Beam Therapeutics reports gene editing breakthrough
By Kyle Marshall | This article orginially appeared via NCBiotech's blog
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.
A story in The New York Times described the results as the first instance of restoring a mutated gene to normal. “This is the beginning of a new era of medicine,” Dr. Kiran Musunuru, a cardiologist and gene therapy researcher at the University of Pennsylvania Perelman School of Medicine, said in the story.
Biomanufacturing center in Durham
As Cambridge, Mass.-based Beam advances BEAM-302 toward commercialization, the company’s Durham facility is expected to play an increasingly large role.
Beam opened its 100,000-square-foot biomanufacturing center at 10 Davis Drive in 2022. The company chose the Triangle over locations in the Philadelphia area, Massachusetts and Maryland, citing Research Triangle Park as a thriving biopharmaceutical hub. Beam currently employs about 100 people in Durham and plans to expand over time.
A spokesman said the facility is designed to support a broad range of research and clinical-stage programs. Clinical-grade in vivo therapies for genetic diseases affecting the liver, including BEAM-302, are made in Durham, as is the company’s ex vivo cell therapy for sickle cell disease.
The center, built to comply with current good manufacturing practices (cGMP), is fully operational for research, development and manufacturing capabilities for precision genetic medicines.
Expansion of the early-stage trial
Based on the success of the BEAM-302 trial to date, Beam is planning further evaluation of the therapy at higher doses. The company said it would report results at a medical conference in the second half of 2025.
Beam also plans to expand the trial to include an additional cohort of AATD patients with mild to moderate liver disease, either with or without lung disease.
According to a news release from Beam, about 100,000 people in the United States have the genetic mutation that causes AATD.
“This landmark result in medicine represents the first clinical evidence of precise correction of a disease-causing mutation by rewriting the genetic code,” Beam CEO John Evans said in the news release. “We look forward to continuing dose escalation and accelerating the development of BEAM-302 for patients with AATD who urgently need more effective therapeutic options.”
