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Biotech in New Jersey: Nine companies leading the charge

New Jersey’s biotech sector is recognized as a major hub of innovation and industry in the pharmaceutical and biotechnological landscapes. According to Genetic Engineering and Biotechnology News’ 2023 ranking, New Jersey is the 4th most important biotech hub in the U.S. with innovative biotech companies in its ecosystem.

With $2.49 billion in venture capital investment in 2022 and the first half of 2023 and over 140,000 biotech jobs in the region, New Jersey is home to industry leaders such as Johnson & Johnson or Bristol Myers Squibb. But industry giants aren’t the only ones to call New Jersey home. Here are nine biotech companies to put on your radar.

Table of contents

    BioAegis Therapeutics

    BioAegis Therapeutics, based in North Brunswick, New Jersey, is a clinical-stage biotech company that focuses on developing therapies based on plasma gelsolin, a naturally occurring human protein known for its role in modulating inflammation and immune response. The company aims to harness plasma gelsolin to treat a variety of conditions where inflammation plays a critical role.

    BioAegis’ flagship project involves the development of recombinant human plasma gelsolin (rhu-pGSN) for the treatment of acute and chronic inflammatory diseases, including acute respiratory distress syndrome (ARDS). ARDS is a severe lung condition often seen in critically ill patients, and it represents a significant area of unmet medical need. The company’s approach leverages plasma gelsolin’s ability to modulate the body’s immune response without causing immunosuppression, offering potential treatments for diseases driven by inflammation and infection.

    The company has received $20 million from the Biomedical Advanced Research and Development Authority (BARDA) to advance its gelsolin therapy. It has also received U.S. Food and Drug Administration (FDA) Investigational New Drug (IND) clearance for its inflammation regulator protein, allowing it to proceed with clinical trials designed to evaluate the efficacy and safety of its therapy.

    Celularity

    Based in Florham Park, New Jersey, Celularity develops cellular therapies derived from the human placenta. These therapies are aimed at treating a variety of diseases, including cancer, immune disorders, and other conditions.

    Celularity’s pipeline features several promising products, including CYCART-19 for lymphoma, and CYNK-301 for acute myeloid leukemia. It is also developing therapies for non-small cell lung cancer and osteoarthritis. The company’s therapies utilize a range of cellular technologies, including CAR-T and NK cell therapies, to target diseases at the cellular level.

    The New Jersey Biotech company has partnerships in place with major pharmaceutical players like Sorrento Therapeutics and more recently Regeneron Pharmaceuticals. Additionally, Celularity received regulatory advancements, such as the Healthcare Common Procedure Coding System (HCPCS) Q Code for one of their biomaterial products, Biovance, facilitating health insurance claims by Medicare and thus its use in clinical settings.

    EpiBone

    EpiBone was founded in 2013, and is soon relocating from Brooklyn to Jersey City, New Jersey. The company is at the forefront of regenerative medicine, focusing on skeletal reconstruction by utilizing stem cell technology and engineering principles to grow bone and cartilage tailored to individual needs.

    EpiBone’s core technology leverages the patient’s stem cells to grow bone and cartilage in the lab. This process begins with a CT scan of the patient’s bone defect, which is used to create a precise digital model. Stem cells harvested from the patient’s fat tissue are then cultured and grown on a scaffold in a bioreactor that mimics the body’s natural conditions. This innovative approach allows the engineered bone to integrate seamlessly with the patient’s body, potentially reducing the risks associated with immune rejection and improving recovery times.

    EpiBone has raised a total of $33.8 million across multiple funding rounds and has also been supported by a $3.5 million Emerge tax credit from the New Jersey Economic Development Authority, aiding its relocation and expansion​. EpiBone is currently involved in clinical trials, including a significant study on knee cartilage replacement, which has received ND clearance from the FDA.

    Iveric Bio

    Iveric Bio, part of Astellas Pharma, is focusing on the development of novel treatments for retinal diseases, including age-related macular degeneration (AMD).

    Iveric Bio’s main product in development is Zimura (avacincaptad pegol), a complement C5 inhibitor, which plays a role in inflammation and cell destruction, aimed at treating geographic atrophy (GA), an advanced form of AMD that lacks FDA-approved treatment options.  Specifically, Zimura is designed to inhibit the activity of complement C5, thereby preventing the formation of a group of proteins known as the membrane attack complex (MAC) that can damage cells and tissues.  Zimura has demonstrated promising results in phase 3 clinical trials GATHER1 and GATHER2, showing potential to slow the progression of GA by targeting the complement pathway, a key factor in the disease’s development.

    Iveric Bio has recently been granted approval by the FDA for Zimura, a significant milestone for the company but also for patients as it is the first FDA-approved treatment for GA.

    Kayothera

    Kayothera develops precision medicines for oncology. The biotech company, established in 2019, is based in Princeton, New Jersey, and focuses on small-molecule drugs that target the retinoid signaling pathway. Retinoids are chemical compounds that are derivatives of vitamin A. This pathway is essential for a variety of biological processes including cell differentiation, cell growth, programmed cell death, and the maintenance of the proper function of the immune system.

    Kayothera’s primary therapeutic candidates include KAYO-1609 and other small-molecule inhibitors. KAYO-1609, its lead compound, is designed to inhibit the heat shock factor 1 (HSF1) pathway, which is crucial for the survival and proliferation of cancer cells. This drug is being developed as a potential treatment for various solid tumors, including ovarian carcinoma and other cancers. Kayothera expects to file an FDA IND application by the end of 2024.

    Kayothera has recently completed a notable extended series A financing round, raising $14 million to support the advancement of its lead candidate into clinical development.

    Nuvectis Pharma

    Nuvectis Pharma is a biopharmaceutical company based in Fort Lee, New Jersey, focusing on the development of precision medicines in oncology. The company was established in 2020, and aims to innovate in the treatment of cancer with targeted therapeutics.

    Nuvectis Pharma is developing novel small-molecule drugs, including NXP800 and NXP900, which are designed to target specific pathways involved in cancer progression. NXP800 is an oral inhibitor of the heat shock factor 1 (HSF1) pathway, currently in phase 1b clinical trials for the treatment of patients with ARID1a-mutated ovarian carcinoma, a condition with few effective treatments. In cancer, the HSF1 pathway is often upregulated, leading to increased expression of HSPs that can protect cancer cells from stressful conditions within tumors, such as hypoxia and nutrient deprivation. By aiding in the survival of these malignant cells, HSF1 contributes to tumor progression and resistance to therapies. 

    NXP900 targets the proto-oncogene c-Src and YES1 kinases, with phase 1a trials underway. c-Src is implicated in numerous cancer-related pathways. It plays a significant role in promoting cancer cell growth, survival, invasion, and metastasis. Over-activation of c-Src has been observed in various cancers, such as colon, liver, lung, breast, and pancreatic cancers, and is often associated with poor clinical prognosis. Similar to c-Src, YES1 is involved in cell growth and survival, and its dysregulation has been linked to the development and progression of cancer. YES1 has been identified as a critical mediator in signaling pathways that contribute to the malignant behavior of cancer cells, including aiding in the evasion of immune surveillance. By targeting both c-Src and YES1 kinases, NXP900 aims to inhibit these critical pathways, potentially leading to reduced tumor growth, decreased metastasis, and improved responses to existing cancer therapies.

    In 2021, Nuvectis Pharma raised $15 million through a series A funding and licensed the rights to develop and commercialize NXP800. 

    PDS Biotechnology

    PDS Biotechnology is a clinical-stage biotech company based in Princeton, New Jersey. It was founded in 2005 with the objective to develop targeted immunotherapies for cancer and infectious diseases using its proprietary Versamune platform. This platform is designed to enhance the body’s immune response against diseases by activating T cells in a way that improves treatment efficacy with manageable toxicity profiles.

    Its lead product, PDS0101, targets human papillomavirus (HPV)-associated cancers by combining the Versamune platform with other immunotherapies to enhance disease control and survival rates. The key mechanism of PDS0101 involves the presentation of HPV-specific antigens to the immune system, enabling the activation of T-cells that can identify and destroy cancer cells expressing these antigens. The company also has other candidates like PDS0301, an interleukin-12 based therapy, and PDS0202, a novel influenza vaccine, under development.

    PDS Biotechnology has reported promising results from phase 2 clinical trials involving PDS0101 for treating HPV-positive head and neck cancers, showing significant survival benefits and clinical responses. Additionally, the company has collaborated with notable institutions such as the National Cancer Institute (NCI).

    Rocket Pharmaceuticals

    Rocket Pharmaceuticals, headquartered in Cranbury, New Jersey, is focused on developing gene therapies for rare genetic disorders. The company is utilizing both adeno-associated viruses (AAV), small viruses that can deliver genetic material into cells without causing disease, and lentiviral vector (LVV) technologies, derived from the human immunodeficiency virus (HIV), with the ability to integrate into the host cell genome, to create treatments for diseases that have historically had limited treatment options.

    Rocket’s therapeutic pipeline includes projects targeting Fanconi anemia (FA), leukocyte adhesion deficiency type I (LAD-I), and Danon disease.

    Rocket Pharmaceuticals has recently made significant strides in advancing its clinical programs. Notably, the company’s therapy for LAD-I, referred to as RP-L201, has been recognized with various FDA designations such as Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Orphan Drug designations, which could facilitate a faster review process by the FDA. It is currently in a phase 1/2 clinical trial. 

    Additionally, the company’s treatment for Danon disease, RP-A501, is notable as it represents one of the first potential gene therapy approaches for this condition, having also received RMAT designation from the FDA and is currently in phase 2 clinical trials.

    Timber Pharmaceuticals

    Timber Pharmaceuticals, located in Basking Ridge, New Jersey, concentrates on developing treatments for rare and orphan dermatological diseases.

    Timber Pharmaceuticals’ pipeline includes several promising candidates, such as TMB-001, a topical formulation of isotretinoin intended for the treatment of congenital ichthyosis (CI), a group of rare genetic skin disorders characterized by dry, thickened, and scaling skin. Isotretinoin, traditionally used in oral form for severe acne, works by reducing the production of sebum and altering the lifecycle of skin cells. The topical application as seen in TMB-001 aims to concentrate its effects on the skin, potentially reducing systemic side effects and focusing the treatment on the areas most affected by the disease. TMB-001 has received Breakthrough Therapy and Fast Track designations from the FDA and is currently in phase 3.

    The company also develops TMB-003, for sclerotic skin diseases. By focusing on the underlying mechanisms of skin fibrosis and sclerosis, TMB-003 seeks to reduce the progression of skin thickening and improve symptoms associated with the disease. 

    In August 2023, LEO Pharma signed an agreement to acquire Timber Pharmaceuticals. This acquisition is intended to enhance LEO Pharma’s pipeline in medical dermatology by adding TMB-001, the main asset of the company to its portfolio.

    New Jersey: A critical biotech hub in the US

    New Jersey is poised to remain a critical hub for biotech innovation due to its dense concentration of pharmaceutical and biotech companies. This environment fosters collaboration and the rapid development of new technologies and therapies. The state government continues to support the biotech industry through incentives such as tax breaks, funding opportunities, and support services for startups. This support helps to maintain a fertile environment for new and existing companies to grow and thrive.

    New Jersey’s strong ties with academic institutions and research facilities contribute to its leading role in biotech research and development. These relationships not only help in the advancement of scientific research but also in the commercialization of new discoveries. The availability of a highly skilled workforce is another key driver. With several top universities and research institutions, New Jersey can provide the biotech industry with skilled professionals.

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