PBM Capital Group, LLC. is a private investment firm primarily focused on healthcare investments. Led by Paul Manning, a successful entrepreneur, it invests across all stages of corporate development– including venture capital, leveraged buyouts, growth capital, distressed/turnaround, and public equity value investing– and create value by applying extensive industry knowledge and contacts, providing superior operational expertise and bringing an entrepreneurial spirit to attractive investment opportunities. We actively partner with our portfolio companies to drive commercial success and enhance profitability.

PBM Capital Group

Nolan Capital is a real estate and other investments company located in Hermosa Beach.

Nolan Capital

Fidelity is a privately owned investment management firm that serves a wide range of clients. It offers services including IRAs, rollovers, annuities, discount brokerage, retirement planning, estate planning, wealth management, securities execution and clearance, and life insurance. They serve individual investors, businesses, financial advisors, and institutions. The firm manages equity, fixed-income, and balanced mutual funds and invests in public equity and fixed-income markets. It also conducts in-house research to inform its investment decisions.

Fidelity

Franklin Resources is a global investment management organization known as Franklin Templeton Investments. They have an extensive global presence, including offices in 35 countries and clients in more than 150. Their common stock is listed on the New York Stock Exchange under the ticker symbol BEN and is included in the Standard & Poor’s 500:registered: Index. Franklin Resources Inc. NYSE: BEN is an investment firm originally founded in New York in 1947 as Franklin Distributors, Inc. It is listed on the NYSE under the ticker BEN as in Benjamin Franklin, whom founder Rupert Johnson, Sr. admired. In 1973 the headquarters moved from New York to San Mateo, California. The firm specializes in conservatively managed mutual funds. It offers products under the Franklin, Templeton, Mutual Series and Fiduciary brand names. Like other large investment companies, the firm offers a wide variety of funds but is traditionally best known for bond funds under the Franklin brand, international funds under the Templeton brand, and value funds under the Mutual Series brand.

Franklin Templeton

Invus recognizes that every company is unique and that extraordinary business performance cannot be achieved by following a formulaic approach. Due to their unique structure, they have a great deal of flexibility in the types of companies and situations where they can get involved. They know that real transformation takes time and that it is never a straight line. They fully align their incentives with their partners and have the wherewithal and long-term commitment to see through any bumps along the way. They have been successfully empowering owner-managers since 1985. They have been reinvesting the same pool of money from the same group of European families (through their Artal investment vehicle) since their founding. Today they manage over $4 billion in an evergreen fund and work out of offices in New York, London, Paris and Hong Kong.

Invus

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

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

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

Casdin Capital is a fundamental research investment firm focused on the life sciences and healthcare industry. It manages a long-short equity fund and makes investments in early-stage to late-stage private investments.  Casdin was launched in 2012 by founder and CIO Eli Casdin and as of October 2020 has approximately $2.2 billion in assets under management.

Casdin Capital

Sands Capital is an active, long-term investor in innovative growth businesses. The approach combines rigorous fundamental analysis with inspired thinking to seek out businesses that are creating the future. They operate through two affiliates: Sands Capital Management, which focuses on investing in companies in the public market, and Sands Capital Ventures, which focuses on investing in companies in the private market. By enabling clients to participate in the growth of these businesses, its mission is to add value and enhance their wealth with prudence over time.

Sands Capital Ventures

BlackRock is an investment management company that provides its services to a diverse range of clients, including institutions, intermediaries, foundations, and individual investors. The firm operates as a global asset manager, overseeing and advising on a wide array of investment portfolios. In addition to its role as an investment manager, BlackRock is also known for its technological innovations and solutions that support various aspects of the financial industry. The company's multifaceted approach encompasses investment management, financial technology, and serving a broad spectrum of clients in the financial ecosystem.

BlackRock

Venrock Healthcare Capital Partners is a fund dedicated to investing in publicly-held and privately-held healthcare companies. It leverages its venture expertise, rigorous data analysis, and long-term supportive orientation to support successful biotechnology companies within the public markets.

Venrock Healthcare Capital Partners

Octagon Capital invests in public and private healthcare companies globally. Octagon Capital focuses on strategic long-term investments and strive to work with our portfolio management teams as partners.

Octagon Capital Advisors

Taysha Gene Therapies

Taysha Gene Therapies is a developer of treatments to eradicate severe & life-threatening monogenic diseases of the central nervous system. Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we are able to rapidly translate our treatments from bench to bedside. We have combined our team’s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to quickly and efficiently build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications.

<div>Taysha Gene Therapies is a gene therapy developer that manufactures gene-based treatments for medical conditions such as neurodegenerative diseases, neurodevelopmental diseases, and genetic epilepsies. The company uses edited genes via its AAV9 Capsid platform (a adeno-associated virus vector), which is used to make engineered genes replace mutated genes. Taysha&rsquo;s solution is administered intrathecally to directly deliver gene therapeutics into the cerebrospinal fluid for more optimal distribution. The company is listed on the Nasdaq and trades under the symbol TSHA.</div>

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Taysha Gene Therapies Announces Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) - December 6, 2024

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 Taysha Gene Therapies is a gene therapy developer that manufactures gene-based treatments for medical conditions such as neurodegenerative diseases, neurodevelopmental diseases, and genetic epilepsies. The company uses edited genes via its AAV9 Capsid platform (a adeno-associated virus vector), which is used to make engineered genes replace mutated genes. Taysha&rsquo;s solution is administered intrathecally to directly deliver gene therapeutics into the cerebrospinal fluid for more optimal distribution. The company is listed on the Nasdaq and trades under the symbol TSHA.
 
 
 In February 2024, the company transferred the IP rights for several gene therapy programs to other organizations under a loan and security agreement. This move was in order to extend the company&rsquo;s cash runway into 2026. 
 
 
 As of May 2024, the company had two gene therapy candidates in development, both for the treatment of Rett syndrome. Both candidates were in Phase I/II of clinical testing. 
 
 
 
 
 Key customers and partnerships
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 Taysha and pharmaceutical company Catalent partnered in November 2020 to support the development and manufacturing capabilities of Tysha&rsquo;d gene therapeutics. Through the partnership Tysha would be able to use Catalent&rsquo;s manufacturing facilities to produce its gene therapies.
 
 
 
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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

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Taysha Gene Therapies, a gene therapy company based in Texas, has declared a planned public offering of common stock and pre-funded warrants up to the value of $75 million. Alongside this, underwriters have been given an option to buy 15% of common shares within a 30-day period.

The proceeds of this public offering aim to bolster operational and developmental programs within the company, though explicit specifics were not revealed. Information such as the offering's size, closing date, and terms were also withheld.

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Taysha Gene Therapies

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