List of Gene Therapy Companies in Florida - 23

Adolore Biotherapeutics, Inc., is a biotechnology company focused on developing novel therapies for the treatment of chronic pain and other pain and nervous system conditions or disorders. Our best-in-class lead programs are long-acting locally acting gene-therapies that are opioid-free Disease Modifying Anti-Pain therapies (DMAPs) for the treatment of a variety of chronic pain indications. The Company’s two current CA8* gene therapy programs are in preclinical development for treatment of patients suffering from erythromelalgia, a life-long heritable chronic pain condition representing an orphan drug disease with no approved therapy, and chronic osteoarthritis knee pain, affecting a large number of patients that is often treated with opioids due to the lack of alternatives, thus contributing to the ongoing opioid crisis.

AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC's product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products Find AGTC on YouTube at goo.gl/sMdJqX.

Ambulero is a cell and gene therapy spin-out of the University of Miami focused on advancing new therapies to fight vascular disease. We are a platform company that can deliver the adhesion molecule E-selectin using different cell and synthetic vector technologies. We work on rare vascular diseases as well as more common indications effecting large patient populations.

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to restore and improve the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease. Lead development candidate AGTC-501, is a gene therapy program in Phase II clinical trials for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss. Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space. The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.

Optimize Product Recovery and Scalability - Since 2009, CARR Biosystems has been providing single-use separation systems to biopharma companies seeking low-shear separation, high-recovery performance, and fast processing time. Avoid the expense and waste of CIP/SIP stainless steel centrifuges and step up to the scalable, industry-leader. Our wide range of separation equipment accelerates the production of high-quality biological products and medicines, for seamless scaling up, down, or out.

Cognigenics develops innovative RNA-based gene therapies targeting memory loss, cognitive decline, anxiety associated with mild cognitive impairment and other neurocognitive and neuropsychiatric disorders. Our intranasal delivery platform targets neuronal receptors in the brain involved in CNS disorders including anxiety, and Mild Cognitive Impairment.

Cytovia Therapeutics aims to accelerate patient access to transformational cell therapies and immunotherapies, addressing several of the most challenging unmet medical needs in cancer. Cytovia focuses on harnessing the innate immune system by developing complementary and disruptive Flex-NK™ bispecific antibody and iPSC-derived, TALEN® gene-edited NK-cell platforms. FLEX-NK™ bispecific antibodies are built on a quadrivalent multifunctional antibody platform designed to engage natural killer cells by targeting NKp46 using Cytovia's proprietary Flex-NK™ technology. The company is also developing three types of iPSC-derived NK (or iNK) cells: unedited iNK cells, TALEN® gene-edited iNK cells with improved function and persistence, and TALEN® gene-edited iNK cells with chimeric antigen receptors (CAR-iNKs) to improve tumor-specific targeting. ​ These two technology platforms are being used to develop treatment of patients with solid tumors such as hepatocellular carcinoma (HCC) and glioblastoma as well as hematological malignancies such as relapsed multiple myeloma. Clinical studies are expected to initiate in 2023.

EXUMA Biotech is a clinical-stage biotechnology company committed to bringing to market cell & gene therapies for the treatment of cancer. Founded in 2015, The company has rapidly built its core technologies and a growing global presence with operations in China (Shanghai and Shenzhen), Grand Cayman, and headquarters in West Palm Beach, FL.

Florida International University is a higher education institution that provides academic programs and conducts research.

Gift of Life Marrow Registry facilitates blood stem cell and marrow transplants for children and adults battling life-threatening diseases, including leukemia, lymphoma, sickle cell and other genetic disorders. Gift of Life is a leading national bone marrow and stem cell registry that serves patients all over the world seeking donors for transplants. It was founded during the grass roots efforts to save the life of New Jersey leukemia patient Jay Feinberg, and has grown to become a leading global registry. Gift of Life is accredited by the World Marrow Donor Association, and is an affiliate donor registry of the National Marrow Donor Program and participant in the international Bone Marrow Donors Worldwide registry. Gift of Life added an in-house apheresis center in 2019 and cellular therapy laboratory in 2020, ensuring its commitment to quality and consistency every step of the way.

LifeSouth is a 501(c)(3) non-profit community blood supplier for more than 125 hospitals in Alabama, Florida and Georgia.

We are a clinical-stage biotechnology company developing cellular therapies for aging-related and life-threatening conditions. Our lead investigational product is Lomecel-B™, which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young healthy adult donors. We believe that by using the same cells that promote tissue repair, organ maintenance, and immune system function, we can develop safe and effective therapies for some of the most difficult diseases and conditions associated with aging. We are currently conducting Phase 1 and 2 clinical trials in the following indications: Aging Frailty, Alzheimer’s disease, the Metabolic Syndrome, Acute Respiratory Distress Syndrome (ARDS), and hypoplastic left heart syndrome (HLHS), which is the current exception to our focus on aging. Our mission is to advance Lomecel-B™ and other cell-based product candidates into pivotal Phase 3 trials, with the goal of achieving regulatory approvals, subsequent commercialization and broad use by the healthcare community. Our philosophy is that healthy aging can be improved through regenerative medicine approaches. Life expectancy has substantially increased over the past century as a result of medical and public health advancements. However, this increase in longevity has not been paralleled by the number of years a person is expected to live in relatively good health, free of chronic disease and disabilities of aging – a period known as healthspan. As we age, we experience a profound decline, in both number and function, in our own MSCs, a decrease in immune system function, decline in blood vessel functioning, chronic inflammation and other issues. Our clinical data suggest that Lomecel-B™ addresses these conditions through multiple mechanisms of action, or MOAs, that simultaneously target key aging-related processes.

Be at the heart of exceptional care. Team MHS Florida is an award-winning group of friends and colleagues at one of the largest not-for-profit health systems in the nation. We're 16,000 strong, advancing towards a brighter future together. We're passionate about the work we do, delivering deep, personalized care for patients and their families. That’s what we call the Memorial Experience. Memorial Healthcare System is proud to be an equal opportunity employer committed to workplace diversity. Memorial Healthcare System recruits, hires and promotes qualified candidates for employment opportunities without regard to race, color, age, religion, gender, gender identity or expression, sexual orientation, national origin, veteran status, disability, genetic information, or any factor prohibited by law. We are proud to offer Veteran’s Preference to former military, reservists and military spouses (including widows and widowers). You must indicate your status on your application to take advantage of this program. Employment is subject to post offer, pre-placement assessment, including drug testing.

OrganaBio was founded in 2018 with the mission to become the hub for tissue sourcing, clinical sample processing and contract manufacturing services to support cell and gene therapy developers around the globe. At our core, we continually apply a data-driven approach with meaningful insights across our strategically located facilities to provide solutions to our clients where they need it most. Headquartered in Miami, Florida, OrganaBio delivers products and services that span the full development lifecycle – from proprietary tissue supply chains and cellular starting materials to expert development, testing, and other support services that expedite the path to clinical translation.

Pacira BioSciences Inc. is a leading provider of non-opioid pain management and regenerative health solutions. We are dedicated to offering patients—and the clinicians who treat them—cutting-edge therapies to treat pain with the potential to reduce or eliminate the need for opioids. Founded in 2006 and headquartered in Tampa, FL, Pacira has offices in Parsippany, NJ as well as San Diego, CA. With more than 700 employees who share a passion for delivering improved patient care and reducing the need for opioids, Pacira is here to challenge and transform the way we think about – and treat – pain. We are committed to providing solutions that address unmet medical need and improve clinical results, and empowering patients and healthcare providers to choose non-opioid options. Our non-opioid product portfolio is aimed at opioid minimization, long-lasting pain control, and enhanced recovery. Find out why we aim for change at Pacira.com/commitments

San Rocco Therapeutics is a biotechnology company with its primary focus on curative gene therapies for Sickle Cell Disease and Thalassemia

The Warrington College of Business is one of the nation’s premier business schools. As a part of a Research 1 institution, Warrington is dedicated to an extensive research agenda in addition to cultivating the young minds of our future leaders of industry. The College is comprised of the Heavener School of Business, the Fisher School of Accounting, and the Hough Graduate School of Business. Together, these three entities provide a full and enriching business education experience that not only prepares our students for business success, but enables them to thrive in the global economy. The University of Florida Warrington College of Business intends to educate, inform and provide updated information on Warrington's activities and news and to support and promote Warrington's objectives for these activities through its social media site. All Warrington comments are made by Warrington designees. This site is not a public forum. Social media users may share ideas through commentary that is consistent with and furthers the objectives of a Warrington post and the University of Florida Warrington College of Business reserves the right to remove any comments that do not fall within this purpose.

U.S. Stem Cell, Inc. is a leader in regenerative medicine and is committed to providing autologous cell therapies for the treatment of degenerative conditions. The company specializes in the development of proprietary, physician-based stem cell therapies; novel regenerative solutions, including its lead product AdipoCell™; physician training and certification; veterinary applications; stem cell banking; as well as creating and managing new stem cell clinics. In the past 20 years, to the company’s knowledge, U.S. Stem Cell, Inc. has completed more clinical treatments than any other regenerative care provider in the world. With more than 700 physicians trained and certified with the company, U.S. Stem Cell, Inc. boasts the largest network of physicians working together to continue its mission of harnessing the body’s own ability to heal from within to treat a wide variety of conditions and to improve quality of life.

U.S. Stem Cell, Inc. is a leader in regenerative medicine and is committed to providing autologous cell therapies for the treatment of degenerative conditions. The company specializes in the development of proprietary, physician-based stem cell therapies; novel regenerative solutions, including its lead product AdipoCell™; physician training and certification; veterinary applications; stem cell banking; as well as creating and managing new stem cell clinics. In the past 20 years, to the company’s knowledge, U.S. Stem Cell, Inc. has completed more clinical treatments than any other regenerative care provider in the world. With more than 700 physicians trained and certified with the company, U.S. Stem Cell, Inc. boasts the largest network of physicians working together to continue its mission of harnessing the body’s own ability to heal from within to treat a wide variety of conditions and to improve quality of life.

Vycellix is a biopharmaceutical company developing novel platforms to optimize and enhance cell & gene based-therapies. The company's platforms include VY-UC to generate immune-privileged universal cells without altering the components that control self-recognition (HLA).