Flagship Pioneering conceives, creates, resources, and grows first-in-category ventures to transform human health and sustainability. The company accelerates Flagship Pioneering’s advances in biology, engineering, medicine, energy, and beyond. The firm harnesses science and entrepreneurialism to envision alternative futures, beginning with seemingly unreasonable propositions and navigating to transformational outcomes through an evolutionary methodology. Flagship Pioneering combines innovative capacity, entrepreneurial leadership, and professional capital management in a single enterprise that systematically produces first-in-category companies.

Flagship Pioneering

Alumni Ventures Group provides high-quality, diversified venture portfolios to individual investors who previously haven't had access to VC. It helps accredited alums from top entrepreneurial schools invest together in the ventures of fellow alums. The firm backs companies with an alumni connection and an institutional lead investor with expertise in the space. AVG also offers focused funds that allow any accredited investor access to a venture portfolio, diversified by type, sector, stage, and geography. Beth Obermiller, Michael Madden, and Mike Collins co-founded the firm in Manchester, New Hampshire in 2013.

Alumni Ventures

T. Rowe Price is an investment management firm dedicated to funding technology startups. The firm serves individuals, financial intermediaries, and institutions and offers a full range of investment strategies across a broad spectrum: debt, equities, and private equities.

T. Rowe Price

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

ARCH invests primarily in companies co-founded by scientists and entrepreneurs, concentrating on bringing to market innovations in life sciences. They enjoy special recognition as a leader in the successful commercialization of technologies developed at academic research institutions and national laboratories. ARCH has raised ten venture funds totaling over $3 billion and has invested in the venture capital rounds for more than 150 companies. ARCH investors include corporations, pension funds, endowment funds, financial institutions, and private investors.

ARCH Venture Partners

Philosophy is to focus on life science markets with strong demand and growth potential, and then back the highest-quality entrepreneurs in those markets to build defensible, strong, and long-term businesses.  build portfolio among attractive life science sectors in a manner that balances risk while maximizing potential synergies and ultimate returns. Our focus is on opportunities in biotechnology, medical devices, and diagnostics, but we are continually responsive to the best areas for investing in order to adapt to changing market conditions.  Invest in companies that:      * are pursuing substantial and growing market opportunities     * possess a unique and proprietary technology, with a clear competitive advantage     * are early-stage opportunities     * have or will attract strong management     * have a strong investment syndicate     * offer a defined path to liquidity

Altitude Life Science Ventures

Abu Dhabi Investment Authority is an investment management agency investing funds on behalf of the Government of Abu Dhabi. ADIA does not directly to manage or take an operational role in the companies in which it invests but through it's PPP Representative Eiosoft Capital Group LLC.

Abu Dhabi Investment Authority

The Alaska Permanent Fund is a constitutionally established permanent fund managed by a state-owned corporation, the Alaska Permanent Fund Corporation. The fund was established in Alaska in 1976 by Article 9, Section 15 of the Alaska State Constitution under Governor Jay Hammond. From February 1976 until April 1980, the Department of Revenue Treasury Division managed the state's Permanent Fund assets, until, in 1980, the Alaska State Legislature created the APFC.  Shortly after the oil from Alaska’s North Slope began flowing to market through the Trans-Alaska Pipeline System, the Permanent Fund was created. It was designed to be an investment where at least 25% of the oil money would be put into a dedicated fund for future generations, who would no longer have oil as a resource. This does not mean the fund is solely funded by oil revenue. The Alaska Permanent Fund sets aside a certain share of oil revenues to continue benefiting current and all future generations of Alaskans.

Alaska Permanent Fund

Morningside Venture Investments is an investment group that provides industry investments and social responsibility. It was founded in 1986 and is based in Shanghai, China.

Morningside Venture Investments

MAPS Capital provides C level executive services for startups, foreign companies seeking US presence, international marketing and business development in addition to seed financing and assistance in venture capital funding.  We specialize in Web 2.0 services, advertising analytics, medical diagnostics and technologies related to renewable energy such as magnetics, thermoelectrics and solar photovoltaics.  We have extensive experience and applications knowledge in social media networks, behavioral targeting, monetization of web traffic, acquisition of users, viral programs for web startups and programming solutions based on the Open Social API platform.  Our primary office is in San Francisco, California with offices in Silicon Valley, Hong Kong and San Diego.

MAPS Capital Management

March Capital is a venture growth firm headquartered in Santa Monica, California and investing globally since 2014. March identifies entrepreneurs with a provocative vision to companies poised, then dares to go all in by leading rounds with deep conviction and concentration risk. With $1B+ in capital over 4 funds and across 30 market technology companies, March accelerates the digital transformation of Cloud-based software, automation, AI, and IT infrastructure. Its vision is to create a technology investment platform by combining intense sector focus, patience, access to a network (including founding The Montgomery Summit), and portfolio engagement to inspire and accelerate extraordinary companies.

March Capital

Amgen is a biotechnology company that develops and manufactures human therapeutics for various illnesses and diseases. The company is values-based and deeply rooted in science and innovation to transform new ideas and discoveries into medicines for patients with serious diseases. They offer products for treating illness in the areas of oncology/hematology, cardiovascular, inflammation, bone health, nephrology, and neuroscience.

Amgen

Pictet Alternative Advisors (PAA) is a leading European alternative investment specialist with expertise in manager selection and portfolio management in hedge funds, private equity and real estate. PAA is an independent unit of the Pictet Group. Founded in 1991, we currently manage over USD 18 billion and our team is composed of more than 50 people.

Pictet Alternative Advisors

Modi Ventures is a Texas-based investment firm investing in fund managers and founders building companies shaping the future of humanity. The company's primary investment focus is into disruptive and emerging AI and Medical technology applications. Investments in successful funds include Khosla Ventures, Section 32, Artis Ventures, Draper Associates and Antler Global. Direct investments include, Starling Medicine and Cyberdontics. For more information please visit www.modivc.com.

Modi Ventures

NVentures is a venture capital firm that invests in early-stage technology companies, focusing on innovations in various sectors. NVentures is the venture capital arm of NVIDIA.

NVentures

Generate Biomedicines

Generate Biomedicines is a biotechnological company focusing on creating breakthrough medicines. It pioneers in the field of Generative Biology, a revolutionary approach to drug development that allows them to program protein-based modalities for the first time.

<div>Generate Biomedicines leverages its AI/ML-powered drug generation platform to develop custom protein-based therapeutics. It creates novel proteins from scratch across a wide range of protein modalities, such as peptides, enzymes, antibodies, and gene therapies.&nbsp;</div>

Artificial intelligence is identifying and testing new therapies faster than ever.

AI Drug Discovery

Generate Biomedicines is a gene therapy developer that offers a platform to generate custom genome sequences through the use of AI and machine learning. The company’s platform, The Generate Platform, uses a library of proteins to generate genomes, based on generalized trends of naturally occurring sequencing. The platform can be used to generate novel medicines with specific therapeutic functions, across specialities and use cases, as through the sequencing of proteins, the platform is therefore able to generate antibodies and peptides, enzymes, and stealth proteins. As of May 2024, the company had generated 42,000 proteins, built them, and tested them.

Novel therapies to reboot cells and organs

Cell & Gene Therapy

The Week’s 10 Biggest Funding Rounds: Databricks And Generate Biomedicines Lead Another AI Surge

Novartis

To use Generate’s GenAI technology, which incorporates ML with high-throughput experimental validation and lab capabilities, to discover novel medical candidates and help reduce preclinical development time. 

Biotechnology companies that have integrated AI as a core component of their in-house drug development process. They typically have a few in-house drug development programs, and also collaborate with pharma companies to develop certain drugs.

<div>
 
 Generate Biomedicines leverages its AI/ML-powered drug generation platform to develop custom protein-based therapeutics. It creates novel proteins from scratch across a wide range of protein modalities, such as peptides, enzymes, antibodies, and gene therapies.&nbsp;
 
 
 The company reports that its platform resolves the historical issue of the immune system's unresponsiveness to new proteins. It learns how proteins are viewed by the immune system and can recode protein sequences to evade the immune system (ones which the immune system would otherwise eliminate because they are non-human proteins entering the body).
 
 
 The company mainly offers its AI platform to other drug developers to simplify and expedite the development process of protein therapeutics. It also has several drug candidates in its in-house pipeline targeting immunology, oncology, and infectious diseases. Two of the company&rsquo;s drug candidates, GB-0669 for SARS-CoV-2 and GB-0895 for asthma, have entered Phase I clinical trials.&nbsp;
 
 
 Key customers and partnerships
 
 
 Generate entered a USD 1.9 billion-worth multi-target collaboration with 
 <a href="https://sp-edge.com/updates/7637">
 Amgen
 </a>
 in January 2022 to jointly develop five novel drug targets across various therapeutic areas and modalities.&nbsp;
 
 
 It also partnered with the 
 <a href="https://generatebiomedicines.com/news/generatebiomedicines-and-md-anderson-enter-co-development-and-commercialization-agreement-to-accelerate-novel-protein-therapeutics-for-oncology-using-generative-ai">
 University of Texas MD Anderson Cancer Center
 </a>
 (April 2023) and 
 <a href="https://www.ns-healthcare.com/news/generatebiomedicines-roswell-park-partner-to-oncology-cell-therapies/">
 Roswell Park Comprehensive Cancer Center
 </a>
 (November 2023) to accelerate the creation of novel therapeutics (protein and chimeric antigen receptor [CAR] T-cell therapies) in oncology.&nbsp;
 
 
 Funding and financials
 
 
 In September 2023, the company raised USD 273 million in a 
 <a href="https://sp-edge.com/updates/21894">
 Series C
 </a>
 funding round led by Amgen to support the advancing of the company&rsquo;s pipeline of protein therapies and enhance its generative biology platform. 
 
</div>

<div>
 
 HiberCell is a biotechnology company that develops novel therapies focused on oncology. It entered the AI drug discovery space when it acquired AI genomics analysis company Genuity Science (and its subsidiaries in Iceland and Ireland) in August 2021.&nbsp;
 
 
 Genuity Science&rsquo;s solutions include end-to-end therapeutic discovery and development services such as building custom disease datasets, discovering and prioritizing targets, identifying biomarkers, and optimizing clinical trials. Genuity Science leverages its AI-powered platform to manage, process, and analyze genomic data. HiberCell intends to use Genuity Science&rsquo;s AI/ML capabilities to advance its oncology pipeline of five programs, of which one has reached the Phase 2 clinical trial stage.
 
 
 As of March 2024, the company&rsquo;s pipeline included three drug candidates, namely: 1) HC-7366, targeting the general control nonderepressible 2 (GCN2) kinase as part of the integrated stress response (ISR) signaling pathway; 2) HC-5404, aimed at inhibiting the PKR-like endoplasmic reticulum kinase (PERK) involved in the unfolded protein response (UPR) signaling pathway; and 3) odetiglucan, a therapy targeting tumor-immune interactions within the tumor microenvironment​.&nbsp;
 
 
 Key customers and partnerships
 
 
 In December 2023, HiberCell and Merck &amp; Co partnered to conduct a Phase Ib trial to evaluate the combination therapy of belzutifan and HC-7366 for treating clear cell renal cell carcinoma. Further, in January 2021, the company partnered with Biodesix to develop an enzyme-linked immunosorbent assay as a companion diagnostic test.
 
 
 Funding and financials
 
 
 In 
 <a href="https://hibercell.com/news/hibercell-closes-67-4-million-series-b-financing-and-secures-30-million-debt-facility-from-hercules-capital/">
 May 2021
 </a>
 , HiberCell completed a Series B financing round, raising USD 67.4 million, and secured an additional USD 30 million in debt financing from Hercules Capital. The funds were expected to be used for clinical advancement of its novel therapeutic candidates. 
 
</div>

HiberCell

<div>
 
 Arbor Biotechnologies specializes in gene editing to develop novel therapeutics for a variety of indications, starting with liver-related diseases. It operates an AI-powered biodiscovery platform to discover novel CRISPR enzymes, which are sequences of repeating genetic code. To do this, its platform deploys computational search techniques, genome sequencing, gene synthesis, and high-throughput screening. This can eventually be turned into drugs or even applied to agriculture and other industrial uses. In 2018, the company announced the discovery of Cas13d, a new type of gene editing enzyme that helps to alter RNA to treat genetic diseases.&nbsp;
 
 
 As of February 2024, the company had five internal drug discovery programs, of which one was in the IND-enabling stage. It also has multiple collaborative programs under development focused on oncology and various other therapeutic areas.
 
 
 Key customers and partnerships
 
 
 The company has a long-standing partnership with Vertex Pharmaceuticals since
 <a href="https://www.businesswire.com/news/home/20190103005194/en/Vertex-and-Arbor-Biotechnologies-Establish-Collaboration-to-Discover-Novel-Proteins-to-Advance-Discovery-of-Gene-Editing-Therapies">
 January 2019
 </a>
 , which focused on developing gene-editing treatments for cystic fibrosis and four other diseases. The alliance was further extended in 
 <a href="https://www.biospace.com/article/vertex-and-arbor-strike-a-1-2-billion-deal-for-gene-editing-programs/">
 August 2021
 </a>
 and 
 <a href="https://sp-edge.com/updates/15741">
 January 2023
 </a>
 .&nbsp;
 
 
 Other partnerships included a collaboration with 
 <a href="https://www.prnewswire.com/news-releases/arbor-biotechnologies-announces-collaboration-with-ginkgo-bioworks-to-advance-the-discovery-and-development-of-precision-gene-editors-302011991.html">
 Ginkgo Bioworks
 </a>
 (December 2023) to leverage Ginkgo's R&amp;D services to further optimize Arbor's novel precision editors, a non-exclusive licensing agreement with 
 <a href="https://www.globenewswire.com/news-release/2022/02/08/2380680/0/en/EdiGene-and-Arbor-Biotechnologies-Announce-Worldwide-Non-exclusive-License-Agreement-to-Advance-Ex-Vivo-Engineered-Cell-Therapy-Programs.html">
 EdiGene
 </a>
 (February 2022) to leverage Arbor&rsquo;s platform for ex vivo oncology therapies, a strategic research and non-exclusive licensing agreement with 
 <a href="https://investors.tcr2.com/news-releases/news-release-details/tcr2-therapeutics-and-arbor-biotechnologies-establish">
 TCR2 Therapeutics
 </a>
 (January 2022) to develop allogeneic TRuC T-cell therapies using Arbor&rsquo;s CRISPR technology, and a license agreement with 
 <a href="https://www.lonza.com/news/2020-12-23-14-00">
 Lonza
 </a>
 (December 2020) to use Arbor&rsquo;s gene-editing technology for Lonza&rsquo;s bioproduction products and services.
 
 
 Funding and financials
 
 
 In its latest Series B funding round in November 2021, which was led by Ally Bridge Group, TCG Crossover, and Temasek, Arbor Biotechnologies raised USD 215 million. The proceeds of the round were earmarked to fuel the company&rsquo;s clinical development pipeline.
 
</div>

Arbor Biotechnologies

Based in the UK, Arctoris provides an automated drug discovery platform, offering fully validated research and development (R&amp;D) processes under an Experiments-as-a-Service model. The company works with biotechnology companies, AI drug discovery companies, pharma and academic institutions enabling these institutions to run experiments in Arctoris’s platform and provides on-demand access to a broad scale of datasets for AI drug discovery companies to train their algorithms.

Arctoris

<div>
 
 Frontier Medicines leverages AI/ML technologies to develop treatments for cancer-causing proteins that are dismissed by traditional pharma companies as undruggable.&nbsp;
 
 
 The company has a proprietary chemoproteomic platform which entails an approach based on the interaction of small-molecule compounds and proteins to screen a large number of targets and combine them with a protein database that contains more than 150,000 novel binding pockets (hotspots). This enables it to access 90% of the human proteome that were not previously targeted due to lack of hotspots. The platform&rsquo;s AI/ML engine incorporates a library of diverse compounds, a hotspot map that serves as the starting point for understanding the druggability of a potential therapeutic target, and lead design and generation.&nbsp;
 
 
 The company focuses on oncology and immunology target and, as of February 2024, it had five wholly-owned therapeutic programs and three AbbVie-partnered programs in its pipeline. All programs were in their discovery stage, except the company&rsquo;s lead candidate, FMC-376 (activated and inactivated forms), focused on treating different cancer types, including non-small cell lung cancer (NSCLC), colorectal carcinoma (CRC), and pancreatic ductal adenocarcinoma (PDAC), which is in the IND-enabling stage.
 
 
 Key customers and partnerships
 
 
 The company entered a USD 100 million partnership with 
 <a href="https://news.abbvie.com/2020-12-02-AbbVie-and-Frontier-Medicines-Establish-Global-Partnership-to-Discover-and-Develop-Novel-Therapies-and-E3-Degraders-Against-Difficult-to-Drug-Targets">
 AbbVie
 </a>
 In December 2020 to develop small-molecule therapeutics against hard-to-drug protein targets including around 600 E3 ligases (the enzymes that drive the ubiquitination and degradation of proteins.&nbsp;
 
 
 Funding and financials
 
 
 In July 2021, the company raised USD 88.5 million in a 
 <a href="https://sp-edge.com/updates/4347">
 Series B
 </a>
 funding round co-led by Woodline Partners LP and RA Capital Management, with equal participation from Deerfield Management Company. The proceeds were earmarked for advancing the company&rsquo;s drug pipeline and expanding operations in Boston, where its new facility was to complement the company&rsquo;s headquarters in California. 
 
</div>

Frontier Medicines

<div>
 
 Verge Genomics is a biotechnology company engaged in developing therapeutics for neurological diseases using human genomics and AI to accelerate drug discovery. The main target areas are Amyotrophic Lateral Sclerosis (ALS), Parkinson&rsquo;s Disease (PD), and Frontotemporal Dementia (FTD).&nbsp;
 
 
 It has built a proprietary all-human platform (unlike standard clinical trials that rely on animal data), called CONVERGE, that uses data sets from brain tissue samples of deceased patients with ALS and PD. While most drugs target only one gene at a time, the company claims its technology can identify hundreds of genes that cause disease and develop drugs that target all the genes at the same time. The company uses AI technology to optimize the drug discovery process as a whole.
 
 
 As of March 2024, Verge had one drug candidate in the clinical stage (Phase I) and 10 drug candidates in the discovery stage.&nbsp;
 
 
 Key customers and partnerships
 
 
 The company has partnerships with various tissue banks (such as Harvard University and University of Miami Miller School of Medicine) and several educational institutes and foundations (such as Target ALS, Answer ALS) that enabled its drug discovery platform.&nbsp;
 
 
 In addition, the company&rsquo;s customers include several pharmaceutical companies, such as 1) 
 <a href="https://www.vergegenomics.com/news-blog/verge-genomics-announces-research-collaboration-with-the-sheffield-institute-for-translational-neuroscience">
 Sheffield Institute for Translational Neuroscience (SITraN)
 </a>
 to accelerate the validation of novel drug targets and screening of drug compounds for Parkinson's disease (February 2021), 2) 
 <a href="https://sp-edge.com/updates/4146">
 Eli Lilly
 </a>
 to research and develop novel therapies to treat ALS (July 2021), 3) 
 <a href="https://sp-edge.com/updates/21707">
 Alexion
 </a>
 to identify novel drug targets for rare neurodegenerative and neuromuscular diseases (September 2023), and 4) 
 <a href="https://www.vergegenomics.com/news-blog/verge-genomics-and-the-pritzker-neuropsychiatric-disorders-research-consortium-form-cutting-edge-schizophrenia-research-collaboration">
 Pritzker Neuropsychiatric Disorders Research Consortium
 </a>
 to analyze data from schizophrenic patient&rsquo;s brain tissue with Verge&rsquo;s technology (October 2023).
 
 
 Funding and financials
 
 
 In December 2021, Verge raised USD 98 million in an oversubscribed 
 <a href="https://sp-edge.com/updates/7426">
 Series A
 </a>
 funding round led by BlackRock. The funds were used to scale up its pre-clinical and clinical candidate programs in the pipeline and enhance its lead therapeutics through clinical testing. 
 
</div>

Verge Genomics

SaaS (Software-as-a-Service) companies providing their ML platform to other pharma companies for drug discovery services such as target selection, validation and lead optimization.

SaaS (Software-as-a-Service) companies providing their ML platform to other pharma companies to design and run preclinical experiments.

SaaS (Software-as-a-Service) companies providing their ML platform to other pharma companies to identify biomarkers (measurable biological signs used to determine disease progression) and predict drug response. 

SaaS (Software-as-a-Service) companies aggregating and analyzing biomedical data with the help of AI to generate insights across the drug development pipeline.

Companies leverage GenAI to support drug discovery through accelerated drug designing

Companies offer AI/ML-based SaaS products to accelerate the discovery and development of novel therapeutics

Offers AI/ML-based solutions to accelerate drug discovery and development

Generate Biomedicines is a gene therapy developer that offers a platform to generate custom genome sequences through the use of AI and machine learning. The company’s platform, The Generate Platform, uses a library of proteins to generate genomes, based on generalized trends of naturally occurring sequencing. The platform can be used to generate novel medicines with specific therapeutic functions, across specialities and use cases, as through the sequencing of proteins, the platform is therefore able to generate antibodies and peptides, enzymes, and stealth proteins. As of May 2024, the company had generated 42,000 proteins, built them, and tested them.Key customers and partnershipsGenerate Biomedicines secured a partnership with Amgen in January 2022 through which both parties signed a multi-modality research collaboration agreement. Through the partnership, Amgen would pay USD 50 million in an upfront payment (with a possible deal value of up to USD 1.9 billion plus future royalties) for Generate Biomedicines to discover and develop protein therapeutics for five clinical targets across several therapeutic areas and modalities.Prior to the above, Generate’s latest partnership was with the University of Texas MD Anderson Cancer Center, which was signed in April 2023. The partnership was secured to accelerate the creation of novel protein therapeutics in oncology through the use of generative AI. Through the partnership, both parties would collaborate to co-discover protein therapeutics for up to five oncology targets in advanced cancers, including small-cell and non-small-cell lung cancer.&nbsp;

<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

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

<div>
 
 Pharmaceutical company Novartis is partnering with Generate Biomedicines to use artificial intelligence (AI) in discovering new targets for treatment in undisclosed disease areas. The agreement will see Novartis making an initial payment of $65 million, including $15 million to purchase equity in Generate. Moreover, Novartis has agreed to pay up to $1 billion in milestone-based payments as well as tiered royalties as high as low double-digit percentages.

The collaboration aims to utilize Generate's GenAI technology which merges machine learning (ML) with high-throughput experimental validation and laboratory capabilities. The goal is to discover innovative medical candidates and aid in cutting down the time taken in preclinical development. Novartis has recognized the success of Generate's proof-of-concept.
 
</div>

Generate Biomedicines

EDGE Insights

About EDGE

Contact Us

Resources

Area of Focus

Technology Research Made Effortless