List of Gene Therapy Companies in New Jersey - 28

Alfa Wassermann Diagnostic Technologies is a provider of medical diagnostic instrumentation and reagents to the physician office, veterinary lab, and biopharmaceutical markets.

Apprentice provides an all-in-one, cloud-native platform (Tempo Manufacturing Cloud) that is purpose-built for regulated industries, and covers a wide range of manufacturing use cases across multiple industry verticals. Within life sciences, Tempo accelerates the end-to-end drug product lifecycle by connecting together distributed teams and sites, helping them execute better on the shop floor, and optimizing their operations through enterprise-wide visibility. Apprentice is trusted by global manufacturing leaders across various regulated industries. Tempo users belong to organizations responsible for contract manufacturing, cell & gene therapy, CRISPR technology, biopharma, biotechnology and adjacent industries.

BioCentriq is a premier contract development and manufacturing organization (CDMO) dedicated to advancing cell therapy. Since 2019, the company has specialized in GMP autologous and allogeneic cell therapies, offering end-to-end clinical-to-commercial capable services tailored to clients’ unique needs. Operating from two state-of-the-art U.S.-based facilities, BioCentriq provides a comprehensive range of services, including technology transfer and new technology evaluations, process and analytical development, cGMP manufacturing, quality control lot release, and stability studies. The company’s expertise spans diverse cell therapy modalities, such as T-cell (CAR-T, TCR), NK-cell, iPSC, and MSC-based products. With its proprietary LEAP™ manufacturing platform, BioCentriq accelerates service timelines while offering standardized and customizable solutions, including traditional fee-for-service, hybrid and person-in-plant operations, dedicated cleanroom capacity, and flexible service and contract arrangements. In addition to its technical capabilities, BioCentriq operates the Cell and Gene Therapy Experiential Learning Center, a training facility which provides hands-on and virtual instruction under the guidance of industry-experienced instructors and subject matter experts. The program has trained hundreds of participants, hosted major pharmaceutical companies, and integrates next-generation technologies and concepts into its curriculum including: Industry 4.0 and Lean principles, predictive analytics, augmented reality, and electronic batch record systems. Backed by GC Corporation, a South Korea-based leader in the healthcare, pharmaceutical and biotechnology industry, BioCentriq combines global expertise with a flexible working relationship, set to empower clients to develop and deliver cutting-edge, personalized therapies with unmatched precision and efficiency.

BioPharm International® covers all aspects of biopharmaceutical development, analysis, processing, and packaging, as well as business strategies and regulatory issues. The print and digital editorial coverage provides technical and business insight and analysis for all biologic-based therapies including monoclonal antibodies, vaccines, biosimilars, protein therapeutics, cell therapies, gene therapies, antibody-drug conjugates, and other emerging therapies.

Celularity, headquartered in Florham Park, N.J., is a clinical stage biotechnology company leading the next evolution in cellular medicine by delivering off-the-shelf allogeneic placenta-derived cellular therapies at unparalleled scale, quality and economics. Celularity’s innovative approach to cell therapy harnesses the unique therapeutic potential locked within the postpartum placenta. Through nature’s immunotherapy engine – the placenta – Celularity is leading the next evolution of cellular medicine with placenta-derived T cells, NK cells, and pluripotent stem cells to target unmet and underserved clinical needs in cancer, infectious and degenerative diseases.

Founded in 2002 and listed on the Hong Kong Stock Exchange in 2015, GenScript has an established global presence across Greater China, North America, the EU, and Asia Pacific. Today, over 200,000 customers from over 100+ countries and regions worldwide have used GenScript's premier, convenient, and reliable products and services. GenScript has over 6,900 employees globally, with about 10% R&D personnel. In addition, GenScript has a number of leading commercial technologies developed in the fields of synthetic biology, immunotherapy, antibody design, chemical synthesis, and bioinformatics, including more than 300 patents and about 900 patent applications. As of December 31, 2023, GenScript's products and services have been cited by 87,745 scientific papers worldwide. GenScript is committed to striving toward its vision of being the most reliable biotech company in the world to make humans and nature healthier through biotechnology.

IMUNON is a fully integrated, clinical stage biotechnology company focused on advancing a portfolio of innovative treatments that harness the body’s natural mechanisms to generate safe, effective, and durable responses across a broad array of human diseases. IMUNON has two platform technologies: Our TheraPlas® platform for the development of immunotherapies and other anti-cancer nucleic acid-based therapies and our PLACCINE platform for the development of nucleic acid vaccines for infectious diseases and cancer. The Company’s lead clinical program, GEN-1, is a DNA-based immunotherapy for the localized treatment of advanced ovarian cancer currently in Phase II development. GEN-1 works by instructing the body to produce safe and durable levels of powerful cancer fighting molecules, such as interleukin-12 (IL-12) and interferon gamma (IFN-γ), at the tumor site. In addition, we are conducting pre-clinical proof-of-concept studies on a nucleic acid vaccine candidate targeting SARS-CoV-2 virus in order to validate our PLACCINE platform. IMUNON’s platform technologies are based on the delivery of nucleic acids with novel synthetic delivery systems that are independent of viral vectors or devices. We will continue to leverage these platforms and to advance the technological frontier of plasmid DNA to better serve patients with difficult to treat conditions.

Generic and innovative specialty pharmaceutical products in injectable, ophthalmic dosage forms

Insmed Incorporated, a biopharmaceutical company, focuses on the development and commercialization of therapies for patients with serious and rare diseases. The company offers ARIKAYCE for the treatment of Mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients in the United States. It also develops INS1007, an oral reversible inhibitor of dipeptidyl peptidase 1 for the treatment of non-cystic fibrosis bronchiectasis and other inflammatory diseases; and INS1009, an inhaled formulation of a treprostinil prodrug for the treatment of rare pulmonary disorders, including pulmonary arterial hypertension. The company was founded in 1988 and is headquartered in Bridgewater, New Jersey.

iVIEW Therapeutics Inc. is a clinical stage biotechnology company focusing on innovative ophthalmic therapeutics. We are driven by the pursuit of innovative therapies for clear vision and healthier eyes.

At Janssen, we never stop working toward a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, and you can count on us to keep working tirelessly to make that future a reality for patients everywhere, by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Arterial Hypertension. Visit us: www.janssen.com Follow us: @JanssenGlobal on Twitter and Instagram Watch our videos on our YouTube channel: http://bit.ly/1LY2xQ5

Kyowa Kirin is a global specialty pharmaceutical company that applies state-of-the-art biotechnologies to discover and deliver novel medicines. We work on some of the hardest to treat diseases where need is high, and potential for life-changing impact is possible. The North America organization includes three offices in New Jersey and California that focus on drug discovery, product development, and commercialization. Together, we work as a collaborative team to understand clinical needs and advance innovations that have a profound impact on patient lives. Our growth in North America relies on entrepreneurial team players who are willing to share their expertise and ideas in an environment that prioritizes innovation, diversity, integrity and “wa.” Each person plays a significant role in shaping the work we do and the results we deliver.

Legend Biotech Corporation, a clinical-stage biopharmaceutical company, engages in the discovery and development of novel cell therapies for oncology and other indications. Its lead product candidate, LCAR-B38M/JNJ-4528, is an autologous CAR-T cell therapy that targets the B-cell maturation antigen. The company is conducting multiple clinical trials to evaluate LCAR-B38M/JNJ-4528 as an earlier line of therapy for multiple myeloma (MM), as well as a comparison of the treatment with standard triplet therapy in Revlimid-refractory MM. It also has a broad portfolio of earlier-stage autologous product candidates targeting various cancers, including non-hodgkins lymphoma (NHL), acute myeloid leukemia, and T cell lymphoma. In addition, the company is developing an allogeneic CAR-T product candidate targeting CD20 for the treatment of NHL, which is currently in an investigator-initiated Phase 1 clinical trial in China. Further, it has various product candidates in early preclinical and clinical development for the treatment of solid tumors, as well as infectious diseases. The company was founded in 2014 and is based in Somerset, New Jersey. Legend Biotech Corporation is a subsidiary of Genscript Biotech Corporation.

Merck & Co., Inc. operates as a healthcare company worldwide. It operates through two segments, Pharmaceutical and Animal Health. The Pharmaceutical segment offers human health pharmaceutical products in the areas of oncology, hospital acute care, immunology, neuroscience, virology, cardiovascular, and diabetes, as well as vaccine products, such as preventive pediatric, adolescent, and adult vaccines. The Animal Health segment discovers, develops, manufactures, and markets veterinary pharmaceuticals, vaccines, and health management solutions and services, as well as digitally connected identification, traceability, and monitoring products. It serves drug wholesalers and retailers, hospitals, and government agencies; managed health care providers, such as health maintenance organizations, pharmacy benefit managers, and other institutions; and physicians and physician distributors, veterinarians, and animal producers. The company has collaborations with AstraZeneca PLC; Bayer AG; Eisai Co., Ltd.; Ridgeback Biotherapeutics; and Gilead Sciences, Inc. to jointly develop and commercialize long-acting treatments in HIV. Merck & Co., Inc. was founded in 1891 and is headquartered in Kenilworth, New Jersey.

Montclair State University is a higher education institution that provides academic programs and services to students.

PhageNova Bio is a company that has developed a targeted gene delivery vector. The company is engaged in pioneering targeted gene expression for gene therapy.

At PMV, we believe that by specifically targeting each mutant p53 we can fundamentally disrupt the course of cancer. Our research and development efforts are dedicated to selectively targeting mutant p53, providing unique therapies for any patient whose tumor harbors these gene mutations.

Porton Advanced is a Cell and Gene Therapy (CGT) CDMO/CRO. We provide end-to-end research grade, process development (PD), analytical development (AD), GMP manufacturing, and testing services of AAV, Lentivirus, mRNA, and plasmid to meet clients’ therapeutic product needs at pre-clinical, clinical, and commercial stages. Our dedicated process and analytical development teams have established robust platform-based USP/DSP and GMP manufacturing capability and capacity, as well as proprietary technologies to further empower our clients’ successes. Our services include gene and cell engineering discovery research and technology licensing, process development and analytical development, testing, drug substances (DS) and drug products (DP), and cGMP manufacturing across plasmids, cell therapy, gene therapy, virotherapy, and mRNA-based therapeutic platforms.

PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for the treatment of rare disorders. The company offers Translarna (ataluren) and Emflaza (deflazacort) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients. It is developing Translarna, which is in Phase 2 clinical study for the treatment of nonsense mutation aniridia and nonsense mutation Dravet syndrome/CDKL5; and RG7916 and RO7034067 for the treatment of patients with spinal muscular atrophy, as well as PTC596 and PTC299, a small molecule dihydrooratate dehydrogenase (DHODH) inhibitor that inhibits de novo pyrimidine nucleotide synthesis, which is in Phase 1 clinical development stage to treat cancer patients. The company is also developing gene therapy product candidate that include PTC-AADC for the treatment of Aromatic L-amino acid decarboxylase deficiency. It has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., and the Spinal Muscular Atrophy Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey.

Rutgers University is an academic institution that offers a wide range of undergraduate and graduate programs, conducts research, and provides educational services.

Sannova is a boutique CRO with a unique Z factor approach, prioritizing high standards and exceptional services for clients. We offer fast, personalized, end to end analytical and bioanalytical services for complex, novel, and generic therapeutics. Our core values of quality, speed, and flexibility enable us to deliver exceptional results via our passionate team of expert scientists. Our commitment to reliable and reproducible results gives clients confidence in their therapeutic development.

Synbio Technologies aims to become the most trusted provider of DNA solutions that empower scientific discoveries by providing highly-accurate and cost-effective synthetic biology services and products for researchers around the world. We have developed a full range of DNA reading (sequencing), DNA writing (synthesis), and DNA editing (engineering) capabilities for various applications, including diagnostic DNA probes, precision medicine, protein production, antibody discovery, vaccine development, novel enzymes, molecular breeding, biofuel implication, and more. Our scientific capabilities cover all facets of DNA synthesis and engineering, offering a full range of support for molecular biology work, including but not limited to, oligo synthesis, gene synthesis, subcloning & PCR cloning, plasmid preparation, mutagenesis, DNA variant libraries, CRISPR sgRNA, protein expression and purification in multiple systems. Relying on our strong DNA sequencing, synthesis, engineering technology platform, and expertise in bioinformatics analysis, we have developed proprietary algorithms to facilitate and expedite the antibody discovery process. Our antibody discovery services include hybridoma sequencing, immune repertoire sequencing, antibody design and production, AI-guided heavy chain and light chain pairing, antibody humanization, affinity maturation, and sdAb.

Tevogen Bio brings together a highly skilled team of preeminent scientists, senior biopharmaceutical leaders, and manufacturing experts with the goal of advancing global health. The company’s patient centric mission is to leverage its proprietary cell and gene therapy platforms and novel business model to rapidly innovate breakthrough immunotherapies for cancers and viral infections with high unmet need. Tevogen Bio’s foundational vision is a healthier world through equitable access to lifesaving innovations.

University Hospital is a healthcare facility that provides medical services and health services.