Cell therapy weekly: license granted for the manufacture and supply of synthetic DNA
This week: 4basebio (Cambridge, UK) received a license to offer clients its synthetic DNA platform, while a pre-clinical gene therapy company signed a global licensing agreement for an AAV gene therapy targeting Pompe disease. Plus, Lexeo Therapeutics (NY, USA) provided an update on its Friedreich’s ataxia (FA) gene therapy trials.
The news highlights:
- License granted for the manufacture and supply of synthetic DNA
- Global licensing agreement to develop Pompe disease gene editing therapy
- Positive interim results from Friedreich ataxia gene therapy trials
License granted for the manufacture and supply of synthetic DNA
4basebio PLC has received a Good Manufacturing Practice certification from the UK Medicine and Healthcare Regulatory Agency (London), authorizing the company to supply GMP-grade synthetic DNA as both a starting material and drug substance for clinical use in cell and gene therapies, as well as DNA- and mRNA-based vaccines.
4basebio’s COO, Amy Walker, commented on how 4basebio is leading the way in providing high-quality DNA products for use in clinical trials and commercialization:
“Our platform is faster, more efficient and offers higher performance products with a better safety profile compared with traditional plasmid DNA and other synthetic DNA products. We are able to offer our clients a rapid and scalable solution for their clinical trials and future product commercialization.”
Global licensing agreement to develop Pompe disease gene editing therapy
Pre-clinical gene therapy company GeneVentiv Therapeutics (NC, USA) has signed a global licensing agreement with Duke University (NC, USA) for GENV-002, the first universal gene editing therapy for both infantile-onset and late-onset Pompe disease. Affecting approximately 120,000 patients in developed countries, Pompe disease is caused by a deficiency of the enzyme acid-alpha-glucosidase, leading to excessive glycogen accumulation in striated muscle. This buildup results in respiratory failure, mobility issues, persistent muscle weakness and cardiac complications, significantly impacting daily life.
GENV-002 was developed by Dwight Koeberl, professor of pediatrics and medical genetics specialist at Duke University, in collaboration with leading experts in genetic and neuromuscular diseases. The therapy utilizes a strategy where the liver acts as a reservoir for the treatment. It uses an AAV to deliver both a healthy donor gene and a CRISPR-Cas9 editor, enabling precise genomic integration within the liver. GENV-002 can be administered to infants, offering a significant advantage over non-genome editing AAV competitors, which are more suitable for older patients.
“GENV-002 surpasses existing AAV gene therapies with the ability to universally treat infantile- and late-onset Pompe disease in infancy via gene editing,” said Paris Margaritis, CEO of GeneVentiv Therapeutics.
Positive interim results from Friedreich ataxia gene therapy trials
Lexeo Therapeutics reported positive interim results for LX2006, its investigational one-time AAV gene therapy for FA cardiomyopathy, based on data from the ongoing SUNRISE-FA and Weill Cornell Phase I trials. Both trials are 52-week, open-label studies using a one-time intravenous infusion of LX2006.
As of March 25, 2025, 16 participants have been treated across both studies, including six with enlarged hearts.
Key findings from the studies include:
- Participants with enlarged hearts experienced an average 25% reduction in heart size within 12 months or sooner
- Most participants demonstrated improved heart function and overall cardiac health
- Frataxin protein levels increased, with a 115% rise in the high-dose group
- Participants tolerated LX2006 well, with no signs of immune reactions or other safety concerns
These results support the continued development of LX2006 as a potential one-time treatment for FA cardiomyopathy.
Additionally, Lexeo plans to begin enrolling participants in a natural history study in Q2 2025, which will serve as an external control for the upcoming registrational study focused on frataxin protein levels and left ventricular mass index. The registrational study is expected to start in early 2026, with results expected in 2027.
