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

Advent Life Sciences is a venture team investing in life sciences businesses. The team consists of 11 professionals with extensive scientific, medical and operational experience, and a long-standing track record of entrepreneurial and investment success across the UK, Europe and the US. The Firm invests in a range of sectors within life sciences, principally in new drug discovery, enabling technologies, and med tech. Realizations in the last three years include Algeta, Avila, CN Creative, EUSA, Micromet. Current investments include Acutus, Biocartis, Cellnovo, f2G, NeRRe,

Advent Life Sciences

The Dementia Discovery Fund (DDF) is the world’s largest family of specialized venture capital funds that invests exclusively in companies developing or enabling novel therapeutics for dementia, managed by SV Health Investors. Dementias including Alzheimer’s Disease are arguably the largest unmet medical need with over 55m patients worldwide. With more than $500m raised for this strategy, and offices in London and Boston, DDF capitalizes on global investment opportunities to fulfill its dual mandate of delivering measurable impact and generating significant financial returns. Utilizing its network of venture partners, entrepreneurs, leading scientists, and strategic partners, DDF invests in and creates new biotech companies and provides thought leadership in the field. DDF is enabled by its limited partners including major pharmaceutical companies (Biogen, Bristol Myers Squibb, Eli Lilly and Co., GSK, Johnson & Johnson, Otsuka (Astex), Pfizer and Takeda), along with AARP, Aegon, Bill Gates, British Patient Capital, NFL Players Association, Quest Diagnostics, UnitedHealth Group, and the non-profits Alzheimer’s Research UK and LifeArc.

Dementia Discovery Fund

LifeArc is an independent life science medical research charity committed to drastically improving positive patient outcomes everywhere. It specializes in the fields of biotechnology, healthcare, and medical. It was founded in 1992 and headquartered in London, England.

LifeArc

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

New Enterprise Associates is a prominent venture capital firm focusing on investments in technology and healthcare startups. They help entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With over $19 billion in cumulative committed capital since the firm’s founding in 1977, NEA invests in technology and healthcare companies at all stages in a company’s lifecycle, from the seed stage through the IPO. The firm's long track record of successful investing includes more than 210 portfolio company IPOs and more than 360 acquisitions.

New Enterprise Associates

Monograph Capital is a life sciences venture firm. We find and fund groundbreaking life sciences companies to transform promising science into real, life-changing treatments for people across the world.

Monograph Capital

EQT life science develops innovative medicines, devices, and tools to address the single important thing that matters to the preservation of health. The organization believes that such opportunities add value to all stakeholders.

EQT Life Sciences

AviadoBio

AviadoBio mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute.

UK-based AviadoBio is a developer of gene-based therapeutics that target neurodegenerative disorders. Specifically, the company develops therapeutics that help treat patients with conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). AviadoBio’s platform uses therapeutics based on gene supplementation and gene knock-down and is based on research carried out at King’s College London and the UK Dementia Research Institute.

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Astellas and AviadoBio announce exclusive gene therapy deal

UK-based AviadoBio is a developer of gene-based therapeutics that target neurodegenerative disorders. Specifically, the company develops therapeutics that help treat patients with conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). AviadoBio’s platform uses therapeutics based on gene supplementation and gene knock-down and is based on research carried out at King’s College London and the UK Dementia Research Institute.As of May 2024, the company had five therapeutic candidates; its lead candidate, AVB-101 for the testing of frontotemporal dementia with GRN mutations, was in its early clinical testing phase. The company’s other candidates were all in discovery stages for the treatment of ALS and Frontotemporal disorders. In April 2024, the company announced that the first patient had been treated with its AVB-101 candidate for frontotemporal dementia with GRN mutations as a part of its Phase I/II clinical trial.

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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

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

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 Astellas Pharma, a global pharmaceutical company, has made a licensing agreement with gene therapy firm AviadoBio. This agreement gives Astellas exclusive rights to AviadoBio's AVB-101, an AAV-based gene therapy under Phase 1/2 development. Astellas is going to invest USD 20 million in equity and an upfront payment of up to USD 30 million for the licensing option to AVB-101. If Astellas decides to exercise this option, AviadoBio could earn USD 2.18 billion from license fees and milestone payments along with royalties.
 AVB-101 is intended to treat frontotemporal dementia with progranulin mutations (FTD-GRN), a dementia often misdiagnosed or undiagnosed. This alliance plays an important role in speeding up the development and global commercialization of a potential therapy for patients living with FTD-GRN and related neurological disorders.
 
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AviadoBio

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