North Carolina is home to Research Triangle Park, an R&D sanctuary situated between the cities of Raleigh and Durham, which just happens to be one of the biggest biotech hubs in the U.S., with a multitude of renowned companies. The southeastern state even rivals the likes of Boston, San Francisco and San Diego, all of which are well-known for their bustling biotech scenes.
There are more than 810 life science companies situated in North Carolina, with some of the biggest names in biotech having manufacturing sites there, including Swiss giant Novartis, who last year acquired a 170,000 square-foot space in Research Triangle Park to start producing Zolgensma, its spinal muscular atrophy therapy.
It helps that the North Carolina Biotechnology Centre also sits in Research Triangle Park, with its aim to transform the U.S. state into a global leader in life sciences by using innovation, education, commercialisation and business growth to help bring about technology-based economic development.
In this article, in alphabetical order, we have listed five of the best biotech companies based in North Carolina, who have either already developed, or are in the process of researching and developing, innovative therapies to tackle various different diseases and conditions.
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One such company based in North Carolina is Altis Biosystems, which has created next-generation in vitro cell-based culture models of the human intestinal epithelium for preclinical modeling of intestinal inflammation, toxicity, bioavailability, and barrier integrity. The stem cell platform is designed to provide companies with relevant preclinical insights and enhance the performance of clinical trials, while also diminishing the need for animal testing.
The company’s models replicate human biology using human donor stem cells to recreate different regions of the intestinal epithelium in vitro. And, by expanding these stem cells, the company is able to create a cryopreserved biobank that allows for multiple studies using the same donor.
According to Altis Biosystems, its models can also help to reduce downstream timelines and costs, and can provide the necessary results in as little as 2 weeks.
Founded in 1996, this biotech company based in North Carolina is committed to the very niche area of combating cisplatin-induced hearing loss in pediatric cancer patients. This can be permanent and severe in young children, and the company’s aim is ultimately to prevent children enduring further hardship while they undergo chemotherapy.
Cisplatin is a platinum-based chemotherapy drug used to treat cancer, and comes with the potential side effect of ototoxicity (hearing loss), damaging the sensory hair cells that trigger the nervous system to communicate with the auditory cortex of the brain.
Fennec’s prescription drug, PEDMARK, was finally approved by the U.S. Food and Drug Administration (FDA) in 2022, after more than ten years of study. It helps protect against cisplatin-induced hearing loss by attaching itself to the cisplatin molecules and reducing the amount of harmful substances that they release.
Dealing in the discovery and development of next-generation cancer therapies, G1 Therapeutics is a North Carolina commercial-stage biopharmaceutical company committed to improving the life of cancer patients.
In 2021, the FDA approved G1 Therapeutics’ drug, trilaciclib – sold under the brand name COSELA – which reduces chemotherapy-induced bone marrow suppression in adults receiving certain types of chemotherapy for extensive-stage small cell lung cancer (SCLC). Cosela may help to protect bone marrow cells from damage caused by chemotherapy by inhibiting cyclin-dependent kinase 4/6, which is a type of enzyme.
The company is also testing trilaciclib in clinical trials for breast cancer and bladder cancer, and recently reported positive preliminary results from its ongoing phase 2 trial of the drug for the treatment of unresectable locally advanced or metastatic triple-negative breast cancer (TNBC). The study found that people who received trilaciclib prior to PD-(L)1 inhibitor sacituzumab govitecan-hziy showed 50% reductions in the incidence of adverse events, such as neutropenia, anemia and diarrhea.
Based in North Carolina, Kriya Therapeutics has research and manufacturing teams located in Research Triangle Park, and concentrates on developing new gene therapies, with a broad pipeline of therapies for ophthalmology, neurology, and metabolic diseases.
The company’s ophthalmology pipeline is focused on addressing some of the most common causes of blindness worldwide, including geographic atrophy, noninfectious uveitis, and diabetic retinopathy.
Meanwhile, its neurology pipeline focuses on severe focal neurological diseases affecting a specific location within the nervous system, such as trigeminal neuralgia and epilepsy, and its metabolic pipeline addresses endocrine diseases of impaired glucose metabolism, including diabetes and glycogen storage diseases.
In one of the biggest funding rounds of July 2023, Kriya announced the addition of more than $150 million in capital as part of its series C financing, which brought the total round to more than $430 million. This financing will be used to support the clinical translation of the company’s gene therapies, as well as continued scaling of its engineering, computational and manufacturing platforms.
North Carolina biotech company Merakris Therapeutics, researches, develops and markets regenerative healthcare products to improve patient care globally, and is pioneering commercially scalable biotherapeutic technologies derived from stem cells that promote homeostasis in damaged tissue.
Two of the company’s current focus areas include: fractionated amniotic fluid, in which the company’s tissue engineering technology is used, isolating amniotic micro-vesicles and soluble amniotic fluid proteins to activate phenotypic changes in target cells; and co-cultured amniotic stem cell-conditioned media, with the aim of generating scalable and targeted manufacturing of amniotic proteins and micro-vesicles that may be used as a platform technology.
Merakris has also already developed Dermacyte Matrix, which provides a protective covering for cell attachment and ingrowth, as well as Opticyte Matrix, which promotes corneal healing by providing a barrier on the surface of the eye to protect it, and prevent scar tissue from occurring, and Opticyte Serum Tears to help support corneal epithelial defects.