Gene Therapy

Category

Gene Therapy for Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Promise 18 Months Post Admin

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), positioning itself as a leader in precision genetic medicine for rare diseases, recently announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the company’s investigational gene therapy for...

Errant Gene Therapeutics (“EGT”) asks European Authorities to Re-examine the Approval Process of Zynteglo, While US Litigation Moves Forward

Picking up on the ongoing “Family Man” saga, in New York Court, EGT claims that Zynteglo, the Bluebird Bio product for Thalassemia, created Clonal Dominance. EGT alleges that Bluebird Bio assisted Memorial Sloan Kettering in orchestrating the sabotage of EGT’s product...

Elite Research Centers Ink Deal with Lonza to Evaluate (Pilot) Cell Therapy Manufacturing for Next Generation Treatments

In a notable partnership, Switzerland-based Lonza inked a partnership with leading research institutes and academic medical centers including Stanford University School of Medicine, Fred Hutchinson Cancer Research Center and Parker Institute for Cancer Immunotherapy...

Bascom Palmer Eye Institute Leads Way in Advanced Gene Therapy Clinical Trials

Researchers at Bascom Palmer Eye Institute, the Department of Ophthalmology at the University of Miami Miller School of Medicine, took part in an international multi-center study titled Initial Results from a First-in-Human Gene Therapy Trial on X-linked Retinitis...

Nationwide Children’s Hospital’s Affiliate Andelyn Biosciences Preparing to Build Major Gene Therapy Production Facility

If not the world’s leading gene therapy research center, Nationwide Children’s Hospital seeks to aggressively expand into gene therapy research with the launch of a $74 million biotechnology manufacturing facility for genetic material used in research and clinical...