Gene Therapy

Adverum Biotechnologies has made significant progress in its lead clinical program for intravitreal gene therapy for retinal diseases, Leone Patterson, CEO and president, said at the J.P. Morgan Healthcare Conference.

Adverum Biotechnologies will change how patients receive steroids to manage inflammation in the third and fourth cohorts of its phase 1 OPTIC trial, the company announced in a press release.

The FDA granted rare pediatric disease designation to ARU-1801, an investigational autologous gene therapy, for the treatment of sickle cell disease, according to the agent’s manufacturer.

Researchers found that increased travel time to cancer care sites due to clinic closings appeared to be associated with greater Medicare spending and patient costs — one of the top stories in hematology/oncology last week.

Gene therapy with valoctocogene roxaparvovec reduced annualized rates of bleeding events and led to discontinuation of prophylactic factor VIII among patients with hemophilia A, according to results of a prospective study with multiyear follow-up published in The New England Journal of Medicine.

ORLANDO — Significant induction of fetal hemoglobin after the infusion of CD34-positive cells occurred among a small cohort of patients with sickle cell disease, according to data from a phase 1 gene therapy feasibility study presented at ASH Annual Meeting and Exposition.

The top retina-related stories on Healio.com/OSN in 2019 included trial results, meeting coverage and FDA approvals.

Spark Therapeutics has accepted a tender offer for all outstanding shares of common stock at a price of $114.50 per share, allowing Roche to complete its acquisition of the company, according to a press release.

The U.K. Competition and Markets Authority has cleared Roche’s pending acquisition of Spark Therapeutics, ruling the deal would not negatively affect competition, according to a press release.

ORLANDO — SB-525 gene therapy resulted in dose-dependent and sustained increases in Factor VIII levels in adult patients with severe hemophilia A, according to follow-up data from the Alta study presented at ASH Annual Meeting and Exposition.