Idinvest Partners is a pan-European private equity manager focused on the low and middle-market segments. With over 6 billion euros under management, the company has developed several complementary areas of expertise, including equity investments in buyout deals focusing on both mid-size as well as young innovative European companies; primary investments in European private equity funds focusing primarily on the middle market segment; secondary investments; mezzanine investments in Europe; and private equity consulting.  Idinvest Partners’ recent success stories include Criteo, Clear2Pay, Meetic, Prosensa, Converteam, Kwik Fit, Lastminute.com, Dailymotion, Deezer, Talend, Synthesio, Menlook, Vestiaire Collective, Pretty Simple, Curse.com, Withings, Kantox, Secret Escapes, Happn, Peakon, Dayuse, Botify, Financefox, Azalead and many more.

Idinvest Partners

BankInvest Biomedical Venture makes investments in research-based biotechnology companies in Europe.

BankInvest Biomedical Venture

ODYSSEE VENTURE is a French Venture Capital and Private Equity firm. It was founded by Mathieu Boillet and Sébastien Sassolas in 1999 and is based in Paris, France. The company has raised over 600M€.

ODYSSEE VENTURE

Andera Partners, formerly known as Edmond de Rothschild Investment Partners (EdRIP) As heir to the traditions and values that have ensured the success of the Rothschild family for 250 years, the Edmond de Rothschild Group has established itself over time as a leading player in European finance. Its activities are grouped around two major areas: Private Banking and Asset Management.  The Group offers wealthy families, entrepreneurs, and large institutions a full range of solutions combining a culture of tailored solutions, stability, performance, and innovation. The Edmond de Rothschild Group’s offering is characterised by strong convictions, with the desire to guarantee to each of its customers: A unique, close relationship, underpinned by the know-how and the variety of expertise of a large international group; Autonomous, experienced teams, who anticipate economic trends and innovate to establish long-term performance; The recognised stability, strength, and independence of a family-owned financial group.  Founded by Baron Edmond de Rothschild in 1953, the Group has been chaired by his son, Baron Benjamin de Rothschild, since 1997.

Andera Partners

Cellectis

Cellectis is a biopharmaceutical company that develops adoptive immunotherapies for cancer. The company’s portfolio includes several lines of proprietary chimeric antigen receptor T-cells targeting individual cancers. Its lead product candidate is UCART19, an engineered T-cell product candidate for the treatment of CD19 expressing hematologic malignancies that developed in acute lymphoblastic leukemia and chronic lymphocytic leukemia. The company’s products also include UCART123 for acute myeloid leukemia indications, and UCART38 and UCARTCS1 for multiple myeloma indications.

<div>France- and US-based Cellectis is a provider of gene-edited therapies to treat patients with cancer. Its therapeutics include editing Chimeric Antigen Receptor (CAR) T-cells to develop immunotherapies that are able to resolve graft-vs-host disease, mitigate treatment rejection, and enhanced patient safety via a T-cell &ldquo;suicide switch.&rdquo; The company edits its therapeutics&rsquo; genetic code through the use of its copyrighted TALEN technology, a class of proteins derived from activator-like effectors that help indicate unique DNA sequences for easier genome editing of cells. The company is listed on the EPA (Euronext Paris) exchange under the ticker symbol ALCLS, the FRA (Frankfurt stock exchange) under the ticker symbol ZVA, and the Nasdaq Global index under the ticker symbol CLLS.</div>

Novel therapies to reboot cells and organs

Cell & Gene Therapy

<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.</div>

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Cellectis: article published in the journal Molecular Therapy

AstraZeneca

To leverage Cellectis' gene-editing technologies to develop up to 10 new cell and gene therapy products in areas of substantial unmet need such as oncology, immunology, and rare diseases.

Chimeric antigen receptor T-cell (CAR-T) therapy involves genetically engineering T cells to target and kill cancer cells

AstraZeneca will leverage the Cellectis proprietary gene editing technologies and manufacturing capabilities, to design novel cell and gene therapy products, strengthening AstraZeneca’s growing offering in this space.

<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>

<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

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 the CRISPR/Cas9 gene-editing technology

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

<div>
 
 France- and US-based Cellectis is a provider of gene-edited therapies to treat patients with cancer. Its therapeutics include editing Chimeric Antigen Receptor (CAR) T-cells to develop immunotherapies that are able to resolve graft-vs-host disease, mitigate treatment rejection, and enhanced patient safety via a T-cell &ldquo;suicide switch.&rdquo; The company edits its therapeutics&rsquo; genetic code through the use of its copyrighted TALEN technology, a class of proteins derived from activator-like effectors that help indicate unique DNA sequences for easier genome editing of cells. The company is listed on the EPA (Euronext Paris) exchange under the ticker symbol ALCLS, the FRA (Frankfurt stock exchange) under the ticker symbol ZVA, and the Nasdaq Global index under the ticker symbol CLLS.
 
 
 As of May 2024, the company had three therapeutics in development; two targeting leukemia, and one for the treatment of non-Hodgkin lymphoma. 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 was also developing therapeutics for Multiple Myeloma, another for Lymphoma, and Renal Cell Carcinoma, which had been licensed out to other pharmaceutical companies.&nbsp;
 
 
 Key customers and partnerships
 
 
 In 
 <a href="https://sp-edge.com/updates/23390">
 November 2023
 </a>
 , Cellectis and AstraZeneca partnered to accelerate the development of gene editing therapeutics, with a focus on oncology, immunology, and rare diseases. The partnership was further extended in 
 <a href="https://sp-edge.com/updates/29145">
 May 2024
 </a>
 with an additional USD 140 million investment from AstraZeneca to develop up to 10 new cell and gene therapy products.&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.&nbsp;
 
 
 Funding and financials
 
 
 The company&rsquo;s latest fundraise was in 
 <a href="https://sp-edge.com/updates/29145">
 May 2024
 </a>
 as part of an ongoing collaboration with AstraZeneca, in which an additional USD 140 million equity investment was made by AstraZeneca resulting in a 44% stake in Cellectis.&nbsp;
 
</div>

<div>
 
 Tenaya Therapeutics is a developer of therapeutics for heart disease. The company develops its therapeutics using precision medicine, gene therapy, and cellular regeneration. Tenaya produces therapeutics that also use proprietary in-vivo and in-vitro models. Additionally, the company has developed in-house adeno-associated virus (AAV) capsid engineering capabilities and has screened over one billion variants from more than 30 diverse, proprietary AAV libraries. The company is publicly listed on the Nasdaq and trades under the ticker symbol TNYA.&nbsp;&nbsp;
 
 
 In October 2023 Tenaya Therapeutics (Tenaya) received FDA clearance for the company&rsquo;s investigational new drug application (TN-401) to initiate clinical testing. TN-401 is Tenaya&rsquo;s adeno-associated virus serotype 9-based investigational gene therapy drug candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy caused by mutations in the plakophilin-2 gene. 
 
 
 As of May 2024, the company&rsquo;s clinical pipeline included two candidates in Phase I clinical trials, a gene therapeutic for the treatment of Hypertrophic Cardiomyopathy, and a small molecule inhibitor for the treatment of heart failure with preserved ejection fraction. Additionally, Tenaya had one gene therapy candidate and two genetic medicine candidates in preclinical testing phases, along with five genetic medicine candidates in the discovery stage. 
 
 
 
</div>

Tenaya Therapeutics

<div>
 
 Sarepta Therapeutics is a gene therapy provider for conditions such as Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy 
 (LGMD), and Charcot-Marie-Tooth Disease. The company&rsquo;s therapeutics incorporate technology related to gene therapy, RNA technologies, and gene editing. The company leverages a proprietary phosphorodiamidate morpholino oligomer (PMO) platform for the development of its RNA-based therapeutics. As of August 2023, the company had four FDA-approved treatments for DMD: ELEVIDYS, EXONDYS 51, VYONDYS 53, and AMONDYS 45. Sarepta Therapeutics is also listed on the Nasdaq, trading under the symbol SRPT.&nbsp;&nbsp;&nbsp;&nbsp;
 
 
 
 
 As of May 2024, the company had several RNA, gene, and gene editing therapeutics in various clinical stages. Sarepta had two RNA therapeutics, both for the treatment of DMD, one in clinical testing, and the other in pre-clinical testing. Additionally, the company had six gene therapeutics, one for the treatment of DMD (both in clinical testing), four for the treatment of LGMD (three in clinical testing, one in preclinical testing) and one for multiple targets (in preclinical testing). Also, Sarepta had two gene editing-based therapeutics in development for the treatment of DMD, both in preclinical testing phases. 
 
 
 
 
 Key customers and partnerships
 
 
 
 
 Over the years since its founding, Sarepta has partnered with several industry and academic partners. The list includes Dyno Therapeutics, Genethon, Genevant Sciences, Hansa Biopharma, Roche, Columbia University, Harvard University, Translational Laboratory of Murdoch University, University of Florida College of Medicine, The Abigail Wexner Research Institute at Nationwide Children&rsquo;s Hospital, University of Massachusetts Medical School, and the University of Western Australia.&nbsp;&nbsp;
 
 
 
 
 In October 2021, Sarepta and healthcare giant Roche announced they would collaborate on carrying out a clinical study of Sarepta&rsquo;s therapeutic candidate SRP-9001 for the treatment of DMD.&nbsp;
 
 
 
 
 Sarepta and pharmaceutical company Catalent announced that in January 2023, the companies were signing a commercial supply agreement. Through the agreement,&nbsp; Catalent would manufacture Sarepta&rsquo;s gene therapeutic for the treatment of DMD; delandistrogene moxeparvovec (SRP-9001).
 
 
 
</div>

Sarepta Therapeutics

<div>
 
 Mesoblast is a cell therapy manufacturer that provides therapeutics for oncology, cardiovascular, orthopedic, hematology, immunity, and inflammation-related conditions. The company is listed on the Australian Stock Exchange and trades under the ticker symbol &ldquo;MSB.&rdquo; Mesoblast develops therapeutics using its proprietary mesenchymal lineage cell technology platform, which is used to produce cryopreserved, off-the-shelf, and cellular-based solutions. The company also incorporates 3D bioreactor technology and automation into its production process to produce higher yields.&nbsp;
 
 
 
 
 As of May 2024, the company had five candidates that are in various stages of clinical testing. Mesoblast&rsquo;s steroid-refractory candidate for Acute Graft Versus Host Disease is currently in late-stage Phase III clinical testing and is the closest candidate to commercialization. The company&rsquo;s drug candidates for acute respiratory distress syndrome, chronic low back pain, and chronic heart failure and reduced ejection fraction are also in Phase III clinical testing while its candidate for inflammatory bowel disease is in Phase II of clinical testing.&nbsp;
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured several agreements with international companies with regard to development and commercialization of Mesoblast&rsquo;s candidates, including Gr&uuml;nenthal GmBH, JCR Pharmaceuticals, Tasly Pharmaceutical Group, TiGenix NV, and the Lonza Group.&nbsp;
 
 
 
 
 Additionally, in April 2020, Mesoblast partnered with The US NIH&rsquo;s National Heart, Lung, and Blood Institute to conduct testing of its allogeneic mesenchymal stem cell (MSC) product candidate, &ldquo;remestemcel-L.&rdquo;&nbsp;&nbsp;&nbsp;&nbsp;
 
 
 
 
 
</div>

Mesoblast

<div>
 
 Ocugen is a gene therapy manufacturer that develops vaccine, cell, and gene-based therapeutics for infectious, eye, and orthopedic diseases. Its cell therapy includes a three dimensional tissue-engineered cartilage disk through the growth of chondrocytes &ndash; the cells responsible for maintaining cartilage health. Its gene therapy solution consists of creating a copy of the patient&rsquo;s gene in order to replace mutated/damaged genes. The company is publicly listed on the Nasdaq, and trades under the symbol &lsquo;OCGN&rsquo;.&nbsp;
 
 
 As of May 2024, the company had five therapeutic solutions being clinically tested. The company was developing four gene therapies for the treatment of retinitis pigmentosa, Leber congenital amaurosis, geographic atrophy, and orphan disease. Its treatment candidate for retinitis pigmentosa was in Phase III of clinical study, while its other gene therapies were in Phase I/II. 
 Ocugen was also developing a biological material, in the IND stage, for diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. The company's cell therapy for the treatment of articular cartilage defects in the knee was in Phase III of clinical study. Finally, the company was also developing three vaccines, all in IND stages, for prevention of diseases caused by the flu, Covid-19, and a combination of the flu and Covid-19.
 
 
 Key customers and partnerships
 
 
 Ocugen partnered with biopharmaceutical manufacturer Jubilant HollisterStier of Spokane in June 2021 to manufacture its Covid-19 therapeutic COVAXIN. Through the partnership, Jubilant would be the potential commercial manufacturer of Ocugen&rsquo;s vaccine for the US and Canadian markets.
 
 
 
 
 
</div>

Ocugen

<div>
 
 MeiraGTx is a developer of gene therapies that aim to treat ocular diseases and problems, neurodegenerative disorders, and salivary gland and autoimmune disorders. MeriaGTx is also developing riboswitch-inducible expression programs for the treatment of inherited obesity and Wilson disease, along with producing small-molecule riboswitch inducers. The company is listed on the Nasdaq and trades under the symbol MGTX.&nbsp;&nbsp;
 
 
 As of May 2024, the company had four gene-based therapeutic candidates in Phase II of their clinical trials for the treatment of (1) xerostomia, Parkinson's Disease, and RPE65-Associated retinal dystrophy and (2) achromatopsia. The company also had one therapeutic being developed to treat Sjogren&rsquo;s Syndrome that was in Phase I/II of its clinical trials. Additionally, MeiraGTx had seven other therapeutics in discovery/preclinical stages. The company was also developing a therapeutic for X-linked retinitis pigmentosa in collaboration with Janssen, which was in Phase III of clinical trials. 
 
 
 Key customers and partnerships
 
 
 MeiraGTx partnered with the International Institute of Risk and Safety Management (IIRSM) in November 2022 to influence risk agendas, raise awareness, the standards of risk, and safety management across the industry.&nbsp;
 
 
 Funding and financials
 
 
 Meira last raised funds in October 2023, when the company secured a strategic investment of USD 30 million from Sanofi. As part of the transaction, Sanofi was granted the right of first negotiation for MeiraGTx's gene regulation technology in certain targets.&nbsp;
 
 
 
</div>

MeiraGTx

Companies producing cells, genes, tissues, and biomaterials such as exosomes and extracellular matrix for drug developers and pharmaceutical companies developing regenerative medicine

Companies conducting R&D on therapies that modify or introduce new genes into the body to treat diseases

Includes therapies that use cells (other than CAR-T and stem cells) such as Adipose-Derived Regenerative Cells (ADRCs), cardiosphere-derived cells, and gamma delta T cells (GDT cells)

Stem cell therapy involves using stem cells usually extracted from the bone marrow and umbilical cord blood to repair a variety of diseases

Startups in the hub consider individual patient profiles, involving variability in genes, environment, and lifestyle

Genomics helps understand the genetic information stored in the DNA to develop targeted treatments

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

<div>
 
 Gene-editing company, Cellectis, has received an additional $140 million in equity investment from AstraZeneca, raising AstraZeneca's stake in Cellectis to 44%. This transaction forms part of the ongoing collaboration between the two firms that began in <a href="https://sp-edge.com/updates/23389"> November 2023 </a>.
 
The investment will further support their joint development of up to ten new cell and gene therapy products. The main focus areas will be oncology, immunology, and rare diseases which have large unmet medical needs. Cellectis stands to gain an option fee and milestone payments ranging from $70 million to $220 million per program.
 
AstraZeneca will have worldwide exclusive license options for the developed candidate products through this collaboration.

</div>

Cellectis

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