This week: Could OTL-102 be used to treat X-CGD, and LakePharma opens new dedicated Vector Center to support gene and cell therapy.
The news highlights:
Commercial gene therapy company Spark Therapeutics (PA, USA) has announced that they have entered into a definitive merge agreement wherein they will be fully acquired by Roche (Basel, Switzerland). Under the terms of the merge agreement, Roche will pay approximately US$4.8 billion for Spark, which will continue to operate as an independent company within Roche Group.
“As the only biotechnology company that has successfully commercialized a gene therapy for a genetic disease in the USA, we have built unmatched competencies in the discovery, development and delivery of genetic medicines. But the needs of patients and families living with genetic diseases are immediate and vast,” commented Spark Therapeutics CEO, Jeffrey Marrazzo. “With its worldwide reach and extensive resources, Roche will help us accelerate the development of more gene therapies for more patients for more diseases and further expedite our vision of a world where no life is limited by genetic disease.”
Orchard Therapeutics (London, UK) has presented clinical proof-of-concept data for OTL-102 as a treatment for X-CGD, a rare, inherited immunodeficiency disorder caused by a genetic mutation that results in the inability of neutrophils to effectively kill bacterial and fungal infections. The safety and efficacy of OTL-102, which utilizes a self-inactivating lentiviral vector (G1XCGD), was assessed in seven patients aged 2—27 years with X-CGD. Results found that six of the patients continued to show sustained levels of functioning neutrophils after 12 months and no longer receive treatment with CGD-related prophylactic antibiotics.
“This proof-of-concept data set for OTL-102 demonstrates efficacy across multiple markers of clinical benefit in 6 of 7 evaluable patients treated for X-CGD at 12 months,” explained Donald Kohn, the University of California (LA, USA). “Of note, patients have shown sustained levels of functioning neutrophils associated with clinical benefit, freedom from infections and resolution of chronic inflammation. We look forward to continuing the development of this potentially transformative gene therapy for patients with X-CGD.”
Biologics contract research, development and manufacturing organization LakePharma (CA, USA) has announced that its Vector Center is now commercially operational. The new center, located in Worcester (MA, USA), offers a range of services for all stages of biologics development including viral vector construction, CAR-T cell line engineering and preclinical process development.
LakePharma CEO, Hua Tu, commented: “Since its founding, LakePharma has been relentless in capability expansion and service integration to provide our valued clients with end-to-end solutions. The dedication of the Vector Center to support the burgeoning gene and cell therapy fields is yet another example of our commitment.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.