Woodford Investment Management is a privately owned investment firm manages equity mutual funds. At Woodford, they don’t want to be just another fund management company. They want to create something different and better for their investors and they feel strongly that existing and potential clients are entitled to understand what they are doing with their money. As investors, they are active, engaged, long-term, disciplined, diligent and focused. That’s a lot of things to say about their selves but each word is important in helping you to understand how they manage money  – they will explain in more detail as they move along. Their investment approach and process are focused on value discovery, not price discovery. They believe that financial markets are inherently inefficient and that, through disciplined and diligent analysis, they can identify investment opportunities where market prices do not reflect long-term fundamental value. By exploiting this pervasive market characteristic, they believe they can create real long-term value for their investors.

Woodford Investment Management

Arix Bioscience is a global venture capital firm that invests in biotechnology firms. It works with exceptional entrepreneurs and provides the capital, expertise, and global networks required to help them turn their ideas into significant new treatments for patients. As a publicly traded company, they can bring this exciting growth phase of the industry to a wider range of investors.

Arix Bioscience

Syncona is a leading healthcare company focused on investing in and building global leaders in life science. Their vision is to deliver transformational treatments to patients in truly innovative areas of healthcare while generating superior returns for shareholders. They seek to partner with the best, brightest and most ambitious minds in science to build globally competitive businesses. They take a long-term view, underpinned by a deep pool of capital, and are established leaders in the development of genetic medicine, particularly gene and cell therapy. They focus on delivering dramatic efficacy for patients in areas of high unmet need. Syncona is aligned with two of the premium charitable funders in UK science, The Wellcome Trust, original founder of Syncona, and Cancer Research UK, both of which are significant shareholders in their business. They make a donation of 0.3% of Net Asset Value to a range of charities each year.

Syncona Partners LLP

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

Google Ventures invests in sectors including life sciences, consumer, enterprise, crypto, climate, and frontier technology. With Alphabet as its sole limited partner, Google Ventures focuses on long-term investments. Its operating partners assist startups in design, equity, diversity & inclusion, talent, and engineering and help them connect with Google for technology and expertise. Founded in 2009 as Google Ventures, Google Ventures manages over $8 billion in assets with 400 active portfolio companies in North America and Europe. Some notable investments include Uber, Nest, Slack, GitLab, and One Medical. Google Ventures is based in the San Francisco Bay Area with offices in Cambridge, New York, and London.

Google Ventures

Nextech Invest is a venture capital and private equity firm specializing in early, mid-stage, late-stage, and emerging growth investments. The firm also invests in incorporation, spin-outs, reverse-take-over, and PIPE transactions. It only considers investments where evidence in preclinical animal models that a drug is efficacious is available or a device prototype fulfills the requirements. The firm seeks to invest in the field of information technology, biotechnology, and healthcare with a focus on oncology companies developing cancer drugs and diagnostics. It prefers to invest in life science and enabling technologies with a focus on interdisciplinary technical and scientific projects, therapies, diagnostics, medical technologies, and services. The firm seeks to invest in companies based in Africa, the Middle East, Asia, North America, and Europe including Switzerland, Germany, and neighboring regions. It also considers co-investments in the rest of Europe. The firm seeks to invest between CHF 1 million ($0.93 million) and CHF 5 million ($4.7 million) per financing round. It seeks a Board position in the portfolio companies. The firm seeks to exit its investments within three years via trade sale.

Nextech Invest

UCLB is the commercialisation company of UCL and its partner NHS Trusts. They bring to market truly world-leading, world-changing technologies, innovations that have a real and positive impact on people’s lives. UCLB the access point to the wealth of innovation and intellectual property emanating from UCL and partner hospitals including University College Hospital, Great Ormond Street Hospital, Royal Free Hospital and Moorfields Eye Hospital. They offer a number of services to assist the technology development process, from consultancy through to collaborative research, IP licensing and the creation of spinout companies and joint ventures to maximise the commercial potential of new discoveries, materials and processes.

UCLB

Autolus

Autolus Therapeutics is working on next-generation programmed T cell treatments for the treatment of cancer. The company's pipeline includes Obe-cel (obecabtagene autoleucel), AUTO1/22, AUTO3, AUTO5, AUTO6, AUTO7 & AUTO8. Autolus was established in 2014 by Tim Funnell and Martin Pule in London, England.

Autolus Therapeutics is a pioneering clinical-stage biopharmaceutical company based in London, specializing in the development of CAR-T cell therapies for the treatment of cancer. Leveraging advanced cell programming technologies, Autolus is committed to creating autologous T cell therapies aimed at delivering significant benefits to cancer patients. The company has 95+ patent families supporting its R&amp;D.

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Autolus Therapeutics Presents Clinical Data Updates at the American Society of Hematology (ASH) Annual Meeting 2024

Cardinal Health

To support the launch and commercialization of CAR T-cell therapies in the United States, subject to FDA approval.

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

Bristol-Myers Squibb

Autolus Therapeutics partners with Bristol Myers Squibb to integrate RQR8 safety switch into cell therapy programs for cancer treatment, enhancing patient safety and therapeutic efficacy.

Autolus Therapeutics is a pioneering clinical-stage biopharmaceutical company based in London, specializing in the development of CAR-T cell therapies for the treatment of cancer. Leveraging advanced cell programming technologies, Autolus is committed to creating autologous T cell therapies aimed at delivering significant benefits to cancer patients. The company has 95+ patent families supporting its R&amp;D.As of November 2024, the company had seven drug candidates in its pipeline across 9 programs, focused on oncology and autoimmune diseases. Of the 9 programs, seven were in Phase I of clinical study, and two were in pre-clinical stages. Its lead candidate, obe-cel, an autologous CD19 CAR-T therapy, received FDA approval in November 2024. The drug became the only CAR-T that did not require a Risk Evaluation Mitigation Strategy (REMS) program by the FDA. Obe-cel uses a “fast-off” kinetic, which has demonstrated reduced levels of cytokine release syndrome (a side effect of CAR-T therapies) in clinical trials.&nbsp;Key customers and partnershipsAutolus Therapeutics collaborates with leading academic institutions and industry partners to advance and deliver its immunotherapies to patients. One notable partnership was with <a href="https://sp-edge.com/updates/26464">BioNTech </a>in February 2024, worth USD 250 million, to support the efficient development of BioNTech's CAR-T BNT211 for pivotal trials in CLDN6+ tumors. In November 2021, the company entered a USD 250 million partnership with <a href="https://autolus.gcs-web.com/news-releases/news-release-details/blackstone-life-sciences-invest-250-million-autolus-therapeutics">Blackstone Life Sciences </a>to develop obe-cel to treat adult Acute Lymphoblastic Leukemia (ALL).&nbsp;

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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

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

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 The FDA has given approval to Autolus Therapeutics for its CAR T therapy, Aucatzyl (obecabtagene autoleucel), designed to treat adult patients suffering from B-cell precursor acute lymphoblastic leukemia that has relapsed or proven refractory. This approval is based on the results of the FELIX clinical trial, where 63% of evaluated patients experienced total remission, with 42% achieving this within a three-month period.
 Aucatzyl sets a precedent as the first CAR T therapy approved sans a required Risk Evaluation Mitigation Strategy (REMS) program. The therapy demonstrated minimal cytokine release syndrome levels with a 3% Grade 3 event rate and zero Grade 4 or 5 events. Production for the treatment will occur at the Stevenage, UK facility of Autolus, with Cardinal Health overseeing all US distribution. 
 With a cost of USD 525,000 for each treatment, Aucatzyl will be in direct competition with Gilead's Tecartus, priced at USD 460,000, and Novartis' Kymriah, priced at USD 580,000. The initial plan for Autolus is to begin with 30 treatment centers, tailored to cover 60% of the target demographic, before expanding to 60 centers catering to 90% of patient population within a 12-month timeframe.
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Autolus

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