The patient, who is in his 30s, got the one-time intravenous infusion at the Infusion and Chemotherapy Treatment Center at ...
Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and ...
Less than a year after approval, Pfizer said it plans to discontinue its hemophilia B gene therapy fidanacogene elaparvovec (Beqvez) across all global markets, citing weak demand. "Pfizer has made the ...
In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a key question remains: How soon will these advances reach children? At the ...
- Based on positive results from Phase 3 AFFINE trial, which met primary and secondary endpoints, Sangamo plans to explore all options to commercialize the asset, including seeking a potential new ...
According to multiple media reports on Thursday, Pfizer Inc. (NYSE:PFE) has terminated the global development and commercialization of its hemophilia B gene therapy, Beqvez. In April 2024, the FDA ...
After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.
At a median 13 years after a single infusion of AAV-mediated gene therapy, factor IX expression remained stable in 10 patients with severe hemophilia B. The median annualized bleeding rate decreased ...
BioMarin Pharmaceutical Inc. pulled Roctavian from the market. Sales of the hemophilia gene therapy fell drastically short of ...
SAN DIEGO — Promising early results from an ongoing randomized, open-label, single-arm, Phase 3 study could pave the way for a Pfizer product to become the second US Food and Drug Administration (FDA) ...
A single intravenous dose of etranacogene dezaparvovec gene therapy maintained factor IX (FIX) activity at 45.7 IU/dL after 5 years in patients with hemophilia B, eliminating the need for prophylaxis ...
Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, ...
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