F-Prime's roots are in one of America’s great entrepreneurial success stories. Fidelity Investments was founded in 1946 and grew from a single mutual fund into one of the largest asset management firms in the world, with over $2 trillion in assets under management. For the last forty years, this venture capital group has had the privilege of backing other great entrepreneurs as they built ground-breaking companies in technology and life sciences, including Atari, MCI, ROLM Corp., Alibaba, Ironwood Pharmaceuticals, and Ultragenyx.  Today, F-Prime's funds are larger and more global, but its teams are still small and local. F-Prime stays true to its entrepreneurial roots. In the US and Europe, F-Prime Capital Partners is investing in healthcare (formerly Fidelity Biosciences) and in technology (formerly part of Devonshire Investors). In other geographies, its sister fund is called Eight Roads (formerly Fidelity Growth Partners), with investment teams in London, Shanghai, Beijing, Hong Kong, Tokyo, and Mumbai.   Together F-Prime brings a world of insight, domain expertise and relationships to support entrepreneurs. Without the pressure of fundraising from outside investors, F-Prime Capital Partners focuses all of its time finding and helping great entrepreneurs build important companies.

F-Prime Capital

The UCL Technology Fund is dedicated to investing in intellectual property commercialisation opportunities arising from UCL’s world-class research base, focusing in particular on the physical and life sciences. The Fund supports UCL in achieving the full potential of innovations that have prospects for outstanding societal and market impact, right through the development journey from initial proof of concept to practical commercial application.

UCL Technology Fund

AlbionVC supports visionary founders with long-term capital and scale-up expertise. Founded in 1996, today we are partners to UK early-stage B2B software and healthcare companies. AlbionVC manages c. $1 billion of venture funds, currently invested in over 50 companies. AlbionVC is the technology investment arm of Albion Capital Group LLP, which is authorised and regulated by the Financial Conduct Authority.

AlbionVC

Baillie Gifford is an independently owned investment management firm. It is owned and run by 39 of its senior executives, who operate as a partnership, a structure that has endured for over a century and provides stability for clients and motivation for employees. It enables them to manage the business for the future as well as the present, with an emphasis on genuine long-term thinking rather than lurching haplessly between short-term targets.

Baillie Gifford

ORI Fund is a venture capital fund that invests in innovative companies with disruptive technologies in the global healthcare industry. The fund is founded by Ms. Simone Song, former investment banker and head of China Healthcare at Goldman Sachs. The team consists mostly of former Goldman Sachs bankers and healthcare scientists. The Fund is supported by a network of most renowned scientists, practitioners, and entrepreneurs.

ORI Capital

Temasek Holdings is a lender provider. The company offers debt financing and focuses on transforming economies, increasing middle-income populations, developing comparative advantages, and emerging champions. Financial services, transportation, industrial, consumer, real estate, telecommunications, media, technology, and life sciences are among the industries served by the firm.

Temasek Holdings

Cowen Group is a financial services firm that provides equity and debt financings, investment banking, and other financial-related services. It also offers mergers and acquisitions, and strategic restructuring advisory services. The firm's sector specializes in healthcare, technology, media and telecommunications, information and technology services, consumer, energy, aerospace and eCommerce, industrials, transportation, and biotechnology industries. Founded in 1918, Cowen also has an investment management division that offers actively managed alternative investment products.

Cowen Group

4BIO Ventures is a London-based firm investing in venture capital and public equities in life sciences. They are focused on advanced therapies addressing high unmet medical and social needs. 4BIO Capital Partners is an emerging asset manager focusing on private equity and venture capital in high growth and high return segments of biotech - biologics, gene and cell therapy and bioinformatics.

4BIO Capital

RTW Investments makes investments in forward-thinking healthcare companies. It invests across the entire company lifecycle, including company creation, early-stage venture capital, alternative financing, and public company investments.  The company is headquartered in New York with offices in London and Shanghai.

RTW Investments

Juda Capital

Agent Capital

Pavilion Capital operates as a private equity firm under Temasek Holdings. Pavilion Capital invests primarily in privately owned firms in North Asia.

Pavilion Capital

Deerfield Capital Management (''DCM'') is a global fixed-income investment adviser that has managed institutional, alternative accounts since 1993.

Deerfield Capital Management

Venrock continues a tradition of partnering with entrepreneurs to establish successful, enduring companies. With a primary focus on technology and healthcare, portfolio companies have included Apple Computer, Athenahealth, Centocor, Check Point Software, DoubleClick, Endeca, Gilead Sciences, Idec Pharma, Imperva, Illumina, Intel, Nest, SlideShare, and Tudou.

Venrock

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

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

Foresite Capital is a healthcare and life sciences investment firm. They take a data-driven approach to invest in startups that employ biology and big data to improve healthcare, such as 10x Genomics, Relay Therapeutics, Element Biosciences, and Inscripta.

Foresite Capital

ArrowMark Partners is an SEC-registered investment adviser. It aims to provide consistent, asymmetric, non-correlated, risk-adjusted returns by utilizing fundamental research across the capital structure. They are committed to compounding wealth by providing one-of-a-kind, client-driven, and absolute return solutions.

ArrowMark Partners

Driehaus is an investment management firm that manages growth equity investment strategies and multi-asset alternative strategies. The firm has a diverse institutional client base comprised of corporate and public pensions, endowments, foundations, sub-advisory, family offices, wealth managers, and financial advisors, globally. Driehaus is a performance-oriented investment management boutique that emphasizes integrity, transparency, and the alignment of the firm’s interests with its clients. The firm was founded in 1982 and is headquartered in Chicago, Illinois.

Driehaus Capital Management

Cormorant Asset Management is an employee-owned hedge fund sponsor that provides its services to pooled investment vehicles. The firm also focuses on both public and private market innovative companies in the biotechnology and life sciences marketplace.

Cormorant Asset Management

Sphera Global Healthcare Management (Investment Manager) is a partnership between Mr. Mori Arkin, former vice chairman of Perrigo (Nasdaq ticker:PRGO, a world leader in generic and OTC drugs), and of Sphera Funds Management.  It is a long/short equity fund that is focused on the global pharmaceutical and biotech industries. The Investment Manager’s objective is to generate consistent and attractive long term returns exceeding the industry while maintaining disciplined risk management. With a highly experienced and cohesive multidisciplinary team, the Fund’s competitive advantage is evident in three key areas:  • Having a rigorous, fundamental, focused and comprehensive approach to healthcare research.  • Investment committee co- led by an industry veteran with a track record of building a pharmaceutical company.  • Unique and unbiased investment approach generated in part from access to massive flow of R &D, innovation and top tier human capital in Israel.

Sphera Global Healthcare Fund

Cowen Healthcare Investments invests fiduciary capital in healthcare companies across the biopharma, diagnostics and digital health sectors.

Cowen Healthcare Investments

Ghost Tree Capital Group is a venture capital firm.

Ghost Tree Capital Group

Medison Ventures’ edge lies in access to over twenty years of clinical and commercial know-how accumulated by Medison

Medison Ventures

Orchard Therapeutics

Orchard Therapeutics is a fully integrated commercial-stage company dedicated to treating the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first autologous ex vivo gene therapy approved by the EMA in 2016, 3 programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott-Aldrich syndrome), and ADA-SCID (adenosine deaminase severe combined immunodeficiency), 2 other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline. The company is partnered with world-leading institutions in gene therapy, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele.

<div>UK-based Orchard Therapeutics focuses on developing gene therapies, specifically hematopoietic stem cell (HSC) gene therapies for serious and life-threatening orphan diseases and rare genetic diseases.&nbsp;</div>

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Orchard Therapeutics Announces Multiple Data Presentations and Publications

<div>
 
 UK-based Orchard Therapeutics focuses on developing gene therapies, specifically hematopoietic stem cell (HSC) gene therapies for serious and life-threatening orphan diseases and rare genetic diseases.&nbsp;
 
 
 As of May 2024, the company&rsquo;s pipeline consisted of five drug candidates focused on neurodegenerative and immunological disorders. Its lead candidate received FDA approval in the US in 
 <a href="https://sp-edge.com/updates/27732">
 March 2024
 </a>
 under the brand name Libmeldy to treat children suffering from metachromatic leukodystrophy (MLD), a rare genetic disease. The therapy is also marketed in the European Union under the brand name Libmeldy since 
 <a href="https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-receives-ec-approval-libmeldytm-treatment">
 December 2020
 </a>
 .
 
 
 It also has programs targeting mucopolysaccharidosis type I and IIIA, frontotemporal dementia, and hereditary angioedema. Orchard Therapeutics is a subsidiary of Kyowa Kirin Co. (since 
 <a href="https://www.kyowakirin.com/media_center/news_releases/2024/pdf/e20240124_02.pdf">
 January 2024
 </a>
 ).
 
 
 Key customers and partnerships
 
 
 Orchard has established partnerships to advance its gene therapy research and commercial outreach. Notably, in January 2021, it expanded its commercial reach in the Middle East and Turkey through exclusive agreements with 
 <a href="https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-secures-partnerships-broaden-access">
 GenPharm Services and GEN
 </a>
 , specializing in rare genetic diseases. The company also entered an agreement with Beneluxa Initiative on Pharmaceutical Policy (
 <a href="https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-agreement-beneluxa-consortium">
 Beneluxa
 </a>
 ) in January 2024 to enable reimbursed access to Libmeldy across several member countries, including Belgium, the Netherlands, and Ireland.&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

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

PROMINENT_CATEGORY

<div>
 
 Orchard Therapeutics, a gene therapy specialist, is partnering with Annogen, an expert in gene expression engineering. The focus of their collaboration is to find immune cell-specific human promoters. These will be used in Orchard's pre-clinical gene therapy initiatives which deal with hematopoietic stem cells (HSCs). The particulars of the agreement, including financial aspects, remain undisclosed.
 Annogen's proprietary SuRE™ technology will analyze the human regulatory genome. Here, the goal is to identify DNA active in distinct cell types and conditions. Ideally, this will result in the discovery of the most effective hybrid promoter that could influence gene expression in a manner specific to certain cells or diseases. This research can greatly benefit Orchard's HSC gene therapy platform. It could lead to its application to a wider range of diseases and potentially enhance certain pre-clinical programs.&nbsp;
 
</div>

Orchard Therapeutics

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