5AM Ventures is a venture capital firm that offers seed- and early-stage investments to its clients. The company’s client portfolio consists of firms in the biopharmaceutical, life sciences, medical technology, and research instrument industries. 5AM Ventures was founded in 2002 by John Diekman and Andrew Schwab and is based in California.

5AM Ventures

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

VenBio invests in promising companies at various stages: from early to late stages, and from academic startups to spinouts. It tends to lead most of its investments, and it enjoys building syndicates and teams. The company is mindful of potential acquisition partners from the outset, and it ensures that its companies are best positioned to meet their requirements. This includes IP, CMC, and the details of clinical trials, including indication, trial design, endpoints, powering, and regulatory considerations. Its team’s expertise and involvement help companies make a bigger impact, and this leads to impactful and lasting relationships with the leaders that they work with.

venBio Partners

Medivate Partners LLC is an investment arm of Posco specializing in cross border, growth capital, early stage and mid stage.

Medivate Partners

GC Green Cross Their goal is to contribute to the healthy life of mankind and become a global leader in the healthcare industry. Recent advances in biotechnology have become a good foundation for achieving their goals. The paradigm of medicine is changing. They are shifting from traditional medicines and synthetic medicines to biopharmaceutical development. GC Green Cross Cellular gene therapy, which is the main pipeline of LAPSEL, is expected to enable the treatment of rare and intractable diseases as well as cancer, because of the fundamental treatment of diseases at the gene and cell level. In particular, successful cases such as approval of the first immunotherapeutic drug (CAR-T) in 2017 and the approval of the first gene therapy (Luxturna) were epoch-making events that transformed the expectations they had in mind into reality. Is added.

Green Cross Lab Cell

GC holding company, which operates pharmaceutical, biotechnology, distribution system, life insurance, and healthcare entities.

Green Cross Holding

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

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

EcoR1 Capital is an investment advisory firm focused on the biotech industry. They aim to identify and evaluate innovative therapeutic solutions that can bring significant improvements to patients' lives. Drawing inspiration from the transformative power of the EcoR1 restriction enzyme in the biomedical field, they invest in biotech companies that offer promising new treatments for untreated diseases. EcoR1 Capital recognizes the importance of building upon the innovative contributions of its predecessors in order to continue advancing medical research.

EcoR1 Capital

Wellington Management focuses on investment management. The company applies its full resources to meet the needs of the institutional clients they serve. Their investment approaches span equity, fixed income, currency, commodity, and alternative markets. They also offer asset allocation, multi-manager, and speciality approaches.

Wellington 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

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

Acuta Capital Partners is a venture capital firm.

Acuta Capital Partners

Janus Henderson Investors focuses on investing performance and services. The firm connects institutional, retail, and high-net-worth clients with investment specialists who specialize in a variety of asset classes, such as equities, fixed income, real estate, and alternatives.

Janus Henderson Investors

Surveyor Capital is a fundamental equities business. Surveyor’s sector-aligned investment teams aim to generate alpha through a deep understanding of each company’s strategy, management teams, drivers of performance, and cyclical and secular industry trends over the medium and long term.

Surveyor Capital

Logos Capital is a fundamental biotechnology-focused fund that seeks to combine in-house data analytics with scientific and clinical expertise to identify transformative therapies in healthcare. Based in San Francisco, Logos combines expertise across medical, scientific, and statistical disciplines to expand the depth of diligence in identifying compelling investment ideas and uncorrelated-market returns. They combine scientific diligence with their statistical modeling of clinical trials to achieve the highest levels of conviction in each position.

Logos Capital

Artiva Biotherapeutics

Artiva Biotherapeutics is a private biotech company advancing a pipeline of off-the-shelf, allogeneic NK cell therapies, for the treatment of hematologic cancers or solid tumors. It was founded on the understanding that NK cells hold enormous therapeutic potential as cancer therapies and the dedication and expertise to overcome technological barriers in the scaling and manufacturing of these cells. At Artiva, its mission is to deliver highly effective cell therapies that are also safe and immediately available and accessible to any cancer patient who stands to benefit. Artiva’s platform delivers scalability, quality, and cryopreservation to support a pipeline of safe, effective, versatile, and accessible product candidates.

<div>Artiva Biotherapeutics is a biotechnology company focused on developing off-the-shelf, allogeneic natural killer (NK) cell-based therapies for cancer and autoimmune diseases. Artiva leverages its AlloNK platform to produce NK cell therapies that can be administered without the need for genetic modification. Its lead product candidate, AB-101, is a cryopreserved NK cell therapy being evaluated for use in combination with B-cell targeted monoclonal antibodies (mAbs) and other innate cell engagers​.</div>

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Artiva Biotherapeutics Appoints Alison Moore, Ph.D., to Its Board of Directors

<div>
 
 Artiva Biotherapeutics is a biotechnology company focused on developing off-the-shelf, allogeneic natural killer (NK) cell-based therapies for cancer and autoimmune diseases. Artiva leverages its AlloNK platform to produce NK cell therapies that can be administered without the need for genetic modification. Its lead product candidate, AB-101, is a cryopreserved NK cell therapy being evaluated for use in combination with B-cell targeted monoclonal antibodies (mAbs) and other innate cell engagers​.
 
 
 The company also develops other oncology candidates, such as AB-201, an allogeneic HER2-targeted CAR-NK cell therapy for the treatment of solid tumors in the outpatient setting with the option for repeat dosing. In 
 <a href="https://www.artivabio.com/artiva-biotherapeutics-announces-fda-allowance-of-ind-for-ab-201/">
 September 2022
 </a>
 ,&nbsp; the US FDA cleared the company&rsquo;s IND application for the treatment.&nbsp;
 
 
 Since July 2024, Artiva Biotherapeutics has operated as a publicly listed company, and is traded on Nasdaq under the ticker symbol ATRA.
 
 
 Key customers and partnerships
 
 
 Since 2021, Artiva has been collaborating with Korean biotechnology company, GC LabCell, co-developing its CAR-NK platform technology. The technology was globally launched through an agreement worth USD 1.9 billion, inked with pharmaceutical company 
 <a href="https://www.businesswire.com/news/home/20210128005198/en/Artiva-Biotherapeutics-Announces-Exclusive-Worldwide-Collaboration-and-License-Agreement-with-Merck-to-Develop-Candidate-CAR-NK-Cell-Therapies">
 Merck Sharp &amp; Dohme Corporation (MSD)
 </a>
 .
 
 
 In April 2022, Artiva entered into a partnership with 
 <a href="https://www.artivabio.com/artiva-biotherapeutics-announces-agreement-with-merck-to-evaluate-combinations-of-nk-cells-with-tri-specific-nk-cell-engagers/">
 Merck
 </a>
 for the development of combination therapies involving AB-101. The collaboration leveraged Artiva&rsquo;s off-the-shelf allogeneic NK cell manufacturing platform and proprietary CAR-NK technology, and included two CAR-NK programs outside Artiva&rsquo;s pipeline, with an option for a third.&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

ANALYST_QUICK_TAKE

<div>

Artiva Biotherapeutics, a cell therapy company, has acquired USD 167 million through its IPO offering of almost 14 million shares priced at USD 12 each. The company will be listed on the Nasdaq under the ticker symbol "ARTV".

The majority of the funds raised are allocated towards developing Artiva's treatment comprised of "natural killer" (NK) cells. The treatment, currently in preliminary testing stages, is aimed at lupus and other inflammatory conditions.

</div> Analyst QuickTake : 
 Artiva's IPO comes amid growing optimism for allogeneic cell therapies in autoimmune diseases, following a recent study in Cell Journal showing potential for donor-derived treatments in this area. Analysts view Artiva's approach using NK cells as a promising alternative to autologous cell therapies, which face challenges like high costs and safety concerns in autoimmune settings.

Artiva Biotherapeutics

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