Takeda Ventures, Inc. (TVI) is the corporate investment arm of Takeda Pharmaceutical Company Limited (TPC). TVI was founded in 2001 and invests in current and future areas of interest for TPC. They are based alongside Takeda’s R&D sites in Cambridge, MA, and San Diego, CA.  As a strategic investor, TVI aims to provide support and guidance to entrepreneurs and early-stage companies driving concepts through clinical proof of concept. They also strive to forge active, collaborative interactions that result in synergies with Takeda's considerable R&D resources. Their goal is to build a portfolio of strategic investments, with an immediate emphasis on oncology, gastroenterology, neuroscience, and rare diseases.

Takeda Ventures

AdBio partners is a European Life Sciences Venture Capital firm dedicated to seeding healthcare’s step forward. We work with leading research institutions and well-established technology transfer offices, with a strong network of scientific and medical partners, exploring new territories to source breakthrough medical discoveries and drive inventive Life Sciences start-ups to success. We embrace European Life Sciences’ dynamic of innovation. Europe harbors a vibrant environment for medical and scientific investigation, with some of the most leading-edge academic and research centers. AdBio partners seizes the opportunity to highlight their undervalued research programs and assets and participate in structuring Europe’s biotechnology ecosystem.

AdBio partners

Invivo Capital is a team of knowledgeable professionals with the scientific, technical, and financial know-how required to analyze and make investments that provide value to the invested company. They also have a network of contacts and key strategic relationships in the health ecosystem.

Invivo Capital Partners

Columbus Venture Partners is Spanish independent venture capital. It focuses on investing in early-stage opportunities in the life science industry.  The firm is a team of investment professionals with a scientific, medical, and business development background combined with experience in building and investing in companies to accelerate their commercialization.

Columbus Venture Partners

The EIC Accelerator is a Horizon Europe funding program that provides assistance to start-ups and SMEs that have an innovative, game-changing product, service, or business model that has the potential to create new markets or disrupt existing ones, and have the ambition and commitment to scale up, are seeking substantial funding, but the risks involved are too high for private investors to invest alone

EIC Accelerator

Integra Therapeutics

Integra Therapeutics is a developer of next-generation of gene editing tools to cure diseases.

<div>Integra Therapeutics is a Spanish biotechnology company spun-out from the Pompeu Fabra University, specializing in CRISPR-based gene editing tools to accelerate the development of novel therapeutics. It uses its gene writing platform FiCAT, which addresses common concerns associated with gene therapy such as gene size, precision, and stability. The platform is applicable for both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapies. It also uses CRISPR-Cas9 proteins and PiggyBac transposase to precisely engineer and insert DNA, reducing off-target occurrences.&nbsp;</div>

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Integra Therapeutics to receive up to €10.5 million from European Commission for its FiCAT advanced therapies platform

CasZyme

To integrate Caszyme's Cas12l nucleases into Integra Therapeutics' gene-writing platform, enabling the development of safer gene and cell therapies, with Caszyme receiving up to EUR 40 million (~USD 43.5 million) in milestone payments and royalties.

Companies that develop novel therapeutics based on the CRISPR/Cas9 gene-editing technology

<div>
 
 Integra Therapeutics is a Spanish biotechnology company spun-out from the Pompeu Fabra University, specializing in CRISPR-based gene editing tools to accelerate the development of novel therapeutics. It uses its gene writing platform FiCAT, which addresses common concerns associated with gene therapy such as gene size, precision, and stability. The platform is applicable for both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapies. It also uses CRISPR-Cas9 proteins and PiggyBac transposase to precisely engineer and insert DNA, reducing off-target occurrences.&nbsp;
 
 
 The company considers its ability to write large messages into genomes using its tech to be its edge. As fo June 2022, it had not disclosed its target indications and intended to work together with its funding investors on its plans going forward.&nbsp;
 
 
 Key customers and partnerships
 
 
 
 In March 2023, Integra partnered with 
 <a href="https://sp-edge.com/companies/714815">
 Predictive Oncology
 </a>
 to advance gene therapy. Through the partnership, the companies would collaborate to help Integra Therapeutics to enhance the way it develops therapeutics for cancer.
 
 
 Funding and financials
 
 
 It raised EUR 6 million (USD 6.3 million) in its last 
 <a href="https://www.biospace.com/article/integra-therapeutics-closes-a-new-investment-of-1-5-million-with-the-addition-of-columbus-venture-partners-to-seed-round/">
 seed round
 </a>
 , which closed in March 2022 and was led by AdBio Partners, Invivo Capital, and Takeda Ventures. The funds were earmarked to further develop Integra&rsquo;s platform and technologies.
 
</div>

<div>
 
 Editas Medicine specializes in gene editing to develop novel therapeutics for a variety of areas including oncology, ocular diseases, and blood diseases. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 and Cas12a enzymes.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, Editas has seven gene editing programs in its pipeline.&nbsp;
 
 
 One of the company&rsquo;s candidates was in the late clinical trial stages as of March 2024. The Phase I/II clinical trials on Editas&rsquo; in vivo gene editing therapy EDIT-101 have been ongoing since 2019 and the company announced that it had achieved proof of concept and identified a responder population in November 2022. For the first time ever (in April 2022), the company delivered its experimental in vivo CRISPR therapy to a 
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-dosing-first-pediatric-patient">
 pediatric patient
 </a>
 to treat Leber congenital amaurosis 10 (LCA10), an inherited early childhood eye disease. Its ex vivo gene editing therapy EDIT-301 holds two rare pediatric disease designations granted by the FDA for transfusion-dependent beta-thalassemia and sickle cell disease. The candidate also received 
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-receives-fda-orphan-drug-designation-edit-301">
 orphan drug designation
 </a>
 in May 2022 for beta-thalassemia.
 
 
 Key customers and partnerships
 
 
 Founded in September 2013, Editas has secured several collaborations with other pharmaceutical and drug developers including 
 <a href="https://www.catalent.com/catalent-news/catalent-enters-into-strategic-partnership-with-editas-medicine-to-support-gene-editing-medicine-pipeline/">
 Catalent
 </a>
 (for therapy development and manufacturing support),
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/adverum-biotechnologies-and-editas-medicine-announce">
 Adverum Biotechnologies 
 </a>
 (to develop technologies to deliver gene editing medicines to the eye), 
 <a href="https://www.askbio.com/editas-medicine-and-askbio-enter-strategic-research-collaboration-to-explore-in-vivo-delivery-of-genome-editing-medicines-to-treat-neurological-diseases/">
 Asklepios BioPharmaceutical
 </a>
 (AskBio) (to research in vivo gene editing therapies for neurological diseases),
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-and-bluerock-therapeutics-enter-strategic">
 BlueRock Therapeutics
 </a>
 (to develop medicines by combining cell therapy and gene editing technologies), and big pharma such as Bristol-Myers Squibb (to develop cell therapies for oncology).&nbsp;
 
 
 The company expanded its partnership with Azzur Cleanrooms on Demand (COD) in July 2023 with regard to continuing to manufacture Editas' candidate EDIT-301. 
 
 
 Funding and financials
 
 
 The company last raised funds in June 2023 when the company raised USD 125 million in a common stock offering. Editas Medicine did not disclose what the raised funds would be used for. The company has been listed on the Nasdaq since February 2016 and was the first CRISPR gene editing company to go public. 
 
</div>

Editas Medicine

<div>
 
 CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, the company had 18 drug candidates in its pipeline. 
 
 
 In a milestone event in November 2023, the company&rsquo;s gene-editing therapy exagamglogene autotemcel, CASGEVY 
 <a href="https://sp-edge.com/updates/24093">
 (exa-cel)
 </a>
 , developed with Vertex Pharmaceuticals to treat sickle-cell disease and beta-thalassemia, received the world&rsquo;s first authorization from the UK's Medicines and Healthcare products, Regulatory Agency (MHRA). In December 2023, it received FDA approval for patients aged 12 and older. The drug for beta-thalassemia patients is also nearing its market entry in the US, with the FDA&rsquo;s decision expected on March 30, 2024.&nbsp;
 
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured strategic collaborations with several companies to co-develop and co-commercialize gene editing medicines that include 
 <a href="https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-and-crispr-therapeutics-amend">
 Vertex Pharmaceuticals
 </a>
 (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and 
 <a href="https://viacyte.com/press-releases/crispr-therapeutics-and-viacyte-announce-strategic-collaboration-to-develop-gene-edited-stem-cell-derived-therapy-for-diabetes/">
 ViaCyte
 </a>
 (September 2018) to develop VCTX210, a regenerative medicine for diabetes.
 
 
 Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.&nbsp;&nbsp;
 
 
 
 
 
 Funding and financials
 
 
 
 
 Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.
 
 
 
 
 
</div>

CRISPR Therapeutics

<div>
 
 Intellia Therapeutics specializes in gene editing to develop novel therapeutics for various areas, including immuno-oncology, autoimmune, and genetic diseases. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, it was working on 3 internal drug programs and four collaborative programs. As of March 2024, one program was in the late clinical stage, one program was in the early clinical stage, and five were in the research and preclinical stage. The company considers its lipid nanoparticle delivery system to be a key strength in minimizing genotoxicity risk and random insertion.
 
 
 Intellia acquired biotechnology company Rewrite Therapeutics for USD 45 million in February 2022 to leverage its gene editing and DNA-writing technologies. Intellia would also pay up to USD 155 million in additional milestone payments.
 
 
 
 
 
 Key customers and partnerships
 
 
 Intellia has partnered with a number of biotech and pharma companies, including: 
 1) Big pharma Novartis (January 2015), to research and co-develop CRISPR/Cas9-edited therapies to develop an ex-vivo treatment for sickle-cell disease and a number of undisclosed programs 
 2) Kyverna Therapeutics (January 2022), for the development of a next-generation CD19 CAR T-cell therapy for autoimmune diseases 
 3) ONK Therapeutics (February 2022), to develop engineered natural killer (NK) cell therapies for cancer treatment.&nbsp;
 
 
 In October 2023, the company expanded its research collaboration with 
 <a href="https://sp-edge.com/updates/22377">
 Regeneron Pharmaceuticals
 </a>
 (entered in April 2020) to develop gene-editing therapies for neurological and muscular diseases.
 
 
 
 
 
 Funding and financials
 
 
 Founded by Caribou Biosciences and Atlas Ventures in November 2014, Intellia has been listed on Nasdaq since May 2016. In 
 <a href="https://sp-edge.com/updates/15140">
 November 2022
 </a>
 , the company raised USD 300 million via an underwritten public offering&mdash;it did not disclose the purpose of the financing. For 2022, the company reported a revenue of ~USD 52&nbsp; million, earned through collaborations.
 
</div>

Intellia Therapeutics

<div>
 
 Based in the US and France, Cellectis develops off-the-shelf gene editing cancer immunotherapies based on allogeneic Chimeric Antigen Receptors T (CAR-T) cells dubbed UCARTs by leveraging the transcription activator-like effector nucleases (TALEN) technology.
 
 
 As of March 2024, the company had seven therapeutics in development; two targeting Leukemia, one for the treatment of non-Hodgkin lymphoma, one for Large B Cell Lymphoma, two for Multiple Myelnoma, and one for Renal Cell Carcinoma. Its Leukemia therapeutics were in Phase I of clinical trials, while its non-Hodgkin lymphoma solution was just entering Phase I of clinical trials. The company's therapeutics for Multiple Myeloma, another for Lymphoma, and Renal Cell Carcinoma, had been licensed out to other pharmaceutical companies.&nbsp;
 
 
 
 
 Key customers and partnerships
 
 
 In 
 <a href="https://sp-edge.com/updates/23389">
 November 2023
 </a>
 , Cellectis entered into a partnership with AstraZeneca to accelerate the development of gene editing therapeutics, with a focus on oncology, immunology, and rare diseases.&nbsp;
 
 
 Cellectis also partnered with Primera in January 2023 to develop in-vivo gene therapies for mitochondrial disease. Through the partnership, both entities would collaborate to genetically edit mutations in mitochondrial DNA and develop an engineering toolbox that can be used to develop therapeutics for mitochondrial diseases. 
 
 
 
 
 
 
 Funding and financials
 
 
 The company entered into several strategic collaboration agreements with AstraZeneca in November 2023. These agreements included an investment agreement relating to an initial equity investment of USD 80 million by subscribing for 16 million ordinary shares. Cellectis stated that it planned to use the proceeds received to develop gene editing tools, for research and development expenses incurred in developing its programs, and other general corporate purposes.
 
 
 
</div>

Cellectis

Caribou Biosciences specializes in gene editing to develop novel therapeutics for hematologic oncology indications using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops allogeneic off-the-shelf gene edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells and CAR Natural Killer (CAR-NK) cells.The company leverages its proprietary platform CRISPR hybrid RNA-DNA (chRDNA), which uses hybrid guides—both ribonucleic acid (RNA) and DNA nucleotides—to edit DNA. As of March 2024, Caribou had four candidates in its clinical pipeline, of which its lead candidate CB-010, a CAR-T therapy to treat relapsed/refractory B cell non-Hodgkin lymphoma, was in the Phase I clinical trial stage (March 2024). Its other CAR-T candidates, CB-011 for the treatment of relapsed/refractory Multiple Myeloma and CB-012, for the treatment of relapsed/refractory Acute Myeloid Leukemia, were also in Phase I of clinical trials. Its alternate application for CB-010 to treat autoimmune diseases was still in the discovery stage.The company intended to extend ex vivo (gene editing done in a specialized laboratory and returned to the body) therapies toward other indications outside oncology and implement its Cas12a chRDNA platform in in vivo (gene editing done inside the body) applications.Key customers and partnershipsThe company entered into a partnership with big pharma company <a href="https://news.abbvie.com/news/press-releases/abbvie-and-caribou-biosciences-announce-collaboration-and-license-agreement-for-car-t-cell-products.htm">Abbvie </a>in February 2021 for licensing and co-development of two CAR-T therapies. Caribou also entered into licensing agreements with <a href="https://www.businesswire.com/news/home/20201118005128/en/Caribou-Biosciences-Announces-Licensing-Agreement-for-scFvs-Targeting-CD371-to-Enable-Development-of-Allogeneic-Cell-Therapies">Memorial Sloan Kettering Cancer Center </a>(MSKCC) (November 2020), Corteva Agriscience, and Intellia Therapeutics. It also has a sales and assignment agreement with ProMab Biotechnologies Inc. (February 2020).Funding and financialsCaribou Biosciences went public on the Nasdaq Global Select Market in July 2021 and raised USD 304 million. The IPO proceeds were earmarked to fuel the company’s clinical development. For 2021, it recorded revenue of USD 9.6 million earned through licensing and collaborations.The company last raised funding in July 2023, when it raised USD 125 million in an upsized underwritten public offering. Caribou did not disclose what the funds would be used for.

Caribou Biosciences

Companies that develop novel therapeutics based on other types of gene editing technologies including TALEN and ZFN

Companies that develop systems that enable the diagnosis of gene-edited therapies. Also includes systems that deliver gene-editing components into the human body

Companies that offer their gene-editing platforms to other pharma companies to develop therapeutics

Companies that offer support services to gene-editing companies, which include genomic research companies, gene-editing component manufacturing, and designing tools

Startups in the hub develop gene-editing treatments to cater to specific patients and patient profiles

Genomics enables precise changes in the DNA sequence of living organisms to develop treatments

PROMINENT_CATEGORY

<div>

Spanish biotechnology firm Integra Therapeutics and Lithuanian gene-editing firm Caszyme have partnered to use Cas12l nucleases for the development of safer gene and cell therapies. Caszyme stands to receive up to EUR 40 million in milestone payments and sales royalties.

As part of the collaboration, Integra Therapeutics will incorporate Caszyme’s Cas12l nucleases into its FiCAT 2.0 gene-writing platform. Cas12l, a CRISPR nuclease family, is approximately 850 amino acids. The partnership will enable the use of these tools in therapy advancement following positive results from in vivo and ex vivo studies. This could improve genetic and oncological disease applications and therapies.

</div>

Integra Therapeutics

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