List of Gene Therapy Companies in North Carolina - 30

Adrenas Therapeutics is a company that was created with a single mission: to work with scientists, physicians, and patients in developing a gene therapy for people affected by CAH. Adrenas is a subsidiary of BridgeBio, a public company dedicated to finding, developing, and delivering breakthrough medicines for genetic diseases to patients as quickly and safely as possible.

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. Learn more at askbio.com. Vision - Pioneering science to create transformative molecular medicines. Mission - Lead innovative science and drive clinical outcomes to transform people's lives. Values: • Be a Pioneer. We are not afraid of the impossible and to innovate to make gene therapies accessible to those in need. • Cultivate Collaboration. Strive to be the best teammate, actively listen, openly communicate, and embrace diverse points of view. • Embrace Responsibility. We are humbled by the enormity of our mission. We hold a relentless commitment to advance science and clinical outcomes for our patients, families, and caregivers. • Raise the Bar. Continuously drive improvements and efficiencies. Seek and provide constructive feedback. Have a bias for learning and action. • Act with Uncompromising Integrity. Be honest, transparent, and committed to doing what’s right in every situation. Make clear commitments and follow through.

Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise.

BA Sciences is a cGMP compliant, FDA/DEA registered, ISO/IEC-17025:2017 certified analytical laboratory located in Salem, NH. BA provides testing services to Pharmaceutical, Biopharmaceutical and Medical Device companies worldwide including: Analytical Testing and Method Development, Microbiological Testing and Environmental Monitoring Services, Biologics, Stability Testing & ICH-compliant Storage, Impurities Testing, Advanced Therapeutics and Extractables & Leachables Studies.

Bedrock Therapeutics is an AAV gene therapy research and development company developing platform and disease specific technologies for the treatment of ocular, hematologic, and other diseases. Drs. Matt Hirsch, Chenwen Li, Bryan Sutton, and Brian Gilger are co-founders of Bedrock. Drs. Gilger and Hirsch oversee the de novo design and validation of ocular assets, and Drs. Li and Sutton oversee basics aspects of the various host immune responses to AAV vectors and protein engineering and design.

Lifechanging breakthroughs like cell and gene therapy are driving a medical revolution. But a lack of purpose-built development and manufacturing tools keeps these treatments from reaching more patients. At Charter Medical, we're on a mission to evolve single-use cell processing step-by-step with the needs of the biopharmaceutical innovators who count on us. With proven customization capabilities and an innovation roadmap that includes pioneering design and biomaterials. With industry-leading expertise and continuous improvement to simplify and standardize biomanufacturing systems so they can scale reliably. And with a deep commitment to responsiveness, collaboration and urgency. Because we know single-use advances are essential to helping our clients make revolutionary therapies more accessible to patients who need them – now.

CLAIRIgene is a preclinical stage biopharmaceutical company focused on development gene therapies for neurodegenerative diseases. Founded by innovative scientists with decades of experience, CLAIRIgene is committed to rapidly advancing their therapeutics to the clinic.

At GeneVentiv, we’re blazing a trail. We’re driven by curiosity. We’re fueled by learning expressed as innovation. Our mission is to discover, develop and deliver gene therapies for patients who had no hope of a cure – until now. We are founded on discovery bringing together insights into Adeno Associated Virus (AAV) directed gene therapy, Hemophilia and how to use the common pathway in the clotting cascade to surmount the obstacle of inhibitors. Patients with inhibitors are unable to respond to current therapies and gene therapies in development due to neutralizing antibodies (inhibitors) formed by the body in response to treatment with missing clotting factor. Our goal is to cure inhibitor patients with a single lifetime infusion, not to offer a half measure of weekly injections of a bypass agent.

Inceptor Bio is developing multiple next-generation cell therapy platforms to cure difficult-to-treat cancers with a focus on novel mechanisms to enhance immune cell performance in the tumor microenvironment.

Isolere's mission is to increase global access to life-changing therapeutics and vaccines by offering novel purification reagents to alleviate manufacturing bottlenecks. The IsoTag™ reagents reimagine bioprocessing by combining target specific affinity ligands with phase separating biopolymers. The Affinity Liquid Phase Separation (ALPS) allows target biologics to be separated on the basis of affinity and size in a single process and can be implemented in a variety of techniques including microfiltration TFF and centrifugation processes. Isolere is currently developing purification solutions for adeno-associated virus (AAV), lentivirus (LV), adenovirus (AdV) and mRNA. Visit our website to learn more! Isolere Bio was acquired by Donaldson Company, Inc. (NYSE: DCI), a leading worldwide provider of innovative filtration products and solutions, in February 2023.

Istari Oncology, Inc. is a clinical-stage biotechnology company focused on novel immuno-oncology and immunotherapy platforms for the treatment of glioblastoma and a wide variety of tumors. The company was founded by Darell Bigner, MD, and Matthias Gromeier, MD, of Duke University Medical Center in 2016. Both are leaders in their respective research fields of virology, immunology, monoclonal development and clinical medicine, particularly in the treatment of brain tumors.

KBI Biopharma is an award-winning biopharmaceutical contract services organization providing fully-integrated, accelerated drug development and biomanufacturing services to pharmaceutical and biotechnology companies globally. With each of our 500+ client partners, we have worked closely to personalize and rapidly accelerate their drug development programs. Built upon a foundation of world-class analytical capabilities, we deliver efficient process development and clinical and commercial cGMP manufacturing services for mammalian and microbial programs. KBI is proud to be a JSR Life Sciences Company.

Next generation CRISPR technology for programmed cell death. Focused on developing therapies for infectious disease and the microbiome. Locus Biosciences’ novel approach to precision antimicrobials works by taking advantage of a part of an immune system present in many bacteria called the CRISPR-Cas system. The CRISPR-Cas system protects bacteria from invaders such as viruses by creating small strands of RNA called CRISPR RNAs, which match DNA sequences specific to a given invader. When the CRISPR RNAs find a match, they activate Cas proteins that cut the DNA.

Marken is the clinical precision logistics and advanced therapy subsidiary of UPS Healthcare. The UPS Healthcare network consists of 200+ locations worldwide. Marken offers a state-of-the-art GMP-compliant depot network and logistic hubs for clinical drug product storage and distribution in 46 locations worldwide, while maintaining the leading position for cell and gene therapy services, direct-to-patient and home healthcare services, biological sample shipments and biological kit production. Marken's dedicated 2,600+ staff members manage 200,000 drug product and biological sample shipments every month at all temperature ranges in more than 220 countries and territories and have orchestrated 17,500+ home healthcare visits. Additional services such as ancillary material sourcing, storage and distribution, shipment lane verification and qualifications, as well as GDP, regulatory and compliance consultancy add to Marken's unique position in the pharma and logistics industry. Delivering What Matters From Clinical to Commercial.

Ocuphire Pharma is a clinical-stage ophthalmic biopharmaceutical company developing and commercializing therapies for the treatment of eye disorders.

Parexel is among the world’s largest clinical research organizations (CROs), providing the full range of Phase I to IV clinical development services to help life-saving treatments reach patients faster. Leveraging the breadth of our clinical, regulatory and therapeutic expertise, our team of more than 21,000 global professionals collaborates with biopharmaceutical leaders, emerging innovators and sites to design and deliver clinical trials with patients in mind, increasing access and participation to make clinical research a care option for anyone, anywhere. Our depth of industry knowledge and strong track record gained over the past 40 years is moving the industry forward and advancing clinical research in healthcare’s most complex areas, while our innovation ecosystem offers the best solutions to make every phase of the clinical trial process more efficient. With the people, insight and focus on operational excellence, we work With HeartTM every day to treat patients with dignity and continuously learn from their experiences, so every trial makes a difference. For more information, visit parexel.com. Community Guidelines Because Parexel’s social media channels are open to the general public and employees, we are not responsible for views expressed other than our own. However, we do not tolerate posts that are: • Abusive, harassing or threatening to others. • Defamatory, offensive, obscene, vulgar or depicting violence. • Hateful targeting by race/ethnicity, age, color, creed, religion, gender, sexual preference or orientation, nationality or political beliefs. • Sexually explicit or pornographic. • Fraudulent, deceptive, libelous, misleading or unlawful. • Referencing criminal or illegal activity. • Spamming. We reserve the right to remove any comments that do not adhere to our guidelines as well as report users who violate the rules of our page.

Phitonex uses a DNA nanotechnology platform to create highly customizable, deterministic fluorescent nanoparticles called Phitons.™

Precision BioSciences, Inc. (Nasdaq: DTIL), is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. Using ARCUS, Precision is developing allogeneic CAR T and in vivo gene correction therapies for cancer and infectious diseases.

Premier Research, a global clinical research, product development, and consulting company, is dedicated to helping innovators transform life-changing ideas and breakthrough science into new medical treatments. We offer strategic solutions across the entire development lifecycle, from pre-clinical through commercialization, specializing in smart study design and full-service clinical trial management. Leveraging technology and therapeutic expertise, we deliver clean, conclusive data with a focus on reducing development timelines, securing access to the right patients, and effectively navigating global regulations to ensure submission-ready results. As an organization that puts patients first, we pride ourselves on helping customers answer the unmet needs of patients across a broad range of medical conditions. Are you ready to make a real difference and help us transform this exciting industry? Follow our company page for more information about us and the work we do, and check out our current openings for your next career move!

ProKidney Corp., a clinical-stage biotechnology, engages in developing cellular therapy candidates. It is developing Renal Autologous Cell Therapy, an autologous homologous cell admixture that is in a Phase III development program, as well as Phase II clinical trials for the treatment of moderate to severe diabetic kidney disease; and Phase I clinical trial for patients with congenital anomalies of the kidney and urinary tract. The company was founded in 2015 and is headquartered in Winston-Salem, North Carolina.

Rainbow Company Youth Theatre is a nationally acclaimed, award-winning youth theatre group funded by the city of Las Vegas. For more than 45 years, the Rainbow Company has been providing quality theatre experiences for young people and their families.

Scorpius Holdings Inc., through its Scorpius BioManufacturing subsidiary, is an integrated contract development and manufacturing organization dedicated to transparent collaboration and flexible, high-quality biologics. With an experienced team and new, purpose-built U.S. facilities, Scorpius Holdings Inc. is committed to excellence in large molecule cGMP manufacturing.

Torque Bio is an ambitious newly launched biotechnology start-up leveraging the power of an innovative technology platform for the treatment of disease. We believe our science represents a novel class of versatile, tunable, and persistent therapeutics that have the potential to transform the development of new medicines.

The vast majority of human disease is governed by the epigenome, the subtle machinery that controls the volume of gene expression in cells. Tune Therapeutics is pioneering the creation of epi-therapeutic medicines with its powerful and precise epigenomic control platform, TEMPO. Unlike genome editing, TEMPO dials the volume of gene expression up or down towards healthy levels, without breaking or permanently rewriting DNA – and it can do this in multiple genes at once. This genetic tuning approach allows us to change cell fate and function at will, unlocking the ability to reverse pathways of cancer, genetic disease and aging. Our approach effectively expands the potential reach of genetic medicine – from targeting a limited range of rare conditions, to overcoming thousands of complex and chronic diseases that currently have no curative treatment.

One of the leading cancer centers in the nation, UNC Lineberger Comprehensive Cancer Center is located in Chapel Hill, North Carolina. UNC Lineberger is one of only 57 National Cancer Institute-designated Comprehensive Cancer Centers and was rated as exceptional - the highest category - following its most recent NCI review. The center brings together some of the most exceptional physicians and scientists in the country to focus on advancing the prevention, early detection and treatment of cancer. With research that spans the spectrum from the laboratory to the bedside to the community, UNC Lineberger faculty work to understand the causes of cancer at the genetic and environmental levels, to conduct groundbreaking laboratory research, and to translate findings into pioneering and innovative clinical trials.

Velocity is the world’s leading organization of fully integrated clinical research sites. With 90 sites and more than 220 investigators, Velocity partners with pharmaceutical and biotechnology companies to research new drugs, medical devices, diagnostics, and combination products that could improve human health and wellbeing. Velocity offers unified research site solutions to efficiently provide the right patients, investigators, and research staff for clinical trials across the U.S. and Europe. The company also operates a technology hub in India, where it is unlocking a new era in clinical research by developing innovative systems to leverage expansive site, patient, and historical performance data. To learn more about how Velocity delivers high-quality data, exemplary patient care, and unprecedented efficiency for clinical trials at any scale, visit VelocityClinical.com.

About Worldwide Clinical Trials  Worldwide Clinical Trials is a leading full-service global contract research organization (CRO) that works in partnership with biotechnology and pharmaceutical companies to create customized solutions that advance new treatments. Our capabilities include bioanalytical laboratory services, Phase I-IV clinical trials, and post-approval and real-world evidence studies – all powered by our team members who bring their expertise and a collaborative, personalized approach to each clinical program. Anchored in our company’s scientific heritage, our dedicated therapeutic focus on cardiovascular, metabolic, neuroscience, oncology, and rare diseases, is applied to develop flexible plans and solve problems quickly for our customers. Our talented team of 3,000+ professionals spans 60+ countries, and embraces a culture of diversity, equity, inclusion, and belonging (DEI&B). We are united in cause with our customers to improve the lives of patients through new, innovative therapies. For more information on Worldwide, visit www.Worldwide.com.