List of Therapeutic Companies in North Carolina - 98

Adaptin Bio, is a pioneering biotechnology company dedicated to revolutionizing drug delivery. Our focus is on developing innovative technologies that enhance how medications and other therapeutic compounds reach their target destinations within the body. Our core mission is to overcome a major hurdle in medicine: ensuring effective delivery of drugs to specific tissues.

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.

Adroit Pharmaceutical Development was founded to efficiently identify new formulations and process technologies for our clients. We also work with clients to identify and correct problems with their existing formulations and processes.

Alcami is a pharmaceutical CDMO that offers contract development and manufacturing services for the healthcare and biotechnology industries.

The AlphaVax pipeline is targeting pandemic influenza and cytomegalovirus (CMV), as well as grant and collaborator-supported programs in HIV, prostate and breast cancer, and several biodefense vaccines. Clinical data from the first alphavaccine human trials have confirmed safety and immunological activity in man. These immune responses are characterized as robust and balanced antibody (B-cell) and cellular (T-cell) immune responses, confirming the promising data we have obtained in over a decade of intensive pre-clinical work with this vector platform.

Areteia Therapeutics is a clinical stage biotechnology company committed to putting respiratory patients in better control of their disease—and back in control of their lives. The company's purpose is to develop and deliver novel inflammation modulating therapies to improve the lives of patients with respiratory diseases. Areteia Therapeutics is dedicated to advancing the treatment of respiratory diseases, including asthma and eosinophilic asthma, through innovative research and development.

Based on an R&D program started in 2005, Orano Med has developed new processes for producing high-purity lead-212 (Pb-212), a rare radioactive isotope.  Lead-212 is currently at the heart of promising projects in nuclear medicine to develop new treatments against cancer.  The innovative approach, known as targeted alpha therapy (TAT), recognizes and destroys cancer cells while limiting the impact on nearby healthy cells. Macrocyclics, Inc., an Orano Med company, is the global leader in high performance chelating agent technology, offering a broad range of services and products.  For more information please visit www.macrocyclics.com.

At Arrivo, we strive to set a new standard in drug development that starts with the selection of drug candidates and runs throughout clinical development. Our approach and decision-making has delivered a history of excellent returns and product successes that makes us the trusted partner for industry and investors.

Ascletis is an innovative R&D driven biotech listed on Hong Kong Stock Exchange (1672.HK). Ascletis is committed to developing and commercializing innovative drugs in the areas of NASH, cancer lipid metabolism and oral checkpoint inhibitors, viral hepatitis and HIV/AIDS for unmet medical needs in China and globally. Led by a management team with deep expertise and a proven track record, Ascletis has developed into a fully integrated platform covering the entire value chain from discovery and development to manufacturing and commercialization. Gannex, a wholly-owned company of Ascletis, is fully dedicated to the R&D and commercialization of new drugs in the field of NASH. Gannex has three clinical stage drug candidates against three different targets – FASN, THR-beta and FXR, and three combination therapies.

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.

Auration Biotech is developing novel therapeutics for ear, nose and throat diseases. The company is based in San Mateo, California and has a focus on developing transformational platform of advanced therapies to cure blindness.

Avicenna Biosciences was founded to solve the intractable challenges that previously stopped drug candidates in their tracks. We have created an ML-driven medicinal chemistry platform that makes Lead Optimization faster, cheaper, and more successful – transforming sub-optimal development candidates into life-saving drugs. The platform has seen rapid success in discovering the first orally available, CNS-penetrant ROCK inhibitors for the treatment of neurodegenerative diseases, with IND-enabling studies initiated just one year since the program kicked off.

Bantam Pharmaceutical is a drug discovery company harnessing the power of mitochondrial dynamics to address unmet needs in oncology. Using its unique expertise in mitochondrial dynamics, Bantam is developing novel, first-in-class small molecule oral therapeutics for difficult-to-treat hematological and solid tumors. Bantam is currently pursuing an IND application for Its lead candidate, BTM-3566, in B-cell malignancies. About BTM-3566 BTM-3566 is an orally-available novel small molecule compound with broad anti-cancer activity in hematologic and solid tumors, initially focused on Diffuse Large B-cell Lymphomas (DLBCL). BTM-3566’s anti-cancer mechanism of action is unique from any other therapeutic, disrupting mitochondrial function in tumor cells to induce apoptosis (cell death). An IND application for BTM-3566 in B-cell malignancies is currently underway and will be completed by Q1 2022.

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.

Belhaven Biopharma is developing a portfolio of dry powder nasal medicines to provide convenient, safe, and rapid treatments in emergency situations

BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema (HAE) and other rare diseases. We leverage our expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. We are a commercial-stage biotech company with US headquarters in Durham, North Carolina. Our European headquarters is in Dublin, Ireland, and our Discovery Center of Excellence is rooted in Birmingham, Alabama, with many other regional offices around the world. As world leaders in structure-guided drug design, we draw on the strengths and expertise of all our colleagues—from those of us in the field or behind the scenes in our labs, to others based in home offices and around the globe. To view our LinkedIn community guidelines, please visit bit.ly/BCRXGuidelines.

BioCytics via the Human Applications Lab is focused on bringing personalized oncology treatments to the market. BioCytics has an ongoing IRB-approved clinical trial (BioCytics 0001; NCT00571389) that allows for the collection and study of blood and tissue samples from consenting cancer patients. We are incubated within Carolina BioOncology - a preferred cancer treatment and Phase I drug testing facility. BioCytics was founded by Dr. John Powderly, MD, medical board certified oncologist and a certified physician investigator (CPI), who is also president of Carolina BioOncology Institute (CBOI).

Brii Biosciences is a biotechnology company developing therapies to address major public health challenges where patients experience high unmet medical needs, limited choice and significant social stigmas. With a focus on infectious and central nervous system diseases, we have built a robust pipeline of potential treatment options based on patient insights and experiences, and are advancing these differentiated investigational therapies to address patient choice. Led by a visionary and experienced leadership team, Brii Bio has deep scientific expertise and a proven ability to progress therapeutic assets from discovery to commercial approval on a consolidated timeline. Established in 2018, Brii Bio now has operations in key biotech hubs, including Raleigh-Durham, the San Francisco Bay Area, Beijing and Shanghai. On July 13, 2021, the Company was officially listed on the Hong Kong Stock Exchange under the stock code 2137.HK.

Bryn Pharma is a privately held pharmaceutical company based in North Carolina. Primary focus is on the development and commercialization of innovative allergy products for the management of anaphylaxis. The mission of Bryn Pharma is to empower patients with a life-saving device that is small enough to comfortably fit in your pocket and easy to use in the event of an anaphylactic attack. Our intranasal delivery system will be an exciting alternative to epinephrine auto-injectors providing patients a needle-free option that is easy to use, portable, and affordable.

Caeregen Therapeutics discovers and develops novel therapies which repair, restore, and protect damaged and diseased tissues by reactivating the pathways and signaling which accompany normal cellular and tissue development. Our revolutionary regenerative medicine approaches have the potential to restore lost or degraded functioning to diseased tissue and organs.

Combination Therapy Pill - Cardiovascular

At Chimerix, we are on a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. With a focus in oncology, we are devoted to filling gaps in the treatment paradigm.

Citranvi Biosciences is a pre-clinical stage company engaged in the design and development of novel systems and tools for immunogenicity enhancement applicable to Vaccines Research and Development. We are developing innovative and proprietary vaccine technology platforms and products, using comprehensive structure-based vaccine design, broadly applicable to a variety of preventable disease targets, where there is an unmet global medical need.

CivaTech Oncology® designs innovative products that bring meaningful improvements to low dose rate brachytherapy. The CivaString® and CivaSheet™ can substantially reduce procedure time, provide homogeneous dose distribution and minimize trauma. Customizable CivaTech products ensure that radiation oncologists can deliver individualized, targeted therapy to treat cancer. http://www.civatechoncology.com/

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.

Clarus Biologics was launched to increase the availability of safe and effective prophylactic and therapeutic technologies. It’s lead product is a next-generation vaccine Virus Like Particle intended to enhance the effectiveness, efficiency and availability of vaccines.Clarus Biologics is Dedicated to the Discovery and Development of Novel Vaccine and Immunology Technologies

Cloud Pharmaceuticals is a pharmaceutical company that specializes in in silico therapeutics, revolutionizing drug discovery and development processes.

Cognosci is a healthcare company that focuses on improving patient care and outcomes through medical technology and health information.

Collaborations Pharmaceuticals, Inc. works on drug discovery for rare and neglected diseases. We develop and license our AI software as well as provide fee for services for pharmaceutical and consumer product services. To date we have won with collaborators $23.5M in grants since 2016.

Concentrx Pharmaceuticals, Inc. Management Team; Gilbert S. Mott, Jr.; Eric C. Richardson; W. James Alexander

Dignify Therapeutics is a drug development company focused on delivering novel drug therapies for bladder and bowel disorders in spinal cord injured individuals.

Domus Diagnostics is working to create infectious disease diagnostic tests that are affordable, user-friendly, accurate, and rapid enough to address the needs raised by current and future public health crises.

Eldec Pharmaceuticals Inc. is a preclinical biotech company focused on developing novel peptide therapeutics to treat inflammatory and autoimmune diseases. The company was spun out of the University of North Carolina at Chapel Hill (UNC-CH) in 2017 and is currently developing peptide-based therapies to treat lung infections and lung inflammation.

Emergo Therapeutics: Bringing the Immune Response back into Balance

Evecxia is the first company dedicated to realizing the therapeutic potential of amplifying serotonin synthesis to treat brain disorders. Serotonin synthesis amplification is distinct from targeting serotonin transporters (e.g., SSRIs) and receptors (e.g., psilocybin). Evecxia deploys 5-HTP, the natural serotonin precursor, delivered via proprietary drug delivery technologies to achieve sustained serotonin synthesis amplification. Evecxia has two Phase 2 clinical-stage drug candidates in development. EVX-101 is being developed as an adjunctive treatment for depression when first-line SSRI/SNRI antidepressants alone are inadequate. EVX-301 is being developed as a rescue therapy for patients in acute suicidal ideation crisis. Evecxia holds a comprehensive portfolio of issued and pending patents on the 5-HTP sustained-release/serotonin amplification method, doses, formulations, and related technologies.

Exela Pharma Sciences LLC is a specialty pharmaceutical company based in Lenoir, North Carolina, founded in 2005. The company develops, manufactures, and markets both generic and proprietary sterile injectable and ophthalmic products. Exela aims to improve patient outcomes and reduce healthcare costs through its innovative offerings. With a workforce of 501–1,000 employees, Exela specializes in products that face high barriers to market entry, utilizing regulatory pathways like Abbreviated New Drug Approvals (ANDA) and 505(b)(2) submissions. The company focuses on delivering high-quality, affordable products that enhance the experiences of healthcare providers and patients, emphasizing ease of use and clinical effectiveness. Exela's product range includes sterile injectables designed for improved safety and faster preparation, as well as sterile ophthalmic solutions that meet strict regulatory standards. The company operates a vertically integrated model, managing development, manufacturing, and commercialization in-house, which supports its commitment to quality control and innovation in the pharmaceutical industry.

Fennec Pharmaceuticals is a biotechnology company focused on improving the lives of children with cancer by addressing chemotherapy-induced hearing loss.

Cisplatin and other platinum compounds are essential chemotherapeutic components for many pediatric malignancies. Unfortunately, platinum-based therapies can cause ototoxicity in many patients, leaving many survivors of childhood cancer with permanent and irreversible hearing loss. Permanent hearing loss, even minimal, acquired at a young age has a devastating impact on development, education, and socialization and a lifelong negative impact on Quality of Life. There are no currently approved treatments for the prevention of platinum-induced hearing loss. At Fennec Pharmaceuticals, we are dedicated to the development of our investigational agent, PEDMARK™, for the prevention of ototoxicity in children, and we hope that this will enable more children to retain their hearing. In honor of our namesake, the fennec fox, we strive to embody adaptability and resourcefulness in everything we do. Please see www.fennecpharma.com for more information.

Focal Medical, Inc. is a privately held, biopharmaceutical company developing novel therapeutic products based on its innovative and patent protected targeted therapeutic system. The Company's lead product is a targeted therapeutic product delivering gemcitabine (an FDA approved chemotherapeutic) actively and directly to the pancreas by non-circulatory pathways to treat pancreatic cancer.

G1 Therapeutics, Inc., is a clinical-stage biopharmaceutical company focused on the discovery, development and delivery of innovative therapies that improve the lives of those affected by cancer.

GeneCentric was founded based on intellectual property from Drs. Neil Hayes and Chuck Perou's laboratories at the Lineberger Comprehensive Cancer Center at the UNC School of Medicine. GeneCentric currently has two unique platform technologies: The Lung Subtype Platform (LSP™) stratifies lung cancer patients into subtypes, which are important for appropriate therapeutic selection. The first application of LSP™ was licensed to and independently developed by Laboratory Corporation of America® Holdings ("LabCorp") and is now available through its specialty testing business, Integrated Oncology, as HistoPlusSM: Lung Cancer. GeneCentric's second platform technology is the Hypoxia Signature which has the potential to identify patients that respond to anti-angiogenesis therapies. GeneCentric utilizes a unique partnership model to translate important cancer discoveries into clinically adopted diagnostics for pathologists, clinicians, and, most importantly, patients. By partnering with the foremost clinical laboratories, in-vitro diagnostic (IVD), pharmaceutical, and biotech companies, GeneCentric can focus on its core strength, exceptional science and clinical studies, building evidence for the utility of its unique assays.

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.

Grid Therapeutics is a biotech company based on the innovative science first developed by Edward F. Patz, Jr. MD and his team of scientists at Duke University Medical Center. Located in Durham, North Carolina, Grid is developing the first human derived targeted immunotherapy for cancer. The antibody was discovered in exceptional outcome early stage lung cancer patients who did not progress to develop metastasis. The antibody was isolated from patient’s B-cells using state of the art molecular genomic techniques. “GT103”, the company’s lead asset, is currently enrolling the first-in-man Phase 1 clinical trial in refractory NSCLC patients.

Humacyte, Inc., is developing a disruptive biotechnology platform to deliver universally implantable bioengineered human tissues and organs designed to improve the lives of patients and transform the practice of medicine. The Company develops and manufactures acellular tissues to treat a wide range of diseases, injuries and chronic conditions. Humacyte’s initial opportunity, a portfolio of human acellular vessels (HAVs), is currently in late-stage clinical trials targeting multiple vascular applications, including vascular trauma repair, arteriovenous access for hemodialysis, and peripheral arterial disease. Pre-clinical development is also underway in coronary artery bypass grafts, pediatric heart surgery, treatment of type 1 diabetes, and multiple novel cell and tissue applications. Humacyte’s HAVs were the first product to receive the FDA’s Regenerative Medicine Advanced Therapy (RMAT) expedited review designation and received priority designation for the treatment of vascular trauma by the U.S. Secretary of Defense.

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.

Inhalon Biopharma is developing an inhaled immunotherapy platform for treating acute respiratory infections (ARI), such as syncytial virus (RSV), influenza, metapneumovirus (MPV), parainfluenza virus (PIV), adenovirus, severe acute respiratory syndrome (SARS) and middle east respiratory syndrome (MERS). Today there are no approved treatments for a wide range of ARIs that can lead to hospitalization, long-term health complications and sometimes death.

Innovate is a publicly traded (Nasdaq: INNT) clinical stage biotechnology company focused on developing novel medicines for autoimmune and inflammatory diseases with unmet needs. Their pipeline includes drug candidates for celiac disease, NASH, Crohn's, and ulcerative colitis.

InSoma Bio is a company that borrows nature's own designs to develop unique products for reconstructive plastic surgery. Their product enables surgeons to use a person's own tissues to enhance the stability of autologous fat grafts and solve problems in tissue engineering and regenerative medicine.

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.

Jubilant Pharma Limited is a global integrated pharmaceutical company offering a wide range of products and services to our customers across geographies. We organize our business into two segments, namely, Specialty Pharmaceuticals, comprising Radiopharmaceuticals (including Radiopharmacies), Contract Manufacturing of Sterile Injectables and Non-sterile Products and Allergy Therapy Products, and Generics & APIs, comprising Solid Dosage Formulations and Active Pharmaceutical Ingredients.

KeraNetics is the global leader in keratin science, purified, human-derived keratin manufacture, research and development for healthier wound healing KeraNetics provides physicians with new options to help patients by exploiting the properties of purified, human-derived keratin. KeraStat® Cream: Better management of radiation dermatitis (RD). KeraStat® Cream harnesses the power of keratin proteins to support the management of dry, light, and moderately exuding partial thickness skin wounds such as radiation dermatitis resulting from a fractionated radiotherapy regimen. FDA cleared and available in 1 oz. and 5 mL tubes. Rx only.

Kinvard Bio is committed to developing a durable new class of antibiotics to treat the most challenging acute and chronic drug-resistant bacterial infections. Our chemistry platform technology is based on groundbreaking research that has been exclusively licensed from the Myers lab in the Department of Chemistry and Chemical Biology at Harvard University and funded by the Kineticos AMR Accelerator Fund I (KAMRA I). The oxepanoprolinamides (OPPs) represent a distinct class of lincosamide antibiotics that are pre-organized for highly effective and differentiated binding to the bacterial ribosome. In preclinical studies, OPPs have proven effective against both Gram-positive and Gram-negative bacterial pathogens associated with clinical needs that remain unmet.

Kuria Therapeutics is a biopharmaceutical company that develops novel Nrf2 activators to treat blinding diseases.

Levee Medical, Inc. designs, develops, and manufactures innovative urological devices with the focus of improving recovery outcomes post-prostatectomy so that patients can quickly and confidently return to normal activity. The company's inaugural product is designed to support and stabilize the post-operative bladder neck and urethra following prostatectomy to accelerate the return of continence and reduce the overall risk of chronic incontinence.

Life Edit, an ElevateBio company, is a next-generation genome editing company that has built a highly innovative platform with one of the world's largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors. The platform allows Life Edit to target any genomic sequence and develop novel human therapeutics for the most challenging genetic diseases by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, Life Edit Therapeutics will continue to strengthen its platform of genome-editing enzymes, provide gene-editing expertise to strategic partners, and form other third-party partnerships to discover and develop new therapies.

Lindy Biosciences is a development-stage protein therapeutic formulations company. Their core technology, Microglassification™, produces spherical, dense, stable particles of a therapeutic protein. These protein particles are ideal for long-term storage, or for incorporation into drug delivery formulations such as suspensions(for high-concentration delivery of antibodies), encapsulation (for sustained/controlled release), or dry powder pulmonary delivery.​

Liquidia Technologies is a biopharmaceutical company transforming the development of new therapies by precisely engineering drug particles. Our proprietary PRINT technology is a simple, elegant solution that solves common problems with drug delivery and efficacy. PRINT technology is a scalable cGMP compliant process that creates particles and can apply to virtually any therapeutic area, molecule or route of administration. Liquidia is bringing more efficient, controlled development capabilities to the industry while enhancing the safety and quality of products for patients. Liquidia is advancing product candidates from its own pipeline. These initial product candidates, LIQ861 and LIQ865, apply the PRINT technology to better drug delivery in inhaled and pain therapeutic areas, respectively. The PRINT technology is also licensed with world-leading pharmaceutical companies that are developing future product candidates.

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.

Madera is the leader in highly potent and specific activators of the mitochondrial protease, ClpP. Together with our academic collaborators and the NIH/NCI, we have established the in vitro and in vivo performance of these agents. Notably, our agents provide a new mechanism of action that is effective on a number of drug-resistant cancer cells and patient-derived tissue samples. This technology is backed by the broadest patent portfolio in the area.

Marius Pharmaceuticals strives to better the lives of men and women by focusing on therapies designed for hypogonadism or Testosterone Deficiency. Our vision is to holistically improve metabolic health and mitigate significant unnecessary costs to the global healthcare system.

First in-class platform for harnessing the synergy between antibodies and mucus to reinforce the mucus barrier for mucosal health Proprietary intravaginal ring technologies for sustained delivery of small molecules to biomacromolecules Unparalleled safety from topical delivery

Nelum is a clinical stage biopharmaceutical company developing first in class and best in class patented molecules. Our area of focus is different forms of cancer. Our first drug, NLM-001, is a best in class small molecule hedgehog inhibitor currently in a phase Ib-IIa clinical trial in pancreatic cancer, after showing a safe profile in the Phase I trial.

Novan, Inc., a clinical development-stage biotechnology company, provides nitric oxide-based therapies to treat dermatological and oncovirus-mediated diseases. Its clinical stage dermatology drug candidates include SB204, a topical monotherapy for the treatment of acne vulgaris; SB206, a topical anti-viral gel for the treatment of viral skin infections; SB208, a topical broad-spectrum anti-fungal gel for the treatment of fungal infections of the skin and nails, including athlete’s foot and fungal nail infections; and SB414, a topical cream-based gel product candidate for the treatment of inflammatory skin diseases. The company also develops SB207, an anti-viral product candidate for the treatment of external genital warts; WH602, a nitric oxide-containing intravaginal gel to treat high-risk human papilloma virus (HPV); WH504, a non-gel formulation product candidate to treat high-risk HPV; and SB019 for the treatment of SARS-CoV-2. Novan, Inc. has a license agreement with Sato Pharmaceutical Co., Ltd.; and a strategic alliance with Orion Corporation. The company was incorporated in 2006 and is headquartered in Morrisville, North Carolina.

Founded by a team of accomplished industry professionals who began working together in 2000, NovaQuest Capital Management is a premier biopharma and life sciences investment firm. NovaQuest pioneered a Product Finance solution for the industry, providing at-risk, nondilutive funding that enables partner companies to advance pivotal clinical trials, launch new brands, license products, and acquire accretive products or companies. NovaQuest has invested in scores of biopharmaceutical assets across therapeutic areas with a clinical success rate significantly higher than the industry average. NovaQuest is actively evaluating and investing into global opportunities with financing needs that range from $30-100 million. For more information, please visit www.novaquest.com

Small Molecules

touchOPHTHALMOLOGY are a leading provider of learning resources, congress highlights and CME accredited activities for ophthalmologists.

OncoTAb, Inc., a North Carolina based biotechnology company, was founded by Mayo Clinic alumna Dr. Pinku Mukherjee, with a mission to improve the quality of cancer care by addressing unmet cancer diagnostic and therapeutic needs. The company has just launched its first product: Agkura™ Personal Score – a simple, non-invasive blood test that aids breast cancer detection in women with dense breast tissue. Roughly half of the women in the US have dense breast tissue and mammograms miss 50% of the breast cancers in these women, resulting in late stage diagnosis and mortality. Agkura™ Personal Score measures the biomarker tumor-associated MUC1 (tMUC1) and shows a trend of increasing value from benign disease through breast cancer stages 1 to 4. Women can monitor their personal score in conjunction with annual mammograms. A score above the normal range or an increase greater than normal variation will trigger a recommendation for diagnostic imaging (ultrasound or MRI), resulting in earlier stage diagnosis of breast cancer. The company is also working on developing treatments for breast and pancreatic cancers.

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

Parion Sciences is a development stage company dedicated to research, development, and commercialization of treatments to restore patient's innate mucosal surface defenses. In the eye and in the lung, our layer of protective mucus plays an important role in keeping our bodies healthy. Sometimes though, either through genetics, aging or environmental stimulus, our innate mucosal defense system can become compromised. Parion's science driven technologies target respiratory and ocular diseases in which the patient's ability to protect their mucosal surfaces is compromised.

Pathalys Pharma is a private, late-stage clinical biopharmaceutical company committed to the development of advanced therapeutics that address unmet needs in the management of late-stage chronic kidney disease (CKD). Pathalys was created in partnership with Catalys Pacific and DaVita to become a multi-asset pharmaceutical company focused on the needs of patients with end-stage renal disease

We are dedicated to creating innovative solutions for the most complex medical challenges, utilizing our unique approach to regenerative medicine. Protego-PD™ is a biologic with anti-inflammatory and immunomodulatory capabilities that work together to orchestrate a complex cellular response to restore and repair organs damaged by inflammation-driven diseases.

Our goal is to replace cigarettes with our reduced-risk nicotine platform.

Polarean is a revenue-generating medical imaging technology company revolutionizing pulmonary medicine through direct visualization of lung function by introducing the power and safety of MRI to the respiratory healthcare community. This community is in desperate need of modern solutions to accurately assess lung function. The Company strives to optimize lung health and prevent avoidable loss by illuminating hidden disease, addressing the global unmet medical needs of more than 500 million patients worldwide suffering from chronic respiratory disease. Polarean is a leader in the field of hyperpolarization science and has successfully developed the first and only hyperpolarized Xenon MRI inhaled contrast agent, XENOVIEW™, which is now FDA-approved in the United States. Polarean is dedicated to researching, developing, and commercializing innovative imaging solutions with its noninvasive and radiation-free pulmonary functional MRI platform. This comprehensive drug-device platform encompasses the proprietary Xenon gas blend, gas hyperpolarization system, as well as software and accessories, facilitating fully integrated modern respiratory imaging operations. Founded in 2012, with offices in Durham, NC, and London, United Kingdom, Polarean is committed to increasing global awareness of and broad access to its XENOVIEW MRI technology platform. For the latest news and information about Polarean, please visit www.polarean.com.

Precise Bio develops and commercializes 4D bio-fabricated functional tissues and organs that can save lives and improve its quality. We combine world-class tissue-engineering and transplantation experience with best-in-class bio-printing, to create a scalable bio-fabrication platform, that can overcome current industry challenges and make complex human organs available for transplantation.

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.

Priovant Therapeutics is a clinical-stage biotechnology company focused on delivering novel therapies for autoimmune diseases with the greatest morbidity and mortality. We are developing oral brepocitinib, a potential first-in-class dual inhibitor of TYK2 and JAK1, across multiple severe autoimmune indications, unlocking new treatment options for patients who are underserved by existing therapies.

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.

RedHill Biopharma Ltd. (NASDAQ: RDHL) is a specialty biopharmaceutical company primarily focused on gastrointestinal diseases and infectious diseases. RedHill’s U.S. commercial operations, headquartered in Raleigh, NC, include a gastrointestinal-focused sales force promoting several specialty gastrointestinal products in the U.S. RedHill’s pipeline includes several drug candidates in advanced clinical development stages addressing clear medical needs and aiming to relieve suffering and save lives. RedHill’s drugs are largely de-risked and are potentially of lower cost and faster to market than new chemical entities under development. RedHill’s products are based on several technology platforms and address several disease targets, thus providing for risk diversification. RedHill’s experienced management team, board of directors and advisory board, based in Israel, the U.S., Canada and Europe, comes with a successful track record of bringing patented drugs to the market, as well as extensive managerial, financial, and transactional expertise.

restor3d enables surgeons to improve the reconstruction and repair of the human body through 3D printed implants with enhanced anatomical fit and superior integrative properties. Leveraging expertise and experience in 3D printing of key biomedical materials spanning a wide range of properties, restor3d seeks to improve medical device solutions.

We know the HPA-T Axis Sanesco Health International is a biomedical company providing licensed healthcare practitioners with innovative, evidence-based and clinically-effective solutions. Assessing, monitoring and correcting neurotransmitter and hormone imbalances is not just what we do, it's ALL we do. We believe in identifying the underlying root causes behind symptoms and conditions affecting HPA-T Axis function. We offer a specialized, clinical model to help health care practitioners see each patient holistically on a neuroendocrine level, and to guide their patients on a healthier path. Sanesco's Communication System Management clinical model includes: -non-invasive functional laboratory testing -personalized patient-centered reporting -all-natural nutritional formulas Our team's clinical expertise, on-staff clinician support, complimentary interpretations, education and hands-on training mean better results for practitioners and their patients. The reason why we choose to specialize in optimizing HPA-T Axis function is because of the pivotal role it plays in the body's Communication System as a whole. Upstream biochemical imbalances found in the critical messengers of this axis can contribute to the manifestation of cognitive function issues, mood disorders and other symptoms and conditions such as: depression, anxiety, insomnia or sleep concerns, Alzheimer's, dementia and memory loss, impairments of focus and attention, PTSD, weight issues, metabolic syndrome, blood sugar regulation, diabetes, chronic pain, fibromyalgia, chronic fatigue, sexual health, menopause, autism, ADD/ADHD, and addictions. Optimizing function of the HPA-T Axis has a positive effect on the rest of the neuroendocrine system and balancing the body's Communication System as a whole. This creates our clinical model which is key to addressing a plethora of symptoms and conditions, and optimizing health and overall quality of life. Privacy Policy https://sanescohealth.com/terms-privacy/

Sapere Bio is a longevity technology company. SapereX, a blood-based measure of cellular senescence and immune function, captures fundamental mechanisms of aging. SapereX allows clinicians and scientists to see early aging and monitor how interventions aimed to increase healthspan are working in individual patients. We are changing the way aging is measured and imagined, for longer healthspan and better lives. Sapere Bio was co-founded by Dr. Norman Sharpless, former Director of the NCI, and Dr Natalia Mitin, current CEO of Sapere Bio.

Our mission is to provide the next generation of sound medicine through proven ablative technologies that precisely deliver care while sparing healthy tissue. This next generation of care involves expanding our current HIFU technologies to treat a range of urological indications that balance treatment effectiveness and quality of life.

Spyryx Biosciences is a biopharmaceutical company developing inhaled therapeutics for severe respiratory diseases, with Cystic Fibrosis (CF) as the first target indication. Our development efforts are based on novel peptides that reduce the airway surface density of epithelial sodium channels (ENaC) via ligand-mediated internalization. This mechanism has a robust and durable effect on enhancing airway and mucus hydration to promote mucociliary clearance. We believe the mechanism of our investigational peptides has the potential to provide disease-modification for the dehydration associated lung disease that is a feature of CF, as well as having utility in multiple other pulmonary and non-pulmonary indications. Spyryx's lead program, SPX-101, has successfully completed a Phase 1 safety study in healthy volunteers and is now being tested in a multinational Phase 2 study in individuals living with CF. The Phase 2, entitled "Hydration for Optimal Pulmonary Effectiveness"​ or HOPE-1, is a 28-day, placebo-controlled study intended to establish treatment proof of concept independent of CFTR mutation (www.clinicaltrials.gov/NCT03229252). This study has initiated recruitment in select sites in Canada and Western Europe. Further information regarding Spyryx Biosciences is available at www.spyryxbio.com.

T3D Therapeutics’ mission is to develop its lead drug product candidate, T3D-959, as a potential ‘First-in-Class’ and ‘Best-in-Class’ disease-modifying, breakthrough medicine for the treatment of Alzheimer’s disease with the potential to slow, stop or reverse the course of the disease. The Company is also seeking to maximize the therapeutic utility of T3D-959 and has initiated pre-clinical studies in Huntington’s Disease models.

Tenax Therapeutics is a Phase 3, development-stage pharmaceutical company focused on identifying and developing therapeutics that address cardio-pulmonary diseases with high unmet medical need.

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.

TregTherapeutics Inc. is a preclinical stage company focused on the treatment and cure of multiple sclerosis. The objective of the TregTherapeutics tolerogenic vaccine is to reverse the inflammatory T cell immune attack in demyelinating lesions of an MS patient, and subsequently, to create an anti-inflammatory tolerogenic response within that patient.

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.

Monoclonal Antibody

Vast is a preclinical airway immunology company focused on breaking the vicious cycle of infection and chronic inflammation in respiratory diseases. The Vast technology platform begins with the unmatched antimicrobial activity of nitric oxide in the form of stable prodrugs. When our new chemical entities are combined with inhalation formulation expertise and proprietary chemistry for delivering drug candidates to the lungs, the result is aerosolized therapies targeting the specific needs of individual patient populations. Nitric oxide is an endogenous molecule that plays a vital role in the natural immune system response to microbial pathogens. Taking cues from human physiology, our nitric oxide based “antibiotic alternative” compounds are not susceptible to antimicrobial resistance mechanisms common to traditional antibiotics.

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.

Veloxis Pharmaceuticals, Inc., an Asahi Kasei company, is a fully integrated specialty pharmaceutical company committed to improving the lives of transplant patients. Headquartered in Cary, North Carolina, USA, Veloxis is focused on the global development and commercialization of medications utilized by transplant patients and by patients with serious related diseases. We know what transplant means to patients, and we’re leading innovation to improve their experience. For patients, transplant is a life-changing procedure. But changes come with challenges. At Veloxis, our mission is to see and respond to the challenges along the transplant journey.

Villaris Therapeutics is a preclinical-stage biopharmaceutical company focused on developing novel antibody therapeutics for the treatment of vitiligo. A recent discovery revealed that an immune pathway, initiated by IL-15 signaling and its formation of autoimmune memory T cells (TRM), was responsible for the return of vitiligo usually soon after stopping treatments. Targeting this pathway therapeutically was very effective at reversing the disease in a mouse model of vitiligo with long-lasting results. Villaris Therapeutics is developing a novel humanized anti-IL-15R antibody to target depletion of TRM for the effective and durable treatment of vitiligo and other tissue-specific autoimmune diseases that also involve these cells.

vTv Therapeutics is a clinical stage biopharmaceutical company focused on developing oral, small molecule drug candidates. vTv has a pipeline of clinical drug candidates led by cadisegliatin, a potential adjunctive therapy to insulin for the treatment of type 1 diabetes. vTv and its development partners are pursuing additional indications, including type 2 diabetes and other chronic conditions.

Developing next generation technologies for precision medicine and drug discovery.

ZY Therapeutics is a biotechnology company that develops novel therapeutics for oncology treatments.