List of Adeno-associated viral vector (AAV) Companies in North Carolina - 14

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.

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.

BioAgilytix is a biotechnology company that specializes in bioanalysis for the biopharmaceutical and life sciences industries.

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.

The Duke University Clinical Laboratoriesemploy a wide range of practices; from high-throughput, highly automated testing in their hematology, chemistry and immunoassay laboratories, to specialized mass spectrometry and molecular based testing in many of their subspecialty labs, to direct patient care bytransfusion services physicians. In total last year, their 850 clinical laboratorians deliveredover 10million resultsto support the care of the many hundreds of thousands of patients who came to Duke.In additional to supporting the clinical mission of DUHS, theyalso support the other two missions of theiracademic health care system: teaching and research.

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.

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.

LigaTrap has developeda patented portfolio of affinity ligands that can be used to purifymonoclonal and polyclonal immunoglobulins from various species.

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.

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.

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.

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.