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

Drive Capital invests in innovative technology, healthcare, and consumer companies in the Midwest. The firm is looking for innovative entrepreneurs addressing big market opportunities. Its goal is to partner with entrepreneurs that have audacious goals and strive to build large sustainable companies. The firm seeks to invest in information technology, financial services, and mobile sectors. It was founded in 2012 and is headquartered in Columbus, Ohio.

Drive Capital

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

Marshall Wace is a hedge fund manager specializing in global long/short equity. The firm has investment management offices in London; New York; and Hong Kong.  Marshall Wace LLP was named Management Firm of the Year by the EuroHedge Awards 2011, reflecting the strong performance delivered by a number of our funds during the year. The firm is dedicated to the objective of providing its clients with a persistent, risk-adjusted return on their investment. Through a range of products, investors can select funds offering variable sources of alpha and beta, in different combinations, from equity markets around the world. Marshall Wace brings together two distinct but complementary approaches to asset management. First, traditional, fundamental long/short investing grounded in stock-specific analytical research, and secondly, Marshall Wace TOPS (‘MW TOPS’), the pioneering, and proprietary, systematic, alpha capture strategy.

Marshall Wace

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

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

Aisling Capital believes the next decade will be marked by a revolution in healthcare driven by new therapeutics generated by biotechnology. The completion of the human genome has given scientists new insights into the causes of human disease. These insights, combined with the past 20 years of developments in the biotechnology industry, are leading to rapid expansion of novel approaches toward the diagnosis, prevention, and treatment of life-threatening illnesses. These advances have led to an ever-increasing demand for capital to complete the development and commercialization of new therapeutics. Our goal is to support the leading global healthcare companies that are building on these technical and medical breakthroughs to commercialize new healthcare products.

Aisling Capital

Forge Biologics

Forge Biologics is a gene therapy development engine, focused on enabling access to life-changing gene therapies and helping bring them from an idea into reality. The company partners with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups. It was founded in 2020 and headquartered in Grove City, Ohio.

Forge Biologics is a genetic medicines manufacturing company, operating a 200,000 square foot facility known as "The Hearth," dedicated to adeno-associated virus (AAV) manufacturing. Forge also develops its own pipeline of gene therapies, including a lead program for infantile Krabbe disease that combines AAV therapy with umbilical cord transplants.

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Ajinomoto

Ajinomoto is acquiring US biotech firm Forge Biologics, expanding its presence in the pharmaceutical sector and gaining access to Forge's gene therapy manufacturing capabilities.

Companies conducting R&D on therapies that modify or introduce new genes into the body to treat diseases

Forge Biologics is a genetic medicines manufacturing specialist and developer based in Columbus, Ohio. The company operates a 200,000-square-foot facility known as "The Hearth," which is dedicated to adeno-associated virus (AAV) manufacturing with 20 custom-designed cGMP suites. Forge offers end-to-end manufacturing services including process and analytical development, cGMP viral vector manufacturing, final fill, plasmid DNA manufacturing, and regulatory consulting support. The company's capabilities span from preclinical through clinical and commercial stage manufacturing for AAV gene therapy programs.
In addition to its contract development and manufacturing organization (CDMO) services, Forge is also developing its own pipeline of gene therapies. The company's lead candidate, FBX-101, is a gene therapy for infantile Krabbe disease currently in Phase 1/2 clinical trials. Early data from two patients treated with FBX-101 showed increased galactocerebrosidase (GALC) enzyme activity and normal white matter myelination. Forge's approach involves administering the AAV gene therapy during full myeloablation after hematopoietic stem cell transplantation, which may help overcome immune challenges seen with other gene therapies.
Forge has made scientific contributions to the field of AAV production. In December 2023, the company published research in Human Gene Therapy demonstrating a novel role for adenovirus L4 region proteins in AAV production, which could lead to more efficient manufacturing strategies. As of November 2023, Forge was acquired by Ajinomoto Co. for USD 554 million, valuing the company at USD 620 million on a cash-free and debt-free basis.

Forge Biologics is a genetic medicines manufacturing company, operating a 200,000 square foot facility known as "The Hearth," dedicated to adeno-associated virus (AAV) manufacturing. Forge also develops its own pipeline of gene therapies, including a lead program for infantile Krabbe disease that combines AAV therapy with umbilical cord transplants.In<a href="https://www.forgebiologics.com/ajinomoto-co-inc-to-acquire-forge-biologics-for-620-million1/"> November 2023</a>, Forge was acquired by Ajinomoto Group for USD 620 million, becoming a fully consolidated subsidiary. The acquisition aimed to expand Ajinomoto's capabilities in AAV and plasmid gene therapy manufacturing. As of September 2024, Forge had over 350 employees and supported nearly 50 clients.Key customers and partnershipsIn September 2024, Forge partnered with the&nbsp;<a href="https://www.biopharma-reporter.com/Article/2024/09/10/Forge-Biologics-in-gene-therapy-collaboration">muscular dystrophy association (MDA)</a> for its Kickstart Program, which focuses on developing gene therapies for ultra-rare neuromuscular diseases. Forge provides its expertise in adeno-associated virus (AAV) manufacturing to help transition therapies from research to clinical trials. Other notable partnerships include that with&nbsp;<a href="https://www.businesswire.com/news/home/20230503005422/en/Forge-Biologics-and-Labcorp-Announce-Strategic-AAV-Gene-Therapy-Development-and-Manufacturing-Collaboration">Labcorp</a>&nbsp; to enhance gene therapy manufacturing (May 2023),&nbsp;<a href="https://www.forgebiologics.com/forge-biologics-and-oculogenex-announce-aav-contract-development-and-manufacturing-partnership/">Oculogenex</a> to develop an AAV gene therapy for age-related macular degeneration (January 2023), and&nbsp; <a href="https://www.biopharminternational.com/view/cirm-picks-forge-biologics-as-aav-manufacturing-partner">California Institute for Regenerative Medicine (CIRM)</a> to manufacture AAV vectors and help accelerate gene therapy projects within California (October 2023).

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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

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

Forge Biologics

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