Published: August 2023
These cell and gene therapy companies are all set to revolutionize the field of cell and gene therapy with their efforts to develop innovative therapeutic candidates. We analyzed close to 480 cell and gene therapy companies that are engaged in the development of cell therapies and gene therapies, across different stages of development. In this article, we unveil 10 leading companies and their capabilities in this industry.
Contemporary medical science has traced thousands of clinical conditions to a genetic cause. Cancer, a life-threatening disease, also has genetic origins, and is considered among the leading causes of death across the globe. Amidst the current initiatives undertaken to develop more targeted anti-cancer therapies, T-cell therapies (specifically CAR T cell therapy, TCR therapy and TIL therapy) have emerged as a promising option, owing to their ability to eradicate tumor cells from the body with minimal treatment-related side effects. It is worth noting that, as of 2023, over 1,035 clinical studies focused on such cell therapy candidates have been initiated. Positive clinical trial results of various T-cell therapy candidates have led the cell and gene therapy developers to partner with other stakeholders in this industry and have captured the interest of potential investors as well.
Moreover, experts believe that there are over 7,000 different types of rare diseases (including some rare forms of cancer), most of which originate as a consequence of genetic anomalies. Driven by numerous developmental breakthroughs and results of studies demonstrating the vast potential of gene therapies in the treatment of rare diseases, this therapeutic modality has garnered considerable attention of various players engaged in the healthcare industry. As of 2023, there are a variety of corrective strategies that have been developed for the treatment of other clinical conditions which develop as a result of genetic abnormalities, such as sickle cell disease, parkinson’s disease, hearing loss, peripheral artery disease, spinal muscular atrophy, autoimmune diseases, and genetic diseases. Further, the growing need for treatment options that can cure a clinical condition, as opposed to those intended for disease management, has created a demand for more effective gene manipulation approaches.
As of 2023, over 1,000 cell and gene therapy candidates are currently being investigated for the treatment of various disease indications. It is worth mentioning that over 40 cell and gene therapies have already been approved by various regulatory authorities across the globe. In 2021, Gilead Sciences received FDA approval of Tecartus (CAR-T cell therapy) for the treatment of relapsed or refractory B-cell Acute Lymphoblastic Leukemia. Moreover, in 2022, Bristol Myers Squibb received the FDA approval of a new generation of personalized CAR-T cell therapy, Breyanzi, for the treatment of relapsed or refractory large B-cell lymphoma. In June 2023, FDA approved Elevidys (a gene therapy developed by Sarepta Therapeutics) for the treatment of Duchenne muscular dystrophy. The development of cell and gene therapies involves a heavy investment in research and development, and manufacturing of such therapeutic modalities. Further, in order to ensure the optimum use of their resources and augment their revenue generation opportunities, a number of cell therapy companies engaged in this market offer contract services, in addition to their in-house manufacturing capabilities. In recent years, several big pharma players have undertaken initiatives, including establishing strategic partnerships, seeking investment from venture firms, and carrying out expansion projects, in order to enhance their cell therapy manufacturing capabilities.
Roots Analysis has conducted an exhaustive study of the Cell Therapy Market and Gene Therapy Market featuring the current market landscape and future opportunity associated with cell therapies and gene therapies, respectively, over the forecasted period.
In the table below, we have listed (in decreasing order of funding amount raised), the top 10 cell therapy companies and gene therapy companies.
Table: Top Cell Therapy and Gene Therapy Companies
|Company||YoE||Headquarters||Number of Employees||Funding Amount (USD Million)1|
|BridgeBio||2014||Palo Alto, CA||201-500||2,903|
|Intellia Therapeutics||2014||Cambridge, MA||201-500||1,658|
|Iovance Therapeutics||2007||San Carlos, CA||501-1,000||1,572|
|Allogene Therapeutics||2017||South San Francisco, CA||201-500||1,425|
|CRISPR Therapeutics||2013||Cambridge, MA||201-500||1,374|
|Atara Therapeutics||2012||Thousand Oaks, CA||201-500||1,333|
|Vir Biotechnology||2016||San Francisco, CA||501-1,000||1,282|
|Fate Therapeutics||2007||San Diego, CA||201-500||1,157|
|Adaptimmune Therapeutics||2008||Abingdon, UK||201-500||1,098|
|Novartis Gene Therapies2||2010||Bannockburn, IL||1,001-5,000||1,024|
Abbreviation: YoE: Year of Establishment
Note 1: The funding instances have been considered till 2021
Note 2: Novartis Gene Therapies was earlier known as AveXis
Source: Roots Analysis
This article highlights the top cell therapy companies and gene therapy companies to watch out for in 2023. These companies were chosen based on the amount of funding raised in capital investment and have the required funds to advance the development of their drug products. It is essential to note that the selection of top players can differ substantially according to the defined criteria.
Note 1: Novartis Gene Therapies is a part of Novartis and the year of establishment corresponds to Novartis Gene Therapies
Interested in exploring the funding and investment activity across all 470+ Cell and Gene Therapy Companies?
BridgeBio is a US-based biotechnology company that develops drugs to treat several genetic diseases and cancer. Additionally, the company has its genetic disease target engine and drug engineering platform which allows to identify, discover, and develop genetic medicines. The company claims to be working in different modalities, including gene therapies, small molecules, therapeutic proteins, and antisense nucleotides. It is worth highlighting that BridgeBio’s subsidiaries, Aspa Therapeutics and Adrenas Therapeutics are actively engaged in the gene therapy industry.
BridgeBio is engaged in the development of following gene therapies:
Market Capitalization (as of July 2023): USD 5.48 billion
Total Funds Raised: USD 2,903 million
In May 2023, BridgeBio presented positive results from a phase 1 / 2 clinical trial of BBP-812 at the 2023 American Society of Gene and Cell Therapy (ASGCT) annual meeting. In June 2023, the company raised USD 150 million through secondary offering. Earlier, in July 2022, the company entered a collaboration with Baylor College of Medicine to identify and translate the innovations into potential therapies for patients suffering from genetic diseases.
BridgeBio is looking at multiple regulatory submissions in the next 5 years for its small molecule portfolio targeting genetic diseases. With the expected launch, the company is also working on setting up its commercial team and network, which will serve as beneficial for any future gene therapy launch.
Intellia Therapeutics was founded in 2014 and is headquartered in Cambridge, Massachusetts, US. The company aims to accelerate the progress of its therapeutic candidates, from laboratory to clinic, based on its CRISPR technology. Over time, Intellia Therapeutics has established a wide intellectual property portfolio for CRISPR / Cas9 technology. The high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability of this technology makes it an efficient gene editing technology.
Intellia Therapeutics is engaged in the development of following gene and cell therapies:
Market Capitalization (as of July 2023): USD 3.82 billion
Total Funds Raised: USD 1.658 billion
In September 2022, the FDA granted orphan drug designation to NTLA-2002, an in vivo CRISPR-based therapy designed to prevent the angioedema attacks in people suffering from hereditary angiodema. Further, in March 2023, the therapy was granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation and became the first in vivo gene therapy to be granted approval for evaluation in a phase 2 clinical trial. More recently, In June 2023, Intellia Therapeutics presented positive clinical data from phase I study of NTLA-2002 at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2023.
With the initiation of phase II clinical trials of its in vivo gene therapy, the company has been able to demonstrate the capability of its CRISPR technology as an in vivo gene editing tool. Additionally, Intellia Therapeutics’ in-house manufacturing facility, which was leased in February 2022, is expected to be operational by 2024, providing the necessary cell and gene therapy manufacturing support to enrich its pipeline.
Iovance Biotherapeutics is a US-based company that specializes in the development and discovery of novel tumor infiltrating lymphocyte (TIL) therapies. The company owns a 136,000 square feet manufacturing facility in Philadelphia dedicated to clinical trial manufacturing of TIL therapies. The company also has two proprietary technology platforms, namely, TIL platform and peripheral blood lymphocyte (PBL) therapy platform. As per the company’s website, the company claims to have more than 50 granted patents related to its TIL platform.
Iovance Biotherapeutics is engaged in the development of following TIL therapies:
Market Capitalization (as of July 2023): USD 1.90 billion
Total Funds Raised: USD 1,572 million
In May 2023, the company announced the acceptance of Biologics License Application (BLA) submission of the TIL therapy Lifileucel for the treatment of patients suffering from advanced melanoma. In addition, in July 2023, the company raised USD 172.5 million through secondary offering.
Post its FDA approval, Lifileucel will be the first one-time cell therapy approved for solid tumors. The treatment includes a short course of high-dose IL-2. In anticipation of the commercial launch of Lifileucel, Iovance Biotherapeutics acquired the worldwide rights to Proleukin (recombinant IL-2) securing its supply chain in advance. The company also has an operational clinical scale manufacturing facility in Philadelphia, US which would help it move faster to the market post the commercial launch.
Allogene Therapeutics, a clinical stage company based in California, US is focused on the development of several CAR-T therapies based on its proprietary allogeneic platform, AlloCAR T™. The firm claims that the products of AlloCAR™ platform exhibit high treatment potential, along with enabling cost effective cell therapy manufacturing and scalability. The company’s proprietary technology platform claims to prevent graft rejection and improve T-cell fitness at the same time. In 2023, the company is developing a series of allogeneic CAR Ts in collaboration with Cellectis and Antion Biosciences.
Allogene Therapeutics is engaged in the development of following CAR T cell therapies:
Market Capitalization (as of July 2023): USD 0.78 billion
Total Funds Raised: USD 1,425 million
In June 2023, the company announced that during the phase I trial, ALLO-501A induced a similar rate of durable complete remission as achieved through approved autologous T-cell therapies. In the same month, the company showed participation in several conferences, such as International Conference on Malignant Lymphoma (ICML), American Society of Clinical Oncology (ASCO) Annual Meeting, European Hematology Association (EHA) Hybrid Congress, in order to present its experimental findings and clinical trial results of its pipeline products.
The company has recently uncovered its proprietary Dagger™ technology which has been designed to resist the rejection of AlloCAR-T cells by the host immune cells. The technology aims at supporting the expansion of prolonged window of persistence wherein the AlloCAR-T cells can target and destroy the tumor cells. This technology will prove helpful for the company to improve the activity of its pipeline products.
CRISPR Therapeutics is a biopharmaceutical company focused on the development of transformative gene-based medicines for serious diseases, particularly somatic (non-germline) diseases. The company has its headquarters in Basel, Switzerland and operates through several centers located across the globe, such as London and Massachusetts. The company claims to be actively working on four focus areas; these include gene-edited hematopoietic stem cells, immune-oncology, in vivo gene editing, and regenerative medicine. It is worth mentioning that the firm states to leverage its proprietary CRISPR / Cas9 gene editing platform for the development of gene therapy products.
CRISPR Therapeutics is engaged in the development of following gene and CAR T therapies:
Market Capitalization (as of July 2023): USD 4.48 billion
Total Funds Raised: USD 1,374 million
In June 2023, the company announced that the FDA has accepted the Biologics License Application (BLA) submission of exa-cel, an ex vivo CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia. In March 2023, the company entered into an agreement with Vertex Pharmaceutical for the use of CRISPR Therapeutics’ technology in the development of Vertex’s hypoimmune cell therapies, intended for the treatment of type-1 diabetes.
Anticipated approval of exa-cel, the first CRISPR based therapy, will be a major milestone for CRISPR Therapeutics. Its drug development partner Vertex Therapeutics is already in talks with the commercial and government reimbursement agencies to ensure market adoption of its gene therapy. The approval of exa-cel will put a stamp of approval on the nobel prize winning technology of CRISPR Therapeutics. Meanwhile, the company continues to enroll and dose the patients undergoing different clinical trials for its product pipeline. CRISPR Therapeutics is expanding its capabilities in various other fields, such as metabolic diseases, through collaboration with other industry stakeholders in the gene editing domain.
Atara Biotherapeutics is a technology-driven company which has a diverse drug portfolio, including T cell immunotherapies, CAR T cell therapies, and Allogeneic CAR-T cell therapies. In addition to this, the company has its in-house manufacturing facility for the development of T cells and other immune cells. It is worth mentioning that Atara Biotherapeutics is the first company that has received the European Commission (EC) approval of Ebvallo™ (tabelecleucel), an allogeneic T-cell immunotherapy. In December 2022, Ebvallo™ became the first monotherapy approved for the treatment of adult and pediatric patients (2 years and older) with relapsed / refractory EBV+ post?transplant lymphoproliferative disease.
Atara Biotherapeutics is engaged in the development of following CAR T cell therapies:
Market Capitalization (as of July 2023): USD 0.21 billion
Total Funds Raised: USD 1,333 million
In February 2023, Atara Biotherapeutics and Pierre Fabre announced the transfer of market authorization of EBVALLO to the latter company; wherein Pierre will be responsible for the commercialization and distribution of the product in Europe. In April 2022, FUJIFILM Diosynth Biotechnologies acquired Atara’s cell therapy manufacturing facility for USD 100 million, leading to the expansion of CDMO business unit of FUJIFILM.
Atara Biotherapeutics has been actively pursuing the CMC aspects with the FDA related to BLA submission of tab-cel. The BLA is expected to be filed in 2023, depending on FDA requests for any additional information. However, the company has extensively started to engage in discussions with potential commercialization partners in the US. Pierre Fabre, its commercialization partner in Europe has begun the drug launch on a country-by-country basis.
Vir Biotechnology is a commercial stage immunology company that was founded in 2016. The firm’s business operations span across various technological platforms, including antibody platform, T-cell platform, innate immunity platform and siRNA platform, for the treatment of infectious diseases, such as Hepatitis B virus, Hepatitis D virus, Influenza A, Covid-19 and HIV infection. The company claims to be working on novel ways to combat these deadly diseases, these include multiple combinatorial regimens of different type of therapeutics.
Vir Biotechnology is engaged in the development of following virus-driven T cell therapies:
Market Capitalization (as of July 2023): USD 1.88 billion
Total Funds Raised: USD 1,282 million
In May 2023, Vir Biotechnology received a grant amounting to USD 10 million from Bill & Melinda Gates Foundation to support its novel T cell vaccine development program. The grant is aimed at supporting the Phase I clinical studies related to VIR-1388. Moreover, the company claims to adopt a novel, combinatorial approach to treat chronic HBV infection. In this regard, in May 2023, the company announced that the first patient has been dosed under Vir’s PREVAIL platform (VIR-2218, VIR-3434, PEG-IFNα and an NRTI).
The company claims to have strong financial strength, amounting to USD 2.3 billion in cash / cash equivalent, to fund company’s ongoing internal research programs. The company is consistently working towards development of impactful therapeutics for treating infectious diseases; it has also received the emergency authorization of Xevudy®? by the European Commission (EC) for the early treatment of COVID-19.
Fate Therapeutics which was established in 2007, is a clinical-stage biopharmaceutical company having its headquarters in California, US. The company is developing a series of induced pluripotent stem cell derived cellular immunotherapies for the treatment of patients suffering from various oncological and auto-immune disorders, by using its proprietary iPSC technological platform. The company uses its platform for the development of several cellular therapies. It claims that the proprietary technology offers unprecedented scalability, product homogeneity, and unlimited cell production in comparison to the patient-sourced cell therapies.
Fate Therapeutics is engaged in the development of following CAR T cell therapies:
Market Capitalization (as of July 2023): USD 0.042 billion
Total Funds Raised: USD 1,157 million
In June 2023, the company participated in 2023 Jefferies Healthcare Conference and 44th Annual Goldman Sachs Global Healthcare Conference to present its findings of various multiplex-engineered cell therapy products to the wider audience. In December 2022, the company revealed the favorable safety profile Phase I results of FT819, the first iPSC-derived CAR T cell therapy, in the patients suffering from aggressive large B-cell lymphoma.
With the intent to transform the cell therapy field, Fate Therapeutics’ facility has the capacity to scale-up the GMP operations and yield 100+ doses in one batch. Moreover, the company claims to maintain the batch-to-batch consistency during the mass production of the multiplex-engineered cell products, in terms of functionality and quality.
Adaptimmune Therapeutics is engaged in the development of various TCR cell therapies for the treatment of patients suffering from cancer. The company has developed its own proprietary technology, namely TCR platform, which aids in the identification of cancer targets and genetically engineer the TCRs to attack the targets. This technology claims to have several advantages over the other conventional platforms, such as targeting of larger number of proteins, extensive preclinical safety, and engineering of natural TCRs.
Adaptimmune Therapeutics is engaged in the development of following TCR cell therapies:
Market Capitalization (as of July 2023): USD 0.021 billion
Total Funds Raised: USD 1,098 million
In June 2023, the company announced the merger with TCR² Therapeutics to create a T-cell therapy company working for the treatment of solid tumors. In the same month, the company presented the results of SPEARHEAD-1 trial, evaluating the safety and efficacy of afami-cel in patients suffering from synovial sarcoma, at American Society of Clinical Oncology's (ASCO) annual meeting.
Adaptimmune Therapeutics expects to complete the rolling BLA submission for afami-cel for treatment of synovial sarcoma in 2023. Completion of the merger with TCR2 Therapeutics is likely to provide the company with necessary funds / cash in hand as well as the experience to undertake a commercial launch. The team at Adaptimmune Therapeutics claims to be well-versed with offering end-to-end capabilities required for the advancement and manufacturing of cell therapies intended to address solid tumors.
Novartis Gene Therapies is a subsidiary of Novartis group that specializes in the development and advancement of gene therapies. In May 2018, Novartis built a footprint in the gene therapy domain through acquisition of AveXis, including its gene therapy Zolgensma. In 2019, the company received the FDA approval of Zolgensma, a one-time infusion treatment, intended for pediatric patients (less than 2 years of age) suffering from spinal muscular atrophy (SMA). Later, in September 2020, as part of its commercialization strategy, the company changed the name of AveXis to Novartis Gene Therapies. The company is also investigating Zolgensma for a similar indication. The drug is being evaluated in phase III trials for the treatment of spinal muscular atrophy in patients, weighing between 8.5 kg and 21 kg and having biallelic mutations in SMN1 gene.
Novartis Gene Therapies is engaged in the development of following gene therapies:
Market Capitalization (as of July 2023): USD 206.56 billion
Total Funds Raised: USD 1,024 million
In May 2023, Novartis acquired AVROBIO’s hematopoietic stem cell gene therapy program being investigated for the treatment of cystinosis, a rare disease. The company announced the payment of USD 87.5 million for this asset acquisition. Earlier, in March 2023, NICE approved the NHS funding of the therapy Zolgensma in England and Wales.
Novartis Gene Therapies has experience with commercialization and reimbursement of cell and gene therapies. Zolgensma generated revenues of USD 1,370 million in FY 2022. Additionally, Novartis has a strong hold on the manufacturing, with 3 manufacturing facilities in the US.
The above presentation features ten cell and gene therapy companies selected from a pool of over 470 companies that we have compiled. If you are interested, you can download the Sample Report on these topics by Roots Analysis on Global T-Cell (CAR T, TCR, and TIL) Therapy Market and Gene Therapy Market. Further, Investor Series: Opportunities in the Cell and Gene Therapy Market report offers a technical and financial perspective on how the opportunity in this industry is likely to evolve, in terms of future business success, over the coming decade. For personalized assistance in identifying the most relevant solutions based on your specific criteria, please don't hesitate to reach out to us at firstname.lastname@example.org.
Prachi holds an M.Tech, degree in Biotechnology from Indian Institute of Technology, Guwahati and B.E. degree in Biotechnology from Panjab University. Upon completing her postgraduate studies, she embarked on her research career by securing the position of Senior Research Fellow at PGIMER, Chandigarh. In her more than two years of scientific career, she honed her skills in the field of cell and molecular biology, pathology and organic chemistry. Prachi has over two years of remarkable experience in the business research and consulting domain. She is equipped with the distinctive skill set to thrive and contribute immensely to the development of syndicate reports and assignments pertaining to the pharmaceutical and biotechnology industry. She has persistently sought to present impactful, critical insights to the clients through several drug-based, product-based, technology-based and service-based research reports
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