangamo Therapeutics Brisbane, CA, USA Zinc finger & AAV gene therapies None Public (SGMO) ZFN platform, Fabry, hemophilia programs
76 SQZ Biotechnologies Watertown, MA, USA Cell therapy for HPV16+ tumors None Public (SQZ) AAC & eAPC platforms, strategic restructuring
77 Arrowhead Pharmaceuticals Pasadena, CA, USA RNAi therapeutics (cardiometabolic & more) None Public (ARWR) SiRNA pipeline, strong partnerships
78 Alnylam Pharmaceuticals Cambridge, MA, USA RNAi therapies for various diseases Onpattro, Givlaari, Oxlumo, Amvuttra Public (ALNY) RNAi pioneer with multiple approved products. Patents (2020–2024): 73 total (73 gene)
79 Aspen Neuroscience San Diego, CA, USA Autologous iPSC for Parkinson’s disease None Private (> $220M raised) iPSC-based personalized cell therapy
80 American Gene Technologies (AGT) Rockville, MD, USA Gene therapy for HIV & rare diseases None ~$78M funding HIV functional cure in Phase 1
81 Ionis Pharmaceuticals Carlsbad, CA, USA Antisense therapies Spinraza, Tegsedi, Waylivra Public (IONS) Antisense leader, broad pipeline. Patents (2020–2024): 62 total (60 gene, 2 vector)
82 Allogene Therapeutics South San Francisco, CA, USA Allogeneic CAR T therapies None Public (ALLO) Off-the-shelf CAR T for hematologic & solid tumors
83 NexImmune Gaithersburg, MD, USA AIM nanoparticle immunotherapy None Public (NEXI) Artificial Immune Modulation platform
84 NextCure Beltsville, MD, USA Immunomedicines for oncology None Public (NXTC) B7-H4 ADC, immunotherapy pipeline
85 Oxford Biomedica Oxford, UK Vector platform (Lentiviral), integrated innovation None Public (LSE:OXB) Leading lentiviral vector developer & collaborator
86 Pluri Inc. Haifa, Israel 3D cell-based technology across industries None Public (PLUR) 3D cell expansion platform
87 ReNeuron Bridgend, UK Exosome-based therapeutics None Public (RENE.L) CustomEX platform for targeted delivery
88 Arcturus Therapeutics San Diego, CA, USA mRNA medicines & vaccines None Public (ARCT) Self-amplifying mRNA vaccines/therapies. Patents (2020–2024): 23 total (23 gene)
89 Silence Therapeutics London, UK RNAi therapeutics targeting liver genes None Public (SLN) GalNAc-siRNA pipeline
90 BioNTech Mainz, Germany mRNA-based immunotherapies Comirnaty (COVID-19 vaccine) Public (BNTX) mRNA cancer immunotherapies, global leader. Patents (2020–2024): 36 total (29 gene, 7 vector)
91 Moderna Cambridge, MA, USA mRNA therapeutics & vaccines Spikevax (COVID-19 vaccine) Public (MRNA) Expanding mRNA platform to rare diseases. Patents (2020–2024): 140 total (124 gene, 16 vector)
92 CureVac Tübingen, Germany mRNA therapeutics & vaccines None Public (CVAC) mRNA platform for prophylactic & therapeutic use. Patents (2020–2024): 65 total (56 gene, 9 vector)
93 Inovio Pharmaceuticals Plymouth Meeting, PA, USA DNA medicines (cancer, infectious diseases) None Public (INO) Electroporation delivery of DNA plasmids. Patents (2020–2024): 28 total (25 gene, 3 vector)
94 Senti Biosciences South San Francisco, CA, USA Gene circuit-engineered cell & gene therapies None Public (SNTI) Programmable gene circuits for cell therapies
95 Graphite Bio South San Francisco, CA, USA CRISPR gene editing therapies None Public (GRPH) Precise gene correction for genetic diseases
96 Century Therapeutics Philadelphia, PA, USA Allogeneic iPSC-derived cell therapies None Public (IPSC) iPSC platform for NK & T-cell therapies
97 ArsenalBio San Francisco, CA, USA Programmable cell therapies for cancer None Private Engineered T-cells with synthetic biology
98 Homestead BioPharma Houston, TX, USA Gene therapies targeting AML & cancers None Private Early-stage gene therapy for oncology
99 Anew Medical Omaha, NE, USA CGT for cancer/neurological None IPO $15M Developing 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 Group
100 Ambulero Miami, FL, USA Vascular gene therapies None Seed $5.5M Gene 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

*International CGT patents based on database search results from 2020 to late August/early September 2024. For more on how CGT was defined in the search, check out the note in “Top 25 global leaders in cell and gene therapy patents.” Every effort was made to ensure the accuracy of data in this table, but mistakes or omissions may be present.

Methodology: This ranking prioritizes organizations that are actively shaping the cell, gene, and RNA therapy landscape through their clinical pipeline, technological contributions, and strategic positioning. Firms with FDA-approved therapies or late-stage (Phase 2/3) clinical candidates were generally elevated in the rankings. For example, Bluebird Bio appears at the top because it has multiple FDA-approved gene therapies and a track record of advancing candidates through clinical trials, including Zynteglo (approved in August 2022 for beta-thalassemia requiring regular transfusions), Skysona (approved in September 2022 for cerebral adrenoleukodystrophy in young boys), and Lyfgenia (approved in December 2023 for sickle cell disease in patients aged 12 and older with a history of vaso-occlusive events).

Companies known for pioneering novel platform technologies or addressing multiple therapeutic areas were positioned more favorably. For instance, Beam Therapeutics, despite the absence of approved products, ranks higher than many earlier-stage ventures because it introduced base editing — a next-generation gene editing approach — and secured significant funding and partnerships.

In addition to the presence of FDA-approved therapies and the stage of clinical development, we considered patent activity (international patents from January 2020 to early September 2024) as a measure of innovation. Acquisitions and integrations were accounted for in aggregated patent data (e.g., Bristol Myers Squibb, Roche). The ranking also takes into account strategic alliances, depth of pipeline, novelty of technology platforms, and funding milestones. Through this multifactorial approach, the list highlights those making significant contributions to advancing the science and commercialization of cell, gene, and RNA therapies.

If you have suggestions or updated data to influence the rankings, feel free to reach out.


Filed Under: Cell & gene therapy
Tagged With: biotech funding, cell therapy, clinical trials, gene therapy, regulatory approvals, RNA 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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