NCBiotech News

The U.S. Food and Drug Administration (FDA) has accepted Durham-based BioCryst Pharmaceuticals’ application to expand the use of ORLADEYO (berotralstat) to treat children ages 2 to 11 with a rare genetic condition known as hereditary angioedema (HAE). The FDA also granted the application Priority Review with a Prescription Drug User Fee Act date of September 12, 2025. HAE causes sudden and severe swelling in various parts of the body, including the face, hands, stomach and airways, which can be painful and sometimes life-threatening.

The recent Accelerate NC Career Celebration at the North Carolina Biotechnology Center brought together key players in the growing ecosystem that makes North Carolina a global leader in life sciences manufacturing. The third biannual event recognized 121 individuals who have successfully leveraged Accelerate NC initiatives to secure a life sciences career. Many of their peers, teachers, employers, family and friends also attended the celebration. Laura Rowley, vice president of life sciences economic development at NCBiotech, opened the ceremony by congratulating the newest members of the state’s life sciences community.

Following a regulatory setback last year, Satsuma Pharmaceuticals Inc. has won approval for its nasal powder migraine treatment device from the U.S. Food and Drug Administration. Satsuma Pharmaceuticals, a unit of a Japanese drugmaker with U.S. headquarters in Durham, said its treatment, called Atzumi, received its new drug application approval from the FDA. The approval covers Atzumi for treatment of acute migraine in adults both with and without aura, or sensory disturbances that sometimes accompany migraine. Atzumi is a device that includes Satsuma’s formulation of a standard, decades-old migraine treatment called dihydroergotamine (DHE).

Durham-based Atsena Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for ATSN-201, its gene therapy product for the treatment of X-linked retinoschisis (XLRS). XLRS is a rare inherited retinal disease with no approved treatments. ATSN-201 uses AAV. SPR, the company’s novel spreading capsid, delivers the gene therapy directly to the photoreceptors in the central retina, achieving therapeutic levels of gene expression while minimizing surgical risks, such as foveal detachment. ATSN-201 has previously received Fast Track, Rare Pediatric Disease and Orphan Drug Designations from the FDA.

When Damon Race, a Raleigh-based biopharmaceutical entrepreneur and investor, wanted to start a new company, he didn’t have to go far to find the founding technologies. After looking at “30 to 40 assets” nationally, in October of 2020 Race chose to in-license a gene therapy devised within a half-hour’s drive, at the University of North Carolina at Chapel Hill. In April 2025 he licensed a gene-editing therapy from Duke University, also a half-hour’s drive away, in Durham. Together, these gene therapy programs make up the product pipeline for GeneVentiv Therapeutics, a pre-clinical gene therapy company founded in 2020 in Raleigh. There was no need to look further or to move elsewhere, said Race, the company’s president and chief executive officer.

The U.S. Food and Drug Administration (FDA) has granted Incyclix Bio Fast Track designation for INX-315 to treat CCNE1-amplified platinum-resistant/refractory ovarian cancer. INX-315, the company’s lead compound, is being evaluated in an ongoing, first-human Phase 1/2 clinical trial. “The FDA’s decision to grant Fast Track designation for INX-315 reflects the best-in-class potential of our CDK2 inhibitor, the strength of our preclinical and early clinical data and the urgency to address significant unmet need in patients with CCNE1-amplified platinum-resistant/refractory ovarian cancer,” said Patrick Roberts, PharmD., Ph.D., chief executive officer and co-founder of Incyclix Bio.

URO-1, a Greensboro medical device company in prostate and soft tissue cancer diagnostics, has secured a new round of equity financing and announced additional milestones since the start of commercialization of its prostate cancer biopsy system in January 2025. GenHenn Capital, a single-family office investor in life sciences companies, led the new round of financing. Terms weren’t disclosed. The financing adds to a Series A round of $8 million announced in early 2024. In addition, URO-1 has made several recent advancements, including new study results, the development of a manufacturing cleanroom, and presentations at national urology and pathology conferences.

Genentech, considered one of the biotech industry’s founding companies, plans to build a $700 million, 700,000-square-foot manufacturing facility in Holly Springs. The company expects to employ 420 people at the new high-volume fill-finish site. Genentech CEO Ashley Magargee announced the investment at its Washington, D.C., headquarters. North Carolina Gov. Josh Stein was at the event for the announcement and praised the company’s investment, which is the first major life sciences announcement of his term. "World-class companies like Genentech recognize that North Carolina is a leading state for biotechnology,” said Governor Josh Stein. “These companies know that our life science workforce is ready to help them deliver their cutting-edge medicines to the world. We are proud to welcome Genentech to North Carolina.”

A statewide partnership to boost workforce training and career opportunities in North Carolina’s life sciences manufacturing industry has won an accolade from a national business publication. Business Facilities, a source of news and trends for site-selection decision-makers, recognized the Accelerate NC Coalition with a 2025 Economic Development Organization award. The Accelerate NC Coalition is a statewide partnership dedicated to strengthening North Carolina’s life sciences manufacturing workforce. It is led by the North Carolina Biotechnology Center with involvement by several industrial, academic and economic development partners.

SonoVascular, a Chapel Hill medical device startup focusing on treating potentially life-threatening blood clots, has earned the top life sciences award from the Angel Capital Association. The association honored SonoVascular with the 2025 Luis Villalobos Award in the life science category at the industry group’s recent conference in Denver. The group cited SonoVascular’s innovations in treating venous thromboembolism (VTE), or a blood clot that forms in a vein. “This award provides recognition of the importance of both the clinical need and the transformative nature of our technology to treat VTE,” Daniel Estay, SonoVascular’s founder and CEO.

Durham-based restor3d, a developer of three-dimensional printed implants for orthopedic use, has raised $38 million in venture capital.  The funding was raised from both existing and new private investors, including Summers Value Partners of Denver. “This funding allows us to further scale our technology, expand access to truly personalized care, and continue setting new standards in orthopedic treatment,” said Kurt Jacobus, chief executive officer and co-founder of restor3d. “We are redefining what’s possible in orthopedics to better serve our surgeons and patients.”

The North Carolina Life Sciences Apprenticeship Consortium (NCLSAC) was founded in 2022 as part of the Accelerate NC Life Sciences Manufacturing coalition, funded by the U.S. Economic Development Administration Build Back Better Regional Challenge (BBBRC). The coalition, led by NCBiotech, promotes life sciences training and job opportunities to distressed communities across North Carolina. The 12 current employer partners include Amgen, Biogen, CSL Seqirus, FUJIFILM Diosynth Biotechnologies, KBI Biopharma, Körber Pharma Software, Lilly, Merck, Novartis, Novonesis, Novo Nordisk, and Pfizer. The NCLSAC has leveraged BBBRC grant funds to award more than 254 BioWork scholarships at 9 community colleges statewide, enabling valuable pre-apprenticeship training. To date, seven of the NCLSAC member companies have hired 38 apprentices.

Phinite, Inc., a sustainable agtech company, has launched a new regional Sustainability Cluster in Sampson County, N.C., advancing its mission to produce high performance, regenerative fertilizers through circular economy principles and innovative drying technology. By transforming the agricultural byproduct hog manure into odorless, nutrient rich, granular fertilizer, Phinite enables farmers to improve soil health and crop yield while supporting long term environmental stewardship. The initiative helps address a pressing global challenge: reducing agriculture’s reliance on mined phosphorus, a non-renewable resource essential to crop production. Phinite’s Sampson County operations are creating a regional hub for advanced hog manure drying technology using advanced robotics. Hog farmers within the county and bordering regions gain an additional revenue stream through providing an agricultural byproduct.

There’s strength in numbers in the global pharmaceutical marketplace, so a small Durham pharmaceutical company is adding numbers to launch its novel prescription drug that won federal marketing approval last year. Pelthos Therapeutics, a Durham-based wholly owned subsidiary of Ligand Pharmaceuticals (Nasdaq: LGND), is combining with Channel Therapeutics (NYSE American: CHRO) to help with the launch of Pelthos’ drug ZELSUVMI. The deal, expected to close by the end of this summer, also includes $50 million in equity capital raised from a group of strategic investors led by Murchinson. Novan, a spinout from the University of North Carolina at Chapel Hill, developed Pelthos’ drug ZELSUVMI. Novan went bankrupt in 2023 before it could commercialize its proprietary nitric oxide-based technology. So Pelthos bought the rights to the drug and ushered it through the 2024 approval by the U.S. Food and Drug Administration (FDA).

Gene therapy continues to advance, with 26 gene therapies approved by the U.S. Food and Drug Administration (FDA) as of April 2025. Yet key challenges remain, especially in managing the level and duration of gene expression following treatment. Epigenos Biosciences Inc., a spinout from the University of North Carolina at Chapel Hill, is addressing this gap by harnessing epigenetics to develop therapies with greater precision and control. “We hope to change the way people do gene therapy in the future,” said Joe Ruiz, co-founder of Epigenos. “By offering a way to control gene expression after treatment, we can make therapies safer and more adaptable — ultimately improving outcomes for patients who need them most.”