GeneTherapyLive’s Weekly Rewind – January 14, 2022

Welcome to GeneTherapyLive’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

Biomarin's investigative gene therapy valoctocogeneroxaparvovec has shown sustained efficacy over 2 years in reducing bleeding in people with severe hemophilia A, according to positive data from the phase 3 GENEr8-1 study (NCT03370913), which has met both its primary and secondary end points.

Guy Young, MD, director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles, discussed improving outcomes in hemophilia with fitusiran. He discussed treatment adherence issues with IV factor therapy and how less frequent fitusiran treatments could reduce patients’ treatment burden.

SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy from Sarepta Therapeutics, statistically improved motor function in children with Duchenne muscular dystrophy (DMD) compared to placebo, according to new data from the phase 2 Study SRP-9001-102 (Study 102; NCT03769116).

Julian Molina, MD, PhD, a hematologist/oncologist at Mayo Clinic, discussed the growing presence of CAR T-cell therapies in lung cancer and melanoma. He discussed the value of immunotherapy in improving the treatment landscape of lung cancer and melanoma and advantages of Tmod technology over traditional CAR T-cell therapy.

Many cell and gene therapy companies are growing and collaborating to develop new innovative therapies while other industry players are establishing cell and gene therapy divisions of their own. We have compiled the latest company news in gene and cell therapies, including partnerships, pipeline updates, and more.