Eurazeo is a private equity and venture capital firm that invests in SMEs and manages a diversified portfolio of assets. With its expertise in private equity, real estate, and private debt, the group supports companies of all sizes in their development commitment through the commitment of its 235 professionals. It provides companies with the necessary investments needed to realize their growth potential. The firm was founded in 1969 and is based in Paris, Ile-de-France, France.

Eurazeo

Novartis Venture Fund's primary focus is on the development of novel therapeutics and platforms. They balance the therapeutic focus with investments in medical devices, diagnostics, or drug delivery systems. In their investments, they look for unmet needs and clinical impact, novel proprietary science, an understanding of mechanism, management and board experience, and capital efficiency in the program. Novartis Venture Fund prefers to have their initial investment at the early stage to build the company and follow with additional investment in pace with the company's progress. Their approach involves larger focused investments, with anticipation of total investments up to USD 30 to 50 million per company over its life, but it can be as little as 100,000 USD to get started. They plan to increase their activities to lead or co-lead deals further and remain open to participating in larger syndicates.

Novartis Venture Fund

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 Roche Venture Fund is the name given to the corporate venture fund of the healthcare company Roche. Roche has allocated CHF 500 million to invest in and develop commercially successful innovative life science companies.  Roche has been investing in early-stage companies as part of collaborations since the early-1990s and independent of collaborations since 2002. All equity investments made by Roche in biotech and diagnostics companies (including collaboration investments) are negotiated and managed by the Roche Venture Fund. In the past 20 years, the Roche Venture Fund has invested in over 60 companies globally. Currently, Roche Venture Fund has a portfolio of around 30 companies located in 10 countries across Europe, North America, and the Pacific Region. The fund is an evergreen fund with CHF 500 million available of which approximately 40% is currently invested. The Roche Venture Fund is a committed long-term stable investor with sufficient money reserved in their fund for follow-on financing rounds. As part of a multinational healthcare company, the Roche Venture Fund has access to considerable expertise both internally and externally. We co-invest with leading venture funds, including other corporate venture funds, on a regular basis.

Roche Venture Fund

HealthCap is a family of multi stage venture capital funds, investing globally in life sciences, with the Odlander Fredrikson Group as their exclusive investments advisor. With committed capital exceeding â‚¬ 800 million, HealthCap is one of the largest specialized provider of venture capital within life sciences Europe.

HealthCap

Ysios Capital is a leading Spanish independent venture capital firm that provides private equity financing to early and mid-stage life science companies that develop disruptive therapeutic products and platform technologies. Founded in 2008, Ysios Capital manages c.a. €350 M through three dedicated funds in life sciences and is led by a team of professionals experienced in building biotech companies.

Ysios Capital

Kurma Partners is a management company based in Paris, specializing in the financing of innovation in healthcare and biotechnology, from pre-creation to development capital. Kurma Partners, which manages the Kurma Biofund I, II funds and the Kurma Diagnostics fund, is one of the main players in the financing of therapeutic and medical innovations in Europe, particularly through links with many institutes. research and prestigious hospitals.

Kurma Partners

Vivet Therapeutics

Vivet Therapeutics is dedicated to developing innovative gene therapy treatments for orphan diseases. Vivet Therapeutics is focused on optimizing gene therapy through a partnership with the Fundacion para la Investigacion Medica Aplicada (FIMA) at the Centro de Investigación Medica Aplicada (CIMA, Universidad de Navarra)  to develop new AAV vectors specifically targeting the liver and generating new technologies to optimize gene delivery and long term expression.

France- and Spain-based Vivet Therapeutics is a developer of adeno-associated virus (AAV) gene-based therapies to treat genetic diseases. Specifically, the company is developing therapeutics to treat individuals with Wilson’s disease, Citrullinemia type I, and progressive familial intrahepatic cholestasis (PFIC).

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Wilson Disease Pipeline Market Research Report 2024: Comprehensive Insights on 5+ Drugs Featuring Vivet Therapeutics, Depymed, Prime Medicine, and Deep Genomics

Pfizer

To enter a manufacturing agreement, under which Pfizer will provide clinical supply for a Phase I/II clinical trial evaluating Vivet’s proprietary, investigational gene therapy, VTX-801, for the potential treatment of Wilson disease, a rare and potentially life-threatening liver disorder.

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

To develop a breakthrough medicine that can meaningfully improve the lives of patients with Wilson disease and accelerate Pfizer's leading AAV-directed gene therapy portfolio.

France- and Spain-based Vivet Therapeutics is a developer of adeno-associated virus (AAV) gene-based therapies to treat genetic diseases. Specifically, the company is developing therapeutics to treat individuals with Wilson’s disease, Citrullinemia type I, and progressive familial intrahepatic cholestasis (PFIC).As of May 2024, the company had eight drug candidates. Among them, the company had one candidate, VTX-801, a therapeutic to treat Wilson’s disease. This candidate was in Phase I/II of clinical testing. The company was also developing two therapeutics, targeting Progressive Familial Intrahepatic Cholestasis (PFIC), VTX-802 and VTX-803, both in preclinical stages. Additionally, the company was developing therapeutics targeting Citrullinemia type I and Cerebrotendinous Xanthomatosis (named VTX-804 and VTX-806), which were also in preclinical stages. Key customers and partnershipsVivet signed a manufacturing agreement with pharmaceutical giant Pfizer in September 2020 through which Pfizer would supply Vivet with clinical supplies to support Vivet’s phase I/II clinical trials for VTX-801.Vivet also partnered with biotechnology provider Mirum Pharmaceuticals in March 2021 to enter a global agreement to allow Mirum to develop and subsequently commercialize Vivet’s candidates VTX-802 and VTX-803. Under the agreement, Vivet would continue to carry out the preclinical trial studies, while Mirum had the exclusive option to license the candidates but was required to provide funding for two gene therapy programs of Vivet. Mirum would then take over any clinical development or future commercialization thereafter.

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 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. 
 
 
 
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Tenaya Therapeutics

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 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).
 
 
 
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Sarepta Therapeutics

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 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;
 
 
 
 
 
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Mesoblast

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 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.
 
 
 
 
 
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Ocugen

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 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;
 
 
 
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MeiraGTx

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

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

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

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Vivet Therapeutics, a Paris-based company, has received a grant of EUR 4.9 million (USD 5.3 million) from the French government under the France Health Innovation Plan 2023 managed by Bpifrance. This funding, to be dispensed over three years, will aid in advancing the development of VTX-806, a novel gene therapy for the rare disease, cerebrotendinous xanthomatosis. 

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Vivet Therapeutics

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