NCBiotech News

RTP-based Opus Genetics has announced several advances in its gene therapy programs designed to restore vision and help prevent blindness in patients with inherited retinal diseases.

R. Jude Samulski, Ph.D., was honored by the American Society of Gene + Cell Therapy (ASGCT) with its Founders Award at the organization’s annual meeting earlier this week in Boston.

It has been known for years by insiders as the “hidden gem” of North Carolina’s life sciences ecosystem. And now, after four decades of unique and valuable service to partners statewide, the North Carolina Biotechnology Center’s (NCBiotech’s) Life Sciences Intelligence (LSI) unit is now a “gem on display.” 

With roots in the research labs at North Carolina State University, ChromaGenix has opened a new $35 million, 6,500-square-foot research and development and manufacturing facility at NC State’s Centennial Campus.

A new Research Triangle company, Persistence Therapeutics, is developing a small, implantable device that generates and delivers genetically engineered T cells directly inside the body. These cells, called chimeric antigen receptor T cells or “CAR-T cells,” are currently administered as a personalized immunotherapy for certain blood cancers but require time-intensive and costly manufacturing.

AskBio Inc., an RTP-based gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has received U.S. Food and Drug Administration (FDA) acceptance of its Investigational New Drug (IND) application for AB-1009, an adeno-associated virus gene therapy in development for late-onset Pompe disease (LOPD).

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has received another pair of regulatory boosts. The FDA recently granted the company’s PBGENE-DMD therapy Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of Duchenne muscular dystrophy (DMD).

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.

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.”

Durham-based Precision BioSciences Inc. has received investigational new drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for PBGENE-HBV, an in vivo gene editing program designed to cure chronic hepatitis B virus (HBV) infection. It is the first investigational in vivo gene editing therapy for treating chronic hepatitis B to be cleared for clinical trials in the U.S. Over a million people in the U.S. have chronic hepatitis B, and while antiviral treatments can suppress the virus and reduce liver damage, they don’t fully eliminate the infection. PBGENE-HBV addresses this challenge by targeting the main source of HBV replication and deactivating integrated HBV DNA in liver cells. “IND clearance to expand the ELIMINATE-B trial for chronic hepatitis B is a first for the gene editing space,” said Michael Amoroso, president and CEO of Precision BioSciences.

The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham operation is poised to help build on that success. On March 10, Beam announced data and proof-of-concept results from an early-stage clinical trial of its potential treatment for a rare genetic disorder affecting the lungs and liver. Nine patients were involved in the trial of BEAM-302, intended to treat alpha-1 antitrypsin (AATD) deficiency. AATD can lead to severe lung and liver complications, including emphysema and cirrhosis. The trial showed that BEAM-302 corrected a disease-causing mutation at the DNA level. AATD patients with lung disease were given a single intravenous infusion of the therapy and reported no significant adverse effects.

Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics and headquartered in Switzerland, announced today the opening of its cutting-edge Center of Excellence for Biologics and Cell and Gene Therapy in the heart of Research Triangle Park (RTP), North Carolina. 

The new facility expands Solvias’ global network of centers and reinforces its commitment to advancing transformative therapies for cancer and rare diseases.  

Tune Therapeutics, an epigenome-editing company with origins at Duke University, has raised over $175 million in financing to advance a potential treatment for chronic hepatitis B and other diseases. The financing – among the largest ever for a North Carolina life science company – was led by New Enterprise Associates, Yosemite, Regeneron Ventures and Hevolution Foundation.

The first Clinical Outsourcing Group: CRO Summit was held in Raleigh on Dec. 3-4, 2024. The event brought together more than 200 attendees to explore opportunities for improving collaboration between sponsors and clinical research organizations (CROs).