OrbiMed is a healthcare-dedicated investment firm, with approximately $5 billion in assets under management. OrbiMed's investment advisory business was founded in 1989 with a vision to invest across the spectrum of healthcare companies: from private start-ups to large multinational companies. OrbiMed manages the Caduceus Private Investments series of venture capital funds and a family of public equity investment funds.

OrbiMed

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

RA Capital Management is an investment advisor based in Boston specializing in the life-sciences and drug development sectors. The company's team has been investing since 2002 and is comprised of professionals with training in biology, chemistry, and medicine and also has industry and business development experience at the executive and board levels. The company invests in companies with promising technologies and products.

RA Capital Management

Vida Ventures’ mission is to bring science to life by advancing transformative biomedical innovations that have the potential to make a meaningful impact on the lives of patients. They want to do well by doing good for others. Vida Ventures is a bicoastal life sciences company building and investment firm, founded in 2017 by a group of scientists, physicians, entrepreneurs, and investors who are passionate about building and funding breakthroughs in biomedicine  .

Vida Ventures

Pfizer Venture Investments is a corporate venture capital firm that invests in areas of current or future strategic interest to Pfizer. It aims to remain at the forefront of life science advances. It identifies and invests in emerging companies that are developing transformative medicines and technologies that have the potential to enhance Pfizer’s pipeline and shape the future of its industry.

Pfizer Venture Investments

Polaris Partners invests in healthcare and biotechnology companies. Their healthcare portfolio companies' care delivery models include digital health, consumer-centric businesses, patient-provider solutions, data science, and analytics. Their life sciences and biotechnology portfolio companies focus on molecules and platforms, therapeutics, and genomics.

Polaris Partners

Bristol-Myers Squibb Company, through its subsidiaries, engages in the discovery, development, licensing, manufacturing, marketing, distribution, and sale of pharmaceuticals and nutritional products.  Bristol Myers Squibb focuses on areas such as oncology, cardiovascular, immunoscience, and fibrosis to develop therapies that address significant unmet medical needs. The company's research and development efforts are centered around discovering new drugs and advancing existing ones to improve patient outcomes. BMS has a diverse portfolio of medicines, including several blockbuster drugs used in the treatment of cancer and other diseases. The company is committed to advancing the science of medicine to help people fight serious diseases and improve their quality of life.

Bristol-Myers Squibb

Alexandria Venture Investments is a venture capital firm that develops work in real estate technology, and healthcare sectors. The company's strategic venture capital platform is Alexandria Venture Investments. Since its inception in 1996, the company has invested in disruptive life science, agrifood tech, and technology companies that are developing transformative new modalities and platforms to meaningfully improve human health.

Alexandria Venture Investments

Leaps by Bayer invests in paradigm-shifting advances in the life sciences and specializes in the fields of biotechnology, agriculture, and scientific breakthrough. The firm's approach is making significant and sustained investments in disruptive biotechnologies that could have the greatest impact on humanity. It was founded in 2015 and headquartered in Leverkusen, Nordrhein-Westfalen.

Leaps by Bayer

Eli Lilly & Company Foundation is an organization that supports programs designed to improve patient outcomes and enhance the quality of life. The organization makes strategic and philanthropic investments consistent with Lilly’s purpose. It also supports improving educational opportunities for children living in underserved communities in Indianapolis.  Eli Lilly & Company Foundation focuses on early childhood education, supporting quality schools and STEM (science, technology, engineering, and math) education.  The foundation was established in 1968 and is based in Indianapolis, Indiana.

Eli Lilly & Company Foundation

Sofinnova Ventures has partnered with entrepreneurs to secure initial funding, build successful teams, win key customers, and navigate acquisitions and IPOs. Sofinnova Ventures invests in Life Science and Technology start-ups. Their professionals are seasoned business operators, possessing deep domain and investment expertise. They know how to help entrepreneurs build successful, global businesses.

Sofinnova Investments

Forbion Capital Partners is a Netherlands-based venture capital firm focused on investing in life sciences companies in drug development as well as MedTech companies addressing high medical needs. Forbion's investment team of nine investment professionals has built an impressive performance track record since the late nineties with successful investments in Rhein Biotech, Crucell, Neutec, Glycart, Borean, Impella, Alantos, Acorda, Fovea and PanGenetics. Current assets under management exceed â‚¬400m ($500m), split between three active funds. Finally, Forbion co-manages BioGeneration Ventures, an early stage fund focused on academic spin-outs and seed investments in the Netherlands.

Forbion Capital Partners

Perceptive Advisors is a privately owned hedge fund sponsor. The firm invests in the public equity markets across the globe. It employs a long/short equity strategy as a hedging technique while investing. The firm typically invests in the biotechnology and life sciences sectors to make its investments. Perceptive Advisors is based in New York, New York.

Perceptive Advisors

Johnson & Johnson Innovation accelerates early-stage innovation through strategic collaboration. It connects potential collaborators to resources with the goal of assisting them in advancing transformation science and technology across the pharmaceutical, medical device, and consumer healthcare spectrums.

Johnson & Johnson Innovation – JJDC

Mubadala Capital’s Ventures business builds on Mubadala’s 10-year track record of being an active investor in advanced technology. The Ventures platform includes a direct investment business, a fund of funds business and the management and oversight of Mubadala’s partnership with SoftBank, including its U.S. $15 billion LP commitment to the SoftBank Vision Fund.  The Ventures team is based in Abu Dhabi, San Francisco, and London with strong connectivity to European and Chinese venture capital markets through other Mubadala Capital managed vehicles.

Mubadala Capital Ventures

Capstan Therapeutics

Capstan Therapeutics is advancing precision in vivo cell engineering to develop therapeutics for a broad range of disease categories with unmet or underserved clinical need.

Capstan Therapeutics is a biotechnology company dedicated to advancing in vivo reprogramming of cells through RNA delivery using targeted lipid nanoparticles (tLNP) technology. The company's proprietary CellSeeker tLNP platform consists of lipid nanoparticles conjugated with a recombinant protein binder, such as a monoclonal antibody, designed to deliver payloads including mRNA or gene editing tools capable of reprogramming specific cell types in vivo. Through this platform, Capstan aims to create therapies that can program immune cells directly inside the body rather than engineering them in a lab. The company's lead candidate, CPTX2309, delivers an mRNA payload encoding for an anti-CD19 CAR to CD8-expressing T cells, effectively engineering CAR-T cells in vivo. The therapeutic goal is to achieve rapid deep B cell depletion in both blood and lymphoid tissues, without the challenges of conventional ex vivo CAR-T. preclinical data presented in October 2023 demonstrated rapid and robust anti-tumor and anti-primary B cell activity in humanized mice through tLNP administration delivering mRNA that encodes an anti-CD19 CAR. Capstan's technology has the potential to generate transformative therapies with applications across autoimmune disorders, oncology, fibrosis, and monogenic blood disorders.

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Capstan Therapeutics Presents Preclinical Data on Lead In Vivo CAR-T Candidate, CPTX2309, at American College of Rheumatology (ACR) Convergence 2024

Capstan Therapeutics is a biotechnology company dedicated to advancing in vivo reprogramming of cells through RNA delivery using targeted lipid nanoparticles (tLNP) technology. The company's proprietary CellSeeker tLNP platform consists of lipid nanoparticles conjugated with a recombinant protein binder, such as a monoclonal antibody, designed to deliver payloads including mRNA or gene editing tools capable of reprogramming specific cell types in vivo. Through this platform, Capstan aims to create therapies that can program immune cells directly inside the body rather than engineering them in a lab. The company's lead candidate, CPTX2309, delivers an mRNA payload encoding for an anti-CD19 CAR to CD8-expressing T cells, effectively engineering CAR-T cells in vivo. The therapeutic goal is to achieve rapid deep B cell depletion in both blood and lymphoid tissues, without the challenges of conventional ex vivo CAR-T. preclinical data presented in October 2023 demonstrated rapid and robust anti-tumor and anti-primary B cell activity in humanized mice through tLNP administration delivering mRNA that encodes an anti-CD19 CAR. Capstan's technology has the potential to generate transformative therapies with applications across autoimmune disorders, oncology, fibrosis, and monogenic blood disorders.The company's approach could make cell therapy more efficient and potentially less costly to manufacture compared to current ex vivo CAR-T therapies. With in vivo CAR-T, patients would not need chemotherapy preconditioning, and treatments could potentially be administered in an outpatient clinic setting rather than requiring specialized centers.Key customers and partnershipsIn March 2024, Capstan formed multiple new partnerships with investors including Forbion, Johnson &amp; Johnson Innovation - JJDC, Mubadala Capital, Perceptive Advisors, and Sofinnova Investments. The company has also maintained ongoing partnerships with existing investors including Alexandria Venture Investments, Bristol Myers Squibb, Eli Lilly and Company, Leaps by Bayer, Novartis Venture Fund, OrbiMed, Pfizer Ventures, Polaris Partners, and Vida Ventures.

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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

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

Capstan Therapeutics

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