List of Cell Therapies Companies in New York - 46

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.

Alphageneron Pharmaceuticals, Inc., is a privately-held clinical stage biotechnology company, developing Antibody Drug Conjugates (ADCs) and natural killer (NK) cell therapies, supported by Companion Diagnostics. Our team are industry veterans with experience in both large pharma and biotechnology startups committed to transforming the lives of cancer patients.

Apertura is a biotechnology company opening opportunities in gene therapy for treating debilitating diseases with limited options. We are pioneering next-generation AAV gene therapies by simultaneously engineering capsids, regulatory elements, and payloads to overcome current limitations in genetic medicine. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, we are located at the Cure in New York City.

Our unique modular closure system is called the Xvivo System model X2. Many people dismiss it as simply another isolator. It does look like one and has glove access where necessary in the workflow. However, unlike isolators it has extensive plug-and-play modularity so it can enclose any production process. Less obvious but just as important, it can easily be reconfigured to quickly accommodate any changes or improvements to the process such as new equipment or automation, or as you need to scale

BrainStorm Cell Therapeutics Inc. (NASDAQ) develops autologous stem cell therapies for neurodegenerative disorders such as ALS, MS, and Parkinson’s disease. These diseases have limited treatment options and represent unmet medical needs. NurOwn® is BrainStorm’s proprietary process for differentiating autologous mesenchymal stem cells (MSC) into neurotrophic factor (NTF)-secreting cells, transplanted at or near the affected tissue site. Our technology converts MSCs into a living drug delivery system for NTFs. We have proof-of-concept in various animal models of neurodegenerative diseases, including Parkinson's, Huntington's, ALS, MS, and peripheral nerve injury. We have completed two single-arm clinical trials in ALS patients in Israel, demonstrating good safety and tolerability profiles and strong efficacy signals. We also finalized a Phase 3 randomized, double-blind, placebo-controlled clinical trial at multiple US sites, supported by a $16 million USD grant from the California Institute for Regenerative Medicine (CIRM) and another grant from the ALS Association. Currently, we are planning a Phase 3b clinical trial of NurOwn® in ALS under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA). For more information, visit www.brainstorm-cell.com

Bristol-Myers Squibb Company discovers, develops, licenses, manufactures, and markets biopharmaceutical products worldwide. It offers products for hematology, oncology, cardiovascular, immunology, fibrotic, neuroscience, and covid-19 diseases. The company’s products include Revlimid, an oral immunomodulatory drug for the treatment of multiple myeloma; Eliquis, an oral inhibitor for reduction in risk of stroke/systemic embolism in NVAF, and for the treatment of DVT/PE; Opdivo for anti-cancer indications; Pomalyst/Imnovid indicated for patients with multiple myeloma; and Orencia for adult patients with active RA and psoriatic arthritis. It also provides Sprycel for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia; Yervoy for the treatment of patients with unresectable or metastatic melanoma; Abraxane, a protein-bound chemotherapy product; Reblozyl for the treatment of anemia in adult patients with beta thalassemia; and Empliciti for the treatment of multiple myeloma. In addition, the company offers Zeposia to treat relapsing forms of multiple sclerosis; Breyanzi, a CD19-directed genetically modified autologous T cell immunotherapy for the treatment of adult patients with relapsed or refractory large B-cell lymphoma; Inrebic, an oral kinase inhibitor indicated for the treatment of adult patients with myelofibrosis; and Onureg for the treatment of adult patients with AML. It sells products to wholesalers, distributors, pharmacies, retailers, hospitals, clinics, and government agencies. The company was formerly known as Bristol-Myers Company. The company was founded in 1887 and is headquartered in New York, New York.

Celyad Oncology is a cutting-edge biotechnology company dedicated to pioneering the discovery and advancement of revolutionary technologies for chimeric antigen receptor (CAR) T-cells. Its primary objective is to unlock the potential of its proprietary technology platforms and intellectual property, enabling to be at the forefront of developing next-generation CAR T-cell therapies. By fully leveraging its innovative technology platforms, Celyad Oncology aims to maximize the transformative impact of its candidate CAR T-cell therapies and redefine the future of CAR T-cell treatments. Celyad Oncology is based in Mont-Saint-Guibert, Belgium. For more information, please visit www.celyad.com.

Citeline powers a full suite of complementary business intelligence offerings to meet the evolving needs of health care professionals to accelerate the connection of treatments to patients and patients to treatments. These patient-focused solutions and services deliver and analyze data used to drive clinical, commercial and regulatory-related decisions and create real-world opportunities for growth. Our global teams of analysts, journalists and consultants keep their fingers on the pulse of the pharmaceutical, biomedical and medtech industries, covering it all with expert insights: key diseases, clinical trials, drug R&D and approvals, market forecasts and more.

Codagenix Inc. is a clinical stage biotechnology company with vaccine products against influenza, respiratory syncytial virus, dengue virus, and others currently under development. Codagenix uses a computer-based algorithm, called SAVE (Synthetic Attenuated Virus Engineering) to design viral genomes that are identical to the wild type strain at the amino acid level, but make less protein in human cells. The highly attenuated viruses make excellent vaccines or oncolytics against solid tumors. Codagenix represents the next wave in vaccine technology, untethered from traditional, time-consuming and unpredictable approaches to attenuating viruses.

https://www.comprehensivecellsolutions.com/contact/ Comprehensive Cell Solutions (CCS), is a business division of the New York Blood Center Enterprises, created to meet the needs of academic institutions, hospitals, biotech, pharmaceutical and startups organizations to launch new products, services, and perform clinical trials. CCS makes clinical and research challenges simple by leveraging NYBCe’s world-class expertise and experience in clinical trials management, regulatory and accreditation history. To date, CCS has: • Collaborated with early stage companies to co-develop innovative products. • Collaborated with diverse business entities and academic institutions to manage clinical trials, assist with budget, contracts, IRB submission, FDA submission, and invoicing. • Initiated and participated in national and international clinical trials. Comprehensive Cell Solutions seeks to expand its loyal customer base by offering an extensive suit of R&D products and services that have been designed to seamlessly facilitate partnerships, innovation support, and joint-ventures. CCS is quickly on its way to becoming a world renown pioneer of research and development in new generations of blood and cell-based therapies that address critical, unmet patient needs. Did You Know? • The CCS team has reviewed and overseen hundreds of research projects, preclinical and clinical studies? • CCS is a subject recruitment expert with access to hundreds of thousands of normal, healthy donors for most clinical trial studies? • CCS provides consultation on FDA requirements, certification and 510(K) application, technical performance, and improvements and adaptations to meet and exceed FDA certification requirements? • CCS has a plethora of ‘Ready to Ship’ and ‘Made to Order’ products available with one-click of a button? Contact us now to learn more about how we can support your endeavors https://www.comprehensivecellsolutions.com/contact/

Embleema's software platform brings precision medicine sooner to patients by collecting and generating regulatory-grade evidence. Our platform natively unifies clinical, molecular and real-world data and is the sole one used by the FDA for its regulatory evaluation of health products involving genomic datasets. The protocols and algorithms contained in our platform are also the basis for future CDISC standards for cell and gene therapies. The FDA also uses our platform to produce regulatory grade genomic sequences for SARS-CoV-2 and other microbial pathogens (project ARGOS). In addition to the FDA, our platform powers the Lupus Landmark Study, the largest clinical study for lupus in the world, the Human Epilepsy Projects and tens of other clinical studies for pharmaceutical companies, biotechs and patient groups. More than half of the top 10 Investigational sites in the US use our platform: John Hopkins, NYU, UCSF, Yale, UPenn, Washington University in St Louis, Cornell and many others also such as Mayo Clinic, Yale, Georgetown.

Emendo Biotherapeutics develops next generation gene editing tools for genetic disorders, addressing the current technological gaps for realizing the promise of gene therapy. We leverage our expertise in protein engineering to bring innovative and disruptive protein tools to resolve the bottleneck of the field.

FOXG1 Research Foundation (FRF) is a US-based 501(c)(3) not-for-profit dedicated to accelerating research to find a cure FOXG1 syndrome. Previously called Brain Factor 1, FOXG1 is one of the first and most fundamental genes formed during brain development. Most children with FOXG1 syndrome cannot walk, talk, crawl, or take care of their most basic needs; most have a feeding tube, most undergo major surgeries, and most suffer from life-threatening seizures. Many leading neuroscientists believe that FOXG1 holds the key to unlocking many brain disorders that affect millions of people including autism, schizophrenia, Alzheimers and brain cancers. They also believe FRF can get to human clinical trials in three to five years. The FOXG1 Research Foundation was founded by FOXG1 moms in 2017 with the support of a worldwide team of FOXG1 parents. FRF is quickly becoming known in the biotech arena as a Patient Organization making tremendous strides. FRF raised more than one million dollars in less than one year and has successfully funded six major research projects fostering the most comprehensive study of FOXG1 to-date. The potential to improve millions of lives is tremendous. The time is now. Join us to pioneer some of the most ground-breaking research in human history. 1 www.foxg1research.org

Gameto is a biotechnology company whose mission is to redefine female reproductive health by developing therapies that improve lives. Women’s health is a vastly underserved therapeutic area. Most conditions have few treatment options, and those available often cause frequent complications and offer limited clinical benefit. We aim to change that. Gameto is committed to using cutting-edge scientific advances to deliver better solutions to patients. Using cellular engineering, we have developed a platform to produce female reproductive cell lines. We use proprietary combinations of transcription factors to reprogram hiPSCs into engineered ovarian and endometrium cell lines that mimic the functions of endogenous cells, producing and responding to all hormones. Our team of cellular engineers is using this platform to advance a portfolio of cellular therapeutics for female reproductive diseases. Our first program, Fertilo, is a therapeutic for IVF and egg freezing, aiming to make the process shorter, safer and more effective. Additional applications of our platform include, an organoid of the female reproductive system, Deovo, and a cell-based therapeutic to ameliorate the health consequences of primary ovarian insufficiency and menopause, Ameno.

The Interactive Advertising Bureau (IAB) empowers the media and marketing industries to thrive in the digital economy. Its membership comprises more than 700 leading media companies, brands, agencies, and the technology firms responsible for selling, delivering, and optimizing digital ad marketing campaigns. The trade group fields critical research on interactive advertising, while also educating brands, agencies, and the wider business community on the importance of digital marketing. In affiliation with the IAB Tech Lab, IAB develops technical standards and solutions. IAB is committed to professional development and elevating the knowledge, skills, expertise, and diversity of the workforce across the industry. Through the work of its public policy office in Washington, D.C., the trade association advocates for its members and promotes the value of the interactive advertising industry to legislators and policymakers. Founded in 1996, IAB is headquartered in New York City.

iCell Gene Therapeutics is developing Chimeric Antigen Receptor (CAR) engineered cells with improved functional properties and expanded applicability. We are a clinical stage company focused to demonstrate clinical proof of concept for unique, patent protected, targets and strategies. Our pipeline includes multiple additional first in man CAR strategies to target hematological cancers, non-hematological cancers and autoimmune disorders.

Immunyx Pharma is focused on changing the landscape of immune modulation by targeting neutrophil toxicity in numerous chronic diseases. The company was built by world leaders in neutrophil biology and nanoparticle delivery to use a targeted neutrophil nanoparticle platform (TENNs) to solve the significant issues in neutrophil therapy. While big pharma has invested heavily in neutrophil targeted drugs, they have failed to bring solutions to the clinic due to the danger of causing neutropenia and the difficulty of modulating such a large & dynamic population of cells. Our TENN platform is intended to manipulate neutrophil toxic behavior without destroying their disease fighting function, while bringing a concentrated payload of drug to all neutrophils throughout the body with a long bioavailability. We are currently focusing on applying our platform to modulate neutrophil activity in cancer and inflammatory disease.

IN8bio, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of gamma-delta T cell therapies for the treatment of cancer. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems, and can intrinsically differentiate between healthy and diseased tissue. IN8bio develops ex vivo-expanded and activated gamma-delta T cells based upon its deep expertise in gamma-delta T cell biology, proprietary genetic engineering, and cell-type specific manufacturing capabilities, which we refer to collectively as our DeltEx platform. IN8bio’s platform employs allogeneic, autologous, and genetically modified approaches to develop novel cell therapies, which are designed to effectively identify and eradicate tumor cells. IN8bio is currently the most clinically advanced gamma-delta T cell company and the first company to bring genetically modified gamma-delta T cells into the clinic. For more information about the Company and its programs, visit www.IN8bio.com.

Based in New York City, Lexeo Therapeutics is a clinical-stage genetic medicines company dedicated to transforming healthcare by applying pioneering science to fundamentally change how disease is treated. Building on groundbreaking research from Weill Cornell Medicine and the University of California San Diego, Lexeo partners with preeminent institutions on the cutting edge of gene therapy research. Using a stepwise development approach, Lexeo is leveraging early proof-of-concept functional and biomarker data to advance a pipeline of cardiovascular and APOE4 associated Alzheimer's disease programs, and is led by pioneers and experts with decades of collective experience in genetic medicines, rare disease drug development, manufacturing and commercialization. For more information, please visit www.lexeotx.com.

LineaRx, an Applied DNA Sciences, Inc. company (NASDAQ: APDN) delivers enzymatically-produced LinearDNA™ as an alternative to current plasmid-based DNA manufacturing processes with advantages of speed, purity and scalability to support the next generation of nucleic acid-based therapies. Manufactured by a proprietary, large-scale polymerase chain reaction (“PCRâ€?) based manufacturing platform, LinearDNA allows for the rapid and efficient cell-free production of high-fidelity DNA sequences.

MeiraGTx is a clinical-stage gene therapy company focused on developing potentially curative treatments for patients living with serious diseases.

Mercer University is a higher education institution offering a variety of academic programs and degrees.

Our vision, to disrupt cancer with the development of highly innovative immunotherapies aimed at increasing patient survival rates MimiVax is a privately held, clinical-stage biotechnology company focused on the development and commercialization of immunotherapeutic vaccines and targeted therapies for the treatment of cancer. SurVaxM, our lead immunotherapeutic vaccine, has completed a Phase 2a clinical trial in adults with newly diagnosed glioblastoma.

Mispro's contract vivarium (CV) solution offers emerging, mid-sized, and even mature biopharma companies access to state-of-the-art vivarium research facilities and laboratory animal services for preclinical drug discovery and development. Our space plus services model enables founding scientists and research teams to keep in in vivo rodent studies in-house without having to incur the costs and operational complexities of internalizing a vivarium facility.

The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services—from community-based facilities to tertiary and quaternary care. WHO WE ARE We are compassionate collaborators—38,000 strong—working to heal, teach, and advance medicine in New York City and throughout the world. WHAT WE BELIEVE We believe in challenging the status quo. Forging a new pathway in clinical excellence is only possible by putting the patient at the center of the experience. WHY WORK WITH US Here, innovation is valued and collaboration is integral. Mount Sinai is full of friendly, helpful people who share a common devotion to delivering exceptional patient care. Yet we’re as diverse as the city we call home—culturally, ethnically, in outlook and lifestyle. When you join us, you become part of Mount Sinai’s unrivaled record of achievement, education, and advancement as we revolutionize healthcare together.

Neural Stem Cell Institute is a regenerative medicine company that focuses on developing stem cell therapies for diseases of the nervous system.

OverT Bio is a biotech company focused on developing cell therapies for solid tumors. The company aims to enable the curative potential of cell therapies through innovative genetic modifications and reprogramming of immune cells.

Pfizer Inc. discovers, develops, manufactures, markets, distributes, and sells biopharmaceutical products worldwide. It offers medicines and vaccines in various therapeutic areas, including cardiovascular metabolic and women’s health under the Premarin family and Eliquis brands; biologics, small molecules, immunotherapies, and biosimilars under the Ibrance, Xtandi, Sutent, Inlyta, Retacrit, Lorbrena, and Braftovi brands; and sterile injectable and anti-infective medicines, and oral COVID-19 treatment under the Sulperazon, Medrol, Zavicefta, Zithromax, Vfend, Panzyga, and Paxlovid brands. The company also provides medicines and vaccines in various therapeutic areas, such as pneumococcal disease, meningococcal disease, tick-borne encephalitis, and COVID-19 under the Comirnaty/BNT162b2, Nimenrix, FSME/IMMUN-TicoVac, Trumenba, and the Prevnar family brands; biosimilars for chronic immune and inflammatory diseases under the Xeljanz, Enbrel, Inflectra, Eucrisa/Staquis, and Cibinqo brands; and amyloidosis, hemophilia, and endocrine diseases under the Vyndaqel/Vyndamax, BeneFIX, and Genotropin brands. In addition, the company is involved in the contract manufacturing business. It serves wholesalers, retailers, hospitals, clinics, government agencies, pharmacies, and individual provider offices, as well as disease control and prevention centers. The company has collaboration agreements with Bristol-Myers Squibb Company; Astellas Pharma US, Inc.; Myovant Sciences Ltd.; Akcea Therapeutics, Inc; Merck KGaA; Valneva SE; BioNTech SE; and Arvinas, Inc. Pfizer Inc. was founded in 1849 and is headquartered in New York, New York.

Phoenix Nest Biotech is a biotechnology company that focuses on treating Sanfilippo Syndrome by collaborating with leading academics and utilizing licensed key technologies.

Prevail Therapeutics was acquired by Eli Lilly and Company. At Prevail Therapeutics, we are developing a pipeline of gene therapies to slow or stop the underlying disease process for patients with Parkinson’s disease and other neurodegenerative disorders. We are leveraging recent breakthroughs in human genetics that point to potential disease-modifying targets for neurodegenerative diseases. Our programs utilize a precision medicine approach targeting patient populations with urgent unmet needs, where there are currently no available therapies that modify the progressive course of their disorders.

Protara Therapeutics, Inc., a clinical-stage company, engages in the identifying and advancing transformative therapies for people with rare and specialty diseases. The company's lead program is TARA-002, an investigational cell therapy for the treatment of lymphatic malformations. It also develops intravenous choline chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure associated liver disease. The company was formerly known as ArTara Therapeutics, Inc. and changed its name to Protara Therapeutics, Inc. in May 2020. Protara Therapeutics, Inc. is based in New York, New York.

Repair Biotechnologies is a preclinical biotechnology company focused on developing drugs for cholesterol and aging-related diseases. Our first-in-class Cholesterol Degrading Platform (CDP) technology is aimed at reversing atherosclerosis, familial hypercholesterolemias, and other conditions in which excess or modified cholesterol drives pathology.

Rocket Pharmaceuticals (NASDAQ: RCKT), Ltd is a biotechnology company focused on seeking cures through molecular and gene therapy. We are developing first-in-class treatments for patients with rare and often devastating orphan diseases using a lentiviral-based and adeno-associated virus (AAV) therapy approach. We have developed rewarding partnerships with a number of leading international research institutions as well as with centers of manufacturing and cell processing expertise, while building an internal team to drive the research, development, clinical and regulatory programs. Ultimately we aim to launch and globally commercialize innovative gene therapies in disease areas with high unmet need.

Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. We do this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology. The therapies in development across our family of companies target a wide range of diseases including uterine fibroids, endometriosis, prostate cancer, Parkinson’s disease, diabetes, pulmonary arterial hypertension, and multiple rare and fatal pediatric conditions. We focus on disease areas where the magnitude of R&D investment from industry is disproportionately low relative to societal medical needs. In addition to our biopharmaceutical subsidiaries, we also build technology-focused Vants focused on improving the process of developing and commercializing medicines.

Dynamic therapeutics immuno-oncology startup in NYC. Synthis’s team is a talented group of scientists with a groundbreaking idea to improve patient care and outcomes. As long-standing tumor immunologists, the team is passionate about the science and therapeutic power behind restoring immune function in cancer patients.

Tactiva Therapeutics is an immuno-oncology company specializing in potent cancer immunotherapy using a dual T cell receptor approach.

TG Therapeutics is a biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for B-cell diseases. In addition to a research pipeline including several investigational medicines, TG has completed a Phase 3 program for ublituximab, an investigational glycoengineered monoclonal antibody that targets a unique epitope on CD20-expressing B-cells, to treat patients with relapsing forms of multiple sclerosis (RMS).

University at Buffalo Center for Entrepreneurial Leadership is an education institution that offers entrepreneurship and leadership development programs.

Waypoint's proprietary spatial multi-omics platform enables us to solve critical issues around understanding how cell therapies navigate the three-dimensional tumor microenvironment and allow the measurement of cell-to-cell interactions.